H.R. 6: The 21st Century Cures Act

Congressional research reportAug 10, 2015

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August 10, 2015

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R44071

H.R. 6: The 21st Century Cures Act

Summary

On July 10, 2015, the House passed H.R. 6, the 21st Century Cures Act, on a vote of 344 to 77.

Eight amendments were offered; five were approved by voice vote, two failed by recorded vote,

and one was withdrawn. The House Energy and Commerce Committee, on May 21, 2015,

unanimously ordered to be reported H.R. 6 and the House Committee on Rules published a

committee print of the bill on July 2, 2015. On July 7, 2015, H.R. 6 was reported by the

Committee on Energy and Commerce (H.Rept. 114-190), and the House Committee on Ways and

Means was discharged from further consideration of the bill.

The bill would reauthorize the National Institutes of Health (NIH) through FY2018 and provide

other funding to the agency through FY2020. In addition, the bill would promote and encourage

more strategic planning for research conducted by NIH; change loan support for young, emerging

scientists; promote pediatric research; and encourage more collaborative research activities. The

bill also focuses on changes to the Food and Drug Administration’s (FDA’s) regulatory

procedures for drugs and devices by requiring the issuance of more guidance and increasing

regulatory flexibility in areas such as precision (or personalized) medicine, types of data that

could serve as evidence of safety and effectiveness, antibiotic drug development, orphan drugs,

and medical devices. The bill proposes additional funding for the FDA to support some of its

efforts in certain specified areas.

H.R. 6 consists of four separate titles. Title I focuses on discovery-related issues and is

concentrated on matters related to the NIH, including developing strategic plans, cultivating

young scientists, and promoting more collaboration among NIH researchers, grant recipients, and

institutions. Title II targets the development of new and more innovative drugs and medical

devices and the regulatory processes in place to consider these products. Title III includes

provisions related to the delivery of health care, including interoperability of electronic health

information technology and the treatment of disposable medical technologies. Title IV includes

Medicare and Medicaid changes being proposed to offset the costs of the NIH- and FDA-related

changes in Titles I, II, and III. These proposed offsets include changes in prior authorization

procedures for power mobility devices under Medicare, as well as a proposed drawdown in the

nation’s strategic petroleum reserve.

This report provides a brief summary of each provision of H.R. 6 as passed by the House on July

10, 2015. Each summary includes a brief description of current law and an explanation of how the

bill would change current law. A list of the abbreviations used throughout this report appears in

Appendix B.

Congressional Research Service

H.R. 6: The 21st Century Cures Act

Contents

Overview ......................................................................................................................................... 1

Summary of Provisions ................................................................................................................... 3

Section 2. NIH and Cures Innovation Fund ........................................................................ 3

Title I—Discovery..................................................................................................................... 7

Subtitle A—National Institutes of Health Funding ................................................................... 7

Section 1001. National Institutes of Health Reauthorization .............................................. 7

Section 1002. Prize Competitions ....................................................................................... 7

Subtitle B—National Institutes of Health Planning and Administration................................... 9

Section 1021. NIH Research Strategic Plan........................................................................ 9

Section 1022. Increasing Accountability at the National Institutes of Health .................. 10

Section 1023. Reducing Administrative Burdens of Researchers ...................................... 11

Section 1024. Exemption for the National Institutes of Health from the

Paperwork Reduction Act Requirements ........................................................................ 11

Section 1025. NIH Travel ................................................................................................. 12

Section 1026. Other Transactions Authority ..................................................................... 13

Section 1027. NCATS Phase IIB Restriction .................................................................... 13

Section 1028. High-Risk, High-Reward Research............................................................ 14

Section 1029. Sense of Congress on Increased Inclusion of Underrepresented

Communities in Clinical Trials ...................................................................................... 15

Subtitle C—Supporting Young Emerging Scientists ............................................................... 16

Section 1041. Improvement of Loan Repayment Programs of National Institutes

of Health ........................................................................................................................ 16

Section 1042. Report......................................................................................................... 17

Subtitle D—Capstone Grant Program ..................................................................................... 17

Section 1061. Capstone Award ......................................................................................... 17

Subtitle E—Promoting Pediatric Research Through the National Institutes of Health .......... 18

Section 1081. National Pediatric Research Network ........................................................ 18

Section 1082. Global Pediatric Clinical Study Network Sense of Congress .................... 18

Section 1083. Appropriate Age Groupings in Clinical Research ...................................... 19

Subtitle F—Advancement of National Institutes of Health Research and Data Access ......... 19

Section 1101. Standardization of Data in Clinical Trial Registry Data Bank on

Eligibility for Clinical Trials .......................................................................................... 19

Subtitle G—Facilitating Collaborative Research .................................................................... 20

Section 1121. Clinical Trial Data System ......................................................................... 20

Section 1122. National Neurological Diseases Surveillance System................................ 21

Section 1123. Data on Natural History of Diseases .......................................................... 21

Section 1124. Accessing, Sharing, and Using Health Data for Research Purposes .......... 22

Subtitle H—Council for 21st Century Cures ........................................................................... 23

Section 1141. Council for 21st Century Cures................................................................... 23

Title II—Development ............................................................................................................ 24

Subtitle A—Patient-Focused Drug Development ................................................................... 24

Section 2001. Development and Use of Patient Experience Data to Enhance

Structured Risk-Benefit Assessment Framework .......................................................... 24

Subtitle B—Qualification and Use of Drug Development Tools ............................................ 25

Section 2021. Qualification of Drug Development Tools ................................................. 25

Section 2022. Accelerated Approval Development Plan .................................................. 28

Subtitle C—FDA Advancement of Precision Medicine .......................................................... 29

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H.R. 6: The 21st Century Cures Act

Section 2041. Precision Medicine Guidance and Other Programs of Food and

Drug Administration ...................................................................................................... 29

Subtitle D—Modern Trial Design and Evidence Development .............................................. 30

Section 2061. Broader Application of Bayesian Statistics and Adaptive Trial

Designs .......................................................................................................................... 30

Section 2062. Utilizing Evidence from Clinical Experience ............................................ 31

Section 2063. Streamlined Data Review Program ............................................................ 32

Subtitle E—Expediting Patient Access ................................................................................... 33

Section 2081. Sense of Congress ...................................................................................... 33

Section 2082. Expanded Access Policy ............................................................................ 34

Section 2083. Finalizing Draft Guidance on Expanded Access ........................................ 35

Subtitle F—Facilitating Responsible Manufacturer Communications.................................... 35

Section 2101. Facilitating Dissemination of Health Care Economic Information ............ 35

Section 2102. Facilitating Responsible Communication of Scientific and Medical

Developments ................................................................................................................ 36

Subtitle G—Antibiotic Drug Development............................................................................. 37

Section 2121. Approval of Certain Drugs for Use in a Limited Population of

Patients........................................................................................................................... 37

Section 2122. Susceptibility Test Interpretive Criteria for Microorganisms ..................... 38

Section 2123. Encouraging the Development and Use of DISARM Drugs ..................... 40

Subtitle H—Vaccine Access, Certainty, and Innovation ......................................................... 41

Section 2141. Timely Review of Vaccines by the Advisory Committee on

Immunization Practices.................................................................................................. 42

Section 2142. Review of Processes and Consistency of ACIP Recommendations........... 43

Section 2143. Meetings Between CDC and Vaccine Developers ..................................... 43

Subtitle I—Orphan Product Extensions Now; Incentives for Certain Products for

Limited Populations ............................................................................................................. 43

Section 2151. Extension of Exclusivity Periods for a Drug Approved for a New

Indication for a Rare Disease or Condition.................................................................... 43

Section 2152. Reauthorization of Rare Pediatric Disease Priority Review Voucher

Incentive Program .......................................................................................................... 44

Subtitle J—Domestic Manufacturing and Export Efficiencies ............................................... 44

Section 2161. Grants for Studying the Process of Continuous Drug

Manufacturing................................................................................................................ 44

Section 2162. Re-Exportation Among Members of the European Economic Area .......... 45

Subtitle K—Enhancing Combination Products Review ......................................................... 45

Section 2181. Enhancing Combination Products Review................................................. 45

Subtitle L—Priority Review for Breakthrough Devices ......................................................... 46

Section 2201. Priority Review for Breakthrough Devices ................................................ 47

Subtitle M—Medical Device Regulatory Process Improvements .......................................... 49

Section 2221. Third-Party Quality System Assessment .................................................... 49

Section 2222. Valid Scientific Evidence ........................................................................... 52

Section 2223. Training and Oversight in Least Burdensome Appropriate

Means Concept .............................................................................................................. 52

Section 2224. Recognition of Standards ........................................................................... 54

Section 2225. Easing Regulatory Burden with Respect to Certain Class I and

Class II Devices ............................................................................................................. 55

Section 2226. Advisory Committee Process ..................................................................... 56

Section 2227. Humanitarian Device Exemption Application ........................................... 58

Section 2228. CLIA Waiver Study Design Guidance for In Vitro Diagnostics ................ 58

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Subtitle N—Sensible Oversight for Technology Which Advances Regulatory

Efficiency ............................................................................................................................. 59

Section 2241. Health Software ......................................................................................... 60

Section 2242. Applicability and Inapplicability of Regulation ......................................... 60

Section 2243. Exclusion from Definition of Device ......................................................... 60

Subtitle O—Streamlining Clinical Trials ................................................................................ 61

Section 2261. Protection of Human Subjects in Research; Applicability of Rules........... 62

Section 2262. Use of Non-Local Institutional Review Boards for Review of

Investigational Device Exemptions and Human Device Exemptions ........................... 62

Section 2263. Alteration or Waiver of Informed Consent for Clinical

Investigations ................................................................................................................. 63

Subtitle P—Improving Scientific Expertise and Outreach at FDA ......................................... 63

Section 2281. Silvio O. Conte Senior Biomedical Research Service ............................... 63

Section 2282. Enabling FDA Scientific Engagement ....................................................... 64

Section 2283. Reagan-Udall Foundation for the Food and Drug Administration............. 64

Section 2284. Collection of Certain Voluntary Information Exempted from

Paperwork Reduction Act .............................................................................................. 65

Section 2285. Hiring Authority for Scientific, Technical, and Professional

Personnel........................................................................................................................ 66

Subtitle Q—Exempting From Sequestration Certain User Fees ............................................. 67

Section 2301. Exempting From Sequestration Certain User Fees of Food and

Drug Administration ...................................................................................................... 67

Subtitle R—Other Provisions .................................................................................................. 68

Section 2321. Sense of Congress ...................................................................................... 68

Title III—Delivery .................................................................................................................. 68

Subtitle A—Interoperability .................................................................................................... 68

Section 3001. Ensuring Interoperability of Health Information Technology .................... 68

Subtitle B—Telehealth ............................................................................................................ 72

Section 3021. Telehealth Services under the Medicare Program ...................................... 72

Subtitle C—Encouraging Continuing Medical Education for Physicians .............................. 73

Section 3041. Exempting From Manufacturer Transparency Reporting Certain

Transfers Used for Educational Purposes ...................................................................... 73

Subtitle D—Disposable Medical Technologies ...................................................................... 75

Section 3061. Treatment of Certain Items and Devices .................................................... 75

Subtitle E—Local Coverage Decision Reforms...................................................................... 76

Section 3081. Improvements in the Medicare Local Coverage Determination

(LCD) Process ............................................................................................................... 76

Subtitle F—Medicare Pharmaceutical and Technology Ombudsman ..................................... 77

Section 3101. Medicare Pharmaceutical and Technology Ombudsman ........................... 77

Subtitle G—Medicare Site-of-Service Price Transparency..................................................... 78

Section 3121. Medicare Site-of-Service Price Transparency ............................................ 78

Subtitle H—Medicare Part D Patient Safety and Drug Abuse Prevention .............................. 79

Section 3141. Programs to Prevent Prescription Drug Abuse Under Medicare

Parts C and D ................................................................................................................. 79

Title IV—Medicaid, Medicare, and Other Reforms ............................................................... 81

Subtitle A—Medicaid and Medicare Reforms ........................................................................ 81

Section 4001. Limiting Federal Medicaid Reimbursement to States for Durable

Medical Equipment (DME) to Medicare Payment Rates .............................................. 81

Section 4002. Excluding Authorized Generics from Calculation of Average

Manufacturer Price ........................................................................................................ 82

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Section 4003. Medicare Payment Incentive for the Transition from Traditional XRay Imaging to Digital Radiography and Other Medicare Imaging Payment

Provision ........................................................................................................................ 83

Section 4004. Treatment of Infusion Drugs Furnished Through Durable Medical

Equipment ...................................................................................................................... 84

Section 4005. Extension and Expansion of Prior Authorization for Power

Mobility Devices (PMDs) and Accessories and Prior Authorization Audit

Limitations ..................................................................................................................... 85

Section 4006. Civil Monetary Penalties for Violations Related to Grants,

Contracts, and Other Agreements .................................................................................. 87

Subtitle B—Other Reforms ..................................................................................................... 90

Section 4041. SPR Drawdown .......................................................................................... 90

Subtitle C—Miscellaneous...................................................................................................... 91

Section 4061. Lyme Disease and Other Tick-Borne Diseases .......................................... 91

Section 4062. Outreach to Historically Black Colleges and Universities ......................... 92

Tables

Table A-1. Guidance, Reports, and Regulations/Rulemaking That H.R. 6 Would Require .......... 93

Appendixes

Appendix A. Guidance, Reports, and Regulations/Rulemaking That H.R. 6

Would Require............................................................................................................................ 93

Appendix B. List of Abbreviations ................................................................................................ 95

Contacts

Author Contact Information .......................................................................................................... 97

Acknowledgments ......................................................................................................................... 97

Congressional Research Service

H.R. 6: The 21st Century Cures Act

Overview

On July 10, 2015, the House passed H.R. 6, the 21st Century Cures Act, on a vote of 344 to 77.

Eight amendments were offered; five were approved by voice vote, two failed by recorded vote,

and one was withdrawn. On May 21, 2015, the House Energy and Commerce Committee

unanimously ordered to be reported H.R. 6. 1 The House Committee on Rules published a

committee print of the bill on July 2, 2015. 2 On July 7, 2015, H.R. 6 was reported by the

Committee on Energy and Commerce (H.Rept. 114-190), and the House Committee on Ways and

Means was discharged from further consideration of the bill.

Amendments to H.R. 6

Amendment 1 (Brat) to reform the NIH and Cures Innovation Fund to make it a discretionary program, failed by

recorded vote Y-141 N-281.

Amendment 2 (Young) to create authority within NIH prize program to incentivize health innovation and create

breakthrough research and technology, approved by voice vote.

Amendment 3 (Lee) to strike the provision that applies policy riders in appropriations bills to NIH & FDA funding in

H.R. 6, failed by recorded vote Y-176 N-245.

Amendment 4 (Castro) to ensure that underrepresented individuals in the sciences (women and minorities) are

included as a focus topic in the report on Supporting Young Emerging Scientists, approved by voice vote.

Amendment 5 (Slaughter) to direct CDC to conduct a study to determine how the additional payments for certain

drugs are affecting usage practices and the development of drug resistance, approved by voice vote.

Amendment 6 (Fitzpatrick) to express a sense of Congress that recording Unique Device Identifiers at the point-ofcare in electronic health record systems could significantly enhance the availability of medical device data for postmarket surveillance purposes, approved by voice vote.

Amendment 7 (Polis) to direct FDA to issue a report on the risks and benefits associated with a two-tiered approval

process that would permit certain medical devices to provisionally come to market if they have demonstrated safety

but not efficacy, withdrawn.

