Regulatory news - February 2021

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Guidance: Management of COVID-19 vaccine deployment for ongoing non-COVID-19 clinical trials

The MHRA have updated guidance on their website regarding the management of clinical trials during the COVID-19 pandemic. A section has been added to advise Sponsors on deployment of a COVID-19 vaccine to trial subjects for ongoing clinical trials for indications other than treatment or prophylaxis of COVID-19.

Sponsors are expected to conduct a specific risk assessment for concomitant use of a COVID-19 vaccine for each investigational medicinal product (IMP) with consideration for the trial population. Non-IMPs and/or combination medicinal products may also need to be considered.

Should the risk assessment concur that a COVID-19 vaccine given to a trial subject is considered a simple concomitant medication, the Sponsor should document this as a non-substantial amendment and notify the MHRA with the next substantial amendment.

However, if potential issues are highlighted which require mitigation (e.g. for trials with immunosuppressive elements, or infection aspects for a high-risk population), then a substantial amendment to both the MHRA and applicable REC is required.

For further information, see here.

MHRA Fees for 2020 - 2021

The MHRA have released their current fees for 2020 – 2021. The fees remain the same as 2019 – 2020, with the exception of the EU Exit fees which came into force on 1st January 2021.

Details of the EU Exit fees are given in Part 18 of The Human Medicines (Amendment etc.) (EU Exit) Regulations 2020 No. 1488.

For the current MHRA fees, see here.

MHRA release updated GCP Inspection Dossier Clinical Trials Spreadsheet

The MHRA carry out inspections at organisations involved in conducting clinical trial research to ensure compliance with requirements of good clinical practice (GCP). The majority of these inspections are carried out under a risk-based compliance plan, and organisations chosen for inspection under the routine inspection programme are required to complete a GCP inspection dossier including the following:

  • a list of clinical trials
  • organisation charts
  • standard operating procedure (SOP) lists
  • contact details
  • overview of facilities
  • service providers

The MHRA have released an updated version of the GCP inspection dossier clinical trials spreadsheet, which is a key component of the GCP inspection dossier.

For further information on MHRA GCP inspections, see here. The updated GCP inspection dossier clinical trial spreadsheet is available here.

European Union

European Medicines Agency (EMA)

Draft guidance to support quality data packages for PRIME marketing authorisation applications

The Priority Medicines (PRIME) scheme is an early access approach pathway developed by the EMA to support the development of medicines that target an unmet medical need with the aim to help patients benefit from these therapies as early as possible. The EMA have released a draft toolbox guidance summarising scientific and regulatory approaches for consideration of PRIME scheme applicants, to facilitate the development and preparation of robust quality data packages.

This guidance has been produced as a result of experience to date demonstrating the challenges that applicants face to complete quality and manufacturing data requirements during development of products in early access approaches. Existing regulatory tools and scientific guidance for Sponsors of medicines holding the PRIME designation is pulled together in this guidance.

The draft guidance has been released for public consultation with a deadline for comments by 31st July 2021.

To read the draft guidance and for details of the public consultation, see here.


Food and Drug Administration (FDA)

CBER Guidance Agenda for 2021

The FDA Center for Biologics Evaluation and Research (CBER) has released a list of draft and final guidance documents the agency intend to issue during 2021.

The list contains 14 guidance documents, with most being carried over from the list released for 2020. Included in this list are the following documents related to regulation of ATMPs:

  • Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Guidance for Industry
  • Human Gene Therapy for Neurodegenerative Diseases; Draft Guidance for Industry (Issued January 2021)
  • Considerations for the Development of Human Gene Therapy Products Incorporating Genome Editing; Draft Guidance for Industry
  • Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Therapies; Draft Guidance for Industry
  • Studying Multiple Versions of a Cellular or Gene Therapy Product in a Clinical Trial; Draft Guidance for Industry
  • Regulation of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) - Small Entity Compliance Guide; Guidance for Industry
  • Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency; Guidance for Industry (Issued January 2021)

To see the full list of guidance documents, see here.