FDA Plan for Statutory Compliance
Federal RegisterNov 24, 1998
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 98N-0339]
FDA Plan for Statutory Compliance
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
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SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a document entitled ``FDA Plan for Statutory
Compliance'' (the plan). This document is the agency's response to
section 406(b) of the Food and Drug Administration Modernization Act of
1997 (FDAMA), which requires the Secretary of the Department of Health
and Human Services (the Secretary) to develop a plan bringing the
agency into compliance with the requirements of the Federal Food, Drug,
and Cosmetic Act (the act).
DATES: Written comments may be submitted at any time.
ADDRESSES: Submit written comments on the plan to Dockets Management
Branch (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm.
1061, Rockville, MD 20852. Submit e-mail comments to
``FDAD[email protected]''. E-mail comments should be labeled as
comments and identified with the docket number found in brackets in the
heading of this document.
Submit written requests for single copies of the ``FDA Plan for
Statutory Compliance'' to the Dockets Management Branch (address
above). Enclose one self-addressed adhesive label to assist that office
in processing your requests. Copies of this plan are available on the
Internet at ``http://www.fda.gov/opacom/7modact''.
FOR FURTHER INFORMATION CONTACT: Steven H. Chasin, Office of Planning
and Evaluation (HFP-20), Food and Drug Administration, 5600 Fishers
Lane, Rockville, MD 20857, 301-827-5207.
SUPPLEMENTARY INFORMATION:
I. Background
On November 21, 1997, the President signed FDAMA into law. Section
406(b) of FDAMA requires the Secretary, after consultation with
appropriate scientific and academic experts, health care professionals,
representatives of patient and consumer advocacy groups, and the
regulated industry, to develop and publish a plan bringing the
Secretary into compliance with each of the obligations of the Secretary
under the act. The plan is to be reviewed biannually and revised as
necessary, in consultation with the groups listed in the previous
sentence. The plan must address the following six objectives: (1)
Maximizing the availability and clarity of information about the
process for review of applications and submissions made under the act;
(2) maximizing the availability and clarity of information for
consumers and patients concerning new products; (3) implementing
inspection and postmarket monitoring provisions of the act; (4)
ensuring access to the scientific and technical expertise needed by the
Secretary to meet the obligations of the Secretary under the act; (5)
establishing mechanisms, by July 1, 1999, for meeting the time periods
specified in the act for the review of applications and submissions
made under the act and submitted after November 21, 1997; and (6)
eliminating backlogs in the review of applications and submissions
described previously by January 1, 2000.
Over the past several months, the agency held a series of meetings
with its stakeholders. The process of consulting with agency
stakeholders began with a careful examination of FDA's stakeholders
vis-a-vis the products regulated by the agency and the perceived
interest of these groups in FDA's processes. A total of eight open
public meetings were held where agency stakeholders had an opportunity
to provide their perspectives on a variety of issues/questions. Six of
the eight meetings were focused specifically on FDA's product centers;
one briefing for health professionals provided an opportunity for
health professionals to offer input to FDA under the broad guidance of
section 406(b) of FDAMA; and an agency-wide meeting was held to capture
the perspectives of those who could not attend previous meetings and to
provide an opportunity to explore recurring themes from previously held
meetings.
In addition to the open public meetings focused specifically on
section 406(b) of FDAMA, agency staff used a variety of ongoing
interactions with stakeholders as opportunities to talk about the
stakeholder consultation process and to invite comments to the docket.
II. The Plan
The agency plan for statutory compliance has been developed in
response to the requirements outlined in section 406(b) of FDAMA. The
plan presents a blueprint for carrying out all of the agency's
statutory obligations, including provisions of the act, as well as its
other mandates.
The plan outlines FDA's strategic directions for the next 5 years
and presents an operational plan for fiscal year 1999 and 2000. The
plan is a dynamic document which will be modified as ongoing
consultations with FDA stakeholders render new and more effective
strategies.
The act itself builds upon a long history of recommendations from
advisory committee members, industry representatives, and consumers to
help the agency respond to new challenges while still fulfilling its
mission and mandates. It was Congress' belief that FDA could address
these challenges by re-engineering several of its regulatory processes
to achieve greater efficiencies and by buttressing its considerable
risk assessment and risk management expertise through productive,
collaborative relationships with key external stakeholders.
III. Comments
Interested persons, may at any time, submit written comments to
the Dockets Management Branch (address above) regarding this plan. Two
copies of any comments are to be submitted, except individuals may
submit one copy. Comments should be identified with the docket number
found in brackets in the heading of this document.
Submit e-mail comments to ``FDAD[email protected]''. E-mail
comments should be labeled as comments and identified with the docket
number found in brackets in the heading of this document. A copy of the
document and received comments are available for public examination in
the Dockets Management Branch between 9 a.m. and 4 p.m., Monday through
Friday.
The text of the plan follows:
BILLING CODE 4160-01-F
Food and Drug Modernization Act of 1997--FDA Plan for Statutory
Compliance
November 1998
Table of Contnets
Executive Summary
Part One: Strategic Framework
Purpose
Scope
The Mandated Strategic Framework
FDA's Strategic Management Approach
Mission Development
Emerging FDA Challenges
Analysis of the Gap Between What is Expected of FDA and Its
Actual Performance
Stakeholder Consultation
Identification of Agency-wide Objectives and Strategic
Directions
[[Page 65001]]
Part Two: FDAMA Plan for FY 1999
Objective A: Information about Review Processes
Objective B: Information about New Products
Objective C: Implementing Inspection and Postmarket Monitoring
Provisions
Subobjective C1: Assuring Product Safety
Subobjective C2: Adverse Event Reporting
Objective D: Science and Research
Objectives E and F: Eliminating Backlogs
Appendices
Executive Summary: FDA Plan for Statutory Compliance
Purpose
The FDA Plan for Statutory compliance addresses requirements set
forth in Section 406 of the Food and Drug Administration Modernization
Act of 1997 (FDAMA). The Plan identifies those actions necessary to
bridge the gap between what FDA is required to do by statute and what
it is able to accomplish with current resources. FDAMA has presented
FDA with an opportunity to close that gap by working in concert with
its community of stakeholders to protect the health and well-being of
the American public. This Plan is a positive first step. It outlines
bold and innovative approaches to meet the increasingly complex public
health challenges of the 21st century.
FDA, however, is unable to meet all of these challenges with its
current level of resources. Innovation and creative collaboration with
stakeholders will enhance this effort, but significant additional
resources, as well as prioritization of FDA activities, are essential
if FDA is to meet its statutory requirements on a sustained basis and
to meet public expectations. The successful implementation of this Plan
depends on commitment of resources by both FDA and its stakeholders.
Scope
The Plan specifically addresses each of the objectives stipulated
by Congress in FDAMA Section 406(b). These objectives, when achieved,
will result in the following outcomes: stakeholders who are well
informed about and involved in the Agency's new products and regulatory
processes; comprehensive monitoring of industry practices and product
use; regulatory decisions that are supported by a sound science base;
and on-time reviews of new products prior to market entry.
To accomplish these objectives the Plan outlines FDA's strategic
directions over the next 5 years and specific performance goals for
Fiscal Year (FY) 1999. The Plan was developed in close consultation
with a wide range of stakeholders, including consumers and patients,
industry, health professionals, and other public sector regulators. The
end product represents the collective views of FDA's senior leaders and
its community of stakeholders.
The Plan
FDA Challenges in Fulfilling Its Mission: FDA must address several
key challenges now and in the future for the Agency to successfully
meet its statutory requirements and to fulfill its health promotion and
protection mission. These include: research and development-fueled
pressures on regulatory responsibilities; greater product complexity
driven by breakthroughs in technology; growth in recognized adverse
effects associated with product use; unpredictable new health and
safety threats; awareness of citizen-stakeholders and their more
targeted needs; emerging regulatory challenges in the international
arena; and increased volume and diversity of imports. The ability to
formulate successful solutions to these challenges depends on
innovative approaches used by FDA, creative collaboration with
stakeholders, prioritization of FDA activities, and an adequate
investment of resources to implement these approaches.
Stakeholder Views: FDA's senior leadership listened carefully to
the viewpoints of its many stakeholders prior to the development of
this Plan. These opinions were expressed during a series of public
meetings held during the summer of 1998. Several productive suggestions
surfaced from these discussions. Two general themes emerged:
(1) Greater stakeholder involvement: Stakeholders want to be
ongoing contributors to FDA's future strategies. Effective
collaboration can raise the likelihood that these strategies will be
successful. Stakeholders also want to be well-informed about FDA's
regulatory processes. Consumers and patients want clear information
about new products, and they want to receive the information in a
timely manner.
(2) Balanced, risk-based FDA decisions: Stakeholders agreed that
FDA priorities should be risk-based, and also believe that the Agency
should balance timely premarket review programs with the need for
effective postmarket inspection and surveillance. They urged the Agency
to continue to develop a strong scientific and analytical basis for
regulatory decisions. Some urged FDA to rely more on third parties and
others want more direct FDA regulation.
Current Innovations/Reinventions: While stakeholders have made
useful suggestions for enhancing Agency programs, FDA had already begun
steps to improve its approach to public health protection and is
continuing this effort. This has been accomplished both through
redesign of internal programs and via collaborative efforts with
outside parties. New, critically important medicines are now reaching
the market more rapidly as a result of more efficient Agency review
processes and the automation of these processes. Since 1993, the medium
approval time for new drugs has been substantially reduced, from 20
months to around 12 months in 1997. FDA is collaborating with its
regulatory colleagues as well as the regulated industry to develop
national systems of consumer protection. Two examples are cited: FDA is
working closely with the U.S. Department of Agriculture, the Center for
Disease Control and Prevention, and the states to develop a
comprehensive network for ensuring safety of the American food supply.
FDA is also coordinating with the international regulatory community
and the U.S. Customs service to increase assurance that imports
entering the country are safe.
Strategic Directions for the Future: FDA's senior leadership
identified the following strategic directions in order to focus the
Agency's energies on meeting the objectives set forth in the Plan:
Establish risk-based priorities--Focus resources on those
health and safety risks that most directly threaten the well-being of
U.S. consumers.
Strengthen the scientific and analytical basis for
regulatory decisions--A strong science base must underpin each of the
Agency's regulatory decisions.
Work more closely with external stakeholders--
Collaboration with stakeholders will result in more effective solutions
to public health problems.
Continue to re-engineer FDA processes--Re-engineering will
result in regulatory simplification and more cost-effective ways to run
FDA's internal processes.
Adopt a systems approach to Agency regulation--Regulatory
approaches in the future will look for total problem solutions, rather
than piecemeal review and enforcement decisions.
Capitalize on information technology--Information
technology will help to improve both internal efficiency and
communication with stakeholders.
The six strategic directions outlined above will guide FDA's
efforts to meet the FDAMA objectives. Many factors over the next
several years will have an impact on FDA's ability to meet these
[[Page 65002]]
objectives including the outcome of a risk-based priority system, the
success of third parties in the regulatory process, improvements in
technology and systems engineering, and the synergies created by
greater collaboration with other federal agencies, as well as FDA's
external stakeholders, new statutory mandates, and emerging public
health responsibilities. Reinvention will enable FDA to make up some of
the difference between current performance and FDAMA objectives.
Additional resources will also be necessary over the next 5 years in
order for the Agency to satisfy its statutory requirements and to meet
public expectations.
The body of this Plan identifies the major areas where FDAMA calls
for FDA to meet statutory requirements, such as premarket reviews,
injury reporting, and product safety assurance. It also discusses areas
where there are not statutory requirements, but where there is general
agreement on what time frames for reviews and inspections are
appropriate and what other work needs to be accomplished to meet FDAMA
objectives. FDA would be hard pressed to meet all of the FDAMA
objectives with current resources and operating procedures. For
example, in FY 1999 the Agency estimates it can accomplish roughly one-
half to three-quarters of its statutory inspectional workload with
current funding (See FIGURE 3).
Plan Organization
Part One of the Plan, the strategic framework, provides the broad
Agency-wide context of the Plan. This includes:
(1) development of a clear mission statement;
(2) assessment of challenges that FDA faces in fulfilling its mission;
(3) analysis to the gap between what is expected of FDA and its actual
performance;
(4) consulting FDA's stakeholders on future directions; and
(5) a statement of Agency-wide objectives (Section 406(b)) and
strategic directions to achieve the objectives.
Part Two of the Plan maps the specific plan for achieving each
406(b) objective, including strategies and performance goals that can
be used to manage toward the objectives. In Part Two, the specific
performance targets for FY 1999 are established based on the Agency's
existing resources, reinventions, and collaborative arrangements. FY
2000 performance targets currently are being developed as part of the
FY 2000 Budget process and are not included in the Plan.
Part One--Strategic Framework
Purpose
The FDA Plan for Statutory Compliance addresses requirements set
forth in Section 406 of the Food and Drug Administration Modernization
Act of 1997 (FDAMA) (see Appendix A). The Plan identifies those actions
necessary to bridge the gap between what FDA is required to do by
statute* and expected to do by the public--and what the Agency
currently is able to accomplish with existing resources. A high-
performing FDA working in concert with its stakeholders is absolutely
crucial to promote and to protect the health and well-being of the
American public. Given the myriad escalating technological, economic,
and health risk challenges, this will not be an easy task for FDA. The
passage of FDAMA presents FDA with an opportunity to demonstrate
innovative and bold approaches in meeting these challenges for the 21st
century. This Plan is one positive step toward moving FDA into
conformance with the views of Congress and the Agency's stakeholders.
This document demonstrates that FDA already is making great
progress in managing health risks--a job that is becoming more complex
and often fraught with uncertainty and unpredictability. The Plan also
highlights the fact that the Agency clearly is unable to meet all of
the challenges it is expected to address with its curent level of
resources. Innovation and creative collaboration with external
stakeholders will certainly enhance the Agency's abilities to reduce
health risks in the long run; but additional resources are essential to
help FDA fulfill its statutory mandates.
[*Statutory requirements encompass all provisions of the Federal
Food Drug and Cosmetic Act (FD&C Act) and its amendments, including
FDAMA.]
Scope
The Plan specifically addresses the six objectives stipulated by
Congress in FDAMA Section 406(b):
Maximize the availability and clarity of information about
the process for review of applications and submissions.
Maximize the availability and clarity of information for
consumers and patients concerning new products.
Implement inspection and postmarket monitoring provisions
of this Act.
Ensure access to needed scientific and technical
expertise.
Establish mechanisms, by July 1, 1999, for meeting time
periods for the review of all applications and submissions.
