Regulatory News - November 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)

Use of Real-World Data in Clinical Research

The MHRA have updated their guidance on management of clinical trials during the COVID-19 pandemic to clarify the process for notification of urgent safety measures (USMs) to the Agency.

As of the 16th November 2021, notification of USMs to the MHRA should be performed following the standard procedure for USM notifications, detailed in the guidance on their website.

The updated guidance on managing clinical trials during COVID-19 is available here. Guidance on the standard notification procedure for USMs can be found here.


European Commission (EC)

EU Extends Scope of Guideline on Clinical Trials with Genetically Modified Cells

The EU has updated the scope of its guidance document good practice on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified.

The guidance is now also applicable to genetically modified human cells without viral vectors and genome edited cells. A section has been added to the manufacturing requirements and containment levels section of the document to account for manufacturing activities with cells modified without a viral vector. The corresponding common application for clinical research with GMOs has been updated alongside the guidance.

This guidance should be read in conjunction with the frequently asked questions document titled medicinal products for human use containing or consisting of GMOs: interplay between the EU legislation on medicinal products and GMOs, available here.

To read the guidance, see here. The updated application form is available here.

European Medicines Agency (EMA)

EU Regulators Vision for Use of Real-World Evidence in Medicines Regulation

Members from the EMA, the Danish Medicined Agency, and the Federal Institute for Drugs and Medical Devices (BfArM) have collaborated to produce an article outlining the vision of European regulators for enabling the use of real-world evidence (RWE) in medicines regulation.

Vital to delivering this vision is the Data Analysis and Real-World Interrogation Network (DARWIN EU). This EU-wide network is due to be launched in early 2022 with the establishment of a coordination centre to provide timely and reliable evidence on the use, safety and effectiveness of medicines for human use from real world healthcare databases across the EU.

The article, published in Clinical Pharmacology & Therapeutics, describes plans to establish methods for high-quality collection and use of RWE in cooperation with stakeholders.

For further information, see here. To read the article, see here.

EMA Guidance on Navigating Regulatory Requirements for ATMP Developers

The EMA have published guidance on their website for ATMP developers to help navigate key regulatory requirements.

The guidance includes flowcharts and checklists covering quality, clinical and non-clinical development, detailing key questions to ask at each stage and common challenges developers should be aware of. There is also guidance on the regulatory support available from the EMA at each stage of development.

The flowcharts and checklists were presented at a webinar given by the EMA and the European Infrastructure for Translational Medicines (EATRIS) on navigating the regulatory requirements for ATMPs.

See here for the flowcharts and checklists. A recording of the webinar is available here.


Food and Drug Administration (FDA)

Cures 2.0 Bill – Follow up to 21st Century Cures Act Released

The follow up legislation to the 21st Century Cures Act, the Cures 2.0 bill, was announced on 16th November 2021 after two years in the making. The Cures 2.0 bill intends to continue the acceleration of the discovery, development and delivery of 21st century cures, and enhance medical innovation. It also proposes to provide new funding for programmes at the FDA and Centers for Medicare and Medicaid Services (CMS), as well as create the Advanced Research Projects Agency for Health (ARPA‑H).

Key provisions of the Cures 2.0 bill include:

  • Acceleration of timelines for Breakthrough and Regenerative Medicine Advanced Therapy (RMAT) designation programmes.
  • Measures to increase clinical trial diversity.
  • Requirements to improve the collection of patient experience data in clinical trials.
  • Grants for novel trial designs and other innovations in drug development.

In addition, a section is included to account for cell and gene therapy development, requiring the Health and Human Services Secretary to report to Congress on the status of cell and gene therapy regulation, and foreseeable regulatory challenges for the FDA.

To read the Cures 2.0 bill, see here. A section-by-section summary is available here.


International Council for Harmonisation (ICH)

Industry Calls for Scope Changes to ICH S12 Guideline

The public consultation of ICH S12 guideline on nonclinical biodistribution considerations for gene therapy products has raised several suggested changes by industry groups Alliance for Regenerative Medicine (ARM) and the American Society of Gene and Cell Therapy (ASGCT).

The two industry groups support the harmonised guidance, but also suggest changing the scope of the guideline, restricting the scope to include in vivo gene therapy, excluding ex vivo genetically modified cells. Clarification is sought on specific technical recommendations given in the guideline, including global expectations for assay methodologies, and regulatory expectations for bioanalytical assays.

See here for further information. See here for a summary of the public consultation comments.

Public Consultations

No relevant public consultations.