Amendment 8 (Jackson Lee) to direct the HHS Secretary to conduct outreach to certain colleges, universities and

other institutions to ensure that health professionals from underrepresented populations are aware of the research

opportunities under this Act, approved by voice vote.

While consisting of many different provisions, the bill is primarily focused on efforts to increase

strategic investments in medical research at the National Institutes of Health (NIH) and change

some aspects of how the Food and Drug Administration (FDA) executes its regulatory oversight

mission with regard to the review and approval of new drugs, biologics, and medical devices.

H.R. 6 is the result of a series of hearings and roundtable meetings hosted by the House Energy

and Commerce Committee dating back to spring 2014.3 The hearings and roundtables focused on

a broad range of topics, including modernizing clinical trials, incorporating patient perspectives

U.S. House of Representatives, Committee on Energy and Commerce, Full Committee Vote on the 21st Century Cures

Act, May 19, 2015, http://energycommerce.house.gov/markup/full-committee-vote-21st-century-cures-act. The

committee website links to more than one version of the bill and several amendments; the committee marked up a

composite of a Committee Print dated May 19, 2015, named UPTON_005 (http://docs.house.gov/meetings/IF/IF00/

20150519/103516/BILLS-1146ih.pdf) and a manager’s amendment dated May 20, 2015, named UPTON_006

(http://docs.house.gov/meetings/IF/IF00/20150519/103516/BILLS-114-6-U000031-Amdt-3.pdf).

Rules Committee Print 114-22, Text of H.R. 6, 21st Century Cures Act, based on H.R. 6 as ordered reported by the

Committee on Energy and Commerce, July 2, 2015, http://docs.house.gov/billsthisweek/20150706/CPRT-114-HPRTRU00-HR6.pdf.

House Energy and Commerce Committee, 21st Century Cures Roundtable, http://energycommerce.house.gov/event/

21st-century-cures-roundtable.

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H.R. 6: The 21st Century Cures Act

into medical research and regulatory processes, precision/personalized medicine, digital health

care, and more. At present, no companion legislation to H.R. 6 has been introduced in the Senate.

However, the Senate Health, Education, Labor, and Pensions (HELP) Committee has started work

on issues related to medical innovation4 and committee leadership has indicated they plan to

continue this work in the 114th Congress.

The July 2, 2015, Rules Committee Print differs from the version that the Committee on Energy

and Commerce ordered to be reported on May 21, 2015. Among the most notable changes is the

reduction in proposed funding for the NIH Innovation Fund, from a level of $10 billion over five

years in the version voted on by Energy and Commerce to a level of $8.75 billion. The other most

significant changes to the bill are found in the proposals to offset the costs for the legislation. A

provision in the May 21 version that would have delayed certain Medicare prepayments for

prescription drug plans has been deleted from the current version of the bill. The latest version

(July 2) includes a new provision to expand the use of prior authorization under Medicare for

power mobility devices (wheelchairs and scooters) along with an expansion of the provision in

the May 21 version for drawdowns from the Strategic Petroleum Reserve, among others.

Some of the key themes in the bill are innovation, flexibility, and transparency. H.R. 6 represents

an effort to maintain or increase medical innovation as reflected in research conducted or funded

by the NIH. The bill has numerous provisions related to increasing regulatory flexibility by FDA

in its processes for reviewing and approving drugs, biologics, and medical devices. In particular,

the bill increases the ability of industries subject to FDA regulation (e.g., pharmaceutical

companies, device makers) and the individuals who are or may become patients who could

benefit from future products, to have a greater voice in the regulatory process and to streamline

the various ways in which FDA ensures safe and effective medications and medical devices enter

the market. The bill attempts to improve the transparency of data—for researchers, consumers,

and regulated entities—by helping to provide enhanced and timelier information for

decisionmakers.

H.R. 6 would reauthorize the NIH through FY2018 and provide $8.75 billion in additional

funding for an innovation fund through FY2020. The bill would promote and encourage more

strategic planning for research conducted by NIH; change loan support for young, emerging

scientists; promote pediatric research; and encourage more collaborative research activities. The

bill focuses on changes to the FDA’s regulatory procedures for drugs and devices by requiring the

issuance of more guidance and increasing regulatory flexibility in areas such as precision (or

personalized) medicine, antibiotic drug development, orphan drugs, and medical devices. The bill

also proposes $550 million in additional funding over five years to support efforts in certain

specified areas, mostly in FDA. FDA estimates it could cost more than $900 million to implement

the legislation; “any unfunded mandates in the bill may require resources to be shifted from other

activities.”5

U.S. Senate Committee on Health, Education, Labor, and Pensions, Full Committee Hearing, “Continuing America’s

Leadership in Medical Innovation for Patient,” March 10, 2015, http://www.help.senate.gov/hearings/continuingamericas-leadership-in-medical-innovation-for-patients. Statement by Senator Lamar Alexander, “Senate Health

Committee Holds First Hearing on Innovation Initiative: How to Get Medical Devices, Drugs, Treatments from

Discovery to the Medicine Cabinet,” March 10, 2015, http://www.help.senate.gov/chair/newsroom/press/-senatehealth-committee-holds-first-hearing-on-innovation-initiative-how-to-get-medical-devices-drugs-treatments-fromdiscovery-to-the-medicine-cabinet.

Derrick Gingery, “FDA Program Cuts Loom if “Cures” Bill Isn't Fully Funded, Ostroff Warns,” The Pink Sheet

Daily, June 3, 2015. See Appendix A for a list of new requirements (e.g., regulations, guidance, reports, etc.) that

would be created by H.R. 6.

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H.R. 6: The 21st Century Cures Act

Many interested groups (manufacturers, medical schools, disease and patient advocates,

researchers, and past and present regulators) have opined publicly on specific provisions in

various drafts of the bill, not all of which have been supportive.6 This report provides summary

descriptions of the bill’s provisions, along with background to give context, not a full analysis of

their impact. The Congressional Budget Office has published a cost estimate.7

Summary of Provisions

Section 2. NIH and Cures Innovation Fund

Background

The National Institutes of Health is the lead federal agency charged with performing and

supporting biomedical and behavioral research. It also has major roles in training biomedical

researchers and disseminating health information. Congress doubled the NIH budget from $13.65

billion to $27.1 billion in the five-year period from FY1998 to FY2003; during that period,

annual increases in the 14%-15% range were the norm. Since then, increases from regular

appropriations have been between 1.0% and 3.2% each year.8 The growth rate of the NIH budget

has been at or below the rate of inflation, which for biomedical research in FY2015 is estimated

to be 2.2%.9 NIH funding in FY2015 is 22% lower than the FY2003 level, the peak of the

doubling period in constant 2012 dollars.10

See, for example, The Editorial Board, “How Not to Fix the FDA,” The New York Times, July 20, 2015,

http://www.nytimes.com/2015/07/20/opinion/how-not-to-fix-the-fda.html?_r=0; Rita F. Redberg and Sanket S. Dhruva,

“The FDA’s Medical Device Problem,” The New York Times, July 17, 2015,

http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html; Jerry Avorn and Aaron S.

Kesselheim, “The 21st Century Cures Act—Will It Take Us Back in Time?” New England Journal of Medicine, June 3,

2015, http://www.nejm.org/doi/pdf/10.1056/NEJMp1506964; Michael Causey, “Congress Crawls Out of 20th Century

to Push Bipartisan ‘Cures’ Legislation,” Quality Digest, June 9, 2015, http://www.qualitydigest.com/inside/fdacompliance-article/060915-congress-crawls-out-20th-century-push-bipartisan-cures#; Editorial Board, “A breakthrough

for biomedicine,” May 29, 2015, Denver Post, May 29, 2015, http://www.denverpost.com/editorials/ci_28216439/

breakthrough-biomedicine; Quardricos Bernard Driskell, “What will it take to cure psoriatic disease?” National

Psoriasis Foundation blog, June 2, 2015, http://blog.psoriasis.org/blog/what-will-it-take-cure-psoriatic-disease; Newt

Gingrich, “21st Century Cures is a Major Breakthrough,” Gingrich Productions, June 3, 2015,

http://www.gingrichproductions.com/2015/06/21st-century-cures-is-a-major-breakthrough/; Gregg Gonsalves, Mark

Harrington, and David A. Kessler, “Don’t Weaken the F.D.A.’s Drug Approval Process,” New York Times, June 11,

2015, http://www.nytimes.com/2015/06/11/opinion/dont-weaken-the-fdas-drug-approval-process.html?ref=opinion;

Bronwyn Mixter, “Watchdog, Consumer Groups Say ‘Cures’ Bill Could Lower Certain Drug Approval Standards,”

BNA, June 3, 2015, http://healthlawrc.bna.com/hlrc/4225/split_display.adp?fedfid=69846101&vname=

hcenotallissues&fn=69846101&jd=69846101; Bernard Muller, “Beyond the Ice Bucket,” The Hill, June 10, 2015,

http://thehill.com/blogs/congress-blog/healthcare/244456-beyond-the-ice-bucket; and Ed Silverman, “Will the 21st

Century Cures Bill Lower Standards for Some Drug Approvals?” Pharmalot blog in Wall Street Journal, May 29, 2015,

http://blogs.wsj.com/pharmalot/search/will%20the%2021st%20century%20cure/?s=will+the+21st+century+cure.

Congressional Budget Office, H.R. 6, 21st Century Cures Act, Cost Estimate for Rules Committee Print 114-22, July

7, 2015, http://www.cbo.gov/publication/50361.

For further information, see CRS Report R43341, NIH Funding: FY1994-FY2016.

The Biomedical Research and Development Price Index (BRDPI) is developed each year for NIH by the Bureau of

Economic Analysis of the Department of Commerce. It reflects the increase in prices of the resources needed to

conduct biomedical research—including personnel services, supplies, equipment—and indicates how much the NIH

budget must change to maintain purchasing power. See http://officeofbudget.od.nih.gov/gbiPriceIndexes.html.

For further information, see CRS Report R43341, NIH Funding: FY1994-FY2016.

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A recent analysis of U.S. expenditures on biomedical research found that “U.S. government

research funding declined from 57% (2004) to 50% (2012) of the global total, as did that of U.S.

companies (50% to 41%), with the total U.S. (public plus private) share of global research

funding declining from 57% to 44%. Asia, particularly China, tripled investment from $2.6

billion (2004) to $9.7 billion (2012) preferentially for education and personnel.”11 The United

States continues to be the top supporter of both public and industry medical research.12 However,

some Members of Congress and many in the biomedical research community have expressed

concern over the rapidly increasing investments being made by other countries in this area of

research.

Many of those who are concerned over the U.S. global position in biomedical research investment

have made frequent calls for increased support for research at the NIH. However, another recent

analysis of U.S. biomedical research funding cautioned that the past pattern of rapid doubling of

the NIH budget followed by slowdowns in federal funding “created an unsustainable

hypercompetitive system that is discouraging even the most outstanding prospective students

from entering our profession—and making it difficult for seasoned investigators to produce their

best work.”13 Rather than short-term infusions of cash that disappear, the authors recommend that

greater emphasis be placed on the predictable and stable growth of federal funds for the research

enterprise.14 In responding to questions raised by Senator Elizabeth Warren during a May 5, 2015,

Senate hearing, NIH Director Francis Collins agreed that continued NIH budget increases—

ranging from 3.7% annually to inflation plus 4% or 5%—would be preferred to a temporary

larger investment that disappears.15

The Food and Drug Administration plays a central role in protecting the public health in the

United States by regulating most of the food supply and vitally important medical products,

including drugs, devices, and biologics that affect American lives on a daily basis. In performing

this role, FDA regulates some of the most successful and innovative companies in the U.S.

economy, such as those in the pharmaceutical and medical device industries. In recent years,

some have argued that FDA is underfunded and at risk of being unable to fulfill all its statutory

responsibilities assigned by Congress. Implementing new statutory provisions involves the

development of new regulations and extensive communication with industry and the public;

carrying out the new responsibilities requires additional FDA staff time as well as agency

resources.16

Hamilton Moses, David H. M. Matheson, Sarah Cairns-Smith, et al., “The Anatomy of Medical Research: U.S. and

International Comparisons,” Journal of the American Medical Association, vol. 313, no. 2 (January 13, 2015), pp. 174189.

Overall medical research funding in the United States was $117.2 billion in 2011. See Figure 8 on page 181 in

Hamilton Moses, David H. M. Matheson, Sarah Cairns-Smith, et al., “The Anatomy of Medical Research: U.S. and

International Comparisons,” Journal of the American Medical Association, vol. 313, no. 2 (January 13, 2015).

Bruce Alberts, Marc W. Kirschner, Shirley Tilghman, and Harold Varmus, “Rescuing U.S. biomedical research from

its systemic flaws,” Proceedings of the National Academy of Sciences, vol. 111, no. 16 (April 22, 2014), pp. 57735777.

Ibid., p. 5775.

U.S. Congress, Senate Committee on Health, Education, Labor, and Pensions, Continuing America’s Leadership:

Realizing the Promise of Precision Medicine for Patients, 114th Cong., 1st sess., May 5, 2015.

A recent example is implementation of the Food Safety Modernization Act (FSMA). The Congressional Budget

Office indicated that FDA would require $580 million between 2011 through 2015 to carry out FSMA; so far Congress

has appropriated less than half of this amount.

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H.R. 6: The 21st Century Cures Act

FDA’s program level, the amount that FDA can spend, is composed of direct appropriations (also

referred to as budget authority) and user fees collected from the regulated industry.17 For FY2015,

FDA’s program level is $4.5 billion, of which 42.3% ($1.902 billion) comes from user fees. By

statute and by five-year agreements between FDA and the regulated industries that pay the fees,

FDA may use user fee revenue only for specified activities. This requirement, along with tasks

that the Committee on Appropriations reports direct FDA to conduct, influences the priorities of

the agency, potentially leaving other tasks inadequately addressed.

Provision

The provision would establish in the U.S. Treasury an NIH and Cures Innovation Fund and would

provide the Fund with $1.86 billion in mandatory funds per year for FY2016 through FY2020.18

The amounts appropriated to the Fund would be in addition to amounts otherwise made available

to the Department of Health and Human Services (HHS).

Of the amounts made available to the NIH and Cures Innovation Fund for each fiscal year, $1.75

billion would be for “NIH Biomedical Research” and $110 million would be for “Cures

Development.” Of the amounts for NIH biomedical research for a fiscal year, not less than $500

million is for the Accelerating Advancement Program. Of the remaining funds in that fiscal year,

not less than 20% is for high-risk, high-reward research, and not less than 35% is for early stage

investigators (defined as the principal investigator of the proposed research, who has been

awarded no more than one substantial, competing grant, and who is within 10 years of having

completed a medical residency or terminal degree). Of the total amount made available for the

NIH Innovation Fund in a fiscal year, not more than 10% is for intramural research.

The NIH and Cures Innovation Fund would not be subject to any transfer authority of the NIH

Director or the Secretary of HHS, such as the PHS Evaluation Set-Aside, the Common Fund, the

1% transfer authority of the NIH Director, or the Nonrecurring Expenses Fund.19

The provision states that amounts in the NIH and Cures Innovation Fund that are allocated for

“NIH biomedical research” would be used only to conduct or support certain biomedical research

activities. The provision specifies some of these activities, such as research carried out by an

early stage investigator, research carried out by a small business, the Accelerating Advancement

Program, and development and implementation of the NIH research strategic plan.

