Regulatory Newsletter

The WuXi Biologics regulatory affairs (RA) team is pleased to provide you with this biannual summary of regulatory updates (i.e., typically product development and CMC-related) that are either in agency draft or final status.

Purpose & Disclaimer: The intent of this newsletter is to provide global regulatory agencies with updates and new or revised documents during the indicated period. The items listed are neither comprehensive nor exhaustive of all updates from regulatory agencies. Rather, the newsletter lists item that the WuXi Biologics RA team deems relevant to our potential or existing clients and partners developing biological therapeutics and vaccines. Therefore, this update is for information purposes only and is provided “as is” without any warranty, expressed or implied, as to the completeness or accuracy of the contents or its use or fitness for a particular purpose.

Without limiting the generality of the preceding content, the document and information contained therein should not be construed as regulatory advice or representing, speaking, or acting for any regulatory agency. The information is provided to support your efforts to remain informed and should not be used as a substitute for your own regulatory due diligence or actions.

Quick Links to Agency Sections:

• FDA (U.S. Food & Drug Administration)
• EU (European Union) / EMA (European Medicines Agency) / EC (European Commission) 
• TGA (Therapeutic Goods Administration) 
• Health Canada 
• WHO (World Health Organization) 
• MHRA (Medicines and Healthcare products Regulatory Agency) 
• PMDA (Pharmaceuticals and Medical Devices Agency) 
• ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use)
• EDQM (European Directorate for the Quality of Medicines & HealthCare)
• PIC/S (Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme)
• Health Products Regulatory Authority (HPRA)
• National Medical Products Administration (NMPA)

FDA (U.S. Food and Drug Administration)

Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C

February 5, 2024

According to the “Decision of the State Council on the Issuance and Implementation of the ‘Interim Provisions on Promoting Industrial Structure Adjustment'” (Council of the state No. 40[2005]), the National Development and Reform Commission, in conjunction with relevant departments, has jointly revised and formed the “Guidance Catalogue for Industrial Structure Adjustment (2024 Edition)” (hereinafter referred to as the “Catalogue (2024 Edition)”), which was approved at the 6th executive meeting on December 1, 2023, and will be implemented from February 1, 2024. The “Catalogue (2024 Edition)” consists of three categories: encouraged, restricted, and eliminated.

Full document: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/notifying-fda-discontinuance-or-interruption-manufacturing-finished-products-or-activefda.gov/regulatory-information/search-fda-guidance-documents/notifying-fda-discontinuance-or-interruption-manufacturing-finished-products-or-active

Human Gene Therapy Products Incorporating Human Genome Editing

January 29, 2024

This guidance provides recommendations regarding information that should be provided in an Investigational New Drug (IND) application to assess the safety and quality of the investigational GE product, as required in Title 21 of the Code of Federal Regulations 312.23 (21 CFR 312.23). In addition, this document includes the agency definition of GE products, current thinking on Human GE products review, and information on product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design. Additional recommendations specific to human GE products regarding design, manufacture, and testing of the GE components, as well as the drug product (DP), are described.

Full document: https://www.fda.gov/media/156894/download

Conducting Remote Regulatory Assessments Questions and Answers

Date: February 5, 2024

FDA is issuing the draft guidance to describe the Agency’s current thinking regarding its use of remote regulatory assessments (RRAs) to increase industry’s understanding of RRAs and facilitate FDA’s process for conducting RRAs. FDA has used RRAs to conduct oversight, mitigate risk, meet critical public health needs and help maximize compliance of FDA-regulated products. The draft guidance provides answers to frequently asked questions regarding what RRAs are, when and why FDA may use them, and how FDA may conduct then, among others.

RRAs have included: (1) mandatory RRAs involving review of records or other information submitted by certain establishments upon request from FDA and review of records from food establishments and (2) voluntary RRAs involving remote requests for records and/or interactive evaluation. FDA’s experiences have identified significant benefits in using RRAs. Based on their experiences, FDA has determined that RRAs are valuable and, therefore, under certain circumstances, should continue to assist FDA in its mission to protect public health, oversee regulated industry, and ensure all types if regulated products comply with FDA requirements.

