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Regulatory Express_Oct. 2025

2025-10-29 19:06:31

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EMA患者体验数据反馈文件草案

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EMA发布了患者体验数据的征求意见稿，供公众咨询。患者体验数据为药品监管机构和医疗系统中的其他决策者提供了宝贵的见解，让他们了解对患者而言最重要的因素，这些因素超出了其他已确立的科学成果。该文件的公众咨询期截至2026年1月31日。

这份征求意见稿是为医药研发人员、患者群体、研究人员和其他决策者准备的。

它鼓励药品研发人员在药品的整个生命周期中收集并纳入反映患者现实生活观点和偏好的数据，并描述如何生成、收集和分析这些数据的一般原则。

EMA Draft Reflection Paper on Patient Experience Data

The EMA published a draft reflection paper on patient experience data for public consultation. Patient experience data provides medicines regulators and other decision-makers in healthcare systems with valuable insights into what matters most to patients, beyond other well-established scientific outcomes. The paper is open for public consultation until Jan. 31, 2026.

The reflection paper is intended for medicine developers, patient groups, researchers and other decision-makers.

It encourages medicines' developers to gather and include data reflecting patients' real-life perspectives and preferences throughout the lifecycle of medicines, and describes general principles on how to generate, collect and analyze the data.

ICH通过了 ICH E2D(R1) 有关上市后安全性数据的准则

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关于“上市后安全性数据：个例安全性报告的管理和报告的定义和标准”的ICH E2D（R1）指导原则，于2025年9月15日进入ICH进程的第四阶段，目前已进入第五阶段。 ICH于2003年11月通过了E2D的第一版。自本指导原则发布后，上市后安全性信息有了新来源或更常被引用。这些来源包括社交媒体、市场研究项目以及患者支持和援助项目。但是，如ICH概念章节中所述，它们在特性和对上市后安全性信息质量的贡献方面各不相同。

本指导原则旨在使不良事件相关术语标准化，并简化收集和报告上市后安全性数据的程序和时间表。

ICH Adopts ICH E2D(R1) on Post-Approval Safety Data

The ICH E2D(R1) Guideline on "Post-Approval Safety Data: Definitions and Standards for Management and Reporting of Individual Case Safety Reports" has reached Step 4 of the ICH Process on 15 September 2025 and has now entered the implementation phase at Step 5 of the ICH Process. The ICH adopted the first version of E2D in November 2003. Since the release of this guideline, new sources of post-approval safety information have emerged or have become more commonly used. These sources include social media, market research programs, and patient support and assistance programs. However, they vary in their characteristics and contributions to the quality of post-approval safety information, as noted in an ICH concept paper.

The guideline aims to standardize terminology related to adverse events and streamline procedures and timelines for collecting and reporting post-approval safety data.

FDA发布关于计算机软件保障的最终指南

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FDA已发布指南，为用作医疗器械生产或质量体系一部分的计算机和自动化数据处理系统提供计算机软件保障建议。

FDA Releases Final Guidance on Computer Software Assurance

FDA has issued guidance to provide recommendations on computer software assurance for computers and automated data processing systems used as part of medical device production or the quality system.

US FDA指南草案：细胞和基因疗法产品上市后获取安全性及有效性数据的方法

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美国FDA发布了一份标题为“细胞和基因疗法产品上市后获取安全性及有效性数据的方法”的指南草案。本指南的目的是讨论获取细胞和基因治疗(CGT)产品上市后安全性和有效性数据的方法和途径。其不涉及为扩展临床适应症而收集的数据。

考虑到CGT产品的潜在持久效应，以及为支持CGT产品上市而进行的临床试验中接受治疗的受试者数量通常有限，上市后监测对于随着时间的推移收集产品安全性和有效性的数据非常重要。

鼓励申办方主动计划稳健的上市后数据收集，以支持CGT产品的持续评价。这对于了解长期安全性和有效性至关重要，尤其是在真实世界中。

US FDA Draft Guidance: Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products

The US FDA has issued a draft guidance titled: Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products. The purpose of this guidance is to discuss methods and approaches for capturing postapproval safety and efficacy data for cell and gene therapy (CGT) products. It does not address data collected for the purpose of expanding clinical indications.

