1.      EMA Assent / Informed Consent Guidance for Paediatric Clinical Trials with Medicinal Products in Europe (January 2021)

EMA关于欧洲儿科药品临床试验赞同/知情同意指南 (2021年1月)

Summary:

The EMA has released a guidance titled: Assent / Informed Consent Guidance for Paediatric Clinical Trials with Medicinal Products in Europe. This guidance was developed by the Ethics Working Group of the Enpr-EMA with the support of the European Young Persons Advisory Group Network, eYPAGnet. The aim of this document is to create an informed consent and assent overview tool, publicly available for all stakeholders, in order to support the design and conduct of high-quality ethical paediatric Clinical Trials (CTs) in Europe.

总结：

EMA发布了“欧洲儿科药品临床试验的赞同/知情同意指南”。本指南由Enpr-EMA伦理工作组在欧洲青年咨询小组网络eYPAGnet的支持下制定。本文件的目的是创建一个知情同意和赞同的概述工具，供所有利益相关者公开使用，以支持欧洲高质量且合乎伦理的儿科临床试验(CT)的设计和实施。

·       This guidance document does not contain consent or assent templates, but merely general guidance about the contents in a form of listing various subject elements to be considered in every paediatric age group when designing consent / assent documentation. All such documents must be always designed case-by-case.

·       The document is based on the current and forthcoming Clinical Trials Regulation(CTR) EU legal framework and ethical requirements, which can be adapted to various paediatric CTs on a case by case basis, and by following local requirements, when necessary.

·       Each Member State (MS) of the European Union (EU) and European Economic Area (EEA) has national legislation and detailed requirements for informed consent and assent that are required for the conduct of paediatric clinical trials.

o   Because these requirements vary amongst countries the information in this document should be used only as a guidance tool together with the local or national requirements, and researchers or sponsors must always check these national regulatory requirements when preparing consent / assent documents.

·       As this document provides the overall “skeleton” of all elements linked to the current legislative framework, all future updates related to the legislation and regulatory documents will be updated by the Enpr-EMA secretariat upon the newest relevant available information.

·       The legal framework of this document, which describes the content for patient information and informed consent and assent documents, is the current EU clinical trial legislation (Directive 2001/20/ECi ) and EU Commission ethical guidance documents, including many elements of the new CTR.

o   After CTR implementation, the detailed definition of informed consent / assent requirements will still remain within the remit of each Member State.

·       The Guidance contains 2 Tables:

o   Table 1: General information for informed consents and assents (agreements)

o   Table 2: Trial specific information for informed consents and assents (agreements)

·       本指南文件不包含知情同意书或赞同书模板，而仅是关于设计知情同意书/赞同书文件时，针对每个儿童年龄组应考虑的各种主题要素内容，以列表形式呈现的通用指南。所有知情同意书/赞同书必须始终根据具体情况设计。

·       本文件基于现行和即将发布的临床试验法规(CTR)欧盟法律框架和伦理要求，可根据具体情况，并在必要时遵循当地要求，适用于各种儿科临床试验。

·       欧盟(EU)和欧洲经济区(EEA)的每个成员国(MS)均有国家立法以及开展儿科临床试验所需的知情同意和赞同的详细要求。

o   由于各国的这些要求各不相同，本文件中的信息应仅作为指导工具与当地或国家要求一起使用，研究人员或申办方在编写知情同意书/赞同文件时必须始终查看这些国家监管要求。

·       由于本文件提供了与当前立法框架相关的所有要素的总体“框架”，因此，Enpr-EMA秘书处将根据最新的相关可用信息更新与立法和监管文件相关的内容。

·       本文件的法律框架所描述的患者须知、知情同意和赞同文件的内容，源自现行欧盟临床试验法规（指令2001/20/ECi）和欧盟委员会伦理指南文件，包括新CTR的许多要素。

o   在CTR实施后，知情同意/赞同要求的详细定义仍将保留在每个成员国的职权范围内。

·       指南包含2个表：

o   表1：知情同意书和赞同书（协议）的一般信息

o   表2：知情同意书和赞同书（协议）的试验特定信息

Key Takeaways:

·       This document is intended to be used as an overview tool of the contents for assent/informed consent forms for all stakeholders (such as patients, sponsors and investigators) to support the conduct of high quality paediatric clinical trials in Europe across all paediatric age groups, from birth to less than 18 years of age.