The full document can be found at the following address:

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/conducting-remote-regulatory-assessments-questions-and-answers

FDA Opens Doors for More Treatments for Rare Diseases through the New START Pilot Program

June 27, 2024

The FDA has launched the Support for clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program to accelerate the development of treatments for rare diseases, recognizing the need for more therapeutic options in this area. CDER selected three development programs for novel drugs intended to treat rare neurodegenerative conditions. CBER selected four development programs for gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first 10 years of life.

Full document: https://www.fda.gov/drugs/our-perspective/fda-opens-doors-more-treatments-rare-diseases-through-new-start-pilot-program

Pharmaceutical Quality – Chemistry, Manufacturing & Controls

April 19, 2024

FDA and Health Level 7 (HL7) held the first ballot for the Stage 1 FHIR Implementation Guide (IG) for Pharmaceutical Quality – Chemistry, Manufacturing & Controls (PQ/CMC) Submissions to the FDA in May 2024. The IG is a set of instructions for implementing the FHIR standard in a specific healthcare context. The IG’s requirements are based on the International Council for Harmonisation (ICH) Quality Guidelines.

This is a project that FDA has undertaken to identify and prioritize PQ/CMC information that would benefit from a structured submission approach.

The goal of this project is to establish electronic standards for submitting PQ/CMC data, which is intended to be included in the Modules 3/2.3 of applications submitted to FDA. The effort is scoped to some of what is currently submitted in Module 3 and Module 2.3 of the eCTD submission. It is not intended to be comprehensive in structuring all eCTD product quality information, only those concepts that were considered amenable to structuring and would bring value to the quality review process.

Full document: https://www.fda.gov/industry/fda-data-standards-advisory-board/pharmaceutical-quality-chemistry-manufacturing-controls-pqcmc

Platform Technology Designation Program

May 29, 2024

The FDA is announcing the availability of a draft guidance for industry entitled “Platform Technology Designation Program for Drug Development.” The purpose is to provide details about the implementation of the Platform Technology Designation Program established by the PREVENT Pandemics Act. This guidance outlines eligibility factors for receiving a platform technology designation, potential benefits of receiving a designation, how to leverage data from designated platform technologies, how to discuss a planned designation request as part of a milestone meeting, the recommended content of a designation request submission, and the review timelines for a designation request.

Full documents: https://www.federalregister.gov/documents/2024/05/29/2024-11686/platform-technology-designation-program-draft-guidance-for-industry-availability-agency-information

https://www.federalregister.gov/documents/2024/05/29/2024-11686/platform-technology-designation-program-draft-guidance-for-industry-availability-agency-information

Other FDA Updates

 EU (European Union) / EMA (European Medicines Agency)

Need for maximum residue limit evaluation for biological substances

May 7, 2024

This guideline addresses the need for a maximum residue limit (MRL) evaluation for biological substances, when it is intended that those substances are included in products for use in food producing species. This guidance was firstly adopted by CVMP in Jan. 17, 2024 and the final version will come into effect till August. 1, 2024.

This guidance is to provide structured and transparent assessment criteria on how to determine the need for an MRL evaluation according to Regulation (EC) No 470/2009 for ‘chemical-unlike’ biologics. Chemical-like biological substances are outside of scope of this guidance. To this end a stepwise (tiered) approach (decision tree) using a set of consecutive questions/criteria has been developed.

Two outcomes can result from the assessment procedure: (1) the ‘chemical-unlike’ biological can be added to the list of biologics if no MRL assessment is considered necessary, (2) an MRL procedure would be necessary if certain properties of the ‘chemical-unlike’ biological reveal that at least some data required for the establishment of MRLs.