Given the potential for long-lasting effects of CGT products, and the generally limited number of participants treated in clinical trials conducted to support approval of CGT products, postapproval monitoring is important for gathering data on product safety and effectiveness over time.

Sponsors are encouraged to proactively plan for robust postapproval data collection to support the ongoing evaluation of CGT products. This is critical for understanding long-term safety and efficacy, especially in real-world settings.

US FDA关于针对小样本量人群的细胞和基因治疗产品在临床试验中的创新设计的指南草案

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美国FDA已经发布了关于针对小样本量人群的细胞和基因治疗产品的临床试验的创新设计的指南草案。该指南为计划开展此类细胞和基因疗法（CGT）产品临床试验的申办方提供了建议，这些产品旨在用于影响小样本量人群的疾病或状况——通常符合《联邦食品、药品和化妆品法案》（FD&C 法案）第 526(a)(2) 条（21 美国法典第 360bb(a)(2) 条）所定义的罕见疾病或状况的标准。该指南描述了 FDA 的要求，并提供了关于使用各种临床试验设计和终点以生成支持产品许可的临床证据的考虑因素。

该指南进一步阐述了FDA此前相关主题指导文件中所阐述的原则，并针对细胞和基因疗法试验的规划、设计、实施和分析提供了更多建议，以协助 FDA 对产品效果进行评估。

本指南草案鼓励申办方尽早与FDA接触，并考虑采用创新、灵活和科学严谨的试验设计，以适应小样本量人群中CGT开发的独特挑战。

US FDA Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations

The US FDA has released a Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. This guidance provides recommendations to sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population—generally one that meets the definition of a rare disease or condition under section 526(a)(2) of the FD&C Act (21 U.S.C. 360bb(a)(2)). It describes FDA requirements and provides considerations for the use of various clinical trial designs and endpoints to generate clinical evidence to support product licensure.

This guidance expands on principles described in FDA's existing guidance documents related to this topic, by providing additional recommendations for the planning, design, conduct, and analysis of cell and gene therapy trials to facilitate FDA's assessment of product effectiveness.

This draft guidance encourages sponsors to engage early with FDA and consider innovative, flexible, and scientifically rigorous trial designs tailored to the unique challenges of CGT development in small populations.

器械检查显示执法力度将加大：Hogan Lovells分析报告

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Hogan Lovells律师事务所发布了一份对美国食品药品管理局（FDA）2025年上半年医疗器械执法活动的分析报告。

该分析和相关见解表明，FDA正在加强监管力度，通过发布更多警告函和依靠人工智能(AI) 来确定检查的优先顺序。

Device Inspections Show Tougher Enforcement: Hogan Lovells Analysis

The law firm Hogan Lovells has released an analysis of the United States Food & Drug Administration’s (FDA) medical device enforcement activities for the first half of 2025.

The analysis and associated insights suggest that FDA is escalating oversight by issuing more Warning Letters and relying on Artificial Intelligence (AI) to prioritize inspections.

TransCelerate 研究分享见解，为优化临床试验数据收集提供策略指导

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TransCelerate BioPharma和塔夫茨大学医学院合作开展了一项新的研究，标题为：为优化临床试验数据收集提供见解并制定策略，旨在分析临床试验中收集的数据的数量、目的和影响——尤其是来自非核心和非必要程序的数据——并找出优化方案设计的机会。

根据来自14家生物制药公司的105项II期和III期方案的数据，研究发现，近三分之一的试验流程和收集的相关数据并不直接支持主要终点或关键次要终点。虽然总体数据量继续增加——III期试验平均数据点数已达 596 万——但这些低值数据显著增加了临床试验机构工作量和患者参与负担。

申办方公司面临收集越来越多临床试验数据的诸多压力。这项研究的结果为未来的数据收集策略提供了令人信服的见解。申办方现在有证据帮助改变传统的方案设计和执行方式，并有助于提高临床试验的绩效、效率、研究中心和患者参与度。

TransCelerate Study Shares Insights Informing Strategies for Optimizing the Collection of Clinical Trial Data

A new new collaborative study with TransCelerate BioPharma and the Tufts University School of Medicine titled : Insights Informing Strategies for Optimizing the Collection of Clinical Trial Data study was designed to analyze the volume, purpose, and impact of data collected in clinical trials — particularly data stemming from non-core and non-essential procedures — and to identify opportunities to optimize protocol design.

