Precision Medicines Have Faster Approvals Based On Fewer And Smaller Trials Than Other Medicines

Health Aff (Millwood). 2018 May;37(5):724-731. doi: 10.1377/hlthaff.2017.1580.


Precision medicines can benefit patients by increasing the probability of a successful treatment response in selected patient populations. The potential for more immediate signals of efficacy during clinical trials suggests such medicines will reach the market more rapidly than traditional drugs will. Using data from the Food and Drug Administration (FDA), we examined the regulatory review and pivotal trial characteristics of precision medicines. We found that in the period January 2013-June 2017, precision medicines were developed and reviewed almost two years faster than nonprecision medicines. In addition, approximately 48 percent of the precision medicines in our study qualified for the FDA's breakthrough therapy designation. Precision medicines were also approved based on fewer pivotal trials with fewer patients, and the trials were less likely to be randomized, blinded, or controlled with either an active or placebo comparator.

Keywords: Clinical trial; Designations; Precision medicines; Speed; Trial size.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biological Products / administration & dosage*
  • Clinical Trials as Topic
  • Cross-Sectional Studies
  • Databases, Factual
  • Drug Approval*
  • Female
  • Humans
  • Male
  • Molecular Targeted Therapy
  • Pharmaceutical Preparations
  • Precision Medicine / methods
  • Precision Medicine / statistics & numerical data*
  • Statistics, Nonparametric
  • United States
  • United States Food and Drug Administration*


  • Biological Products
  • Pharmaceutical Preparations