Full document: https://www.ema.europa.eu/en/need-maximum-residue-limit-mrl-evaluation-biological-substances-scientific-guideline

Implementation of ISO standards for the identification of medicinal products (IDMP) in Europe Introduction

May 14, 2024

The ISO IDMP standards ensure standardized definitions for identifying and describing medicinal products for human use, facilitating consistent and reliable information exchange among stakeholders. Mandated by EU regulations, these standards must be used by member states, marketing authorization holders, and the EMA for medicinal product communication. EMA supports this implementation through SPOR (Substance, Product, Organization, and Referential Management Services), providing technical specifications, messaging standards, and EU-specific guidance. The EU Implementation Guide (EU IG) outlines registration requirements, data elements for submission, data access levels, and migration rules for existing systems, with periodic updates reflecting the latest agreements and improvements. User access to the SPOR system is governed by a detailed access policy, and a subset of data will be publicly accessible, ensuring transparency while maintaining confidentiality.

The EMA is implementing ISO standards for the identification of medicinal products (IDMP) in the EU, as mandated by Commission Implementing Regulation (EU) No 520/2012. This phased implementation involves standardizing data across four domains: Substance, Product, Organisation, and Referential (SPOR) master data, to enhance the exchange and reliability of medicinal product information. The document outlines the onboarding process for SPOR data services, including user registration, access management, and training requirements for submitting and maintaining product information in the Product Management Service (PMS). Standardized data will improve regulatory efficiency, reduce data silos, and provide operational savings by reusing data across different procedures and regulators. Access to PMS data is managed through SPOR APIs and a Product User Interface, with specific roles and permissions defined for various stakeholders, ensuring compliance with data protection regulations.

Full document: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/products-management-services-implementation-international-organization-standardization-iso-standards-identification-medicinal-products-idmp-europe-introduction-eu-implementation-guide_en.pdf

Other EMA Updates

TGA (Therapeutic Goods Administration)

Pilot GMP Single Inspection Program

February 20, 2024

Health Canada, the Therapeutic Goods Administration (TGA) and the Medicines & Healthcare products Regulatory Agency (MHRA), all members of the Pharmaceutical Inspection Co-operation Scheme (PIC/S), International Coalition of Medicines Regulatory Authorities (ICMRA) and Access Consortium, have begun piloting a GMP Single Inspection Program (SIP). This pilot aims to establish a coordinated global approach to GMP inspections of foreign manufacturing sites of common interest. Using their collective inspection resources, each authority has agreed to extend the scope of an inspection to cover products of interest to one another, where possible, reducing the need for multiple inspections of the same site. This builds on the success of their existing collaborative GMP arrangements and will allow for more efficient inspection reliance processes, reduced regulatory burden on industry and enhanced collaboration in their regulatory oversight of common global supply chains.

Full document: https://www.tga.gov.au/news/notices/pilot-gmp-single-inspection-program

Other TGA Updates

Health Canada

No updates provided by WuXi Biologics for the time period. To see past updates for this agency, go to our regulatory newsletter archive.

WHO (World Health Organization)

WHO launches Pharmaceutical Quality Assurance guidelines, 10th edition

February 29, 2024

WHO launches Pharmaceutical Quality Assurance guidelines (10th edition·Volume 2) which guides the manufacturing and quality control of pharmaceuticals, vaccines and other biologicals and other medical products to ensure their quality, safety, and efficacy, focusing on good manufacturing practices (GMP) and inspection.

The compendium is organized into sections: GMP main principles, starting materials, specific medical products, related guidelines, laboratory guidelines and inspections. The guidelines feature several updates and new texts to ensure the recommendations are fit-for-purpose and current. The latest edition of the Compendium includes 45 guidelines, of which 10 guidelines have been revised to incorporate the latest advancements and address emerging challenges in the pharmaceutical industry, while 8 new guidelines have been introduced to address previously unexplored areas. These new topics include GMP for investigational radiopharmaceuticals and medicinal gases, recommendations on environmental aspects for the prevention of antimicrobial resistance, health-based exposure limits in cleaning validation and good practices for research and development facilities.

