Regulatory Round-up - October 2024

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Business Plan 2024/25

On 7^th October 2024 the MHRA published its new business plan for 2024/25, setting out the agency’s core focus of enabling access to innovative healthcare products while ensuring delivery for all who need its services – patients and the public, healthcare professionals, researchers and developers. 

Statutory Instrument laid in Parliament provides first regulatory framework of its kind for the manufacture of innovative medicines at the point of patient care

A draft Statutory instrument was laid in Parliament on 21^st  October. The legislation aims to transform the manufacture of innovative medicines at the point of care. The UK will be the first country globally to introduce a tailored framework for the regulation of innovative products manufactured at or close to the location where a patient receives care. This new legislation will:

• Allow medicines with a very short shelf life and highly personalised medicines to be manufactured in or near a hospital setting so that patients can access them more effectively 
• Allow manufacture of innovative medicines in small, portable units that can be set up close to patients who may be unable to travel or where rapid medicine availability is best served.
• Help relieve pressures on hospitals by enabling delivery of care where it is most appropriate for the patient, 
• Enable the safe development of innovative, personalised products such as cell or gene therapy that need to be manufactured close to the individual patient. 

The legislation proposes a six-month implementation period once Parliamentary processes have been concluded. This regulation could, therefore, become law in Summer 2025.

Statutory Instrument laid in Parliament sets out first steps in delivering Medical Device Regulatory Reform and strengthening patient safety

The Post-market Surveillance (PMS) Statutory instrument laid in Parliament on 21^st October 2024 is the first major update to the framework of medical device regulations in Great Britain. Please find the draft statutory instrument, explanatory memorandum and impact assessment here. A summary of the key changes to the regulation is available here. 

Health Research Authority (HRA)

HRA and MHRA draft inclusion and diversity guidance

The Health Research Authority (HRA) have launched an informal consultation on a draft guidance being produced with the MHRA which aims to help increase the inclusion and diversity of people taking part in clinical trials and clinical investigations. The deadline for comments is 9^th December 2024. 

National Health Service (NHS) England

National contract value review; advanced therapeutic medical products (ATMPs) will be incorporated

From October 2024, as part of work to accelerate commercial research setup in the UK, the National Contract Value Review (NCVR) will incorporate clinical trials of ATMPs. The NCVR is a standardised, national approach to costing for commercial contract research. To support this change, a new model clinical trial agreement for ATMPs and a new clinical research organisation (CRO) model clinical trial agreement for ATMPs are introduced. The new templates have been developed by the Four Nations Contracts Leads, which consists of representatives from the HRA and the Devolved Administrations of Northern Ireland, Scotland and Wales. With this, ATMP clinical trials now have new pre-agreed template agreements, which should be used without modification for all phases of ATMP clinical trials.

EUROPE

European Commission (EC)

Implementation of the Hospital Exemption in the EU and its role in boosting innovation and patient access to innovative therapies

An online event will be held by the European Health and Digital Executive Agency (HaDEA) on 21^st November 2024 to address the knowledge gaps on the implementation and functioning of the Hospital Exemption in EU Member States. This event provides an opportunity to bring together experts and representatives from academia, the biotech industry, professional organisations and national authorities to explore the impact of the Hospital Exemption pathway on patient access to ATMPs and to enable conversations that matter. Please find further information including how to register here.

European Directorate for the Quality of Medicines (EDQM)

Webinar – Advancements in gene therapy: the European Pharmacopoeia’s new approach

In response to the recent approval of many new gene therapy medicinal products (GTMPs), the European Pharmacopoeia Commission (EPC) has defined a new approach to these innovative medicinal products. This webinar will provide guidance on the two new Ph. Eur. texts that form the basis of this approach: the general chapter 5.34, ‘Additional information on gene therapy medicinal products for human use’, and the general monograph 3186, ‘Gene therapy medicinal products for human use’, which, in combination, replace and supplement the previously published general chapter 5.14, ‘Gene transfer medicinal products for human use’. The webinar will explain the links between them and other Ph. Eur. texts, highlighting the key changes versus general chapter 5.14, and explain the flexible framework of requirements necessary for these rapidly evolving products. The webinar is scheduled for 3 December 2024, 14-16:00 (CET). Please find further information including how to register and send questions in advance here.