The provision states that amounts in the NIH and Cures Innovation Fund that are allocated for

“cures development” would only be used for activities of the following nine provisions:



PHSA Section 229A, as added by Section 1123 (the natural history of diseases);

Section 2001 and the amendments made by such section (development and use of

patient experience data to enhance structured risk-benefit assessment

framework);

Beginning with the Prescription Drug User Fee Act (PDUFA, P.L. 102-571) in 1992, Congress has authorized FDA

to collect fees from industry sponsors of certain FDA-regulated products and to use the revenue to support statutorily

defined activities, such as the review of product marketing applications.

During floor debate on H.R. 6, Amendment 1 (Brat) was defeated on a vote of Yea-141 Nay-281. Amendment 1

would have made the NIH and Cures Innovation Fund a discretionary spending program.

Nonrecurring Expenses Fund (NEF) is an account within the Department of the Treasury. The HHS Secretary is

authorized to transfer to the NEF unobligated balances of expired discretionary funds. NEF funds are available until

expended for use by the HHS Secretary for capital acquisitions, including facility and information technology

infrastructure. Congressional appropriators must be notified in advance of any planned use of NEF funds. NEF was

created by Section 223 of Division G of the Consolidated Appropriations Act, 2008 (42 U.S.C. 3514a).

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H.R. 6: The 21st Century Cures Act



Section 2021 and the amendments made by such section (qualification of drug

development tools);

Section 2062 and the amendments made by such section (utilizing evidence from

clinical experience);

Section 2161 (grants to study the process of continuous drug manufacturing);

Section 2201 and the amendments made by such section (priority review for

breakthrough devices);

Section 2221 and the amendments made by such section (third-party quality

system assessments);

Sections 2241, 2242, and 2243 and the amendments made by such sections

(health software); and

FFDCA Section 513(j), as added by Section 2223 (training and oversight in least

burdensome appropriate means concept).

Of the biomedical research funded under the provision, the NIH Director would ensure

coordination among the various research institutes, centers, agencies, departments, offices of the

federal government and minimize unnecessary duplication. This section requires the NIH

Director to establish the Accelerating Advancement Program under which for every $1 of NIH

Innovation Fund made available by the NIH Director to an NIH research Institute or Center, the

Institute or Center contributes $1 of other funding to accomplish important biomedical research

objectives. The scientifically based strategic plan would identify focus areas in which the

resources of the NIH Innovation Fund can be used on biomedical research to expand knowledge,

find more effective treatments, and address unmet needs in the United States. Focus areas include

biomarkers, precision medicine, infectious diseases, and antibiotics. The strategic plan would

include objectives for each strategic focus area and ensure that basic research remains a priority.

The strategic plan would be updated not less than every 18 months.

The House and Senate Committees on Appropriation could provide for the transfer of funds in the

NIH and Cures Innovation Fund for the authorized uses specified in the provision (NIH

biomedical research and cures development).

Funds appropriated to the NIH and Cures Innovation Fund would be used to supplement, not

supplant, the funds otherwise made available to HHS, are subject to the requirements and

limitations of the most recently enacted regular or full-year continuing appropriation Act or

resolution for NIH or FDA programs, and may be used only for the activities specified in the

provision.20

Amendment 1 (Brat) to H.R. 6 would have struck subsection (f) and all the terms and conditions listed here.

Amendment 1 was defeated on a vote of Yea-141 Nay-281. Amendment 3 (Lee), defeated on a vote of Yea-176 Nay245, would have struck paragraph (f)(2). Paragraph (f)(2) would require that the funds appropriated to the NIH and

Cures Innovation Fund be subject to the requirements and limitations—also called policy riders—of the most recently

enacted regular or full-year continuing appropriation Act or resolution for NIH or FDA programs.

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H.R. 6: The 21st Century Cures Act

Title I—Discovery

Subtitle A—National Institutes of Health Funding

Section 1001. National Institutes of Health Reauthorization

Background

NIH derives its statutory authority from the Public Health Service Act of 1944 (PHSA), as

amended.21 Section 301 of the PHSA grants the Secretary of HHS broad permanent authority to

conduct and sponsor research.22 In addition, Title IV of the PHSA, “National Research Institutes,”

authorizes in greater detail various activities, functions, and responsibilities of the NIH Director

and the institutes and centers.23 The last major NIH reauthorization was the NIH Reform Act of

2006.24 The NIH Reform Act authorized total funding levels for NIH appropriations for FY2007

($30,331,309,000), FY2008 ($32,831,309,000), and such sums as necessary for FY2009. Overall

NIH authorization expired at the end of FY2009 and has not been extended by Congress. Annual

appropriations, together with Section 301 of the PHSA, provide authority for NIH programs to

continue from FY2009 to the present.

Provision

The provision would authorize appropriations for NIH in FY2016 ($31,811,000,000), FY2017

($33,331,000,000), and FY2018 ($34,851,000,000).

Section 1002. Prize Competitions

Background

Section 105 of the America COMPETES Reauthorization Act of 2010 (P.L. 111-358) provided

federal agencies with broad authority to carry out programs designed to stimulate innovation

through prize competitions.25 Before passage of P.L. 111-358, only certain federal agencies had

the authority to initiate prize competitions. The White House Office of Science and Technology

Policy (OSTP) has published annual reports on the implementation of Section 105 as required by

P.L. 111-358. Currently a number of federal government agencies, including NIH, sponsor

challenges or prize competitions in science and medical research. A current list of such challenges

can be found at a federal government website.26 A search of the website on July 15, 2015, resulted

in seven competitions conducted by the “National Institutes of Health.” Examples of research

topics covered in the various challenges: breast cancer genetics, antimicrobial resistance, and

drug abuse and addiction research.

42 U.S.C. §§201-300mm-61.

42 U.S.C. §241.

42 U.S.C. §§281-290b.

P.L. 109-482

For more information, see CRS Report R43880, The America COMPETES Acts: An Overview.

https://www.challenge.gov/list/.

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H.R. 6: The 21st Century Cures Act

Provision

The provision would amend the PHSA by adding a new Section 409K that would require the

Director of NIH to establish an Innovation Prizes Program.27 The goal(s) of the NIH prize

program would be “identifying and funding areas of biomedical science that could realize

significant advancement through the creation of a prize competition,” and/or “improving health

outcomes, particularly with respect to human diseases and conditions for which the public and

private investment in research is disproportionately small relative to federal government

expenditures on prevention and treatment activities.”

Within six months of enactment, the Director of NIH would be required to: (1) design the prize

competitions; (2) ensure the design is realistic (given the funds to be awarded), does not reflect

any bias (concerning which innovations would be the best solution), allows any person to

participate; and (3) submit a report to Congress on the design of the competitions.

The Director of NIH would be required to establish the “I-Prize Board” composed of 9 board

members who would be appointed by the NIH Director and certain specified Members of

Congress. The I-Prize Board would provide advice on identifying areas of biomedical science per

the goals of the prize program and make recommendations on establishing the criteria for the

prize competitions as well as how to conduct the prize competition. Board members would be

appointed within 120 days of enactment, each for a 5-year term.

The provision specifies restrictions on financial conflict of interest that would be imposed on the

members of the I-Prize board and any other NIH officer or employee involved in carrying out the

prize competition. The provision also would require that the NIH Director, “with respect to an

innovation,” not award a prize “to any individual or entity that has a vested financial interest in

any product or procedure that is likely to be developed or marketed because of such innovation.”

The provision would allow for one or more contracts to be awarded by the NIH Director to

perform a simulation of the prize competitions and use the simulation to assess the effectiveness

of the competition design; a report to Congress on the simulation results would be submitted

within 4 months of awarding such a contract. The provision would allow the NIH Director to

enter into an agreement with one or more “tax exempt” entities to implement the prize

competition. However, no more than 15% of funds or other assistance “shall be for administration

of the prize competition and not less than 85% of such assistance shall be for activities in direct

support of competitors.”

The Director of NIH would be required to collect information on the medical efficacy of

innovations funded via the prize program as well as the actual and potential effect on federal

expenditures and submit reports to Congress as specified in the provision.

The provision would prohibit the federal government from acquiring the intellectual property

rights from a participant in a prize competition without his or her written consent. The provision

would allow the federal government to negotiate a license for the use of such intellectual

property.

Amendment 2 (Young), agreed to by voice vote during floor debate on H.R. 6, added this provision.

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H.R. 6: The 21st Century Cures Act

Subtitle B—National Institutes of Health Planning and

Administration

Section 1021. NIH Research Strategic Plan

Background

Section 402(b)(5) of the PHSA specifies that the NIH Director “shall ensure that scientifically

based strategic planning is implemented in support of research priorities as determined by the

agencies of the National Institutes of Health.” NIH provides access to many of its strategic plans

on the agency’s website.28

The focus of NIH research, and to some extent its organizational structure, have been criticized

by some in the academic literature.29 A point often made is that the United States spends more on

health care than any of the other 30 countries that make up the Organization for Economic

Cooperation and Development (OECD)—in fact, U.S. health care spending is more than 2.5

times the OECD average—and yet, the health of the U.S. populace, as measured by life

expectancy, is ranked 24th of the 30 countries.30 “Despite its name, NIH’s mission has not

generally been current health, per se, but rather research for tomorrow’s health.... An agency

devoted to current health would do well to focus on tobacco control, exercise, nutrition,

sanitation, and more cost-effective delivery of health care—prevention and efficiency, rather than

research on diseases currently not treatable.”31 Questioning or making changes to the focus of

NIH research (whether basic, clinical, prevention, health care delivery, or patient-centered

outcomes research) is perhaps “especially pertinent in light of the nation’s continually mediocre

public health outcomes, and their stark contrast to the sophistication and productivity of the

biomedical research enterprise.”32

Provision

The provision would add a new subsection (m) to Section 402 of the PHSA, which describes in

further detail a Research Strategic Plan for NIH. Every five years, beginning in 2016, the NIH

Director, along with the directors of the national research Institutes and Centers, as well as

researchers, patient advocacy groups, and industry leaders, would be required to develop and

maintain a biomedical research strategic plan. The strategic plan would be used to identify

research opportunities and develop individual strategic plans for the research activities of each of

the NIH Institutes and Centers. The Institute and Center (IC) plans would have a common

template and identify strategic focus areas. The IC plans would consider and identify the return

on investment to the U.S. public of such biomedical research and identify contributions to

improving U.S. public health through biomedical research. Overarching and trans-NIH focus

areas—or Mission Priority Focus Areas—would be identified that best serve the goals of

preventing or eliminating the burden of a disease or condition and scientifically merit enhanced

and focused research over the next five years. Rare and pediatric diseases would remain a

See for example http://report.nih.gov/strategicplans/#tab2.

See for example Michael M. Crow, “Time to rethink NIH,” Nature, vol. 471 (March 31, 2011), pp. 569-571; and,

Robert Cook-Deegan, “Has NIH lost its halo?,” Issues in Science and Technology, Winter 2015, pp. 37-47.

Michael M. Crow, “Time to rethink NIH,” Nature, vol. 471 (March 31, 2011), p. 570.

Robert Cook-Deegan, “Has NIH lost its halo?,” Issues in Science and Technology, Winter 2015, p. 43.

Ibid.

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H.R. 6: The 21st Century Cures Act

priority. In developing the strategic plan, the NIH Director would be required to ensure that

maintaining the biomedical workforce, including the participation of scientists from traditionally

underrepresented groups, would remain a priority. The initial strategic plan would be completed

not later than 270 days after enactment. The NIH Director, in consultation with the directors of

the national research Institutes and Centers, would be required to conduct annual progress

reviews for each strategic focus area in the IC plans. The plans would be reviewed and updated

every five years.

Section 1022. Increasing Accountability at the National Institutes of Health

Background

Section 405 of the PHSA specifies that the Director of the National Cancer Institute is appointed

by the President and the Directors of the other NIH Institutes are appointed by the Secretary. Each

NIH Institute Director reports directly to the NIH Director.

Section 202 of the Labor/HHS/ED Appropriations Act, 1993, states at the end of the section that

the payment of compensation to consultants or individual scientists appointed for limited periods

of time is “not to exceed the per diem rate equivalent to the maximum rate payable for seniorlevel positions,” which is “not less than 120% of the minimum rate of basic pay payable for GS–

15 of the General Schedule; and ... not greater than the rate of basic pay payable for level III of

the Executive Schedule.”33

Provision

The provision would amend Section 405 of the PHSA with regard to the appointment and terms

of the Director of the National Cancer Institute and the directors of other NIH Institutes and

Centers (ICs). It would require that directors of ICs be appointed by the NIH Director, with the

exception of the Director of the National Cancer Institute (who would continue to be appointed

by the President). It would add a new requirement that the term of office for the director of an IC

be five years and authorize the NIH Director to remove an IC Director prior to the end of a fiveyear term. It would permit the director of an IC to be reappointed at the end of a five-year term,

with no limit to the number of terms served. It would require that, if the office of a director of an

IC becomes vacant before the end of a five-year term, the director appointed to fill the vacancy

begin a new five-year term (as opposed to finishing the five-year term of the previous director).

Each current IC Director would be deemed to be appointed for a five-year term as of the date of

enactment.

The provision would remove compensation limitations for consultants and individual scientists as

stipulated by Section 202 of the Labor/HHS/ED Appropriations Act, 1993.34

The provision would add a new requirement that before a new research grant is made, the IC

Director will review and approve the award, taking into consideration the mission of the IC, the

scientific priorities identified in the strategic plan, and “whether other agencies are funding

programs or projects to accomplish the same goal.”

The provision would require the Secretary to enter into an arrangement with the Institute of

Medicine35 (or other appropriate entity) to complete a study, not later than two years following

P.L. 102-394 and 5 U.S.C. 5376.

Ibid.

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H.R. 6: The 21st Century Cures Act

enactment, “on the extent to which biomedical research supported by the federal government is

duplicative” and would require a report be submitted to Congress including recommendations on

how to prevent such duplication.

Section 1023. Reducing Administrative Burdens of Researchers

Background

The Federal Demonstration Partnership (FDP) is “a cooperative initiative among 10 federal

agencies and 119 institutional recipients of federal funds, sponsored by the National Academies,

with a purpose of reducing the administrative burdens associated with federal research grants and

contracts.”36 In 2005 and 2012, FDP conducted surveys of principal investigators of federally

funded projects to determine the impact of federal regulations and requirements on the research

process. In both surveys, researchers reported spending 57% of their time engaged in research and

42% of their time in completing pre- and post-award requirements. “The most commonly

experienced administrative responsibilities included those related to federal project finances,

personnel, and effort reporting. These were also among the most time-consuming responsibilities.

For researchers engaged in projects that required human or animal subjects, the related

Institutional Review Board (IRB) and Institutional Animal Care and Use Committee (IACUC)

requirements were by far the most time-consuming. Other areas viewed as particularly timeconsuming were those involving clinical trials, subcontracts, and cross-agency differences.”37

Provision

The provision would require the NIH Director to implement measures to reduce the

administrative burden of NIH-funded researchers, taking into account the recommendations of the

NIH Scientific Management Review Board, the National Academy of Sciences, the Faculty

Burden Survey conducted by the Federal Demonstration Partnership, and the Research Business

Models Working Group. Not later than two years following enactment, the NIH Director would

be required to submit a report to Congress on the measures that have been implemented to reduce

the administrative burden of NIH-funded researchers.

