Regulatory News - September 2021

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

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

Updated Guidance on the Management of Clinical Trials During the COVID-19 Pandemic

The MHRA have provided updated guidance for Investigators on managing clinical trials during the COVID-19 pandemic, detailing information for new and ongoing trials, including additional considerations for remote monitoring.

Sponsors planning to restart trials or initiate new clinical trials should consider the principles described in the NIHR guidance on Managing Research Recovery. Following a risk assessment, any risks to trial participants should be addressed in the benefit-risk section of the protocol along with risk mitigation measures.

A risk/benefit assessment should be performed on a trial-by-trial basis regarding continuation of the trial for both ongoing participants and recruitment of new participants. Where sites have to make decisions about adapting or stopping trials, sponsors should assist in the decision of whether any possible actions can be taken to adapt the trial (i.e. to reduce the burden on investigator sites). This should be done alongside the Chief Investigator and local trial teams. If a trial is put on hold due to issues related to COVID-19, the MHRA does not need to be informed. However, if the hold is required due to a direct participant safety issue, or a medicine supply issue, the MHRA needs to be informed by phone or e-mail.

Remote monitoring and auditing activities (such as source data review/verification) necessary for the protection of participant safety and reliability of trial results, require remote access to health records of trial participants. Information on the Access to Electronic Health Records (EHRs) by Sponsor representatives in clinical trials has been published by the MHRA, providing guidance for sponsors on accessing and viewing EHRs remotely and their responsibilities.

For further information, see here.

MHRA Initiates Consultation on Medical Device Regulation

The MHRA has released a public consultation document seeking feedback on proposed changes to the regulatory framework for medical devices in the UK. The aim of the consultation is to help develop a future regime for medical devices enabling improved patient and public safety, greater transparency of regulatory decision making and medical device information, close alignment with international best practice, and more flexible, responsive and proportionate regulation of medical devices.

The consultation has been released alongside an update on plans for establishing a work program for the regulation of software and artificial intelligence (AI) as a medical device.

To support the consultation, the MHRA have scheduled two webinars, one aimed at industry, and one aimed at patients and the wider public, to provide further background.

To read more about the MHRA consultation see here. The online survey is available here.

Updated Guidance on GxP Data Integrity

The MHRA have provided updated guidance regarding GxP data integrity, which now includes a section on Good Laboratory Practice (GLP).

This new section captures the release of the OECD’s Advisory Document on GLP Data Integrity (dated 20th September 2021), which takes precedence over the MHRA GxP Data Integrity Guidance, released in March 2018.

For further details, see here.

 

EU

European Medicines Agency (EMA)

Call for Expressions of Interest to Represent Patient and Healthcare Professionals in Committee for Advanced Therapies

The EC has launched a call for expressions of interest for representatives to participate in the EMA’s Committee for Advanced Therapies (CAT). The Commission is looking to appoint two members (and two alternatives) representing healthcare professional organisations and two members (and two alternatives) representing patient organisations.

The additional patient organisation representatives will be responsible for ensuring the patients’ needs and views are taken into consideration within all CAT discussions. Healthcare professional organisation representatives will be responsible for ensuring the requirements of practitioners, clinicians, pharmacists, or other health professionals are also considered in discussions held within the committee, as well as any implications of CAT recommendations to such health professionals.

For further information, see here.

 

US

Food and Drug Administration (FDA)

FDA’s Advisory Committee Discuss Limitations to Animal Models for Safety Assessment of Gene Therapies

The FDA’ Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) held a virtual meeting on 2nd – 3rd September 2021 to discuss the toxicity risks of gene therapies derived from adeno-associated virus (AAV) vectors. The meeting covered topics including oncogenicity risks due to vector genome integration and safety issues identified during preclinical and/or clinical evaluations.

A key topic of discussion for the meeting was around the merits and limitations of animal studies to characterise the risk of AAV-mediated oncogenicity, and to provide recommendations on specific preclinical study design elements. This arose some debate around the use of animal models and their potential to show what is possible, but also how they can be problematic as they are not necessarily a sufficient representative of the human situation.

Further details of the meeting, including the recordings of the sessions and presentations, are available here.

Public Consultations

MEDICINES & HEALTHCARE PRODUCTS REGULATORY AGENCY (MHRA)

 

Title

Consultation

Period

Category

1

Consultation on the future of medical devices in the United Kingdom

16 Sep 2021

To

25 Nov 2021

Public Consultation

 

EUROPEAN MEDICINES AGENCY (EMA)

 

Title

Consultation

Period

Category

1

ICH S12 guideline on nonclinical biodistribution considerations for gene therapies

24 Jun 2021

To

24 Oct 2021

Public Consultation

2

Guideline on core SmPC, Labelling and Package Leaflet for ATMPs containing genetically modified cells

30 Jul 2021

To

31 Oct 2021

Public Consultation

 

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