Regulatory Round-up - September 2024

UNITED KINGDOM

National Institute for Health and Care Excellence (NICE)

Exagamglogene autotemcel for treating transfusion-dependent beta-thalassaemia in people 12 years and over

NICE is recommending exagamglogene autotemcel (Casgevy) with managed access as an option for treating transfusion-dependent beta-thalassaemia in people 12 years and over:

• when a haematopoietic stem cell transplant (HSCT) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available
• only if the conditions in the managed access agreement for exagamglogene autotemcel are followed.

Please find further information here.

Medicines and Healthcare products Regulatory Agency (MHRA)

Medicines Pipeline data

The MHRA is requesting that current or potential medicines Marketing Authorisation Holders (MAH) provide information on planned or potential future submissions to the MHRA. Knowledge of expected submissions of all types, and requests for advice is key for the MHRA to effectively deliver their services to the highest quality and to be predictable with their assessment and advice timelines. In addition, it has benefits for industry timeframes, patient access and system colleagues. Please find further information here.

Implementing the new UK Clinical Trials regulations

On 15^th October 2024, the MHRA in partnership with the Health Research Authority (HRA), is hosting an online webinar to introduce their joint plans to share draft guidance with stakeholders and to set out the anticipated timelines for implementing the new UK Clinical Trials regulations.

The agenda will include:

• Updates on the legislative timeframes
• High-level details of the plan for developing and sharing draft guidance
• Introducing the ‘Implementing the new Clinical Trials regulations’ roadmap.

Please find further information including how to register here.

Application route for performance studies under the In Vitro Diagnostics Regulations in Northern Ireland

The first submission and approval of the application for In Vitro Diagnostic devices under the EU In Vitro Diagnostic Medical Devices Regulation (IVDR) in Northern Ireland has been completed due to recent implementation of a submission route for manufacturers for performance studies. This process was developed over the past eighteen months in collaboration with colleagues at Health and Social Care Northern Ireland (HSCNI) and Health Research Authority (HRA). Please find further information here.

MHRA opens applications from AI developers to join the AI Airlock regulatory sandbox

The MHRA, is calling for applications for manufacturers and developers of Artificial Intelligence (AI) medical devices, to join the AI Airlock regulatory sandbox. This pilot project will help the MHRA identify and address the challenges for regulating AI medical devices so that in the future, innovative AI medical devices can be brought into use safely in the shortest time possible.

Applications are open for submission for two weeks until Monday, 7 October, and candidates will be provisionally recruited into the pilot covering a wide range of regulatory challenges, from different healthcare or clinical disciplines and at various stages of product and regulatory development. Please find further information here.

Health Research Authority (HRA)

HRA - Tell us about your older studies

HRA is checking whether older studies in Integrated Research Application System (IRAS) are still active and need sponsors’ help. Over the next six weeks the HRA will be getting in touch with sponsors to find out whether studies from before 2018 have been completed. Please find further information here.

EUROPE

European Directorate for the Quality of Medicines (EDQM)

EDQM Webinar; Everything you've always wanted to know about the certification of suitability (CEP) procedure

The EDQM invites CEP holders and users to participate in a webinar to facilitate the understanding of the Certification Procedure on 21 November 2024. Please find further information including how to register here.

European Medicines Agency (EMA)

Harnessing AI in medicines regulation: use of large language models (LLMs)

EMA and the Heads of Medicines Agencies (HMA) have published high-level principles and recommendations for all staff across the European medicines regulatory network (EMRN) using large language models (LLMs) in their work. The aim of the guiding principles is to build understanding of the capabilities and limitations of these applications among staff at regulatory agencies across the EU so that they can harness the potential of LLMs effectively and avoid pitfalls and risks.

Reflection paper on establishing efficacy based on single arm trials submitted as pivotal evidence in a marketing authorisation application

This reflection paper, adopted by the CHMP on 9^th September 2024 outlines perspectives on single arm trials (SATs) that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications. It aims to identify and critically reflect on specific and important features of SATs.