Section 1024. Exemption for the National Institutes of Health from the

Paperwork Reduction Act Requirements

Background

The Paperwork Reduction Act (PRA, 44 U.S.C. Chapter 35), enacted in 1980 and amended in

1995, established the Office of Information and Regulatory Affairs (OIRA) in the Office of

Management and Budget (OMB). Congress required that agencies seek OIRA permission before

(...continued)

In April 2015, the Institute of Medicine of the National Academies announced that, effective July 1, 2015, it would

change its name to the National Academy of Medicine. Institute of Medicine of the National Academies, “Institute of

Medicine to Become National Academy of Medicine,” press release, April 28, 2015, http://www.iom.edu/Global/

News%20Announcements/IOM-to-become-NAM-Press-Release.aspx.

Sandra L. Schneider et al., Federal Demonstration Partnership (FDP) 2012 Faculty Workload Survey: Executive

Summary, April 2014.

http://sites.nationalacademies.org/cs/groups/pgasite/documents/webpage/pga_087823.pdf;

http://sites.nationalacademies.org/PGA/fdp/PGA_055749.

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collecting information from the public. The first of 11 stated purposes was to “minimize the

paperwork burden for individuals ... and other persons resulting from the collection of

information by and for the Federal Government.”38 The PRA requires that federal agencies

receive clearance from OIRA before requesting most types of information from the public.39 PRA

clearance is required when standardized information is collected from 10 or more respondents

within a 12-month period.40 PRA does not apply to certain types of scientific research, including

collections that are neither sponsored nor conducted by the agency and those that are subject to a

clinical exception.41

Provision

The provision would amend 44 U.S.C. Chapter 35 to exempt NIH research from the requirements

of the PRA.

Section 1025. NIH Travel

Background

Following allegations of misspent funds during a 2010 General Services Administration meeting

held in Las Vegas, the Office of Management and Budget imposed restrictions on conference

travel for federal employees in a May 11, 2012, memorandum.42 The memorandum directed

agencies, beginning in FY2013, to spend at least 30% less than what was spent in FY2010 on

travel expenses, and stated that agencies “must maintain this reduced level of spending each year

through FY 2016.” Senior level agency approval is required for all conferences sponsored by an

agency where the conference expenses to the agency are over $100,000. Agencies are prohibited

from spending more than $500,000 on a single conference. However, this restriction may be

waived if the agency head “determines that exceptional circumstances exist whereby spending in

excess of $500,000 on a single conference is the most cost-effective option to achieve a

compelling purpose.”43

Provision

The provision would express the sense of Congress that “participation in or sponsorship of

scientific conferences and meetings is essential to the mission of the National Institutes of

Health.”

44 U.S.C. §3501.

For further information about the PRA, see CRS Report RL30590, Paperwork Reduction Act Reauthorization and

Government Information Management Issues, and CRS Report RL32397, Federal Rulemaking: The Role of the Office

of Information and Regulatory Affairs.

See NIH, Office of Science Policy, Genetics, Health and Society, What is the Paperwork Reduction Act?, at

http://osp.od.nih.gov/faq/what-paperwork-reduction-act; and HHS, Frequently Asked Questions About PRA /

Information Collection, at http://www.hhs.gov/ocio/policy/collection/infocollectfaq.html.

Cass R. Sunstein, Facilitating Scientific Research by Streamlining the Paperwork Reduction Act Process, Executive

Office of the President, Office of Management and Budget, December 9, 2010, https://www.whitehouse.gov/sites/

default/files/omb/memoranda/2011/m11-07.pdf.

Promoting Efficient Spending to Support Agency Operations, http://www.whitehouse.gov/sites/default/files/omb/

memoranda/2012/m-12-12.pdf.

Ibid.

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H.R. 6: The 21st Century Cures Act

Section 1026. Other Transactions Authority

Background

Section 480 of the PHSA establishes the Cures Acceleration Network (CAN). The purpose of the

CAN is to support revolutionary advances in basic research and facilitate FDA review of CANfunded high-need cures. A high-need cure is a drug, biological product, or device that, as

determined by the Director of the NIH National Center for Advancing Translational Sciences

(NCATS), “is a priority to diagnose, mitigate, prevent, or treat harm from any disease or

condition [and] for which the incentives of the commercial market are unlikely to result in its

adequate or timely development.”44

Under current law, if the Director of NCATS determines that the goals and objectives of this

section cannot be adequately carried out through a contract, grant, or cooperative agreement, then

the Director has “flexible research authority to use other transactions to fund projects in

accordance with the terms and conditions of this section. Awards made under such flexible

research authority for a fiscal year shall not exceed 20 percent of the total funds appropriated” for

a fiscal year.45 Other transaction (OT) authority is a special vehicle used by certain federal

agencies for obtaining or advancing research and development (R&D).46 Generally, OT authority

is created because the government needs to obtain leading-edge R&D from commercial sources,

but some companies (and other entities) are unwilling or unable to comply with the government’s

procurement regulations.

Current law stipulates that any “grant, cooperative agreement, or contract awarded under this

section shall be awarded on a competitive basis.”47

Provision

The provision would replace the current subparagraph on other transactions authority with a new

subparagraph that would provide other transactions authority with fewer restrictions. The OT

authority would not be conditional on a determination that the goals and objectives of this section

cannot be adequately carried out through a contract, grant, or cooperative agreement. The

provision would not limit OTs to 20% of the total funds appropriated.

The provision would also delete the requirement that grants, contracts, and cooperative

agreements be awarded on a competitive basis.

Section 1027. NCATS Phase IIB Restriction

Background

Prior to FDA approval, medical products are tested in a clinical trial using human volunteers to

see how the products compare to standard treatments or to no treatment. FDA uses the data from

clinical trials to determine whether to approve a manufacturer’s application for marketing a

PHS Act §480(a)(3).

PHS Act §480(e)(3)(C).

An OT is not a contract, grant, or cooperative agreement, and there is no statutory or regulatory definition of “other

transaction.” Only those agencies that have been provided OT authority may engage in other transactions. For further

information, see CRS Report RL34760, Other Transaction (OT) Authority.

PHS Act §480(f).

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medical product. Clinical trials are conducted in phases, which are described by FDA in the

paragraphs below. Sometimes Phase II clinical trials are divided into Phase IIA (to assess dosing

requirements) and Phase IIB (to study efficacy).

Phase I trials try to determine dosing, document how a drug is metabolized and excreted,

and identify acute side effects. Usually, a small number of healthy volunteers (between

20 and 80) are used in Phase I trials.

Phase II trials include more participants (about 100-300) who have the disease or

condition that the product potentially could treat. In Phase II trials, researchers seek to

gather further safety data and preliminary evidence of the drug’s beneficial effects

(efficacy), and they develop and refine research methods for future trials with this drug. If

the Phase II trials indicate that the drug may be effective—and the risks are considered

acceptable, given the observed efficacy and the severity of the disease—the drug moves

to Phase III.

In Phase III trials, the drug is studied in a larger number of participants with the disease

(approximately 1,000-3,000). This phase further tests the product’s effectiveness,

monitors side effects and, in some cases, compares the product’s effects to a standard

treatment, if one is already available. As more and more participants are tested over

longer periods of time, the less common side effects are more likely to be revealed. 48

Under current law, although NCATS may develop and provide infrastructure and resources for all

phases of clinical trials research, it may support clinical trial activities only through the end of

Phase IIA, with one exception. NCATS may support clinical trial activities through the end of

Phase IIB for a treatment for a rare disease or condition if (1) it gives public notice for a period of

at least 120 days of NCATS intention to support the clinical trial activities in Phase IIB; (2) no

public or private organization provides credible written intent to NCATS that the organization has

timely plans to further the clinical trial activities or conduct clinical trials of a similar nature

beyond Phase IIA; and (3) NCATS ensures that support of the clinical trial activities in Phase IIB

will not increase the federal government’s liability beyond the award value of the center’s

support.

Provision

The provision would extend NCATS’s authority to support clinical trial activities through the end

of Phase IIB (instead of Phase IIA), and extend the exception for treatment of a rare disease or

condition through the end of Phase III (instead of Phase IIB).

Section 1028. High-Risk, High-Reward Research

Background

The NIH Common Fund, within the Office of the NIH Director, supports research in emerging

areas of scientific opportunity, public health challenges, and knowledge gaps. These are often

large, complex research efforts that involve the collaboration of two or more research institutes or

centers. The Common Fund also supports the High-Risk, High-Reward Research Program, which

has “four unique funding opportunities for exceptionally creative scientists who propose highly

innovative approaches to major challenges in biomedical research.”49 These awards are intended

FDA, Inside Clinical Trials: Testing Medical Products in People, What Happens in a Clinical Trial?, at

http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143531.htm.

NIH, Office of Strategic Coordination, The Common Fund, High-Risk Research, at https://commonfund.nih.gov/

(continued...)

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“to encourage creative, outside-the-box thinkers to pursue exciting and innovative ideas about

biomedical research.” The four funding opportunities are (1) the NIH Director’s Pioneer Award,

(2) the New Innovator Award, (3) the Transformative Research Award, and (4) the NIH Director’s

Early Independence Award. This last award was created in FY2011 “to support exceptional early

career scientists who possess the intellect, scientific creativity, drive, and maturity to flourish

independently immediately following their graduate training, eliminating the need for traditional

post-doctoral training.”50 NIH announced 78 High-Risk, High-Reward awards in FY2013.51 A

total of 85 such awards were made in FY2014.52

Provision

The provision would add a new Section 409K to the PHSA that would require the Director of

each NIH institute to “establish programs to conduct or support research projects that pursue

innovative approaches to major contemporary challenges in biomedical research that involve

inherent high risk, but have the potential to lead to breakthroughs.” The NIH Director would

determine a specific percentage of funding for each institute for such projects.

Section 1029. Sense of Congress on Increased Inclusion of Underrepresented

Communities in Clinical Trials

Background

Minorities have been underrepresented in clinical trials. For example, according to a 2011 report

from an FDA-sponsored conference, “African Americans represent 12% of the U.S. population

but only 5% of clinical trial participants and Hispanics make up 16% of the population but only

1% of clinical trial participants.”53 There can be biological differences in how people process or

respond to medical products. For example, genetic differences can make a treatment less effective

or perhaps even more toxic in one particular ethnic group. Therefore, it is important to study in

clinical trials the safety and effectiveness of medical products in all people who will use the

products following FDA approval.

Provision

The provision would express the sense of Congress that the NIH National Institute on Minority

Health and Health Disparities “should include within its strategic plan ways to increase

representation of underrepresented communities in clinical trials.”

(...continued)

highrisk/index.

NIH, Office of Strategic Coordination, The Common Fund, High-Risk Research, at http://commonfund.nih.gov/

highrisk/overview.

NIH, News Releases, at http://www.nih.gov/news/health/sep2013/od-30.htm.

NIH, News Releases, at http://www.nih.gov/news/health/oct2014/od-06.htm.

FDA, For Consumers, Clinical Trials Shed Light on Minority Health, at http://www.fda.gov/ForConsumers/

ConsumerUpdates/ucm349063.htm.

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Subtitle C—Supporting Young Emerging Scientists

Section 1041. Improvement of Loan Repayment Programs of National

Institutes of Health

Background

NIH funds seven loan repayment programs for researchers.54 Three of these are intramural

programs that provide loan repayment to researchers in exchange for undertaking research while

employed by NIH. Intramural loan repayment programs support researchers from disadvantaged

backgrounds, those who are investigating AIDS, and those undertaking general research

(including general research by physicians during their fellowship training). NIH also funds four

programs to repay the loans of extramural researchers. These funds are awarded competitively to

researchers who are employed by a qualifying educational institution. Specific programs are

available to extramural researchers investigating health disparities, undertaking contraception and

infertility research, engaging in clinical research, and examining pediatric-related topics.

Researchers may receive up to $35,000 per year in loan repayment under each of these programs,

and the NIH loan repayment follows (where not inconsistent with the specific program) the

regulations that govern the National Health Service Corps Loan Repayment Program55 with

regard to participant eligibility, application procedures, selection criteria, loan repayment contract

terms, tax liability for payments received, service obligation, and penalties for breach of contract.

Provision

The provision would authorize a new NIH loan repayment program by adding a new PHSA

Section 487H “Loan Repayment Program.” The new section would require the Secretary to

establish a new extramural loan repayment program for the NIH, based on the agency’s scientific

and workforce needs, under which the federal government would pay not more than $50,000 per

year on the principal and educational loans of health professionals who engage in research.

Beginning in FY2017, the provision would allow amounts repaid under this new program to be

adjusted annually for inflation. Individuals eligible for loan repayment must have a substantial

amount of educational loans relative to income and must complete at least two years of research

service. The provision would also require that the new program, except where inconsistent with

the program’s purpose, be subject to the regulations that govern the National Health Service

Corps Loan Repayment Program56 with regard to participant eligibility, application procedures,

selection criteria, loan repayment contract terms, tax liability for payments received, service

obligation, and penalties for breach of contract. The provision would also allow amounts

appropriated for new loan repayment contracts to remain available until the end of the second

fiscal year after they are appropriated.

For description of these programs, see Appendix A of CRS Report R43571, Federal Student Loan Forgiveness and

Loan Repayment Programs.

For program description, see CRS Report R43920, National Health Service Corps: Changes in Funding and Impact

on Recruitment. For program regulations, see U.S. Department of Health and Human Services, Health Resources and

Services Administration, “National Health Service Corps: Loan repayment Program,” http://nhsc.hrsa.gov/downloads/

lrpapplicationguidance.pdf.

Ibid.

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Finally, the provision would amend the existing NIH loan repayment programs by increasing

annual loan repayment limits from $35,000 to $50,000 and by permitting an annual adjustment of

loan repayment amounts for inflation, beginning in FY2017.

Section 1042. Report

Provision

The provision would require the NIH Director to submit to Congress a report, not later than 18

months following enactment, on NIH efforts “to attract, retain and develop emerging scientists,

including underrepresented individuals in the sciences, such as women and other minorities.”57

Subtitle D—Capstone Grant Program

Section 1061. Capstone Award

Background

In February 2015, the NIH Deputy Director for Extramural Research, Sally Rockey, posted a

description of a new NIH emeritus award that would “help senior investigators who wish to

transition out of a position that relies on funding from NIH research grants, and facilitate the

transfer of their work, knowledge and resources to junior colleagues.”58 According to Science,

“most of the more than 120 comments” responding to Sally Rockey’s blog were critical of the

emeritus award.59 At the same time, NIH also published a formal request for public comment—

also known as a Request for Information (RFI)—on the emeritus award.60 Jeremy Berg, former

director of the NIH National Institute of General Medical Sciences stated that he is “skeptical that

it would have the desired impact,” that it may become “an entitlement for senior investigators,”

and that “[t]here is absolutely no need to create a new mechanism.”61

Amendment 4 (Castro), agreed to by voice vote during floor debate on H.R. 6, added the language ensuring that

underrepresented individuals in the sciences (women and minorities) would be included as a focus topic in the NIH

report to Congress.

Sally Rockey, Rock Talk, “Seeking Your Input on Sustaining the Workforce Through an Emeritus Award,” February

3, 2015, at http://nexus.od.nih.gov/all/2015/02/03/emeritus-rfi/. The Federation of American Societies for Experimental

Biology (FASEB) states that the award “reflects an idea initially suggested during a meeting of the NIH Advisory

Committee to the Director.” Yvette Seger, “NIH requests feedback on potential emeritus award,” The Washington

Update, February 11, 2015, http://washingtonupdate.faseb.org/?p=1225. The transition award idea is also a

recommendation in a January 2015 FASEB report, Sustaining Discovery in Biological and Medical Sciences: A

Framework for Discussion. See recommendation 2.10 on page 64 of the FASEB report, Sustaining Discovery in

Biological and Medical Sciences: A Framework for Discussion, at

http://www.faseb.org/pdfviewer.aspx?loadthis=http%3A%2F%2Fwww.faseb.org%2FPortals%2F2%2FPDFs%2Fopa%

2F2015%2FSustaining%2520Discovery%2520Report%2520Final.pdf.

