Latest Regulatory Updates

January 2025

Happy New Year and welcome back to the Boyds Regulatory Intelligence Digest. To kickstart 2025, we are sharing the latest guidance, insights and key developments from the regulatory agencies - FDA, EMA, MHRA, and EC.

Food and Drug Administration (FDA)

Considerations for the Use of Artificial Intelligence (AI) To Support Regulatory Decision-Making for Drug and Biological Products

FDA has issued draft guidance on the use of artificial intelligence (AI) in regulatory decision-making for drugs and biological products. This guidance offers recommendations to sponsors and other stakeholders on using AI-generated information or data to support regulatory evaluations of safety, effectiveness, or quality.

The document introduces a risk-based credibility assessment framework designed to establish and evaluate the credibility of AI models within specific contexts of use (COU).

The draft guidance is currently open for public comment, and interested parties are encouraged to submit their feedback by April 7, 2025.

Artificial Intelligence-Enabled Device Software Functions: Lifecycle Management and Marketing Submission Recommendations

This draft FDA guidance document provides recommendations regarding the contents of marketing submissions for devices that include artificial intelligence (AI)-enabled device software functions including documentation and information that will support FDA’s evaluation of safety and effectiveness. The recommendations reflect a comprehensive approach to the management of risk throughout the device total product life cycle (TPLC). The guide also proposes recommendations for the design, development, and implementation of AI-enabled devices that manufacturers may wish to consider using throughout the TPLC.

Comments can be submitted until April 7, 2025.

FDA issued Draft Guidance: Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway. 

For drugs granted accelerated approval, sponsors are required to conduct post-approval confirmatory studies to verify and describe the anticipated effects on irreversible morbidity, mortality, or other clinical benefits. This draft guidance outlines the FDA's interpretation of the term "underway" and discusses policies for implementing this requirement. It also details the factors the FDA will consider when determining whether a confirmatory trial is underway before granting accelerated approval.

Comments can be submitted until March 10, 2025.

FDA issued draft guidance:  Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)

The purpose of this guidance is to assist establishments in making donor eligibility determinations in compliance with the requirements in Title 21 of the Code of Federal Regulations, part 1271, subpart C (21 CFR part 1271, subpart C). This guidance applies to human cells and tissues recovered on or after May 25, 2005, the effective date of the regulations outlined in 21 CFR part 1271, subpart C, and updates the August 2007 guidance, Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps), Guidance for Industry.

When finalized, the guidance will provide establishments with updated recommendations for appropriately screening and testing HCT/P donors for communicable disease risks, clarify recommendations that have been the subject of frequent stakeholder inquiries, and correct outdated references, URLs, physical addresses, and FDA contact information.

Comments can be submitted until February 6, 2025.

Protocol Deviations for Clinical Investigations of Drugs, Biological Products, and Devices

This draft guidance provides recommendations to assist sponsors, clinical investigators, and institutional review boards (IRBs) in defining, identifying, and reporting protocol deviations in clinical investigations. The guidance includes definitions for protocol deviations and important protocol deviations, recommendations on the types of protocol deviations that sponsors should report to FDA in clinical study reports for drugs and devices, recommendations on the types of protocol deviations that investigators should report to sponsors and to IRBs, and recommendations for IRBs in their evaluation of protocol deviations.

Comments can be submitted until February 28, 2025.

Accelerated Approval – Expedited Program for Serious Conditions | FDA

The purpose of this draft guidance is to provide information on FDA’s policies and procedures for accelerated approval as well as threshold criteria generally applicable to concluding that a drug is a candidate for accelerated approval. This guidance also describes the procedures for expedited withdrawal of approval of a product approved under accelerated approval and the revisions Congress made through the Consolidated Appropriations Act, of 2023. 

Comments can be submitted until February 02, 2025.

European Medicines Agency (EMA) and the European Union (EU)

New EU rules for health technology assessments become effective

The new regulation on health technology assessment (HTAR) (Regulation (EU) 2021/2282) became applicable on 12 January 2025. The new rules will initially apply to new active substances to treat cancer and to all advanced therapy medicinal products (ATMPs). They will be expanded to orphan medicinal products in January 2028, and to all centrally authorized medicinal products as of 2030. Selected high-risk medical devices will also be assessed under the HTAR as of 2026.

The regulation is an important step forward in accelerating and widening access to new medicines. The EMA has been supporting the preparation for the implementation of the regulation by working closely with the European Commission, EU Member States and stakeholders representing the pharmaceutical industry, healthcare professionals, patients, and academia. Further details are available here.

EMA and HMA update principles for identification and disclosure of commercially confidential information and personal data

The EMA and HMA (Heads of Medicines Agencies) have updated guidance on identifying and disclosing commercially confidential information (CCI) and personal data in marketing authorization applications for human medicines.

