FDA罕见病框架倡议公开会议——2026年10月28日
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会议 | 混合形式 活动标题 FDA罕见病框架倡议公开会议 2026年10月28日 更多会议、 大会和 研讨会 FDA会议、大会和研讨会 FDA会议、大会和研讨会：往期活动 FDA会议年度报告 公开日历——与FDA官员的会议 日期： 2026年10月28日 会议录像 观看完整会议录像（视频文字记录即将发布）。 2026年10月28日，美国食品药品监督管理局（FDA）与里根-尤德尔FDA基金会合作，召开了一次混合形式的公开会议，就FDA罕见病框架倡议征求输入意见，以重新构想该机构对罕见病药物开发的处理方式。这一新倡议的意图是制定一个适用的框架，通过考虑每种疾病的细微差异、患者需求、产品类型以及在小型患者人群中现实上能够产生的证据，来反映罕见病药物开发的现实。 此次公开会议已在《联邦公报》上宣布，为罕见病社区成员——包括患者、疾病倡导者、申办方及其他人士——提供了一个就框架的方向和重点提供意见的机会。公众发言中的见解将为该机构即将开展的工作提供参考。 FDA欢迎与框架倡议相关的所有反馈，尤其征求以下主题领域的意见： 基于可获得的证据、特定疾病状态和人群对罕见病疗法进行适用性审评。 FDA药物开发与审评监管要求的一致性与可预测性。 FDA与外部各方（包括患者、疾病专家和研究人员）的接触，以为审评决策提供信息。 针对罕见病疗法的化学、生产和控制（CMC）方面的考虑。 内容截至日期： 2026年11月05日 受监管产品 药品
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Meeting | Mixed Event Title FDA Rare Disease Framework Initiative Public Meeting October 28, 2026 More Meetings, Conferences, and Workshops FDA Meetings, Conferences and Workshops FDA Meetings, Conferences, and Workshops: Past Events FDA Annual Reports on Conferences Public Calendar - Meetings With FDA Officials Date: October 28, 2026 Meeting Recording Watch the full meeting recording (video transcript coming soon). On October 28, 2026, the U.S. Food and Drug Administration, in partnership with the Reagan-Udall Foundation for the FDA, convened a hybrid public meeting to seek input related to the FDA’s Rare Disease Framework Initiative to reimagine the agency’s approach to rare disease drug development. The intent of this new initiative is to develop a fit-for-purpose Framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type, and the evidence that realistically can be generated in small patient populations. The public meeting, announced in the Federal Register, was an opportunity for members of the rare disease community – including patients, disease advocates, sponsors, and others – to provide input on the direction and focus of the Framework. Insights from the public’s remarks will inform the agency’s upcoming work. The FDA welcomes all feedback related to the Framework Initiative and in particular seeks input on the following topic areas: Fit-for-purpose review of rare disease therapies based on attainable evidence, particular disease state, and population. FDA consistency and predictability of drug development and review regulatory requirements. FDA engagements with external parties – including patients, disease experts, and researchers – to inform review decision-making. Considerations for chemistry, manufacturing, and controls (CMC) specific to rare disease therapies. Content current as of: 11/05/2026 Regulated Product(s) Drugs