Hostname: page-component-586b7cd67f-gb8f7 Total loading time: 0 Render date: 2024-11-28T23:48:37.148Z Has data issue: false hasContentIssue false
  • English
  • Français

Recombinant Erythropoietin: Orphan Product with a Silver Spoon

Published online by Cambridge University Press:  10 March 2009

John M. Coster
John M. Coster
Affiliation:
U.S. Senate Special Committee on Aging
Get access Rights & Permissions [Opens in a new window]

Abstract

The U.S. Food and Drug Administration's (FDA) approval of the orphan biological product recombinant erythropoietin (rEPO) in June 1989 resulted both in a breakthrough treatment for the chronic anemia of people who suffer from chronic renal failure and a powerful argument for change in the legislation that spawned its development: the Orphan Drug Act of 1983. At a cost of over $6,000 per patient per year, Congress could not understand how a product that no manufacturer wanted to produce was suddenly costing the federal government hundreds of millions of dollars each year. Congress attempted to change the act in 1990 to preclude a manufacturer from using its provisions to secure lucrative monopolies in certain drug markets. In early 1991, the FDA finally issued regulations to implement the act that addressed some of the very concerns that were caused by rEPO.

Type
Special Section: Orphan Technologies
Information
International Journal of Technology Assessment in Health Care , Volume 8 , Issue 4 , Fall 1992 , pp. 635 - 646
Copyright
Copyright © Cambridge University Press 1992

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1.Amgen lnc. vs. Chugai Pharmaceuticals, Ltd. and Genetics Institute, Inc. Civil Action 87 2617-Y, 12 11, 1989.Google Scholar
2.Evans, R., Manninen, D., & Garrison, L.The quality of life of patients with end stage renal disease. New England Journal of Medicine, 1985, 312, 553–59.CrossRefGoogle ScholarPubMed
3.Meyers, A. S., Executive Director, National Organization of Rare Disorders, Inc., New Fair field, CT. Statement: The Orphan Drug Act, drug pricing, competition and reauthorization. Hearing before the Subcommittee on Health and the Environment, Committee on Energy and Commerce, House of Representatives, U.S. Congress, Washington, DC, 02 7, 1990.Google Scholar
4.Miyake, T., Kung, C. K.-H., & Goldwasser, E.Purification of human erythropoietin. Journal of Biological Chemistry, 1977, 252, 5558–64.Google Scholar
5.Reissman, K.Studies on the mechanism of erythropoietic stimulation in parabiotic rats during hypoxia. Blood, 1950, 5, 372–80.Google Scholar
6.Stohlman, F., Rath, C, & Rose, J.Evidence for humoral regulation of erythropoiesis: Studies on a patient with polycythemia secondary to regional hypoxia. Blood, 1954, 9, 721–33.Google Scholar
7.United States Congress. Office of Technology Assessment. New developments in biotechnology: U.S. investment—special report. OTA-BA-360. Springfield, VA: National Technical Information Service, 07 1988.Google Scholar
8.United States Congress. Office of Technology Assessment. Recombinant erythropoietin: Payment options for Medicare—Special report. OTA-H-451. Washington, DC: 05 1990.Google Scholar
9.United States Department of Health and Human Services, Health Care Financing Administration. End stage renal disease quarterly statistical summary. Baltimore, MD: 11 1989.Google Scholar
10.United States Department of Health and Human Services, Public Health Service, Food and Drug Administration. Summary basis of approval for epoetin-alfa. ELA #87–0535, PLA #87–0536. Washington, DC: 06 1989.Google Scholar
11.Unites-States House of Representatives. Report of the Subcommittee on Health and the Environment. Report 101–635, Orphan Drug Amendments of 1990. Washington, DC: 07 1990.Google Scholar
12.United States International Trade Commission. In the matter of recombinant erythropoietin, investigation 337-TA-281. 01 1989.Google Scholar
13.United States Securities and Exchange Commission. 10K report for Amgen for the fiscal year ending March 1988. Washington, DC: #88157399, 06 29, 1988.Google Scholar