As a general rule, the overwhelming majority of data in marketing-authorization applications is not considered CCI. While considered CCI at the time of the initial guidance, general information related to quality is now mostly considered releasable.

The updated guidance considers information as releasable by default. It provides detailed practical orientations as to which specific points could be redacted or anonymized within each section of the dossier. The annex of the guidance document has been updated and now includes examples of information that may be considered CCI or protected personal data. The guidance also sets out how personalized data will be protected if it can lead to the identification of a person.

HMA/EMA multi-stakeholder workshop on artificial intelligence (AI) - enabling the safe and responsible use of AI

EMA has posted the video of a workshop on artificial intelligence (AI) held in November 2024, available here.

Medicines and Healthcare products Regulatory Agency (MHRA), HRA & GOV.UK

Real-World Evidence (RWE) and proactive approaches to safety surveillance

MHRA is launching a pilot RWE Scientific Dialogue Program in 2025. This initiative is designed to help innovators refine their evidence-generation strategies while providing clear guidance on regulatory expectations. The program aims to facilitate robust decision-making across the entire lifecycle of products, benefitting both regulatory and health technology assessment (HTA) evaluations relevant to the UK.
Details of the program are available here.

New MHRA Chair Anthony Harnden outlines priorities on start of new role

Professor Anthony Harnden has officially started his role as Chair of the Medicines and Healthcare products Regulatory Agency (MHRA).

Medical devices: post-market surveillance Updates

MHRA has published guidance to help medical device manufacturers prepare for the new Post-market Surveillance (PMS) regulation in Great Britain, effective June 16, 2025. Key requirements include enhanced data collection, shorter timelines for reporting serious incidents, and clearer obligations for risk mitigation and communication to protect patients and users.

Businesses are encouraged to begin using the guidance immediately to ensure they understand their obligations and are ready to comply when the regulations come into effect.

European Commission (EC)

Commission launches a public consultation and a call for evidence for EU Medical Devices evaluation

The Commission has launched a public consultation and a call for evidence on the EU’s legislation on medical devices and in vitro diagnostic medical devices, as part of the targeted evaluation of these rules. The consultation provides stakeholders with the opportunity to express their views on how the current rules are performing and to highlight possible shortcomings. The consultation and call for evidence will be open until 21 March 2025 and are accessible here.

MDCG 2023-3 rev.2 - Questions and Answers on vigilance terms and concepts

MDCG has issued an update to MDCG 2023-3 rev.2 - Questions and Answers on Vigilance Terms and Concepts under Regulations (EU) 2017/745 (MDR) and 2017/746 (IVDR), dated January 2025. This document clarifies key terms and concepts from Section 2 of Chapter VII of the MDR and IVDR, incorporating relevant updates from the Guidelines on a Medical Devices Vigilance System with modifications for alignment.

Key updates are to Q1 has been amended to align with Regulation (EU) 2024/1860 amending Regulations (EU) 2017/745 and (EU) 2017/746 as regards a gradual roll-out of Eudamed, the obligation to inform in case of interruption or discontinuation of supply, and transitional provisions for certain in vitro diagnostic medical devices. Question 2 Reference to ‘Eudamed vigilance (VGL) module’ is amended to ‘Eudamed Postmarket surveillance and Vigilance module (VGL module)’.  Footnote 34   ’48 working hours’ replaced with ‘allow 48 hours (equivalent to two weekdays)’.

The guide is available here.

Combined studies and COMBINE strategy
December 16^th, 2024 EU National authorities in Member States have endorsed a new strategy for the COMBINE program, (a cross-sector initiative to streamline combined studies of medicines and medical devices, including diagnostics).

The strategy can be found on the Commission’s webpage: Combined studies and COMBINE strategy

The COMBINE program will be rolled out over the coming years through seven cross-sector projects. Among the goals of this program are to:

• Pilot a single assessment process for multi-country combined studies across device and medicinal product regulations
• Harmonise serious adverse event reporting processes
• Clarify questions on the interface between clinical trials and medical device regulations
• Explore new opportunities for advising sponsors and facilitating knowledge exchange among national authorities

Podcast: Conversations in Drug Development

Understanding EU Medical Device Regulations: Key Insights for Developers and Innovators

In our latest episode of Conversations in Drug Development, host Harriet Edwards is joined by Eamonn McGowran, Associate Director of Regulatory Affairs at Boyds, to explore the complex and rapidly evolving regulatory landscape for medical devices, particularly within the EU.

Join us as we discuss the challenges of developing combination products that integrate devices and pharmaceuticals, the roles of notified bodies and competent authorities, and the importance of early stakeholder engagement. In this episode we also cover the integration of artificial intelligence in medical devices and the evolving UK regulatory framework post-Brexit.

Visit, Podcast | Conversations in Drug Development | Boyds (boydconsultants.com) or listen on Spotify, Amazon Music or Apple Podcasts by searching for "Conversations in Drug Development".