414:(sometimes referred to as the author of the Act), chairman of the Energy and Commerce Subcommittee on Health. It passed the House of Representatives on 14 December 1982, and was similarly approved by voice vote in the Senate on 17 December. On 4 January 1983, President Ronald Reagan signed the ODA into law. Under the ODA drugs, vaccines, and diagnostic agents would qualify for orphan status if they were intended to treat a disease affecting less than 200,000 American citizens. In order to encourage the development of drugs for orphan diseases, the ODA included a number of incentives including seven-year market exclusivity for companies that developed orphan drug, tax credits equal to half of the development costs, later changed to a fifteen-year carry-forward provision and a three-year carry-back that can be applied in profitable year, grants for drug development, fast-track approvals of drugs indicated for rare diseases, and expanded access to the
38:
333:
developing new medicines. Pharmaceutical companies responded by focusing on developing treatments for common diseases in order to maximize the possibility of recouping research and development costs and generating significant profits. As a result, rare diseases were largely ignored due to poor economic potential and were thus said to be "orphaned." The gap between drugs for common versus rare diseases eventually widened to the point where few or no treatments were available for some rare conditions such as
464:
stimulated the production of truly non-profitable drugs; the act also received some criticism for allowing some pharmaceutical companies to make a large profit off of drugs that have a small market but still sell for a high price. While orphan drug status is given to drugs with "no reasonable expectation" of profitability, some orphan drugs have gone on to net large profits and/or receive widespread use. The topic of profit in the aftermath of the ODA was addressed in
November 2013 in the
426:
current patent status. Furthermore, if a market competitor wishes to introduce a drug for the same indication, the onus is on the competitor to prove that their drug is therapeutically superior (e.g. increased efficacy, less toxicity, etc.) when compared to the present drug indicated for the rare disease of interest. This incentive creates an attractive monopolistic market for companies interested in developing a product for any given rare disease.
456:
the Office of Orphan
Products Development (OOPD) and 249 orphan drugs have received marketing authorization. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds. As of 2010, 200 of the roughly 7,000 officially designated orphan diseases have become treatable. In 2010, drugmaker
370:, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be affected by the disease in question.
332:
was passed in 1962 as an amendment to the
Federal Food, Drug, and Cosmetic Act. Kefauver-Harris required that all drugs approved for sale be proven safe and effective via rigorous scientific studies. While this legislation improved drug safety, it also dramatically increased the costs associated with
455:
Drug companies nearly universally believe the ODA to be a success. Before
Congress enacted the ODA in 1983 only 38 drugs were approved in the USA specifically to treat orphan diseases. In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by
310:
to pass the Orphan Drug Act (ODA) in early 1983. Only thirty-eight orphan drugs had been approved prior to the 1983 Act; by 2014 "468 indication designations covering 373 drugs have been approved." Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the
European Union
1075:
In an attempt to simplify the process for obtaining orphan status for medications targeting rare diseases, the FDA and the
European Medicines Agency (EMA) have created a common application. ... U.S. and European regulators still will conduct independent reviews of application submissions to ensure
425:
Market exclusivity is particularly appealing to pharmaceutical firms as an incentive to pursue orphan drug development. The seven-year market exclusivity period differs from traditional patent law in that it does not begin until the drug is granted FDA approval and is independent of the drug's
463:
Some critics have questioned whether orphan drug legislation was the real cause of this increase (claiming that many of the new drugs were for disorders that were already being researched anyway, and would have had drugs developed regardless of the legislation), and whether the ODA has really
373:
Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable, government intervention is often required to motivate a manufacturer to address the need for an orphan drug.
406:
The plight of patients with rare diseases became an important political issue in the late 1970s and early 1980s. The US government was subject to pressure from activist groups such as NORD and many others.
290:
Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States. That process is handled through other offices in the US
569:
210:
460:
established a division to focus specifically on the development of orphan drugs as other large pharmaceutical companies focused greater efforts on the orphan drug research.
502:(EMA) agreed in late 2007 to utilize a common application process for both agencies. However, the two agencies will continue to maintain separate approval processes.
1123:
295:. Instead, the designation means only that the sponsor qualifies for certain benefits from the federal government, such as market exclusivity and reduced taxes.
202:
48:
An Act to amend the
Federal Food, Drug, and Cosmetic Act to facilitate the development of drugs for rare diseases and conditions, and for other purposes.
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The industry has taken advantage of the incentives to charge excessive profits and to reap windfalls far in excess of their investments in the drug.
