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Clinical Approval Success Rates for Investigational Cancer Drugs
Author(s) -
DiMasi J A,
Reichert J M,
Feldman L,
Malins A
Publication year - 2013
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1038/clpt.2013.117
Subject(s) - clinical pharmacology , medicine , investigational drugs , cancer , clinical oncology , clinical trial , pharmacology , intensive care medicine
We examined development risks for new cancer drugs. For the full study period, the estimated clinical approval success rate for cancer compounds was 13.4% (9.9% for the first half of the study period, 19.8% for the second half). Small molecules had a somewhat higher clinical approval success rate than did large molecules (14.3 vs. 11.5%). Compounds studied solely in hematologic indications had markedly higher estimated clinical approval success rates than did compounds studied only in solid tumor indications (36.0 vs. 9.8%). The first, second, and third cancer indications pursued had estimated clinical approval success rates of 9.0, 8.2, and 6.9%, respectively. Success rates of second and third indications were found to be highly dependent on the success or failure of the first indication pursued (54.9 and 42.4%, respectively, for second and third indications if the first indication is a success, but 2.5 and 1.8%, respectively, if the first indication is a failure). Clinical Pharmacology & Therapeutics (2013); 94 3, 329–335. doi: 10.1038/clpt.2013.117