Eliminate backlogs in the review of applications and
submissions by January 1, 2000.
To achieve these objectives, the Plan identifies Agency-wide
strategic directions for the next 5 years, and specific performance
goals for Fiscal Year (FY) 1999. Thus, the total plan presents a
picture of the Agency's long- and short-term future that will be
reviewed and modified as part of ongoing discussions with FDA's
stakeholders, with future Department of Health and Human Services
(DHHS) leadership and other parts of the Administration, and with
Congress.
The Mandated Strategic Framework
This Plan is one element of a total strategic framework mandated by
FDAMA that enables FDA to address increasingly complex public health
challenges. This framework, outlined in Section 903 of the Federal
Food, Drug, and Cosmetic Act as amended by FDAMA (see Appendix A),
contains the following key elements:
1. An augmented mission statement for FDA, which places new
emphasis on more resource-intensive consultation and cooperation with
stakeholders as a crucial ingredient in public health protection and
promotion [Sec. 903(b)(4)].
2. A charge to the Secretary of Health and Human Services to foster
collaboration among science-based agencies throughout the federal
government. Such coordination is necessary to strengthen the science
capabilities that underpin federal responsibilities to ensure a safe
food supply and related to development, evaluation, and monitoring of
new medical therapies [Sec. 903(c)].
3. Stipulation of general powers that are necessary for carrying
out Agency responsibilities, including research and education [Sec.
903(d)].
4. A requirement that FDA develop, after consulting with
stakeholders, a plan for bringing the Agency into compliance with each
of the obligations under the Act (The FD&C Act), and revise that plan
as appropriate with stakeholder input [Sec. 903(f)].
5. A provision for FDA to prepare and publish an annual report that
compares planned versus actual performance [Sec. 903(g)].
These elements reflect certain broad themes. First, the Agency
should devise and implement strategies in a more open, multi-
organizational environment. Congress emphasized throughout FDAMA that
consultation, collaboration, and synergy-building
[[Page 65003]]
with external organizations are paramount to FDA achieving its mission
of protecting and promoting public health. Simply put, FDA cannot do
the job alone.
Second, Section 903 provides FDA with a more systematic approach to
strategic management. The essential elements are clearly articulated: a
clear mission, consultation with stakeholders, a plan based on
stakeholder input to carry out the intent of the mission, and provision
for ongoing feedback, accountability, and adjustment to the plan. The
Agency recognizes the importance of this plan for action
accountability, as outlined in Section 406(b) of FDAMA, and in
establishing an ongoing dialogue with stakeholders to continually
improve strategies.
Third, Congress has recognized that an array of capabilities
including public education and research [Section 903(d)(2)] are
essential elements required to carry out its responsibilities under the
Act. The six objectives outlined in FDAMA 406(b) also explicitly
stipulate education and scientific expertise as being central to the
Agency's modernization plan. Successful public health promotion and
protection decisions depend upon a well-developed science
infrastructure and an informed public. Without these two elements,
desired health outcomes are not possible.
FDA's Strategic Management Approach
Figure 1 illustrates how FDA is integrating the mandates in Section
903 to form the components of an effective strategic management
process. As the figure illustrates, effective implementation of the
FDAMA plan depends upon several elements:
(1) development of a clear mission statement;
(2) assessment of challenges that FDA faces in fulfilling its mission;
(3) analysis of the gap between what is expected of FDA and its actual
performance;
(4) consulting FDA's stakeholders on future directions;
(5) a statement of Agency-wide objectives [406(b)] and strategic
directions to achieve the objectives;
(6) a specific plan for achieving each 406(b) objective, including
strategies and performance goals that can be used to manage toward the
objectives; and
(7) a budget that adequately funds the plan.
Part One of the Plan provides the broad Agency-wide context--steps
1 through 5 above. Part Two of the Plan maps the specific plan for
achieving objectives. In Part Two, the specific performance targets for
FY 1999 are established based on the Agency's existing resources,
reinventions, and collaborative arrangements. FY 2000 performance
targets currently are being developed as part of the FY 2000 Budget
process and are not included in the Plan. Many factors influence FDA's
choice of performance levels, including: extrapolations of past
performance, anticipated workload, creative re-engineering to improve
internal efficiencies, successful collaboration with FDA's outside
stakeholders, and strategic priorities.
BILLING CODE 6160-01-M
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BILLING CODE 4160-01-C
[[Page 65005]]
Mission Development
Over the years, Congress has dramatically expanded the
responsibilities of the FDA. The Federal Food and Drugs Act of 1906,
the first national statute enacted by Congress to regulate the American
food and drug supply, gave FDA's predecessor agency the authority to
remove adulterated or misbranded foods and drugs. In ensuing years,
Congress enacted a series of statutes that expanded FDA's
responsibilities in a number of directions, including: new product
areas (cosmetics, biologicals, and medical devices.); additional
product characteristics (e.g., efficacy as well as safety); and
additional perspectives from which to monitor products (e.g.,
monitoring prior to market introduction as well postmarket monitoring).
Beginning in 1996 with the passage of the Animal Drug Availability
Act (ADAA) and continuing in 1997 with the passage of FDAMA, Congress
enhanced FDA's mission in ways that recognized the Agency would be
operating in a 21st century characterized by increasing technological,
trade, and public health complexities. To meet these challenges,
Congress added explicit phrasing to the Agency's mission statement to
ensure that FDA would coordinate its own efforts with regulatory
counterparts worldwide. In addition, Congress recognized that external
scientists, medical experts, and public health experts must play an
increasing role in Agency responsibilities. It defined a new emphasis
to be placed on regulatory processes and required more interaction with
stakeholders. Through FDAMA, Congress intends to ensure timely
availability of safe and effective new products that benefit the
public, and to ensure that our nation continues to lead the world in
new product innovation and development.
DAMA defines FDA's new mission as follows:
The Administration shall--
(1) promote the public health by promptly and efficiently
reviewing clinical research and taking appropriate action on the
marketing of regulated products in a timely manner;
(2) with respect to such products, protect the public health by
ensuring that--
(A) foods are safe, wholesome, sanitary, and properly labeled;
(B) human and veterinary drugs are safe and effective;
(C) there is reasonable assurance of the safety and
effectiveness of devices intended for human use;
(D) cosmetics are safe and properly labeled; and
(E) public health and safety are protected from electronic
product radiation;
(3) participate through appropriate processes with
representatives of other countries to reduce the burden of
regulation, harmonize regulatory requirements, and achieve
appropriate reciprocal arrangements; and
(4) as determined to be appropriate by the Secretary, carry out
paragraphs (1) through (3) in consultation with experts in science,
medicine, and public health, and in cooperation with consumers,
users, manufacturers, importers, packers, distributors, and
retailers of regulated products.
Emerging FDA Challenges
FDA must address a wide range of challenges that serve as potential
obstacles to successfully carrying out its health protection mission in
the 21st century. To the extent that these challenges remain
unaddressed, a gap between expectation and performance will persist.
This Plan represents a blueprint for addressing these challenges,
thereby narrowing the gap.
Key challenges that FDA faces now and in the near future include:
1. Research and development-fueled pressures on regulatory
responsibilities;
2. Greater product complexity driven by breakthroughs in
technology;
3. Growth in recognized adverse effects associated with product
use;
4. Unpredictable, new health and safety threats;
5. More targeted needs and awareness of citizen-stakeholders;
6. Emerging regulatory challenges in the international arena;
7. Increased volume and diversity of imports; and
8. Federal budget constraints.
Each of these challenges is discussed briefly below.
Research and Development-fueled Pressures on Regulatory
Responsibilities
Each year, FDA-regulated firms add more than $2 billion to domestic
research and development efforts. For pharmaceuticals alone, this
effort currently exceeds $20 billion total, which is triple the effort
of only 10 years ago. The growth in research budgets at public agencies
such as NIH surely will result in a greater number and wider variety of
products that FDA must, by statute, regulate. More importantly, the
speed of product development also is accelerating. By streamlining the
commercial review process, FDA has helped to reduce the time between
discovery and Agency evaluation. But this streamlining also gives the
Agency very little time to develop a regulatory framework to handle new
technologies. Thus, it is imperative for FDA to continue to engage in
close interaction with industry in the early stages of product research
and development.
The volume, variety, and speed of new product development presents
FDA with the twofold goals of: (1) ensuring that consumers enjoy timely
public health benefits from these products; and (2) minimizing the
health risks associated with consumers' use of these products. FDA
resources devoted to premarket review of these products must be
carefully allocated so that both goals are addressed. The Agency's
current level of resources, however, cannot adequately address both
goals in all of the product areas for which the Agency has
responsibility.
Greater Product Complexity Driven by Breakthroughs in
Technology
Product complexity continues to increase. FDA-regulated products
will be characterized by unprecedented technological sophistication,
while also providing unparalleled health benefits for the U.S. public.
The continued benefits of genetic engineering warrant particular
attention. New products generated by the biotechnology revolution cover
a broad spectrum, including: genetic probes that serve as powerful
diagnostics; genetically engineered drug and gene therapies; and
biotechnology-based food modifications such as protein-enhanced
vegetables. Increased understanding of the human genome, as well as of
the genetic make-up of other organisms (genomes of other animals and
plants), will yield many new and different products and applications.
The number of sources that produce these new genetically engineered
products continues to escalate. The number of biotechnology firms grew
dramatically from the early 1980s through 1993, so that by 1993 there
were 1,272 firms, more than a threefold increase over the pre-1981
number. By April 1997, nearly 300 biotechnology drugs were in
development, tripling the number that were in development in 1989. FDA
must have access to the necessary scientific expertise to be able to
address the complexity of these new products, and to provide sound
regulatory decisions.
Microprocessor and miniaturization technologies are rapidly
expanding and enabling significant improvements in implantable medical
devices such as pacemakers, cochlear implants, and closed-loop medicine
delivery systems that monitor conditions within the body and administer
treatments as required. Progress in artificial intelligence has
increased companies' ability to apply pattern recognition techniques in
such
[[Page 65006]]
products as Pap smear readers and neural net classifiers.
New combination products, such as food-drug and drug-device
combinations, will continue to be generated through the application of
biotechnology techniques. Such developments foster improved versions of
products already developed and approved, as well as entirely new
products. New biological-based products will require the development of
new data profiles, because the data used to determine the safety of
chemical-based products of the past are neither sufficient nor
appropriate for predicting the safety of these new products.
Biotechnology also is being used to develop new assessment tools.
More emphasis is being placed on new approaches to assess the product
safety of food, dietary supplements, and health care products. These
tools include bioassays to improve safety assessments of carcinogencity
and to address emerging concerns of neurotoxicity, immunotoxicity, and
developmental toxicity.
Growth in Recognized Adverse Effects Associated With Product
Use
New technologies have provided an explosion of innovative
diagnostic and therapeutic health products. The consequences of this
explosion, however, include a parallel expansion of adverse effects
associated with product use. Although the benefits realized from these
products still greatly outweigh the problems associated with
consumption, these problems must be addressed. To illustrate, FDA
received more than one-quarter million reports of suspected drug-
related adverse effects in 1997, and this number of adverse reports
continues to increase annually. FDA estimates that nearly one million
patient injuries and deaths each year are associated with the improper
use of FDA-regulated products. Additional injuries and deaths occur
under conditions of proper use and accidental injury. For example, of
the more than 70,000 injury reports related to medical devices received
annually, approximately 25 to 40 percent of the injury or death reports
may be attributed to device misuse or operator error. Injury reports
received by FDA only represent between 1 and 10 percent of all injuries
associated with the use of medical devices. Using these figures, as
many as 400,000 incidents per year resulting in patient injury or death
may, at least in some way, be attributed to the user-device
interaction.
Currently, the FDA Center for Food Safety and Applied Nutrition
(CFSAN) receives reporting on food additives, cosmetics, and special
nutritionals from the field offices and other sources. To achieve
efficiency in monitoring and responding to adverse events, the Center
is proposing the establishment of an integrated adverse event reporting
system for food and cosmetic products. As the Agency develops more
comprehensive adverse event reporting systems, particularly in
collaboration with other institutions, the number of reported adverse
events likely will increase. If surveillance capability does not
expand, the magnitude and severity of product use problems will, to a
large extent, remain unknown, and the health risks will be unaddressed.
Unpredictable, New Health and Safety Threats
FDA continues to face a range of threats to public health that
appear in a random and discontinuous pattern. For example, crippling
infectious diseases such as tuberculosis are reemerging, bovine
spongiform encephalopathy (BSE) became epidemic in the United Kingdom
and was unexpectedly linked to the human disease, Creutzfeld-Jakob
disease (nvCJD), and more virulent and antibiotic-resistant bacteria
have been discovered in food products around the world. These
unpredictable threats, coupled with the growing incidence of disease-
causing organisms' resistance to existing drug therapies, challenge
both industry and FDA to bring innovative, safe, and effective
treatments to the market rapidly. The Agency also must address crises
that require emergency responses, whether they are the discovery of
pesticides in selected imported products, Escherichia coli outbreaks,
or intentional product tampering. These events are byproducts of
several factors, including continually expanding global trade; new
entrants into domestic industries--particularly where emerging
technologies are present; and economic pressures on regulated firms to
reduce costs in order to ensure short-term survival.
The unpredictable nature of a significant portion of FDA's
compliance activity also acts as a severe limitation to fulfilling
statutory mandates of inspectional coverage. FDA is attempting to
augment its inspection capability with strategies that call for
collaboration with states, use of third parties to verify industry
compliance, and augmenting industry quality control mechanisms. But
even these augmentation strategies require front-end investments to
develop systemic capabilities such as data validation, data sharing,
and auditing to determine whether protocols are in place. In addition,
some stakeholders oppose other third-party involvement. Consequently,
in the short run FDA--even in conjunction with collaborators--will not
be able simultaneously to satisfy statutory inspection requirements and
address all current health and safety threats.
More Targeted Needs and Awareness of U.S. Citizens-
stakeholders
A more knowledgeable and diverse consumer population is escalating
expectations for more information, as well as information that is more
tailored to their particular needs, concerning the safety of FDA-
regulated products. American consumers have become more health-
conscious during the 1990s and are seeking more information on the
impact of medical products and food on their health. FDA must
distinguish between the risks perceived by consumers and their actual
risks, and respond accordingly. Based on the additional information
that FDA provides, consumers are playing a larger role in protecting
their own health.
