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Principles of FDA Reform

February 13, 1996

A note from The field of medicine is constantly evolving. As a result, parts of this article may be outdated. Please keep this in mind, and be sure to visit other parts of our site for more recent information!

The Patient's Perspective

It is the duty of the Federal Government to regulate the safety and efficacy of drugs marketed in the United States. It exercises this duty under the authority of the Food, Drug and Cosmetic Act, as amended, through the Food and Drug Administration whose mission it is to insure that drugs, biological products and medical devices are safe and effective for the treatment of specific health conditions and that evidence relating to that safety and efficacy is accurately represented to the American people.

Basing their decisions regarding these products upon an intensive analysis of scientific data, the FDA strives to be a positive force in making safe and effective products available to the citizens of this country, focusing special attention on drugs to benefit those suffering from serious and lifethreatening diseases. In addition to ensuring the safety of the American people by the careful oversight of new products, the FDA protects our health by removing unsafe products and drugs from the market.

In fulfilling its role, the FDA promulgates regulations to improve public health, protecting Americans from unsafe, ineffective or impure drugs. The agency also helps to define cost-effective treatment strategies by establishing minimum efficacy standards through the drug regulation and approval process. It is important to remember that most drugs under FDA review are not breakthrough drugs that will have a major impact on public health. These drugs are given "standard" reviews that take more time than the "accelerated" reviews given to drugs for serious and lifethreatening diseases. It is the length of time for reviews of the former -- the standard review -- that is the pharmaceutical industry's primary concern. These drugs include antihypertensive, anti-inflammatory and antidepressant drugs.

The FDA should be applauded for its continued efforts to protect the American consumer through oversight of private industry's drug development, the assessment of health risks to Americans, and the acceleration of the drug approval process, with special emphasis on those drugs that may benefit those who suffer from serious and lifethreatening illnesses. As stated in the FDA's Vision For The Year 2000, the agency will continue to uphold safety and efficacy standards related to products on the market. In addition, FDA's mandate must encourage innovation and progress in medical research. In order to continue to perform its duties and attain these goals, however, the FDA must be given sufficient resources to handle its expanding responsibilities including monitoring the nation's food supply, expediting new drug approval, monitoring existing drugs and manufacturing facilities, and insuring the safety of medical devices, x-ray and mammography facilities, laboratories, food imports, and cosmetics. Most importantly, in regulating many of the core elements of consumers' lives, the FDA must operate at very high ethical standards, free from conflict of interest.

Finally, Industry must improve its performance by designing and implementing high quality research and submitting timely applications with supporting data that will facilitate efficient review and approval. The undersigned organizations ask that Congress join them in recognizing the central importance of the patient's voice in any discussion of potential reform of the FDA . The following principles outline some crucial concerns for patients in these discussions.

Principle 1:

  • The FDA must review drugs for serious and lifethreatening diseases differently than drugs for routine and treatable disease.

    Drugs for serious and lifethreatening diseases require different risk-benefit calculations. They should be reviewed more quickly and considered for marketing at the earliest possible time. Some people with cancer, Alzheimer's Disease, Parkinson's disease, or AIDS, for example, will accept the risks a drug might pose for the benefits it might provide.

    The FDA should take all steps necessary to ensure that effective new drugs are made available to patients with these conditions as soon in the development process as is practical.

Principle 2:

  • In order to assure the best possible care for patients with serious or life-threatening conditions, pre-approval access to experimental drugs that show reasonable safety and promise of efficacy should be encouraged.

    The FDA must balance premarket access with the need for sound scientific research, which benefits all present and future patients. Under current law, the FDA has pioneered parallel track and expanded access programs, which have helped thousands of people with serious diseases obtain experimental drugs. While the FDA should encourage manufacturers to provide early access to promising therapies, the agency cannot compel the industry to do so. Industry initiative is required for the success of such programs.

Principle 3:

  • Marketing approval is one point on the continuum of drug development. Especially in the context of drugs for serious or lifethreatening diseases, which may receive approval based on preliminary efficacy data, post-marketing research is critical.

    The data required by the FDA for marketing approval is the foundation upon which present and future doctors and patients decide how, when, and whether to use any drug. The FDA must develop a post-marketing follow-up system bolstered by clearly defined enforcement powers.

Principle 4:

  • While validation and third-party reimbursement of nonFDA approved ("off label") uses of drugs are of critical importance to people with serious or lifethreatening diseases, the promotion or validation of such uses must not entail a reduction in efficacy standards.

