Accelerating orphan drug development

Nature Reviews Drug Discovery

Volumn 9, Issue 12, 2010, Pages 901-902

  Coté, Timothy R ^a   Xu, Kui ^a   Pariser, Anne R ^a  

^a Office of Orphan Products Development ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

3 [1 (2,6 DICHLORO 3 FLUOROPHENYL)ETHOXY] 5 [1 (4 PIPERIDINYL) 1H PYRAZOL 4 YL] 2 PYRIDINYLAMINE; BOTULINUM TOXIN A; CRIZOTINIB; ORPHAN DRUG; UNCLASSIFIED DRUG;

BLEPHAROSPASM; CERVICAL DYSTONIA; DRUG EFFICACY; DRUG LEGISLATION; DRUG MARKETING; DRUG RESEARCH; DRUG SAFETY; DRUG SCREENING; HEALTH CARE POLICY; HUMAN; KIDNEY CARCINOMA; LUNG NON SMALL CELL CANCER; MEDICAL SOCIETY; NOTE; PREVALENCE; PRIORITY JOURNAL; RARE DISEASE; RISK BENEFIT ANALYSIS; UNITED STATES;

ANIMALS; DRUG APPROVAL; DRUG DISCOVERY; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 78649689236     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd3340     Document Type: Note

References (9)

1. Braun, M. M. et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nature Rev. Drug Discov. 9, 519-522 (2010).
2. National Organization for Rare Disorders (NORD). About NORD. NORD website [online] http://www.rarediseases.org/info/about.html (2010).
3. Department of Health and Human Services. Food and Drug Administration. 21 CFR Part 316. Orphan Drug Regulations: Proposed Rule. Federal Register 56, 3338-3351 (1991).
4. US FDA. New resource for drug developers: the rare disease repurposing database (RDRD). US FDA website [online], http://www. fda.gov/ForIndustry/ DevelopingProductsforRareDiseasesConditions/ HowtoapplyforOrphanProductDesignation/ucm216147.htm (2010).
5. US FDA. Code of Federal Regulations Title 21: Adequate and Well-Controlled Studies Pt. 314.126 (US Government Printing Office, Washington, DC, 2009).
6. US FDA. Code of Federal Regulations Title 21: Approval of an Application and an Abbreviated Application Pt. 314.105 (US Government Printing Office, Washington, DC, 2009).
7. Hamburg, M. A. Announcement of FDA/NIH collaboration on advancing regulatory science. FDA Website [online], http://www.fda.gov/NewsEvents/ Speeches/ucm201687.htm (2010).
8. Institute of Medicine of the National Academies. Rare Diseases and Orphan Products: Accelerating Research and Development (eds Field, M. J. and Boat, T. F.) (The National Academies Press, Washington, DC, 2010).
9. Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010, Pub. L. No. 111-180, §740 (2010).