1. 英国关于早期药物准入获取计划使用真实世界数据的指南（2022年5月）
UK Issues Guidance on Use of Real-World Data Under Early Access Scheme (May 2022)
总结：
英国卫生研究机构发布了更新的指南，以促进公司根据政府早期药物准入计划（EAMS）收集真实世界数据（RWD），该计划允许生命垂危或严重衰弱的患者在缺乏现有治疗选择的情况下，在新药获批前使用有前景的新药。
Summary:
The UK's Health Research Authority has issued updated guidance to facilitate the collection of real-world data (RWD) by companies under the government's Early Access to Medicines Scheme (EAMS), which allows patients with life-threatening or seriously debilitating conditions without existing treatment options to use promising new medicines before they are approved.
关键信息：
· 根据新的英国指南，收集在早期准入计划下接受未批准药物的患者的临床管理数据，公司不必申请临床试验批准或伦理审查。。
· 对于同意根据该计划收集其数据的患者，还应告知数据可能用于未来研究的可能性，并询问其是否同意将其数据用于此类目的。
Key Takeaways:
· New UK guidance says companies do not have to apply for clinical trial approval or ethics review to collect data on the clinical management of patients receiving unapproved drugs under an early access scheme.
· Patients consenting to the collection of their data under the scheme should additionally be provided with information about the potential for future research use and asked whether they consent to their data being used for such purposes.
2. 监测欧洲临床试验环境的关键性能指标（KPI）（2022年5月）
Key Performance Indicators (KPIs) to Monitor the European Clinical Trials Environment (May 2022)
总结：
欧洲药品监管网络已经同意每月发布一组关键参数或关键性能指标(KPI)，以深入了解临床试验法规如何改变欧盟/欧洲经济区的临床试验环境。
第一组KPI于本月公布。KPI将跟踪：
· 申办方类型（商业或非商业）
· 是否正在一个或多个成员国进行试验
· 临床试验阶段
· 临床试验针对的治疗领域
Summary:
The European medicines regulatory network has agreed on a set of key parameters – or key performance indicators (KPI) – that are to be published every month to provide an insight into how the CTR might be transforming the clinical trial environment in the EU/European Economic Area.
The first set of KPIs was published this month. The KPIs are to track:
· The sponsor type (commercial or non-commercial).
· Whether the trial is taking place in one member state or more.
· The clinical trial phase.
· The therapeutic area being addressed by the clinical trial.
关键信息：
· 欧洲药品监管网络已开始发布月度指标，以深入了解临床试验法规如何影响欧盟研究环境。
· 欧盟加速临床试验（ACT EU）寻求改变临床试验的启动、设计和运行方式。ACT EU的优先行动之一是监测临床试验法规的执行情况。
Key Takeaways:
· The European medicines regulatory network has started publishing monthly metrics to offer insight into how the Clinical Trial Regulation is affecting the EU research space.
· Accelerating Clinical Trials (ACT) EU seeks to transform how clinical trials are initiated, designed, and run. One of the priority actions of ACT EU focuses on monitoring the implementation of the CTR.
3. EMA更新药物临床试验质量管理规范检查程序附件（2022年5月）
EMA Updates Good Clinical Practice Inspection Procedure Annexes (May 2022)
总结：
EMA已经发布了其药物临床试验质量管理规范检查程序的几个修订附件。根据欧洲药品管理局(EMA)人用药品委员会(CHMP)在集中程序背景下的要求，药物临床试验质量管理规范(GCP)检查员工作组制定了协调、准备、实施和报告的GCP检查程序。
本附录汇编了可在研究中心验证的具体项目，但其选择将取决于检查范围，并可在当地检查计划中确定。
本指南可能适用于对临床试验中采集的人体样本进行分析或评价的实验室进行检查。
本附录汇编了可在申办方或履行申办方试验相关职责的合同/临床研究组织(CRO)中验证的具体项目。
· 附件VI：CHMP要求的GCP检查程序：文件的记录保存和存档
本文件的范围是为欧盟成员国主管当局代表EMA在集中程序背景下进行的所有药物临床试验质量管理规范(“GCP”)检查相关文件的记录保存和存档提供指南。
· CHMP要求的GCP检查程序附录VII：生物分析部分，生物等效性试验的药代动力学和统计分析
本附录是生物分析部分和生物等效性试验的药代动力学和统计分析检查中需要验证的具体项目的汇编。检查项目的选择将取决于检查范围，并应在检查计划中详细说明。
Summary:
The EMA has released several revised Annexes to their Good Clinical Practice Inspection Procedures. The Good Clinical Practice (GCP) Inspectors Working Group has developed procedures for the coordination, preparation, conduct and reporting of GCP inspections requested by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) in the context of the centralised procedure.
