Expedited programs for drug development and approval

Expert Opinion on Orphan Drugs

Volumn 1, Issue 7, 2013, Pages 507-510

  Pariser, Anne R ^a   Robb, Melissa ^a   Sherman, Rachel E ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Drug approval; Food and Drug Administration; Orphan drugs; Rare diseases

Indexed keywords

NEW DRUG;

DRUG APPROVAL; DRUG DEVELOPMENT; DRUG EFFICACY; DRUG LEGISLATION; DRUG RESEARCH; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; HUMAN; REVIEW;

EID: 84896689199     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2013.805124     Document Type: Review

References (14)

1. Drugs intended to treat life-threatening and severely-debilitating illnesses, 21 Code of Federal Regulations (C.F.R.) Subpart E Sect. 312.80-312.84
2. The Food and Drug Administration Modernization Act (FDAMA) of 1997. Public Law. 1997;105-15
3. Accelerated approval of new drugs for serious or life-threatening illnesses, 21 C. F.R. Subpart H Sect. 314.500-314.560 and 21 C.F.R. Subpart E Sect. 601.40-601.46
4. The Prescription Drug User Fee Act (PDUFA) of 1992, Public Law 1992;102-571
5. The Food and Drug Administration Safety and Innovation Act (FDASIA). Public Law. 2012;112-44
6. Cassino C, Orfali M, Charnigo RJ, Marsden DL. Rare and orphan diseases challenges: clinical development and clinical practice. J Rare Disorders 2013;1:1-3
7. Independent evaluation of FDAs first cycle review performance-retrospective analysis final report text. Booz Allen Hamilton, Inc., FDA, Washington, DC. 2006. Available from: http://www.fda. gov/ForIndustry/UserFees/ PrescriptionDrugUserFee/ucm119469. htm [Last accessed 11 April 2013]
8. FDAVoice, Early communication: A key to reduced drug development and approval times. Pariser A. FDA/CDER, Washington, DC; 2013. Available from: http://blogs.fda.gov/fdavoice/index.php/ 2013/02/early-communication-a-key-toreduced- drug-development-and-approvaltimes/ [Last accessed 11 April 2013]
9. Pariser AR, Slack DJ, Bauer LJ, et al. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010. Drug Discov Today 2012;17:898-904
10. About FDA, FY 2012 innovative drug approvals. FDA, Washington, DC; 2012. Available from: http://www.fda. gov/AboutFDA/ReportsManualsForms/ Reports/ucm276385.htm [Last accessed 11 April 2013]
11. CDER new drug review: 2012 update. Jenkins JK. FDA, Washington, DC; 2012. Available from: http://www.fda. gov/downloads/AboutFDA/ CentersOffices/OfficeofMedical ProductsandTobacco/CDER/ UCM331454.pdf [Last accessed 11 April 2013]
12. Downing NS, Aminawung JA, Shah ND, et al. Regulatory review of novel therapeutics-comparison of three regulatory agencies. N Engl J Med 2012;366:2284-93
13. Regulatory information, Frequently asked questions: breakthrough therapies. FDA, Washington, DC; 2012. Available from: http://www.fda. gov/RegulatoryInformation/Legislation/ FederalFoodDrugandCosmeticActFDC Act/SignificantAmendmentstothe FDCAct/FDASIA/ucm341027.htm [Last accessed 11 April 2013]
14. Sasinowski FJ. Quantum of effectiveness evidence in FDAs approval of orphan drugs: cataloguing FDAs flexibility in regulating therapies for persons with rare disorders. Drug Inf J 2012;46:238-63