European Rare Diseases Supplement 2021

A special supplement looking at European and global efforts tackling rare diseases.

The last year has been unlike any other. As the world responds to COVID-19, many of us face uncertainty, disconnect, and an evolving new normal. Unfortunately, these are not new experiences for those living with or caring for someone with a rare disease. We know this journey, and we can build on it to foster empathy and solidarity in our society for people living with a rare disease.

We also know that many people living with a rare disease are facing greater health challenges during the pandemic: severely disrupted essential treatment and care; and lives faced with heightened risk and anxiety. Everyone in our community has felt this extra vulnerability, this need for extra support, and, too often, this extra physical or mental suffering. We cannot lose sight of what we want to achieve within a changing environment. Throughout this year, health has been thrust into the spotlight and become a political priority.

Our experience shows that collaboration benefits European patients. Rare diseases are a model of innovation; driving progress in therapies, healthcare organisation, and digital health. Tomorrow, rare diseases can become a model of performance in public health and healthcare.

The EU4Health programme shows that health has become an EU political priority, with a tenfold budget increase and a seven-year funding commitment for European Reference Networks (ERNs). Citizens can benefit from pan-European cooperation in diagnostic strategies, newborn screening, health technology assessments, patients’ access to medicines, addressing unmet medical needs, and cross-border access to care.

We can build on recent experience with vaccines for SARS-Cov2: the fast-track evaluation by the European Medicines Agency, the joint negotiation & procurement, and equal access on the same day. We want all citizens living outside the European Union to benefit too.

We are also completing the Rare 2030 Foresight Study in rare disease policy, which provides policy recommendations for improving the lives of people living with a rare disease. With the development of the Pharmaceutical Strategy, Europe has the opportunity to develop a new ecosystem.

We too have a collective responsibility to shape a new approach which will accelerate the transition of major scientific advancements into new therapies, in a predictable and sustainable way. The rare disease community can be proud of its achievements over the past two decades, and we look to the future with great energy and drive. Building on our collective expertise, we will continue to tackle any challenges that lie ahead.

- Yann Le Cam is the Chief Executive Officer of EURORDIS