FDA启动罕见病框架倡议：按病种与患者需求重塑药物审评 10月28日召开公众会议
华盛顿——美国食品药品监管局（FDA）10月14日宣布启动「罕见病框架倡议」，并发布配套白皮书，拟按疾病特点、患者需求、产品类型、以及小规模患者人群能够现实产生的证据，重塑罕见病药物与生物制品的开发及审评框架。FDA指出，目前仅约5%的罕见病有经FDA批准的疗法。
该倡议由FDA罕见病创新中心（Rare Disease Innovation Hub）牵头，联合专员办公室、生物制品审评与研究中心（CBER）与药品审评与研究中心（CDER）共同推进，目标包括使监管决策更清晰、更一致，在药物开发早期纳入患者声音，并对生产质量与检查采取风险导向的安排。
FDA将于10月28日在其位于马里兰州白橡（White Oak）园区的总部召开公众会议征求意见，会议由里根-尤德尔基金会（Reagan-Udall Foundation）协助举办；此后还将举行分行业圆桌并发布征求信息通知，患者、倡导组织与业界人士可参与提交意见。
FDA Launches Rare Disease Framework Initiative to Tailor Drug Development; Public Meeting Set for October 28
Washington — The Food and Drug Administration on October 14 launched its Rare Disease Framework Initiative and released an accompanying white paper, proposing to reshape how drugs and biologics for rare diseases are developed and reviewed, with standards tailored to each disease's characteristics, patient needs, product type and the evidence that small patient populations can realistically generate. The FDA noted that only about 5 percent of rare diseases currently have an FDA-approved therapy.
The initiative is led by the FDA's Rare Disease Innovation Hub, working with the Office of the Commissioner, the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER). Its goals include clearer and more consistent regulatory decisions, embedding the patient voice earlier in development, and risk-based approaches to manufacturing quality and inspections.
The FDA will hold a public meeting on October 28 at its White Oak campus in Maryland, hosted with support from the Reagan-Udall Foundation, to gather input from patients, advocates and industry, followed by sector roundtables and a request for information.
发布：GSM·美国（发布链接）
图片来源：图：位于马里兰州银泉市白橡园区的美国食品药品监管局总部入口（资料图）。图片来源：美国食品药品监管局，公共领域 / Photo: Entrance sign at the FDA White Oak campus in Silver Spring, Maryland (file photo). Source: U.S. Food and Drug Administration, public domain.
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