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What Is the Purpose of the Orphan Drug Act?
Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.
Vyšlo v časopise: What Is the Purpose of the Orphan Drug Act?. PLoS Med 14(1): e32767. doi:10.1371/journal.pmed.1002191
Kategorie: Perspective
prolekare.web.journal.doi_sk: https://doi.org/10.1371/journal.pmed.1002191Souhrn
Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.
Zdroje
1. Orphan Drug Act of 1983. Pub L. No. 97–414, 96 Stat. 2049.
2. Lazonick W, Tulum Ö. US biopharmaceutical finance and the sustainability of the biotech business model. Research Policy. 2011 Nov;40(9):1170–87.
3. Silverman, Ed. FDA designated a record number of orphan drugs last year [Internet]. STAT. 2016 [cited 2016 Sep 12]. https://www.statnews.com/pharmalot/2016/02/11/fda-designates-record-number-of-orphan-drugs/
4. Kesselheim A, Treasure CL, Joffe S. Biomarker-defined subsets of common diseases: policy and economic implications of Orphan Drug Act coverage. PLoS Med. 2016;14(1):e1002190. doi: 10.1371/journal.pmed.1002190
5. Rai AK. Pharmacogenetic interventions, orphan drugs, and distributive justice: The role of cost-benefit analysis. Social Philosophy and Policy. 2002;19(02):246–70.
6. Loughnot D. Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses. Am JL & Med. 2005;31 : 365–80.
7. Meekings KN, Williams CSM, Arrowsmith JE. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discovery Today. 2012 Jul;17(13–14):660–4. doi: 10.1016/j.drudis.2012.02.005 22366309
8. Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7 : 74. doi: 10.1186/1750-1172-7-74 23013790
9. Fellows GK, Hollis A. Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach. Orphanet Journal of Rare Diseases. 2013;8 : 180. doi: 10.1186/1750-1172-8-180 24237605
10. Waxman HA. The history and development of the Orphan Drug Act. In: Scheinberg IH, Walsh JM, editors. Orphan Diseases and Orphan Drugs. Manchester University Press; 1986.
11. Orphan Drug Act of 1983, Pub. L. No. 97–414, 96 Stat 2049 (Jan. 4, 1983). § 526(a)(2).
12. Health Promotion and Disease Prevention Amendments of 1984, Pub. L. 98–551, 98 Stat 2815 (1984), § 4.
13. Simoens S. Pricing and reimbursement of orphan drugs: The need for more transparency. Orphanet Journal of Rare Diseases. 2011;6 : 42. doi: 10.1186/1750-1172-6-42 21682893
14. Wellman-Labadie O, Zhou Y. The US Orphan Drug Act: Rare disease research stimulator or commercial research opportunity? 2010;95(2):216–228. doi: 10.1016/j.healthpol.2009.12.001 20036435
15. Kesselheim AS. An Empirical Review of Major Legislation Affecting Drug Development: Past Experiences, Effects, and Unintended Consequences. Milbank Quarterly. 2011 Sep 1;89(3):450–502. doi: 10.1111/j.1468-0009.2011.00636.x 21933276
16. Orphan Drug Regulations (Final Rule) 57 Fed. Reg. 62,076 (Dec. 29, 1992).
17. Gibson S, Tigerstrom B von. Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada. J Law Biosci. 2015 Jul 13;2(2):263–91. doi: 10.1093/jlb/lsv013 27774196
18. Orphan Drug Regulations (Final Rule) 78 Fed. Reg. 35,117 (June 12, 2013).
19. Warren KS. The great neglected diseases of mankind, or All the world's an orphanage. In: Scheinberg IH, Walshe JM, editors. Orphan Diseases and Orphan Drugs. Manchester University Press; 1986.
20. Baylis F. Pregnant women deserve better. Nature. 2010 Jun 10;465(7299):689–90. doi: 10.1038/465689a 20535185
21. Kapczynski A, Syed T. The Continuum of Excludability and the Limits of Patents. Yale Law Journal. 2013;122(7):1900–63.
22. Trouiller P, Torreele E, Olliaro P, White N, Foster S, Wirth D, et al. Drugs for neglected diseases: a failure of the market and a public health failure? Trop Med Int Health. 2001 Nov;6(11):945–51. 11703850
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