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01
US FDA White Paper on Key Considerations for the Development and Use of Digitally Derived Measures for Clinical Investigations
美国 FDA 关于临床研究中数字化衍生测量指标开发与使用关键考虑因素的白皮书
The US FDA has released a white paper on Key Considerations for the Development and Use of Digitally Derived Measures for Clinical Investigations.
美国 FDA 已发布白皮书：《临床研究中数字化衍生测量指标开发与使用的关键考虑因素》。
The document focuses on outcomes that matter to patients to ensure the technology is fit-for-purpose. FDA emphasized that digitally derived endpoints must be validated in the actual study population, including relevant age groups, disease severity, and patient characteristics. Sponsors should define how missing data will be handled at every level of the trial, not just the patient level, to ensure endpoints are patient-centered and clinically meaningful. FDA stressed that a cleared device does not automatically validate a clinical endpoint.
该文件聚焦于对患者重要的结局指标，以确保相关技术适合其预期用途。FDA强调，数字化衍生终点必须在实际研究人群中进行验证，包括相关年龄组、疾病严重程度和患者特征。申办方应明确如何处理试验每个层级（而不仅仅是患者层级）的缺失数据，以确保终点以患者为中心且具有临床意义。FDA强调，器械获得许可（cleared）并不自动意味着某个临床终点已经得到验证。
02
US FDA Releases Final Guidance on FAQ: Developing Potential Cellular and Gene Therapy Products
美国 FDA发布关于“开发潜在细胞和基因治疗产品”常见问题的最终指导原则
The US FDA has released a final guidance titled: Frequently Asked Questions —Developing Potential Cellular and Gene Therapy Products. FDA is providing greater transparency and operational clarity for cellular and gene therapy (CGT) sponsors, while simultaneously raising expectations around development readiness, manufacturing control, and evidence generation. Companies that engage FDA early and build regulatory, CMC, and clinical strategies in parallel are likely to be better positioned for accelerated development and approval.
美国 FDA已发布题为《常见问题——开发潜在细胞和基因治疗产品》的最终指导原则。FDA正在为细胞和基因治疗（CGT）申办方提供更高的透明度和更明确的操作说明，同时提高对开发准备程度、生产控制和证据生成的期望。尽早与 FDA 沟通并同步构建法规、CMC 和临床策略的公司，可能更有利于加速开发和获批。
03
EMA Releases Sponsor Guidance: New Safety Module on the Annual Safety Report
EMA 发布申办方指南：年度安全报告的新安全模块
This EMA guidance introduces the new CTIS Safety Module for Annual Safety Reports (ASRs, formerly DSURs), which will become the mandatory submission route for new ASRs beginning 28 September 2026, enabling harmonized submission, assessment, and management of safety reports across EU/EEA clinical trials. It provides sponsors with detailed instructions on user roles, ASR creation and submission, management of Requests for Information (RFIs), transition from the legacy ASR process, and key system requirements and known issues within CTIS. This guidance is operational in nature and is intended to help sponsors prepare for the September 2026 CTIS Safety Module go-live.
本EMA指南介绍了用于年度安全报告的新CTIS安全模块（ASR，原为DSUR）。自2026年9月28日起，该模块将成为新ASR的强制提交途径，从而实现欧盟/欧洲经济区（EU/EEA）临床试验安全报告提交、评估和管理的协调统一。指南向申办方详细说明了用户角色、ASR的创建与提交、信息请求（RFI）的管理、从原有ASR流程向新流程的过渡，以及CTIS内的关键系统要求和已知问题。本指南具有操作性，旨在帮助申办方为2026年9月CTIS安全模块上线做好准备。
The module introduces dedicated Safety roles (Sponsor Safety Admin and ASR Submitter) with role-based access controls.
该模块引入专门的安全角色（申办方安全管理员〔Sponsor Safety Admin〕和ASR提交者〔ASR Submitter〕），并采用基于角色的访问控制。
The new CTIS Safety Module replaces the current ASR functionality, with a 2-month transition period ending 28 November 2026. Sponsors can submit either Clinical Trial-centric or Substance Group-centric ASRs, with SG-centric submissions expected to be used most frequently. After submission, ASRs undergo coordinated Member State assessment, and sponsors must respond to any RFIs within the defined timelines.
新的CTIS安全模块取代现有ASR功能，并设有为期2个月、于2026年11月28日结束的过渡期。申办方可提交以临床试验为中心（Clinical Trial-centric）或以物质组为中心（Substance Group-centric）的ASR，预计以SG为中心的提交方式使用最为频繁。提交后，ASR将接受成员国协调评估，申办方必须在规定时限内回复任何RFI。
ASR documents and supporting materials submitted through the module are not publicly disclosed, although they are available to relevant EU Member State authorities.
通过该模块提交的ASR文件和支持材料不向公众公开，但相关欧盟成员国主管机构可以查阅。
04
US FDA Issues 3 Final Guidance on Cancer Clinical Trial Eligibility Criteria
美国FDA发布3项关于癌症临床试验入选标准的最终指导原则
The FDA has released a series of guidance that provide recommendations regarding eligibility criteria for clinical trials of investigational drugs regulated by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research for the treatment of cancer, although many of the concepts may apply more broadly to other clinical areas. They are intended to assist interested parties, including sponsors, clinical investigators and institutional review boards, who are responsible for the development and/or oversight of clinical trials.
FDA已发布一系列指导文件，就由药物评价与研究中心（Center for Drug Evaluation and Research）和生物制品评价与研究中心（Center for Biologics Evaluation and Research）监管、用于癌症治疗的研究性药物临床试验入选标准提出建议，尽管其中许多概念也可能更广泛地适用于其他临床领域。这些指导文件旨在协助负责临床试验开发和/或监督的相关方，包括申办方、临床研究者和机构审查委员会。
Cancer Clinical Trial Eligibility Criteria: Performance Status
Cancer Clinical Trial Eligibility Criteria: Washout Periods and Concomitant Medications
Cancer Clinical Trial Eligibility Criteria: Laboratory Values
05
Recent FDA Draft Guidance Documents: Master Protocols, Substantial Evidence & Quantitative Systems Pharmacology
FDA近期指导原则草案：主方案、充分的有效性证据与定量系统药理学
The US FDA has released several FDA draft guidance recently:
美国 FDA 近期发布了数份 FDA 指导原则草案：
Master Protocols for Drug and Biological Product Development provides recommendations on the design and analysis of trials conducted under a master protocol as well as guidance on the submission of documentation to support regulatory review.
《药物和生物制品开发的主方案》就依据主方案开展的试验的设计和分析提出建议，并就提交支持监管审评的文件提供指导。
Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry. Advances in our understanding of biological processes and the increasing availability of high-quality data have transformed the evidentiary landscape for drug development. Given these advances, the draft guidance discusses the many factors that can impact the strength of evidence of effectiveness for a drug and clarifies how sponsors can rely on one adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the substantial evidence of effectiveness standard. When final, this guidance will replace the 1998 guidance titled "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products."
《证明人用药物和生物制品有效性的充分证据：行业指导原则草案》。随着我们对生物过程认识的进步以及高质量数据可获得性的提高，药物开发的证据环境已经发生转变。鉴于这些进展，该指导原则草案讨论了可能影响药物有效性证据强度的诸多因素，并阐明申办方如何依靠一项充分且良好对照的临床研究，结合确证性证据，来满足充分的有效性证据标准。该指南定稿后，将取代1998年题为《为人用药物和生物制品提供有效性的临床证据》的指南。
声明：本文信息以内容定稿当日为准，如后续有所变更，以官方信息为准。
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