title: Participation of women and sex analyses in late-phase clinical trials of new molecular entity drugs and biologics approved by the FDA in 2007-2009.
abstract: BACKGROUND
  Biological sex differences may contribute to differential treatment outcomes for therapeutic products. This study tracks women's participation in late-phase clinical trials (LPCTs), where efficacy and safety of drugs and biologics are evaluated, of new molecular entity (NME) drugs and biologics approved by the U.S. Food and Drug Administration (FDA) in 2007-2009. Furthermore, presentations of sex-based analyses were assessed from the FDA reviews.
  
  
  METHODS
  New drug applications (NDAs) and biologics license applications (BLAs) were accessed from the U.S. FDA database and evaluated for women's participation in LPCTs. Sex-based analyses for efficacy and safety contained in FDA reviews were surveyed. Ratios for women's LPCT participation (PROPORTION OF STUDY SUBJECTS) to their proportion in the disease population were calculated for each approved therapeutic product and grouped into therapeutic categories.
  
  
  RESULTS
  Sex-specific (n=5) and pediatric (n=3) drug applications were excluded. Women's participation in LPCTs was 39%, 48%, and 42% in NDAs (n=50) and 49%, 62%, and 58% in BLAs (n=11) for 2007, 2008, and 2009, respectively. Sixty-four percent of NDAs and 91% of BLAs had participation to proportion ratios of ≥0.80. Seventy-four percent of NDA reviews and 64% of BLA reviews included safety and efficacy sex analysis. Ninety-six percent of NDA reviews and 100% of BLA reviews included efficacy sex analysis.
  
  
  CONCLUSIONS
  Women's participation in LPCTs averaged 43% for NDAs and 57% for BLAs in 2007-2009 and varied widely by indication. As a comparison, the 2001 U.S. Government Accountability Office (GAO) reported 52% of women's participation for drug clinical trials in1998-2000 and an FDA study reported 45% for BLAs approved from 1995 to 1999. This study showed that sex-analysis of both safety and efficacy in NDA has increased to 74% since the GAO report of 72%, while those for BLAs increased to 64% from 37% reported for therapeutic biologics approved in 1995-1999. Knowledge of disease prevalence and participation in clinical trials provides an understanding of recruitment and retention patterns of patients in these trials.
authors.affiliation: Office of Women's Health (OWH), Center for Drug Evaluation and Research (CDER), Food and Drug Administration, Silver Spring, MD 20993, USA.
authors.forename: Rita
authors.initials: R
authors.lastname: Poon
authors.forename: Keshav
authors.initials: K
authors.lastname: Khanijow
authors.forename: Sphoorti
authors.initials: S
authors.lastname: Umarjee
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Fadiran
authors.forename: Monica
authors.initials: M
authors.lastname: Yu
authors.forename: Lei
authors.initials: L
authors.lastname: Zhang
authors.forename: Ameeta
authors.initials: A
authors.lastname: Parekh
mesh_terms: D000328:Adult; D001688:Biological Products; D002986:Clinical Trials as Topic / Q000379:methods*; D062313:Databases, Pharmaceutical; D017277:Drug Approval / Q000706:statistics & numerical data*; D005260:Female; D006805:Human Experimentation / Q000706:statistics & numerical data; D006801:Humans; D008004:Licensure / Q000706:statistics & numerical data*; D008297:Male; D010358:Patient Participation / Q000706:statistics & numerical data; D018579:Patient Selection; D010372:Pediatrics; D004364:Pharmaceutical Preparations; D018570:Risk Assessment; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D001688:Biological Products; D004364:Pharmaceutical Preparations