Regulatory Round-up - February 2025

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Draft guidance on individualised mRNA cancer immunotherapies

The MHRA is seeking feedback on the new draft guideline on individualised mRNA cancer immunotherapies, a new type of cancer treatment being tested in clinical trials. This is intended to clarify and streamline pathways for bringing these therapies through to patients, without compromising on robust safety principles. The deadline for consultation is 31^st March 2025. Further details and the draft guideline can be found here.

Decision tree for navigating nanotechnology-based products for medical application

In response to the increased number of scientific advice queries on nanomedicines, the MHRA has published a decision tree for navigating nanotechnology-based products for medical application. The decision tree is to help support researchers and developers to understand how different guidelines from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and European regulatory agencies can be applied to support the development of nanomedicines. It is not considered as a substitute for a regulatory or scientific advice meeting with the MHRA when specific issues may also need to be considered for medicinal products or medical devices. Consideration of medical devices is also outside the scope of this decision tree.

UK-wide licensing for human medicines: supplementary guidance

The MHRA published a guidance on 31^st January 2025 to provide supplementary information on the licensing of medicines for human use in the UK following the agreement of the Windsor Framework. The supplementary information is intended to help understand the main changes affecting the licensing of medicines for human use in the UK, introduced by the Windsor Framework’s medicines arrangements which came into effect on 1^st January 2025.

UK patients enabled access to transformative new medicines in shortest time possible via new, integrated Innovative Licensing and Access Pathway

The new Innovative Licensing and Access Pathway (ILAP) has been launched by the MHRA, the Health Technology Appraisal Bodies the All Wales Therapeutics and Toxicology Centre (AWTTC), the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and the NHS. The aim of this new pathway is to support the rapid development of transformative medicines that can be introduced into the NHS to address unmet clinical needs for patients and healthcare professionals at the earliest opportunity, without compromising on standards of safety, quality, and effectiveness. It is the only example globally of an end-to-end access pathway, where a medicine developer can work collaboratively with the national health system, the regulator, and health technology assessment bodies from the early stages of clinical development. Full details of the refreshed UK-wide ILAP have been published to provide a clearer, more streamlined and integrated process for developers to help get transformative new medicines to patients in the NHS in the shortest time possible.

Health Research Authority (HRA)

General Data Protection Regulation: transparency wording for all sponsors

To help ensure research participants have all the information they need to make an informed decision about the use of their data the HRA developed a general data protection regulation (GDPR) template that is recommend for sponsors to use. There is a transition period until 31^st March 2025 during which previous wording or the sponsors own bespoke wording can be used if it has been approved by the HRA. From 1^st April 2025, any new research applications submitted via IRAS, sponsors will be expected to either:

• use the new GDPR transparency wording template for all sponsors
• demonstrate how the sponsor’s own bespoke wording meets the four principles for meaningful involvement of patients and the public in health and social care research

Please find further information including the new GDPR transparency wording template here.

A National Institute for Health and Care Excellence (NICE)

Casgevy: NICE approval for severe sickle cell disease

NICE has approved the CRISPR gene editing therapy, exagamglogene autotemcel (exa-cel, brand name Casgevy) for use in the NHS in England, providing a potential cure for some people with severe sickle cell disease (SCD). It will be made available under a managed access scheme for people 12 years and over with certain types of severe SCD. SCD is the name for a group of inherited health conditions that affect the red blood cells and is particularly common in people with an African or Caribbean family background. Prior to the Casgevy approval, the only curative treatment for people with SCD had been a donor stem cell transplant. Casgevy will now be an option when a stem cell transplant is suitable, but no matched donor can be found. Please find further information here.

EUROPE

European Commission (EC)

Commission launches new Biotech and Biomanufacturing Hub to support innovative companies

The EC has launched a new Biotech and Biomanufacturing Hub to support companies – particularly start-ups and SMEs - in bringing innovative products to the EU market and increase their competitiveness. 

The EU Bio Hub, hosted on the Commission's Your Europe portal, will serve as an operational tool for easy and accessible information on the relevant EU legislation, financing opportunities and business support networks, such as the Enterprise Europe Network and European Cluster Collaboration Platform.

Please find further information here.

European Medicines Agency (EMA)

Overview of the current regulatory testing requirements for medicinal products for human use and opportunities for implementation of the 3Rs - Scientific guideline

The EMA has published a draft reflection paper which provides an overview of the main tests that involve the use of animals that are required for the regulatory testing of human medicines. It also includes new approaches for further improvement in the application of 3Rs. The deadline for comments is 30 June 2025.

Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials

The guideline titled ‘Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials’ has been adopted and will be legally effective on 1^st July 2025. The guideline provides information on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with investigational advanced therapy medicinal products (ATMPs) and a perspective towards MAA. The guideline addresses development, manufacturing and quality control as well as non-clinical and some clinical aspects of investigational ATMPs. 

EMA proposes new guideline on model-informed drug development

Mechanistic models, i.e. mathematical or computer models that integrate biopharmaceutical, physico- mechanical, (patho)physiological and pharmacological processes, along with population characteristics, are increasingly used in all phases of the drug research and development life cycle. The EMA is planning to issue a draft guideline on the reporting and evaluation of new mechanistic models to encourage their broader use in model-informed drug development (MIDD), according to a concept paper published on 14th February. The current EMA guidance document (EMA/CHMP/458101/2016) which was adopted by the Committee for Medicinal Products for Human Use (CHMP) in 2018 only includes physiologically based pharmacokinetic (PBPK) modelling and simulation. To keep abreast with methodological developments in the field of MIDD, the scope of a new guideline on assessment and reporting of mechanistic models will also apply to other mechanistic models currently not covered by regulatory guidance documents, such as Physiologically Based Biopharmaceutics (PBBM) and Quantitative Systems Pharmacology (QSP) models.

Data protection notice regarding personal data processing in the Clinical Trials Information System 

The EMA has issued a data protection notice on 13^th February 2025, to provide information on personal data processing in the Clinical Trial Information System (CTIS). This data protection notice explains the most essential details of the processing of personal data in the context of the operation of the CTIS, including the EU Portal and the EU Database established in accordance with the requirements of Article 80 and 81, respectively, of the Regulation (EU) No 536/2014,2 hereinafter the Clinical Trials Regulation.

Swiss Agency for Therapeutic Products (Swissmedic)

Swissmedic updates position on decentralized clinical trials

The Swiss Agency for Therapeutic Products (Swissmedic) has updated its position paper on decentralized clinical trials (DCTs), including a comparison of what decentralized aspects of trial design are allowed in Switzerland and other European countries. Swissmedic published the second version of its DCT position paper with the Swiss Association of Research Ethics Committees, late in 2022. This document described DCTs, the legal framework in Switzerland and the acceptability of specific decentralized design elements such as recruitment through digital channels and remote data capture. In the latest paper, Swissmedic and Swissethics have added two new sections to the list of DCT elements, created three appendices, and made changes to the existing text. The two new sections cover trial oversight and responsibilities and organizational aspects for good clinical practice inspections. The updated position paper is available to be downloaded here.

INTERNATIONAL

African Union Development Agency (AUDA-NEPAD) 

Pharmaceutical Inspection Co-operation Scheme establishes cooperative agreement with African Union Development Agency

The Pharmaceutical Inspection Co-operation scheme (PIC/S) recently announced a new agreement with the African Union Development Agency – New Partnership for Africa’s Development (AUDA-NEPAD) to share information on good manufacturing practices (GMP) for pharmaceuticals and active pharmaceutical ingredients (APIs).

Key Areas of Focus in the Agreement:

• Development of Guidelines: Collaborative efforts to create and harmonize GMP guidelines that align with global best practices while addressing Africa’s unique needs.
• Capacity Building: Initiatives aimed at enhancing the technical expertise of regulatory authorities and pharmaceutical manufacturers across the continent.
• Work Sharing: Frameworks designed to improve efficiency in GMP inspections by reducing duplication and fostering regulatory convergence.

AUDA-NEPAD has been granted the status of associate member, which is the first step towards becoming a PIC/S Participating Authority. Please find further information here.

Singapore's Health Sciences Authority (HSA)

Singapore introduces online system for cell, tissue, and gene therapy product filings

Singapore’s HSA has launched an online system for handling cell, tissue, and gene therapy product licenses and certificates. Companies can now log in to SHARE (Singapore Health Product Access and Regulatory E-System), with Corppass to carry out certain regulatory activities. Companies can use the system to apply for a dealer’s license or to change or cancel an existing license. HSA stopped accepting submissions via the FormSG platform on 7^th February and activated SHARE on 10^th February. Please find notice issued by HSA here.

Public consultations

European Medicines Agency (EMA)

The British Pharmacopoeia (BP)

Medicines and Healthcare products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

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