Clinical and regulatory features of drugs not initially approved by the FDA

Clinical Pharmacology and Therapeutics

Volumn 94, Issue 6, 2013, Pages 670-677

  Wang, B ^a   Avorn, J ^a   Kesselheim, A S ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BINODENOSON; CETHROMYCIN; DAPAGLIFLOZIN; DENOSUMAB; ECALLANTIDE; ETORICOXIB; FERRIC CARBOXYMALTOSE; FOSPROPOFOL; ICLAPRIM; IOFLUPANE I 123; MARAVIROC; MIFAMURTIDE; NEW DRUG; ORITAVANCIN; PEGLOTICASE; PERFLUBUTANE; RIMONABANT; RIVAROXABAN; SATRAPLATIN; SERTINDOLE; SUGAMMADEX; TEDISAMIL; TETRABENAZINE; TOCILIZUMAB; TORCETRAPIB; TRABECTEDIN; UNINDEXED DRUG; USTEKINUMAB; VAPREOTIDE; VERNAKALANT;

ADVISORY COMMITTEE; DIABETES MELLITUS; DRUG APPROVAL; DRUG EFFICACY; DRUG FATALITY; DRUG INDICATION; DRUG INDUSTRY; DRUG RESEARCH; DRUG SAFETY; DRUG SCREENING; EUROPEAN UNION; FOOD AND DRUG ADMINISTRATION; HUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); POSTMARKETING SURVEILLANCE; PRIORITY JOURNAL; REVIEW; UNITED STATES;

ADVISORY COMMITTEES; BIOLOGICAL PRODUCTS; DATABASES, PHARMACEUTICAL; DRUG APPROVAL; DRUG-RELATED SIDE EFFECTS AND ADVERSE REACTIONS; PHARMACEUTICAL PREPARATIONS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84888000492     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2013.165     Document Type: Review

References (45)