The elderly population provides a good illustration of why FDA must
target its information and regulatory policies to fit the needs of
particular market segments. Although the elderly are by no means the
only segment with special needs, their numbers have become much more
prominent in the general population. By the year 2000, Americans aged
75 and older will be the fastest growing group. The elderly (those over
65) have disproportionately high health care demands. Challenges
associated with this patient subpopulation, such as multiple drug
interactions, different physiological characterizations and reactions
to drug regimens, and the need for better medical device design for
home self-diagnostics and therapies, will become more acute. These
challenges will require greater inclusion of the elderly in clinical
testing for drugs, medical devices, and other FDA-regulated products.
Further, the increasing educational needs of the elderly will require
more focused education programs, including specific dietary information
and foods targeted to their nutritional requirements. The elderly
population and food service workers who prepare food for the elderly
also will require special education initiatives concerning proper food
handling, because as the population ages it becomes more susceptible to
foodborne diseases.
[[Page 65007]]
Emerging Regulatory Challenges in the International Arena
FDA participates in the world community of developed,
underdeveloped, and developing economies and regulatory authorities.
Radical changes in the dynamics of the world structure are underway,
driven by several forces: (1) an increasing number of global and
multinational firms that produce FDA-regulated products; (2) increasing
sophistication of unified economic, political, and regional entities
(e.g., the European Union [EU] and Pacific Rim countries); and (3) the
response to these conditions on the part of regulatory/standard-setting
entities.
The larger drug, biological, device and food firms now operate as
multinational companies. New products will be developed, produced, and
marketed through a highly networked and global commercial system. The
system will have great power to satisfy consumer needs, but will be
much more complex to monitor for potential risk than has been the case
in the past. This situation will require sophisticated international
regulatory responses. Further, the regulatory response by U.S.
interests must preserve the delicate balance at the international level
between preventing unnecessarily high-risk products from entry into the
country, while providing access to novel, important therapies or foods
to the American public.
The multinational and global firms are sharing center stage with an
increasingly organized set of regional economic and political entities
such as the EU, Pacific Rim organizations, North America Free Trade Act
(NAFTA) participants, etc. These entities are amassing the economic and
political power to attract world trade. The pace of their development
is often uneven, but the longer term direction is clear. Raw materials
and joint ventures that stretch across national borders are all
becoming international elements for FDA to regulate where previously
these were purely domestic phenomena. The Agency must now make new
decisions on how (or if) to manage each of these new elements.
Increasingly, FDA must take into account the global trade implications
of its decisions.
Organizations such as the International Committee on Harmonization
(ICH), the International Standards Organization (ISO), the Global
Harmonization Task Force, the International Cooperation on
Harmonization of Technical Requirements of Registration for Veterinary
Medicinal Products (VICH), and Codex are becoming increasingly
important in the determination of the level of acceptable product
safety, quality, and efficacy for products trading in the international
arena. FDA must maintain a viable voice as standards are prepared and
speak with a voice that represents the interests of all of its
stakeholders, whether they are consumers, patients, health
practitioners, or the regulated industry.
Increased Volume and Diversity of Imports
Imported products regulated by FDA represent a significant
component of total U.S. consumption. In some sectors, such as seafood,
the percentage of total consumption represented by imports is
approximately 50 percent. FDA's responsibilities in the import arena
continue to expand, without a corresponding increase in resources to do
the job. To illustrate: The volume of imports has grown steadily over
the past few decades. By 1998 an estimated 4 million FDA-regulated
import line items arrived in the U.S. The number of food items,
representing the majority of those imports, increased by 21 percent
over the last year alone! During that same period, FDA resources to
address imports remained essentially level.
And the complexity is increasing--the reality of a truly global
economy is adding significant regulatory challenges for FDA. These
products are originating in countries that often have less developed
health/safety regulatory structures. The increase in volume, variety,
and sources of imports may be accompanied by increases in novel
pathogens, microbial contamination, and other public health concerns
and regulatory challenges for FDA. Developing countries, which once
provided raw materials for U.S. manufacturers, and assemblers are
increasingly providing finished products to the U.S. market. This
conversion could increase the risks associated with such products.
Federal Budget Constraints
Recent budget proposals and appropriations acts have addressed
emerging public health issues (such as AIDS) and long-standing public
health problems that received insufficient attention in the past
(including reducing youth tobacco use, improving food safety, and
accelerating prescription drug approvals). While those problems
continue to need attention, inflation has reduced real resources
available for FDA's other public health responsibilities, which are
necessary to meet the obligations delineated in FDAMA. These include
inspections to ensure product safety; review of devices, food
additives, blood products, animal drugs, and generic drugs; and adverse
event reporting and followup.
Analysis of the Gap Between What is Expected of FDA and Its Actual
Performance
FDA faces a critical issue today. Because of a convergence of
challenges outlined in previous sections, the Agency has been unable to
fully meet its explicit statutory obligations; nor has it been able to
completely guarantee the more implicit health and safety
responsibilities the statute requires and the public demands. Figure 2
illustrates that a sizable gap still exists between statutory
requirements of ``on-time review'' for several product areas, and what
FDA currently is able to deliver. Figure 3 shows a similar gap between
mandated and actual inspectional coverage for FDA-regulated industries.
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The agency has listened carefully to its stakeholders over the past
several months and has combined their views with its own emerging
strategies to develop a plan for narrowing the gap. The following
section provides a summary of stakeholder views.
Stakeholder Consultation
FDA's assessment of the challenges it faces in fulfilling its
mission and the identification of the disparity between expectations
and what is achievable given the current climate set the stage for
consultations with its external stakeholders. This consultation is
necessary to determine the most effective ways of narrowing the gap.
FDA depends on the views of its stakeholders for two crucial reasons:
(1) stakeholders are affected by the outcomes of FDA's strategies and
should therefore play a role in formulating them; and
(2) stakeholders are also the collaborators that are necessary for
successful implementation of the Plan.
In the sections that follow, the process of stakeholder
consultation is discussed, and a summary of their views is provided.
The Process
Section 406(b) of FDAMA prescribes that the plan for statutory
compliance be developed:
after consultation with appropriate scientific and academic experts,
health care professionals, representatives of patient and advocacy
groups, and the regulated industry.
The experts and representatives referenced in Section 406(b)
comprise the constituency of the FDA. The Agency informally consults
with these constituents on a regular basis. Section 406(b) codifies
this process and provides a mechanism for formal input from and
feedback to its constituency.
In response to this requirement, the Agency designed a process that
provided multiple avenues for input, including the following:
Public meetings were held and tailored to address concerns
associated with each of FDA's product centers: foods, human drugs,
animal drugs, biologics, and medical devices. In addition, there was a
meeting focusing on health professionals and an Agency-wide meeting
addressing cross-cutting issues.
Dockets were provided for stakeholders to make additional
comments subsequent to the public meetings. These dockets will remain
open indefinitely.
Electronic communication vehicles were established that
allow stakeholders to communicate with FDA via Internet responses to
the Agency's home page as well as through e-mail.
District Consumer Forums were held to solicit comments
from stakeholders.
On going communication vehicles were used to actively
solicit stakeholder views on current and future directions for the
Agency. These vehicles include speeches made by the Agency's senior
leadership, ongoing exchanges in smaller forums such as workshops, and
one-on-one conversations.
FDA adopted a uniform approach in framing the stakeholder
discussions and comments. Agency officials first outlined the
stakeholder consultation process. The leadership then provided a
framework outlining the emerging technological and public health
challenges faced by FDA. Finally, to focus stakeholder comments and
discussion, questions (Appendix B) were developed that related to each
of the six objectives addressed by the 406(b) plan and were available
to stakeholders prior to the meetings.
The process of engaging the Agency's stakeholders and receiving
useful feedback is an ongoing one. This initial round of stakeholder
views will continue to be analyzed and interpreted during Fall 1998.
Results of the analysis will be shared with FDA's external as well as
internal audiences. The next round of formal stakeholder meetings is
being scheduled for Spring 1999, and regular contacts will continue to
be maintained. Although longer term assessment is forthcoming, a
preliminary evaluation of stakeholder views has been conducted. An
overview of these views is provided in the next section. Stakeholder
comments are assessed in greater detail in Part Two of the Plan and are
related to Agency strategies.
Summary of Stakeholder Viewpoints
FDA's stakeholders commented on many aspects of the Agency's
operations. The recommendations made by stakeholders regarding the
Agency's priorities and the strategies FDA should use in carrying out
its responsibilities reflect a wide range of concerns and perspectives.
The full context of stakeholder views expressed at public meetings and
in written comments are captured in transcripts and dockets that are
available on FDA's Internet Web page http://www.fda.gov/oc/fdama/comm.
Appendix B-4 also provides a compendium of stakeholder recommendations,
classified both by 406(b) objectives and by the strategic directions
that are identified in the next section of the Plan. Major themes that
emerged from the stakeholder comments are summarized below.
Areas of Consensus
Most stakeholders agree on several broad issues. Many agreed that
FDA priorities should be risk-based, scientifically rational, and
focused on protecting public health. In addition, the Agency should
view meeting its statutory obligations as a high priority. A number of
organizations cautioned that the Agency should limit its participation
in new activities, especially those that go beyond the scope of its
core statutory requirements. Although stakeholders varied in their
interpretations of core responsibilities, some stakeholders highlighted
the importance of preserving FDA's regulatory role and encouraged the
Agency to develop more creative strategies to exercise its regulatory
responsibilities. Many stakeholders acknowledged the difficulties
inherent in making trade-offs among program activities when resources
are constrained.
Making new safe and effective treatments available to patients in a
timely manner is also a high priority for FDA. To optimize the
performance of the premarket review and approval system, stakeholders
recommended that FDA continue to re-engineer its systems and strive for
internal efficiencies; communicate earlier in the premarket review
process, more frequently, and more openly with industry and other
stakeholders; and make FDA policies and procedures more consistent and
more transparent to industry and the public. Several groups would like
FDA to adopt a more uniform and consistent approach to addressing risks
of public health significance. Consistency of FDA policies and
procedures seemed to be a greater concern than their transparency.
Requests for improved communication emphasized two-way
communication--not only from the FDA to its stakeholders but also from
stakeholders to FDA beyond adverse event reporting. Stakeholders value
FDA developing a strong scientific and analytic base for its regulatory
decisions. They believe that FDA should use the expertise of other
organizations to help meet its goals. For example, delegating or
collaborating on certain functions (such as research, standard-setting,
and some aspects of product review) to third parties were offered as a
means of leveraging limited resources.
Several stakeholder groups want to be more involved in FDA advisory
committees. These views are consistent with FDA's transition to a more
open and collaborative relationship with its
[[Page 65010]]
regulatory counterparts and industry. Continued FDA leadership and
participation in the international arena was encouraged to ensure that
international standards and guidelines are consistent with U.S.
requirements. Even through it was recognized that FDA had limited
resources to meet all of its statutory obligations and to meet public
expectations, industry representatives opposed the collection of user
fees for medical devices and the blood banking industry, as well as for
veterinary products, as a means of funding premarket review activities.
Similarly the concept of an ``FDA seal'', viewed as a form of user
fees, was not supported.
Areas of divergence
Although the first order of concern of all stakeholders is consumer
health protection and availability of medical products, there is no
consensus on the role FDA should play nor what approach should be taken
in this daunting task. Key differences among stakeholders include the
following:
FDA's Role in Education
Stakeholders differed sharply in their opinions on the legitimacy
and primacy of FDA's role in consumer education. While some stakeholder
groups believe that industry and health professionals should be
responsible for consumer education, others assert that FDA should play
an essential role in providing objective information about regulated
products to consumers and in facilitating patient participation in
ongoing clinical trials of promising new therapies. One consumer
advocacy group, the National Council on Patient Information and
Education, requested FDA's support in developing a collaborative,
national consumer
FDA's Enforcement Activities
Some stakeholders called for expanded FDA authority and additional
resource appropriations to allow the Agency to carry out its
responsibilities, for example, in the areas of drug safety monitoring
and monitoring the sale of unapproved veterinary products. Other
stakeholders acknowledged that FDA would need to share enforcement
responsibilities with others. For example, one group supported a
division of tasks in the inspection arena, with FDA covering the
imports, and states being responsible for domestic inspections.
Use of Third Parties
There were mixed views in this area as well. Many consumers
preferred that FDA regulate the industry more directly, while several
industry representatives advocated for greater use of third parties, as
long as the arrangement was carefully monitored by the Agency.
Advisory Committees
Views regarding the composition of FDA advisory committees diverged
greatly. Some pressed for broader presentation of interested persons
while others advocated that FDA place greater emphasis on the depth of
knowledge of advisory committee members. The Agency was urged to
recruit renowned experts to serve on advisory committees. Some advisory
committees were criticized for favoring nonscientific issues over
sciences when they make recommendations.
Unresolved Issues
Perhaps the issue that remains most problematic is the overall
question of balance among FDA's functions. The appropriate mix of
premarket review, post-market inspection, and surveillance activity is
an ongoing topic of debate among the Agency's stakeholders. One
stakeholder summed up the issue:
``How should FDA balance the need for strong and timely
premarket review programs with the need for effective postmarket
inspection, surveillance, and enforcement programs? That is like
asking the American people to find a balance between building safe
aircraft and providing adequate maintenance over the course of a
plane's life.'' (Patient Group)
Although stakeholders expressed their views regarding the emphasis
FDA should place on various issues, these comments frequently focused
on a single FDA Center or two Competing issues. FDA does not have
sufficient information at this time about the priority Agency
stakeholders wish to assign to a particular issue relative to other
issues competing for resources within an FDA Center or within the
Agency as a whole. In some instances the proposed strategies appear to
be contradictory. For example, how should the Agency balance setting
risk-based priorities or meeting public expectations when doing so
directly competes with meeting its statutory obligations?
Identification of Agency-Wide Objectives and Strategic Directions
The six objectives specified in FDAMA Section 406(b) and outlined
on page 1 of this Plan, provide FDA with a broad framework for meeting
its statutory requirements and public expectations. The Agency's senior
leadership believes the following strategic directions are necessary to
focus its efforts in achieving the objectives set forth by Congress.
These directions represent an amalgam of approaches that have been
emerging for several years, and which have been modified both by new
FDA challenges and by the productive suggestions made by external
stakeholders. Figure 4 identifies the link between key stakeholder
themes and the strategic directions outlined in this section of the
plan.
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[[Page 65012]]
The strategic directions are broad in scope and cross-cut
components of the organization. As such, they provide a context to
guide all of the Agency's more specific goals and programs. They also
serve as a way to galvanize diverse activities into a set of unified
directions for the long-term.
