The Mary Horrigan Connors Center for Women's Health and Gender Biology, Brigham & Women's Hospital, Boston, MA, United States of America; Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham & Women's Hospital, Boston, MA, United States of America; Department of Epidemiology, Harvard T.H. Chan School of Public Health, Boston, MA, United States of America.
The Mary Horrigan Connors Center for Women's Health and Gender Biology, Brigham & Women's Hospital, Boston, MA, United States of America; Department of Epidemiology, Harvard T.H. Chan School of Public Health, Boston, MA, United States of America; Division of Women's Health, Department of Medicine, Brigham & Women's Hospital, Boston, MA, United States of America; Harvard Medical School, Boston, MA, United States of America.
Contemp Clin Trials. 2022 Apr;115:106718. doi: 10.1016/j.cct.2022.106718. Epub 2022 Mar 2.
Historically, females have been underrepresented in clinical trials evaluating the safety and efficacy of investigational drugs and devices. We assessed participation by sex in recent clinical trials.
We extracted data over a 4-year period (2016-2019) from ClinicalTrials.gov on US-based, pharmaceutical industry or government-funded Phase 1-3 clinical trials of drugs and devices. We included trials with adult cardiovascular, psychiatric, and cancer endpoints whose protocol planned to enroll both sexes. Average proportions of females enrolled per trial were described overall and by disease area.
Across 1433 trials including 302,664 participants in our analysis, on average, 41.2% were female. Females were underrepresented compared with their proportion of the disease population in cardiovascular disease trials (41.9% female participants vs. 49% female population with cardiovascular disease). In psychiatry, where females comprise 60% of patients, the mean participation of females in clinical trials was 42.0%. Similarly, for cancer trials, where 51% of patients are female, only 41.0% of cancer clinical trial participants were female. For each therapeutic area analyzed, the participation of females in clinical trials fell short of the benchmark derived from national prevalence data.
While the participation of females in clinical trials has improved compared to previous reports, sex-based gaps still persist between trial populations and those expected to use these drugs/devices based on distributions of diseases in the population. Given potential sex-based differences in treatment responses and toxicities, adequate inclusion of females in clinical trials remains critical.
历史上，女性在评估研究药物和器械安全性和疗效的临床试验中代表性不足。我们评估了近期临床试验中女性的参与情况。
我们从 ClinicalTrials.gov 提取了 4 年（2016-2019 年）的数据，包括美国制药业或政府资助的 1-3 期药物和器械临床试验，这些试验有成人心血管、精神和癌症终点，方案计划招募男女患者。总体和按疾病领域描述了每个试验中纳入的女性比例。
在我们分析的 1433 项试验中，有 302664 名参与者，平均有 41.2%是女性。与心血管疾病患者中女性的比例相比，女性在心血管疾病试验中代表性不足（女性参与者占 41.9%，而心血管疾病女性患者占 49%）。在精神科，女性占患者的 60%，临床试验中女性的平均参与率为 42.0%。同样，在癌症试验中，51%的患者是女性，只有 41.0%的癌症临床试验参与者是女性。对于分析的每个治疗领域，临床试验中女性的参与都低于根据人群中疾病分布得出的基准。
尽管与之前的报告相比，女性在临床试验中的参与有所提高，但试验人群与预计根据人群中疾病分布使用这些药物/器械的人群之间仍然存在基于性别的差距。鉴于治疗反应和毒性方面可能存在基于性别的差异，充分纳入女性参与临床试验仍然至关重要。
