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

PLoS Medicine

Volumn 14, Issue 1, 2017, Pages

  Kesselheim, Aaron S ^a   Treasure, Carolyn L ^a   Joffe, Steven ^b,c  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AFATINIB; ALECTINIB; ANAPLASTIC LYMPHOMA KINASE; BIOLOGICAL MARKER; CABOZANTINIB; CERITINIB; COBIMETINIB; CRIZOTINIB; DABRAFENIB; IDELALISIB; IVACAFTOR; IVACAFTOR PLUS LUMACAFTOR; ORPHAN DRUG; OSIMERTINIB; PONATINIB; VEMURAFENIB;

ACT; ADENOCARCINOMA; ARTICLE; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DATA EXTRACTION; DRUG APPROVAL; DRUG COST; DRUG DESIGN; DRUG RESPONSE; ECONOMIC ASPECT; FOOD AND DRUG ADMINISTRATION; GENE MUTATION; GENETIC VARIABILITY; HEALTH CARE POLICY; INFORMATION PROCESSING; NON SMALL CELL LUNG CANCER; OFF LABEL DRUG USE; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROGRESSION FREE SURVIVAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SQUAMOUS CELL CARCINOMA; DRUG MANUFACTURE; ECONOMICS; HUMAN; LEGISLATION AND JURISPRUDENCE; RARE DISEASE; UNITED STATES;

BIOMARKERS; DRUG APPROVAL; HEALTH POLICY; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85011008969     PISSN: 15491277     EISSN: 15491676     Source Type: Journal    
DOI: 10.1371/journal.pmed.1002190     Document Type: Article

References (23)

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: 10.1038/508016a24695293
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: 10.1080/08989621.2013.79312023805831
5. Daniel MG, Pawlik TM, Fader AN, Esnaola NF, Makary MA, The Orphan Drug Act: Restoring the Mission to Rare Diseases. Am J Clin Oncol2015;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 Med2014;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.2012.42.725223423747
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/mdm51718083693
16. Fugh-Berman A, Melnick D, Off-label promotion, on-target sales. PLoS Med2008;5(10):e210. doi: 10.1371/journal.pmed.005021018959472
17. Wellman-Labadie O, Zhou Y, The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?Health Policy2010;95(2–3):216–28. doi: 10.1016/j.healthpol.2009.12.00120036435
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.2011.76921642684
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.2167619743448
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/lsv01327774196
22. Kesselheim AS, Avorn J, Sarpatwari A, The high cost of prescription drugs in the United States: origins and prospects for reform. JAMA2016;316(8):858–871. doi: 10.1001/jama.2016.1123727552619
23. Bach PB, Indication-specific pricing for cancer drugs. JAMA2014;312(16):1629–1630. doi: 10.1001/jama.2014.1323525279433