·       The guidance document can be used to prepare paediatric Clinical Trial submissions in the EU, and to support and promote the inclusion of children in the consent/assent processes.

·       This will also enhance raising awareness about children´s basic rights to be heard and to express their opinion about participating in a Clinical Trial, according to their maturity.

关键信息：

·       本文件旨在作为所有利益相关者（如患者、申办方和研究者）的一个赞同/知情同意书内容的概述工具，以支持在欧洲所有儿童年龄组（从出生至18岁以下）中开展高质量的儿童临床试验。

·       本指南文件可用于准备在欧盟提交的儿科临床试验申请，并支持和促进将儿童纳入同意/赞同过程。

·       这也将提高对儿童基本权利的认识，使他们能够根据自己的成熟度表达自己对参加临床试验的意见。

2.      EMA Updates Post-Authorisation Efficacy Studies: Q&A (January 2021)

EMA更新许可后疗效研究问答（2021年1月）

Summary:

The EMA has updated their Post-authorisation efficacy studies: questions and answers website.  This page lists questions that marketing-authorisation holders (MAHs) may have on post-authorisation efficacy studies (PAESs). It provides an overview of the EMA position on issues that are typically addressed in discussions or meetings with MAHs in the post-authorisation phase.

总结：

EMA更新了其许可后疗效研究：问答网站。网页列出了上市许可持有人(MAH)可能对上市后疗效研究(PAESs)提出的问题。本文件概述了EMA对上市许可后阶段与MAH讨论或会议中通常涉及的问题的立场。

They have updated Question 2: How and where the PAES imposed in accordance with the Commission Delegated Regulation will be reflected in the marketing authorization?

对问题2进行了更新：根据委员会授权法规，PAES如何以及在何处反映在上市许可中？

·       For centrally authorised medicinal products (“CAPs”), a PAES imposed as a condition to the MA is reflected in Annex II under section D “Obligation to conduct post-authorisation measures”.

·       The study objective and the deadline for the submission of the final study results are specified in the Annex II. At the beginning of the description of the study, such efficacy study imposed in accordance with the Delegated Regulation is explicitly named 'Post-Authorisation Efficacy Study (PAES)'.

·       The imposition of such PAES shall meet one of the criteria set out in the Delegated Regulation. A justification will be provided in the Committee for Medicinal Products for Human Use (CHMP) assessment report.

·       If the MAH has to submit the protocol for endorsement by the European Medicines Agency, this will be reflected in Annex II in the wording of the condition (e.g. “according to an agreed protocol”).

·       Any post-approval amendments to the conditions in Annex II (objective and/or due date) should be duly justified and submitted as a variation, type IB C.I.11.z) for change in the due date or type II C.I.11.b) for changes other than the due date.

·       As for any imposed post-authorisation efficacy studies, those imposed in accordance with the Delegated Regulation should also be reflected in the risk management plan (“RMP”), part IV ‘Plans for post-authorisation efficacy studies’.

·       对于集中许可药品(“CAPs”)，作为MA条件的PAES反映在附录II第D节“实施许可后措施的义务”中。

·       附录II规定了研究目的和最终研究结果的提交期限。在本研究描述开始时，根据授权法规实施的此类疗效研究明确命名为“许可后疗效研究(PAES)”。

·       此类PAES的实施应符合授权法规中规定的标准之一。将在人用药品委员会（CHMP）评估报告中提供依据。

·       如果MAH必须提交方案以获得欧洲药品管理局的认可，这将在附录II的条件措辞中反映（例如，“根据商定的方案”）。

·       对附件II中条件（目的和/或到期日）的任何批准后修订均应进行适当论证，并作为IB类C.I.11.z变更提交）截止日期变更或II类C.I.11.b）截止日期以外的变更。

·       对于任何强制实施的许可后疗效研究，根据授权法规强制实施的研究也应反映在风险管理计划(“RMP”)第IV部分“许可后疗效研究计划”中。

Key Takeaways:

·       The EMA has updated their Post-Authorisation Efficacy Studies: Questions and Answers website to revise Question 2.

·       These questions and answers have been produced for guidance only and should be read in conjunction with the rules governing medicinal products in the European Union, volume 2, notice to applicants.

关键信息：

·       EMA已更新其上市后疗效研究：问题和答案网站，以修订问题2。

·       这些问题和答案仅用于指导，应结合欧盟药品管理条例第2卷申请人须知一起阅读。