Regulatory news - December 2018

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


European Commission (EC)

Additional competent authorities endorse GMO requirements for investigational ATMPS

It has been announced that additional member states have endorsed guidance on the EU GMO requirements for investigational ATMPs. The additional endorsements have been reflected in the documents and new versions released, see below for links to the updated documents:

Regulatory Science to 2025 – EMA launch six-month public consultation

The EMA has launched a six-month public consultation on its proposed plan for advancing the Agency’s engagement with regulatory science over the next five to ten years, including both human and veterinary medicines.

Stakeholders are invited to feedback their comments via an online questionnaire by 30 June 2019.

For further information, links to the consultation document and feedback questionnaire, click here.

United Kingdom

Letter to the health and care sector: update on preparations for a potential no-deal Brexit

The Secretary of State for Health and Social Care, Matt Hancock, has provided an update on what the health and care system needs to consider in the period leading up to March 2019. The update includes plans relating to the continued supply of medicines and vaccines; medical devices and clinical consumables; blood and other products of human origin; non-clinical consumables, goods and services. It also includes information on the health and care workforce, research and clinical trials and business continuity plans.

Click here to read the letter.

Be The Match BioTherapies announces collaboration with National Health Service Blood and Transplant of the United Kingdom

Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, has announced a collaboration with the National Health Service Blood and Transplant (NHSBT), a public organization responsible for the donation and storage of blood in England, and organ, tissue, bone marrow and stem cells in the UK. The collaboration aims to streamline access to authorized collection and processing centres through the creation of a framework for cell and gene therapy companies to gain access to partnerships with NHSBT’s authorized cell collection centres and close-to-patient cell manufacturing facilities in the UK.

This collaboration is key step forward in helping ensure the harvest of high-quality starting materials for cellular therapy manufacture.

For more information and to read the full article, click here.


Food and Drug Administration (FDA)

FDA releases strategic framework to advance use of real-world Evidence to support development of drugs and biologics

The FDA announced their new 2019 strategic framework on how the Agency will continue to advance opportunities to leverage real-world data (RWD) to improve regulatory decision making. With vast amounts of RWD being collected during the routine care of patients from a variety of sources, this offers a great opportunity to utilise such data to generate evidence and better understand clinical outcomes.

The framework is aimed at leveraging information gathered from patients and the medical community to inform the FDA’s decisions across drug and biologic development. It outlines a number of important RWE-related efforts to explore the potential for using RWE to support approval of new indications for approved drugs or to support post-approval study requirements. To help implement this strategy, the FDA are also working to develop new guidance to assist sponsors interested in developing and using RWE.

The FDA’s Centre for Drug Evaluation and Research (CDER) and Centre for Biologics Evaluation and Research (CBER) currently perform safety monitoring studies through pharmacoepidemiologic research projects using electronic health data stored in the Sentinel System. CDER also uses the Clinical Practice Research Datalink (CPRD) which captures UK electronic health data.

Click here to read the full article. The PDF Framework for FDA’s Real-World Evidence Program is available here.

FDA sends warning to company for marketing dangerous unapproved stem cell products that put patients at risk and puts other stem cell firms providers on notice

The FDA has warned Genetec, Inc. of San Diego, California and its president Edwin N. Pinos for marketing stem cell products without FDA approval and for significant deviations from current good tissue practice (cGTP) and current good manufacturing practice (cGMP) requirements; including some violations that may have led to microbial contamination, potentially causing significant blood infections in patients. Genetech processed umbilical cord blood into unapproved human cellular products, which were distributed by Liveyon, LLC.

The agency is sending letters to other manufacturers and health care providers who may be offering stem cell treatments to reiterate the FDA’s compliance and enforcement policy.

Click here to read the full article.

Patient Experience Data: FDA Drafts Guidance

The FDA has published guidance to support stakeholders in the development of proposed draft guidance on patient experience data. The draft guidance provides in a Q&A format addressing questions related to both guidance development and other potential pathways for contributing patient experience data.

FDA Commissioner, Scott Gottileb, said “today’s guidance document is part of our commitment to advance patient-focused drug development and is one of several guidance's that we’re developing regarding the collection of patient experience data, and the use of such data and related information in drug development”.

The draft guidance can be found here.

FDA Finalises Data Integrity Q&A Guidance

The FDA finalised its questions and answers guidance on complying with data integrity requirements under current good manufacturing practice (cGMP) for drugs and biologics. The guidance has been revised to aid drugmakers in identifying lapses in data integrity processes and implement best practices to prevent these gaps.

The guidance was developed in response to an increase in data integrity violations observed and includes 18 questions and answers listing their relevant sections of the regulations governing data integrity under 21 CFR parts 210, 211 and 212.

For further information and to read the guidance, click here.

Public consultations



Consultation period


Evaluation of the legislation on medicines for children and rare diseases (medicines for special populations)

12 Oct 2018 to 4 Jan 2019

Public consultation




Consultation period


Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells

31 July 2018 to 31 July 2019

Draft guidance

EMA Regulatory Science to 2025 – Strategic reflection

19 Dec 2018 to 30 Jun 2019

Public consultation




Consultation period


Meta-Analyses of Randomized Controlled Clinical Trials to Evaluate the Safety of Human Drugs or Biological Products Guidance for Industry

06 Nov 2018 to 07 Jan 2019

Draft guidance




Consultation period


Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018


Draft guidance




Consultation period


Good Practices for Data Management and Integrity in Regulated GMP/GDP Environments

30 Nov 2018 to 28 Feb 2019


Draft guidance


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