Rare diseases must remain a political priority in the EU and beyond

Rare diseases present unique challenges that demand our immediate attention and action — not only because of the financial and emotional burden they impose on families[i] but also because of the intrinsic inequities faced in access to treatment and care.

Our system needs to change. The current approach does not deliver the right solution for most rare conditions. The main challenges for the global rare disease ecosystem remain on how to further unlock science, improve the time to diagnosis and ensure people have faster and more equitable access to treatments.

The urgent need for a unified action plan

It is vital that the progress of the Orphan Medicinal Products legislation - which has authorised over 200 orphan medicines since 2000 - is not lost in the current review of the Pharmaceutical Legislation. According to a study by Dolon, proposed changes to the orphan incentives framework would mean a loss of 45 products in Europe by 2035, impacting 1.5 million European rare disease patients and foregoing €4.5 billion in R&D spending.[ii]

To truly harness the existing strengths of Europe’s rare diseases and life sciences sector, an EU Action Plan for Rare Diseases is needed. A shared commitment to unify and amplify strategies across Member States would deliver transformational value to patients and families. 

A pan-European solution that shortens the pathway to diagnosis and access to treatment is more than just a policy priority, it's our moral obligation to the 36 million people living with a rare disease. The new ‘Cancer and Rare Diseases Intergroup’ in the European Parliament presents an opportunity to accelerate the discussion around rare diseases across the political spectrum.

A renewed focus on rare diseases

At Chiesi Group, we have aligned our strategy with our ambition of revolutionizing the way health innovation is delivered to patients. Through our new Biotech Center of Excellence in Parma, Italy, we have demonstrated that groundbreaking biotech advancements can happen right here in Europe. We hope this can be a catalyst for the rest of the continent showcasing we have the capacity to produce the life-saving therapies of tomorrow, but also foster the talent to better meet the needs of patients globally—especially in rare diseases.

A renewed political focus on rare diseases will contribute to the EU’s competitiveness as a new frontier for life sciences innovation, and the prosperity of its citizens. By investing in rare diseases, we are positioned to address a wider range of healthcare needs. Now we need policymakers to complement this work and keep the spotlight on rare diseases.

This year’s Rare Disease Day reminds us that the complexities and challenges facing people living with a rare disease are more than we can imagine. Now it’s time for collective action - to incentivize investment and research that will transform scientific challenges into opportunities and ensure a favourable environment for the millions of people living with rare diseases — in the EU and beyond.

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