Aroga Biosciences, Inc, 10717 Sorrento Valley Road, San Diego, CA, 92121, USA.
Ther Innov Regul Sci. 2024 Jul;58(4):704-713. doi: 10.1007/s43441-024-00643-4. Epub 2024 Apr 3.
Recent Food and Drug Administration (FDA) draft guidelines are intended to improve representation and formalize the assessment of race and ethnicity in drug development, but how regulators and industry stakeholders plan to implement and enforce new requirements is still being determined.
Here, a 10-question survey was developed to assess the experiences of industry stakeholders in developing diversity plans. These survey results informed an analysis of postmarketing studies to understand how diversity requirements have been enforced to date.
Among 13 survey responders, experience submitting and receiving feedback on diversity plans was limited. A variety of challenges have been associated with developing these plans, including questions regarding regulatory guidance. Sponsors have utilized several data sources, including real-world datasets, to define enrollment goals. Diversity-related postmarketing studies most commonly related to oncologic diseases, and endpoints frequently related to efficacy. Most marketing applications associated with diversity-related postmarketing studies received Orphan drug designation (ODD) and Accelerated Approval.
These results show that industry experience with diversity plans remains limited in the absence of finalized regulatory guidance. Sponsors are beginning to develop strategies for submitting diversity plans, which include identifying key functions and data sources to support enrollment goals, although definitive conclusions were difficult to draw from the small responder pool. In the postmarketing setting, studies are already underway to improve the understanding of racial and ethnic differences in responses to approved drugs. Development programs relating to oncology, which has historically suffered from a lack of diverse representation, have been a primary focus of such studies thus far.
最近美国食品和药物管理局（FDA）的指导草案旨在改善药物开发中种族和民族的代表性，并使对其的评估正式化，但监管机构和行业利益相关者计划如何实施和执行新要求仍在确定中。
在这里，开发了一个包含 10 个问题的调查来评估行业利益相关者制定多样性计划的经验。这些调查结果为理解迄今为止如何执行多样性要求的上市后研究分析提供了信息。
在 13 名调查回应者中，提交和接收多样性计划反馈的经验有限。制定这些计划存在各种挑战，包括对监管指导的疑问。赞助商利用了多种数据源，包括真实世界数据集，来定义入组目标。与多样性相关的上市后研究最常涉及肿瘤疾病，终点通常与疗效相关。与多样性相关的上市后研究相关的大多数营销申请都获得了孤儿药指定（ODD）和加速批准。
在没有最终监管指导的情况下，这些结果表明行业在多样性计划方面的经验仍然有限。赞助商正在开始制定提交多样性计划的策略，其中包括确定支持入组目标的关键功能和数据源，尽管从小的回应者群体中很难得出明确的结论。在上市后环境中，已经有研究正在进行，以提高对批准药物反应中种族和民族差异的理解。迄今为止，此类研究的主要重点一直是涉及肿瘤学的开发项目，该领域历来缺乏多样性代表性。