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for helping the ODA pass in the USA: "Seldom Silent, Never Heard" (1981) and "Give Me Your Weak" (1982). The show's star,
785:"Quincy, M.E.: Seldom Silent, Never Heard (1981) - Jeffrey Hayden; Synopsis, Characteristics, Moods, Themes and Related"
358:
generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on
152:
137:
124:
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627:
999:
Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J (February 21, 2012). Alessi-Severini S (ed.).
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the data submitted meet the legal and scientific requirements of their respective jurisdictions, the agencies said.
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95:
87:
17:
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814:"Quincy, M.E.: Give Me Your Weak (1982) - Georg Fenady; Synopsis, Characteristics, Moods, Themes and Related"
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329:
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In an effort to reduce the burden on manufacturers applying for orphan drug status, the FDA and the
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In 1982 an informal coalition of supporters and families of patients with rare diseases who formed
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813:
377:
The intervention by government on behalf of orphan drug development can take a variety of forms:
268:
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180:
809:
780:
434:
718:"Orphan Drug Policies: Implications for the United States, Canada, and Developing Countries"
37:
1012:
906:
Rich Daly (5 September 2002). "House Offers
Incentives For Development of 'Orphan' Drugs".
8:
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302:(NORD) and others, called for change to legislation to support development of
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Creating a government-run enterprise to engage in research and development.
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1118:. Washington, D.C.: National Archives and Records Service. pp. 9–10.
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which affect small numbers of individuals residing in the United States.
27:
Law passed in the United States to facilitate development of orphan drugs
1001:"The prevalence and cost of unapproved uses of top-selling orphan drugs"
99:
192:
716:
Cheung, Richard Y.; Cohen, Jillian; Illingworth, Patricia (Jan 2004).
566:"Abbey Meyers, President of NORD, announces her retirement (July 07)"
306:, or drugs for treating rare diseases. They succeeded in getting the
272:
923:"Bookmark and Share Pfizer creates orphan disease research division"
230:
on
December 14, 1982 (agreed voice vote) with further amendment
487:
447:, even testified before Congress concerning the orphan drug issue.
430:
1092:
The Orphan Drug Act and
Catalyst Pharmaceuticals, Inc., v. Becerra
418:. The law was also later amended to waive user fees charged under
259:
is a law passed in the United States to facilitate development of
998:
457:
388:
367:
419:
1112:"Statement on Signing the Orphan Drug Act - January 4, 1983"
864:
Jack Klugman calls it quits after seven seasons as 'Quincy'
769:(paper). Vol. 31, no. 12. Dec 2013. p. 1062.
224:
on October 1, 1982 (passed voice vote) with amendment
381:
276:
715:
1126:. U.S. Food and Drug Administration. 20 October 2020.
676:
674:
490:
was an orphan drug and went on to be a blockbuster.
595:"Millions Around World to Observe Rare Disease Day"
324:In response to incidents such as difficulties with
891:Armstrong, Walter (May 2010). "Pharma's Orphans".
671:
890:
559:
557:
555:
1138:
1060:"U.S., EU Will Use Same Orphan Drug Application"
563:
319:
975:"Drug earning millions despite 'orphan' label"
773:
552:
437:credits two episodes of the television series
410:The chief sponsor of the bill (H.R. 5238) was
218:on September 28, 1982 (passed voice vote)
680:
623:Orphan Drug Law Matures into Medical Mainstay
236:on December 17, 1982 (agreed voice vote)
886:
884:
802:
1124:"FDA Office of Orphan Products Development"
1057:
493:
949:"Big Pharma Reassesses Orphan Drug Sector"
1051:
1034:
1024:
905:
881:
728:. Canada: Health Law Institute: 183–200.
1157:United States federal health legislation
570:European Organization for Rare Disorders
300:National Organization for Rare Disorders
972:
681:Hadjivasiliou, Andreas (October 2014),
587:
14:
1139:
1109:
946:
920:
619:
564:Parisse-Brassens, Jerome (June 2007).
1110:Reagan, Ronald W. (January 4, 1983).
908:Congressional Quarterly Daily Monitor
711:
709:
707:
705:
613:
876:Klugman winds up his 'Quincy' career
866:, St. Petersburg Times - Jul 3, 1983
831:
808:
779:
752:
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470:where the following quote appeared:
115:Federal Food, Drug, and Cosmetic Act
24:
1084:
973:Schulte, Fred (18 December 2007).
702:
523:
25:
1183:
1103:
947:Conway, Benjamin (1 March 2010).
878:, The Deseret News - Mar 23, 1983
747:
628:U.S. Food and Drug Administration
1172:Drug policy of the United States
450:
416:Investigational New Drug Program
391:protection and marketing rights.
234:Senate agreed to House amendment
228:House agreed to Senate amendment
211:Senate Labor and Human Resources
36:
992:
966:
940:
914:
899:
869:
857:
538:US Food and Drug Administration
1096:Congressional Research Service
921:Grogan, Kevin (15 June 2010).
