Latest Regulatory Updates

October 2023

20 years of European and American collaboration on medicines regulation

The European Commission, EMA and FDA signed confidentiality agreements in 2003. The last 20 years of collaboration enabled the Agencies to streamline efforts, share best practices and avoid duplication across the whole lifecycle of medicinal products to promote human health.

Important milestones achieved are highlighted here.

Horizon Europe

UK scientists can once more participate in Horizon Europe, the world’s largest research funding scheme. Horizon will give UK companies and research institutions unrivalled opportunities to lead global work to develop new technologies and research projects, in areas from health to artificial intelligence (AI).

See press release; Professor Alan Boyd responds to the announcement.

Food and Drug Administration (FDA)

New pilot programme for rare disease therapies

FDA launched a pilot programme to accelerate the development of novel drug and biological products for rare diseases. This will be open to a limited number of sponsors of products currently in clinical trials under an active Investigational New Drug application (IND), regulated by the Center for Biologics Evaluation and Research (CBER) and/or the Center for Drug Evaluation and Research (CDER).

Final guidance released – Real World Data:

Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products

This guidance discusses the applicability of FDA’s investigational new drug application (IND) regulations to various clinical study designs that utilize RWD and clarifies the Agency’s expectations regarding the effectiveness and safety of a drug when such studies are not subject to part 312, 21 CFR. The focus is primarily on non-interventional clinical study designs.

Final guidance released - Devices:

Use of International Standard ISO 10993-1, "Biological evaluation of medical devices - Part 1: Evaluation and testing within a risk management process" The new attachment included in this guidance (Attachment G) provides recommendations about the type of biocompatibility information that should be provided in a medical device premarket submission for certain devices in contact with intact skin.

Final guidance released - Devices:

Cybersecurity in Medical Devices: Quality System Considerations and Content of Premarket Submissions This advice applies to devices with cybersecurity considerations, including those with a software function or programmable logic. Recommendations include device design, labelling, and what information to include in premarket submissions.

Final guidance released – Compassionate Use:

Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products This guidance provides recommendations to clinical investigators and institutional review boards (IRBs) regarding the key factors and procedures to consider when reviewing individual patient expanded access submissions. It provides further clarity for IRBs and relates to previous draft guidance released on expanded access for individual patients (November 2022).

Draft guidance released for comment – FDA meetings for PDUFA products:

Formal Meetings Between the FDA and Sponsors or Applicants of Prescription Drug User Fee Act (PDUFA) Products This guidance discusses the principles of good meeting management practices and describes standardised procedures for requesting, preparing, scheduling, conducting, and documenting such formal meetings.

Submit comments by 21 December 2023.

European Medicines Agency (EMA)

Questions and answers for biological medicinal products

This Q&A page is maintained by the CHMP Biologics Working Party providing agreed positions on issues that can be subject to different interpretation or require clarification.

MA guideline template update

The 4^th revised guideline of the electronic application form for a Marketing Authorisation (MA) is available here (version September 2023).

Update to questions and answers (Q&As) guidance on pre-authorisation procedural advice for users of the centralised procedure

The update concerns advice on acceptable combination packs and the Risk Management Plan (RMP) assessed in initial marketing authorization procedures.

Launch of ACT EU Website

A dedicated website was launched on the Accelerating Clinical Trials in the European Union (ACT EU) initiative, containing key resources, latest news and upcoming events, including updates on the Multi-stakeholder platform, the implementation of the regulation, and information on scientific advice procedures such as the Simultaneous national scientific advice (SNSA) pilot.

Transitioning multi-national clinical trials to the Clinical Trials Regulation / CTIS

Revised versions of the Best Practice Guide and cover letter template are available. A major change is that a consolidated Investigator’s Brochure/Product Dossier may now be submitted for transitional trials. The Clinical Trials Coordination Group (CTCG) also agrees on an expedited, harmonised Member State evaluation procedure focusing on the validation of minimum application dossiers restricted to documents already authorised under the Clinical Trials Directive.

Newsletter:  Human medicines highlights – Issue 173, September 2023

Primarily addressed to organisations representing patients, consumers and healthcare professionals, this newsletter provides a summary of key information relating to medicines for human use.

Good Clinical Practice inspection procedures

The Guideline on computerised systems and electronic data in clinical trials, adopted in March, has now entered into effect. Describing generally applicable principles and key concepts the guideline also focuses on validation, user management, security, and the electronic data life cycle for computerised systems.

Medicines and Healthcare products Regulatory Agency (MHRA)

New Innovative Devices Access Pathway (IDAP) pilot 

Guidance is available for this new integrated and enhanced regulatory and access pathway supporting innovative technologies and solutions to the UK National Health Service (NHS) to help with unmet medical needs. IDAP is available to UK and international commercial and non-commercial developers with new health technology solutions. The pilot scheme will test the main elements of the pathway whist providing informative learning and feedback for future IDAP.

The scheme was launched at the end of September and the submission deadline is 29 October 2023.

New international regulatory recognition routes for medicines approvals

A new scheme will replace the current European Commission Decision Reliance Procedure (ECDRP) on 01 January 2024. The guidance informs how the new international recognition framework can be used to apply for a medicine licence in the UK, following approval by trusted regulatory partners in Australia, Canada, the European Union, Japan, Switzerland, Singapore, and the United States.

Licensing and applications

A comprehensive new MHRA webpage is available compiling information about new applications, post-licensing, advertising, product information and fees in one place.

Good practice

MHRA published also a collection of guidance on good laboratory, manufacturing and distribution practice, good pharmacovigilance practice, out-of-specification investigations, inspection metrics and fees.

Changes to National Contract Value Review (NCVR) affecting commercial sponsors

As of 01 October 2023, the new, unmodifiable financial appendix is now mandated for use in commercial contracts, thereby removing local contract-value negotiation for commercial research.

Podcast: Conversations in Drug Development, brought to you by the team at Boyds

We are very excited to announce the launch of our new podcast "Conversations in Drug Development", brought to you by the team at Boyds. This podcast is for our fellow community of scientists and clinicians working in the wonderful world of cell and gene therapy and drug development.

As loyal readers of our Regulatory Intelligence Digest, we invite you to be the first to listen to our first episode, "Recent Trends in Oncology Drug Development" hosted by Dr Julie Warner who is joined by oncology expert Dr Katherine Bowen. Click here to find out how to listen on your preferred podcast platform and don't forget to subscribe for more episodes.