Event report: European Health Forum Gastein: A better tomorrow for rare disease care and research

EUCOPE, in partnership with Sanofi, participated in the European Health Forum Gastein 2021 (EHFG) on 29 September in a session on rare diseases to discuss what is needed to stimulate basic research, clinical development, and access to care across the EU in light of the EU Orphan Medicinal Product (OMP) Regulation Review.

The OMP Regulation has proven a tremendous success for patients, promoting research and innovation in the rare disease space since its adoption. Today, there are about 200 approved treatments for rare diseases, giving hope to more than 20 million people. At the same time, the pandemic has highlighted fundamental challenges faced by rare disease patients, such as the need for disease screening, access to care, and stress.

To continue supporting patients and provide treatment during the crisis, important lessons have been learnt regarding access to care and digitalisation. As the OMP Regulation undergoes review in 2021, another pivotal legislative moment for rare diseases is on the horizon, and with it an opportunity to tackle remaining gaps in rare disease research, investment and care.

The first panel looked at the achievement of the EU OMP Regulation to date and the broader context this review is set into, including the ambitious plan of the European Commission with its Pharmaceutical Strategy for Europe.

In the second panel, experts focused on solutions to tackle challenges discussed in the first session and fleshed out actionable ideas to strengthen the OMP development environment in Europe. The panel built on the recommendations of the  European Orphan Drug Expert Group (OD Expert Group) presented earlier this year [Link].

PANELLISTS

1st Panel

• Yann Le Cam, Chief Executive Officer, EURORDIS-Rare Diseases Europe
• Kaya Kantorska, Policy Officer, Pharmaceuticals, Directorate-General for Health and Food Safety, DG SANTE, European Commission
• Manuela Buxo, Head of Region Europe, Sanofi Genzyme

2nd Panel

• Michela Gabaldo, Head of Translational Projects Management & Regulatory Affairs, Fondazione Telethon
• Alexander Natz, Secretary General, EUCOPE (European Confederation of Pharmaceutical Entrepreneurs)
• César Hernández García, Head of the Department of Medicines for Human Use, Spanish Medicines Agency

 

MODERATOR

• Christian Jervelund, Health Care & Life Sciences Partner, Copenhagen Economics

Re-watch the event here