Full document: https://www.who.int/news/item/29-02-2024-who-launches-pharmaceutical-quality-assurance-guidelines–10th-edition

WHO GMP for excipients used in pharmaceutical products

April 12, 2024

On 12 April, the World Health Organization (WHO) published the draft guidance on “WHO GMP for Pharmaceutical Excipients – Annex 1: Risk Management for the Production and Control of Pharmaceutical Excipients” and “WHO GMP for excipients used in pharmaceutical products – Appendix 2: List of examples of high-risk excipients” to provide guidance to manufacturers, packers and distributors of pharmaceutical excipients to identify risks and take steps to ensure the safety, purity and quality of such excipients and finished formulations. Now they’re inviting comments and posting of the working document on the WHO website for public consultation. The appendix 1 introduces risk identification and risk assessment, individual excipient risk assessment, aggregate excipient risk assessment, nitrosamine contamination, examples, risk control, risk communication, and risk review. The purpose of appendix 2 is to provide an example list of high-risk excipients and contaminants, and to remind manufacturers of such excipients, as well as producers of finished formulations containing such excipients, to take steps to ensure the safety, purity, and quality of such excipients and finished formulations.

Full document: https://cdn.who.int/media/docs/default-source/medicines/norms-and-standards/current-projects/qas24_945_excipients_appendix1_forpublic.pdf?sfvrsn=38184f27_2

MHRA 

MHRA’s new International Recognition Procedure (IRP) goes live from 1 January 2024

January 2, 2024

Since the Brexit, the MHRA has created a new International Recognition Procedure (IRP) recognition rout to accelerate patient access to new medicines on the NHS and putting the UK on the forefront of medical innovation. This new international recognition procedure will speed up the licensing process by allowing access to the expertise of other trusted regulatory partners including Australia, Canada, the European Union, individual EU member states and those in the EEA (European Economic Area), Japan, Switzerland, Singapore and the United States. From 1 January 2024, applicants can submit applications via this new route, International Recognition procedure (IRP). The new IRP will replace the previous EC Decision Reliance Procedure (ECDRP). There are two recognition timetables for initial MAAs: 60-day timetable and 110-day timetable (Recognition A and Recognition B). To be eligible for Recognition A, the Reference Regulators (RR) approval must have been granted within the previous 2 years. To be eligible for Recognition B, the RR approval should have been granted within the previous 10 years.

Full document: https://www.gov.uk/government/news/mhras-new-international-recognition-procedure-irp-goes-live-from-1-january-2024

Project Orbis

January 4, 2024

Project Orbis is a programme to review and approve promising cancer drugs helping patients access treatments faster. It provides a framework for concurrent submission and review of oncology products among international partners. The programme is coordinated by the US Food and Drug Administration (FDA). Alongside MHRA, it involves the regulatory authorities of Australia (Therapeutic Goods Administration (TGA)), Canada (Health Canada), Singapore (Health Sciences Authority (HSA)), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA)), Israel (Ministry of Health).

Full document: https://www.gov.uk/guidance/guidance-on-project-orbis#full-publication-update-history

Project Orbis

January 4, 2024

Project Orbis is a programme to review and approve promising cancer drugs helping patients access treatments faster. It provides a framework for concurrent submission and review of oncology products among international partners. The programme is coordinated by the US Food and Drug Administration (FDA). Alongside MHRA, it involves the regulatory authorities of Australia (Therapeutic Goods Administration (TGA)), Canada (Health Canada), Singapore (Health Sciences Authority (HSA)), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA)), Israel (Ministry of Health).