Jocelyn Kaiser, NIH proposal to create grant for aging scientists hits a nerve, ScienceInsider, February 6, 2015, at

http://news.sciencemag.org/funding/2015/02/nih-proposal-create-grant-aging-scientists-hits-nerve.

http://grants.nih.gov/grants/guide/notice-files/NOT-OD-15-064.html; NIH uses RFIs as a mechanism to gather

community feedback on proposed ideas; the draft concepts described in RFIs are subject to change as a result of public

input and internal NIH discussions.

Kaiser, “NIH proposal to create grant for aging scientists hits a nerve,” ScienceInsider.

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Provision

The provision would add a new Section 490 to the PHSA creating a capstone award to support

outstanding scientists who have received NIH funding. The purpose of the award would be to

“facilitate the successful transition or conclusion of research programs.” The duration and amount

of each award would be determined by the NIH Director in consultation with the IC Directors.

Individuals who have received a capstone award would not be eligible to be the principal

investigator on subsequent NIH awards.

Subtitle E—Promoting Pediatric Research Through the National

Institutes of Health

Background

Section 409D(d) of the PHSA authorized in 2013 the establishment within NIH of a Pediatric

Research Network “in order to more effectively support pediatric research and optimize the use of

Federal resources.”62

Section 1081. National Pediatric Research Network

Provision

The provision would require the establishment of the National Pediatric Research Network

(NPRN). In establishing the NPRN, the provision would (1) eliminate language telling the NIH

Director to consult with the Director of the Eunice Kennedy Shriver National Institute of Child

Health and Human Development but (2) retain language telling the NIH Director to collaborate

with the ICs that carry out pediatric research. The provision would allow that the NPRN “may be

comprised of, as appropriate, the pediatric research consortia” that are receiving grants under this

section of the PHSA, and deletes that the NPRN may be comprised of other consortia, centers or

networks focused on pediatric research. The provision would now require the NIH Director to

award funding to support the pediatric research consortia; the duration of such support “shall be

for a period not to exceed 5 years.” Each consortium receiving an award under this section of the

PHSA would be required to “provide assistance to [CDC] for activities related to patient registries

and other surveillance systems.”

Section 1082. Global Pediatric Clinical Study Network Sense of Congress

Provision

The provision would express the sense of Congress that NIH “should encourage a global pediatric

clinical study network through the allocation of grants, contracts, or cooperative agreements to

supplement the salaries of new and early investigators who participate in the global pediatric

clinical study network.”

Section 409D(d) was added to the PHS Act by P.L. 113-55, the Prematurity Research Expansion and Education for

Mothers who deliver Infants Early Reauthorization Act, or the PREEMIE Reauthorization Act, which was signed into

law on November 27, 2013.

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The provision would express the sense of Congress that NIH “grants, contracts, or cooperative

agreements should be awarded, solely for the purpose of supplementing the salaries of new and

early investigators, to entities that participate in the global pediatric clinical study network.”

The provision would express the sense of Congress that FDA “should engage the European

Medicines Agency and other foreign regulatory entities during the formation of the global

pediatric clinical study network to encourage their participation.”

The provision would express the sense of Congress that “once a global pediatric clinical study

network is established and becomes operational, [FDA] should continue to engage the European

Medicines Agency and other foreign regulatory entities to encourage and facilitate their

participation in the network with the goal of enhancing the global reach of the network.”

Section 1083. Appropriate Age Groupings in Clinical Research

Provision

The provision would require the NIH Director, within 180 days of enactment, to convene a

workshop of experts on pediatrics and geriatrics and then publish guidelines regarding

“appropriate age groupings to be included in research studies.” The Director would also be

required to make available to the public, within 180 days after the end of the workshop, the

findings and conclusions of the workshop. At least every other year, the Director would be

required to disclose to the public the number of children included in NIH-supported research,

“disaggregated by developmentally appropriate age group, race, and gender.”

Subtitle F—Advancement of National Institutes of Health Research

and Data Access

Section 1101. Standardization of Data in Clinical Trial Registry Data Bank on

Eligibility for Clinical Trials

Background

Sponsors of clinical trials for drugs, biologics, and devices regulated by the FDA are required to

submit registration and summary results information to ClinicalTrials.gov, the clinical trial

registry and results data bank operated by NIH’s National Library of Medicine (NLM) pursuant to

Sections 402(i)-(j) of the PHS Act. Subparagraph 402(j)(2)(B) requires the NIH Director to

ensure that the public may, in addition to key-word searching, search the entries in the data bank

by various specified criteria, including the disease or condition being studied, the name of the

drug or device under investigation, and the location of the clinical trial. The NIH Director is

instructed to add search categories as deemed necessary and to ensure that the data bank is easy to

use, and that its entries are easily compared.

Provision

The provision would add new language to Section 402(j) of the PHS Act (“Expanded Clinical

Trial Registry Data Bank”) requiring the NIH Director to ensure that (1) the registry and results

data bank is easily used by the public; (2) the registry and results data bank entries are easily

compared; (3) information is submitted to the registry and results data bank in a standardized

format, including certain specified data; and (4) standard terminologies and code sets are used, to

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the extent possible, to facilitate electronic data matching. The provision would strike

subparagraph 402(j)(2)(B).

Within 90 days of enactment, the Secretary would be required to seek the advice of relevant

stakeholders and experts on enhancements to the clinical trial registry data bank that are

necessary to implement the provision. The Secretary would have to begin implementation of the

provision within 18 months of enactment.

Subtitle G—Facilitating Collaborative Research

Section 1121. Clinical Trial Data System

Background

Sponsors of clinical trials for drugs, biologics, and devices regulated by the FDA are required to

submit registration and summary results information to ClinicalTrials.gov, the clinical trial

registry and results data bank operated by NIH’s National Library of Medicine (NLM). Under

Section 402(j) of the PHS Act, those responsible for specified clinical trials of FDA-regulated

products have been required to submit registration information to ClinicalTrials.gov since

December 2007, submit summary results information for clinical trials of approved products

since September 2008, and submit adverse events information since September 2009. The

Secretary is required, by rulemaking, to expand the requirements for submission of summary

results information, and authorized to use rulemaking to make other changes in the requirements

for submission of registration and results information. In November 2014, HHS published a

proposed rule to clarify and expand requirements for the submission of clinical trial registration

and results information to ClinicalTrials.gov.63

Provision

The provision would instruct the Secretary to enter into a seven-year cooperative agreement,

contract, or grant—the Clinical Trial Data System Agreement—with one or more eligible entities

(i.e., tax-exempt academic institutions) to implement a pilot program to enable registered users to

conduct further research on reported clinical trial data. Eligible entities seeking funding would

have to submit an application that contains certain specified information including, among other

things, (1) information demonstrating that the eligible entity can compile clinical trial data in

standardized formats; (2) a description of the system the eligible entity will use to store and

maintain such data; (3) a certification that the eligible entity will allow only registered users to

access and use de-identified clinical trial data; (4) evidence demonstrating the ability of the

eligible entity to ensure that registered users disseminate the results of their research; and (5)

evidence demonstrating that the eligible entity has a proven track record of protecting

confidential data.

Within six years of establishing the pilot program, the Comptroller General would have to study

and report to the Secretary and Congress on the impact and effectiveness of the program,

including recommendations for improving it. Among other things, the report would have to

include information on new discoveries, research inquiries, or clinical trials that resulted from

having access to clinical trial data under the pilot program, as well as an analysis of whether the

Department of Health and Human Services, National Institutes of Health, “Clinical Trials Registration and Results

Submission,” 79 Federal Register 69566, November 21, 2014.

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program had helped reduce adverse events in clinical trials. The Secretary would be able to

extend (including permanently), expand, or terminate the pilot program, in whole or in part, after

the seven-year period expires.

Section 1122. National Neurological Diseases Surveillance System

Background

The PHSA does not explicitly authorize or require surveillance of neurological diseases in

general, although the Secretary may conduct such activities under general authorities in PHSA

Title III. Surveillance is explicitly authorized for certain specified neurological disorders (e.g.,

amyotrophic lateral sclerosis64 and autism spectrum disorder).65

Provision

The provision would add a new PHSA Section 399V-6, “Surveillance of Neurological Diseases.”

It would require the Secretary, acting through the Director of the Centers for Disease Control and

Prevention (CDC)—in consultation with specified stakeholders, and coordinated with other

agencies—to establish a National Neurological Diseases Surveillance System, to include

surveillance of multiple sclerosis and Parkinson’s disease. Required system elements would

include demographic information and risk factors associated or possibly associated with

neurological diseases, and information about diagnosis and progression markers.66 Optional

system elements would include information about the epidemiology, natural history, prevention,

detection, management, and treatment approaches for the diseases; the development of outcomes

measures; and any additional matters identified by stakeholders.

The provision also would authorize the Secretary to furnish grants, contracts, or cooperative

agreements with public or private nonprofit entities to implement this provision. The Secretary

would be required to make information and analysis obtained from the system available to other

federal health agencies (as listed) and state and local agencies, and, subject to HIPAA privacy and

security protections, to the public, including researchers. The Secretary would be required to

report to Congress regarding the system within four years of enactment. The provision would

authorize the appropriation of $5 million for each of fiscal years FY2016 through FY2020.

Section 1123. Data on Natural History of Diseases

Background

The natural history of a disease is its course over time from inception to its eventual end in full

recovery or death. Natural history encompasses exposure or another inciting event, onset and

types of symptoms, and any resulting disability, among other things.

PHSA Section 399S; 42 U.S.C. §280g-7.

PHSA Section 399AA; 42 U.S.C. §280i.

A disease marker is a substance or other measurable parameter that can be used to identify the presence or severity

of a health condition. A progression marker is one that could indicate worsening or improvement in the condition over

time.

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Provision

The provision would express the sense of Congress, in subsection (a), that “studies on the natural

history of diseases can help facilitate and expedite the development of medical products for such

diseases.” Subsection (b) would establish a new PHSA Section 229A. It would authorize the

Secretary to engage in public-private partnerships and award grants in order to gather, analyze,

and interpret data on the natural history of diseases, with a focus on rare diseases. It also would

authorize the Secretary, through these public-private partnerships, to support disease registries

and a secure, flexible information management system, and to provide advice to researchers,

advocacy groups, and others on matters regarding disease studies.

The new PHSA Section 229A also would require the Secretary to make data obtained or

maintained pursuant to this section available to the public (including patient advocacy groups,

researchers, and drug developers), consistent with federal and state privacy laws, in order to

facilitate medical product development. The Secretary would be required to follow applicable

laws protecting privileged or confidential trade secret, commercial, or financial information.

Finally, the provision would authorize the appropriation of $5 million for each of fiscal years

FY2016 through FY2020 to implement this section.

Section 1124. Accessing, Sharing, and Using Health Data for Research

Purposes

Background

The Health Information Portability and Accountability Act (HIPAA) privacy rule describes the

circumstances under which HIPAA-covered entities such as health plans and health care

providers are permitted to use or disclose individually identifiable health information (i.e.,

protected health information, or PHI) without an individual’s written authorization.67 In general,

covered entities may use or disclose PHI for the purposes of treatment, payment, and other

routine health care operations with few restrictions.68 Covered entities also may disclose PHI for

certain public health purposes, including disclosing information about an FDA-regulated product

or activity to an individual subject to FDA’s jurisdiction.69 However, the privacy rule’s definition

of health care operations excludes using or disclosing PHI for the primary purpose of conducting

research (i.e., systematic investigation designed to develop or contribute to generalizable

knowledge).70

The disclosure of PHI to researchers generally requires an individual’s authorization unless an

Institutional Review Board (or equivalent Privacy Board) waives the authorization.71 A covered

entity may, however, allow researchers access to PHI to prepare a research protocol, provided the

PHI is not removed from the covered entity. The privacy rule traditionally has required

authorizations to be study-specific; authorizations for future research were prohibited. In a

January 2013 final rule, HHS permitted authorizations for future research if a sufficiently clear

description of the future research is provided.72 While covered entities may not sell PHI to

The HIPAA privacy rule is codified at 45 C.F.R. Part 164, Subpart E.

45 C.F.R. §164.506.

45 C.F.R. §164.512(b)(1)(iii).

45 C.F.R. §164.501.

45 C.F.R. §164.512(i)(1)(i).

Department of Health and Human Services, Office of the Secretary, “Modifications to the HIPAA Privacy, Security,

(continued...)

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researchers, they are permitted to charge researchers a cost-based fee to cover the preparation and

transmission of the information.73

Provision

This provision would add a new Part 4 to Subtitle D of the HITECH Act instructing the Secretary

to make several revisions or clarifications to the HIPAA privacy rule within 12 months of

enactment. These changes are intended to ease some of the rule’s restrictions on accessing,

sharing, and using health information for research purposes.

First, the Secretary would be required to revise or clarify the privacy rule to allow the use or

disclosure of PHI by a covered entity for research purposes to be treated as health care operations.

However, such disclosures would be treated as health care operations only if they were made to

another covered entity (or to a business associate under contract with the disclosing covered

entity) to perform health care operations, or to a business associate under contract to perform data

aggregation.

Second, the Secretary would be required to revise or clarify the privacy rule to permit the sale of

PHI for research purposes by removing the provision that limits the remuneration to an amount

that covers the cost of preparing and transmitting the information. The Secretary would be

required to further amend the rule so that research on the quality, safety, or effectiveness of FDAregulated products is treated as a public health activity for the purpose of disclosing PHI to an

individual subject to FDA’s jurisdiction.

Third, the Secretary would be required to revise or clarify the privacy rule’s provision that

prohibits researchers from removing PHI during preparation of a research protocol to permit

remote access to PHI by researchers, provided appropriate security and privacy safeguards are in

place and the PHI is not copied or retained by the researchers.

Finally, the Secretary would be required to revise or clarify the privacy rule to allow an

authorization for the use or disclosure of PHI for future research purposes, provided the

authorization (1) sufficiently describes the purposes such that it would be reasonable for an

individual to expect that the PHI could be used or disclosed for future research; (2) states that the

authorization will expire on a particular date or on the occurrence of a particular event; and (3)

states that the authorization will remain valid unless revoked, and provides revocation

instructions.

Subtitle H—Council for 21st Century Cures

Section 1141. Council for 21st Century Cures

Provision

The provision would add to Title II of the PHSA a new Part E, “Council for 21st Century Cures.”

The council would be a non-profit public-private partnership. The purpose of the council would

(...continued)

Enforcement, and Breach Notification Rules Under the Health Information Technology for Economic and Clinical

Health Act and the Genetic Information Nondiscrimination Act; Other Modifications to the HIPAA Rules; Final Rule,”

78 Federal Register 5566, 5611, January 25, 2013.

45 C.F.R. §164.502(a)(5)(ii)(B)(2).

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be to “accelerate the discovery, development, and delivery in the United States of innovative

cures, treatments, and preventive measures for patients.” To accomplish its purpose, the council

would foster collaboration and coordination of those “engaged in the cycle of discovery,

development and delivery of life-saving and health-enhancing innovative interventions.” Other

duties of the council would include communication and dissemination activities; establishing a

strategic agenda to accelerate the discovery, development, and delivery of cures, treatments, and

preventive interventions; developing recommendations based on the identification of gaps and

opportunities within the discovery, development, and delivery cycle; and identifying opportunities

to work with other entities within the United States as well as internationally, such as the

Innovative Medicines Initiative of the European Union.

