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Regulatory Express Details

Regulatory Express_Mar. 2023

2023-03-29 14:09:45


EMA Final Guidance Annex V to Guidance for the Conduct of Good Clinical Practice Inspections – Phase I Units



The EMA has released the final guidance on First in Human (FIH) and early clinical trials with the objective of managing and minimizing potential risks to trial participants (healthy volunteers and patients) who take part in these types of trials.


· 本指南是核查I期病房的参考资料。

The guidance is listed in the references and should be taken into account during the inspection of the Phase I unit.

· 本指南改编自2008年版本。

The guidance is largely rewritten from the previous version issued in 2008.




EMA Final Guideline on Computerized Systems and Electronic Data in Clinical Trials



The EMA has released the final version of the guideline: Guideline on computerized systems and electronic data in clinical trials. The scope of this guideline is computerized systems (including instruments, software and 'as a service') used in the creation/ capture of electronic clinical data and to the control of other processes with the potential to affect participant protection and reliability of trial data, in the conduct of a clinical trial of investigational medicinal products.  


· 本指南提供一般原则和关键概念定义、计算机系统一般要求(包括验证、用户管理、安全、电子数据生命周期管理等)、以及某些特定类型的计算机系统、过程和数据的要求。

This guideline has described some generally applicable principles and definition of key concepts. It also covers requirements and expectations for computerized systems, including validation, user management, security, and electronic data for the data life cycle. Requirements and expectations are also covered related to specific types of systems, processes, and data.

· 基于风险的质量管理模式也适用于计算机系统和电子数据的管理。

The risk-based approach to quality management also has an impact on the use of computerized systems and the collection of electronic data.

· 根据不同的数字技术,还应考虑是否满足药品监管框架中其它现行法律法规的要求,比如医疗器械、数据保护、电子识别和电子签名等。

Consideration should also be given to meeting the requirements of any additional current legal and regulatory framework that may in addition apply to the medicinal product regulatory framework, depending on the digital technology. These may include e.g., medical devices, data protection legislation, and legislation on electronic identification and electronic signatures.




FDA Releases Revised Draft Guidance on Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Q&A



The U.S. Food and Drug Administration has issued a revised draft guidance. While the new guidance continues to use the questions and answers format of the previous version, the topics addressed have been updated to account for technological advances.


· 本指南草案的读者为申办方、研究者、伦理委员会、合同研究组织及其他相关方,涉及FDA监管下的食品、医疗产品、烟草产品和新兽药的临床研究。

This draft guidance provides information for sponsors, clinical investigators, institutional review boards, contract research organizations, and other interested parties on the use of electronic systems, electronic records, and electronic signatures in clinical investigations of foods, medical products, tobacco products, and new animal drugs under FDA regulations.

· 本指南草案修订自20176月发布的在临床研究中使用电子记录和电子签名问答;定稿后,本指南将取代20075月发布的临床研究中使用的计算机化系统指南

This draft guidance revises the draft guidance for industry issued in June 2017 entitled Use of Electronic Records and Electronic Signatures in Clinical Investigations Under 21 CFR Part 11 — Questions and Answers and, when finalized, will supersede the guidance for industry entitled Computerized Systems Used in Clinical Investigations (May 2007).




UK PMCPA Publishes Guidance on Use of Social Media



The UK Prescription Medicines Code of Practice Authority (PMCPA) has published a new social media guidance to help pharmaceutical companies apply the high standards demanded by the industry's Code of Practice to all their online communications channels. The PMCPA Social Media Guidance 2023 has been produced in consultation with the Medicines and Healthcare Products Regulatory Agency (MHRA), the British Pharmaceutical Industry (ABPI), and pharmaceutical companies.


· 该指南涵盖了公司使用各类社交渠道以及员工个人使用公司渠道,特别提及公司在使用/与社交媒体互动的不同情况下,适用的相关法律法规和原则。

The guidance covers companies' use of corporate social media channels and employees' personal use of those channels. In particular, it reminds companies of the relevant laws and Code provisions that apply in different cases, and the principles that companies have to be mindful of when using or interacting with social media.

· 它提及在特定情况下如何使用社交媒体,如临床试验招募、患者支持、与意见领袖合作、职位广告、宣布产品或管线里程碑事件等。

It looks at the use of social media in specific situations, such as clinical trial recruitment, patient support, working with influencers, job advertising, and announcing product or pipeline milestones.

· PMCPA希望借此帮助制药公司看清影响合规使用的沟沟坎坎,更加自信而清晰地理解原则、遵从要求。

PMCPA hopes this guidance will help companies navigate the potential pitfalls of social media, with more confidence, a clearer knowledge of the principles to follow and the legal requirements.




FDA Final Guidance on Format and Content of a REMS Document and REMS Document Technical Conformance Guide



FDA has issued a final guidance titled: Format and Content of a Risk Evaluation and Mitigation Strategy Document. This guidance provides updated recommendations for the format and content of a Risk Evaluation and Mitigation Strategy (REMS) Document for a prescription drug product, including a biological drug product.


FDA发布了REMS文档技术符合性指南。本指南针对REMS文档格式提供详细说明和常用标准化语句,帮助确保一致性并提高审阅效率。本指南支持以结构化产品标签(SPL)格式提交REMS文档。此外,本指南还概述了申请人如何起草Bifurcated REMS文档。

FDA has released a REMS Document Technical Conformance Guide. This Guide provides updated, detailed instructions on the format of a REMS Document, along with standardized language that describes common REMS requirements for applicants to use whenever possible, to help ensure consistency and facilitate efficient review of the REMS Document. This Guide supports submission of a REMS Document in Structured Product Labeling (SPL) format. In addition, this Guide provides an outline to assist applicants in drafting a Bifurcated REMS Document.