(1) Establish risk-Based Priorities
Although the importance of setting risk-based priorities was a
concept repeatedly endorsed by many stakeholder groups, there was not
consensus regarding what constituted the highest risk areas. FDA must
listen to its stakeholder community, but then it must decide, based on
continuing consultation with its stakeholders, which health and safety
risks most directly threaten the well-being of U.S. consumers, and
allocate its resources accordingly. In the harsh light of limited
resources, FDA simply cannot meet everyone's demands and cannot address
all risks with the same degree of urgency or intensity. For example,
the Agency is unable to respond to its highest priority health risks
and at the same time fully meet its biennial statutory inspection
requirements for drugs, biologics, and medical devices. it may be
appropriate to reassess the practicality of mandates that emphasize
industry coverage, regardless of risk, when those mandates may divert
limited resources away from addressing serious health and safety
concerns. The Agency has and will continue to increase the efficiency
of ``fast track'' processes to address the most urgent needs for
therapies so that these therapies can enter the marketplace rapidly.
Resources will continue to be redirected toward the review of these
products. Surveillance and compliance efforts also will continue to be
directed toward identifying and taking action to correct the most
serious health and safety problems associated with products that are in
the marketplace or about to enter the market. The Presidential Food
Safety Initiative will continue to focus attention and devote resources
to those areas of the food supply that pose the greatest risk of
illness and/or death to consumers.
(2) Strengthen the Scientific and Analytical Basis for Regulatory
Decisions
A strong science base continues to underpin each of the Agency's
regulatory decisions. Such decisions must be made throughout the
lifespan of FDA-regulated products from initial research, development
and testing, through production, marketing and consumption. A strong
science base consists of the expertise, the risk assessment protocols,
the test methods, product guidance and performance standards, and the
facilities and equipment necessary for conducting excellent science.
The emerging emphasis in this strategic area is to seek means for
achieving synergies in science capability through access to and
collaborative efforts with sources of scientific expertise beyond FDA.
A recent example that the Agency hopes will achieve research synergies
through collaboration is the pharmaceutical quality and drug
development science initiative that the Agency has begun to pursue
under a cooperative research agreement among FDA, professional
societies, and industry. The initiative will provide a venue to conduct
research on pressing questions about pharmaceutical manufacturing that
can inform regulatory decisions regarding needs in such areas as
supplement submission requirements or bioequivalence studies after
there are manufacturing changes. Such collaborative efforts are
reinforced in the objectives identified in FDAMA Section 406(b). The
key lies in ``ensuring access to the expertise,'' wherever it is most
cost-effective.
(3) Work More Closely With External Stakeholders
FDA will need to multiply the Agency's capability to address
complex public health problems by working with stakeholders in
planning, implementing, and evaluating solutions to these problems. The
solutions don't lie solely in expanding the mass of the Agency.
Consumers, the regulated industry, health professionals, and FDA's
regulatory counterparts in the U.S. and abroad each represent
components of a total network that can potentially improve health
outcomes. To help ``activate'' that network, FDA is engaged in several
strategies some just emerging and others in a more mature phase. These
`'activation strategies'' include: collaboration with stakeholders to
create synergies in protecting the public health; ensuring that
stakeholders are well informed about the Agency's regulatory processes
[the processes should be as transparent as possible] and the products
that are affected by these processes; involving stakeholders early in
the Agency's processes; and ensuring that all affected stakeholder
groups' interests are well represented in product testing and approval
decisions.
FDA is striving to create synergies through collaboration with
appropriate outside colleagues in product research and testing,
development, production, marketing, and consumption/use to ensure
safety, quality, and efficacy.The Agency's Joint Institute for Food
Safety and Applied Nutrition [JIFSAN] (with the University of Maryland)
and the Moffett Center in Illinois are illustrative of such synergies
working at the level of applied research and development to ensure safe
foods.
Industry representatives and health professionals made it clear to
FDA during the stakeholder consultation process that they can be more
effective colleagues in improving health outcomes in their role as
product developers and users if they are (1) well informed about the
Agency's regulatory review, surveillance, and compliance processes; and
(2) consulted prior to regulatory decisions on both the pre- and post-
market side of product commercialization. FDA will continue
implementing strategies to engage in preventive problem solving, as
well as initiatives that will make the Agency's processes as clear and
understandable as possible to participants.
Consumers and patients expressed a need to have prompt, complete,
understandable, and unbiased information about products that FDA
regulates, particularly new therapies. Well-informed consumers are more
effective contributors to the management of their own health risks. FDA
has launched several initiatives that are intended to keep the consumer
well-informed through such vehicles as publishing the availability of
important new drugs on the Internet. FDA is also attempting to ensure
that the interests of all affected patients are well represented in
such areas as clinical trial designs for new therapies. In addition,
FDA will ensure that the interests of the consumer are represented in
such deliberative bodies as advisory committees when recommendations on
new products are being considered.
(4) Re-Engineer FDA Processes
FDA has used both an internal and an external focus in redesigning
many of its regulatory review processes. From the external perspective,
FDA is implementing several protocols that will result in simplified
regulatory approaches and, as a result, a reduced burden for the
regulated industry. Many of these regulatory reinventions are embodied
in provisions in FDAMA. For example, the Agency may start review of a
``fast-track'' drug application before the application is complete if
preliminary clinical data demonstrate that the product may be
effective. Fast-track status also is being established for humanitarian
medical devices, and new product development protocols will allow
medical device sponsors to use
[[Page 65013]]
recognized study results that have been generated by other sources as
part of their own application submission. Other regulatory
simplification strategies have been instituted independent of FDAMA.
For example, a phased review process for animal drugs has been designed
that enables the Agency to provide periodic feedback to product
sponsors throughout the drug review process to foster ``continuous
improvement'' in the application.
FDA is also focusing internally to achieve greater efficiencies and
effectiveness in its review and tracking processes. For example,
implementation of project management techniques allows an opportunity
for convergent thinking and action to occur so that multiple
disciplines can coordinate their efforts in providing thorough but
timely reviews of product sponsors' applications.
(5) Adopt a Systems Rather Than a Piecemeal Approach to Agency
Regulation
Several stakeholders during the public meetings noted that they
could be more efficient and effective participants in promoting and
protecting public health if they could understand the total context of
what the Agency was trying to do and what its future directions were.
The establishment of a systems approach within FDA is closely related
to the establishment of risk-based priorities. Use of a systems
orientation is an effective way to identify what is truly high-priority
risk and then to address that risk in a systemic manner. Systems
solutions, such as the Food Safety Initiative, the integrated adverse
event reporting initiative, and the important monitoring system, are
examples of FDA acting in concert with other collaborators to address
the highest priority, most pervasive risks facing consumers.
The Agency also has adopted a systems orientation in many of its
individual programs. To illustrate, medical device inspectors have
embarked on a new approach to determine industry compliance with Good
Manufacturing Practices (GMPs). They are pilot-testing a systems-
oriented inspectional strategy whereby medical device plants are given
guidance on the establishment of a total Device Quality System, so that
the control of product safety and quality is owned by the firm, rather
than their having to respond to a series of external compliance
requirements that must be responded to one at a time. The seafood
Hazard Analysis and Critical Control Points (HACCP) initiative provides
another example where FDA worked with the seafood industry to implement
a systems approach to ensure the safety of seafood consumed by the
American public.
(6) Capitalize on Information Technology
FDA has been on a long course of improvement in taking advantage of
the opportunities offered by a rapidly evolving information technology
environment. Information technology has been used for quite some time
by the Agency in order to improve internal efficiencies. For example, a
key element in accelerating the review of new drug therapies has been
automating major portions of the drug review process. When both product
sponsor and Agency reviewer can use electronic communication to
establish a common ground of understanding, then all parties benefit.
It is a critical element that has become pervasive in all mission-
oriented as well as support activities.
More recently, the Agency has turned its attention to using
information technology as a way of improving communication with
external stakeholders. One of the most powerful examples of how
stakeholders are assisted is in the rapid provision of information on
new drug therapies via the Internet to consumers and patients. FDA's
home page provides an opportunity for all of FDA stakeholders to be
aware of recent Agency regulatory decisions, and, just as important, to
receive input in the form of suggestions and other opinions from Agency
officials. The Agency will expand use of information technology to
bring relevant information to bear in the area of produce surveillance
and adverse event reporting. Well-designed and integrated information
systems will dramatically reduce the gap between adverse effects
associated with consumption and problem correction.
Making the Transition From Strategic Context to Targeted Planning
The strategic directions outlined above provide the context for
understanding Part Two of the 406(b) Plan. In Part Two, specific
performance targets and associated strategies re outlined for FY 1999.
Part Two is organized into sections that correspond to the six
objectives outlined in Section 406(b) of FDAMA (Section 903(f) of the
FD&C Act as amended). Thus, specific performance targets can be
directly related to achieving the objectives of the Act.
Within each objective, strategies for FY 1999 relfect the Agency-
wide strategic directions identified in Part One. Thus, the Agency's
targeted planning for FY 1999 is strategically aligned with its
intended directions over the next several years.
Part Two--FDAMA Plan For FY 1999
This Plan outlines key performance goals and strategies designed to
achieve these goals during FY 1999. The Plan serves several purposes:
(1) It provides a blueprint for narrowing the gap between what FDA
is expected to do by law and by the stakeholder community and what FDA
currently can accomplish given its existing Agency resources.
(2) It responds to Section 406(b) of FDAMA, which requires the
Agency to develop such a plan:
``The Secretary, after consultation with appropriate scientific
and academic experts, health care professionals, representatives of
patient and consumer advocacy groups, and the regulated industry,
shall develop and publish in the Federal Register a plan bringing
the Secretary into compliance with each of the obligations of the
Secretary under this Act.''
(3) It moves FDA closer to fulfilling its strategic goals, and
thus, its mission of consumer health protection and promotion.
(4) Finally, the Plan provides a specific set of performance
commitments that will serve as a basis for managing towards results and
for reporting progress.
The Plan is organized according to the six objectives outlined in
Section 406(b) of FDAMA.
These objectives address critical components of FDA's
responsibilities. The Agency, working in collaboration with key players
in both the public and private sector, will pursue each objective as
part of a total consumer health protection and enhancement system. The
process begins with the research and development of new products with
great health- and life-sustaining potential, and ends with the safe and
effective consumption of these products. Figure 5 illustrates how FDAMA
objectives are crucial elements of FDAs total contribution to
beneficial public health outcomes.
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Part Two--FDAMA Plan For FY 1999
This plan outlines key performance goals and strategies designed to
achieve these goals during FY 1999. The Plan serves several purposes:
(1) It provides a blueprint for narrowing the gap between what FDA
is expected to do by law and by the stakeholder community and what FDA
currently can accomplish given its existing Agency resources.
(2) It responds to Section 406(b) of FDAMA, which requires the
Agency to develop such a plan:
``The Secretary, after consultation with appropriate scientific
and academic experts, health care professionals, representatives of
patient and consumer advocacy groups, and the regulated industry,
shall develop and publish in the Federal Register a plan brining the
Secretary into compliance with each of the obligations of the
Secretary under this Act.''
(3) It moves FDA closer to fulfilling its strategic goals and thus,
its mission of consumer health protection and promotion.
(4) Finally, the Plan provides a specific set of performance
commitments that will serve as a basis for managing towards results and
for reporting progress.
The Plan is organized according to the six objectives outlined in
Section 406(b) of FDAMA.
These objectives address critical components of FDAs
responsibilities. The Agency, working in collaboration with key players
in both the public and private sector, will pursue each objective as
part of a total consumer health protection and enhancement system. The
process begins with the research and development of new products with
great health- andlife-sustaining potential, and ends with the safe and
effective consumption of these products. Figure 5 illustrates how FDAMA
objectives re crucial elements of FDA's total contribution to
beneficial public health outcomes.
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The six 406(b) objectives are addressed in five sections below. The
five sections examine the FDAMA objectives in order by objective (A, B,
C, D, and E&F). Each section provides:
Identification of Needs--Outlines the unmet demands stated
by law and expressed by the Agency's stakeholders, which FDA must
address to achieve the FDAMA objective and to fulfill its mission.
Stakeholder Views--Selected Stakeholder opinions on the
importance of the need being addressed.
Current Innovations and Reinventions--Creative
improvements FDA has underway that will help achieve objectives.
Plan for Meeting Statutory Requirements and Public
Expectations--Key strategies that are planned for the future that will
narrow the gap between expectations and current capabilities.
Performance Goals for FY 1999--FY 1999 goals are based on
final Congressional appropriations and may be subject to adjustment
pending Agency resource allocation decisions.
Objective A--Maximizing the Availability and Clarity of Information
About the Process for Review of Applications and Submissions
(Including Petitions, Notifications, and any Other Similar Forms of
Requests) Made Under This Act
1. Identification of Needs
FDA's ability to provide clear, adequate, and timely information on
its application review processes must be improved by making FDA
processes transparent to stakeholders and involving stakeholders early
in the review process.
Make FDA Processes Transparent
While the Agency has developed written information (i.e.,
regulations, guidance documents, or internal procedures) on its review
processes and requirements, more needs to be done to ensure that
stakeholders understand FDA requirements. This lack of understanding is
reflected in the quality of regulatory submissions received by FDA.
Transparent processes also include openness on how FDA develops its
requirements and how those requirements are applied within the agency
during the review process.
Collaborate with Stakeholders Early in the Regulatory Decisionmaking
Processes
In passing FDAMA, the Congress expected major improvements on how
products are reviewed and approved by FDA. To meet this expectation,
FDA must change how it responds to the product applicants during the
review process--from being reactive to proactive through early
applicant consultations. By consultation with product sponsors, the
Agency will be able to help define the critical issues that must be
addressed in a product application, to define the types of clinical
trials that appear necessary, and to avoid unnecessary effort. This
shifting of resources is not, however, without cost, and additional
resources will be needed to meet the increasing number of product
submissions generated by the doubling of biomedical research funding at
the National Institutes of Health and by the regulated industry.
2. Stakeholder Views
Stakeholders endorsed the concept of a more open and collaborative
relationship between FDA and its regulatory colleagues and industry.
Many stakeholders commended FDA for the efforts the Agency has already
made to address this objective. Requests for improved communication
about application review processes emphasized not only communication
from FDA to industry, but also greater stakeholder participation in
regulatory decisionmaking. The examples below illustrate some of the
further improvements stakeholders requested:
Make FDA policies and procedures more transparent,
particularly those related to Good Review Practices [trade
association].