Full document: https://www.gov.uk/guidance/guidance-on-project-orbis#full-publication-update-history

Impact of AI on the regulation of medical products

April 30, 2024

The MHRA is considering the opportunities and risks of Artificial Intelligence (AI), which has been identified as one of the five critical technologies in the UK Science and Technology Framework, from three different perspectives: as a regulator of AI products, as a public service organization delivering time critical decisions, and as an organization that makes evidence-based decisions that impact on public and patient safety. There are five key principles introduced by the Pro-innovation approach to the regulation of AI: 1) safety, security and robustness, 2) appropriate transparency and explainability, 3) fairness, 4) accountability and governance, and 5) contestability and redress.

MHRA are committed to taking a propriate approach to regulating AI medical products, which takes into account the unique challenges of these products. MHRA’s collaboration with the IMDRF and in particular our partnership with the US FDA and Health Canada, will ensure safe and responsible use of AI in medical products and keep the UK at the forefront of international best practice.

Full document: https://www.gov.uk/government/publications/impact-of-ai-on-the-regulation-of-medical-products

PMDA (Pharmaceuticals and Medical Devices Agency)

PMDA Updates

ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use)

ICH Updates

EDQM (European Directorate for the Quality of Medicines & HealthCare)

New General Chapter on Extractable Elements in Plastic Materials for Pharmaceutical Use

June 26, 2024

The European Pharmacopoeia has adopted a new general chapter, titled “Extractable elements in plastic materials for pharmaceutical use” (2.4.35), which focuses on the assessment of extractable elements in plastic materials used in pharmaceutical containers and closures. This development is in line with the ICH Q3D guideline on elemental impurities, which advocates for a comprehensive control strategy for elemental impurities at the product level rather than just substance-based testing. Although the plastics used in pharmaceutical containers are not directly covered by the ICH Q3D guideline, their quality significantly affects the final product’s quality. The new procedure developed by the Ph. Eur. Group 16 aims to provide a framework for assessing extractable elements in plastics, enhancing the safety and quality of pharmaceutical products. The chapter was adopted in March 2024 and will be published in September 2024, with its requirements soon to be referenced in specific chapters related to individual plastic materials.

Full document: https://www.edqm.eu/en/-/new-general-chapter-on-extractable-elements-in-plastic-materials-for-pharmaceutical-use-2.4.35-adopted

EDQM Updates

PIC/S (Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme)

No updates provided by WuXi Biologics for the time period. To see past updates for this agency, go to our regulatory newsletter archive.

Health Products Regulatory Authority (HPRA)

HPRA Updates

National Medical Products Administration (NMPA)

Industrial Structure Adjustment Guidance Catalogue

December 27, 2023

According to the “Decision of the State Council on the Issuance and Implementation of the ‘Interim Provisions on Promoting Industrial Structure Adjustment'” (Council of the state No. 40[2005]), the National Development and Reform Commission, in conjunction with relevant departments, has jointly revised and formed the “Guidance Catalogue for Industrial Structure Adjustment (2024 Edition)” (hereinafter referred to as the “Catalogue (2024 Edition)”), which was approved at the 6th executive meeting on December 1, 2023, and will be implemented from February 1, 2024. The “Catalogue (2024 Edition)” consists of three categories: encouraged, restricted, and eliminated.

Full documents: https://www.ndrc.gov.cn/xxgk/zcfb/fzggwl/202312/t20231229_1362999.html

https://www.ndrc.gov.cn/xxgk/jd/jd/202312/t20231229_1363020.html

Technical Guidance for the Validation of Viral Clearance Processes for Therapeutic Recombinant Protein Products in Clinical Trial Applications

January 16, 2024

Under the deployment of the National Medical Products Administration, the Center for Drug Evaluation has organized and drafted the “Technical Guidance Principles for Viral Clearance Process Platform Validation in Clinical Trial Applications of Therapeutic Recombinant Protein Products (Trial)” to guide the viral safety control of biological products. This guidance was issued and implemented on January 16, 2024.

