Orphan drug incentives in the pharmacogenomic context: Policy responses in the US and Canada

Journal of Law and the Biosciences

Volumn 2, Issue 3, 2016, Pages 263-291

  Gibson, Shannon ^a   von Tigerstrom, Barbara ^b  

^a UNIVERSITY OF TORONTO   (Canada)

^b UNIVERSITY OF SASKATCHEWAN   (Canada)

Author keywords

Food and Drug Administration; Orphan drug policy; Pharmaceutical policy; Pharmacogenomics; Rare diseases

Indexed keywords

EID: 85010970480     PISSN: None     EISSN: 20539711     Source Type: Journal    
DOI: 10.1093/jlb/lsv013     Document Type: Article

References (61)

1. Abbey S.Meyers, History of the American Orphan Drug Act, NATIONAL ORGANIZATION FOR RARE DISORDERS, Feb. 18, 2000, at 1, https://www.rarediseases.org/docs/policy/HistoryoftheAmericanOrphanDrugAct.pdf (accessed Dec. 10, 2014)
2. Timoty R. Coté, Kui Xu & Anne R. Pariser, Accelerating Orphan Drug Development, 9 NAT. REV. DRUG DISC. 901, 901 (2010)
3. Paul D. Maher & MarleneHaffner, Orphan Drug Designation and Pharmacogenomics: Options and Opportunities, 20 BIODRUGS 71, 72 (2006)
4. US Food and Drug Administration, Developing Products for Rare Diseases & Conditions, http://www.fda.gov/ forindustry/DevelopingProductsforrareDiseasesConditions/default.htm (accessed Dec. 10, 2014)
5. Olivier Wellman-Labadie & Youwen Zhou, The US Orphan Drug Act: Rare Disease Research Stimulator or Commercial Opportunity?, 95HEALTH POL'Y 216, 221 (2010)
6. Reuters, The Economic Power of Orphan Drugs, 2012, at 4, http://thomsonreuters.com/business-unit/ science/subsector/pdf/the-economic-power-of-orphan-drugs.pdf (accessed Dec. 10, 2014)
7. Jonathan D. Rockoff, Drug Makers See Profit Potential in Rare Diseases, WALL STREET JOURNAL, Jan. 30, 2013, http://www.wsj.com/articles/SB10001424127887323926104578273900197322758 (accessed Apr. 2, 2015)
8. Christopher-Paul Milne & Joyce Tait, Evolution Along the Government-Governance Continuum: FDA's Orphan Products and Fast Track Programs as Exemplars of "What Works" for Innovation and Regulation, 64FOODDRUG LAW J. 733, 742-43 (2009)
9. Ian Kerr, Jennifer Chandler & Timothy Caulfield, Emerging Health Technologies, in 501 CANADIAN HEALTH LAW AND POLICY, (Jocelyn Downie et al. eds, 4th ed. 2011), at 504
10. National Human Genome Research Institute, Frequently Asked Questions About Pharmacogenomics, http://www.genome.gov/27530645 (accessed Dec. 10, 2014)
11. Roger Collier, Bye, Bye Blockbusters, Hello Niche Busters, 183 CAN. MED. ASS'NS J. e697 (2011)
12. Personalized Medicines Coalition, Personalized Medicine by the Numbers, 2014, http://www. personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/pmc personalized medicine by the numbers.pdf (accessed Dec. 10, 2014)
13. DavidLoughnot, Potential Interactions of the Orphan Drug Act andPharmacogenomics:AFlood of Orphan Drugs and Abuses? 31 AM. J. L.&MED. 365 (2005)
14. Matthew Herder, When Everyone is an Orphan:Against Adopting aU.S.-styled Orphan Drug Policy in Canada, 20ACCT.RES.227 (2013)
15. Marlene E. Haffner, Josep Torrent-Farnell & Paul D. Maher, Does Orphan Drug Legislation Really Answer the Needs of Patients?, 371 LANCET 2041, 2043 (2008)
16. Dov Greenbaum, Incentivizing Pharmacogenomic Drug Development: How the FDA Can Overcome Early Missteps in Regulating PersonalizedMedicine, 40 RUTGERS L. J. 97 (2008)
17. Health Canada, Office of Legislative and RegulatoryModernization, Initial Draft Discussion Document for a Canadian Orphan Drug Regulatory Framework, Dec. 13, 2012, at 4, http://www.orpha.net/national/data/ CA-EN/www/uploads/Initial-Draft-Discussion-Document-for-A-Canadian-Orphan-Drug-Regulatory-Framework.doc (accessed Dec. 11, 2014)
18. TheWhiteHouse, FACT SHEET: President Obama's PrecisionsMedicine Initiative, Jan. 30, 2015, http://www. whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative (accessed Feb. 27, 2015)
19. Valerie G. Koch, Incentivizing Utilization of Pharmacogenomics in Drug Development, 15 J.HEALTH CARE L. & POL'Y 263 (2012)
20. Christopher A. Lipinski, Drug Repurposing, 8 DRUG DISC.TODAY 57, 57 (2011)