Provide requested clear, concise, and up-to-date guidance
to product sponsors. Where the existing guidance is deemed inadequate
or scientifically outdated, FDA should issue guidance about the
specific product applications [trade association].
Work closely with product sponsors to ensure submissions
are properly formatted [trade association].
Provide a sample submission guide to applicants and make
available more templates, prototypes, and examples of submissions to
clarify FDA's expectations of the regulated industry and to expedite
the review process [trade association].
Provide as much feedback to industry as possible in the
earliest time frame because many of the questions that are generated
will result in long-term experiments or clinical trials [industry
representative].
Industry input in developing guidance documents, such as
the one on inclusion of women in clinical trials, and regulations is
key in maintaining the integrity of the clinical trials process and of
the application review process [consumer advocacy group].
Collaborate and interact more with the regulated
industries to avoid issuing guidance documents that do not adequately
take into account useful perspectives that can be provided by industry
to the FDA [trade association].
Use the formal binding presubmission consultations to
reduce backlogs and to speed the approval process. [trade association].
``Expedite the approval of appropriate nutrient content
claim and health claim petitions and citizen petitions related to food
labeling.'' [trade association].
3. Current Innovations/Reinventions
FDA is improving its review processes and specific product
applications through collaborative agreements, process re-engineering,
and information technology.
Agreements Among FDA, Industry, and Others Enhance Review Processes
FDA, academia, and industry are working to establish a program to
provide research to inform and assist FDA in developing regulations and
guidance regarding the types of product quality information that should
be submitted in a product application (e.g., Collaboration for Drug
Development Improvement and Product Quality Research Initiative).
FDA collaborates with regulatory authorities of Europe and Japan on
drug development requirements (e.g., International Harmonization).
FDA Continues to Improve Review Processes Through Process Re-
engineering
FDA's medical device program improved by providing manufacturers
with regulatory options to reduce regulatory burden for lower risk
products and by improving communication with manufacturers. As part of
the Reinventing Government Initiative (REGO), FDA has simplified the
filing process by consolidating review application forms for
biotechnology-based drugs, blood, vaccines, and other drugs into just
one form. This enables companies to provide higher quality submissions
to the FDA and reduces their application preparation time.
During FY 1997 and early FY 1998, the Foods Program conducted under
contract a review of deficiencies in over 600 industry-submitted food
and color additive petitions. CFSAN currently is reviewing the
contractor's report and expects to use the information to improve
guidance to petitions and to
[[Page 65017]]
implement a stronger refusal to file policy.
FDA Uses Information Technology To Improve Access of Review Processes
The FDA website (www.fda.gov) provides specific information to
particular stakeholder groups: consumer, industry, state and local
officials, patients, health professionals, women, and children.
FDA has published information on its review processes to assist
applicants. For example, the FDA Center for Drug Evaluation and
Research (CDER) Handbook is available on the Internet.
The Foods Program is completing testing on a document management
and workflow system that will replace the current tracking system for
petition reviews and will make petition data available on demand in
electronic format on reviewer's and administrator's desktops. The new
workflow tracking system will permit realtime access to detailed
information on petition status and tasks.
4. Plan for Meeting Statutory Requirements and Public Expectations
Section 903 of the FD&C Act, as amended by FDAMA, authorizes the
Commissioner to conduct educational and public information programs
relating to the responsibilities of FDA. Under FDAMA (Section 406),
FDA's mission is expanded to include the prompt review of clinical
research and regulatory submissions, harmonization of regulatory
requirements with other countries, and consultation of various experts
in fulfilling the mission.
FDA's plan for meeting these statutory requirements will encompass
a variety of actions intended to make Agency processes transparent and
to improve collaboration between product sponsors and the agency. These
include:
Continuation of developing appropriate regulations,
guidance documents, and internal operating policies and procedures.
Expansion of the use of communication media and
information technology (e.g., the FDA website) to provide written
materials and information on FDA regulatory review processes.
Improvement of the efficiency and effectiveness of Agency
review processes through process re-engineering, project management,
performance management, and electronic technology.
Development of innovative approaches to facilitate sponsor
and Agency consultations.
5. Performance Goals for FY 1999
The table provided in this section links the performance goals and
measures with statutory requirements addressing information about the
review processes. Under the FD&C Act, the Commissioner is authorized to
conduct educational and public information programs relating to FDA's
responsibilities. These performance goals illustrate two types of
efforts. The first type identifies the development of a method that can
be applied to a review process. An example would be to recognize a
standard used for a medical device review. The second type identifies
an improvement to enhance the Agency's ability to provide updated
information or to achieve greater capability and capacity for accepting
electronic regulatory submissions.
Highlighted below are key performance goals for FY 1999 in the area
of electronic regulatory submissions. These goals are critical to the
Agency's ability to provide timely review of clinical research and
regulatory submissions, which is the intent of FDAMA. For more complete
identification of performance goals and statutory requirements see the
table at the end of this section.
FY 1999 Performance Goals
Complete the development of industry guidance required for
electronic submission by the end of FY 2002.
Achieve electronic submission capability for certificates to foreign
governments.
Achieve capability and capacity for electronic submission and
archiving of information required to submit New Drug Applications
(NDAs) without paper copy by the end of FY 2002.
Achieve capability and capacity for electronic submission and
archiving of Abbreviated New Drug Applications (ANDAs) by the end of
FY 2002.
----------------------------------------------------------------------------------------------------------------
Relevant statute and/or Relevant FY 1999 FY 1997 performance
Statutory authority regulation performance goals baseline
----------------------------------------------------------------------------------------------------------------
Applicants are invited to meet with FD&C Act, Section 505 By the end of FY 2002, In FY 1997, electronic
FDA before submitting an application and 21 Code of Federal CDER will complete signature guidance was
to discuss the presentation and Regulations (CFR) development of published.
format of supporting information. If 314.50(f)(4). industry guidance
the applicant and FDA agree, the required for
applicant may submit tabulations of electronic submission.
patient data and case report forms
in a form other than hard copy, for
example, on microfiche or computer
tapes.
Before 30 days after the date of FD&C Act, Section By the end of FY 1999, In FY 1998, develop and
submission of an application to 801(e) and 802, 21 CFR CDER will achieve pilot Export
export a drug, the FDA must review 210, Drug Export electronic submission Certificate Program.
the application to determine if it Amendments Act of 1986 capability for
meets all applicable requirements. (PL. 99-660), FDA certificates to
Export Reform & foreign governments.
Enhancement Act of
1996.
For records submitted to the Agency, FD&C Act, Sections 201- By the end of FY 2002, By FY 1997, establish
persons may use electronic records 903; PHS Act Section CDER will achieve the structure of the
in lieu of paper records or 3512, 21 CFR 11. capability and Electronic Document
electronic signatures in lieu of capacity for Room (EDR).
traditional signatures, in whole or electronic submission
in part, provided that certain and archiving of
requirements are met. information required
to submit NDAs without
paper copy.
By the end of FY 2002, By FY 1997, establish
CDER will achieve the structure of EDR.
capability and
capacity for
electronic submission
and archiving of ANDAs.
[[Page 65018]]
Any record of the FDA that is FD&C Act, Sections 201- By the end of FY 2002, By FY 1998, the
disclosed in an authorized manner to 903, 5 United States CDER will make Electronic Document
any member of the public is Code 552, 21 CFR 20. publicly releasable Room, as required by
available for disclosure to all information available the Electronic Freedom
members of the public, except that via Internet. of Information Act,
data and information subject to the will be initiated.
exemptions established in 21 CFR
20.61 for trade secrets and
confidential commercial or financial
information, and in Section 20.63
for person privacy, shall be
disclosed only to the persons for
the protection of whom these
exemptions exist.
Publish regulations for adequate and Animal Drug FDA Center for ADAA enacted by 10/9/96
well-controlled clinical trials by 4/ Availability Act Veterinary Medicine
9/98 and substantial evidence by 10/ (ADAA), (P.L. 104-250) (CVM) will revise
9/98. Section 2(e). Investigational New
Animal Drug
Application procedural
regulations and
implement provisions
of the ADAA and CVM's
REGO initiatives.
Recognize and approve list of FD&C Act, Sections 514 FDA Center for Devices 0 recognized
standards suitable for use in (b) and (c). and Radiologic Health
application review. (CDRH) will recognize
over 415 standards for
use in application
review and update the
list of recognized
standards.
----------------------------------------------------------------------------------------------------------------
FY 2000 Performance Goals are not identified in this Plan. Specification of these goals is dependent upon final
determination of the President's FY 2000 Budget submission to Congress.
Objective B--Maximize the Availability and Clarity of Information
for Consumers and Patients Concerning New Products
1. Identification of Needs
FDA is reviewing applications for new drugs, biologics, medical
devices and food additives more quickly. Dissemination of information
that will enhance consumption decisions about these new products must
keep pace with the products' earlier availability. The Agency would
like to provide timely information to consumers and patients, however,
in some instances products are reaching the market faster than FDA can
inform its stakeholders. The Agency's ability to disseminate
information must be enhanced by upgrading its technology, its
computers, and the training of its employees to keep abreast with the
latest developments in technology. FDA is under pressure from Congress,
the medical community, patients, and industry to provide timely
unbiased information to its stakeholder.
Dissemination of information to consumers and patients
concerning new products must keep pace with the earlier availability of
products.
The Agency is aware of the growing diversity of consumer
health needs and interests. To respond to this diversity, FDA is
attempting to target product information that it is tailored, as much
as possible, to appropriate patient and professional audiences.
The growth in health benefits made possible by scientific
advances and new product technology is a tremendous benefit to U.S.
consumers. The speed of technology development, combined with
increasing product complexity, requires creative approaches in keeping
everyone rapidly and accurately informed.
FDA recognizes that consumers and patients want and
deserve active input and participation in the Agency's policy and
product decisions. The Agency is receiving rapid input from consumers.
FDA considers collaborations with others in the public and
private sector critical to achieving synergies in information
technology. FDA has accepted the challenge of dissemination of accurate
and timely information, although at times it can be daunting,
particularly because of the widespread audiences the Agency serves.
Use of the Internet has become increasingly central in FDA
communication with its stakeholders. FDA must upgrade its capabilities
in this area.
2. Stakeholder Views
Stakeholders strongly agree that maximizing the availability and
clarity of information to consumers and patients about new FDA-
regulated products is a priority. A selection of stakeholder comments
is provided below:
``We have consistently argued that efforts to reform the
Agency must build on, not dismantle, the ability of the FDA to
safeguard drug products . . . As the FDA's authority has been relaxed,
we feel that safety has been relaxed as well.'' [consumer advocacy
group]
``We see the FDA . . . as a data warehouse, as an
information source.'' [professional association]
``. . . FDA should aggressively educate patients' advocacy
groups, disease-specific organizations, disease experts, and new
biotech companies about FDA's function, process, and scope.'' [consumer
advocacy group]
Ensure the validity and integrity of drug information
provided on the Internet. [State, local, or federal government]
Re-evaluate [FDA's] policy on direct-to-consumer
advertising. [professional association and consumer advocacy group]
``Do not depend upon scientists to review the direct-to-
consumer advertising.'' [State, local, or federal government]
``Although Congress imposed this requirement, or at least
asked FDA to come up with ways to maximize information about new
products, our feeling on this was that this is really not
[[Page 65019]]
a function for FDA to promote new products. Rather, FDA's obligation
would be to refer inquiries about new products, new drugs, etc. to the
appropriate parties, and that might be professional societies,
physicians, medical device companies, and drug companies. [trade
association]
Use plain language on product labels. [consumer]
Make risk and safety data and statistics available to the
public via the toll-free Consumer Information Line. [consumer advocacy
group]
Inform the public when companies have been asked to revise
or pull ads, and explain why. [consumer advocacy group]
3. Current Innovations/Reinventions
FDA is currently expanding its information for consumers and
patients. The following are illustrations of the information exchange:
Collaboration
The Agency is collaborating with industry to inform patients and
consumers of the availability of new drugs (prescription and over-the-
counter [OTC] drugs). FDA engages in cooperative research with industry
for new food items as well as collaborates with industry to bring
better food labels and information to its stakeholders.
The Agency is collaborating with industry to provide technical,
non-financial assistance to manufacturers to enable them to bring their
products that meet FDA standards to the market more quickly.
Outreach
FDA has an outreach program to keep physicians informed of new
drugs available to their patients. The Agency is working cooperatively
with the drug industry, consumers, and patients to inform them of new
drugs and emerging new drugs. Patients are able to receive information
on new therapies approved by foreign countries before they are approved
by the Agency. Additionally, the Agency's Public Affairs Specialists in
the field offices furnish information to interested consumers and
patients concerning new drugs, devices, etc.
FDA delivers educational and technical assistance in the area of
food safety messages and uses. The FDA Consumer/Fact Sheets and
National Food Safety Hotlines are part of the Agency's outreach. The
Internet is used to bring new information to consumers and patients.
Each Center has its own web page. Many of these pages are interactive
and allow the user to communicate with the Agency directly. Printed
materials are provided to those that are without Internet capabilities,
and many of the materials are in several languages. These materials
help to inform consumers and patients about new drugs. The Veterinary
Newsletter, exhibits, and Public Affairs Specialists programs keep the
veterinary community abreast of the newest drugs and technology being
developed.
During the 20th century, the nation has witnessed a more dramatic
extension of longevity than humankind has ever seen. The Agency is
making a concerted effort to ensure that older persons, their families,
and their communities are aware of FDA's responsibilities and how the
Agency can be a resource for them in improving the quality of their
lives.
FDA's consumer protection and public health mission plays a
particularly important role in building a sound health foundation for
ensuring quality of a long life for older persons. The needs of the
U.S. aging population are stimulating innovative research and
technological advancements for both preventing and treating disease.
The Agency makes a meaningful contribution to this research by
facilitating the timely availability of safe and effective products,
keeping unsafe or ineffective products off the market, and providing
easily understandable and meaningful information about the availability
of new products, as well as how to use products safely and effectively.
In October 1998, the United Nations launched the International Year of
the Older Person 1999 to bring global attention to the phenomenon of an
aging world and the need to begin to establish the policies, programs,
and services needed to meet the needs of an aging world. The Agency is
an active participant in this initiative.
