Regulatory Round-up - January 2025

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Implementation of medical devices future regime

On 21 October 2024, MHRA published a guidance to help medtech manufacturers comply with its updated post-market surveillance (PMS) regulation. The draft Post-Market Surveillance Statutory Instrument was laid in Parliament, and it was signed into law on 16^th December 2024. These regulations will come into force following a 6-month transition period on 16 June 2025. The aim of the new regulations is to introduce clearer and more robust requirements for PMS that improve patient safety. Please find further information here.

MHRA Scientific Advice: advance notice of changes

From 21 January 2025, the MHRA will review all new requests for scientific advice meetings against whether the requirements can be addressed with existing guidance, written advice, or a scientific advice meeting. Therefore, temporarily an increased number of requests will be progressed as written-only advice from MHRA specialists. Full guidance on this change will be published on 31 January 2025. Please find the MHRA advance notice here.

A National Institute for Health and Care Excellence (NICE)

New guidance on the use of surrogate endpoints in cost-effectiveness analysis for use in Health Technology Assessment (HTA)

NICE collaborated with international HTA agencies from the US, Canada, Australia, the Netherlands, and Columbia to produce best practice recommendations for using surrogate endpoints in health economic models to guide HTA decisions. Surrogate endpoints are biomarkers or other intermediate outcomes that predict a treatment effect on a final clinical outcome and these outcomes are increasingly being used in licensing. The new guidance provides clarity and support for technology developers when using surrogate endpoints for analysing the cost-effectiveness of the drugs or devices they are developing. The white paper, available to download here, outlines how to select and check the surrogate endpoints, the selection of the longer-term benefits, and how to display them in evidence submissions. Please find further information here.

EUROPE

European Medicines Agency (EMA)

Q&A clinic on web-based electronic Application Form (eAF) functionalities for centrally authorised products (CAPs) and non-CAPs variations

EMA will hold an online event on 13^th March 2025 where the web-based electronic Application Form (eAF) team answers questions on the eAF form functionalities for CAPs and non-CAPs' variations. Please find registration details here.

Pharmacovigilance risk assessment committee (PRAC): Work plan 2025

Please find the PRAC work plan which outlines the pharmacovigilance objectives and activities for 2025.

USA

Food and Drug Administration (FDA)

Recommendations to reduce the risk of transmission of mycobacterium tuberculosis (Mtb) by human cells, tissues, and cellular and tissue-based products (HCT/Ps)

FDA has issued a guidance to provide recommendations for making donor eligibility (DE) determinations, in line with the requirements in 21 CFR part 1271, subpart C (21 CFR part 1271, subpart C). The regulations under 21 CFR part 1271, subpart C, set out requirements for determining DE, including donor screening and testing, for donors of human cells, tissues, and cellular and tissue-based products (HCT/Ps). This guidance provides recommendations for screening donors for evidence of, and risk factors for, infection with mycobacterium tuberculosis (Mtb), the organism that causes tuberculosis. The guidance also recommends additional steps that HCT/P establishments should take to reduce risk of transmission of Mtb until such time as appropriate FDA-licensed, approved, or cleared donor screening tests are available for use to test donors for Mtb infection.

Considerations for the use of artificial intelligence to support regulatory decision-making for drug and biological products

FDA has issued a draft guidance to provide recommendations to sponsors and other interested parties on the use of artificial intelligence (AI) and to produce information or data intended to support regulatory decision- making regarding safety, effectiveness, or quality for drugs. It discusses the use of AI models in the drug product life cycle, where the specific use of the AI model is to produce information or data to support regulatory decision-making regarding safety, effectiveness, or quality for drugs. Comments can be submitted until 7^th April 2025.

Considerations for complying with 21 CFR 211.110

The FDA has issued a draft guidance, which describes considerations for complying with the requirements in 21 CFR 211.110 to ensure batch uniformity and drug product integrity. It discusses related quality considerations for drug products that are manufactured using advanced manufacturing as well as provides recommendations on how manufacturers can incorporate process models into commercial manufacturing control strategies. Comments can be submitted until 7^th April 2025.

Communications from firms to health care providers (HCPs) regarding scientific information on unapproved uses of approved/cleared medical products: questions and answers guidance for industry

The FDA has issued a final guidance describing the FDA’s enforcement policy regarding certain firm-initiated communications of scientific information on unapproved use(s) of the firm’s approved/cleared medical products to HCPs engaged in prescribing or administering medical products to individual patients.

Study of sex differences in the clinical evaluation of medical products

FDA has issued a draft guidance to provide recommendations for increasing enrolment of females in clinical trials, analysing and interpreting sex-specific data, and including sex-specific information in regulatory submissions of medical products. When finalized, this guidance will replace the guidance titled ‘Guideline for the Study and Evaluation of Gender Differences in the Clinical Evaluation of Drugs’ issued in July 1993. Comments can be submitted until 7^th April 2025.

Joint US FDA – Health Canada International Conference on Harmonisation (ICH) public meeting 2025

FDA and Health Canada will be co-hosting a regional public meeting to provide information to stakeholders and solicit input prior to the next ICH Biannual Assembly meeting scheduled for 13-14 May 2025. This joint public meeting will include presentations by FDA, Health Canada, and Pharmaceutical Research and Manufacturers of America experts on ICH guidelines recently reaching significant ICH milestones. Please find the agenda and information on how to register here.

Combined FDA and sponsor oncologic drugs advisory committee briefing document

The FDA has announced the availability of a draft guidance for industry entitled “Combined FDA and Sponsor Oncologic Drugs Advisory Committee (ODAC) Briefing Document.” It provides recommendations to sponsors regarding the use and development of a combined version of the ODAC briefing document, as part of the Oncology Center of Excellence’s Project Point/Counterpoint initiative. This document includes information that customarily would be contained in separate briefing documents prepared individually by the Sponsor and FDA. Comments can be submitted until 28^th February 2025.

Considerations for including tissue biopsies in clinical trials

FDA has issued a draft guidance to provide recommendations to industry, investigators, institutions, and institutional review boards regarding considerations for tissue biopsies that may be conducted in adults and in children as part of clinical trials that evaluate investigational medical products and/or that are conducted or supported by the Department of Health and Human Services. Comments can be submitted until 10^th March 2025.

INTERNATIONAL

International Council for Harmonisation (ICH)

The ICH E6(R3) Guideline reaches Step 4 of the ICH Process

ICH E6(R3) principles and Annex 1 have been adopted on 6^th January 2025 and published on the ICH website. This is a rewrite and reorganisation of the R2 version of the guideline on Good Clinical Practice (GCP), which provides a unified standard to facilitate the mutual acceptance of clinical trial data for ICH member countries and regions by applicable regulatory authorities. Further information can be found on the E6(R3) page, including the Guideline and the Step 4 Introductory Training Presentation. Annex 2 of the guideline is being developed in parallel. It provides additional GCP considerations focusing on examples of trials that incorporate decentralised elements, pragmatic elements, and/or real-world data. A draft of Annex 2 is currently under public consultation and will conclude on 28 February 2025.

Public consultations

The British Pharmacopoeia (BP)

Medicines and Healthcare products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

International Council for Harmonisation (ICH)

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