食品药品监管局发布罕见病药物研发年度报告：加速罕见病治愈计划公布 2026 年进展
美国食品药品监管局（FDA）10 月 22 日宣布，旗下药品审评与研究中心的「加速罕见病治愈计划」（Accelerating Rare disease Cures，简称 ARC）发布 2026 年年度报告，题为《持续进展，在罕见病药物研发中拓展可能》（Sustaining Progress, Expanding Possibilities in Rare Disease Drug Development）。
FDA 介绍，该报告记录了过去一年罕见病药物研发领域在科学与监管创新、与患者和研发方的沟通协作以及计划运营方面取得的进展。ARC 计划是 FDA 药品审评与研究中心设立的跨部门计划，目的是通过加强科学、监管与政策层面的协作，加快为罕见病患者开发安全有效的治疗药物。
罕见病在美国指患病人数少于 20 万的疾病。据 FDA 以往公布的资料，美国有 7,000 多种罕见病，患者总数估计在 2,500 万至 3,000 万之间，但其中绝大多数疾病尚无 FDA 批准的治疗药物。FDA 表示，公众可通过其官网查阅 ARC 计划 2026 年年度报告全文，了解该计划本年度的工作与未来展望。
FDA Releases 2026 Annual Report on Rare Disease Drug Development: ARC Program Documents a Year of Progress
The U.S. Food and Drug Administration (FDA) announced on October 22 that the Accelerating Rare disease Cures (ARC) Program, run by its Center for Drug Evaluation and Research, has released its 2026 Annual Report, titled Sustaining Progress, Expanding Possibilities in Rare Disease Drug Development.
According to the FDA, the report documents a year of scientific and regulatory innovation, meaningful engagement with patients and drug developers, and operational progress in rare disease drug development. The ARC Program is a cross-center FDA initiative intended to speed the development of safe and effective treatments for rare diseases by strengthening collaboration across scientific, regulatory, and policy work.
In the United States, a rare disease is defined as one affecting fewer than 200,000 people. FDA materials published previously put the number of known rare diseases at more than 7,000, with an estimated 25 million to 30 million Americans affected in total, while the large majority of those conditions still have no FDA-approved treatment. The FDA said the full text of the ARC Program's 2026 Annual Report is available on its website, covering the program's work over the past year and its outlook.
来源：GSM·美国（原文链接）
图片来源：图：实验室科研人员进行病毒样本转移操作（资料图）。照片：James Gathany，美国疾病控制与预防中心，公共领域，经 Wikimedia Commons。 / A laboratory scientist transfers virus samples (file photo). Photo: James Gathany, CDC, public domain, via Wikimedia Commons.
