Regulatory Round-up - April 2025

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Clinical Trials regulations signed into law

New regulations for running clinical trials in the UK have now been signed into law. The new regulations will take full effect from 10 April 2026, following a 12-month implementation period starting 11 April 2025. The regulations deliver the most significant update to UK clinical trials regulation in two decades – with the aim of strengthening patient safety, accelerating approvals from 250 to 150 days, enabling innovation and helping more people benefit from taking part in vital research.

The reforms will:

• Put patients and their safety are at the focus of all clinical trials and bring the benefits of clinical trials to everyone.
• Cut duplication and unnecessary delays, while maintaining robust oversight of the safety of trials.
• Create a proportionate and flexible regulatory environment, reducing bureaucracy for lower-risk trials.
• Cement the UK as a destination for international trials.
• Provide a framework that is streamlined, agile and responsive to innovation.

Please find further information here. 

National assessment procedure for medicines

MHRA has published a guidance on the new national assessment procedure for marketing authorisation (MA) applications. The new guidance is now in effect for MA applications received from 3^rd April 2025. It applies to both innovative and established medicines and sets out for the first time how decisions will be delivered on MA applications within the 210 days target to support timely access to medicines, while being transparent, predictable and reliable. 

A webinar will be held on Tuesday 6 May 2025 to provide an overview of the guidance and highlight the key process changes. Please find further information including how to register here.

First-ever MHRA analysis of UK clinical trial applications finds new opportunities to drive medical breakthroughs for patients

The report published on 9^th April 2025 in the British Journal of Clinical Pharmacology, offers the most detailed picture yet of the UK’s clinical trials landscape, setting a baseline to track progress and inform future funding, policy and regulation. This is the first-ever analysis of the UK clinical trial landscape by the MHRA and the University of Liverpool that reveals the UK is a global leader in clinical research – and sets out key opportunities to deliver even more life-changing treatments for patients. Please find further information here.

MHRA conditional approval: Aucatzyl 

On 25^th April 2025, the MHRA granted a conditional MA for obecabtagene autoleucel (Aucatzyl), a chimeric antigen receptor (CAR) T-cell therapy, to treat adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

B-cell precursor ALL is a type of blood cancer that affects the white blood cells, specifically the B-lymphocytes. In the condition, the bone marrow produces a large number of abnormal, immature B-lymphocytes, often known as blast cells which grow and divide quickly. 

Please find further information here.

EUROPE

European Commission (EC)

Stability testing of drug substances and drug products Q1

This International Council for Harmonisation (ICH) draft guideline outlines the stability data expectations for drug substances and drug products. It is applicable to marketed drug products, including those associated with registration and lifecycle/post-approval changes and, when applicable, master files. ICH Q1 is a consolidated revision that supersedes ICH Q1A-F and Q5C guidelines and provides additional guidance on principles relating to stability. The document has been endorsed by the Members of the ICH Assembly under Step 2 and is now released for public consultation.

European Directorate for the Quality of Medicines (EDQM)

The European Pharmacopoeia Commission adopts new general chapter for high-throughput detection of viral extraneaous agents 

The new general chapter, High-throughput sequencing for the detection of viral extraneous agents (2.6.41), recently adopted by the European Pharmacopoeia Commission (ECP), describes high-throughput sequencing methodologies used for the detection of viral extraneous agents in biological products, including vaccines, recombinant proteins, viral vectors used for gene therapy and cell-based preparations for cell therapy. Please find further information here.

The European Pharmacopoeia Commission adopts a new general chapter for cell-based preparations for human use

The new general chapter, Cell-based preparations for human use (5.32), provides a comprehensive framework for the production and quality control of cell-based preparations, while offering the flexibility required to address the rapidly evolving nature of the field. While not legally binding, the chapter provides an extensive set of general requirements common to all cell-based preparations, together with four detailed sections outlining specific requirements for human haematopoietic stem cells, human chondrocytes, human limbal stem cells, and human mesenchymal stromal cells. Please find further information here.

European Medicines Agency (EMA)

Clinical Trials Information System designated as World Health Organisation (WHO) primary registry

The Clinical Trials Information System (CTIS) has been designated as a primary registry by the WHO within the International Clinical Trials Registry Platform. As a registry, CTIS adheres to specific criteria for content, data quality and validity, accessibility, unique identification, technical capacity, and administration. This ensures comprehensive research information is accessible to healthcare decision-makers globally. The list of current registries that meet the requirements of the ICMJE can be found here. Please find further information here.

Guidance on good manufacturing practice and good distribution practice: Questions and answers

Please find the update regarding 'EU GMP guide part II: Basic requirements for active substances used as starting materials: GMP compliance for active substances' section here.

Highlight report from the 13^th Industry stakeholder platform on research and development support

The thirteenth meeting between regulators and representatives of industry stakeholders took place to address topics of evidence generation along the medicine’s life cycle and related product-development support activities, such as scientific advice and qualification, as well as specifics for paediatric and orphan medicines. The aim of the platform is to provide an opportunity to foster a constructive dialogue with industry stakeholders. Please find the highlight report here.

EMA roundtable with stakeholders on the 20^th anniversary of the small and medium-sized enterprises (SME) Regulation

Representatives from the European Commission and the European Medicines Regulatory Network join EMA in presenting the achievements of the SME Regulation and perspectives on EU support to SMEs in the pharmaceutical sector in online roundtable meeting with stakeholders on 17^th October 2025 to mark the 20^th anniversary of EMA’s SME regulation.  

The SME regulation was adopted in December 2005 to promote innovation and development of new medicines by SMEs. Its primary objective is to provide financial and administrative assistance to these companies through the EMA’s SME Office.The video recording will be available after the event. Please find further details here.

Anonymisation of personal data and assessment of commercially confidential information during the preparation and redaction of risk management plans (body and annexes 4 and 6) 

EMA has issued a general guidance to provide information to applicants/marketing authorisation holders on the retention/transformation of personal data and identification of commercially confidential information when preparing risk management plans (RMPs) in the pre-approval process, and for the redaction of the RMPs for publication post-approval. 

Quality Review of Documents (QRD) annotated template v11

EMA has published a public consultation on the revision of the QRD template on 11^th April 2025. The deadline for comments is 31^st August 2025.

 The main changes in the QRD template intend to tackle the following aspects:

• The recurrent issue of the length of the PL, hence the deletion of the introductory bullets and the optionality of the table of contents.
• The creation of new standard statements aimed at improving patient-friendliness as well as consistency across products.
• The relocation of some information that is deemed important enough to appear at the beginning of the leaflet.
• The grouping of information by subject, as patients tend to look for information related to the same topic in the same place (e.g. interactions with food and drink merged with instructions on taking the medicine with food and drink).
• The reorganisation of warnings and precautions throughout the PL so that they follow the logics of what is important before, while, and after taking the medicine.
• The inclusion of a new optional section 7 for instructions for use when these are too extensive to be accommodated in section 3 or if they are related to a medical device accompanying the medicine.

Please find version 11 of the QRD annotated template here. 

Public consultations

International Conference on Harmonisation (ICH)

European Medicines Agency (EMA)

Click here to download the PDF