Regulatory Intelligence Digest - February 2024

Latest Regulatory Updates

February 2024

Hello and welcome to the Boyds Regulatory Intelligence Digest. This month we are sharing the latest guidance and key updates from the regulatory agencies - FDA, EMA, MHRA, EC and Swissmedic.

To discuss any of these updates, please?contact a member of the Boyds Regulatory teamwho will be happy to help.

Food and Drug Administration (FDA)

Pharmaceutical Quality Documents

FDA launched a new search function relating to the Pharmaceutical Quality Resources. The new search webpage scans existing FDA guidance documents, manuals of policies and procedures (MAPPs), and compliance programs to provide users with relevant and up-to-date resources and information.

ClinicalTrials.gov Reporting

FDA announced a newly published report on the findings to understand the barriers to timely, accurate, and complete registration and reporting of clinical trial summary results information on ClinicalTrials.gov. The report provides strategies and recommendations for improving registration and results information reporting for applicable clinical trials.

Chimeric Antigen Receptor (CAR) T Cell Products

In November 2023, the FDA?posted?a safety communication with information related to findings of T cell malignancies reported in patients who received treatment with CAR T cell immunotherapies. In January 2024 this lead to a class wide change of safety labelling and black box warning for all six commercial CAR T therapies, and now the release of the final guidance on? Considerations for the Development of CAR T Cell Products, providing recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and clinical study design.

Final Guidance Issued

Human Gene Therapy Products Incorporating Human Genome Editing (GE) This guidance provides recommendations on what should be provided in an Investigational New Drug (IND) application in order to assess the safety and quality of investigational GE products, and includes information on product design, manufacturing and testing, nonclinical safety assessment, and clinical trial design.

Final Guidance Issued

Q5A(R2) Viral Safety Evaluation of Biotechnology Products Derived From Cell Lines of Human or Animal Origin, which describes risk-based principles and mitigation strategies to assure the viral safety of biotechnology products, including the data necessary to submit in a marketing application.

Draft guidance out for comment

The following draft guidance is available for comment: Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products. Providing FDA’s expectations for, and recommendations on, use of a standardized approach for collecting and reporting race and ethnicity data in submissions including information collected and reported from clinical trials and clinical studies for FDA-regulated medical products. Submit comments by 29 April 2024.

Revised Draft Guidance

FDA revised the draft guidance, Requests for Reconsideration at the Division Level Under GDUFA, provides recommendations on the procedures for applicants of abbreviated new drug applications (ANDAs) who wish to pursue a request for reconsideration within the review discipline at the division level or original signatory authority.

This revision reflects the most recent reauthorization of the Generic Drug User Fee Amendments (GDUFA) and clarifies what matters are appropriate for requests for reconsideration.

Draft Guidance Issued

The draft guidance Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act is intended to assist applicants and manufacturers in providing FDA timely, informative notifications about changes in the production of finished products that are life supporting, life sustaining, or intended for use in the prevention or treatment of a debilitating disease or condition. This applies to applicants with approved new drug applications (NDAs), approved abbreviated new drug applications (ANDAs), or approved biologics license applications (BLAs), plus manufacturers of finished drug products marketed without approved NDAs or ANDAs.

European Medicines Agency (EMA)

Revised SME User Guide

Following a major revision of its user guide for micro, small, and medium-sized enterprises (SMEs), EMA has released an updated version. Two new sections have been added on the Clinical Trials Regulation and on the Medical Devices Regulation. Other revised topics are IT systems, borderline products, environmental risk assessment (ERA), big data, information on EMA collaboration with non-EU regulators (OPEN initiative) plus guidance around access to high-priority medicines for patients outside of the EU (EU-M4all).

Revised Guidance

Guideline on the clinical evaluation of anticancer medicinal products This 6th revision addresses biomarker-guided medicinal product development and recent designs in oncology, such as designs of master protocol studies.

Paediatric Investigation Plans

Revised versions of the following two documents were published in January: Policy on the determination of the condition(s) for a Paediatric Investigation Plan/Waiver (scope of the PIP/waiver) and Guidance on paediatric submissions

Brexit

EMA explains how Regulation (EU) 2023/1182 impacts on centrally authorised medicinal products (CAPs) intended to be placed on the market in Northern Ireland. See Questions and answers to Stakeholders

Newsletter:? Human medicines highlights – Issue 176, January 2024

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

Medicines and Healthcare products Regulatory Agency (MHRA)

Medical Devices

The MHRA published a roadmap which outlines the intended timelines for delivering the future regulatory framework for medical devices. See Press Release plus Roadmap for the Implementation of the Future Regulatory Framework for Medical Devices’ and information about future core regulations.

Health Research Authority

The EU Clinical Trials Information System (CTIS) does not allow information to be submitted about UK trials conducted as part of a multinational trial. To fulfil the Research Ethics Committee (REC) standard approval conditions for trial registration on a public registry within six weeks of recruiting the first research participant in the UK, the HRA provided clarification on the meaning of a “public registry”, which is a registry mentioned on the World Health Organisation (WHO) list of primary registries or the International Committee of Medical Journal Editors (ICMJE) list of registries which facilitates public access, including ?ISRCTN and ClinicalTrials.gov.

European Commission (EC)

Another extension to transition period for In Vitro Diagnostic Medical Devices Regulation (IVDR) To allow companies more time to apply the IVDR, applicable since May 2022, the EC proposed an extension to the rules. The new proposal for high-risk (Class D) diagnostics extends the deadline to December 2027; for high individual and/or moderate public health risk tests (Class C) to December 2028, and for low-risk Class A sterile and class B tests the deadline is now December 2029. See Press Release and the Proposal to amend the Regulation.

Swissmedic

The Swiss Agency for Therapeutic Products (Swissmedic) has announced a new organizational structure to make the responsibilities for medical devices and market surveillance clearer. Swissmedic’s objectives include making legal responsibilities for medical devices more visible and stepping up international collaboration in this area.

Fast-track Authorisation procedure (FTP)

Swissmedic also optimised the process for applying for the implementation of an FTP for products which prevent or treat severe, disabling, or life-threatening diseases. Differing processes will be employed in future based on the outcome of the assessment of the documentation submitted with the application. For more information, including the amended guidance, see here.

Updated Guidance for Products with known active pharmaceutical ingredient (API)

Swissmedic has updated its guidance on APIs explaining that additional documentation?and additional data (pharmacodynamics, pharmacokinetics or toxicity) may be required for medicinal products with known APIs in order to prove that the reference medicinal product's safety and/or efficacy data are transferable.

Podcast: Conversations in Drug Development

Have you tuned in to our recent episode of Conversations in Drug Development, where Professor Alan Boyd and Harriet Edwards discuss trends and predictions for the drug development space in 2024?

In this podcast episode, our host?Harriet Edwards, and Alan Boyd discuss trends from 2023?in the drug development industry and its potential evolution in 2024. Join us as we explore the financial downturn affecting early-stage investment, the rise of cell and gene therapy approvals, and the use of artificial intelligence in various areas of drug discovery and development.

To tune in and listen now, search 'Conversations in Drug Development' on Spotify, Apple Podcasts or Amazon Music or visit Podcast | Conversations in Drug Development | Boyds.