401:
13:
1:
820:. All Media Network. Overview
791:. All Media Network. Overview
517:
344:
314:
1167:Food and Drug Administration
1026:10.1371/journal.pone.0031894
482:, primary sponsor of the ODA
394:Clinical research subsidies.
320:Emergence of orphan diseases
293:Food and Drug Administration
7:
1152:97th United States Congress
1131:99% Invisible: Orphan Drugs
839:"Episode 329: Orphan Drugs"
505:
58:97th United States Congress
10:
1188:
1058:Donna Young (2007-11-28).
348:
736:– via ResearchGate.
683:"Orphan Drug Report 2014"
531:"Orphan Drug Act of 1983"
512:Rare Diseases Act of 2002
500:European Medicines Agency
429:Television historian and
330:Kefauver-Harris Amendment
203:House Energy and Commerce
172:
163:
148:
130:
125:21 U.S.C.: Food and Drugs
120:
110:
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71:
63:
52:
44:
35:
893:Pharmaceutical Executive
494:Regulatory harmonization
1098:. Aug 17, 2023. R47653.
257:Orphan Drug Act of 1983
199:Committee consideration
149:U.S.C. sections amended
31:Orphan Drug Act of 1983
1162:Pharmaceuticals policy
485:
308:United States Congress
195:) on December 15, 1981
741:registration required
620:Henkel, John (1999).
472:
1147:1983 in American law
766:Nature Biotechnology
269:Huntington's disease
207:House Ways and Means
1017:2012PLoSO...731894K
953:Wall Street BioBeat
166:Legislative history
142:ch. 9, subch. V
32:
845:. 13 November 2018
722:Health Law Journal
601:. 13 February 2009
285:muscular dystrophy
246:on January 4, 1983
30:
637:978-1-4223-2677-0
281:Tourette syndrome
253:
252:
222:Passed the Senate
89:Statutes at Large
16:(Redirected from
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1119:
1116:Internet Archive
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1079:
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758:"IN their words"
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572:. Archived from
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550:
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540:. 4 January 1983
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527:
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364:pharmacodynamics
360:pharmacokinetics
339:Hansen's disease
216:Passed the House
179:in the House as
168:
134:sections created
90:
40:
33:
29:
21:
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1085:Further reading
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335:Crohn's disease
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240:Signed into law
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67:January 4, 1983
53:Enacted by
28:
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22:
18:Orphan Drug Act
15:
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1066:. Washington
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982:. Retrieved
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927:Pharma Times
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693:, retrieved
689:
641:. Retrieved
622:
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603:. Retrieved
589:
578:. Retrieved
574:the original
542:. Retrieved
537:
525:
497:
486:
480:Henry Waxman
473:
465:
462:
454:
445:Jack Klugman
440:Quincy, M.E.
438:
435:Hal Erickson
433:contributor
428:
424:
412:Henry Waxman
409:
405:
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356:Orphan drugs
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304:orphan drugs
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261:orphan drugs
256:
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185:Henry Waxman
176:
111:Acts amended
106:Codification
1133:, (podcast)
849:18 November
661:|work=
643:14 February
605:14 February
599:PR Newswire
402:Legislation
384:incentives.
351:Orphan drug
326:thalidomide
263:—drugs for
1141:Categories
1094:(Report).
1070:2008-01-06
824:2010-06-07
795:2010-06-07
580:2024-04-10
544:27 October
518:References
345:Key issues
315:Background
177:Introduced
77:Public law
45:Long title
663:ignored (
653:cite book
387:Enhanced
311:in 2000.
273:myoclonus
153:21 U.S.C.
138:21 U.S.C.
72:Citations
64:Effective
1045:22363762
1005:PLOS ONE
984:28 March
958:28 March
932:28 March
818:AllMovie
789:AllMovie
763:. News.
734:16539081
506:See also
488:Provigil
477:—
431:Allmovie
267:such as
94:96
1036:3283698
1013:Bibcode
695:28 June
341:, etc.
1043:
1033:
732:
634:
458:Pfizer
389:patent
368:dosing
132:U.S.C.
98:
81:97-414
761:(PDF)
686:(PDF)
534:(PDF)
420:PDUFA
157:ch. 9
155:
140:
96:Stat.
1041:PMID
986:2011
960:2011
934:2011
851:2018
730:PMID
697:2015
665:help
645:2009
632:ISBN
607:2009
546:2015
362:and
328:the
255:The
100:2049
56:the
1031:PMC
1021:doi
382:Tax
283:or
277:ALS
201:by
183:by
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