    FDA-required safety and efficacy data that form the basis for initial approval cannot provide a full picture of a drug's usefulness. Additional uses for drugs, discovered through future research, may be appropriate for some patients as determined by medical experts and as prescribed in accepted medical compendia and journals. However, such uses are frequently not reimbursed by third-party payers based on their lack of FDA approval.

    While the Department of Health and Human Services should accept the responsibility to establish medical practice standards and third-party reimbursement guidelines, it would be a mistake to focus such mechanisms solely in the context of FDA reform. While the FDA should encourage sponsors to submit supplemental approval applications, research to validate "off-label" uses remains the responsibility of industry, government and academia.

Principle 5:

  • The FDA must retain regulatory flexibility.

    FDA regulation must remain flexible and must not be obstructive if the agency is to efficiently pursue the goals of ensuring the safety and efficacy of those products under its jurisdiction. Legislative changes to the FDA's authority, therefore, must not restrict the agency's ability to evaluate each drug's unique safety and efficacy profile.

Principle 6:

  • The FDA should ensure that applications for new drugs include data on all populations likely to use such drugs.

    The FDA should require data on all relevant populations; currently, drugs are often approved with little or no information on their use for women, racial minorities, older Americans or children.

Principle 7:

  • The FDA must be responsive to the concerns of patient groups, industry, physicians and the American people.

    The FDA must develop a consistent approach to drug regulation across all its centers and divisions. To remain responsive, the FDA should solicit nonscientist patient representatives to serve on advisory committees. The unique dynamics of intellectual property rights for drugs, which constitute a critical component of the health care system, must be recognized. In the context of reviewing applications for marketing approvals, the FDA must be allowed to balance its responsibility to protect industry trade secrets against the need for public disclosure.

Principle 8:

  • There must be close communication between the FDA and industry during all stages of clinical drug development.

    Drug development and regulation proceeds best and most efficiently when industry and the FDA work together from the early stages of research. Communication should be enhanced between the agency, companies, physicians and the public.

Principle 9:

  • Robust pharmaceutical research is critical to the development of drugs to treat serious or lifethreatening diseases. Where appropriate, incentives to encourage industry, academic or government-sponsored research should be considered.

    Incentives might incorporate a variety of mechanisms, including market exclusivity or regulatory relief for breakthrough drugs.

AIDS Action Council
AIDS Policy Center for Children, Youth and Families
Alzheimer's Association
American Cancer Society
American Foundation for AIDS Research
American Health Information Management Association
American Laryngeal Papilloma Foundation
American Social Health Association
Arthritis Foundation
Association of Schools of Public Health
Brain Injury Association
Center For Research in Sleep Disorders
Critical Path AIDS Project
Cushing Support & Research Foundation
Foundation for Icthiosis and Related Skin Types
Gay Men's Health Crisis
Guillain Barre Syndrome Foundation International
Help Hospitalized Children's Fund
Hemochromatosis Foundation, Inc.
Histiocytosis Association of America
Human Rights Campaign
International Patient Advocacy Association
International Tremor Foundation
Late Onset Tay-Sachs Foundation
Mobilization Against AIDS
MPS Society
Narcolepsy Network
National AIDS Fund
National Association of Developmental Disabilities Councils
National Association of People With AIDS
National Association of Protection and Advocacy Systems
National Catholic AIDS Network
National Episcopal AIDS Coalition
National Foundation for Ectodermal Dysplasias
National Health Care for The Homeless Council
National Health Council
National Hemophilia Foundation
National Latino/Latina Lesbian and Gay Organization
National Mental Health Association
National Minority AIDS Council
National Network for Youth
National Organization for Rare Disorders
National Puerto Rican Coalition
National Tuberous Sclerosis Association
National Sjogren's Syndrome Association
Osteogenesis Imperfecta Foundation
Pediatric AIDS Foundation
Prader-Willi Syndrome Association
Search For A Cure
Sickle Cell Disease Association
Sturge-Weber Foundation
Tourette Syndrome Association, Inc.
Treatment Action Group
United Leukodystrophy Foundation
United Patients' Association for Pulmonary Hypertension
United Parkinson Foundation
Wilson's Disease Association

For more information, contact
AIDS Action Council
Gary Rose
1875 Connecticut Avenue NW #700
Washington DC 20009
202-986-1345 (fax)
202-332-9614 (tty)

A note from The field of medicine is constantly evolving. As a result, parts of this article may be outdated. Please keep this in mind, and be sure to visit other parts of our site for more recent information!

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This article was provided by AIDS Action Council.
See Also
More on the FDA Drug Approval Process and Other Regulatory Issues