Latest Regulatory Updates

December 2023

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

We would like to say a big thank you to all our clients and contacts for your continued support in 2023. We hope you have a restful December and best wishes for the New Year.

Food and Drug Administration (FDA)

Medical Devices:

FDA announced its recognition and encouragement for use of a key consensus standard to support device sponsors as they address cybersecurity concerns. ANSI/AAMI SW96:2023 Standard for medical device security - Security risk management for device manufacturers aligns with existing international safety risk management standards and quality systems expectations and provides direction on how to consider and address cybersecurity risks in device design and development. The list of all currently recognized consensus standards for medical devices is available here.

FDA also announced a new MITRE report, Next Steps Toward Managing Legacy Medical Device Cybersecurity Risks, which identifies near-term solutions to address the challenges posed by legacy medical devices.

Final Guidance Published:

Real-Time Oncology Review (RTOR) provides recommendations on the process for submission of selected new drug applications (NDAs) and biologics license applications (BLAs) with oncology indications for review under RTOR.

Final Guidance Published:

Submitting Patient-Reported Outcome Data in Cancer Clinical Trials provides technical specifications for submitting patient-reported outcome (PRO) data collected in cancer clinical trials to support a marketing authorisation application (MAA) for a medical product in oncology, where a PRO is a type of clinical outcome assessment (COA) used to collect patient experience data.

Draft Guidance Published:

Translation of Good Laboratory Practice Study Reports: Questions and Answers

The concepts described in this guidance provide information on the translation of GLP reports from studies intended for submission to FDA to support a MAA.

Safety communication:

FDA issued a safety communication regarding reports of T-cell malignancies, including chimeric antigen receptor (CAR)-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies.

European Medicines Agency (EMA)

IRIS Guide for Applicants

An updated version IRIS guide is available. Version 3.0 (21 November 2023) merges the IRIS guide to applicants and IRIS guide to parallel distribution applicants.

EMA Guide – What EMA Publishes and When

This guide was last updated in November. Major changes concern the publication of full risk management plans (RMPs) for all medicines.

Newsletter: SME Office – Issue 60, November 2023

The bulletin highlights news, documents, and activities of interest to SMEs and their stakeholders.

Newsletter: Clinical Trials Highlights – Issue November 2023

Provides insights on clinical trials topics, including Accelerating Clinical Trials in the EU (ACT EU) and CTIS

Clinical Trials Information System (CTIS)

An updated version CTIS Sponsor handbook was released 06 November 2023

Clinical Trials Information System (CTIS)

Member State competent authorities for clinical trials and medical devices and the European Commission published information on the "COMBINE" project, which addresses the challenges at the interface between the regulations on clinical trials of medicinal products, medical devices and in vitro diagnostics.

Clinical Trials Information System (CTIS)

There will be a CTIS winter clock stop from 22 December 2023 at midnight until 08 January 2024 at 00:00:01 CET, during which evaluations of clinical trial applications will stop. Tasks will not have their due date falling during the clock stop and any days during that period are not counted towards the timelines.

Guidance for notification on arrangements for requesting EMA certificates through urgent and standard procedure for December 2023 is available here.

EMA Website Relaunch

From 05 December 2023, EMA’s website will feature a new, simple medicines search, clearer navigation and a revamped “What’s new” page. All the content on the current website will remain available and the current website’s addresses (URLs), such as links to pages, documents, and images, will work after the relaunch.

Medicines and Healthcare products Regulatory Agency (MHRA)

Rare Therapies Launch Pad

In the Chancellor’s Autumn statement, a new programme was announced to develop a pathway for children with ultra-rare conditions to access individualised therapies. The programme’s first project will explore the use of individualized therapies known as antisense oligonucleotides (ASOs) to treat children with ultra-rare and life-threatening brain conditions. For more information visit Genomics England.

Common issues identified during clinical trial applications

The non-clinical section was updated with a new link to guidance on Recommendations related to contraception and pregnancy testing in clinical trials.

Decentralised trial methods

The Health Research Authority (HRA) published a position statement to confirm the current UK position on the use of decentralised trial methods. This includes key considerations for sponsors and those working in research teams, as well as links to useful resources.

International Recognition Procedure (IRP)

Additional guidance is available that addresses common queries about the IRP for medicines licensing applications using pre-existing approvals from Australia, Canada, the European Union, Japan, Switzerland, Singapore, and the United States.

Before submitting an IRP application applicants should use the new eligibility checker to determine whether their MAA is suitable for IRP and to identify which route to follow.

The new IRP itself becomes operational on 1 January 2024.

Confidentiality Advisory Group (CAG) support

From 18 January 2024, applicants seeking Section 251 support or advice from CAG regarding the use of confidential patient data without consent for research or non-research purposes will be expected to attend the online meeting at which their application is discussed.

For further information see HRA website. The CAG mainline will close on 29 December 2023.

TOPRA Awards for Regulatory Excellence 2023

Double win for Boyds at TOPRA Awards 2023

The TOPRA Awards 2023 saw the Boyds team take home two awards, with our regulatory affairs team crowned winners of the Futures Award and our Senior Director of Regulatory Affairs Katherine Bowen awarded the Inspiration Award. Read the full story here.