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Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
09:19

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo

Published on: February 6, 2015

Clinical approval success rates for investigational cancer drugs

J A DiMasi1, J M Reichert, L Feldman

  • 1Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA. joseph.dimasi@tufts.edu

Clinical Pharmacology and Therapeutics
|June 7, 2013
PubMed

Insights

New cancer drug development faces significant risks, with an overall clinical approval success rate of 13.4%. Hematologic cancer drug development shows higher success rates than solid tumors, and early success significantly impacts later-stage drug development.

Area of Science:

  • Oncology
  • Pharmaceutical Development
  • Drug Discovery

Background:

  • Developing new cancer drugs is a complex and high-risk process.
  • Understanding clinical approval success rates is crucial for pharmaceutical investment and strategy.
  • Historical data provides insights into factors influencing drug development outcomes.

Purpose of the Study:

  • To analyze the clinical approval success rates of new cancer drug compounds.
  • To identify factors associated with higher success rates in cancer drug development.
  • To evaluate the impact of indication type and prior trial outcomes on drug approval probabilities.

Main Methods:

  • Retrospective analysis of clinical trial data for cancer drug compounds.
  • Calculation of estimated clinical approval success rates based on study period and drug type.
  • Comparison of success rates across different cancer indications (hematologic vs. solid tumors).
  • Assessment of the influence of the first indication's success on subsequent indications.

Main Results:

  • The overall clinical approval success rate for cancer compounds was 13.4% across the study period.
  • Small molecule drugs demonstrated a higher success rate (14.3%) compared to large molecules (11.5%).
  • Compounds targeting hematologic indications had significantly higher success rates (36.0%) than those for solid tumors (9.8%).
  • Success rates for second and third indications were substantially higher if the first indication was successful (54.9% and 42.4%) versus if it failed (2.5% and 1.8%).

Conclusions:

  • Cancer drug development is characterized by low overall success rates, highlighting significant risks.
  • Drug development strategies may benefit from focusing on hematologic indications and small molecule drugs.
  • The success of initial clinical trials is a critical predictor for the success of subsequent indications for the same drug compound.

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