Yang Yongsheng, Carlin Alan S, Faustino Patrick J, Motta Mónica I Pagán, Hamad Mazen L, He Ruyi, Watanuki Y, Pinnow E E, Khan Mansoor A
Food and Drug Administration, Center for Drug Evaluation and Research, Silver Spring, Maryland 20993, USA.
J Womens Health (Larchmt). 2009 Mar;18(3):303-10. doi: 10.1089/jwh.2008.0971.
This study aimed to track the inclusion of women in clinical trials for new drugs approved by the Food and Drug Administration (FDA) between 2000 and 2002 and to evaluate the extent of analyses by sex.
Data were extracted from FDA reviewers' reports, summaries of clinical trials in New Drug Applications (NDAs), and product labeling and organized into a Microsoft Access database. The information collected includes subject enrollment by sex per clinical phase and sex differences in pharmacokinetics, safety, and efficacy as determined by either sponsors or reviewers.
There were 67 New Molecular Entities (NMEs) approved by the FDA between 2000 and 2002. A total of 397,825 subjects were enrolled in 2,323 clinical trials. If 9 sex-specific NMEs are excluded, 297,697 subjects were enrolled in 1,974 clinical trials. Forty-seven percent of participants were male, 49% were female, and 4% of subjects were not specified. Of the 58 sex-nonspecific products in the study, 71% (41 of 58) of sex analyses were performed either by the sponsor or FDA reviewers. Twenty-five NMEs were found to have sex differences in pharmacokinetics, efficacy or adverse events. However, no recommendation was made to adjust dosage based on sex differences.
The percentages of women and men participating in clinical trials varied by year, phase, and product type. However, the overall participation by women and men was comparable, suggesting an improvement in including more women in clinical trials when compared with the previous FDA study evaluating women's participation from 1995 through 1999. As with the previous study, however, a significant underrepresentation of women in early phase trials and in certain areas, such as cardiovascular products, was observed and continues to be an issue of concern. Lack of appropriate analyses by sex should also be noted as an issue of concern.
本研究旨在追踪2000年至2002年间美国食品药品监督管理局（FDA）批准的新药临床试验中女性受试者的纳入情况，并评估按性别进行分析的程度。
数据从FDA审评员报告、新药申请（NDA）中的临床试验总结以及产品标签中提取，并整理到一个Microsoft Access数据库中。收集的信息包括每个临床阶段按性别划分的受试者入组情况，以及由申办方或审评员确定的药代动力学、安全性和疗效方面的性别差异。
2000年至2002年间，FDA共批准了67种新分子实体（NME）。2323项临床试验中共纳入了397,825名受试者。如果排除9种针对特定性别的NME，1974项临床试验中共纳入了297,697名受试者。47%的参与者为男性，49%为女性，4%的受试者性别未明确。在本研究的58种非性别特异性产品中，71%（58种中的41种）的性别分析由申办方或FDA审评员进行。发现25种NME在药代动力学、疗效或不良事件方面存在性别差异。然而，未基于性别差异提出调整剂量的建议。
参与临床试验的女性和男性比例因年份、阶段和产品类型而异。然而，女性和男性的总体参与情况相当，这表明与FDA之前评估1995年至1999年女性参与情况的研究相比，在临床试验中纳入更多女性方面有了改善。然而，与之前的研究一样，在早期试验以及某些领域，如心血管产品领域，女性受试者的代表性明显不足，这仍然是一个令人担忧的问题。缺乏适当的性别分析也应作为一个令人担忧的问题加以关注。
