The US Orphan Drug Programme 1983-1995

PharmacoEconomics

Volumn 12, Issue 3, 1997, Pages 312-326

  Shulman, Sheila R ^a   Manocchia, Michael ^b  

^a TUFTS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b TUFTS MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA2A INTERFERON; BETA INTERFERON; BOTULINUM TOXIN; CARNITINE; ORPHAN DRUG; RECOMBINANT ERYTHROPOIETIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; SOMATOSTATIN; THALIDOMIDE; TRIMETREXATE;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; BIOTECHNOLOGY; CANCER; DRUG DEVELOPMENT; DRUG INDICATION; DRUG LEGISLATION; DRUG USE; FOOD AND DRUG ADMINISTRATION; GENETIC DISORDER; INFECTION; MARKETING; PRESCRIPTION; PRIORITY JOURNAL; REVIEW;

EID: 0030882809     PISSN: 11707690     EISSN: None     Source Type: Journal    
DOI: 10.2165/00019053-199712030-00004     Document Type: Review

References (51)

1. Orphan Drug Act. Public Law No. 97-414, 96 Stat 2049 (1983)
2. Uchida K. Orphan drugs in Japan. Drug Inf J 1996; 30 (1): 171-5
3. Fracchia GN, Haavisto KH. Orphan drugs: European Union priorities for research and regulatory actions. Cambridge: European Conference Pubs., 1997: 1-6
4. EU orphan medicinal products. Scrip 1996 Sep 20: 9 (2165): 6
5. Orphan Drug Act. Report from the Committee on Energy and Commerce. Report no.: 97-840, Part 1. 97th Congress, 2nd Session (Serial set #13490), US House of Representatives: 1982 Sep 17; Washington D.C., 6
6. Asbury CH. The Orphan Drug Act: the first 7 years. JAMA 1991; 265 (7): 893-7
7. Shulman SR, Bienz-Tadmor B, Seo PS, et al. Implementation of the Orphan Drug Act: 1983-1991. Food and Drug Law J 1992; 47 (4): 363-403
8. Davidson S. An orphan drug worth $250 million? Nature Biotechnology 1996; 14 (4): 420
9. Orphan drugs. 21 Code of Federal Regulations (CFR) 316, 316.2(b)(6)
10. Gosse ME, Manocchia M. First biologicals approved in the U.S.: 1980-1994. Drug Inf J 1996; 30 (4): 991-1001
11. Testimony of John McLaughlin, General Counsel, Genentech, Inc., before the Subcommittee on Health and the Environment of the House Committee on Energy and Commerce. 101st US Congress, 2nd Sess.; 1990 Feb 7
12. Davidson S. Orphan drugs: European biotechnology waits for EC act. Nature Biotechnology 1996; 14 (4): 419
13. The Biotechnology Process Patent Protection Act, PL 1044, Nov 1, 1995
14. Utility examination guidelines. 60 Federal Register 1995 Jul 14: 36263
15. Orphan drugs. Syndicat National de l'Industrie Pharmaceutique (SNIP) Paris: Nov 1995
16. 21 Code of Federal Regulations (CFR) 316.2(c)
17. 21 Code of Federal Rgulations (CFR) 316.2(b)(8)(i),(ii)
18. Data provided to Tufts Center for the Study of Drug Development by Robert Steeves, U.S. Food and Drug Administration Orphan Drug Office, 10 Apr 1996
19. Statement by Michael Friedman, Deputy Commissioner for Operations, Food and Drug Administration, Department of Health and Human Services. Before the Subcommittee on Human Resources and Intergovernmental Relations, Committee on Government Reform and Oversight, U.S. House of Representatives: 12 Sep 1996
20. Small Business Job Protection Act of 1996. P.L. 104-88, Sec. 1205, 20 Aug 1996
21. 21 Code of Federal Regulations (CFR) 316, 316.3(b)(12,13)
22. Treatment use of an investigational new drug. 21 CFR 316.34
23. Drugs intended to treat life-threatening and severely-debilitating illnesses. 21 Code of Federal Regulations (CFR) 312.8
24. Accelerated approval of new drugs for serious or life-threatening illnesses. 21 Code of Federal Regulations (CFR) 314.5
25. Accelerated approval of biological products for serious or life-threatening illnesses. 21 CFR 601.4
