Regulatory Round-up - November 2023

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia

The MHRA has granted conditional marketing authorization for Casgevy, a CRISPR/Cas9 gene-edited therapy for the treatment of sickle-cell disease and transfusion-dependent β-thalassemia of patients aged 12 and over. Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. Please find further information here.

MHRA to launch the AI-Airlock, a new regulatory sandbox for AI developers

The MHRA has announced it is launching its new ‘regulatory sandbox’, the AI-Airlock, that will provide a regulator-monitored virtual area for developers to generate robust evidence for their advanced technologies. The AI-Airlock will assist in the development and deployment of software and AI medical devices, providing patients with earlier access to cutting-edge innovations.

This initiative shows the MHRA’s commitment to building on its existing capabilities and investing its regulatory expertise to enable highly innovative areas of medical product development, bringing cutting-edge products to UK patients faster, without compromising on safety and performance standards. Please find further information here.

MHRA launches online Eligibility Checker tool for applications via the new International Recognition Procedure (IRP)

The MHRA has launched the Agency’s new online ‘Eligibility Checker’ tool. From the 20^th of November 2023, applicants should can use this tool to determine whether their Marketing Authorisation Application (MAA) is suitable for the International Recognition Procedure (IRP) and to identify which route (A or B) to follow, before submitting an IRP application. The new IRP itself becomes operational in six weeks’ time, on 1^st of January 2024. Please find further information here.

EUROPE

European Commission (EC)

First electronic product information (ePIs) published for selected human medicines

The Heads of Medicines Agencies (HMA), the EC and EMA have published, for the first time, electronic product information (ePI) for selected human medicines harmonised across the EU. The product information of a medicine includes its summary of product characteristics, labelling, and package leaflet. The creation and testing of ePIs in real regulatory procedures is being explored through a one-year pilot initiative by HMA, EMA and the EC to enable the transition to the electronic system for medicines evaluated both nationally and at the European level. The ePI initiative is an action under the Pharmaceutical Strategy for Europe supported by the EU funding programme EU4Health. Please find further information here.

European Directorate for the Quality of Medicines (EDQM)

EDQM launches updated version of the EuroGTP II tool to ensure good tissue and cell practices for human application

To help ensure quality and safety in the field of substances of human origin (SoHO), the EDQM has been assigned to manage the EuroGTP II tool for good tissue and cell practices. This interactive assessment tool is now available on the EDQM SoHO platform, along with the Microbiological Risk of Contamination Assessment (MiRCA) tool. Identifying, quantifying and assessing the level of risk associated with novel SoHO preparations using a standard process is highly important as any modification in processes associated with the donation, procurement, testing, processing, storage and distribution of such preparations may impact the quality and/or safety of SoHO-related therapies and products. The EuroGTP II tool can help assess these risks through three interactive steps:

• determine if a SoHO preparation or process has any novelty;
• assess the risks associated with the SoHO preparation or process;
• determine the extent of any studies and/or follow-up required to guarantee the safety and efficacy of the SoHO preparation or process.

Please find further information here.

Next-generation sequencing for adventitious virus detection in biologics: EDQM collaborates on article published in Biologicals

The EDQM has co-authored a report with academics and interested parties, entitled “Report of the third conference on next-generation sequencing for adventitious virus detection in biologics for humans and animals”. It reports on an International Alliance for Biological Standardization (IABS) conference held in September 2022 that emphasized the significant progress made in next-generation sequencing (NGS, also called high-throughput sequencing, or HTS) overcoming limitations of current testing methods for adventitious virus detection in biologics. The conference, organised by the EDQM and FDA gathered international representatives from regulatory and public health authorities and other government agencies, industry, contract research organizations, as well as academia to present the current status of NGS applications and the progress on NGS standardization and validation for detection of viral adventitious agents in biologics, including human and animal vaccines, gene therapies, and biotherapeutics.

European Medicines Agency (EMA)

Committee for Advanced Therapies (CAT): minutes of the meeting 4-6 October 2023

The European Medicines Agency (EMA) publishes the agendas, minutes and meeting reports of the plenary meetings of CAT. These reports summarise the main findings and outcomes of the meetings. The minutes of the 4-6^th October CAT meeting are now available here.

USA

Food and Drug Administration (FDA)

Process to Request a Review of FDA's Decision Not to Issue Certain Export Certificates for Devices

The FDA has reissued a guidance that describes the information that the Center for Devices and Radiological Health (CDRH), the Center for Biologics Evaluation and Research (CBER), in collaboration with the Office of Regulatory Affairs (ORA), will provide to a person whose request for a CFG (Certificate to Foreign Government) or CFG-NE ( Certificate to Foreign Government for Device Not Exported) for a device is denied, and the process for seeking review of such a denial. Section 801(e)(4)(A)(ii) of the FD&C Act applies to export certificates for devices, as well as other FDA-regulated products, that are exported from the United States. Such FDA export certifications provide information on a product and/or establishment’s regulatory or marketing status, based on available information at the time FDA issues the certification.

FDA announced its recognition of a key consensus standard to address cybersecurity concerns

The FDA has announced its recognition 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 provides specific requirements for managing security across a product's entire life cycle. Published by the American National Standards Institute, Inc., and the Association for the Advancement of Medical Instrumentation, the Recognized Consensus Standards: Medical Devices aligns with existing international safety risk management standards and quality systems expectations. It also provides direction to sponsors on how to consider and address cybersecurity risks in device design and development. The FDA recommends the use of this new standard to enhance quality and support product performance.

New FDA Guidance Describes Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies

The Food and Drug Administration released final guidance in October that describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) to recognize and use voluntary consensus standards (VCS) for the development and evaluation of regenerative medicine therapy (RMT) products. The guidance also describes how CBER intends to review for recognition in the SRP-RMT.

Through the program, CBER aims to promote the adoption of appropriate standards to streamline regulatory compliance and increase predictability for RMT products, following a model similar to the one used for medical devices.

Those interested in submitting online or written comments on the guidance may do so at any time. Instructions are available online.

FDA reiterates guidance plans on distributed and point-of-care manufacturing

The FDA has reiterated its plan to issue draft guidance spelling out how it intends to regulate distributed manufacturing (DM) and point-of-care (POC) manufacturing of drugs and biologics. FDA expects that issuing such guidance may clear up regulatory ambiguities and spur the adoption of these technologies. Please find further details here.

INTERNATIONAL

International Coalition of Medicines Regulatory Authorities (ICMRA)

ICMRA anniversary: 10 years of strategic leadership and cooperation

The ICMRA has celebrated its 10th anniversary in 2023. The establishment dates to December 2013 when eight regulatory authorities came together to form the Coalition which has now expanded to 38 members with the World Health Organization (WHO) as an observer. The ICMRA Regulatory Authorities is a forum to support international cooperation among medicines regulatory authorities. ICMRA helps address emerging regulatory science issues being faced by medicines regulatory authorities, provide direction and support to challenges across all areas within the regulatory mandates of national medicines regulatory authorities, and respond to regional and global major health crisis.

The anniversary was celebrated during ICMRA's annual summit and Plenary in Melbourne on 13-16 November, hosted by the Therapeutic Goods Administration (TGA) of Australia. ICMRA members exchanged views and discussed topics such as the use of artificial intelligence and machine learning in medicine regulation, evolution of clinical trials and advanced medical products based on genes, cells or tissue engineering.

Public consultations

EUROPEAN MEDICINES AGENCY (EMA)

Food and Drug Administration (FDA)

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