Regulatory Intelligence - Year Round Up
An overview of the key regulatory intelligence updates from FDA, EC, EMA & MHRA released in 2024.
Authored by:
Nohman Mohammad, Associate Consultant
Basia Grodyńska, Associate Consultant
Mauricio Cano Mena, Associate Consultant
Dr Hamish Poli, Associate Consultant
Savannah Orth, Associate Consultant Intern
Dr Tulasi Yadati, Associate Consultant
Dr Demi Wiskerke, Associate Consultant
Brenna Devlin, Associate Consultant
European Medicines Agency (EMA)
Clinical Trials’ Transition to New EU System
Under the Clinical Trials Regulation (CTR), European Union (EU) Member States, and European Economic Area (EEA) countries use the Clinical Trials Information System (CTIS) to carry out their legal responsibilities to assess and oversee clinical trials from 31st January 2022. From 31st January 2025, any trials approved under the Clinical Trials Directive that continue running will need to comply with the Clinical Trials Regulation and their sponsors must have recorded information on them in CTIS. The revised CTIS transparency rules became applicable on 18th June 2024 with the launch of a new version of the CTIS public website and the CTIS secure workspaces. A quick guide for users of the revised CTIS transparency rules can be accessed via this link.
EMA Publishes New Fee Regulation Working Arrangements
The European Medicines Agency (EMA) has released a document on fees and charges payable to the EMA applicable from 1st January 2025. The document aims to clarify the new regulation’s requirements and terminology, implement fee reductions for specific services, and detail payment methods. Adopted by EMA’s management Board in June 2024, these documents are part of the agency’s broader efforts to prepare for the new regulatory framework.
Two New Advice Pilots to Improve Clinical Trials in Europe
The EMA has released two advice pilots aimed at improving the quality of clinical trial applications. The first pilot provides medicinal product developers with scientific advice on clinical trials and requirements for marketing authorisation applications (MAA). The second pilot, coordinated by the Clinical Trials Coordination Group (CTCG), offers technical and regulatory support on the dossier of a clinical trial application (CTA) prior to its submission through the Clinical Trials Information System (CTIS). Developers seeking advice on MAA or CTA requirements can apply to these pilot programs.
Guidance for Applicants on Simultaneous National Scientific Advice (SNSA) – Phase 2 Pilot
The EU Innovation Network (EU IN) has launched the second phase of the Simultaneous National Scientific Advice (SNSA) pilot, running until the end of 2024, to streamline the process of obtaining national scientific advice from multiple National Competent Authorities (NCAs) simultaneously. This phase focuses on facilitating clinical trials within Europe under the Accelerating Clinical Trials in the EU (ACT-EU) initiative, offering improved procedures such as a unified application process, predictable timelines, and expanded NCA participation to reduce administrative burdens. Applicants, including pharmaceutical companies, small and medium enterprises (SMEs), and academic researchers, can seek advice on topics such as clinical trials spanning multiple Member States, early-stage development of innovative products, and repurposing authorised medicines. The process promotes collaboration among NCAs to provide joint advice, ensuring alignment while addressing any divergences transparently, and supports the integration of national and centralised European advice systems.
EMA Revises Draft Guideline on Quality, Non-clinical and Clinical Requirements for Investigational Advanced Therapy Medicinal Products in Clinical Trials
The EMA revises draft guideline on advanced therapy medicinal products (ATMPs) in clinical trials. The guideline provides guidance on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with investigational ATMPs. The guideline is multidisciplinary and addresses development, manufacturing and quality control as well as non-clinical and clinical development of investigational ATMPs. Requirements for exploratory trials (including First in Human studies) and confirmatory trials are described throughout the guideline, and a perspective towards Marketing Authorization Application (MAA) is provided.
European Commission (EC)
Regulation on Standards of Quality and Safety for Substances of Human Origin Intended for Human Application Published on the Official Journal of the EU
The European Commission (EC) has published the Regulation (EU) 2024/1938 of the European Parliament and of the Council of 13 June 2024, on the standards of quality and safety for substances of human origin (SoHOs) intended for human application and repealing Directives 2002/98/EC and 2004/23/EC. The regulation covers all SoHOs, including blood, tissues, cells, human breast milk, and intestinal microbiota. Additionally, it applies to activities from donor registration to human application.
EC Adopts the European Health Data Space and Regulation on Substances of Human Origin
The EC has welcomed the adoption by European Parliament of the European Health Data Space (EDHS) and new rules to increase the safety and quality of substances of human origin (SoHO). New EU rules ensure citizens across the EU have easy access to their digital health data regardless of location, benefiting healthcare coordination and patient care. Additionally, comprehensive regulations covering substances of human origin, except solid organs, are introduced to enhance safety, quality, and oversight, fostering innovation and ensuring supply continuity through a coordinated EU platform. Further information on the adoption of EHDS and SoHO can be found in the Q&A on the European Health Data Space and Factsheet EHDS.