· Annex I To Procedure For Conducting GCP Inspections Requested By The CHMP: Investigator Site
This annex compiles specific items that may be verified at the investigator site but their selection will depend on the scope of the inspection and can be established in the local inspection plan.
· Annex II To Procedure For Conducting GCP Inspections Requested By The CHMP: Clinical Laboratories
This guidance may be applied to the inspection of laboratories that perform the analysis or evaluation of human samples collected as part of a clinical trial.
· Annex IV To Procedure For Conducting GCP Inspections Requested By The CHMP: Sponsor And CRO
This annex compiles specific items that may be verified at the sponsor site or the Contract/Clinical Research Organisations (CROs) performing sponsor's trial-related duties.
The scope of this document is to provide guidance for the record keeping and archiving of documents in relation to all Good Clinical Practice ("GCP") inspections carried out by the competent authorities of Member States of the European Union on behalf of the EMA in the context of a centralized procedure.
This annex compiles specific items that may be verified during the inspection of the bioanalytical part and of the pharmacokinetic and statistical analyses of bioequivalence trials. The selection of items to be inspected will depend on the scope of the inspection and should be detailed in the inspection plan.
4. EMA和FDA在GCP检查结果上基本一致（2022年6月）
EMA and FDA Mostly Align on GCP Inspection Findings (Jun 2022)
总结：
美国FDA和EMA进行的一项研究发现，监管机构在联合药物临床试验质量管理规范(GCP)检查中发现了相同类型的缺陷。本研究比较了2009年至2015年两个机构进行的与上市许可申请材料中提交的临床研究相关的49次GCP检查的决定，并概述了常见检查缺陷结果的相似性和差异性。在分析之前，排除了与两家机构之间已知的操作和监管差异相关的一些发现。
Summary:
A study conducted by the US FDA and EMA found that the agencies are identifying the same types of deficiencies in joint good clinical practice (GCP) inspections. This study compared decisions on 49 GCP inspections conducted by the two agencies related to clinical investigations submitted in marketing authorization packages between 2009 and 2015 and outlined the similarities and differences in findings for common inspection deficiencies. Prior to their analysis, a number of findings related to known operational and regulatory differences between the two agencies were excluded.
关键信息：
· 总体而言，两家监管机构在临床研究者检查和CRO检查方面发现了相似的问题。
· 今后，EMA&FDA和PMDA计划通过指南制定和联合培训计划，在持续流程改进方面加强现有的GCP合作，加强监管融合，并形成全球GCP检查合作，以支持共享上市申请审评。
Key Takeaways:
· Overall, the two regulators identified similar problems with respect to the clinical investigator inspections and the CRO inspections.
· Moving forward, EMA & FDA along with PMDA plan to enhance their existing GCP collaborations in terms of continuous process improvement through guidance development and joint training programs, strengthen regulatory convergence, and form global GCP inspection collaboration in support of shared marketing application review.
5. EMA发布复杂临床试验的最终问答指南（2022年6月）
EMA Issues Final Q&A Guidance on Complex Clinical Trials (Jun 2022)
总结：
EMA发布了关于复杂临床试验的最终问答文件。
Summary:
The EMA has released a final Question and Answer document on Complex Clinical Trials.
关键信息：
· 本文件的范围是为复杂的临床试验提供指导和支持，这些临床试验被定义为非常规试验，因为它们具有令其设计、实施、分析或报告变得复杂的元素、特征、方法或其组合，包括新颖的方法。
· 该文件旨在回答一些基本问题，例如在复杂临床试验的计划和实施中需要考虑什么，如何向监管机构证明贝叶斯方法的合理性，以及如何使用生物标志物。
· 针对复杂临床试验与标准临床试验的不同，监管机构提出了7个广泛的问题，例如复杂临床试验的计划和实施中需要考虑什么，研究设计中需要考虑什么，以及在研究受试者的安全性、权利和健康方面需要考虑什么。
Key Takeaways:
· The scope of this document is to provide guidance and support for complex clinical trials which are defined as being non-conventional in the sense that they have elements, features, methods or combination thereof, including novel approaches, that confer complexity of their designs, conduct, analyses or reporting.
· The document aims to answer some basic questions, such as what to consider in the planning and conduct of CCTs, how to justify Bayesian approaches to regulators, and how to use biomarkers.
· To address how CCTs may operate differently than standard clinical trials, the regulators ask seven broad questions, such as what needs to be considered in the planning and conduct of CCTs, what to consider in the study design, and what to consider in terms of the safety, rights, and well-being of study participants.
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