European Medicines Agency (EMA)

EMA conditional MA approval: Beqvez

EMA has granted a conditional marketing authorisation for Beqvez (previously Durveqtix) to treat adults with severe or moderately severe haemophilia B. Beqvez is an adeno-associated virus (AAV)-based gene therapy designed to introduce a functional copy of the factor IX (FIX) gene encoding a high-activity FIX variant to the target cells. It is intended for use in adults without a history of FIX inhibitors who do not have antibodies against AAV. 

Improving efficiency of approval process for new medicines in the EU

EMA and the European medicines regulatory network are working to further improve efficiency in the assessment and approval processes for new medicines in the EU. The initiative aims to better manage the use of the network's expert resources, streamline assessment processes and encourage better and more comprehensive application dossiers from applicants at the time of initial submission. Please find further information here.

Q&A Clinic on Product Management Service (PMS), Product User Interface(UI), and Application Programming Interface (API)

EMA will hold a Q&A session on 22^nd November 2024 which will cover this year's release of read-only data for centrally authorised products (CAPs) and non-centrally authorised products (non-CAPs), available through the Product Management Service (PMS) via both the PUI and the API. 

The first iteration of the PMS covers a subset of the authorised medicinal product part of the International Organisation for Standardisation (ISO) standards for the identification of medicinal products (IDMP). As part of this iteration, the new ISO IDMP compatible data submission format (HL7 FHIR) replaces the current data submission format, the eXtended EudraVigilance Product Report Message (XEVPRM). Further information can be found here. 

USA

Food and Drug Administration (FDA)

Revision of USP Chapter <1047> on Gene Therapy Medicinal Products

This chapter covers the clinical applications, manufacturing processes and analytical methods of gene therapy products, which are constantly evolving. 

The current amendment proposals include, for example, the following points: 

• Delete the reference to "Safety Tests-Biologicals" in "Biological Reactivity Tests, in vivo" from the chapter
• Delete the Safety Tests-Biologicals section from Chapter <88>, as the corresponding FDA Code of Federal Regulations, 21 CFR §610.11, was revoked on August 3, 2015.
• Some editorial changes.

Please find further information here.

Workshop on Integration Site Analysis During Long Term Follow-Up for Gene Therapies with Integrating Viral Vectors

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a virtual scientific public workshop on the clinical use of integration site analysis (ISA) during long term follow-up following administration of gene therapies with integrating viral vectors. This workshop is intended for research scientists with experience in vector design and ISA, as well as clinician-scientists with expertise in clinical interpretation of ISA data. The webinar is on 14^th November 2024, Please find further information including registration details here. 

OTP Town Hall: Best Practices for Regulatory Interactions with OTP

The FDA’s CBER OTP is hosting its next virtual town hall on 12^th December 2024. Experts from OTP’s Office of Review Management & Regulatory Review (ORMRR) will answer questions regarding best practices for interacting with OTP. The aim is to provide answers to questions from developers of advanced therapies, regarding best practices throughout the product development lifecycle. Please find further information including how to register and submit questions prior to the event here.

Drug Interaction Information in Human Prescription Drug and Biological Product Labeling—Content and Format; Draft Guidance for Industry

This draft guidance provides recommendations to applicants of human prescription drug and biological products on appropriate placement and content of drug interaction (DI) information in labeling as described in the regulations for the content and format of labeling for human prescription drug and biological products. Comments can be submitted until 22^nd January 2025.

Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases

CBER will host a public patient listening meeting to better understand patient and carer perspectives on enrollment of rare disease patients into gene therapy clinical trials in the pre-symptomatic or early symptomatic stages of their disease. The aim of this meeting is to explore what patients, and caregivers of patients in the pre-symptomatic or early stages of disease take into consideration when deciding whether to enroll in a gene therapy clinical trial and potentially receive an investigational gene therapy product. The meeting is on 4^th December 2024, Please find further information including how to register here.

Public consultations

European Medicines Agency (EMA)

Food and Drug Administration (FDA)

Click here to download the PDF