This guidance discusses the considerations for clinical trial applications of therapeutic recombinant protein products using platform validation as an alternative to conducting viral clearance process validation with specific products.

Full document: https://www.cde.org.cn/main/news/viewInfoCommon/52daa6be5e529f9e3b1f5c89fc5607ce

Guidance for the Preparation of the Site Master File

March 27, 2024

The CFDI has formulated the “Guidance for the Preparation of the Site Master File (Trial).” This guidance document details the format and specific contents of the Site Master File, which mainly includes 10 sections: Company Overview, Quality Management System, Organization and Personnel, Plant Facilities and Equipment, Documentation, Production, Quality Control, Distribution/Complaints and Recalls, Self-Inspection, and Revision History.

Full document: https://www.cfdi.org.cn/resource/news/15871.html

Regulations on the Management of Key Personnel in Quality and Safety for Pharmaceutical Manufacturing and Distribution Enterprises in Shanghai

January 16, 2024

The CFDI has formulated the “Guidance for the Preparation of the Site Master File (Trial).” This guidance document details the format and specific contents of the Site Master File, which mainly includes 10 sections: Company Overview, Quality Management System, Organization and Personnel, Plant Facilities and Equipment, Documentation, Production, Quality Control, Distribution/Complaints and Recalls, Self-Inspection, and Revision History.

Full documents: https://yjj.sh.gov.cn/zx-yp/20240116/26a470e2cbc84bde8baa376c054bd162.html

https://yjj.sh.gov.cn/zcjd/20240117/2fb121d8ba734123b18b2eddfdadac9c.html

The Provincial Drug Administration, in collaboration with the Provincial Free Trade Office and other departments, issued a pilot program to support the import of “white list” items for biomedical research and development in Nanjing and Wuxi

January 18, 2024

On January 15, the Jiangsu Provincial Drug Administration, in conjunction with the Provincial Free Trade Office, Provincial Science and Technology Department, and Nanjing Customs, jointly issued the “Notice on the Issuance of the Pilot Scheme for the ‘Whitelist’ System for Biomedical Research and Development Materials in Nanjing and Wuxi.” The aim is to enhance the level of customs clearance facilitation for biomedical research and development materials in the free trade pilot zones and pilot cities for cross-border trade facilitation, and to support the innovative and high-quality development of the provincial biomedical industry. From the date of the document’s issuance, six product specifications from four pharmaceutical companies in Nanjing and Wuxi can be directly imported without the need for an “Import Drug Customs Clearance Form.”

Full document: https://da.jiangsu.gov.cn/art/2024/1/18/art_84602_11128736.html

Technical Guideline for Pharmaceutical Research and Evaluation of Clinical Trial Applications for Recombinant Adeno-Associated Virus Vector-Based In Vivo Gene Therapy Products

January 19, 2024

The Center for Drug Evaluation organized the formulation of the “Technical Guideline for Pharmaceutical Research and Evaluation of Clinical Trial Applications for Recombinant Adeno-Associated Virus Vector-Based In Vivo Gene Therapy Products,” which was published and implemented on January 19, 2024. This guideline primarily details the pharmaceutical information required in the submission documents for recombinant adeno-associated virus (rAAV) vector products, covering aspects such as scope of application, general principles, production materials, production processes, quality research and quality standards, stability studies, packaging, and the container closure system.

Full document: https://www.cde.org.cn/main/news/viewInfoCommon/635323ee319dacab26eb783b843b8bd3

Technical Guidance Principles for the Pharmaceutical Research and Evaluation of Antibody-Drug Conjugates

February 2, 2024

To standardize and guide the research and development as well as the application process of Antibody-Drug Conjugate (ADC) products, the Center for Drug Evaluation has organized and drafted the “Technical Guidance for the Pharmaceutical Research and Evaluation of Antibody-Drug Conjugates.” This document outlines the general requirements for ADC products in terms of product design, production process, quality research and control, and formulation development. It also summarizes considerations for different stages of ADC product development. This guidance is effective from the date of publication.

