Regulatory News - October 2021

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Update Guidance for UK Clinical Trial Authorisation Submissions

The MHRA have updated their guidance on applying for clinical trial authorisation, and management of an approved clinical trial authorisation in the UK, which now incorporates the combined review service (previously known as Combined Ways of Working (CWOW)).

As of 1^st January 2022, the combined review service will become the mandatory process under which all new clinical trials of investigational medicinal products (CTIMPs) applications are prepared, submitted and managed. CTIMP applications must be submitted using a new part of the Integrated Research Application System (IRAS) and should not be started in the standard part of IRAS.

The combined review service is currently open to CTIMP sponsors and applicants via the Health Research Authority (HRA) website. It is recommended that any applicants planning to complete new CTIMP submissions over the coming months should submit via the combined review process. It will not be possible to create new CTIMP applications in the standard system of IRAS from 14^th December 2021.

For the full updates on the MHRA website, see here (clinical trial authorisation submission) and here (management of an authorisation). For further details on the combined review service, see here.

EU

European Commission (EC)

EC Release Updated Clinical Trial Regulation Q&A Document

The EC Clinical Trials Regulation (EU) No 536/2014 Questions & Answers document has been updated in order to clarify certain areas of the Regulation. The following clarifications have been added:

• Documentation regarding the good manufacturing practice compliance of active pharmaceutical ingredients is not required by the Regulation in the clinical trial application.
• Details on the required content of the protocol synopsis.
• How sponsors should report dates annual safety reports are to be submitted for investigator-initiated trials.

To read the updated Q&A document, see here.

European Medicines Agency (EMA)

EMA Release CTIS Go-Live Plan

The EMA have published a go-live planning document in preparation for the launch of the Clinical Trial Information System (CTIS), planned for 31^st January 2022.

An overview of the components of the CTIS, including the submission workspace, authority workspace and public website is provided. The document outlines key steps to be completed before the release of the system covering the following areas:

• IT/Technical/Infrastructure Related Activities
• Business Processes and Operational Activities
• Cooperation in Safety Assessment
• CTIS Change Management Activities
• Delivery of Training
• Communication Planning
• CTIS Engagement

To read the EU CTIS go-live plan, see here.

European Federation of Pharmaceutical Industries and Associations (EFPIA)

EFPIA Call for Harmonisation of GMO-IMP Studies

The EFPIA have release a discussion paper in a call for greater harmonisation of genetically modified organism (GMO) procedures for investigational medicinal products.

Advanced therapy medicinal products (ATMPs), such as gene therapies, consisting of, or containing GMOs, and viral based vaccines are currently required to comply with the EU GMO legislation before a clinical trial can commence. The EU Clinical Trials Regulation (due to go-live on 31^st January 2022) requires a single clinical trial application dossier to be submitted via the CTIS submission portal, which is sent to all Member States involved in the clinical trial. However, the Regulation has not yet addressed GMO documentation required under EU GMO legislation (Environmental Risk Assessment and common application form) for a GMO-IMP.

This paper highlights the upcoming challenges for sponsors of GMO-IMP clinical trials under the Clinical Trial Regulation and calls for competent authorities to be pragmatic and adopt a simple approach to facilitate GMO assessment in coordination with the approval of clinical trials.

To read the full article and access the discussion paper, see here.

US

Food and Drug Administration (FDA)

FDA Announce Partnership to Accelerate Gene Therapy Development

The FDA have announced that they are joining forces with the National Institutes of Health (NIH), ten pharmaceutical companies and five not-for-profit organisations to accelerate the development of gene therapies for Americans suffering from rare diseases.

The NIH have recently launched the Bespoke Gene Therapy Consortium (BGTC) as part of their Accelerating Medicines Partnership (AMP) programme, which intends to streamline the gene therapy development process. A key aim of the BGTC is to advance the current knowledge of adeno-associated virus (AAV) vectors, examining the biological and mechanistic steps of AAV vector production, vector delivery of genes into human cells and activation of therapeutic genes in target cells. Research in this area will help improve vector manufacture efficiency and enhance the overall therapeutic benefit of AAV gene therapy.

The program will also investigate current regulatory requirements for gene therapies and FDA approval process to assess whether a more efficient process can be established.

For further information, see here.

INTERNATIONAL

International Council for Harmonisation (ICH)

Finalisation of ICH E8(R1) Guideline

The ICH has announced that the E8(R1) guideline on general considerations for clinical studies has reached Step 4 of the ICH process and the guideline is now pending sign off by regulators.

The much anticipated guideline incorporates quality by design principles, modernising the approach to the design, conduct and reporting of clinical trials.

To read the full announcement see here. For further information and access to ICH E8(R1) see here.

Public Consultations

MEDICINES & HEALTHCARE PRODUCTS REGULATORY AGENCY (MHRA)