盘点→最新国际医药政策合集[SEP 2022]

文摘   健康医疗   2022-09-23 16:07   上海  

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  本篇摘要  

以下整理分享最新发布的18项国际医药政策法规时事,包括:

FDA发布《以患者为中心的药物开发:选择、开发或修改适合目的的临床结果评估》指南草案、《Q9(R1)质量风险管理》指南草案、《开发膀胱癌的辅助治疗药物和生物制剂》指南、《开发肾细胞癌的辅助治疗药物和生物制剂》指南、《开发寡核苷酸治疗药物的临床药理学考虑》指南草案、《在IND和NDA中评估食物对药物的影响-临床药理学考虑》指南、《再生医学疗法自愿共识标准认可计划》指南草案、《放射设备上市前提交的定量成像技术性能评估》指南EMA发布《药品数据质量框架监管研讨会报告》、《强制使用ISO/ICH E2B(R3)ICSR安全报告:使用EudraVigilance系统》实践培训课程、《对集中程序用户的上市批准前程序建议》指南、《对集中程序用户的上市批准后程序建议》指南、《更改申办者的名称或地址》、《罕病药:监管和程序指南及表格》、《EudraVigilance用户界面安装》指南、《eXtended EudraVigilance药品字典(XEVMPD)数据输入工具(EVWEB)》用户手册;EC发布《复杂临床试验问与答》指南;MHRA发布《提交150天上市申请(MAA)的英国和欧盟委员会决策程序》指南。

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We are drowning in information but starved for knowledge.

我们被信息淹没,却渴望知识。

– John Naisbitt


1

以患者为中心的药物开发:选择、开发或修改适合目的的临床结果评估

Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments

On 29 June 2022 the FDA published the draft guidance on the patient-focused drug development. 

This guidance (Guidance 3) is the third in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making. When finalized, Guidance 3 will represent the current thinking of CDER, CBER, and CDRH on this topic.
https://www.fda.gov/media/159500/download





2

Q9(R1)质量风险管理

Q9(R1) Quality Risk Management

On 14 June 2022 the FDA issued the draft guidance on quality risk management. 

In the pharmaceutical sector, the principles and framework of ICH Q9, coupled with the official ICH training material that supports this guideline, are instrumental in enhancing the application of effective quality risk management by industry and regulators. The importance of quality systems has been recognized in the pharmaceutical industry, and it is evident that quality risk management is a valuable component of an effective quality system.
https://www.fda.gov/media/159218/download






3

开发膀胱癌的辅助治疗药物和生物制剂

Bladder Cancer: Developing Drugs and Biologics for Adjuvant Treatment

On 27 June 2022 the FDA published the final guidance on the adjuvant treatment of bladder cancer for industry.

This guidance provides recommendations to sponsors regarding the development of drugs and biological products regulated by CDER and CBER for the adjuvant treatment of muscle-invasive bladder cancer. The guidance includes recommendations regarding eligibility criteria, choice of comparator, follow-up imaging assessments, determination of disease recurrence, analyses of disease-free survival (DFS), and interpretation of trial results. Although FDA may consider endpoints other than DFS for the adjuvant treatment of muscle-invasive bladder cancer, this guidance is focused on clinical trials with DFS as the primary efficacy endpoint.
https://www.fda.gov/media/159509/download





4

开发肾细胞癌的辅助治疗药物和生物制剂

Renal Cell Carcinoma: Developing Drugs and Biologics for Adjuvant Treatment

On 27 June 2022 the FDA published the final guidance on the adjuvant treatment of renal cell carcinoma for industry.

This guidance provides recommendations to sponsors regarding the development of drugs and biological products regulated by CDER and CBER for the adjuvant treatment of renal cell carcinoma. The guidance includes recommendations regarding eligibility criteria, choice of comparator, follow-up imaging assessments, determination of disease recurrence, analyses of disease-free survival (DFS), and interpretation of trial results. Although FDA may consider endpoints other than DFS for the adjuvant treatment of renal cell carcinoma, this guidance is focused on clinical trials with DFS as the primary efficacy endpoint.
https://www.fda.gov/media/159510/download






5

开发寡核苷酸治疗药物的临床药理学考虑

Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics

On 27 June 2022 the FDA published the draft guidance for industry. 

