Raising Orphans: How Clinical Development Programs of Drugs for Rare and Common Diseases Are Different

We compared clinical trials described in package inserts from noncancer orphan and nonorphan drugs from 1 January 2001 to 31 December 2011. Among the 37 orphan and 58 nonorphan drugs approved by the US Food and Drug Administration (US FDA) during this period, orphans had fewer clinical trials (2.8 v...

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Veröffentlicht in:Clinical pharmacology and therapeutics 2012-08, Vol.92 (2), p.262-264
Hauptverfasser: Orfali, M, Feldman, L, Bhattacharjee, V, Harkins, P, Kadam, S, Lo, C, Ravi, M, Shringarpure, D T, Mardekian, J, Cassino, C, Coté, T
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container_end_page 264
container_issue 2
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container_title Clinical pharmacology and therapeutics
container_volume 92
creator Orfali, M
Feldman, L
Bhattacharjee, V
Harkins, P
Kadam, S
Lo, C
Ravi, M
Shringarpure, D T
Mardekian, J
Cassino, C
Coté, T
description We compared clinical trials described in package inserts from noncancer orphan and nonorphan drugs from 1 January 2001 to 31 December 2011. Among the 37 orphan and 58 nonorphan drugs approved by the US Food and Drug Administration (US FDA) during this period, orphans had fewer clinical trials (2.8 vs. 3.5, P < 0.05) and fewer total participants (390 vs. 2,566, P < 0.001), but proportions with randomization, blinding, and placebo‐controlled clinical end points were similar, as were development times. We conclude that small studies of appropriate design can support US FDA approval of new medicines for rare diseases. Clinical Pharmacology & Therapeutics (2012); 92 2, 262–264. doi:10.1038/clpt.2012.87
doi_str_mv 10.1038/clpt.2012.87
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subjects Biological and medical sciences
Drug Approval - legislation & jurisprudence
Drug Approval - methods
Drug Discovery - economics
Drug Discovery - legislation & jurisprudence
Drug Discovery - methods
Humans
Medical sciences
Orphan Drug Production - economics
Orphan Drug Production - legislation & jurisprudence
Orphan Drug Production - methods
Pharmacology. Drug treatments
Randomized Controlled Trials as Topic
Rare Diseases - drug therapy
Research Design - legislation & jurisprudence
Research Design - standards
Sample Size
United States
United States Food and Drug Administration
title Raising Orphans: How Clinical Development Programs of Drugs for Rare and Common Diseases Are Different
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