29 Feb 2024

Rare Disease Moonshot supports the EURORDIS-Rare Diseases Europe’s call for an EU action plan for Rare Diseases

On the rarest of days, we, the members of the Rare Diseases Moonshot, stand together to celebrate the resilience, strength, and perseverance of those living with rare diseases. This day serves as a poignant reminder of the unique challenges faced by individuals and families affected by rare conditions, and it underscores our collective commitment to making a difference.

Our coalition recognizes that progress is achieved through collaboration, shared knowledge, and an environment that fosters and supports innovation. By bringing together patient advocates, dedicated researchers and research infrastructures, and forward-thinking industry partners, we create a powerful force for positive change.

Today, despite progress in recent years, the reality is stark, with an estimated 95 percent of the 7,000 identified rare diseases still lacking a cure or treatment. In fact, in the worst cases, for many diseases there is no “science” at all. The knowledge and infrastructure required to unlock these “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradigm.

Our vision is to build deep and diverse collaborations on the scale witnessed during the COVID-19 pandemic: collectively, we can generate new scientific knowledge, build upon existing infrastructures, and reimagine the translational research ecosystem for better patient outcomes. On this day, we come together to raise awareness, foster understanding, encourage investments and inspire action to improve the lives of those facing the challenges of rare diseases. Public-private partnerships (PPPs) like the Rare Disease Moonshot can help pool resources to solve problems more quickly, reduce fragmentation and scale up existing initiatives to make a real difference for patients. In the last year, we set ourselves on a path for a transformative change through a series of collaborative exercises: three sets of Research Needs Recommendations focusing on clinical trials, diagnostics, and translational medicines (upcoming) that show the value of speaking with one voice and providing capacity to turn these recommendations into actions. Furthermore, our common work has led to the shaping of Innovative Health Initiative (IHI) calls such as on establishing novel approaches to improve clinical trials for rare and ultra-rare diseases, or ensuring the participation of the industry in the upcoming European Rare Diseases Research Alliance (ERDERA) – also known as RD Partnership – which should build the critical mass of expertise and knowledge required to speed up rare diseases research and development.

On Rare Disease Day 2024, let us renew our commitment to collaboration, innovation, and compassion. By standing together, we can create a brighter future for those affected by rare diseases, where every individual receives the care, understanding, and support they need to live their lives to the fullest. Together, we can turn challenges into opportunities, obstacles into stepping stones, and inspire hope for a world where rare diseases are not defined by their rarity, but by our collective determination to make a difference.

With the new European legislative term kicking off later this year, policymakers have a rare opportunity to redefine policies to drive the scientific, technological, and societal shifts that will ultimately improve the lives of the 30 million Europeans living with a rare disease.

We stand once again behind EURORDIS-Rare Diseases Europe by renewing the Call for a European Action Plan for Rare Diseases already endorsed by 21 Member States in the Employment, Social Policy, Health and Consumer Affairs Council and included in the Recommendations from the Rare 2030 Foresight Study, underlining the value of PPPs in addressing research needs. Nobody can do it alone for rare diseases.