The council would have a Board of Directors composed of eight ex officio members and 17

appointed members. The ex officio members would consist of the NIH Director, the FDA

Commissioner, the CMS Administrator, and “the heads of five other federal agencies deemed by

the Secretary to be engaged in biomedical research and development.” Within six months of

enactment, the Comptroller General would select the appointed board members. From a list of

nominations made by the leading trade associations, the Comptroller General would select four

representatives of the biopharmaceutical industry, two from the medical device industry, and two

from the information and digital technology industry. In addition, the Comptroller General would

select two academic researchers, three patient representatives, two representatives of health care

providers, and two representatives of health care plans and insurers. The term of appointed

members would be five years. Within 90 days of incorporation of the council and board

appointment, the members of the board would select a Chair, establish the by-laws and policies

for the council, and issue an agenda outlining how it will achieve its purpose. This agenda would

be reviewed and updated annually. The board would be required to meet quarterly, and its minutes

would be publically available and submitted to Congress. The day-to-day management of the

council would be the responsibility of the Executive Director, whose specific duties would be

established by the Board of Directors.

The council would be required to terminate on September 30, 2023. For each fiscal year, FY2016

through FY2023, the provision would authorize appropriations of $10 million to the council. The

council would also be able to “accept financial or in-kind support from participating entities or

private foundations or organizations when such support is deemed appropriate.”

Title II—Development

Subtitle A—Patient-Focused Drug Development

Section 2001. Development and Use of Patient Experience Data to Enhance

Structured Risk-Benefit Assessment Framework

Background

FFDCA Section 505(d), in its instructions on new drug applications, requires the Secretary to

implement a structured risk-benefit assessment framework in the new drug approval

process to facilitate the balanced consideration of benefits and risks, a consistent and

systematic approach to the discussion and regulatory decision making, and the

communication of the benefits and risks of new drugs. Nothing in the preceding sentence

shall alter the criteria for evaluating an application for premarket approval of a drug.

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Provision

The provision would amend FFDCA Section 505 by deleting a clause from Section 505(d) and

adding new subsections (x) and (y). The new 505(x) would restate the deleted 505(d) requirement

for the Secretary to “implement a structured risk-benefit assessment framework in the new drug

approval process.” The new 505(y) would require the Secretary to “establish and implement

processes under which” entities “seeking to develop patient experience data” could submit ideas

and data to the Secretary and the Secretary could request materials from those entities, which

could include the manufacturer and nonmanufacturer groups. This provision would define

“patient experience data” as

data collected by patients, parents, caregivers, patient advocacy organizations, disease

research foundations, medical researchers, research sponsors, or other parties determined

appropriate by the Secretary that is intended to facilitate or enhance the Secretary’s riskbenefit assessments, including information about the impact of a disease or a therapy on

patients’ lives.

The new subsection would also require the Secretary to issue implementation guidance after

holding several methodological workshops and a public meeting.

Subtitle B—Qualification and Use of Drug Development Tools

Section 2021. Qualification of Drug Development Tools

Background

The pharmaceutical industry claims the cost of drug discovery and development is high,

estimated at $1.3 billion to $1.6 billion to bring a drug to market for use in humans.74 Others have

criticized these estimates, claiming they are “false and built on seriously flawed methods” and

that the “true cost is likely to be below $100 million.”75 Lengthy clinical trials have been blamed

as one factor contributing to the high cost of drug development.

Surrogate endpoints—based on the measurement of biomarkers—may be used to determine the

clinical benefit of a product instead of using clinical endpoints. This is because surrogates “enable

smaller, faster, and thus cheaper clinical trials. In addition, pharmaceutical companies argue that

using surrogates means that fewer patients are exposed during testing, and beneficial new

medications reach the market faster. Their main disadvantage is that favorable effects on

surrogates do not automatically translate into benefits to health.”76 For example, Avastin “delayed

Stephen Whitehead, “Making medicines evergreen,” (rapid response), BMJ, December 17, 2012.

Peter C. Gotzsche, “Making medicines evergreen,” (rapid response), BMJ, December 17, 2012. See also: Arnold S.

Relman and Marcia Angell, “America’s other drug problem: how the drug industry distorts medicine and politics,” The

New Republic, December 16, 2002, pp. 27-41; Marcia Angell, “How much does the pharmaceutical industry really

spend on R&D?,” in The truth about the drug companies: How they deceive us and what to do about it (New York:

Random House, 2004), pp. 37-41; Merrill Goozner, The $800 million pill: The truth behind the cost of new drugs

(Berkeley: University of California Press, 2005); and, Donald W. Light, “Misleading Congress about Drug

Development,” Journal of Health Politics, Policy and Law, vol. 32, no. 5 (October 2007), pp. 895-913. One recent

estimate states that the “median costs were a third less than the average, or $60 million. Deconstructing other inflators

would lower the estimate of costs even further.” Donald W. Light and Joel R. Lexchin, “Pharmaceutical research and

development: what do we get for all that money?,” BMJ, August 7, 2012.

Staffan Svensson, David B. Menkes, and Joel Lexchin, “Surrogate Outcomes in Clinical Trials—A Cautionary Tale,”

JAMA Internal Medicine, vol. 173, no. 8 (April 22, 2013), pp. 611-612.

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tumor progression in advanced breast cancer but was not shown to benefit patients.”77 Likewise

Avandia lowered a biomarker level in patients with diabetes but also increased their risk of heart

attack. A number of drugs have been approved on the basis of surrogate endpoint data and, after

adoption into medical practice, have been shown to be harmful through clinical trials or other

subsequent analysis.78 The FDA uses surrogate endpoints in about half of new drug approvals.79

The Institute of Medicine defines a clinical endpoint as “a characteristic or variable that reflects

how a patient [or consumer] feels, functions, or survives. Death is one example of a clinical

endpoint.”80 IOM defines “surrogate endpoint” in the following way:

a biomarker that is intended to substitute for a clinical endpoint. A surrogate endpoint is

expected to predict clinical benefit (or harm or lack of benefit or harm) based on

epidemiologic, therapeutic, pathophysiologic, or other scientific evidence. For example,

blood pressure has served as a surrogate endpoint for morbidity and mortality due to

cardiovascular disease in trials of several classes of antihypertensive drugs. A surrogate

endpoint represents a special use of a biomarker, in which the biomarker substitutes for a

clinical endpoint.81

The Food and Drug Administration Safety and Innovation Act (FDASIA, P.L. 112-144) amended

FFDCA Section 506 by adding the following: “The Secretary shall ... establish a program to

encourage the development of surrogate and clinical endpoints, including biomarkers, and other

scientific methods and tools that can assist the Secretary in determining whether the evidence

submitted in an application is reasonably likely to predict clinical benefit for serious or lifethreatening conditions for which significant unmet medical needs exist.”82

Provision

The provision would add a new FFDCA Section 507, “Qualification of Drug Development

Tools,” which would require the Secretary to establish a process for the qualification of drug

development tools. A drug development tool would be defined to include (1) a biomarker; (2) a

clinical outcome assessment; and (3) any other method, material, or measure that the Secretary

determines aids drug development and regulatory review.

Under new FFDCA Section 507, the Secretary would be allowed to accept a qualification

submission based on factors that include its scientific merit or the available resources of the FDA

to review the submission, and the Secretary would be allowed to prioritize review of a

qualification submission based on factors including, for example, the severity, rarity, or

prevalence of the disease being targeted or the availability or lack of an alternative treatment. The

Secretary would be allowed, through grants or other specified mechanisms, to consult with

biomedical research consortia and may consider the consortia’s recommendations in review of the

qualification submission. “Biomedical research consortia” would be defined as collaborative

Jerry Avorn and Aaron S. Kesselheim, “The 21st Century Cures Act—Will It Take Us Back in Time?,” The New

England Journal of Medicine, June 3, 2015, http://www.nejm.org/doi/full/10.1056/NEJMp1506964.

Staffan Svensson, David B. Menkes, and Joel Lexchin, “Surrogate Outcomes in Clinical Trials—A Cautionary Tale,”

JAMA Internal Medicine, vol. 173, no. 8 (April 22, 2013), Supplementary Online eTable.

Jerry Avorn and Aaron S. Kesselheim, “The 21st Century Cures Act—Will It Take Us Back in Time?,” The New

England Journal of Medicine, June 3, 2015, http://www.nejm.org/doi/full/10.1056/NEJMp1506964.

IOM, Perspectives on Biomarker and Surrogate Endpoint Evaluation: Discussion Forum Summary, January 18,

2011, p.6.

Ibid.

FFDCA §506(d)(2).

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groups that may take the form of public-private partnerships and may include, among others,

government agencies, institutions of higher education, patient advocacy groups, industry

representatives, clinical and scientific experts, and other relevant individuals.83 The Secretary

would be required to carry out a full review of the qualification package and to determine if the

drug development tool at issue is qualified for its proposed context of use.

A qualified drug development tool would be allowed to be used to obtain approval or licensure of

a drug or biologic or to support the product’s investigational use. The Secretary would be allowed

to rescind or modify the granted qualification if she determines the drug development tool is not

appropriate for the proposed context, and, if the Secretary does this, the requestor would be

granted a meeting with the Secretary to discuss the basis of the decision.

New FFDCA Section 507 would require the Secretary to make public, and update at least

biannually, certain information, including, for example, information about the qualification

submissions, the Secretary’s determinations in response to the submissions, and any subsequent

modifications to the Secretary’s determinations. It also specifies that nothing in this section would

be construed to allow the Secretary to release any information contained in an application for

approval or licensure of a drug or biologic that is confidential commercial or trade secret

information; in addition, nothing in the section would be allowed to be construed as altering the

standards of evidence for approval or licensure of a drug or biologic or to limit the Secretary’s

authority to approve or license such products.

New FFDCA Section 507 would authorize to be appropriated $10 million for each of fiscal years

FY2016 through FY2020.

The provision would also require the Secretary, not later than 24 months after enactment, to

publish draft guidance to implement new FFDCA Section 507, in consultation with the

biomedical research consortia and other interested parties through a collaborative public process.

The guidance would be required to, for example, make recommendations for demonstrating that a

surrogate endpoint is reasonably likely to predict clinical benefit for the purpose of supporting

accelerated approval of a drug. The Secretary would be required to issue final guidance not later

than six months after the comment period for the draft guidance closes. In order to inform the

guidance, the Secretary would be required, in consultation with the biomedical research consortia,

to develop a taxonomy for the classification of biomarkers for use in drug development. The

Secretary would be required to make this publicly available not later than 12 months after

enactment and finalize the taxonomy not later than 12 months after the public comment period

closes.

The provision would require the Secretary, not later than 12 months after enactment, to convene a

public meeting regarding the qualification process under new FFDCA Section 507. The Secretary

would also be required to publish a report on FDA’s website, not later than five years after

enactment, to include information, as specified.

Funds from the Cures Innovation Fund, as would be established by Section 4041 of this Act,

would be allowed to be made available to be used to carry out the activities in this provision and

amendments made by this provision.

The provision includes a finding that these consortia can play a valuable role in helping develop and qualify drug

development tools, and a sense of Congress stating that an entity seeking to qualify a drug development tool should be

encouraged to consult with these consortia.

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Section 2022. Accelerated Approval Development Plan

Background

For drugs that address unmet needs or serious or life-threatening conditions, have the potential to

offer better outcomes or fewer side effects, or meet other criteria associated with improved public

health, FDA uses several formal mechanisms to expedite development or review processes.84

These include “priority review,” “breakthrough therapy,” and “fast track” designations, and the

accelerated approval pathway.85 FFDCA Section 506(c) authorizes accelerated approval for a

product to treat a serious or life-threatening disease or condition; this pathway allows the

Secretary to approve an application based on the product’s effect on a “surrogate endpoint that is

reasonably likely to predict clinical benefit” or on a clinical endpoint meeting specified criteria.

The Institute of Medicine defined “surrogate endpoint” as

a biomarker that is intended to substitute for a clinical endpoint. A surrogate endpoint is

expected to predict clinical benefit (or harm or lack of benefit or harm) based on

epidemiologic, therapeutic, pathophysiologic, or other scientific evidence. For example,

blood pressure has served as a surrogate endpoint for morbidity and mortality due to

cardiovascular disease in trials of several classes of antihypertensive drugs. A surrogate

endpoint represents a special use of a biomarker, in which the biomarker substitutes for a

clinical endpoint.86

Provision

The provision would add to FFDCA Section 506 a new subsection (g), which would allow the

sponsor of a drug or biological product to request that the Secretary agree to an accelerated

approval development plan if an application for investigation of the product has been submitted

under FFDCA Section 505(i) or PHSA Section 351(a)(3) and the Secretary determines that the

product may be eligible for accelerated approval under FFDCA 506(c). An accelerated approval

development plan would be defined as a plan agreed on by the Secretary and the sponsor that

contains study parameters for the use of a surrogate endpoint that is reasonably likely to predict

clinical benefit and is intended to be the basis of the accelerated approval of a product under

FFDCA 506(c).

An accelerated approval development plan would include, among other things, agreement on the

surrogate endpoint to be assessed and the design of the study that would utilize the surrogate

endpoint. The provision would authorize the Secretary to require the product sponsor to modify

or terminate the plan if data indicate that the plan is no longer sufficient to demonstrate the safety

of the drug involved or the drug is no longer eligible for accelerated approval; in this case, the

sponsor would be granted a request for a meeting to discuss the basis of the Secretary’s decision.

FDA, “DRAFT Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics,” Center

for Drug Evaluation and Research and Center for Biologics Evaluation and Research, June 2013, http://www.fda.gov/

downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf; and FDA, “Review

Designation Policy: Priority (P) and Standard (S),” MAPP 6020.3 Rev. 2, Manual of Policies and Procedures, Center

for Drug Evaluation and Research, Office of New Drugs, June 25, 2013, http://www.fda.gov/downloads/AboutFDA/

CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM082000.pdf.

See FDA, “DRAFT Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics” table,

pp. 7-8.

Institute of Medicine, 2011, “Perspectives on Biomarker and Surrogate Endpoint Evaluation: Discussion Forum

Summary,” p. 6, http://www.iom.edu/Reports/2011/Perspectives-on-Biomarker-and-Surrogate-EndpointEvaluation.aspx.

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Subtitle C—FDA Advancement of Precision Medicine

Section 2041. Precision Medicine Guidance and Other Programs of Food and

Drug Administration

Background

Precision medicine is a relatively new term for what has traditionally been called personalized

medicine, the idea of providing health care to individuals based on specific patient characteristics.

This approach relies on companion diagnostics to target drugs and biological products to specific

subsets of patients.

Precision drugs and biologicals, because they may be treating small subsets of patients,

sometimes qualify as “orphan drugs.” Such drugs are called orphan drugs because firms may lack

the financial incentives to sponsor products to treat small patient populations. Orphan drugs

receive their designation pursuant to FFDCA Section 526(a),87 a designation that was created by

the Orphan Drug Act88 to encourage firms to develop pharmaceuticals to treat rare diseases and

conditions by providing an extended period of market exclusivity.