This guidance provides recommendations to assist industry in the development of oligonucleotide therapeutics under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) and 21 CFR parts 312 and 314. Specifically, this guidance represents the FDA's recommendations for certain evaluations including pharmacokinetic, pharmacodynamic, and safety assessments during oligonucleotide therapeutic development, including: (1) characterizing the potential for QTc interval prolongation, (2) performing immunogenicity risk assessment, (3) characterizing the impact of hepatic and renal impairment, and (4) assessing the potential for drug-drug interactions. This guidance provides recommendations on when to conduct these assessments and what types of assessments are suitable to address these questions.
https://www.fda.gov/media/159414/download





6

在IND和NDA中评估食物对药物的影响-临床药理学考虑

Assessing the Effects of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations

On 24 June 2022 the FDA published the final guidance on food-effect on drug in INDs and NDAs.

This guidance provides recommendations to sponsors planning to conduct food-effect (FE) studies for orally administered drug products under investigational new drug applications (INDs) to support new drug applications (NDAs) and supplements to these applications for drugs being developed under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355).  This guidance revises and replaces part of the 2002 FDA guidance entitled Food-Effect Bioavailability and Fed Bioequivalence Studies (December 2002).

https://www.fda.gov/media/121313/download






7

再生医学疗法自愿共识标准认可计划

Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies

On 15 June 2022 the FDA published the draft guidance for industry. 

This guidance describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) designed to identify and recognize Voluntary Consensus Standards  (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate.  CBER encourages the use of appropriate standards in the development of CBER-regulated products.  The use of recognized VCS can assist stakeholders in more efficiently meeting regulatory requirements and increasing regulatory predictability for RMT products.  This program is modeled after the formal standards and conformity assessment program or S-CAP for medical devices.
https://www.fda.gov/media/159237/download





8

放射设备上市前提交的定量成像技术性能评估

Technical Performance Assessment of Quantitative Imaging in Radiological Device Premarket Submissions

On 16 June 2022 the FDA published the final guidance on a premarket submission for radiological devices for industry and FDA staff.

This guidance document provides FDA's recommendations on the information, technical performance assessment, and user information that should be included in a premarket submission for radiological devices that include quantitative imaging functions. The recommendations reflect current review practices and are intended to promote consistency and facilitate efficient review of premarket submissions for radiological devices that include quantitative imaging functions.
https://www.fda.gov/media/123271/download






9

药品数据质量框架监管研讨会报告

Report of Workshop on Data Quality Framework for Medicines Regulation 

On 29 June 2022 the EMA published the report of the workshop on data quality framework for medicines regulation. 

The workshop was an initiative of HMA, EMA and TEHDAS meant to both share the current progress on building a data quality framework for medicines regulation and to solicit the comments and ideas of experts in this field to help shape the drafting process. Breakout sessions focused on particular use cases with fruitful discussions on the current data quality landscape and how the future of data quality in medicines regulation should look in the clinical and non-clinical areas. 

https://www.ema.europa.eu/en/documents/report/report-data-quality-framework-medicines-regulation_en.pdf






10

强制使用ISO/ICH E2B(R3)ICSR安全报告:使用EudraVigilance系统的实践培训课程

Mandatory Use of ISO/ICH E2B(R3) Individual Case Safety Reporting in the EU: Hands-on Training Course on Using the EudraVigilance System

On 15 June 2022 the EMA announced the online training course on using the EudraVigilance system.

This training course covers the use of the ISO ICSR/ICH E2B(R3) format and related ISO standard terminology for pharmaceutical form and route of administration for reporting individual cases of suspected side effects to EudraVigilance.

https://www.ema.europa.eu/en/events/mandatory-use-iso-ich-e2br3-individual-case-safety-reporting-eu-hands-training-course-using-22






11

EMA对集中程序用户的上市批准前程序建议

European Medicines Agency Pre-authorisation Procedural Advice for Users of the Centralised Procedure 

On 20 June 2022 the EMA published the updated pre-authorisation Q&A guidance.

This guidance document addresses a number of questions which users of the centralised procedure may have. It provides an overview of the European Medicines Agency’s position on issues, which are typically addressed during the course of pre-submission meetings. 
The following sections were revised:
•3.3.3. What information regarding transfer of test methods is required for different active substance or finished product release testing sites in the MAA? Rev. Jun 2022 
•5.1.1. How long does it take for my application to be evaluated? Rev. Jun 2022 
•5.1.11. Can EMA assessment or inspection documents be shared with third parties? Rev. Jun 2022
•5.2.1. When can I expect a pre-authorisation GMP inspection and how are they conducted? Rev. Jun 2022 
•5.2.2. When can I expect a pre-approval GCP inspection and how are they conducted? Rev. Jun 2022 
•5.2.3. What is the fee for a GMP/GCP/GLP pharmacovigilance inspection? Rev. Jun 2022 

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-pre-authorisation-procedural-advice-users-centralised-procedure_en-0.pdf







12

EMA对集中程序用户的上市批准后程序建议

European Medicines Agency Post-authorisation Procedural Advice for Users of the Centralised Procedure 

On 20 June 2022 the EMA published the updated Q&A guidance on extensions of marketing authorisations.