4. Plan for Meeting Statutory Requirements and Public Expectations
Section 406(b) requires the Agency to maximize the availability and
clarity of information for consumers and patients concerning new
products. FDA is engaged in a variety of activities to fulfill this
requirement that revolve around four themes. First are Agency efforts
to ensure that product information is tailored to meet the special
needs of diverse populations. One example is the implementation of
public awareness campaigns for consumers, i.e., Take Time to Care,
Office of Women's Health; Mammography Awareness Seminars; Food Safety
Programs (Fight BAC!TM); Over the Counter Labeling Changes
(OTC) Campaign; and the Partnership for Food Safety Education. As the
population becomes more culturally diverse, FDA must reach out to
consumers in ways they will understand. For instance, Public Affairs
Specialists give seminars on new drug therapies, health fraud,
labeling, etc. in different languages to fulfill the needs of diverse
populations.
The Agency is entering into an increasing number of stakeholder
``collaborations'' to achieve a multiplier effect (e.g., with print
media, radio, television, industry, other federal agencies, consumers,
health professionals, and associations). Another example is
implementation of the Pharmacist Education Outreach Program to assist
pharmacists in explaining the drug approval process to consumers.
Another approach is focusing FDA resources so that patients are an
integral part of the health care decisionmaking process. FDA has
established programs to make promising investigational drugs,
therapies, and devices available to patients with serious and life-
threatening conditions. For example, FDA has also included patient
representatives on advisory committees considering products for HIV/
AIDS, cancer, and other serious diseases.
The technological revolution provides the Agency the tools to offer
quick access to a wide range of information to consumers through
various methods. The Internet is being used as a means for two-way
communication--both to disseminate information about new products and
to quickly answer questions about new and existing products.
Additionally, the Agency will participate with NIH in the establishment
of (under Section 402 of the Public Health Service Act) a registry of
publicly and privately funded clinical trials for experimental drugs
and biologics being tested for serious or life-threatening medical
conditions. This registry will simplify the process of obtaining
information.
[[Page 65020]]
5. Performance Goals for FY 1999
The table provided in this section links the performance goals and
the measures with statutory requirements to regulate information
provided to consumers and to ensure that consumers understand OTC drug
information. The FY 1999 performance goals focus on both OTC and
prescription drugs. FDA wants consumers and patients to receive and to
be able to refer to the highest quality information when taking either
OTC or prescription medications.
Highlighted below are key performance goals for FY 1999. These
goals seek to provide drug information, in easily understood language,
to consumers and patients faster through various outreach efforts. For
more complete identification of performance goals and statutory
requirements see the table at the end of this section.
FY 1999 Performance Goals
Evaluate drug information provided to 75 percent of individuals
receiving new prescriptions.
Improve OTC information and consumers' ability to understand it by
2001.
BILLING CODE 4160-01-M
[[Page 65021]]
[GRAPHIC] [TIFF OMITTED] TN24NO98.008
BILLING CODE 4160-01-C
[[Page 65022]]
Objective C--Implementing Inspection and Postmarket Monitoring
Provisions of this Act
A central part of FDA's responsibilities to protect the public
health includes: (1) ensuring that manufacturing establishments and the
products being produced by these establishments--both domestic and
imported--are meeting safety and quality standards that are acceptable
to the U.S. and (2) monitoring these products to identify and correct
any problems associated with their consumption and use. Through
inspection and monitoring activities, potential hazards are identified
and corrected in time to prevent or minimize public exposure.
The discussion that follows is divided into these two areas of
postmarket responsibility.
Subobjective C1.--Assuring Product Safety
A. Domestic Inspections
1. Identification of Needs
FDA is responsible for ensuring the safety of products produced and
distributed by more than 100,000 domestic establishments. The Agency
uses its inspection authority, as directed by the statute, to provide
this assurance. Approximately 45,000 of these establishments
manufacture or process regulated product. FDA inspected 30 percent of
these facilities in FY 1997. A sizable number of the remaining
establishments (23,000) are distribution facilities, of which FDA
inspected 10 percent in FY 1997. The remainder includes 10,000
mammography facilities, which FDA inspects at a nearly annual rate, and
a varied assortment of other establishment types, e.g. control
laboratories, importer/brokers, clinical investigators, and
conveyances, of which FDA inspected about 14 percent in FY 1997.
Overall, approximately 40 percent of FDA's current inspectional
coverage is provided through contracts with states.
As these varying inspectional coverage statistics indicate, FDA
exercises considerable discretion regarding the frequency and
comprehensiveness of inspections. For approximately 25 percent of this
inventory, however, the law requires FDA to conduct inspections at
specified maximum time intervals. Certain manufacturing facilities must
be inspected at least once every 2 years, and mammography facilities
must be inspected at least once each year. In recent years, inspection
coverage has fallen short of meeting these statutory requirements. The
table below summarizes the Agency's recent coverage of the domestic
inventory including the segment subject to statutory minimum inspection
coverage as well as the segment over which the Agency has discretion
regarding inspection frequency. To meet the statutory requirements, 100
percent of the mammography facilities and at least 50 percent of the
other statutory establishments should have been inspected in FY 1997.
As the data show, with the exception of mammography facilities, neither
goal was reached.
----------------------------------------------------------------------------------------------------------------
Statutory coverage Non-statutory coverage
-----------------------------------------------------------------
Program area Inventory Coverage in FY Coverage in FY
Establishments 1997 (percent) Establishments 1997 (percent)
-------------------------------------------------------*--------------------------------*-----------------------
Biologics..................... 5,685 2,787 46 2,898 13
Human Drugs................... 19,749 6,408 23 13,341 12
Devices (excluding
mammography)................. 27,638 4,870 28 22,768 9
Mammography............... 10,000 10,000 96 ............... ..............
Foods......................... 49,000 NA NA 49,000 23
Animal Drugs and Feeds........ 6,414 1,688 27 4,726 13
----------------------------------------------------------------------------------------------------------------
* Status as of May 1998.
2. Stakeholder Views
Agency stakeholders expressed strong support for more regulatory
enforcement in general, and the continued focus on risk-based
inspections in particular.
``Stratify the inspections based upon past history of
compliance of companies, the degree of risk of the product, and various
other elements.'' [trade association].
FDA should increase its efforts to monitor the marketplace
to remove unapproved products and also those that provide unfair
competition. [trade association]
Inspections should take a comprehensive approach and
``focus on the health impact of the regulations, not just the `black-
and-white' of the regulations. [state, local or Federal government]
There should be more enforcement efforts to prevent
distribution of illegally marketed and compounded drugs, unapproved
drugs not manufactured in accordance with current GMPs, illegal
extralabel use practices, illegal distribution of veterinary
prescription drugs, marketing of unapproved feed ingredients, and
extraordinary claims on animal feed labels. [trade and professional
associations]
Stakeholders endorsed HACCP systems for seafood and retail
settings and the possible expansion of HACCP into other food-related
areas, but only when supported by science and a high consumer safety
priority. [trade association]
``Move towards a voluntary HACCP-based system for dairy
products and away from checklist inspections and prescriptive plant
processing regulations.'' [trade association]
HACCP would be applicable in general for ``foods with a
demonstrated high risk (e.g., unpasteurized juice).'' In contrast,
stakeholders urged the Agency not to ``promote the HACCP process for
device conformance,'' but to consider ISO certifications [standard
setting organization].
Stakeholders encouraged FDA to work closely with the
states and to ``be a leader (i.e., leadership in science, setting
standards, evaluating state programs, certifying inspectors).'' [state,
local or federal government].
The Agency should provide more guidance and training to
state investigators to minimize inconsistency between investigations in
different states and districts, thereby contributing to a level playing
field for regulated firms. The Agency should involve states in the
development of enforcement strategies related to animal drugs and
feeds. [state, local or federal government]
[[Page 65023]]
Stakeholders tended to support the idea of third-party inspections,
especially noncritical inspections.
The Agency should identify more functions that could be
performed by third parties. [trade association]
In some cases, particularly the manufacture of animal
feeds, voluntary self-inspection with third-party oversight might be
appropriate. [state, local or federal government]
At the same time, however, the Agency needs to be careful
to avoid duplication of effort and to ensure consistency between FDA
inspectors and third parties. [trade association]
3. Current Innovations/Reinventions
The Agency's domestic inspection program is an integral part of the
strategy for monitoring the compliance status of the regulated
industry. The goals of an inspection may be many and varied, i.e., to
verify data submitted to the FDA in a new drug or biologic application,
and to ensure continued compliance with application commitments.
Inspections monitor the regulatory control over manufacturing
operations including compliance with current GMP regulations. The
results of inspections form the basis for many of the Agency's
administrative and regulatory decisions, including new drug, device, or
biologic approvals, as well as detecting industry problems or
objectionable conditions and practices.
Establish Risk-Based Priorities
Given the large inventory of establishments it must inspect with
limited resources, FDA targets the highest risk products and those
facilities whose violations of standards would most likely expose the
public to unnecessary risk. The cornerstone of the Agency's drug (human
and animal), medicated feed, biological, and medical device inspection
strategy is the biennial inspection requirement, which mandates the
inspection of critical establishments in the Agency's inventory,
primarily manufacturers, at least once every 2 years. While FDA has no
such legal mandate for food inspections, it is moving toward
establishing a vertically integrated food safety system that is risk-
based and which would allow it to inspect high-risk establishments
every 1 to 2 years and moderate-to-low risk establishments every 4
years.
Adopt a System Rather Than a Piecemeal Approach to Agency Regulation
Manufacturing processes are becoming more complex due to the rapid
advancement of science and technology. This trend continues to
accelerate. This increasing complexity is mirrored in FDA's approach to
ensuring comprehensive, consistent, and fair inspections.Where, in the
past, the Agency often perceived its role as providing quality control
for the industries it regulated, today, it recognizes the essential
role that establishments themselves must play to ensure product quality
assurance.The Agency is focusing more on ensuring that the systems the
industry has in place to monitor the quality of its products are
adequate. This approach stresses the importance of HACCP-type
inspections and frequently requires that the Agency take a
multidisciplined, team approach to inspections.
the FDA Center for Biologics Evaluation and Research
(CBER), which used to conduct many inspections on its own, joined with
the FDA Office of Regulatory Affairs (ORA) to form `Team Biologics'
whereby teams of CBER product specialists and specially trained
investigators from ORA's field force work together to conduct
surveillance inspections. Follow-up compliance actions are handled
under a streamlined system that provides concurrent review by CBER and
ORA.
CDER, to ensure inspection consistency, is developing
standards for investigator training and certification for performance
of pharmaceutical inspections.
CFSAN has developed and implemented HACCP controls for
seafood and has proposed HACCP controls for the juice industry. All
seafood processors had been inspected by the end of FY 1998 to verify
proper use of HACCP, and 6,681 industry officials and federal and state
inspectors have been trained in seafood HACCP through the Seafood
Alliance.
CDRH, whose quality systems regulations ask manufacturers
to take more responsibility for assuring the quality of devices, is
moving toward systems-oriented inspections and developing HACCP-type
programs for firms with a good compliance history.
Work More Closely With External Stakeholders
The Agency increasingly has emphasized communication and education
as alternatives that are at times preferable to and more effective in
achieving and maintaining compliance than the more traditional
enforcement approaches used in isolation. It accomplishes this by
providing training and workshops for industry groups, seeking the views
of stakeholders, and sharing information with stakeholders and
colleagues. Some examples of the Agency working closely with external
stakeholders include:
CBER produced a satellite broadcast on blood establishment
inspections to educate the industry and held a workshop for
manufacturers of licensed in vitro diagnostics.
CDRH undertook education efforts on quality systems
requirements.
CFSAN issued guidance on GMPs and Good Agricultural
Practices (GAPs), worked with the U.S. Department of Agriculture (USDA)
to achieve adoption of the Food code by an increasing number of states,
collaborated with JIFSAN/World Health Organization (WHO) for risk
assessment, and cooperated with USDA and the Centers for Disease
Control and Prevention (CDC) to implement a national education program
on retail food preparation practices.
CDER, ORA, and a major industry scientific trade
organization in conjunction with a university developed a new approach
for training field investigators in pharmaceutical manufacturing
operations and the application of GMP and other FDA regulations to new
drug development.
CVM, in cooperation with stakeholder groups, sponsored
satellite teleconferences concerning compliance with the BSE feed
regulation and the Animal Medicinal Drug Use Clarification Act, which
concerns extralabel drug use.
District offices conduct ``grass roots'' meetings and
industry exchange meetings on a variety of regulatory matters as a
means of facilitating an ongoing dialogue with various constituencies.
4. Plan for Meeting Statutory Requirements and Public Expectations
Under provisions of the Food, Drug and Cosmetic Act and the Public
Health Service Act, FDA is required to conduct biennial inspections of
approximately 16,000 registered drug, biologic and device production
facilities. Although there is no statutory requirement that mandates a
particular frequency for the inspection of any food establishment, or
those drug, biologic and device facilities excluded from the biennial
requirement, the statute obliges the Agency to ensure the safety of
regulated products within these establishments. Accordingly, goals have
been set within these establishment categories to achieve an average
inspection cycle of once every 4 years, with appropriate risk-based
variations in this cycle where warranted.
FDA fell short of meeting its statutory biennial and annual
inspection obligations by approximately 4,000
[[Page 65024]]
inspections in FY 1997. In an effort to improve its performance in
these critical areas, FDA plans to rely increasingly on states and
other third parties, both for direct help with some statutory
inspections and for other important inspectional obligations, thus
freeing some of FDA's own resources to cover additional statutory
obligations. Because all public and private sector organizations in the
future will be subject to the same resource-constrained environment,
FDA may have to consider that even a highly collaborative inspectional
network may not be adequate to completely meet existing statutory
inspectional requirements. A strategic reassessment may be in order to
determine the kinds of statutory flexibility that would be desirable to
preserve the comprehensive consumer protection intent of the FD&C Act,
and at the same time, allow FDA to address the most critical health and
safety priorities. Some examples of Agency initiatives either planned
or already underway include the following:
Developing contracts with states and public health
agencies to inspect unlicensed blood banks.
Reinstating state contracts for medical gas inspections,
oxygen bars, and emergency medical services. FDA is considering a pilot
First Party Audit Program (FPAP).
Concentrating its own resources on the highest risk
devices such as cardiac implantables and relying on third parties for
inspection of lower risk products.
Continuing to develop contracts and collaborations with
states for both statutory and non-statutory animal drug and feed
inspections.
Conducting joint surveillance work with CDC and USDA and
working with the Association of American Feed Control Officials (AAFCO)
to develop a model program for medicated feed manufacturers that
includes self inspection.