21. Thomas Eissing, Jörg Lippert & Stefen Willmann, Pharmacogenomics of Codeine, Morphine, and Morphine-6-glucuronide: Model-based Analysis of the Influence of CYP2D6 Activity, UGT2B7 Activity, Renal Impairment, and CYP3A4 Inhibition, 16MOL. DIAGN. THER. 43 (2012)
22. http://www.accessdata.fda.gov/ scripts/opdlisting/oopd/index.cfm (accessed Dec. 11, 2014)
23. US Food and Drug Administration, Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products (Draft Guidance), 2012, http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/UCM332181.pdf (accessedMay 6, 2014)
24. Julian R. Molina et al., Non-Small Cell Lung Cancer: Epidemiology, Risk Factors, Treatment, and Survivorship, 83MAYO CLIN. PROC. 582 (2009)
25. American Lung Association, Lung Cancer Fact Sheet, http://www.lung.org/lung-disease/lung-cancer/ resources/facts-figures/lung-cancer-fact-sheet.html (accessed Dec. 11, 2014)
26. J. Korpanty et al., Biomarkers That Currently Affect Clinical Practice in Lung Cancer: EGFR, ALK,MET, ROS-1, and KRAS, 4 FRONT. ONCOL. 204 (2014)
27. James H. Reese, FDA Orphan Drug Designation 101, at 23, http://www.ema.europa.eu/ docs/en GB/document library/Presentation/2014/03/WC500164160.pdf (accessed Jan. 13, 2015)
28. National Cancer Institute, SEER Stat Fact Sheets: Melanoma of the Skin, http://seer.cancer.gov/ statfacts/html/melan.html (accessed Dec. 18, 2014)
29. Jai Shah, Economic and Regulatory Considerations in Pharmacogenomics for Drug Licensing and Healthcare, 21 NAT. BIOTECHNOL. 747, 749 (2002)
30. National Cancer Institute, SEER Stat Fact Sheets: Breast Cancer, http://seer.cancer.gov/statfacts/html/ breast.html (accessed Dec. 18, 2014)
31. Leukemia and Lymphoma Society, Facts, 2014, at 8, https://www.lls.org/content/nationalcontent/resourcecenter/ freeeducationmaterials/generalcancer/pdf/facts.pdf (accessed Dec. 18, 2014)
32. National Cancer Institute, SEER Stat Fact Sheets: Pancreas Cancer, http://seer.cancer.gov/statfacts/html/pancreas.html (accessedDec. 18, 2014)
33. Gary A. Pulsinelli, The Orphan Drug Act: What's Right with It, 15 SANTA CLARA HIGH TECH. L. J 299, 310 (1999)
34. Kurt R. Karst & Michelle L. Butler, FDA Issues Post-Depomed Policy Statement; Agency Doubles Down on Clinical Superiority Requirement, FDA LAW BLOG, Dec. 22, 2014, http://www.fdalawblog.net/fda law blog hyman phelps/2014/12/fda-issues-post-depomed-policystatement-agency-doubles-down-on-clinical-superiority-requirement.html (accessed Dec. 23, 2014)
35. Steven Grossman, President of health policy consultancy HPS Group, quoted in Fiona Barry, FDA Defiant on Orphan Exclusivity Rules after Court Judgement, IN-PHARMA, Jan. 7, 2015, http://www.in-pharmatechnologist.com/Regulatory-Safety/FDA-defiant-on-orphan-exclusivity-ruleseven-after-court-judgement (accessed Apr. 2, 2015)
36. Tal Burt & Savita Dhillon, Pharmacogenomics in Early-phase Clinical Development, 12 PHARMACOGENOMICS 1085, 1090 (2013)
37. Rebecca S. Yoshitani & Ellen S. Cooper, Pharmaceutical Reformulation: The Growth of Lifecycle Management, 7HOUS. J. HEALTH L. & POL'Y 379, 380 (2007)
38. http://www.cff.org/aboutcf/faqs (accessed Dec. 18, 2014)
39. http://www.cff.org/aboutCFFoundation/NewsEvents/2-21-FDA-Approves-Expanded-Use-of-Kalydecofor-CF.cfm (accessed Dec. 18, 2014)
40. Felix W. Frueh, Back to the Future: Why Randomized Controlled Trials Cannot Be the Answer to Pharmacogenomics and PersonalizedMedicine, 10 PHARMACOGENOMICS 1077 (2009)
41. US Food and Drug Administration, The Future of Drug Safety-Promoting and Protecting the Health of the Public (FDA's Response to the Institute of Medicine's 2006 Report), Jan. 2007, at 3, http://www.fda.gov/ downloads/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders/UCM171627. pdf (accessed Dec. 18, 2014)
42. US Food and Drug Administration, A Valuable Resource for Drug Developers: The Rare Disease Repurposing Database (RDRD), http://www.fda.gov/ForIndustry/Developing ProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm216147.htm (accessed Dec. 19, 2014)