This guidance document addresses a number of questions which marketing authorisation holders (MAHs) may have on post-authorisation procedures. It provides an overview of the Agency’s position on issues, which are typically addressed in discussions or meetings with MAHs in the post-authorisation phase. The following sections were revised:
•3.21. Do I need to confirm the maintenance of my orphan designation when applying for a type II variation? Rev. Jun 2022 
•4.7. Do I need to confirm the maintenance of my orphan designation when applying for an extension application? Rev. Jun 2022 
•10.6. When, how and to whom shall I submit my annual re-assessment application? Rev. Jun 2022 
•19.5. How to choose the implementation date? Rev. Jun 2022 

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-post-authorisation-procedural-advice-users-centralised-procedure_en.pdf






13

更改申办者的名称或地址

Changing the Name or Address of a Sponsor

On 9 June 2022 the EMA announced the update for submitting a request to change the name or address.

Sponsors need to use EMA's IRIS platform to request changes to their name or address. The EMA is not able to process any requests outside of IRIS.

https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/changing-name-address-sponsor






14

罕病药:监管和程序指南及表格

Orphans: Regulatory and Procedural Guidance and Forms

On 9 June 2022 the EMA announced the updates for the regulatory and procedural guidance documents on orphan designation.

The following regulatory and procedural guidance and forms on orphan designation were updated:
•Template - Translations required with the submission of an application for orphan medicinal product designation
•Procedural advice for post-orphan medicinal product designation activities:
Guidance for sponsors 
•Checklist for sponsors applying for the transfer of Orphan Medicinal Product (OMP) designation 
•Template - Translations required with the submission of an application for transfer of orphan medicinal product designation

https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/applying-designation/orphans-regulatory-procedural-guidance-forms






15

EudraVigilance用户界面安装指南

Article 57 User Interface (UI) Installation Guide 

On 7 June 2022 the EMA published the updated the installation guide on article 57 UI components and initial set up. 

This document provides detailed steps to follow to install Article 57 components on users' computers to support the use of the Extended EudraVigilance Medicinal Product Dictionary (XEVMPD) data-entry tool user interface known as EVWEB. 

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/article-57-user-interface-ui-installation-guide-installation-article-57-ui-components-initial-set_en.pdf







16

eXtended EudraVigilance药品字典(XEVMPD)数据输入工具(EVWEB)用户手册

eXtended EudraVigilance Medicinal Product Dictionary (XEVMPD) Data-Entry Tool (EVWEB) User Manual 

On 7 June 2022 the EMA published the updated EVWEB user manual, version 5.7.

The below sections were amended following the publication of version 5.6 in October 2021: 
•1.5. EVWEB 
•4.8. Save, Reload and Send an XEVPRM 
•4.9. Use EV Post functionality 
•4.10.1. Exporting results of a simple query

https://www.ema.europa.eu/en/documents/other/extended-eudravigilance-medicinal-product-dictionary-xevmpd-data-entry-tool-evweb-user-manual_en.pdf






17

复杂临床试验问与答指南

Questions and answers - Complex clinical trials

On 2 June 2022 the European Commission published the updated Q&A guidance on complex clinical trials, version 2022-05-23.

This Q&A document provides guidance and seeks to support sponsors, clinical trialists and applicants regarding scientific aspects and the planning, set-up, submission for obtaining CT authorisation (CTA), conduct, reporting and transparency, analysis and interpretation of complex clinical trials (CCTs) under the EU Clinical Trials Regulation (EU CTR) as well as their use in submissions for marketing authorisation.

https://health.ec.europa.eu/latest-updates/questions-and-answers-complex-clinical-trials-2022-06-02_en






18

提交150天上市申请(MAA)的英国和欧盟委员会决策程序

Marketing Authorisation Application (MAA) Submission Dates for 150-days National and European Commission Decision Reliance Procedures

On 20 June 2022 the MHRA published the updated guidance on MAA submission dates.

The MHRA will operate a 150-day Assessment route for high-quality marketing authorisation applications (MAAs). Submission deadlines were updated.

https://www.gov.uk/guidance/marketing-authorisation-application-submission-dates-for-150-days-national-and-european-commission-decision-reliance-procedures#full-publication-update-history







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