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Clinical Trials and Commercialisation Workshop:

Tuesday 24 October

08:30-09:00 Gare Maritime

9:00-11:05 Auditorium, BEL

From research to market approved therapies: exploring the challenges of clinical evidence generation and patient access

Chair: Martina Schüssler-Lenz, Alessandro Aiuti

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


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 Atrium, BEL
Coffee Break

11:30-13:00 Auditorium, BEL

Round table discussion

The challenges of clinical evidence generation, registries and patient access

Chair: Martina Schüssler-Lenz

Antje Behring, DE (G-BA), Mencía de Lemus Belmonte, SMA Europe, Francis Pang, Orchard Therapeutics, Frank Marx, Gilead, Simone Boselli, Eurordis

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