26. Shulman SR, Brown JS. The Food and Drug Administration's early access and fast-track approval initiatives: how have they worked? Food Drug Law J 1995; 50 (4): 503-32
27. Prescription Drug User Fee Act of 1992. Pub Law 102-571 (1992 Oct 29); 21 USC 379; 106 Stat 4491
28. Interim guidance document for waivers and reductions in user fees. Rockville (MD): Food and Drug Administration, 1993
29. Shulman SR, Kaitin KI. The Prescription Drug User Fee Act of 1992: a five-year experiment for industry and the FDA. Pharmacoeconomics 1996 Feb; 9 (2): 121-33
30. List of orphan product designations and approvals. Rockville (MD): Office of Orphan Products Development, Food and Drug Administration, Jan 1983-Dec 1995
31. Tufts CSDD Biopharmaceutical Database and Tufts Early Access and Fast-track Database. Boston: Tufts Center for the Study of Drug Development Investigational and Marketed NCE Databases
32. Prescription Drug User Fee Act of 1992: Waivers and reductions; small business exception. Report no.: 3 (July 16, 1993 through July 16, 1996). Rockville (MD): Office of the Chief Mediator and Ombudsman, Food and Drug Administration, 1996
33. 21 Code of Federal Regulations (CFR) 316.2(a), 316.23(b)
34. Smith Jr RC, editor. Medical & health marketplace guide, 5th and 6th ed. New York: Investment Dealers' Digest (IDD), 1989 and 1990
35. Smith Jr RC, editor. Medical & health marketplace guide. 11th ed. New York: Investment Dealers' Digest (IDD), 1995
36. BioScan. Vol. 9. Phoenix: Oryx Press, 1995
37. Centers fur Disease Control and Prevention. 1993 revised classification system for HIV infection and expanded surveillance case definition for AIDS among adolescents and adults. JAMA 1993; 269 (6): 729-30
38. Haffner ME. Orphan products: ten years later and then some. Food and Drug Law J 1994; 49 (4): 593-601
39. Kaitin KI, Manocchia M, Seibring M, et al. The new drug approvals of 1990, 1991, and 1992: trends in drug development. J Clin Pharmacol 1994; 34 (2): 120-7
40. Kaitin KI Manocchia MA. The new drug approvals of 1993, 1994, and 1995: trends in drug development. Am J Ther. In press
41. DiMasi JA, Seibring MA, Lasagna L. New drug development in the United States 1963 to 1992. Clin Pharmacol Ther 1994; 55 (6): 609-22
42. Genentech Inc. v. Bowen, 676 Federal Supplement 301 (D.D.C. 1987).
43. 21 Code of Federal Regulations (CFR) 316
44. 21 Code of Federal Regulations (CFR) 316.3(b)(3)(i-iii)
45. Orphan drug regulations. 57 Federal Register 1992 Dec 29: 62076
46. Berlex Laboratories, Inc. v. Food and Drug Administration, et al. Civil No. 96-0971, US District Court for the District of Columbia, 1996 Apr 26
47. Berlex sues Biogen over beta-interteron. Scrip 1996 Jul 16; 7 (2146): 8
48. Berlex Laboratories, Inc. v Food and Drug Administration, et al. Civil No. 96-0971, U.S. District Court for the District of Columbia (1996 WI. 590649 D.D.C.) 1996 Oct 7
49. Van Brunt J. Early sales predict some big winners in the class of 1996. Bio World Financial Watch 1996 Nov 25; 4 (48): 1
50. Institute of Medicine (U.S.). Committee to Study Medication Development and Research at the National Institute on Drug Abuse. In: Fulco CE, Liverman CT, Earley LE, editors. Development of medications for the treatment of opiate and cocaine addictions: issues for the government and private sector. Washington, DC: National Academy Press, 1995
51. Ludlam C, Beier D. Tax incentives/disincentives: maximizing AIDS drug research by the biotechnology industry. A Joint Meeting of the Public/Private Issues and Drug Development Subcommittee of the National Task Force on AIDS Drug Development. Biotechnology Industry Organization; 1995 Apr 25; Washington, DC