Drawing on data from 105 Phase II and III protocols across 14 biopharmaceutical companies, the study finds that nearly one-third of procedures and the associated data collected doesn't directly support primary objectives or key secondary endpoints. While overall data volume continues to climb — Phase III protocols now average 5.96 million data points — these lower-value data contribute significantly to the site workload and patient participation burden.

Sponsor companies face a myriad of pressures to gather increasing amounts of clinical trial data. The results of this study provide compelling insights informing future data collection strategies. Sponsors now have evidence to help transform legacy protocol design and execution practices, and help improve clinical trial performance, efficiency, site, and patient participation.

美国FDA生物仿制药开发的最终指南：比较性分析评估和质量标准

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美国FDA发布了生物仿制药开发的最终指南：比较性分析评估和质量标准。本指南描述了监管机构关于对比分析研究设计和评价的建议，旨在支持支持拟定治疗性蛋白质产品与公共卫生服务法案（PHS法案）第351(a)节许可的参比产品进行生物相似性证明的论证。此外，本指南旨在为申办方提供关于根据《公共卫生法案》第351(k)部分提交的拟定产品上市申请的化学、生产和控制(CMC)部分的科学和技术信息的建议。本指南最终确定并替代2019年5月22日发布的同一标题的指南草案，并替代2015年4月30日发布“证明治疗性蛋白产品与参比产品生物相似性的质量考虑” 的最终指南。

本指南为生物仿仿制药产品的开发提供了建议产品的开发提供了建议，重点关注全面比较分析评估的重要性，以证明与已批准的参比制剂的生物相似性。它强调质量属性和分析数据是建立生物相似性的核心。总体目标是通过严格的分析评估确保生物仿制药符合高质量标准，最终为参比生物制剂提供安全有效的替代品，并增加生物治疗的可及性。

US FDA Final Guidance on Development of Biosimilars: Comparative Analytical Assessment and Quality

The US FDA has released a final guidance on the Development of Biosimilars: Comparative Analytical Assessment and Quality. This guidance describes the Agency's recommendations on the design and evaluation of comparative analytical studies intended to support a demonstration that a proposed therapeutic protein product is biosimilar to a reference product licensed under section 351(a) of the Public Health Service Act (PHS Act). Additionally, this guidance is intended to provide recommendations to sponsors on the scientific and technical information for chemistry, manufacturing, and controls (CMC) portion of a marketing application for a proposed product submitted under section 351(k) of the PHS Act. This guidance finalizes and replaces the draft guidance of the same title issued on May 22, 2019, and replaces the final guidance "Quality Considerations in Demonstrating Biosimilarity of a Therapeutic Protein Product to a Reference Product" issued on April 30, 2015.

This guidance provides recommendations for the development of biosimilar products, focusing on the importance of a comprehensive comparative analytical assessment to demonstrate biosimilarity to an approved reference product. It emphasizes that quality attributes and analytical data are central to establishing biosimilarity. The overall goal is to ensure that biosimilars meet high-quality standards through rigorous analytical assessment, ultimately providing safe and effective alternatives to reference biologics and increasing access to biologic therapies.

FDA发布关于使用远程监督工具以协助帮助批准药物的最终指南

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FDA已经发布了关于该机构打算如何提前使用替代工具，代替或辅助预先审批和预先许可检查的最终指南，以远程评估在待批申请中所提及的药物生产设施，作为FDA药物审查流程的一部分。

FDA Issues Final Guidance on Using Remote Oversight Tools to Help Approve Drugs

FDA has issued a final guidance on how the agency intends to use alternative tools in advance, in lieu of, or in support of preapproval and prelicense inspections to remotely evaluate drug manufacturing facilities named in pending applications as part of FDA’s drug review process.