Food and Drug Administration (FDA)
FDA Publishes Final Guidance on Drug Interaction Information in Human Prescription Drug and Biological Product Labeling
This guidance is intended to assist applicants of human prescription drug and biological products in determining the 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. The purpose of this guidance is to provide recommendations to help ensure that appropriate DI information is consistently placed in the proper sections and subsections within labeling so that the information is clear and accessible to health care practitioners (HCPs) and includes content that guides the safe and effective use of the drug.
FDA Releases Final Guidance on Conducting Clinical Trials With Decentralized Elements Guidance for Industry, Investigators, and Other Interested Parties
The FDA issued a final guidance on decentralized clinical trials (DCTs) addressing some key issues from the draft guidance raised by clinical experts and industry groups, including providing more information on requirements to keep a task log of healthcare providers (HCPs), physical inspection requirements, and clarifying challenges around data variability in DCTs.
FDA Issues Draft Guidance on Diversity Action Plans Required for Certain Clinical Studies
The FDA has issued a draft guidance to assist sponsors in conducting clinical studies involving drugs, biological products, and devices in compliance with the Diversity Action Plans required under sections 505(z) and 520(g)(9) of the Food Drug and Cosmetics (FD&C) Act, as amended by the Food and Drug Omnibus Reform Act of 2022 (FDORA). These plans aim to increase the enrolment of historically underrepresented populations in clinical studies, enhancing the generalizability of evidence for the intended use population. Sponsors must specify enrolment goals, provide rationales, and detail methods to achieve these goals.
FDA Launches Center for Clinical Trial Innovation
The Center for Drug Evaluation and Research (CDER) has established the CDER Center for Clinical Trial Innovation (C3TI) as a central hub for fostering innovation and coordinating communication internally and externally. Led by Kevin Bugin, PhD, C3TI aims to promote innovative approaches to clinical trial design and implementation, facilitating knowledge sharing and collaboration across industry and therapeutic areas. Through initiatives like the C3TI Demonstration Program, CDER seeks to integrate novel trial approaches into drug development, addressing the evolving landscape of clinical trials and improving regulatory decision-making for new medicines. This initiative stems from a recognition of the need to bridge the gap between policy and implementation, with a focus on providing support, training, and dissemination of lessons learned in clinical trial innovation.
FDA Finalizes Guidance on Developing, Manufacturing CAR T Therapies
The FDA has finalized guidance which provides chimeric antigen receptor (CAR) T cell-specific recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and design of clinical studies for oncology indications (including hematologic malignancies and solid tumors). Recommendations specific to autologous or allogeneic CAR T cell products are noted in this guidance. This guidance also provides recommendations for analytical comparability studies for CAR T cell products.
Medicines and Healthcare Products
Regulatory Agency (MHRA)
MHRA Updates Guidance on Innovative Licensing and Access Pathway
The MHRA has updated its page on Innovative Licensing and Access Pathway (ILAP). The current ILAP scheme stopped accepting applications as of 17:00 Greenwich Mean Time (GMT) on 20th November 2024. This will enable the ILAP partners and developers to transition to the new refreshed pathway which will open to applications in March 2025. Please see The Statement of Policy Intent: relaunch of the ILAP for further information.
MHRA Updates Guidance for Early Access to Medicines Scheme
The MHRA has updated its guidance for applicants wishing to apply for a Promising Innovative Medicines (PIM) designation, pre-submission meeting, or an Early Access to Medicines Scheme (EAMS) Scientific Opinion. The information provided, however, should be considered as a guide only, and the final content and structure of applying for EAMS should be considered at the pre-submission meeting (PSM).
MHRA Updates Guidance on Clinical Trials for Medicines
The MHRA has revised its guidance for clinical trials of medicines and the application process for authorisation in the United Kingdom (UK). The guidance outlines how to apply for a clinical trial, covering eligibility, phases, model Investigational Medical Product Dossier (IMPDs), costs, and how to make amendments to applications. The latest update includes a modification to the text on In Vitro Diagnostic Medical Devices (IVDs) on clinical trial/investigation webpages.
MHRA Updates Guidance for International Recognition Procedure
The MHRA has updated its guidance describing how to use the International Recognition Procedure (IRP) for medicines licensing applications. This has also included its electronic Common Technical Document (eCTD) guidance for marketing authorisation and post-authorisation applications which can be found in the link here. The IRP has replaced the European Commission’s Decision Reliance Procedure (ECDRP); submissions received before 1st January 2024 will be processed under the existing practices. The IRP will allow the MHRA to take into account the expertise and decision-making of trusted regulatory partners for the benefit of UK patients.
MHRA Updates Guidance on ATMP: Regulation and Licensing in Great Britain
The MHRA has released a new guidance for process changes for the assessment of established medicines. The changes will enable the MHRA to assess applications more efficiently, helping to ensure a smoother and more rapid approval process for applicants. This aligns with the agency’s broader role as the UK’s national competent authority for medicinal products, including ATMPs, which encompass gene therapies, somatic cell therapies, and tissue-engineered products.
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