Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage

Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.

Vyšlo v časopise: Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage. PLoS Med 14(1): e32767. doi:10.1371/journal.pmed.1002190
Kategorie: Policy Forum
prolekare.web.journal.doi_sk: https://doi.org/10.1371/journal.pmed.1002190

Souhrn

Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.

Zdroje

1. Orphan Drug Act of 1983. 21 U.S.C. 360bb(a)(2) (2008). https://www.gpo.gov/fdsys/pkg/STATUTE-96/pdf/STATUTE-96-Pg2049.pdf Accessed 2 Sept 2016.

2. Reardon S. Regulators adopt more orphan drugs. Nature. 2014;508:16–17. doi: 10.1038/508016a 24695293

3. Field MJ, Boat TF. Committee on Accelerating Rare Diseases Research and Orphan Product Development Board on Health Sciences Policy. Washington, DC: National Academies Press. 2010.

4. Herder M. When everyone is an orphan: against adopting a U.S.-styled orphan drug policy in Canada. Account Res. 2013;20(4):227–69. doi: 10.1080/08989621.2013.793120 23805831

5. Daniel MG, Pawlik TM, Fader AN, Esnaola NF, Makary MA. The Orphan Drug Act: Restoring the Mission to Rare Diseases. Am J Clin Oncol 2015;00(00):1. http://content.wkhealth.com/linkback/openurl?sid=WKPTLP:landingpage&an=00000421-900000000-99097

6. Food and Drug Administration. Drugs@FDA: FDA approved drug products. Drugs@FDA. 2016. http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.DrugDetails

7. Food and Drug Administration. Fast track, breakthrough therapy, accelerated approval and priority review. Sept 14, 2015. http://www.fda.gov/forpatients/approvals/fast/ucm20041766.htm

8. Darrow JJ, Kesselheim AS. Drug Development and FDA Approval, 1938–2013. N Engl J Med 2014;370(26).

9. Conti R, Bernstein A, Villaflor V, Schilsky R, Rosenthal M, Bach P. Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists. J Clin Oncol. 2013;31(9):1134–9. doi: 10.1200/JCO.2012.42.7252 23423747

10. Memorial Sloan Kettering Cancer Center Abacus Database. 2015. http://app.drugabacus.org/abacus-mskcc

11. Nocera J. The $300,000 Drug. The New York Times. July 19, 2014. http://www.nytimes.com/2014/07/19/opinion/joe-nocera-cystic-fibrosis-drug-price.html?_r=0

12. Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J. The prevalence and cost of unapproved uses of top-selling orphan drugs. PLoS ONE. 2012;7(2):1–7.

13. Faden L, Huskamp H. Medicare Part D Coverage and Reimbursement of Orphan Drugs. National Academies Press; 2010.

14. Gupta SK, Nayak RP. Off-label use of medicine: Perspective of physicians, patients, pharmaceutical companies and regulatory authorities. Journal of Pharmacology & Pharmacotherapeutics. 2014;5(2):88–92.

15. Casali PG. The off-label use of drugs in oncology: a position paper by the European Society for Medical Oncology (ESMO). Ann Oncol. 2007;18(12):1923–1925. doi: 10.1093/annonc/mdm517 18083693

16. Fugh-Berman A, Melnick D. Off-label promotion, on-target sales. PLoS Med 2008;5(10):e210. doi: 10.1371/journal.pmed.0050210 18959472

17. Wellman-Labadie O, Zhou Y. The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? Health Policy 2010;95(2–3):216–28. doi: 10.1016/j.healthpol.2009.12.001 20036435

18. Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA. 2011;305(22):2320–6. doi: 10.1001/jama.2011.769 21642684

19. Mitsumoto J, Dorsey ER, Beck CA, Kieburtz K, Griggs RC. Pivotal studies of orphan drugs approved for neurological diseases. Ann Neurol. 2009;66(2):184–90. doi: 10.1002/ana.21676 19743448

20. Herder M. Orphan drug incentives in the pharmacogenomic context: policy responses in the USA and Canada. J Law Biosci. 2016;lsv060.

21. Gibson S, Tigerstrom B von. Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada. J Law Biosci. 2015;2(2):263–91. doi: 10.1093/jlb/lsv013 27774196

22. Kesselheim AS, Avorn J, Sarpatwari A. The high cost of prescription drugs in the United States: origins and prospects for reform. JAMA 2016;316(8):858–871. doi: 10.1001/jama.2016.11237 27552619

23. Bach PB. Indication-specific pricing for cancer drugs. JAMA 2014;312(16):1629–1630. doi: 10.1001/jama.2014.13235 25279433