Translating innovation to access for ATMPs: 3rd EU-Innovation network multi-stakeholder meeting

The EU-Innovation Network (EU-IN) and the Italian Medicines Agency (AIFA) are organising a multi-stakeholder online meeting on 15^th November 2024, 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.

The objectives of this meeting are:

• To seek feedback from developers on the main challenges and barriers currently encountered from early development to access,
• To provide insights on scientific and regulatory advice tools at European and national level available for developers,
• To discuss opportunities for promoting mutual exchange and interactions between stakeholders with the aim of strengthening the European Innovation Ecosystem.

Please find further information including how to register here.

USA

Food and Drug Administration (FDA)

Convergence: FDA officials offer updates on START and STAR pilots

FDA has shared an update on the status and progress of two pilot programs that aim to promote the development of innovative medicines at RAPS Convergence 2024.The two pilots are the Support for clinical Trials Advancing Rare disease Therapeutics (START) program and the Split Real Time Application Review (STAR). The aim of these pilots are to accelerate the regulatory review phase. Please find further details here.

Providing regulatory submissions in electronic format — Certain human pharmaceutical product applications and related submissions using the eCTD specifications

The FDA has issued a final guidance which describes how sponsors and applicants must organise the content that they submit to the agency electronically for all submission types under section 745A(a) of the FD&C Act. This guidance also references several technical specification documents and the electronic common technical document (eCTD) Technical Conformance Guide, which provides additional details regarding the organization of content for electronic submissions.

Incorporating voluntary patient preference information over the total product life cycle

The FDA has issued a draft guidance to propose revisions to the 2016 guidance to reflect the current scope of FDA’s benefit-risk paradigm, which may under appropriate circumstances, include patient preference information (PPI), and to provide additional considerations and practical recommendations based on additional experience evaluating patient preferences regarding devices. This draft guidance also fulfils a commitment in Section V.E. of the Medical Device User Fee Amendments Performance Goals and Procedures, Fiscal Years 2023 Through 2027 (MDUFA V). This guidance, when finalized, is intended to provide updated recommendations to industry and FDA staff for designing, collecting, and evaluating PPI in the context of benefit-risk assessments of devices. This includes practical recommendations intended to address common questions for those interested in the voluntary inclusion of PPI for FDA consideration. This guidance is applicable to voluntary PPI for consideration by FDA staff in decision making relating to devices. Voluntary PPI can, if it meets applicable legal standards, be considered by FDA during all stages of the total product life cycle.

Comments can be submitted until 6^th December 2024.

Control of nitrosamine impurities in human drugs

The FDA has issued a final guidance for industry entitled “Control of Nitrosamine Impurities in Human Drugs.” This guidance recommends steps manufacturers of active pharmaceutical ingredients (APIs) and drug products should take to detect and prevent unacceptable levels of nitrosamine impurities in pharmaceutical products.

Integrating randomized controlled trials for drug and biological products Into routine clinical practice

As part of FDA’s Real-World Evidence (RWE) Program, this draft guidance is intended to support the conduct of randomized controlled drug trials (RCTs) with streamlined protocols and procedures that focus on essential data collection, allowing integration of research into routine clinical practice. Such trials have sometimes been referred to as point of care trials or large simple trials.

Comments can be submitted until 17^th December 2024.

Workshop on integration site analysis during long term follow-up for gene therapies with integrating viral vectors

On 14^th November 2024, 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. In this workshop a panel of external experts will discuss the risk of insertional mutagenesis and best practices for ISA method design, data analysis, and clinical interpretation. Please find further information including how to register here.

Public consultations

European Medicines Agency (EMA)

Food and Drug Administration (FDA)

New Zealand Medicines and Medical Devices Safety Authority (Medsafe)

Click here to download the PDF.