食品药品监管局发布细胞与基因治疗效力保障最终指南：取代2011年旧指南 逐批验证效力
美国食品药品监督管理局（FDA）2026年11月2日在华盛顿发布最终指南，要求细胞与基因治疗（CGT）产品生产商实施一套全面、以科学和风险为基础的效力保障计划，确保产品在抵达患者之前，每一批次都达到既定的效力标准。
这份题为《细胞与基因治疗产品效力保障》的最终指南取代 FDA 2011 年的效力检测指南，适用于治疗严重乃至危及生命疾病的疗法。指南给出「效力保障策略」的建议，引导生产商通过检测与验证，确认每一批次产品都能达到其预期治疗作用所定义的效力标准。依法，生产商须从产品获批时起、直至上市后监测全程，验证产品按预期发挥作用。
FDA 生物制品评价与研究中心主任 Karim Mikhail 表示：「接受细胞与基因治疗的患者往往正与癌症、罕见病等严重或危及生命的疾病抗争，他们理应确信所用产品会按预期起效。这份指南体现了 FDA 的承诺：确保美国始终是世界上最适合创新的地方，并让创新与最高的产品质量和患者安全标准相匹配。」
指南为生产商提供了灵活、基于风险的建议，涵盖如何把效力考量融入研发与商业化的每个阶段：从早期临床试验申请（IND）、生物制品许可申请（BLA）获批，一直到上市后的持续验证；重点包括原材料变异的控制、生产过程中的检测，以及确认每批产品生物活性与预期疗效相符的实验室检测。指南还针对保质期短的产品、病毒载体基因疗法和患者个体化疗法给出专门考量。
FDA 表示，清晰可预期的路线图有助于让没有其他治疗选择的患者更快获得安全有效的疗法，其中许多产品是为单个患者单独生产的。FDA 将于2026年12月10日举办网络研讨会，帮助相关方理解并实施这份指南。
（来源：美国食品药品监督管理局）
FDA Issues Final Potency Assurance Guidance for Cell and Gene Therapies, Replacing 2011 Guidance
The U.S. Food and Drug Administration (FDA) on November 2, 2026, in Washington issued final guidance requiring manufacturers of cellular and gene therapy (CGT) products to implement a comprehensive, science- and risk-based plan to make sure their products meet potency standards, verifying before a product reaches a patient that every batch meets a defined standard of potency to do what the product is supposed to do therapeutically.
The final guidance, titled Potency Assurance for Cellular and Gene Therapy Products, replaces the FDA's 2011 potency testing guidance and applies to treatments for serious and often life-threatening conditions. By law, manufacturers are expected to verify that their products work as intended, from the time of approval through post-approval monitoring, and the guidance sets out the FDA's current thinking and recommendations on how manufacturers can meet that requirement.
"Patients who receive cellular and gene therapies are often battling serious or life-threatening conditions, such as cancers and rare diseases, and they deserve the assurance that the product administered to them will perform as intended," said Karim Mikhail, director of the Center for Biologics Evaluation and Research. "This guidance reflects the FDA's commitment to ensuring America remains the best place in the world to innovate — and that innovation is matched by the highest standards of product quality and patient safety."
The guidance provides flexible, risk-based recommendations on integrating potency considerations at every stage of development and commercialization, from early-phase investigational new drug (IND) submissions through biologics license application (BLA) approval and continued post-market verification. Key topics include controls for variability in raw materials, testing during manufacturing, and lab tests that confirm each batch is biologically active in a way that matches its intended therapeutic effect. It also addresses specialized considerations for products with short shelf lives, viral vector gene therapies, and patient-specific therapies.
The FDA said the clear, predictable roadmap supports faster access to safe and effective therapies for patients who often have no other treatment options, many of whom receive products made individually for a single person. The agency will host a webinar on December 10, 2026, to help stakeholders understand and implement the guidance.
(Source: U.S. Food and Drug Administration)
来源：美国食品药品监督管理局
发布：GSM·美国（发布链接）
图片来源：图：美国食品药品监督管理局总部大楼（固定图库）。 / Photo: Headquarters of the U.S. Food and Drug Administration (agency file photo).
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