LUNCH WORKSHOP 6
Organised by Shire
New innovations require fresh tools and policy approaches to ensure we can adequately and equitably meet the needs of those with rare diseases. This is particularly true given their small patient populations and lack of data which can lead to late or no access. The potential of real world evidence must be exploited to address uncertainties that occur during the evidence generation pathways. The TRUST-4RD multi-stakeholder initiative has created a practical tool to address evidentiary requirements and the use of RWE to enable earlier patient access.
This session will:
Views and insights from academia, the ERNs, national/European policy-makers, patient representatives and industry will be shared during the workshop. The panel will be followed by Q&A with the audience.
SIMONE BOSELLI, Public Affairs Director, EURORDIS
LIISA-MARIA VOIPIO-PULKKI, Head of the Management Support Unit, Ministry of Social Affairs and Health, Finland
HEIDRUN IRSCHIK-HADJIEFF, Vice President DACH Cluster, Shire
RUTH LADENSTEIN, Professor of Paediatrics, Head of the Clinical Trials Unit S²IRP - CCRI (Studies & Statistics for Integrated Research and Projects) St. Anna Kinderkrebsforschung e.V.
ALEXANDER NATZ, Secretary General, EUCOPE
VINCIANE PIRARD, Task-force Member, Senior Public Affairs Director, SANOFI Genzyme
MAURIZIO SCARPA, Past Chairman of the ERN Coordinator Group, European Reference Networks
JOHN BOWIS, Special Adviser to FIPRA, Former MEP and Member of the UK Parliament