Regulatory Round-up - December 2024

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Statement regarding the new Clinical Trials Framework

The Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2024 were laid before Parliament on 12 December 2024. When approved by Parliament, this legislation will represent the most significant reform of UK clinical trials regulation in over 20 years, addressing the sector's need for a more efficient and adaptable regulatory framework. The amended regulatory framework will aim to speed up trial approvals and encourage innovation in trial design without compromising patient safety.

The reforms will:

• Create a proportionate and flexible regulatory environment 
• Cement the UK as a leading destination for international trials
• Move away from a one-size-fits all approach, to be responsive to innovation
• Ensure patients and their safety are at the focus of all clinical trials and supported by greater transparency bring the benefits of clinical trials to everyone

Please find the statement made by Baroness Merron Parliamentary Under-Secretary of State for Patient Safety, Women's Health and Mental Health.

Medical devices regulatory reform roadmap to implementation

The MHRA published a revised roadmap towards the future regulatory framework for medical devices on 11^th December 2024. This provides a further update on the intended timelines to implement the future medical device regulations. New guidance on post-market surveillance is expected to be published in June 2025, guidance on pre-market requirements is expected in 2026, and an in vitro diagnostic roadmap is expected to be published by September 2025.

The British Pharmacopoeia (BP)

Draft guidance on replication competent virus assays

The BP has prepared draft best practice guidance on replication competent virus assays. This guidance is intended to ensure product quality is upheld throughout the product’s lifecycle. The deadline for comments is 28^th February 2025. The consultation document and response form can be downloaded here.

Consultation for Vector Copy Number guidance revision and addition of Polymerase Chain Reaction validation annexes

The BP has prepared an update to the guidance for Vector Copy Number, which includes a revision to the guidance itself as well as new Polymerase Chain Reaction validation plans. This update is intended to a range of stakeholders including those in GMP regulated environments, research and development, academia, and clinical trials. The deadline for consultation is 19^th March 2025. The consultation document and response form can be accessed here.

EUROPE

European Directorate for the Quality of Medicines (EDQM)

Collaborative study on validation of an ELISA method for determination of physical particle titre of AAV2 vectors: outcome published

The Gene Therapy Working Group (GTWG) of the General European Official Medicines Control Laboratory (OMCL) Network (GEON) organises collaborative studies to validate analytical methods for the quality control of gene therapy products.

A recent study focused on the validation of an ELISA method for the determination of the physical particle titre of AAV2-based vector preparations. The outcome of this study is now available online in Pharmeuropa Bio & Scientific Notes.

European Medicines Agency (EMA)

Successful pilot paves the way for implementation of electronic product information

The report of a pilot exploring the creation and testing of ePIs (electronic product information) in real regulatory procedures has been published. The pilot found that the EU regulatory system is generally prepared for the introduction of ePI and can move towards its phased implementation in regulatory procedures, however recognising the need for more development, including additional functionalities and integration with current IT systems. Work on this additional development will be carried out in 2025. The report then recommends a phased implementation beginning with voluntary adoption for centrally authorised products, progressively expanding to nationally authorised products based on Member States’ readiness and available resources. 

A common EU approach to data transparency in medicine regulation

EMA and the Heads of Medicines Agencies (HMA) have published a revised guidance on the identification of commercially confidential information (CCI) and personal data in marketing authorisation applications for human medicines. The update of the initial 2012 guidance reaffirms the commitment of regulatory authorities across the European Economic Area (EEA) to extensive transparency when disclosing information, both in response to access-to-documents requests and in the proactive publication of data once a medicine is authorised. Please find further information here.

Translating innovation into access for Advanced therapy medicinal products: third EU-Innovation network multi-stakeholder meeting

The video recording of the 15^th November 2024 multi-stakeholder meeting, organised by The EU-Innovation Network (EU-IN) and the Italian Medicines Agency (AIFA) to discuss how innovation in the development of Advanced Therapy Medicinal Products (ATMPs) can be translated into effective and safe therapies at the point of care in Europe is now available here.

