1 Office of Translational Sciences, Center for Drug Evaluation and Research , Food and Drug Administration, Silver Spring, Maryland.
2 Office of Women's Health, Office of the Commissioner, Food and Drug Administration , Silver Spring, Maryland.
J Womens Health (Larchmt). 2018 Apr;27(4):418-429. doi: 10.1089/jwh.2016.6272. Epub 2017 Oct 19.
The U.S. Food and Drug Administration (FDA) has made efforts to encourage adequate assessment of women, racial/ethnic minorities, and geriatric participants in clinical trials through regulations and guidance documents. This study surveyed the demographics of clinical trial participants and the presence of efficacy and safety analyses by sex for new drugs approved between 2013 and 2015 by the FDA Center for Drug Evaluation and Research.
New drug marketing applications submitted to FDA were surveyed for demographic data (sex, race, ethnicity, and age) and the presence of sex-based analyses for efficacy and safety. The Ratio of the Proportion of women in clinical trials for the indicated disease population relative to the estimated Proportion of women in the disease population (PPR) was calculated for new drug indications.
Of the 102 new drugs in this cohort (defined as new molecular entity drugs and original therapeutic biologics), sex was reported for >99.9% of trial participants, and women accounted for 40.4% of these participants. An estimated 77.2% of participants were White, 6.4% were Black/African American, and 29.1% were aged ≥65 years. Sex-based analyses for both efficacy and safety were conducted for 93.1% of applications. PPR was calculated for 82 new drugs for a total of 60 indications, of which 50 indications (83.3%) had a PPR ≥0.80.
Sex data are now collected for almost all study participants, and this study shows appropriate sex participation for most new drugs when estimated disease prevalence by sex (PPR) is considered. Therapeutic area and disease indication are important considerations when assessing the sex of participants because variation occurs depending on the disease under study. Some racial minorities, especially Blacks/African Americans, are still not well represented in most drug development programs and remain an area where improvement is needed.
美国食品和药物管理局（FDA）通过法规和指导文件，努力鼓励在临床试验中充分评估女性、种族/少数民族和老年参与者。本研究调查了 2013 年至 2015 年期间 FDA 药物评价与研究中心批准的新药临床试验参与者的人口统计学特征，以及按性别进行疗效和安全性分析的情况。
对提交给 FDA 的新药营销申请进行了调查，以获取人口统计学数据（性别、种族、民族和年龄）以及按性别进行疗效和安全性分析的情况。计算了新适应证药物的临床试验中女性比例（PPR）与估计疾病人群中女性比例的比值。
在该队列的 102 种新药（定义为新分子实体药物和原始治疗性生物制剂）中，试验参与者的性别报告比例超过 99.9%，女性占这些参与者的 40.4%。估计 77.2%的参与者为白人，6.4%为黑人/非裔美国人，29.1%为年龄≥65 岁。93.1%的申请都进行了疗效和安全性的性别分析。共对 82 种新药的 60 个适应证计算了 PPR，其中 50 个适应证（83.3%）的 PPR≥0.80。
现在几乎所有研究参与者的性别数据都已收集，当考虑按性别估计的疾病流行率（PPR）时，本研究表明大多数新药的性别参与是适当的。治疗领域和疾病适应证是评估参与者性别的重要考虑因素，因为这取决于所研究的疾病。一些少数族裔，尤其是黑人/非裔美国人，在大多数药物开发项目中仍然代表性不足，这仍然是需要改进的领域。
