title: Clinical approval success rates for investigational cancer drugs.
abstract: 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).
authors.affiliation: Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA. joseph.dimasi@tufts.edu
authors.forename: J A
authors.initials: JA
authors.lastname: DiMasi
authors.forename: J M
authors.initials: JM
authors.lastname: Reichert
authors.forename: L
authors.initials: L
authors.lastname: Feldman
authors.forename: A
authors.initials: A
authors.lastname: Malins
mesh_terms: D000970:Antineoplastic Agents / Q000737:chemistry / Q000627:therapeutic use*; D001688:Biological Products / Q000737:chemistry / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000706:statistics & numerical data*; D004345:Drug Industry / Q000639:trends*; D015507:Drugs, Investigational / Q000737:chemistry / Q000627:therapeutic use*; D006801:Humans
chemical_list: D000970:Antineoplastic Agents; D001688:Biological Products; D015507:Drugs, Investigational