The Committee for Advanced Therapies (CAT) has established a temporary working party on gene therapy, following the discontinuation of the existing Gene Therapy Working Party of the Committee for Medicinal Products for Human Use (CHMP).
The Gene Therapy Working Party (GTWP) is a multidisciplinary group of European experts that provides recommendations to the CAT on all matters relating directly or indirectly to gene therapy, including:
- preparing, reviewing and updating guidelines and other guidance documents, such as reflection papers, in conjunction with other working parties as appropriate;
- ensuring that the scientific committees and working parties are informed about the scientific issues specific to gene therapy medicinal products;
- providing scientific reports on matters of public interest and emergency issues pertaining to gene therapy.
At the request of CAT, the GTWP can also:
- contribute to scientific advice procedures, marketing authorisation evaluation, post-authorisation procedure evaluation, certification, paediatric investigation plan evaluation and orphan designation related to gene-based products;
- contribute to briefing meetings related to gene therapy with external parties, such as pharmaceutical companies, academia, public-private partnerships and patient associations, often in collaboration with the Innovation Task Force;
- liaise with interested parties, such as learned societies, trade organisations, industry, academia and patient organisations;
- contribute to international co-operation on gene therapy-related matters including scientific contribution to the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH);
- act as a focus and catalyst for training for the quality, preclinical and clinical assessment of gene therapy products;
- contribute to and organise workshops and training courses on gene therapy;
- constitute a rapid-acting crisis group to take on specific issues related to gene therapy products with the objective of exchanging information on a European level and co-ordinating responses to the public in a timely manner.
Mandate, rules of procedure and work programme
More information on the GTWP's responsibilities and composition are available in these documents:
Up to 10 European experts constitute the core group of the GTWP. These experts are identified and nominated by the CAT on the basis of their specific scientific expertise or regulatory experience on the subjects covered within the scope of the working party’s mandate. The core group includes one representative from the Biologics Working Party (BWP), one from the Safety Working Party (SWP), and one from the CAT, all of whom have experience in gene therapy.
The members of the CAT ensure that information on the activities of the GTWP is disseminated at a national level.
Below (in alphabetical order of surname) are the current members of the GTWP. The members' declarations of interests are available in the European expert list.
- Alessandro Aiuti
- Nathalie Cartier
- Pablo de Felipe
- Niklas Ekman
- Maria Cristina Galli (Chair)
- Vincent Irwin
- Dominique Masset
- Elizabeth Pollitt
- Matthias Renner (Vice-Chair)
- Axel Ståhlbom