Clinical Trials and Commercialisation Workshop:
Tuesday 24 October
08:30-09:00
Registration
Chairs: Martina Schüssler-Lenz, Alessandro Aiuti
From research to market approved therapies: exploring the challenges of clinical evidence generation and patient access
Gene therapies are evolving rapidly as treatment options for patients with monogenic and so far untreatable diseases. The EU regulatory frame for gene and cell therapies (ATMPs), set up in 2008, provides tailored regulatory provisions and has contributed to the authorisation of safe and efficacious ATMPs. The streamlining of complex authorization processes is planned with the revision of the pharmaceutical frame, but new challenges have come up. In this workshop you will hear about clinical trial approval, hospital exemption and patient access. The workshop will address regulatory and HTA-related aspects and provide a forum for exchange between the different stakeholders involved in the development of ATMPs.
09:00 - 09:05 Martina Schüssler Lenz, PEI Langen, Alessandro Aiuti, SR-TIGET, Milan
Welcome
09:05 - 09:25 CTC01: Martina Schüssler Lenz, PEI Langen
Pre-and post-authorisation - clinical data requirements in the ATMP life cycle
09:25-09:45 CTC02: Alessandro Aiuti, SR-TIGET, Milan
A gene and cell therapy case study of an academic developer. From clinical trials to patient access – what needs to be improved in the EU
9:45-10:05 CTC03: Claire Beuneu, Belgium
How to get a clinical trial with a gene therapy approved in the EU – experience of regulators with the CTR/CTIS system, view from Belgium
10:05-10:25 CTC04: Antje Behring, G-BA, Germany
Patient access to gene and cell therapies – the German HTA experience and aspects related to the EU HTA regulation (including Eunetha21)
10:25-10:45 CTC05: Marcos Timon, Spain
Clinical trial approval and hospital exemption of a CART cell product --experience of a regulator
10:45-11:05 CTC06: Paschalia Koufokotsiou, European Commission
The EU regulatory frame for gene and cell therapies in transition – aspects related to hospital exemption from a European Commission representative
11:05-11:30
Coffee Break
11:30-13:00
Round table discussion
The challenges of clinical evidence generation, registries and patient access
Chair: Martina Schüssler-Lenz
Antje Behring, DE (G-BA), Johan Prevot, IPOPI, Astrid Pechmann, University Hospital Freiburg, Francis Pang, Orchard Therapeutics, Frank Marx, Gilead,