1. Lowry, F. Dapagliflozin declined by FDA. Heartwire, 19 January 2012.http://www.theheart.org/article/1342425.do.
2. Pollack, A. Diabetes drug dapagliflozin rejected by F.D.A. panel. New York Times, 19 July 2011.http://www.nytimes.com/2011/07/20/business/diabetes- drug-dapagliflozin-rejected-by-fda-panel.html?scp=5&sq=fda%20 rejection&st=cse .
3. Wehling, M. Drug development in the light of translational science: shine or shade? Drug Discov. Today 16, 1076-1083 (2011).
4. Brown, D. & Superti-Furga, G. Rediscovering the sweet spot in drug discovery. Drug Discov. Today 8, 1067-1077 (2003). (Pubitemid 38124472)
5. Goodyear, M. Learning from the TGN1412 trial. BMJ 332, 677-678 (2006). (Pubitemid 44365678)
6. Moses, H. 3rd, Dorsey, E.R., Matheson, D.H. & Thier, S.O. Financial anatomy of biomedical research. JAMA 294, 1333-1342 (2005). (Pubitemid 41345812)
7. Mullard, A. Learning lessons from Pfizer's $800 million failure. Nat. Rev. Drug Discov. 10(3):), 163-164 (2011).
8. Stein, R. FDA rejects another diabetes drug. Washington Post, 1 February 2011.http://voices.washingtonpost.com/checkup/2011/02/fda-rejects-another-diet- pill.html .
9. O'Riordan, M. Bariatric docs disappointed in latest setback: another antiobesity drug fails to make FDA cut. Heartwire, 14 February 2011,http://www.theheart.org/article/1184837.do .
10. Dobson, R. Biotechnology company's shares dive 67% after drug failure. BMJ 321, 1039 (2000).
11. Pollack, A. F.D.A. panel votes against obesity drug. New York Times, 1 May 2012.http://query.nytimes.com/gst/fullpage.html?res=9C01E3D9153CF935A257 54C0A9669D8B63&ref=vivusinc .
12. Hron, BM. Placebo or panacea: the FDA's rejection of ImClone's Erbitux licensing application.http://dash.harvard.edu/bitstream/handle/1/8889445/Hron. pdf .
13. Flook, B. The biotech sector fights for survival as its regulator tightens the reins, but some say there's hope for change. Washington Business Journal, 19 August 2011.http://www.bizjournals.com/washington/printedition/2011/ 08/19/the-biotech-sector-fights-for-survival.html .
14. Food and Drug Administration Safety and Innovation Act.http://www.gpo. gov/fdsys/pkg/BILLS-112s3187enr/pdf/BILLS-112s3187enr.pdf .
15. Advisory Committees: Drugs. Food and Drug Administration.http://www.fda. gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/default.htm .
16. LexisNexis. Total research system.http://www.lexisnexis.com/en-us/home. page .
17. US Food and Drug Administration. Medical Devices: PMA review process.http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/ HowtoMarketYourDevice/PremarketSubmissions/PremarketApprovalPMA/ucm047991.htm .
18. US Food and Drug Administration. Drugs: Questions and answers regarding the complete response letter.
19. Drug Product Database. Health Canada.http://www.fda.gov/Drugs/ GuidanceComplianceRegulatoryInformation/LawsActsandRules/ucm084143.htm . Accessed 18 July 2013.
20. European Medicines Agency.http://www.ema.europa.eu/ema/index.jsp?curl=/ pages/home/Home-page.jsp&jsenabled=true . Accessed 18 July 2013.
21. Munos, B. Lessons from 60 years of pharmaceutical innovation. Nat. Rev. Drug Discov. 8, 959-968 (2009).
22. Pharm Exec 50. Pharmaceutical Executive. http://www.pharmexec.com/ pharmexec/data/articlestandard//pharmexec/302008/531367/article.pdf
23. Pharm Exec 50. Pharmaceutical Executive. http://www.pharmexec.com/ pharmexec/data/articlestandard/pharmexec/202009/597526/article.pdf
24. Clinton, P., Mozeson, M. Pharm Exec 50. Pharmaceutical Executive.
25. US Food and Drug Administration. Fast track, accelerated approval and priority review. http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/ speedingaccesstoimportantnewtherapies/ucm128291. htm>
26. US Food and Drug Administration. Orphan Drug Act.http://www.fda.gov/ regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/ significantamendmentstothefdcact/orphandrugact/default.htm
27. US Food and Drug Administration. BLA Approval Letter.http://www. accessdata.fda.gov/drugsat.fda-docs/nda/2009/125277s000Approv.pdf
28. Ioannides-Demos, L.L., Piccenna, L. & McNeil, J.J. Pharmacotherapies for obesity: past, current, and future therapies. J. Obes. 2011, 179674 (2011).
29. Government Accountability Office. Prescription drugs: FDA has met most performance goals for reviewing applications.http://www.gao.gov/assets/590/ 589762.pdf
30. Carpenter, D. Reputation and Power: Organizational Image and Pharmaceutical Regulation at the FDA (Princeton University Press: Princeton, NJ, 2010).
31. Arrowsmith, J. Phase II failures: 2008-2010. Nat. Rev. Drug Discov. 10(2), 1 (2011).
32. Arrowsmith, J. Phase III and submission failures: 2007-2010. Nat. Rev. Drug Discov. 10(5), 1 (2011).
33. Downing, N.S., Aminawung, J.A., Shah, N.D., Braunstein, J.B., Krumholz, H.M. & Ross, J.S. Regulatory review of novel therapeutics-comparison of three regulatory agencies. N. Engl. J. Med. 366, 2284-2293 (2012).
34. Doshi, P. & Jefferson, T. Drug data shouldn't be secret. New York Times, 10 April 2012.http://www.nytimes.com/2012/04/11/opinion/drug-data- shouldntbe-secret.html>
35. Thomas, K. Breaking the seal on drug research. New York Times, 29 June 2013.http://www.nytimes.com/2013/06/30/business/breaking-the-seal-on- drugresearch.html?pagewanted=all.
36. Code of Federal Regulations Title 21, Vol 5, s312.47.http://www. accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.47>
37. US Food and Drug Administration. Guidance for industry: special protocol assessment.http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/ucm080571.pdf .
38. Booz Allen Hamilton. Evaluating the approval path for safe and effective new drugs and biologics.http://www.boozallen.com/media/file/FDA-Approval-Path. pdf> .
39. Pariser, A.R., Slack, D.J., Bauer, L.J., Warner, C.A. & Tracy, L.A. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010. Drug Discov. Today 17, 898-904 (2012).
40. US Food and Drug Administration. PDUFA reauthorization performance goals and procedures fiscal years 2013 through 2017.http://www.fda.gov/downloads/ ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf> .
41. Moore, T.J. & Furberg, C.D. The safety risks of innovation: the FDA's Expedited Drug Development Pathway. JAMA 308, 869-870 (2012).
42. Saltonstall, P.L. Clinical trials of orphan drugs for cancer. JAMA 306, 1545; author reply 1545-1545; author reply 1546 (2011).
43. Volokh, E. Medical self-defense, prohibited experimental therapies, and payment for organs. Harv. Law Rev. 120, 1813-1846 (2007).
44. Woodcock, J. Evidence vs. access: can twenty-first-century drug regulation refine the tradeoffs? Clin. Pharmacol. Ther. 91, 378-380 (2012).
45. Avorn, J. Powerful Medicines: The Benefits, Risks, and Costs of Prescription Drugs (Knopf: New York, 2004).