Drugs Against Rare Diseases: Are The Regulatory Standards Higher?

Clinical Pharmacology and Therapeutics

Volumn , Issue , 2016, Pages 322-323

  Gobburu, J ^a   Pastoor, D ^a  

^a UNIVERSITY OF MARYLAND SCHOOL OF PHARMACY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DRUG EFFICACY; DRUG INDUSTRY; DRUG LEGISLATION; FOOD AND DRUG ADMINISTRATION; GOLD STANDARD; HUMAN; LICENSING; NOTE; PRIORITY JOURNAL; PROFESSIONAL STANDARD; RARE DISEASE; REGISTRATION; STANDARD;

EID: 84986587805     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1002/cpt.415     Document Type: Note

References (5)

1. European Medicines Agency. Guideline on Clinical Trials in Small Populations. Adopted guideline, CHMP/EWP/83561/2005. . Accessed 15 May 2015.
2. U.S. Food and Drug Administration. Rare Diseases: Common Issues in Drug Development Guidance for Industry [Internet]. Center for Drug Evaluation and Research (CDER). . Accessed 12 May 2016.
3. U.S. Food and Drug Administration. Targeted Drug Development: Why Are Many Diseases Lagging Behind? . Accessed 15 May 2015.
4. Sasinowski, F.J. Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs. . Accessed 6 June 2016.
5. Cornu, C. et al. Experimental designs for small randomised clinical trials: an algorithm for choice. Orphanet. J. Rare Dis. 8, 48 (2013).