For drugs that address unmet needs or serious or life-threatening conditions, have the potential to

offer better outcomes or fewer side effects, or meet other criteria associated with improved public

health, FDA uses several formal mechanisms to expedite development or review processes.89

These include “priority review,” “breakthrough therapy,” and “fast track” designations, and the

accelerated approval pathway.90

Provision

The provision would add a new Subchapter J, Precision Medicine, to Chapter V of the FFDCA;

this subchapter would include two new sections: (1) Section 591, “General agency guidance on

precision medicine,” and (2) Section 592, “Precision medicine regarding orphan-drug and

expedited-approval programs.” New FFDCA Section 591 would require the Secretary, not later

than 18 months after enactment, to issue and periodically update guidance to help sponsors

develop a precision drug or biological product. The guidance would have to, among other things,

define the term “precision drug or biologic product,” and address topics such as the evidence

needed to support the use of biomarkers to identify subsets of patients for streamlining clinical

trials, the design of studies to demonstrate a biomarker’s validity, and considerations for inclusion

of biomarker information in prescription drug or biological labeling.

FFDCA Section 526, “Designation of Drugs for Rare Diseases or Conditions”; 21 U.S.C. 360bb.

P.L. 97-414, 96 Stat. 2049 (1982).

FDA, “DRAFT Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics,” Center

for Drug Evaluation and Research and Center for Biologics Evaluation and Research, June 2013, http://www.fda.gov/

downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf; and FDA, “Review

Designation Policy: Priority (P) and Standard (S),” MAPP 6020.3 Rev. 2, Manual of Policies and Procedures, Center

for Drug Evaluation and Research, Office of New Drugs, June 25, 2013, http://www.fda.gov/downloads/AboutFDA/

CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM082000.pdf.

The fast track and breakthrough therapy designations and the accelerated approval pathway are authorized under

FFDCA Section 506, “Expedited approval of drugs for serious or life-threatening diseases or conditions”; 21 U.S.C.

356. See FDA, “DRAFT Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics,”

table, pp. 7-8.

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For a precision drug or biological product application where the product is for the treatment of a

serious or life-threatening disease or condition and has been designated as an orphan drug under

FFDCA Section 526, the new FFDCA Section 592 would allow the Secretary to do two things.

First, the Secretary would be allowed to rely on information about the drug or biological product

that has been previously submitted, either by the same or a different sponsor (with permission), in

approval of an application. This may be for either a new product, or for a different indication for

an existing product. Second, it would allow the Secretary to consider the application for expedited

review programs, including accelerated approval. New Section 592 should not be construed to

limit the Secretary’s product approval authorities, or to entitle sponsors to obtain information in

another sponsor’s application without permission of the other sponsor.

Subtitle D—Modern Trial Design and Evidence Development

Section 2061. Broader Application of Bayesian Statistics and Adaptive Trial

Designs

Background

The traditional approach to clinical trials for drugs has focused on a design planned in advance

that includes specific treatments and doses and durations, specified decision rules for

patient/subject assignment to treatment groups, and prespecified statistical analysis to test a

prespecified qualitative and quantitative hypothesis. Because the analytic plan is set in advance, it

does not lend itself to unintentional (or intentional) bias as data are reviewed. A researcher may

feel strongly about a hypothesis and hope that the results will confirm an idea, but he or she must

carry out the analysis so the results can be understood and replicated by others. A drawback to

trials with this kind of static design is that they tend to take a long time and cannot adapt to new

information learned during the trial. In recent years, some clinical and methodological researchers

have looked to adaptive trial designs and statistical analyses using techniques (such as Bayesian

statistics) that can provide mid-course feedback. Because a mistaken finding of effectiveness or

safety could put a dangerous drug on the market or delay the approval of a useful drug, FDA has

acted cautiously in accepting alternative trial designs. In 2010, FDA published draft guidance on

the use of adaptive trial design.91

Provision

The provision would require the Secretary to (1) update and finalize the draft guidance and (2)

“issue draft guidance on the use of Bayesian methods in the development and regulatory review

and approval or licensure of drugs and biological products.” It would require that the guidances

address the use of adaptive designs and Bayesian methods to meet the “substantial evidence”

standard in FDA’s review of safety and effectiveness data in marketing applications, technical

feedback to drug sponsors, “the types of quantitative and qualitative information that should be

submitted for review,” and “recommended analysis methodologies.” The provision would require

the Secretary to conduct a public meeting of stakeholders before “updating or developing” these

two guidances. The provision would require the Secretary to publish the first guidance no later

FDA, “DRAFT Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics,” Center for Drug

Evaluation and Research and Center for Biologics Evaluation and Research, February 2010, http://www.fda.gov/

RegulatoryInformation/Guidances/default.htm.

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than 18 months after the date of the public meeting, and the second guidance no later than 48

months after the date of the public meeting.

Section 2062. Utilizing Evidence from Clinical Experience

Background

To approve a new drug for marketing in the United States, FDA reviews the sponsor’s new drug

application (NDA) to assess, among other things, whether the drug is safe and effective for its

intended purpose. FFDCA Section 505(d) refers to “substantial evidence,” which it defines as

evidence consisting of adequate and well-controlled investigations, including clinical

investigations, by experts qualified by scientific training and experience to evaluate the

effectiveness of the drug involved, on the basis of which it could fairly and responsibly

be concluded by such experts that the drug will have the effect it purports or is

represented to have under the conditions of use prescribed, recommended, or suggested in

the labeling or proposed labeling thereof. If the Secretary determines, based on relevant

science, that data from one adequate and well-controlled clinical investigation and

confirmatory evidence (obtained prior to or after such investigation) are sufficient to

establish effectiveness, the Secretary may consider such data and evidence to constitute

substantial evidence for purposes of the preceding sentence. The Secretary shall

implement a structured risk-benefit assessment framework in the new drug approval

process to facilitate the balanced consideration of benefits and risks, a consistent and

systematic approach to the discussion and regulatory decisionmaking, and the

communication of the benefits and risks of new drugs. Nothing in the preceding sentence

shall alter the criteria for evaluating an application for premarket approval of a drug.

The associated rules (21 C.F.R. 314.126) describe characteristics of “adequate and well-controlled

studies,” which include a statement of objectives, an analytic plan, a control group, quantification

of treatment duration and timing, and method of sample size determination. The study design

would lead to the identification of appropriate research subjects and include methods to minimize

bias in the assignment of subjects to treatment groups as well as in data analysis.

These characteristics basically describe a controlled (often randomized) clinical trial. The rule,

however, places these characteristics in the context of having “been developed over a period of

years and are recognized by the scientific community as the essentials of an adequate and wellcontrolled clinical investigation.” The rule states that FDA “consider” these characteristics in its

determination of effectiveness claims.

Provision

The provision would add a new Section 505F to the FFDCA, requiring the Secretary to “establish

a program to evaluate the potential use of evidence from clinical experience to help support the

approval of a new indication for a drug approved under Section 505(b) and to help support or

satisfy postapproval study requirements.” The provision would define “evidence from clinical

experience” as “data regarding the usage, or the potential benefits or risks, of a drug derived from

sources other than randomized clinical trials, including from observational studies, registries, and

therapeutic use.” It would require the Secretary to establish a draft framework for implementing

the program to include specified content. Required consultation with interested parties could be

done via a public-private partnership or a contract, grant, or other appropriate arrangement. The

Secretary would be required to use the new “program to evaluate the potential use of evidence

from clinical experience” to “inform” the development of guidance for industry. The provision

would also state that “[t]this section shall not be construed to alter the standards of evidence

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under” Section 505(c) or (d) of the FFDCA, including the substantial evidence standard; Section

351(a) of the PHSA; or “the Secretary’s authority to require postapproval studies or clinical trials,

or the standards of evidence under which studies or trials are evaluated.”

This provision would add a new Section 505G to the FFDCA, requiring the Secretary to design

and implement pilot demonstrations to use “data captured through the Sentinel System92

surveillance infrastructure” to generate evidence of drugs’ risks or benefits, protect the public

health, and advance patient-centered care. The provision would allow the Secretary to “make

strategic linkages with sources of complementary public health data and infrastructure.”

Regarding these pilots, the Secretary would be required to “(A) consult with regulated industry,

academia, medical professional organizations, representatives of patient advocacy organizations,

disease research foundations, and other interested parties through a public process; and (B)

develop a framework to promote appropriate transparency and dialogue about research.”

In addition, the new FFDCA Section 505G would allow the Secretary to deem such pilot

demonstrations to be “public health activities” for purposes of permitting the use and disclosure

of protected health information (as specified)93 and placing them “outside the scope of

‘research’”94 for purposes of human subjects research protections (as specified).

The provision would also authorize to be appropriated $3 million for each of fiscal years FY2016

through FY2020.

Section 2063. Streamlined Data Review Program

Background

FFDCA Section 505 and accompanying regulations provide the framework for FDA’s approval of

sponsors’ drug marketing applications. For a drug whose active ingredient has never been FDAapproved, the law requires the sponsor to submit a new drug application that includes data to

provide evidence of the drug’s safety and effectiveness for its intended use, information about the

manufacturing process, and the drug labeling. Once a product has an approved NDA, FDA

requires that the manufacturer submit a supplemental NDA each time the manufacturer wants to

change the labeling, the manufacturing process, or the dosing, or when it wants to add a new

indication (a new intended use) of the drug. Regulations at 21 C.F.R. Sections 314.50 and 314.54

describe the required contents of those applications. Regarding clinical data, the regulations direct

the applicant to submit, in addition to descriptions and analysis of controlled and uncontrolled

clinical studies,

(iv) A description and analysis of any other data or information relevant to an evaluation

of the safety and effectiveness of the drug product obtained or otherwise received by the

applicant from any source, foreign or domestic, including information derived from

clinical investigations, including controlled and uncontrolled studies of uses of the drug

other than those proposed in the application, commercial marketing experience, reports in

the scientific literature, and unpublished scientific papers.

(21

C.F.R.

314.50(d)(5)(iv))

In May 2008, FDA launched the Sentinel Initiative “to develop and implement a proactive system”—the Sentinel

System—to actively query existing sources of healthcare data (e.g., electronic health record systems and insurance

claims databases) “to evaluate possible medical product safety issues quickly and securely.” HHS, FDA, FDA’s

Sentinel Initiative, http://www.fda.gov/Safety/FDAsSentinelInitiative/default.htm.

45 C.F.R. §164.512(b)(1).

45 C.F.R. §46.102(d).

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The clinical data submission must also include an “integrated summary of the data demonstrating

substantial evidence of effectiveness for the claimed indications.”

Provision

The provision would add a new Section 505H to the FFDCA that would address the data

requirements in a supplemental NDA that a sponsor of an approved drug would submit when

seeking to add to the approval a new indication that is “qualified” (defined in this section as

treating cancer or other indications as determined by the Secretary). FFDCA Section 505H would

require the Secretary to “establish a streamlined data review program” through which a sponsor

could submit a “qualified data summary,” defined as “a summary of clinical data intended to

demonstrate safety and effectiveness with respect to a qualified indication for use of a drug,”

when “there is an existing database acceptable to the Secretary regarding the safety of the drug

developed for one or more indications” of the approved drug. The sponsor would also be required

to submit “the full data sets used to develop the qualified data summaries ... unless the Secretary

determines that the full data sets are not required.”

The provision would state a sense of Congress that the new streamlined data review program

“should enable the Food and Drug Administration to make approval decisions for certain

supplemental applications based on qualified data summaries (as defined in such section 505H).”

The provision would require that the Commissioner of Food and Drugs issue implementation

guidance for the streamlined data review program and would allow the Commissioner to issue

regulations for implementation.

Subtitle E—Expediting Patient Access

Section 2081. Sense of Congress

Background

FDA uses several formal mechanisms to expedite the development or review processes for drugs

that address unmet needs or serious conditions, that have the potential to offer better outcomes or

fewer side effects, or that meet other criteria associated with improved public health. FFDCA

Section 506, which Congress added in 2012,95 introduced the breakthrough therapy designation

for a drug that would treat a serious condition and for which preliminary clinical evidence

indicates that the drug may demonstrate substantial improvement over available therapies on a

clinically significant endpoint (or endpoints). FDA provides breakthrough therapies with

intensive guidance during drug development and organizational commitment involving senior

managers. The requirements for drug approval, however, do not change.96 Breakthrough therapy

designation, therefore, affects the timing and smoothness of the application process. Such

designation does not alter the types of evidence required to demonstrate safety and effectiveness.

Section 902 of the Food and Drug Administration Safety and Innovation Act (FDASIA), P.L. 112-144.

The FFDCA and agency regulations allow alteration of the evidence required for approval in other circumstances;

these alterations are not connected to breakthrough designation.

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Provision

The provision would express the sense of Congress that FDA should approve drugs designated as

breakthrough therapies “as early as possible in the clinical development process, regardless of the

phase of development,” provided that the applications meet existing standards of evidence of

safety and effectiveness, as determined by the HHS Secretary.

Section 2082. Expanded Access Policy

Background

FDA regulates the U.S. sale of drugs and biological products, basing approval or licensure on

evidence of the safety and effectiveness for a product’s intended uses. Without that approval or

licensure, a manufacturer may not distribute the product except for use in the clinical trials that

will provide evidence to determine that product’s safety and effectiveness. Under certain

circumstances, however, FDA may permit the sponsor to provide an unapproved or unlicensed

product to patients outside that standard regulatory framework. One such mechanism is expanded

access to investigational drugs, commonly referred to as compassionate use.

If excluded from a clinical trial because of its enrollment limitations, a person, acting through a

physician, may request access to an investigational new drug outside of the trial. FDA may grant

expanded access to a patient with a serious disease or condition for which there is no comparable

or satisfactory alternative therapy, if, among other requirements, probable risk to the patient from

the drug is less than the probable risk from the disease; if there is sufficient evidence of safety and

effectiveness to support the drug’s use for this person; and if providing access “will not interfere

with the ... clinical investigations to support marketing approval.”97 The widespread use of

expanded access is limited by an important factor: whether the manufacturer agrees to provide the

drug, which—because it is not FDA-approved—cannot be obtained otherwise. FDA does not

have the authority to compel a manufacturer to participate. Manufacturers consider several factors

in deciding whether to provide an investigational drug, such as available supply, perceived

liability risk, limited staff and facility resources, and need for data to assess safety and

effectiveness. Although FDA reports the number of investigational drug requests it receives,

manufacturers do not.

Provision

The provision would add a new Section 561A to the FFDCA to require the manufacturer or

distributor of an investigational drug to make publicly available its policy “on evaluating and

responding to requests ... for provision of such a drug.” Required elements of the policy would

include contact information for the manufacturer or distributor of the drug, request procedures,

“the general criteria the manufacturer or distributor will consider or use to approve such

requests,” and anticipated time to acknowledge request receipts. The new section would state that

posting of policy would not guarantee patients access to an investigational drug. The provision

would also allow the manufacturer or distributor to revise its policy at any time.

FFDCA Section 561(b).

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Section 2083. Finalizing Draft Guidance on Expanded Access

Background

FFDCA Section 561(b) allows a person, acting through a licensed physician, to request a

manufacturer or distributor of an investigational product to provide that product under specified

circumstances and conditions. The sponsor or clinical investigator must provide the HHS

Secretary with information as required by regulations. Although FDA has approved patient access

in over 99% of the requests to which the sponsor has agreed, some sponsors have been reluctant

to provide investigational drugs outside of the standard investigational new drug (IND) processes

because of the uncertainty of how FDA would consider potential adverse events associated with

the expanded access use in its assessment of the drug’s safety, which could influence whether an

NDA is approved.