Special Emphasis on Food Safety: The Agency recognizes its
obligation to ensure the safety of the food supply, and the public
expects food to be safe. To met this expectation, FDA needs to inspect
high-risk establishments every 1 to 2 year and moderate-to-low risk
establishments every 4 years. This level of inspection coverage will
require an additional 4,000 to 6,000 annual inspections. FDA's own food
safety assurance efforts is being integrated with a national risk-based
food safety system. This will require close collaboration with USDA,
CDC, the states, food manufacturers and food retailers. Key elements of
the initiative are:
Surveillance activities that enhance electronic
communication with states and other agencies to permit rapid
identification of and response to foodborne hazard outbreaks;
A cooperative inspection and monitoring effort with states
that focuses on high-risk firms, and emphasizes enforcement of
initiatives such as FDA's BSE Feed regulation.
Education emphasizing safe handling practices for
consumers and retailers through FDA's Model Food Code; and
Research to develop improved methods of detecting and
identifying pathogens and formulating preventive interventions.
5. Performance Goals for FY 1999
This section contains two tables. The first table summarizes the
Agency's domestic inspection performance goals for FY 1999. The second
table links these performance goals to the statutory requirements.
FY 1999 Performance Goals
Inspect 46 percent of registered biologic firms
Inspect 23 percent of registered drug manufacturers, propagators,
compounders, or processors
Inspect 28 percent of registered class II and III medical device
manufacturers, propagators, compounders, or processors
Conduct 8,898 inspections of mammography facilities
Ensure that 50 percent of seafood industry operating under HACCP
Develop HACCP final rule for fruit and vegetable juices
Inspect 50 percent of registered animal drug and feed establishments
----------------------------------------------------------------------------------------------------------------
Relevant statute and/or Relevant FY 1999 FY 1997 performance
Statutory authority regulation performance goals baseline
----------------------------------------------------------------------------------------------------------------
Biennial GMP inspections of biologic FD&C Act--Sec. 510(h).. Coverage: 46 percent... Coverage: 46 percent.
firms (50 percent annually).
Biennial inspections of registered FD&C Act--Sec. 510(h).. Coverage: 23 percent... Coverage: 23 percent.
drug manufacturers, propagators,
compounders, or processors (50
percent annually).
Biennial inspections of registered FD&C Act--Sec. 510(h).. Coverage: 28 percent... Coverage: 28 percent.
class II and III medical device
manufacturers, propagators,
compounders, or processors (50
percent annually).
Annual inspections of mammography PHS Act--Sec. 354...... Conduct 8,898 Conduct 8,280
facilities. inspections. inspections.
General authority to inspect food, FD&C Act--Sec. 704..... Ensure that 50 percent .......................
drugs, devices, or cosmetic of seafood industry
establishments. operating under HACCP.
Develop the HACCP
final rule for fruit
and vegetable juices.
Biennial inspections of registered FD&C Act--Sec. 510(h).. Coverage: 20 percent... Coverage: 27 percent.
animal drug and feed establishments
(50 percent annually).
----------------------------------------------------------------------------------------------------------------
FY 2000 Performance Goals are not identified in this Plan. Specification of these goals is dependent upon final
determination of the President's FY 2000 Budget submission to Congress.
Subobjective C1--Assuring Product Safety (Continued)
B. Imports
1. Identification of Needs
Imported products pose multiple challenges to FDA. These include
the sheer volume and diversity of products, the difficulty of
ascertaining exactly which establishments are shipping products to the
United States, and the difficulty of verifying conformity with GMPs
quality systems. Each of these challenges, is described in the
following paragraphs.
The Volume and Diversity of Products
FDA is responsible for ensuring the safety of nearly 4 million line
entries that cross our borders annually, or over 12,000 entries per
day. Imports of all products that FDA regulates have been increasing;
pharmaceuticals, both finished and bulk, are increasing very
[[Page 65025]]
rapidly. Approximately $57 billion of FDA-regulated product was
imported in 1997. The sources are diversifying and including more
products from countries that are typically categorized as emerging
economies, with emerging regulatory infrastructures. The products
include, among others, food products that have been implicated in
serious disease outbreaks in the United States, food products that
could pose health threats if not processed and handled properly, over-
the-counter drugs that do not require a new drug application with the
Agency, as well as approved drugs, biologics, and medical devices.
Difficulty in Ascertaining Establishments Shipping to the United States
Section 417 of FDAMA [510(i) of the Act] now requires all foreign
manufacturing establishments whose drug and device products are
imported into the United States to register. There is, however, no
universal registration requirement for producers of imported food
products. Manufacturers/packers of low-acid canned food, acidified
foods, and infant formula (all of which products are considered at high
risk) register or list with the FDA; other food producers and
processors are not required to register or list with FDA, making
identification of sources of product difficult.
Difficulty of Verifying Conformity with GMPs/Quality Systems
There are two ways that typically are used to confirm that product
has been produced properly--end point product testing (which for
imports could be analysis of border samples) and on-site inspections.
There are difficulties with both of these approaches. To date, no
effective, scientifically based method has been established for general
screening of foreign drug product for adherence to GMPs. Analysis of
product samples is reasonably effective in assuring conformity, but the
volume of trade and resource limitations preclude high rates of
analysis. On-site inspections, the way of affirming conformity with
good manufacturing practices/quality systems, are expensive and pose a
host of logistical and practical difficulties. All foreign firms are
aware that an FDA inspection is planned well in advance of the
inspection, unlike the inspection of domestic establishments.
Regardless of these challenges, there is consistent expectation from
the Congress that FDA assure foreign product safety, and there is
recurring congressional focus on FDA inspections of foreign
manufacturing facilities.
2. Stakeholder Views
Stakeholders want assurances that foreign products meet the high
standards expected of domestic products, and encourage FDA to conduct
foreign inspections and periodic testing of product to confirm quality.
Stakeholders strongly support FDA's activities in Codex and
international harmonization, reflecting a desire to minimize regulatory
burden while assuring that foreign produced food products are safe and
therapeutic products are safe and effective. Stakeholders especially
stress the importance of effective participation in Codex, because of
the special place Codex holds in resolving international trade issues:
the international standards that are adopted must reflect the standards
and the high level of safety required in the United States. Support for
pharmaceutical GMP mutual recognition agreements (MRAs) was predicated
on the likelihood of there being equivalent standards as well as truly
effective regulatory programs in MRA countries. The need for expanded
funding support for Codex activities and for monitoring of imports was
noted. A few typical comments are as follows:
Assurance that Foreign Product Meets High Standards Expected of
Domestic Product
``Realizing this would require improved resources and
budgets, it would still seem appropriate to perform periodic [foreign]
quality assurance inspections and [border] laboratory analyses for
identity, potency, and purity to ensure the quality of the drugs
manufactured in foreign countries, do, in fact, equal ours.'' [state,
local, or federal government]
``We do think more emphasis needs to be placed on
inspections of imports for safety and purity, with the important caveat
that such inspections should not constitute non-tariff trade
barriers.'' [trade association]
``We have concerns regarding imported foods. In many
cases, the hygienic requirements for production and processing of a
food in the United States are more stringent than in countries with
competing foods that are exported into the United States. More effort
needs to be focused by CFSCAN in reducing the risk to the consuming
public from the imported foods.''. [trade association]
Support for Codex Activities
``* * * the Codex has grown in significance as more and
more of our nation's food supply is either imported or exported. Food
regulatory bodies around the world, including the FDA, have begun to
recognize that harmonized international standards are not just a good
idea. They are essential if the country is going to compete in today's
global marketplace.'' [trade association]
``Codex quality and safety standards are being utilized
increasingly to resolve food safety disputes between nations in the
World Trade Organization. Therefore, FDA must play an active role in
Codex to ensure international standards and guidelines are consisent
with US requirements.'' [trade association]
Support for Mutual Recongition Agreements (MRAs)
``CVM needs to determine whether foreign countries'
requirements and systems for animal drug approvals ae equivalent to
those in the United States.'' [trade association]
``While the MRA is attempting an honorable and desirable
result, we would like to stress that the foreign countries should not
only have equivalent standards but effective regulatory programs as
well.'' [state, local, or federal government]
* * * but a Cautionary Note
``FDA needs to be a spokesperson for public health. The
whole drive behind international harmonization is trade concerns * * *
That may be fine from an economic standpoint, but it has nothing to do
with FDA's public health mission. FDA needs to be there * * * to put
public health * * * if not first, at least equal to trade concerns.''
[consumer advocacy group]
``* * * there is no question that we are bound by
international agreements to harmonize regulatory standards in the area
of food regulation * * * [T]his presents not only a threat but an
opportunity because if we are going to go about harmonizing regulatory
requirements, we can go up or down * * * When our current requirements
may not be that high, we should raise our requirements and advocate the
stronger requirements to become the international standard and a model
for the U.S.''. [consumer advocacy group]
3. Current Innovations/Reinventions
FDA must ensure that the structure in place at the point of origin
results in product being shipped to the United States meeting FDA
requirements for safety, quality and/or therapeutic efficacy. This is a
prevention-based strategy. A secondary strategy is detection based:
conduct inspections of establishments shipping product to the United
States, and screen product at the border for more intensive review.
[[Page 65026]]
Electronic screening allows conforming product to more quickly into
commerce, while identifying product that may need more review at the
border.
To deal with an explosively expanding workload and flat resources,
FDA has directed its non-Prescription Drug User Fee Act of 1992 (non-
PDUFA) foreign inspection activities toward higher risk products and is
expanding PDUFA inspections to include more comprehensive inspections
of facilities. More screening of product at the border is being
accomplished through electronic means. And finally, analysis of product
at the border is increasingly targeted toward product that is expected
to pose high risk, as identified in the electronic screening. This
risk-based prioritization means that many medium-risk product
manufacturing facilities are not inspected, and most lower risk product
facilities are not inspected.
4. Plan for Meeting Statutory Requirements and Public Expectations
With additional resources, FDA expects to strengthen the safety net
that extends from the point of production in source countries through
their entry into the U.S. These strategies encompass: (1) Reducing the
probability that violative products will be exported to the United
States; (2) Making rapid and reliable decisions on product entry at the
border; and (3) Targeting violative products at the border and
preventing their entry.
To reduce the probability that violative products will be exported
to the United States, FDA will continue to participate in international
negotiations and establishment of mutual recognition agreements with
other nations. These activities will assure that products from those
nations are meeting FDA standards, and will also increase the number of
foreign inspections. As international regulatory agreements are
negotiated among trading nations, the Agency will explore new and
innovative institutional arrangements, such as a third-party
certification of both imports and exports. These arrangements will have
to be cost-effective, with statutory mandates, and enforce health and
safety standards. To allow rapid entry of safe products, FDA continues
to enhance its electronic screening process. To target violative
products at the border, the Agency will maintain its ability to conduct
laboratory analysis on a small percentage of products with potential
problems, by increasing its sample analysis. The Agency will also
enhance the electronic import entry system to provide for a broad-scope
collection and analysis of information on product-country intersects
that will allow development of national profiles. These profiles will
provide the basis for establishing systematic risk-based priorities in
examining import entries. Many of these efforts are obviously resource
intensive, and linked closely with the steadily rising volume of
imports.
5. Performance Goals for FY 1999
Consistent with the strategic directions noted above, FDA has
established performance goals that support moving toward higher
assurance of imported product safety in a time of increasing imports,
as noted in the table below. The FD&C Act provides for sampling of
product at import, and FDAMA modifications require the Agency to engage
in activity designated to harmonize regulatory requirements with the
objective of reducing the burden of regulations. Goals to support these
activities address the short-term screening of imports at the border as
well as longer term infrastructure development internationally, and
these are noted in the table below. A more comprehensive table,
illustrating legislative provisions, follows.
Associated with the immediate need at the border, the performance
goals relate broadly to assuring the integrity of the screening system,
such as by confirmation of the accuracy of entries and continual
updating of the screening criteria and by improving the overall
sampling and the targeted sampling rates at the border. Goals relating
to international infrastructure development reflect ongoing commitment
and heavy investment in international standard setting forums and
negotiating equivalence agreements and mutual recognition agreements.
Success in these realms would allow FDA to rely more on the regulatory
structures in place at the point of origin of products being shipped to
the United States. And finally, there are times when direct FDA
inspections of foreign manufacturing sites are necessary to ensure the
quality of product being shipped to the United States, and several
performance goals reflect this need.
FY 1999 Performance Goals
Enhance the safety of imported products through increased
surveillance of imported food products at the border, increased
foreign inspections (from a target level of 40 to 75-100), through
providing education, outreach, and technical assistance to foreign
countries on the use of GAP/GMP guidance for produce, and through
the evaluation of food production systems in foreign countries.
Enhance import screening capabilities for public health while
ensuring that 55 percent of entries are released within 15 minutes.
Assess potentially violative imports through direct examination of 3
percent of entries.
Accept at least 20 percent of imports into the U.S. market through
evidence that source country quality systems/standards/ audits meet
the requirements of the FD&C Act.
BILLING CODE 4160-01-M
[[Page 65027]]
[GRAPHIC] [TIFF OMITTED] TN24NO98.009
[[Page 65028]]
[GRAPHIC] [TIFF OMITTED] TN24NO98.010
[[Page 65029]]
[GRAPHIC] [TIFF OMITTED] TN24NO98.011
BILLING CODE 4160-01-C
[[Page 65030]]
Subobjective C2--Adverse Event Reporting
1. Identification of Needs
FDA needs to work with its community of stakeholders and develop a
systematic approach to address the problem of over 2 million injuries
and deaths a year occurring as a result of consuming/using FDA-
regulated products. The ideal approach should be comprehensive,
involving the participation of regulatory agencies, health care givers,
the regulated industry, and the consumers/patients themselves.
Components of this system include:
A full understanding of the causes of product-related
deaths and injuries: FDA needs to ensure that causes attributable to
product labeling, design, or composition are addressed in the premarket
review programs, where required. FDA currently receives yearly
thousands of reports of injuries and deaths associated with the misuse
or failure of FDA-regulated products. FDA should improve the quality of
information on adverse events and product failure and develop methods
to enhance understanding of causes of product-related injuries.
Currently, for example, the FDA's ability to identify and track the
causes of food-borne illness is very limited.
New postmarket information-gathering programs: FDA often
has little date with which to make fundamental decisions about some
products. This is especially true for products like foods and cosmetics
for which no premarket approval is required. New programs must be
initiated, in collaboration with other agencies, to provide such data.
The Agency also needs to implement new ways of gathering data. The
National Sentinel Reporting System, a nationally representative sample
of medical device user-facilities, is expected to be a less expensive
way of providing better and quicker data on medical device-related
problems than the 100 percent mandatory reporting system now used. This
system cannot be implemented without the necessary funds.
