A better tomorrow for rare disease care and research

A better tomorrow for rare disease care and research

The ongoing Orphan Medicinal Product (OMP) Regulation review represents a unique opportunity to discuss what is needed to stimulate basic research, clinical development, and access to care across the EU. It is essential that the fight against rare diseases remains at the heart of Europe’s recovery. This was the core theme of EUCOPE’s session organized in partnership with Sanofi at the 2021 European Health Forum Gastein.

The first part of the meeting 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. 

Kaja Kantorska, policy officer at the European Commission working on the OMP Regulation review, highlighted the tremendous success of the legislation in bringing 200 authorised treatments to the market and transforming the lives of many patients in Europe. However, the current lack of treatment and accurate diagnosis for the majority of rare diseases represents an enormous unmet medical need (UMN). It is, therefore, the perfect moment to look at possible solutions that will foster development addressing those unmet needs.

Different challenges were identified such as the need to strike a balance between needed change and a system that continues to attract companies to invest in rare diseases. Manuela Buxo, Head of Europe Region, Sanofi Genzyme, pointed out that the EU cannot afford to lose ground to other regions of the world such as the US or Japan in terms of patient care and research and development efforts. Going forward, the EU should provide incentives to develop medicines addressing all rare diseases. She added that the real challenge lies in creating access conditions that reward innovation and ensure rare disease patients get access to the medicines they need.

While Yann Le Cam, Chief Executive Officer, EURORDIS-Rare Diseases Europe, stressed the importance of upstream dialogue and coordination among regulators, patients, HTA bodies and developers to pave to way to faster access to rare diseases therapies. “Dialogue upstream to reduce uncertainty is vital. The EMA and HTA agencies should be companions of success, and not gatekeepers”, said Mr Le Cam.

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. 

Michela Gabaldo, Head of Translational Projects, Management & Regulatory Affairs, Fondazione Telethon, César Hernández García, Head of the Department of Medicines for Human Use, Spanish Medicines Agency and Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs, (EUCOPE) all touched upon the importance of cooperation and multistakeholder cooperation, including regulators, researchers, payers, industry, patients and policymakers. Speakers agreed that creating ‘pilots’ of multistakeholder cooperation from research to access would be key to tackle rare diseases’ challenges, and cooperation should build on existing infrastructures such as the European Reference Networks (ERNs) and cross-border healthcare cooperation.

In a final call to action, all speakers agreed on the need to keep working together in the coming years in order to improve patient access to treatments across the EU and to keep rare diseases a public health priority.