Alofisel withdrawn from the EU market

The available data from a recent study (ADMIRE-CD II) have shown that the use of Alofisel, a medicine that treats complex anal fistulas (abnormal passages between the lower parts of the gut and the skin near the anus) in adults with Crohn’s disease, does not demonstrate a favourable benefit-risk ratio. As a result, the company that markets Alofisel decided to withdraw the medicine from the EU market stating that it was not possible for them to provide additional data on the effectiveness of Alofisel, as expected by the EMA. Please find further information here.

USA

Food and Drug Administration (FDA)

FDA approval: Ryoncil

FDA has approved Ryoncil (remestemcel-L-rknd), an allogeneic (donor) bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients 2 months of age and older. Steroid-refractory acute graft-versus-host disease is a serious and life-threatening condition that can occur as a complication of allogeneic hematopoietic (blood) stem cell transplantation (allo-HSCT). MSCs are a type of cell that can have various roles in the body and can differentiate into multiple other types of cells. These MSCs are isolated from the bone marrow of healthy adult human donors. Ryoncil is the first MSC therapy to treat steroid-refractory acute graft-versus-host disease. Please find further information here.

Accelerated approval – expedited program for serious conditions

FDA has issued a draft guidance to provide information on FDA’s policies and procedures for accelerated approval as well as threshold criteria for whether 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, 2023 (Public Law 117-328). The deadline for comments is 4^th February 2025.

RegenMedEd roundtable with FDA’s office of therapeutic products (OTP)

The FDA’s Center for Biologics Evaluation and Research (CBER), OTP will be hosting a webinar on 30^th January 2025 which is part of an educational series, RegenMedEd. This series aims to provide foundational information about regenerative medicine therapies, such as cell and gene therapy products, and explore opportunities for patients, care partners, and advocates to engage with FDA to help advance product development of OTP-regulated products. Leaders from across OTP will provide information about how the office is helping to advance cell and gene therapy product development through innovation, engagement, and collaboration. Further information on this event can be found here.

Protocol deviations for clinical investigations of drugs, biological products, and devices

FDA has issued a draft guidance which provides recommendations to assist sponsors, clinical investigators, and institutional review boards (IRBs) in defining, identifying, and reporting protocol deviations in clinical investigations. This guidance includes the following: 

• 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 
• Recommendations for IRBs in their evaluation of protocol deviations

Comments can be submitted until 28 February 2025.

Advanced Manufacturing Technologies Designation program

FDA has issued a final guidance for industry entitled “Advanced Manufacturing Technologies Designation Program.” This guidance provides recommendations to persons and organisations interested in participating in the program, which facilitates the development of drugs manufactured using Advanced Manufacturing Technologies (AMT) that has been designated as such under the program. Early adoption of AMTs by the pharmaceutical industry is encouraged by the FDA as it can improve the reliability and robustness of the manufacturing process by:

• enhancing product quality
• reducing drug development time
• increasing or maintaining the supply of drugs

Cell therapies and tissue-based products: a public workshop on generating scientific evidence to facilitate development

The FDA’s CBER OTP is hosting a virtual scientific public workshop on 25^th February 2025, titled “Cell Therapies and Tissue-based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development.” The purpose of the workshop is to identify and discuss the current state of the science, development, and regulation for cellular therapies and tissue-based products. Please find further information including how to register here.

INTERNATIONAL

International Council for Harmonisation (ICH)

ICH Q9(R1) IWG updated Q9(R1) Annex 1- Q8/Q9/Q10 Questions & Answers

ICH has published the updated Q9(R1) Annex 1- Q8/Q9/Q10 Questions & Answers (R5) related to ICH Q9(R1) Quality Risk Management (QRM). Please find further information here.

Public consultations

The British Pharmacopoeia (BP)

Medicines and Healthcare products Regulatory Agency (MHRA)

European Medicines Agency (EMA)

Food and Drug Administration (FDA)

International Council for Harmonisation (ICH)

Click here to download the PDF