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For a patient-driven evolution of the Orphan Medicinal Product Regulation: Addressing unmet needs

janvier 2023

Join this European Parliament event, hosted by MEP Frédérique Ries and organised in collaboration with EURORDIS – Rare Disease Europe, in the framework of Brussels Rare Disease Week 2023, to reflect on how the EU can ensure that more and better treatments are available, accessible and affordable for people living with rare diseases.

The event will seek the perspectives of experts such as patients, clinicians, regulators and developers, whilst ensuring an honest conversation on the evolution of the incentives frameworks starting from the needs of people living with a rare disease. 

The revision of the Orphan Medicinal Products (OMP) Regulation is an unprecedented opportunity to revisit the current model, recognising its strengths and weaknesses, and to drive success by design. EURORDIS published its proposal of how to create a robust regulatory framework that will drive innovation and improve access to transformative treatments for this population in the years to come.

8 February 2023 from 2 PM to 4 PM (CET), European Parliament


14:00Welcome remarks by
Frédérique Ries MEP, co-Chair of the network of Parliamentary Advocates for Rare Diseases
14:10For a patient-driven evolution of the OMP regulation Avril Daly, President, EURORDIS Rare Diseases Europe  
14:20The Evaluation of the Regulation on Orphan Medicinal Products: what’s next?
Florian Schmidt, Deputy Head of Unit D1, DG SANTE, European Commission  
Roundtable discussion
14:30-14:55Addressing unmet medical needs through the regulation  
14:30-14:45Preliminary remarks by:
Rebecca Skarberg, Osteogenesis Imperfecta Federation Europe (OIFE)
Kristina Larsson, Head of Orphan Drugs at EMA -European Medicines Agency
14:45-14:55Q&A – Discussion (10min)
14:55-15:20Calibrating incentives for a thriving and innovative European ecosystem for Rare Diseases
14:55-15:10Preliminary remarks by:
Yann Le Cam, Chief Executive Officer EURORDIS Rare Diseases Europe
Maurizio Scarpa, MD, PHD Director, Coordinating Center for Rare Diseases, Azienda Sanitaria Universitaria Friuli Centrale, Udine
Deirdre Clune MEP  
15:10-15:20Q&A – Discussion (10min)
15:20-16:00Improving access to existing and future therapies for rare diseases
15:20-15:30Preliminary remarks by:
Petia Stratieva, Retina International
Nathalie Moll, Director General, EFPIA
Tilly Metz MEP
15:30-15:40Q&A -Discussion
15:40-15:50Moving forward – Message from the upcoming Spanish Council Presidency
Enrique Terol Garcia, Spain Permanent Representation in Brussels, Coordinating advisor for health