美国FDA承诺向申办方发放回复函后会立即公布完整回复内容

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美国食品药品监督管理局(fda)今天宣布，将在发送给申办方之后立即发布未来出具的完整回复函(CRL)。在 7 月初已发布了约 200 份针对先前获批产品的 CRL。

该机构还发布了89份2024年至今未发表的与待定或撤回申请相关的CRL。这些CRL中的每一个都详细描述了FDA确定的导致申请无法获得批准的具体安全性和有效性缺陷。今后，监管机构将及时发布新发布的CRL，并在批准申请时发布与该申请相关的所有CRL。该机构还将公布之前发布的与撤回或放弃申请相关的CRL批次。

FDA认为，发布CRL对公共健康具有重要意义，包括赋予药物研发者权力以避免常见的错误，并有效地为美国公众带来更多的治愈药物和有意义的治疗方法；为患者和治疗他们的医疗保健提供者提供更深入的了解；确保申办方在与投资者和股东的沟通中提供完整且具有背景信息的内容。将对所有CRL进行编辑，以删除机密商业信息、商业秘密和个人隐私信息，但将保留公司名称。

决策信函作为集中数据集的一部分，可在开放食品药品信息平台（openFDA）上供公众查阅。

US FDA Pledges to Release Complete Response Letters Promptly After Issuing to Sponsors

The U.S. Food and Drug Administration today announced that it will release future complete response letters (CRLs) promptly after they are issued to sponsors. This follows the release of approximately 200 CRLs in early July of previously approved products.

The agency also released 89 previously unpublished CRLs issued from 2024 to the present associated with pending or withdrawn applications. Each of these CRLs detail specific safety and effectiveness deficiencies identified by the FDA as preventing the application from receiving approval. Going forward, the agency will promptly release newly issued CRLs, and when approving applications will release all CRLs associated with that application. The agency will also publish batches of previously issued CRLs associated with withdrawn or abandoned applications

The FDA believes that publishing CRLs offers important benefits for public health, including empowering drug developers to avoid common missteps and efficiently bring more cures and meaningful treatments to the American public; delivering greater insight to patients and the health care providers who treat them; and ensuring sponsors provide complete and contextualized information in communications to investors and shareholders. All CRLs will be redacted to remove confidential commercial information, trade secrets, and personal private information, but will contain company names.

Decision letters are accessible to the public as a centralized dataset at openFDA.

美国FDA提供了7年临床试验质量管理规范检查结果分析

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美国食品药品监督管理局（FDA）发布了一份关于美国食品药品监督管理局在药品上市申请中执行良好临床实践（GCP）情况的七年分析报告。该研究对 2017 年至 2023 年期间进行的 GCP 检查进行了分析，分析内容包括检查的特征和结果。

大多数最终OAI分类是基于有因GCP检查，而不是基于审查的GCP检查
从2017年(23.5%)至2023年(10.4%)，FDA 483表发布的机构百分比下降
7年期间每年完成的检查总数各不相同，2017年最多（527次检查），2020年最少（284次检查）
在7年期间，按机构类型的检查分布保持相对一致；每年，大多数GCP检查针对临床研究者(82.3%-90.0%)、申办方(7.2%-11.9%)、CRO(2.8%-6.0%)、申办方研究者(0%-0.4%)
在 7 年的时间段内，完成的 GCP 检查中有 17.5%被开具了 FDA 483 表格。。按年份统计，2017年FDA 483表发布的检查比例最高(23.5%)。该百分比通常随时间推移而降低，2022 年和 2023 年的最低比例分别为 12.4%和 10.4%

US FDA provides a 7-Year Analysis of Good Clinical Practice Inspection Outcomes

The US FDA has published a 7-Year Analysis of U.S. FDA Good Clinical Practice Inspection Outcomes for Marketing Applications. The study reviewed GCP inspections conducted from calendar year 2017 to 2023 were analyzed by characteristics and outcomes.

The majority of final OAI classifications are driven by for-cause GCP inspections rather than review-based GCP inspections
The percentage of establishments issued a Form FDA 483 decreased from 2017 (23.5%) to 2023 (10.4%).
The total number of inspections completed per year varied over the 7-year time period, with the maximum occurring in 2017 (527 inspections) and the minimum in 2020 (284 inspections).
The distribution of inspections by establishment type remained relatively consistent over the 7-year period; each year, the majority of GCP inspections were for Clinical Investigators (82.3 to 90.0%), Sponsors (7.2 to 11.9%), CROs (2.8 to 6.0%), Sponsor Investigators (0 to 0.4%)
A Form FDA 483 was issued for 17.5% of GCP inspections completed during the 7-year time period. By year, the highest percent of inspections with a Form FDA 483 issued occurred in 2017 (23.5%). This percentage generally decreased over time, with the lowest percent issued in 2022 (12.4%) and 2023 (10.4%).

