2007-2009 年 FDA 批准的新分子实体药物和生物制品的后期临床试验中女性参与情况和性别分析。
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.
机构信息
Office of Women's Health (OWH), Center for Drug Evaluation and Research (CDER), Food and Drug Administration, Silver Spring, MD 20993, USA.
出版信息
J Womens Health (Larchmt). 2013 Jul;22(7):604-16. doi: 10.1089/jwh.2012.3753. Epub 2013 Jun 14.
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.
CONCLUSION
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.
背景
生物性别差异可能导致治疗产品的治疗效果存在差异。本研究跟踪了女性参与新药临床试验（LPCT）的情况，这些试验评估了 2007-2009 年美国食品和药物管理局（FDA）批准的新分子实体（NME）药物和生物制剂的疗效和安全性。此外，还评估了 FDA 审查中基于性别的分析报告。
方法
从美国 FDA 数据库中获取新药申请（NDA）和生物制品许可申请（BLA），评估女性参与 LPCT 的情况。调查了 FDA 审查中包含的疗效和安全性的基于性别的分析。为每个批准的治疗产品计算了女性 LPCT 参与率（研究受试者比例）与其在疾病人群中的比例之比，并按治疗类别进行分组。
结果
排除了特定性别（n=5）和儿科（n=3）药物申请。2007 年、2008 年和 2009 年，女性在 NDA（n=50）中的 LPCT 参与率分别为 39%、48%和 42%，在 BLA（n=11）中的 LPCT 参与率分别为 49%、62%和 58%。64%的 NDA 和 91%的 BLA 有参与比例≥0.80。74%的 NDA 审查和 64%的 BLA 审查包括安全性和疗效的性别分析。96%的 NDA 审查和 100%的 BLA 审查包括疗效的性别分析。
结论
2007-2009 年，女性在 NDA 的 LPCT 参与率平均为 43%，在 BLA 的 LPCT 参与率为 57%，不同适应症的参与率差异很大。相比之下，2001 年美国政府问责局（GAO）报告称，1998-2000 年女性在药物临床试验中的参与率为 52%，而 FDA 的一项研究报告称，1995 年至 1999 年批准的生物制剂中，女性的参与率为 45%。本研究表明，自 GAO 报告的 72%以来，NDA 中安全性和疗效的性别分析增加到 74%，而生物制剂的性别分析从 1995-1999 年批准的治疗性生物制剂的 37%增加到 64%。了解疾病的流行程度和参与临床试验可以帮助我们了解这些试验中患者的招募和保留模式。
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