Presentations from the 6th EBE Annual Conference on ATMPs – 5 December 2017, London
The remarkable opportunities that cell and gene therapies can offer, but also the seemingly increasing number of obstacles to their wide application, were presented to a full audience in London’s Canary Wharf on December 5th, 2017.
It was the sixth annual regulatory conference organised by the European Biopharmaceutical Enterprises, with the participation of the European Medicines Agency, the European Commission, of industry and patients representatives, and many other stakeholders.
The conference was a rare opportunity for industry, regulators, payers, and patients to meet and discuss what has to happen to ensure that therapies that can effectively treat unmet medical needs are available to patients.
Aiming to support the development of next generation treatments through gene, somatic cell and tissue-engineered therapies, the European Commission established a specific regulatory framework for Advanced Therapies and Medicinal Products (ATMP) in 2007. The EMA’s Committee for Advanced Therapies (CAT) delivers scientific recommendations on ATMP classification and ensuing applications for marketing authorisation. After authorisation, those therapies then need to be made available to patients and be reimbursed at the national level. This year’s conference showed that despite the promise of these new therapeutics, there are challenges to overcome in the development and supply of the ATMPs, as well as in addressing regulatory requirements, where there is now found to be a growing gap between the EMA and the
EU Member States.
Session 1: Learning from the past – recommendations to foster development of ATMPs and patient access:
The EMA view – presented by Ana Hidalgo-Simon
EBE/EFPIA Initiative: Escher Project Results – presented by Renske ten Ham, Utrecht University
European Commission Perspective – presented by Rocio Salvador-Roldan, DG Santé
Session 2: Challenges for ATMP development and supply to the patients
Comparability challenges – presented by Christina Basford, GSK
Challenges associated with supply to larger patients populations: Elements of the CTL019 – presented by Daniel Stark, Novartis
New guideline on requirements for ATMPs in clinical trials – presented by Ilona Reichl, CAT Vice-Chair
Session 3 – How to translate gene editing from the laboratory to the clinic
Industry perspective – presented by Mick Fellows, Astra Zeneca
European Regulators view – presented by Paolo Gasparini, CAT member
FDA views – presented by Anna Kwilas (by Webex)
Update on the revision of guideline “ Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (CHMP/GTWP/671639/2008) – presented by Marcos Timon, CAT alternate delegate
Session 4 – Ensuring safety for patients throughout the lifecycle of an ATMP
A case study from Industry – presented by Myriam Fuchs, Novartis
A case study from Industry – presented by Jens Hasskarl, Celgene and Nick Trede, Juno Therapeutics
Guideline on Safety and Efficacy Follow-up – Risk Management of ATMPs – presented by Caroline Voltz, EMA
More pictures from the event are available here