Demographics of clinical trials participants in pivotal clinical trials for new molecular entity drugs and biologics approved by FDA From 2010 to 2012

Am J Ther. 2015 Nov-Dec;22(6):435-55. doi: 10.1097/MJT.0000000000000177.


To fully assess the safety and efficacy of therapeutics before approval, the US Food and Drug Administration (FDA) has encouraged adequate representation and assessment of demographic subgroups in clinical trials through guidance documents and regulations. This study aimed to survey the demographics of participants in pivotal clinical trials, as well as the presence of analyses by sex on efficacy and safety for FDA-approved new drug applications (NDAs) and biologics license applications (BLAs) from 2010 to 2012. Medical and statistical reviews for new molecular entity drugs and biological products approved during this period were obtained from Drugs@FDA. All pivotal clinical trials referenced in the FDA reviews were evaluated for the participation of different demographic subgroups (such as sex, race/ethnicity, and age). Pivotal trials were defined as those phase 2 and/or phase 3 trials described in the labeling or the FDA medical reviews in support of the drug/biological approval. Eighty-three new molecular entities (66 NDAs and 17 BLAs) were approved by the FDA from 2010 to 2012. Overall, women constituted 45% of trial participants for NDAs and 65% for BLAs. Sex analysis related to safety and efficacy was reported in 92% of the surveyed FDA medical and statistical reviews. Most NDAs and BLAs (82%) had a study population that was representative of the sex distribution for the intended patient population; however, most study participants were whites (77%), and minority racial/ethnic groups had lower participation rates in the study population than would be representative of the US racial group populations.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Biological Products*
  • Child
  • Clinical Trials as Topic*
  • Drug Approval*
  • Female
  • Humans
  • Male
  • Middle Aged
  • Patient Participation*
  • Time Factors
  • United States
  • United States Food and Drug Administration


  • Biological Products