Full document: https://www.cde.org.cn/main/news/viewInfoCommon/923172e1cf1c6eb92737c21ef3b93bc4

Announcement of the National Medical Products Administration on Optimizing the Drug Marketing Application for Transferring Domestic-marketed Drugs that are Overseas-produced to Domestic Production

April 23, 2024

The optimization items for transferring overseas-produced and domestic-marketed drugs to domestic production include: The application should be made by the domestic applicant in accordance with the requirements and procedures for drug marketing registration applications; It is possible to submit the original registration application materials for the drugs produced overseas and submit relevant research materials for the transfer to domestic production to support their drug marketing registration application; For the application for drug marketing registration of original research chemical drugs and biological products transferred to domestic production, the National Medical Products Administration includes it in the scope of priority review and approval.

Full document: https://www.nmpa.gov.cn/xxgk/ggtg/ypggtg/ypqtggtg/20240423144218148.html

Dossier Requirements for Drug Marketing Application for Transferring Domestic-marketed Drugs that are Overseas-produced to Domestic Production (Therapeutic Biological Products)

June 14, 2024

To transfer domestic-marketed therapeutic biological products that are overseas-produced to domestic production, the domestic applicant should submit a marketing registration application in accordance with category 3.4 for therapeutic biological products. The main body introduces simplified situations and requirements for application dossiers. The general requirements for simplified dossiers are introduced from Module 1 to Module 5, with a focus on Module 3 where applicants can submit change research materials related to raw materials for production, prescription processes, quality studies, stability, packaging materials in direct contact with the drug, and related review and approval, according to the characteristics of different categories of therapeutic biological products. Simplifications or exemptions can be made based on the actual situation of the transfer from overseas to domestic production.

Full document: https://www.cde.org.cn/main/news/viewInfoCommon/f37332951c50ea97f7b642b2cf42b3a4

Technical Guidance for Pharmaceutical Research and Changes of Biological Products During Clinical Trials (Trial Implementation) and Technical Guidance for Pharmaceutical Changes of Marketed Vaccines (Trial Implementation)

June 14, 2024

The CDE has released announcements for the “Technical Guidance for Pharmaceutical Research and Changes of Biological Products During Clinical Trials (Trial Implementation)” and the “Technical Guidance for Pharmaceutical Changes of Marketed Vaccines (Trial Implementation)”.

The “Technical Guidance for Pharmaceutical Research and Changes of Biological Products During Clinical Trials (Trial Implementation)” covers the basic considerations for changes in biological products during clinical trials, pharmaceutical change risk assessment, comparability studies and communication. The phased requirements for pharmaceutical research are introduced separately for drug substance and drug product according to the CTD format, detailing the basic considerations and phased requirements for changes in each section. Changes in drug substance include production, characterization, quality control, reference standards, packaging systems, and stability, while changes in drug product include product development and manufacturing, excipient control, quality standards, reference standards, packaging systems, and stability. Finally, the document outlines the assessment of pharmaceutical changes and their corresponding application methods, listing changes that may increase safety risks.

The “Technical Guidance for Pharmaceutical Changes of Marketed Vaccines (Trial Implementation)” is drafted based on the “Technical Guidance for Pharmaceutical Changes of Marketed Biological Products (Trial Implementation)” and focuses on the specificity and characteristics of vaccines, especially regarding production process changes. It introduces related changes, adjuvants, quality characteristic studies, bridging studies, and considerations for seasonal influenza vaccines. The “Categories and Technical Requirements of Vaccine Pharmaceutical Changes” section lists common vaccine pharmaceutical changes, especially production process changes, defines the categories of specific changes, the prerequisites that need to be met, and the basic technical requirements.

Full document: https://www.cde.org.cn/main/news/viewInfoCommon/e56700d8d2aaf1394610b2c47d8c6a5c

Other NMPA Updates