#ActRare2024: How the EU elections can help tackle rare diseases

The fate of more than 30 million Europeans living with rare diseases hinges on the outcomes of the upcoming elections. Policymakers must use this moment to build a more inclusive future for all EU citizens, including the most vulnerable

By Valentina Bottarelli

Valentina Bottarelli is the Public Affairs Director & Head of European and International Advocacy at EURORDIS-Rare Diseases Europe.

26 Feb 2024

As we edge closer to the European Union elections in June 2024, the spotlight intensifies on a critical yet often overlooked population: the rare disease community. Representing more than 30 million Europeans, this community's fate hinges on the upcoming electoral outcomes, making this a pivotal moment for rare disease policy and advocacy. 

The current outlook for rare diseases in Europe paints a picture of disparity and urgency. With more than 6,000 rare diseases affecting the EU population, a staggering 94 per cent lack approved treatments, underscoring a significant gap in medical research and drug development. The diagnostic journey is equally daunting, where patients face an average wait of five years for a correct diagnosis, a period marked by uncertainty and diminished quality of life. This scenario is further complicated by variances in healthcare systems across Member States, leading to a 'healthcare lottery' based on geography, rather than need. 

Building the engine of the European Health Union 

Responding to this, the European Organisation for Rare Diseases (EURORDIS) has launched the manifesto campaign “Championing the Rare – Building the Engine of the European Health Union”. It urges future EU policymakers to prioritise rare diseases, aiming to transform the current fragmented approach into a unified, goal-oriented and EU-wide strategy. The campaign's goals are ambitious yet essential: halving the average diagnosis time by 2030, increasing the percentage of rare diseases with approved treatments, and ensuring equitable access to care and therapies across all EU nations. 

The EURORDIS campaign focuses on eight specific policy asks designed to address both the immediate and long-term needs of the rare disease community. 

1 European policy framework on rare diseases 

The proposal for a EU policy framework on rare diseases represents a crucial step forward. It seeks to unify and streamline efforts across the EU, ensuring that policies are consistently applied and that they effectively address the needs of rare disease patients. This framework is about creating a common ground where all Member States collaborate towards shared goals. 

2 Earlier, faster and more accurate diagnosis 

The call for improvement in diagnostic processes addresses a fundamental need. Quicker and more precise diagnoses mean that patients can receive appropriate care sooner, reducing the anxiety of uncertainty and potentially improving outcomes. This policy ask is a push towards integrating advanced diagnostic tools and expertise across Europe. 

3 Integrated national and European healthcare pathways 

Developing integrated healthcare pathways at both national and European levels is about ensuring equal access to specialised care. By strengthening connections between local and EU-wide networks, such as the European Reference Networks, patients can receive the best possible care regardless of where they live. 

4 Timely access to affordable and innovative treatments 

This policy ask focuses on bridging the gap between the development of new treatments and their availability to patients. It's about creating a more efficient pathway from research to clinical use, ensuring that patients have access to the latest and most effective treatments without prohibitive costs. 

5 Integrated, person-centred, and lifelong holistic care 

Recognising the diverse needs of rare disease patients, this ask advocates for care that goes beyond medical treatment. It calls for a holistic approach that considers psychological, social and lifelong support needs, ensuring comprehensive care for patients. 

6 Innovative and needs-led research and development 

The emphasis on targeted research and development is about directing efforts and resources to where they are most needed. By involving patients in research and prioritising funding for rare diseases, this approach aims to drive innovation that directly benefits those affected. 

7 Optimised data for patient and societal benefit 

Effective use of health data is key in advancing research and treatment. This policy ask is about harmonising data collection and usage across Europe, ensuring that it's used ethically for the benefit of patients while maintaining their privacy and security. 

8 Sustainable and resilient treatment development 

Focusing on sustainable and resilient treatment development, this policy ask considers the environmental and economic impacts of medical advancements. It's about ensuring that the development of new treatments is as sustainable as it is effective, looking towards long-term viability. 

ECRD 2024: catalysing policy action 

Aligned with the objectives of the EURORDIS campaign, the European Conference on Rare Diseases (ECRD) 2024 will bring these issues to the forefront. Taking place just before the EU elections and organised under the auspices of the Belgian presidency of the European Council, it will shape the community's aspirations for 2030. The conference will synthesise the collective experiences of over 1,000 participants – including patients, academics, industry professionals and policymakers – into actionable, data-driven policy recommendations. 

As the EU elections loom, policymakers will recognise the crossroads at which the rare disease community stands: without strategic, informed action, millions will continue to navigate healthcare and societal challenges in their daily lives.  

It is time for policymakers to build a more inclusive future for all EU citizens, in solidarity with the more and most vulnerable, such as the rare disease population.  

It is time to establish another cornerstone of a European Health Union that leaves no one behind: a new European strategy for rare diseases. 

It is time to join the call of #ActRare2024.   

Read more about Rare Disease Day on our Rare Disease Hub or in our Rare Disease supplement.