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Evaluation of Orphan Products by the U.S. Food and Drug Administration

Published online by Cambridge University Press:  10 March 2009

Marlene E. Haffner  and
John V. Kelsey
Marlene E. Haffner
Affiliation:
U.S. Food and Drug Administration
John V. Kelsey
Affiliation:
U.S. Food and Drug Administration
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Abstract

Orphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

Type
Special Section: Orphan Technologies
Information
International Journal of Technology Assessment in Health Care , Volume 8 , Issue 4 , Fall 1992 , pp. 647 - 657
Copyright
Copyright © Cambridge University Press 1992

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References

REFERENCES

1.21 CFR 312.80–312.88. Vol. 5, 1990.Google Scholar
2.Food and Drug Administration. Regulatory procedures manual, part 9–71. Bethesda, MD: Food and Drug Administration, 1989.Google Scholar
3.Food and Drug Law Institute. Future regulation of foods, drugs, devices and cosmetics. Washington, DC: Hill and Knowlton, Inc., Institute for Alternative Futures, The Wirthlin Group, 1988.Google Scholar
4.Freidman, M., et al. Group C drugs: Experience of the Division of Cancer Treatment. Bethesda, MD: National Cancer Institute, n.d.Google Scholar
5.Meinert, C. L.Clinical trials, design, conduct and analysis. New York: Oxford University Press, 1986.Google Scholar
6.Minamida, Y.Good Drugs That Have Been Abandoned: Orphan Drugs in Japan (Drugs for Rare Diseases) in Japanese. Osaka: Medical Review Co., 1990.Google Scholar
7.Orphan Drug Act, P.L. 97–414, 1983.Google Scholar
8.Safe Medical Devices Act of 1990, P.L. 101–629, 1990.Google Scholar
9.21 USC 505(d), Federal Food, Drug, and Cosmetic Act, Chapter 5, 1938.Google Scholar
10.U.S. Congress, House of Representatives. Committee report on H.R. 5238. 97th Congress, 2nd Session, 1982.Google Scholar
11. U.S. Department of Health and Human Services, Public Health Service, Office of the Assistant Secretary for Health. Report of the National Committee of Orphan Diseases. 02 1989.Google Scholar
12.U.S. Department of Health and Human Services. PHS response to the report of the National Commission on Orphan Diseases. 07 1990.Google Scholar
13.U.S. Vice President's Council on Competitiveness. Council on Competitiveness fact sheet— Improving the nation's drug approval process. Washington, DC, 1991.Google Scholar