US FDA更新MAPP 6030.2 申报指南：对知情同意书的审查

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美国FDA发布了修订后的政策和程序手册（MAPP）6030.2，内容为“新药申请：对知情同意书的审查”。

本政策和程序手册（MAPP）描述了：何时应审查试验用新药申请(IND)中提交的知情同意文件（ICD）；何时药物评价和研究中心（CDER）应要求将ICD提交IND；以及审查ICD的程序。

本《指南》并未涉及治疗用药物临床试验申请中的知情同意事宜，也未涉及在紧急研究中免除知情同意要求的情况。此版本已进行更新，以与当前OND组织结构、适用的用户费用协议（UFA）承诺以及CDER最佳实践和工作流程保持一致。

US FDA Updates MAPP 6030.2 INDs: Review of Informed Consent Documents

The US FDA has released a revised MANUAL OF POLICIES AND PROCEDURES (MAPP) 6030.2, Rev. 2, INDs: Review of Informed Consent Documents.

This Manual of Policies and Procedures (MAPP) describes: when an informed consent document (ICD) submitted under an investigational new drug application (IND) should be reviewed; when the Center for Drug Evaluation and Research (CDER) should request that an ICD be submitted to an IND; and procedures for reviewing an ICD.

This MAPP does not address consent for treatment INDs or exception from informed consent requirements for emergency research.

This version has been Updated to align with current OND organizational structure, applicable user fee agreement (UFA) commitments, and CDER best practices and workflow procedures.

MHRA发布了许多新的指南文件

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MHRA发布了几份指南文件：

MHRA Publishes Many New Guidance documents

The MHRA has published several guidance documents:

ICH发布了E20临床试验适应性设计指南草案

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人们对使用适应性方法进行药物开发越来越感兴趣。人用药品注册技术要求国际协调会(ICH)最近宣布发布E20指南草案：临床试验适应性设计。

本指南的重点是计划、实施、分析和解释具有适应性设计的试验的原则，旨在证实疗效并支持治疗的获益-风险评估。草案提出将“适应性设计”统一定义为“基于对试验受试者累积数据的期中分析，允许对试验的一个或多个方面进行前瞻性计划的修改的临床试验设计”。本指南不包括计划外修改的试验，如IDMC提出的方案修订。它还排除了对试验的常规监测，如入组率、数据质量或受试者退出。

ICH已经发布了其高度预期的指导原则草案，该指导原则协调了新药开发中适应性临床试验的设计、分析和报告原则。该指南指出，适应性设计可以有意义地加速药物开发并优化资源，同时不影响科学或监管的严谨性。

ICH Releases E20 Draft Guideline on Adaptive Design for Clinical Trials

There is growing interest in using an adaptive approach to drug development. The International Council for Harmonisation (ICH) recently announced the release of its E20 Guideline draft: Adaptive Designs for Clinical Trials.

The focus of this guideline is on principles for the planning, conduct, analysis, and interpretation of trials with an adaptive design intended to confirm the efficacy and support the benefit-risk assessment of a treatment. The draft proposes a unified definition of "adaptive design" as a "clinical trial design that allows for prospectively planned modifications to one or more aspects of the trial based on interim analysis of accumulating data from participants in the trial." The guidance does not cover trials with unplanned modifications, such as protocol amendments proposed by an IDMC. It also excludes routine monitoring of trials such as enrollment rate, data quality, or participant withdrawals.

The ICH has released its highly anticipated draft guideline that harmonizes principles for the design, analysis, and reporting of adaptive clinical trials in new drug development. The guideline indicates that adaptive designs can meaningfully accelerate drug development and optimize resources without compromising scientific or regulatory rigor.

审阅｜刘海涛、施媛媛、张淼、宋婷婷

编辑｜宋婷婷、李茜然、乐园