43. M. Angeles Villarreal, Orphan Drug Act: Background and Proposed Legislation in the 107th Congress (CRS Report for Congress), July 25, 2001, at 2, http://www.law.umaryland.edu/marshall/crsreports/crsdocuments/RS20971.pdf (accessed Dec. 19, 2014)
44. ChristopherM. Wittich, ChristopherM. Burkle, & William L. Lanier, Ten Common Questions (and Their Answers) About Off-label Drug Use, 87MAYO CLIN. PROC. 982 (2012)
45. Food and Drug Administration, Abbreviated New Drug Application (ANDA): Generics, updated Sept. 18, 2014, http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsare DevelopedandApproved/ApprovalApplications/AbbreviatedNewDrugApplicationANDAGenerics/ (accessedMar. 9, 2015)
46. Terry Mahn, Protecting New Investments in Old Drugs,Fish and Richardson PC, updated Mar./Apr. 2009, http://www.fr.com/files/Uploads/bios/Mahn Terry/Protecting New Investments.pdf (accessed Apr. 2, 2015)
47. DurhaneWong-Reiger, Canada's Long Journey toward an Orphan Drug Framework, ADVOCATE, 2013, at 20
48. Health Canada, Orphan Drug Policy (letter sent to associations, file no: 96-037419), Jan. 16, 1997, https://web.archive.org/web/20130722055233/, http://www.hc-sc.gc.ca/dhp-mps/prodpharma/applicdemande/ pol/orph?pol-eng.php (accessed July 2013)
49. Canadian Organization for Rare Disorders, Canada's Orphan Drug Policy, July 6, 2005, at 4, http://paperzz.com/download/1749313#pdf (accessed Dec. 19, 2014)
50. BIOTECanada, Helping Canadians with Rare Diseases: Government of Canada Rare Disease Strategy Offers Hope and Solutions for Patients, http://www.biotech.ca/en/policy-matters/health-bio/rare.aspx (accessed Dec. 19, 2014)
51. External Advisory Committee on Smart Regulation, Smart Regulation: A Regulatory Strategy for Canada (Report to the Government of Canada), Sep. 2004, at 93, http://publications.gc.ca/collections/Collection/ CP22-78-2004E.pdf (accessed Dec. 19, 2014)
52. Canadian Organization for Rare Disorders, Current Issues, http://www.raredisorders.ca/currentIssues.html (accessed Dec. 19, 2014)
53. ShannonGibson & Trudo Lemmens, NicheMarkets and Evidence Assessment in Transition: A Critical Review of Proposed Drug Reforms 22 MED. LAW REV. 200 (2014)
54. Dirk Czarnitzki, Petr Hanel & JulioMiguel Rosa, Evaluating the Impact of R & D Tax Credits on Innovation: A Microeconometric Study on Canadian Firms 40 RES. POL'Y 217, 218 (2011)
55. US Food and Drug Administration, User Fee Waivers, Reductions, and Refunds for Drug and Biological Products (Guidance for Industry), Sep., 2011, at 11, http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ ucm079298.pdf (accessed Dec. 19, 2014)
56. Health Canada, Health Canada's Proposal to Parliament for User Fees and Service Standards for Human Drugs and Medical Devices Programs, Apr., 2010, at Table 1A, http://www.hc-sc.gc.ca/dhp-mps/finance/costs-couts/fee-propo-frais-eng.php pdf (accessed Dec. 19, 2014)
57. US Food and Drug Administration, Postmarketing Requirements and Commitments: Introduction, http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIV Commitments/default.htm (accessed Dec. 20, 2014)
58. European Commission, Orphan Refusals, http://ec.europa.eu/health/documents/community-register/ html/orh others.htm (accessed Dec. 20, 2014)
59. Health Canada, Guidance Document: Data Protection under C.08.004.1 of theFood and Drug Regulations (Guidance document), http://www.hc-sc.gc.ca/dhp-mps/prodpharma/applic-demande/guide-ld/data donnees protection-eng. php (accessed Dec. 20, 2014)
60. Kurt R. Karst, Exclusivity Creep: OPEN ACT Would Open Up Hatch-Waxman and the BPCIA to Add a 6-Month Extension to Existing Exclusivities, FDA LAW BLOG, Dec. 4, 2014, http://www.fdalawblog.net/ fda law blog hyman phelps/2014/12/exclusivity-creep-open-act-would-open-up-hatch-waxman-and-thebpcia-to-add-a-6-month-extension-to-ex.html (accessed Dec. 20, 2014)
61. Health Canada, An Orphan Drug Framework for Canada, http://www.hc-sc.gc.ca/ahc-asc/media/nr-cp/ 2012/2012-147a-eng.php (accessed Dec. 20, 2014)