Rapid dissemination of findings: FDA needs to be an active
participant in a multi-institutional network that can detect adverse
effects quickly and can disseminate information to health professionals
industry, and consumers quickly.
Outreach and education: A significant component of
improving the current situation is to improve the feedback to health
care personnel and consumers. Requested resources will be devoted to
developing strategies , such as consumer publications and public
service announcements, to reduce the number of injuries from food and
cosmetic products.
2. Stakeholder Views
There is strong stakeholder support for improving the data
collection, analysis, and dissemination of information from the
existing Adverse Event Reporting System and for some of the news data
collection initiatives. A few indications of these views follow:
``The process for adverse event/injury reporting is
perhaps the most urgent task facing FDA today. The process by which
adverse injury report data is captured and converted to agency and
consumer use must be addressed.'' [consumer advocacy group]
``Perform analysis and trend reporting on error and
accident reports and make this available to the industry.'' [trade
association]
``Improve the handling of adverse event reports for
dietary supplements to involve the industry earlier.'' [trade
association]
``Consumer safety is being threatened by funding cuts in
1996 that eliminated the adverse-reaction report part of the voluntary
reporting program for cosmetics. [trade association]
``Accurate food safety statistics are vital to developing
an effective strategy for enhancing the safety of our nation's food
supply.'' [trade association]
3. Current Innovations/Reinventions
FDA has initiated several programs for gathering information on
adverse events/injuries associated with the misuse or failure of FDA-
regulated medical products and foods. These include the following:
MedWatch
MedWatch covers drugs, biologics, medical and radiation-emitting
devices, and special nutritional products, such as medical foods,
dietary supplements, and infant formulas. The MedWatch form is used for
voluntary and mandatory reporting of adverse events and product
problems by health professionals; the reports are sent on to the
appropriate FDA component for analysis and follow-up action. Over 140
health professional and industry organizatios have joined the MedWatch
effort as MedWatch Partners and actively support the program by
promoting the importance of reporting serious adverse events or product
problems to their members.
Adverse Events Reporting System (AERS)
With its new computer system, the Adverse Events Reporting System
(AERS) is expected to form the basis for a revitalized
pharmacovigilance program for the United States. AERS continues to be
developed and will be relied upon by both CDER and CBER over ensuring
years to provide accurate, accountable data for the performance goals
identified for injury reporting.
FDA is responsible for monitoring the market for adverse effects of
medical devices. FDA expects to receive over 63,000 postmarket reports
in FY 1998, including mandated reports from medical device
manufactures; voluntary reports from medical device professionals
received through the problem reporting program (MedWatch); and results
of field inspections. FDA currently is managing the huge numbers of
reports in three phases. During the first phase, the reports are
screened for completeness and entered into the data management system.
During the second phase, the reports are analyzed for similar events,
judged for severity, and searched for trends. The final phase focuses
on action, such as issuing safety alerts and notifications to users
(i.e., health professionals and patients) warning them of concerns and
advising them how to prevent future occurrences.
Some manufacturers have been granted approvals to submit summary
reports quarterly for adverse events involving specific devices. This
summary erporting system is bieng expanded and will produce usable
information at a small cost to both FDA and the industry.
FoodNet
FoodNet is the product of a cooperative venture among USDA, CDC,
and FDA; it attempts to estimate the incidence of foodborne illness
that is not revealed in obvious outbreaks. Most foodborne illness
occurs in ways that appear sporadic and unrelated to each other.
FoodNet, which has the ability to provide more comprehensive
information through sources such as case-control studies and surveys of
laboratories and physicians, can help FDA and its federal colleagues
link illnesses that have a common cause, no matter where they occur.
National Antimicrobial Resistance Monitoring System (NARMS)
The National Antimicrobial Resistance Monitoring System (NARMS) was
established in January 1996 as a collaborative effort among the FDA,
USDA, and CDC. The system was
[[Page 65031]]
initiated in response to public health issues associated with the
approval of fluoroquinolone products for use in poultry. The NARMS
program monitors changes in susceptibilities to 17 antimicrobial drugs
of zoonotic enteric pathogens from human and animal clinical specimens,
from healthy farm animals, and from carcasses of food-producing animals
at slaughter. The objectives of the system include: to provide
descriptive data on the extent and temporal trends of antimicrobial
susceptibility in Salmonella and other enteric organisms, to facilitate
the identification of resistance in humans and animals as it arises,
and to provide timely information to veterinarians and physicians. The
ultimate goal of these activities is to prolong the lifespan of
approved drugs by promoting prudent and judicious use of antimicrobials
and taking appropriate public health action.
Vaccine Adverse Events Reporting System (VAERS)
CBER and CDC jointly overseas the Vaccine Adverse Events Reporting
System (VAERS), which receives mandatory reports as required by the
National Vaccine Injury Act about adverse effects from vaccines. CBER
and its colleagues are discussing electronic submission of reports,
which would provide more rapid access of the VAERs data to
manufacturers.
4. Plan for Meeting Statutory Requirements and Public Expectations
Prompt identification of new, previously unrecognized problems with
FDA-regulated products has the potential to decrease morbidity and
mortality associated with those products and maximize the safety of
approved products. Thousands of deaths and injuries could possibly be
avoided, or their consequences reduced, through a comprehensive
strategy aimed at finding out why incidents occur and implementing
strategies to prevent them from occurring again.
One of the Agency's primary objectives is the development and
implementation of a system for improving the quality of information on
adverse events and product defects associated with FDA-regulated
products. This system needs to address issues of injury reporting by
focusing on three areas: surveillance and epidemiology; research; and
education and outreach. FDA believes that such a system would maximize
the safety of FDA-regulated products through increased reporting of
potentially dangerous adverse events or product problems to FDA or the
manufacturer. Increased reporting provides greater assurance that a
potential problem with a marketed product will be discovered and
appropriate corrective action will be taken, and it ensures systematic
feedback to the health care community and the public. None of these
systemic improvements are possible without adequate funding.
Surveillance and Epidemiology
With sufficient resources, FDA continues to develop and
revitalize its system for reporting, monitoring, and evaluating adverse
events associated with FDA-regulated products. AERS is the basis for
this revitalized program.
FDA is also developing active reporting systems for foods
and for medical devices. These active systems use statistical selection
of sites to provide better estimates of adverse events from the events
that are reported.
FDA will implement a National Sentinel Reporting System to
provide an alternative to 100 percent mandatory reporting by medical
device user-facilities. The system will use a nationally representative
sample of user-facilities to track postmarket adverse events and is
intended to save the industry millions of dollars in reporting costs.
The system also will provide FDA clinicians and analysts with more
timely, and better quality, postmarket data, thus improving FDA's
ability to detect and to analyze medical device-related problems. In
addition, this system is intended to provide FDA with ready access to a
network of clinical facilities that could offer clinical insight into
problem investigation and participate in specific research and
educational efforts on product problems. However, this cannot be
implemented without the necessary funds.
Research
Methodologic and surveillance research efforts designed to
understand the causes of, and the factors contributing to, product-
related injuries are critical to reducing the number of FDA-regulated
product injuries. Research will be initiated in ``human factors
sciences'' to identify labeling and product interface design features
that may cause or contribute to use error, a leading cause of avoidable
deaths and injuries.
Education and Outreach
Improving feedback to health care professionals and consumers is
critical to the improvement of adverse event reporting. Rapid
dissemination of findings on injuries to the relevant stakeholders and
the education of the medical community require additional resources.
The Agency has begun to collaborate with other agencies and
professional groups to produce teleconferences that convey general
information or product-specific information, nationwide.
An integrated science-based system for reporting, monitoring, and
evaluating food and cosmetics-based adverse events is necessary to make
fundamental regulatory decisions and policies. This system will depend
on a research program aimed at understanding how health care
professionals, as well as the public, can better recognize product-
problems, and on a related research program on methods of analyzing the
data. The clinical evaluation of adverse events and the determination
of risk assessment requires medical officers and other trained
personnel to take follow-up actions, make clinically-based decisions,
and report activities to FDA's existing staff.
5. Performance Goals for FY 1999
The table provided in this section links FDA's statutory
requirements with performance goals in the FY 1999 Performance Plan,
illustrating the Agency's efforts to consolidate several systematic
approaches into one performance system.
Highlighted below are key performance goals for FY 1999 in the area
of adverse event reporting. These performance goals deal with creating
new, active surveillance systems, or with improving passive reporting
programs to make them more useful and available. For more complete
identification of performance goals and statutory requirements see the
table at the end of this section.
FY 1999 Performance Goals
Implement AERS for the electronic receipt and review of Adverse Drug
Report (ADR) reports
Evaluate pilot efforts for new postmarket surveillance system
Increase the number of reports on device events that are received
and processed in summary form by using electronic reporting
Develop baseline surveillance data on foodborne illness under the
FootNet program
Improve public access to information on adverse events with Special
Nutritionals
Increase the number of human and animal isolates in National
Antimicrobial Resistance Monitoring System (NARMS)
[[Page 65032]]
----------------------------------------------------------------------------------------------------------------
FY 1997 FY 1998
Statutory authority Relevant statute Relevant FY 1999 performance performance
and/or regulation performance goals baseline baseline
----------------------------------------------------------------------------------------------------------------
Applicants must report to FDA FD&C Act, Section By the end of FY 1999, Implementing the FY 1998: Pilot,
adverse drug experience 505; Public implement the AERS core system is five firms
information. Health Service for the electronic currently under electronic entry
Act, Section receipt and review of way and will be uncoded only.
2101-2134; 21 voluntary and completed by FY Periodic reports
CFR 314.50, mandatory ADR reports. 1998. only.
314.80-81,
314.98, 314.540,
and 600.80.
Plan and implement a sentinel FD&C Act Section Evaluate pilot efforts Not applicable... Recruit 24 pilot
user reporting system. 519(b)(5). for new sentinel facilities.
device reporting
system as alternative
to universal user
facility reporting.
CDRH
Device user-facilities are FD&C Act Section Increase the number of Not applicable... FY 1998: 20,000
required to report adverse 519(b)(1). low-risk postmarket reports received
events. reports received and in summary form.
processed in summary
form. The total
number of summary
reports will be
increased from 20,000
in FY 98 to over
25,000 in FY 99. This
will be done by using
innovative
surveillance methods
and improving quality
and analysis needed
for Safety Alerts and
other actions..
CDRH
CFSAN.......................... ................. Work with CDC and Sentinel Sites Expand the
other federal expanded to demographic
agencies to develop provide better diversity and
baseline surveillance coverage of the size of the
data on foodborne representative population
illnesses required to areas of the covered by
evaluate the United States. FoodNet by
effectiveness of, set increasing the
better priorities number active
for, and determine surveillance
appropriate outcomes sites from 7 to
for the Food Safety 8. Begin
Initiative. implementation
of PulseNet,
which provides
data required to
do more rapid
and accurate
tracebacks to
determine the
causes of
foodborne
outbreaks.
CFSAN.......................... ................. By the end of FY 1999, Two releases in The requisite
improve public access FY 1997. hardware and
to timely information software systems
on adverse events need to be
related to dietary purchased for
supplements, infant integration of
formulas, and medical current Center-
foods by increasing based limited
the frequency of capability
public releases of systems.
information in the
Special Nutritionals
Adverse Events
Monitoring System
from two per year to
four per year.
CVM............................ ................. Assure that food Salmonella Salmonella
derived from animals isolates: 1,287 isolates: 2,000
and animal products human, 2,391 human, 3,000
is safe for human veterinary. veterinary.
consumption by
increasing the number
of human and animal
isolates in the NARMS
database.
----------------------------------------------------------------------------------------------------------------
FY 2000 Performance Goals are not identified in this Plan. Specification of these goals is dependent upon final
determination of the President's FY 2000 Budget Submission to Congress.
[[Page 65033]]
Objected D--Ensuring access to the scientific and technical
expertise needed by the Secretary--
1. Identification of Needs
FDA's ability to access the scientific and technical expertise
necessary to carry out its mission must be enhanced, i.e., improving
the science infrastructure, by upgrading the status of its facilities
and equipment; augmenting and targeting its science expertise toward
important new health enhancing technologies; and linking its science
information to external sources.
Upgrade Facilities and Equipment
FDA's current science capability, both internally generated and
externally coordinated, supports a wide range of risk management
activities, covering the life cycle of Agency-regulated products. The
integrity of the science base should be sustained by state-of-art
equipment and facilities, but at a minimum they must be in good repair.
The present status of this infrastructure, in many cases, is
considerably less than adequate. For instance, replacing the FDA's Los
Angeles laboratory and expanding the Arkansas regional facility will
provide the physical tools necessary to meet FDA's obligations.
Augment and Target Science Expertise
Although FDA's science efforts are supporting current efforts in
premarket review, postmarket safety assurance, and product use
monitoring, these programs are falling short of meeting the Agency's
statutory mandates and public expectations. As the programs are
enhanced to meet expectations, the Agency's access top state-of-the-art
science must be expanded. This will be accomplished both through
strategic recruitment of needed expertise and through creative
collaboration with outside institutions. Because FDA must regulate
increasingly complex products, the Agency's science capabilities must
be able to keep pace with new scientific developments. Further, the
science expertise must be positioned so that appropriate risk
assessments can be targeted toward emerging technologies that are
significant in protecting public health and which must reach the market
place quickly.
Link Science Information to External Sources
FDA must make strides in linking its science information bases to
external sources so that synergies can be realized and appropriate
information can be brought to bear on risk assessment and risk
management decisions promptly. If FDA does not enhance its ability to
link its science information with other outside sources, it will lose
comparability and communicability with these sources. Further, it will
not be as able to capitalize on cost-effective use of science
information to support regulatory decisions.
2. Stakeholder Views
Stakeholders strongly support the need for FDA maintaining a strong
and well-linked science base to support increasingly complex regulatory
judgments. A few illustrations of these views are indicated below:
``These needs to be a continuing strong commitment within
the Food and Drug Administration towards maintaining an appropriate
scientific base. It has been the experience of our member companies,
with numerous examples relating to both clinical development and
complex manufacturing issues, that these are speedily resolved because
of the scientific expertise within [FDA]. [trade association]
``Our company's long history in biotechnology has
repeatedly shown the value of active research scientists at [FDA].
[FDA's] personnel that are involved in research related to safety,
efficacy, basic biology, mechanism of action, and other associated
areas provide an important component for in-depth understanding of
issues and bring an understanding and response to issues in a
scientifically and regulatory responsible and appropriate manner.''
[industry representativ
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