EMA Final Guideline on Clinical Evaluation of Vaccines



The EMA has released a final guidance titled: Guideline on clinical evaluation of vaccines. This guideline is focused on the clinical development of vaccines, where vaccines are defined as medicinal products intended for prevention, post-exposure prophylaxis and/ or treatment of disease caused by an infectious agent and which contain antigen(s) or genetic information for an antigen(s), either of biological or synthetic nature, that induce a specific immune response against the causative infectious agent(s) or its toxins. 



This guideline replaces Guideline on the clinical evaluation of new vaccines (EMEA/CHMP/VWP/164653/05) including its Annex on SPC requirements (EMEA/CHMP/VWP/382702/06) and Guideline on adjuvants in vaccines for human use (EMEA/CHMP/VEG/134716/04)




Updated Position Paper on Decentralized Clinical Trials (DCTs) with Medicinal Products in Switzerland



Swissmedic has updated their position paper on DCTs with medicinal products in Switzerland.


· Swissmedicswissethics总结了当前开展去中心化临床试验的主要挑战,明确在瑞士开展该类试验适用的条件。

In this position paper, Swissmedic and swissethics have summarized the main current challenges of DCTs with medicinal products and show under which conditions such clinical trials could be conducted in Switzerland.

· 更新以澄清包括签名和数据服务器的信息

Updates were made to clarify text including information on signatures and data servers.




FDA Issues Draft Guidance: Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products



FDA has issued a draft guidance to makes recommendations to sponsors and investigators considering the use of externally controlled clinical trials to show evidence of the safety and effectiveness of a drug.


· 外部对照试验中,根据方案接受试验治疗的参与者数据与未接受该治疗的一组人群数据进行比较。外部对照组可以是早期接受治疗或未接受治疗的一组人(历史对照),或同期在另一条件下接受治疗或未接受治疗的一组人(同期对照)。

In an externally controlled trial, outcomes in participants receiving the test treatment according to a protocol are compared to outcomes in a group of people external to the trial who had not received the same treatment. The external control arm can be a group of people, treated or untreated, from an earlier time (historical control), or a group of people, treated or untreated, during the same time period (concurrent control) but in another setting.

· 外部对照试验使用患者水平的数据(即个体信息,如病史、治疗史)来研究药物的有效性和安全性。对此,本指南提供了设计和实施外部对照试验的考虑因素包括各种来源的潜在的偏倚对试验结果的影响。

The guidance provides stakeholders with considerations for designing and conducting externally controlled trials that use patient-level data (i.e., information on individual people, such as medical and treatment history) to study the effectiveness and safety of drugs, including threats to the validity of trial results from sources of potential bias.

· 外部数据可以包括来自其他临床试验或真实世界数据源的数据,例如登记研究、电子病历或医疗索赔。

The external data can include data from other clinical trials or from real-world data sources such as registries, electronic health records, or medical claims.

· 本指南还描述了与FDA沟通的考虑因素,并确保该机构可以访问外部对照试验的数据。

This guidance also describes considerations for communicating with FDA and ensuring the agency has access to data from an externally controlled trial.



ICH Q9(R1):质量风险管理指南

ICH Adopts Q9(R1) Guideline on Quality Risk Management


ICH Q9(R1)质量风险管理指南于2023118日签署,将由ICH成员国实施。该文件于126日被EMA采纳并将于今年726日生效。

The ICH Q9(R1) Quality Risk Management Guideline has been signed off as a Step 4 document (18 January 2023) to be implemented by the ICH Regulatory Members. The document was adopted by the EMA on 26Jan2023 and will come into effect on 26 July 2023.



The guideline has been revised to address four areas for improvement in relation to the current application of QRM:    

· 在风险评估和质量风险管理输出中的高主观性。

High levels of subjectivity in risk assessments and in QRM outputs.

· 未能充分管理供应和产品可及性的风险。

Failing to adequately manage supply and product availability risks.

· 对质量风险管理工作的构成形式缺乏理解。

Lack of understanding as to what constitutes formality in QRM work.

· 对基于风险的决策缺乏澄清。

Lack of clarity on risk-based decision-making.




FDA Reviews Alternative Tools Used to Evaluate GCP during the COVID-19 Pandemic



A recent study was published by staff at the FDA titled: The United States Food and Drug Administration's Innovative Alternative Tools to Evaluate Good Clinical Practice During the COVID-19 Public Health EmergencyThe COVID-19 public health emergency limited the U.S. Food and Drug Administration's ability to conduct on-site good clinical practice (GCP) inspections. In this paper, they present their experience in using alternative tools to evaluate data integrity for pivotal Phase II/III clinical trials supporting marketing applications during the COVID-19 public health emergency in the Office of Scientific Investigations (OSI) in the Center for Drug Evaluation and Research (CDER). In addition, they analyze the impact of the pandemic on on-site GCP inspections and discuss the contribution of alternative tools to GCP activities and the implications for the future.


· 作者认为FDA远程评估有助于评估研究者、申办者/CRO的数据完整性、患者安全性和临床试验实施合规性。

The authors believe the study shows that the remote evaluation of clinical trials from FDA locations was useful in evaluating CI and sponsor/CRO data integrity, subject safety, and clinical trial conduct.

· 未来,这些替代工具可作为GCP现场核查的补充,尤其在出行受限时。还可以考虑使用这些工具扩大核查覆盖范围。

Looking forward, alternative tools can be complementary to on-site GCP inspections, particularly when travel limitations exist. In addition, these tools could be considered to expand the breadth of the inspection coverage.