Provision

This provision would require that the HHS Secretary finalize the guidance “Expanded Access to

Investigational Drugs for Treatment Use—Qs & As,” issued in draft form in May 2013.98 The

provision would require that the final guidance “clearly define how the Secretary of Health and

Human Services interprets and uses adverse drug event data reported by investigators in the case

of data reported from use under a request submitted under” FFDCA Section 561(b).

Subtitle F—Facilitating Responsible Manufacturer

Communications

Section 2101. Facilitating Dissemination of Health Care Economic Information

Background

FFDCA Section 502 includes “[i]f its labeling is false or misleading in any particular” in the list

of circumstances under which a drug is “deemed to be misbranded.” It allows a drug’s sponsor

(usually its manufacturer or distributor) to provide health care economic information to entities

such as formulary committees for use in decisions regarding drug selection for managed care. The

section defines “health care economic information” to mean “any analysis that identifies,

measures, or compares the economic consequences, including the costs of the represented health

outcomes, of the use of a drug to the use of another drug, to another health care intervention, or to

no intervention.” The information must be “based on competent and reliable scientific evidence.”

Provision of this information is allowed only regarding indications that are included in the drug’s

approval; the provision of health care economic information regarding unapproved indications

could be considered false and misleading.

Provision

The provision would amend the description of the recipient of the information to include a

“payor” and to refer to the use of the information in the “selection of drugs for coverage or

FDA, “DRAFT Guidance for Industry: Expanded Access to Investigational Drugs for Treatment Use—Qs & As,”

May 2013, http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm351261.pdf

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reimbursement.” The requirement that information be “based on competent and reliable scientific

evidence” would be expanded to include “where applicable, a conspicuous and prominent

statement describing any material differences between the health care economic information and

the labeling approved for the drug.”

The provision would amend the definition of “health care economic information” to include “the

clinical data, inputs, clinical or other assumptions, methods, results, and other components

underlying or comprising the analysis.” It would specify that the economic consequences “may be

based on the separate or aggregated clinical consequences of the represented health outcomes, of

the use of a drug.” Lastly, the revised definition would rephrase the reference to comparisons,

without an apparent change in authority or policy.

Section 2102. Facilitating Responsible Communication of Scientific and

Medical Developments

Background

FFDCA Section 505 governs approval of new drugs; approval is linked to the intended use

(indication) of the drug, for which FDA reviews evidence of safety and effectiveness that the

sponsor provides. Approval is also linked to the drug labeling provided by the sponsor. Intended

uses not covered by the approval are not included in the labeling.

FFDCA Section 502 describes circumstances under which a product is to be deemed misbranded.

Among those are when labeling is false or misleading. FDA has interpreted the FFDCA,

therefore, to prohibit a manufacturer from promoting or advertising a drug for any use not listed

in the FDA-approved labeling, which contains those claims for which FDA has reviewed safety

and effectiveness evidence.99 However, FDA’s interpretation has been challenged and is in

dispute.100

FDA has acknowledged that the manufacturer has information that may be useful to clinicians in

their treatment of patients. In a 2009 guidance,101 for example, the agency noted that “[o]nce a

drug or medical device has been approved or cleared by FDA, generally, healthcare professionals

may lawfully use or prescribe that product for uses or treatment regimens that are not included in

the product’s approved labeling (or, in the case of a medical device cleared under the 510(k)

process, in the product’s statement of intended uses).” FDA, therefore, recognized the “public

health and policy justification” in allowing certain information on unapproved uses of approved

products. FDA has released several draft guidance documents102 to characterize the circumstances

Materials from FDA’s Bad Ad Program describe elements of false or misleading ads. These include promotion of an

unapproved use. See FDA, “Truthful Prescription Drug Advertising and Promotion,” http://www.fda.gov/Drugs/

GuidanceComplianceRegulatoryInformation/Surveillance/DrugMarketingAdvertisingandCommunications/

ucm209384.htm#ExamplesofViolations. See also FFDCA §§ 301 and 502(a).

100

A December 2012 Court of Appeals decision (United States v. Caronia) “reversed the criminal conviction of a

pharmaceutical representative who had promoted an off-label use of a drug on First Amendment grounds” ((name r

edacted), “Is There a Constitutional Right to Promote an Unapproved Use for a Drug?” Legal Sidebar, Congressional

Research Service, April 2, 2013, http://www.crs.gov/LegalSidebar/details.aspx?ID=436&Source=search).

101

FDA, “Guidance for Industry: Good Reprint Practices for the Distribution of Medical Journal Articles and Medical

or Scientific Reference Publications on Unapproved New Uses of Approved Drugs and Approved or Cleared Medical

Devices,” Office of the Commissioner, Office of Policy, January 2009, http://www.fda.gov/RegulatoryInformation/

Guidances/ucm125126.htm.

102

FDA, “‘Off-Label’ and Investigational Use Of Marketed Drugs, Biologics, and Medical Devices - Information

Sheet,” Guidance for Institutional Review Boards and Clinical Investigators, http://www.fda.gov/

(continued...)

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in which it would allow manufacturers to provide information on off-label uses of drugs approved

for other uses without triggering the misbranding provision and without using a manufacturer’s

dissemination of the information as evidence of an intended new indication.

Provision

The provision would require the HHS Secretary to “issue draft guidance on facilitating the

responsible dissemination of truthful and non-misleading scientific and medical information not

included in the approved labeling of drugs and devices.”

Subtitle G—Antibiotic Drug Development

Section 2121. Approval of Certain Drugs for Use in a Limited Population of

Patients

Background

According to the CDC, each year in the United States, at least 2 million people become infected

with bacteria that are resistant to antibiotics, and at least 23,000 of them die from these

infections.103 Antibiotics are intended for short-term use, making the development of new ones

potentially less attractive to drug developers. Addressing barriers to antibiotic drug approval may

help counter this problem. One such proposal is the so-called Limited Population Antibacterial

Drug (LPAD) approval pathway for new antibacterial drugs.104 Such a pathway would involve

smaller clinical trials in a limited population of patients that have serious or life-threatening

infections and unmet medical needs due to the lack of an effective approved antibiotic. This

streamlined approach would result in more uncertainty about potential risks posed by the product,

and therefore a greater need for post-market scrutiny.105

(...continued)

regulatoryinformation/guidances/ucm126486.htm; FDA, “DRAFT Guidance for Industry: Distributing Scientific and

Medical Publications on Risk Information for Approved Prescription Drugs and Biological Products—Recommended

Practices,” Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for

Veterinary Medicine, June 2014, http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/

Guidances/UCM400104.pdf; FDA, “Revised DRAFT Guidance for Industry: Distributing Scientific and Medical

Publications on Unapproved New Uses—Recommended Practices,” Center for Drug Evaluation and Research, Center

for Biologics Evaluation and Research, and Center for Devices and Radiological Health, February 2014,

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM387652.pdf; and

FDA, “DRAFT Guidance for Industry: Responding to Unsolicited Requests for Off-Label Information About

Prescription Drugs and Medical Devices,” Center for Drug Evaluation and Research, Center for Biologics Evaluation

Research, Center for Veterinary Medicine, and Center for Devices and Radiological Health, December 2011,

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm285145.pdf.

103

Centers for Disease Control and Prevention (CDC), “Antibiotic Resistance Threats in the United States, 2013,”

http://www.cdc.gov/drugresistance/threat-report-2013/.

104

See for example testimony of Janet Woodcock, Director, FDA Center for Drug Evaluation and Research, U.S.

Congress, House Committee on Energy and Commerce, Subcommittee on Health, 21st Century Cures: Examining

Ways to Combat Antibiotic Resistance and Foster New Drug Development, 113th Cong., 2nd sess., September 19, 2014,

http://www.fda.gov/newsevents/testimony/ucm415387.htm.

105

Ibid. See also Executive Office of the President, President’s Council of Advisors on Science and Technology

(PCAST), Report to the President on Combating Antibiotic Resistance, “Goal 4.2. Drug approval based on clinical

trials in limited patient populations,” September 2014, pp. 32 ff., https://www.whitehouse.gov/administration/eop/ostp/

pcast.

Congressional Research Service

H.R. 6: The 21st Century Cures Act

Provision

The stated purpose of this provision, in subsection (a), is

to help expedite the development and availability of treatments for serious or lifethreatening bacterial or fungal infections in patients with unmet needs, while maintaining

safety and effectiveness standards for such treatments, taking into account the severity of

the infection and the availability or lack of alternative treatments.

It would do so by adding, in subsection (b), a new FFDCA subsection 505(z), an expedited

review pathway, effective upon enactment, for certain antibacterial and antifungal drugs

(including biologics) intended for use in limited, defined populations of patients that have severe,

life-threatening infections for which current treatment options may be limited or absent, and for

which the benefits of a product could outweigh harms that would not be acceptable in broader

population use. This review pathway would include the following elements, among others:



Upon a sponsor’s request, FDA may enter into written agreement with the

sponsor to define the process and data needed to review the limited population

use application. The process could not proceed without such written agreement.

The Secretary may consider limited data sets and non-clinical data as substantial

evidence of safety and effectiveness, recognizing the smaller populations

available for study of an LPAD drug, and the different balance of benefit versus

harm in these populations.

The process must adhere to existing goals and procedures agreed upon by

sponsors and FDA in the Prescription Drug User Fee Amendments of 2012 (P.L.

112-144, Title I).

Products approved using this pathway must carry prominent labeling noting the

intended use for a limited and specific population of patients.

Sponsors must submit promotional materials to FDA for review 30 days prior to

dissemination.

Sponsors may pursue this pathway concurrently with other specified streamlined

approval pathways, as applicable.

This provision would not alter current prescribing or other medical practices

(such as off-label prescribing).

Subsection (c) of this provision would require FDA to issue draft implementation guidance within

18 months of enactment. Subsection (d) provides conforming amendments. Subsection (e) would

require the Secretary to conduct and publish an assessment of the program within 48 months of

enactment, and seek public input. Subsection (f) would allow the Secretary to expand the limited

population use pathway if deemed beneficial by the assessment above. Subsection (g) would add

a new subsection 317U to the PHSA to establish a monitoring system for the use of antibacterial

and antifungal drugs, including products approved under the limited population use pathway, as

well as changes in bacterial and fungal resistance to drugs. The Secretary would be required to

make summaries of data from this system publicly available.

Section 2122. Susceptibility Test Interpretive Criteria for Microorganisms

Background

Laboratory tests can help clinicians determine whether a drug is likely to work against a specific

infection by showing whether the infectious organism is susceptible (vs. resistant) to that drug.

Congressional Research Service

H.R. 6: The 21st Century Cures Act

The criteria that distinguish susceptibility from resistance are called “breakpoints.” Under current

law and regulation, breakpoint information must be provided on antimicrobial drug labels, and

labels for Antimicrobial Susceptibility Testing (AST) devices must reflect the relevant drug

label(s). Generally, sponsors must apply to make changes to information contained in these drug

and device labels, and FDA must approve label changes for these drugs and devices. However,

the susceptibility of infectious organisms may change over time, rendering label information

inaccurate for clinical decision-making purposes. FDA, clinicians, and others have sought to

streamline FDA’s process to ensure that antimicrobial drug and AST device labels reflect current

information.106

Provision

Subsection (a) of this provision would replace the existing language of FFDCA Section 511

(which requires the Secretary to publish guidance for industry regarding the review of antibiotic

drugs) with new language. It would require the Secretary to establish, within one year of

enactment, a public “Interpretive Criteria Website,” and to review and revise the content of such

website every six months thereafter. The stated purpose of the website is to identify and publish

current, generally accepted standards, including breakpoint information (i.e., interpretive criteria),

used to guide testing of test bacteria, fungi, or other microorganisms for susceptibility to

antimicrobial drugs, and to use these criteria to inform the use of AST devices.

The Secretary would be required to identify appropriate susceptibility test interpretive criteria

(“criteria”) for approved or licensed antimicrobial drugs through review of preclinical, clinical,

and statistical information and other available evidence. Within one year of enactment, the

Secretary would be required to establish, and thereafter maintain, the Interpretive Criteria Website

containing two lists: (1) a list of any criteria standards established by a nationally or

internationally recognized standard development organization, where such organization meets

specified requirements for transparency and management of potential conflicts of interest, among

other things; and (2) a list of criteria that, although determined by the Secretary to be appropriate

with respect to approved or licensed antimicrobial drugs, lack a recognized standard, for one of

several stated reasons. The website would have to include several specific disclaimers regarding

the uses and limitations of the information presented. The Secretary would be required to publish

in the Federal Register a notice of establishment of the website not later than the date on which it

is established.

The Secretary would be required to review any new or updated criteria standards from a

recognized standard development organization, revise the website accordingly, and make public a

notice of any such revisions on the FDA agency website, at least every six months. Any such

notices would be required to be compiled and published in the Federal Register at least annually,

with a request for public comments. The Secretary would be allowed to consider public

comments, among other things, in revising website content.

Both criteria standards and non-standard criteria listed on the website would be considered to be

recognized standards for the purpose of premarket review and other legal requirements for

devices, pursuant to FFDCA Section 514(c)(1). However, sponsors would be allowed to use

106

See, for example, testimony of Janet Woodcock, Director, FDA Center for Drug Evaluation and Research, U.S.

Congress, House Committee on Energy and Commerce, Subcommittee on Health, 21st Century Cures: Examining

Ways to Combat Antibiotic Resistance and Foster New Drug Development, 113th Cong., 2nd sess., September 19, 2014,

http://www.fda.gov/newsevents/testimony/ucm415387.htm; and FDA, “Guidance for Industry, Updating Labeling for

Susceptibility Test Information in Systemic Antibacterial Drug Products and Antimicrobial Susceptibility Testing

Devices,” June 2009.

Congressional Research Service

H.R. 6: The 21st Century Cures Act

standards other than those listed by FDA under this section in seeking approval or clearance of a

drug or device. The provision would require that antimicrobial drugs sold after the Interpretive

Criteria Website is established carry a reference to the website on the label, and that sponsors of

antimicrobial drugs sold before the website was established submit, within one year of

establishment of the website, supplemental applications to similarly change the label. The

provision would clarify that reference to the website in the labeling of an antimicrobial drug

would not constitute misbranding, and state that FFDCA Section 511 should not be construed to

allow the Secretary to disclose protected trade secret or confidential information.

The provision would allow the Secretary to authorize the marketing of an AST device for which

the label references information from the website in lieu of information from clinical trials, and

directs practitioners to information on the labels of drugs tested using such device.

Subsection (b) of the provision would make conforming amendments to the FFDCA. Subsection

section. Subsection (d) would exempt FDA from requirements under the Paperwork Reduction

Act when updating the list of susceptibility test interpretive criteria standards.107 Subsection (e)

states that provisions of Subtitle G of the bill should not be construed to restrict antibiotic or other

drug prescribing or administering practices by health care practitioners.

Section 2123. Encouraging the Development and Use of DISARM Drugs

Background

Under Medicare’s Hospital Inpatient Prospective Payment System (IPPS), reimbursement is often

predetermined for each discharge based on a patient’s condition and related treatment strategy,

and other factors. To account for patients’ needs, Medicare assigns discharges to Medicareseverity diagnosis related groups (MS-DRGs). The capitated MS-DRG payment can discourage

the use of new technologies if they are more expensive than standard care. To address this,

Sections 1886(d)(5)(K) and (L) of the Social Security Act (SSA) authorize additional payments

for new medical services and technologies under the IPPS in addition to the MS-DRG

reimbursement.108

In 2012, Congress passed the Generating Antibiotic Incentives Now

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