Stronger together: Fighting rare diseases requires collaboration

To address the unique needs of affected individuals and improve patients lives, a multifaceted approach involving all stakeholders is needed

By Tomislav Sokol

Tomislav Sokol (HR, EPP) is a member of Parliament’s Internal Market and Consumer Protection Committee

29 Feb 2024

Affecting up to 36 million people in the EU, rare diseases have a significant impact on the lives of millions of individuals. Often overlooked in the context of public health, it’s paramount we address these conditions. 

We are currently in the process of revising EU pharmaceutical legislation. Regulation on OMPs, introduced in 2000, aimed to address significant challenges that held back innovation. 23 years after the regulation came into effect, more than 200 new therapies have been developed.  

Gap remains, however. We must address the needs of patients who have no existing treatment options. It is imperative to safeguard and enhance the stability and predictability of orphan designation, approvals, marketing exclusivity and regulatory data protection to foster further innovation and support the rare disease ecosystem.  

We are also creating the European Health Data Space (EHDS). By facilitating the secure, responsible sharing of health data across Member States, the EHDS will contribute to a deeper understanding of rare diseases, boosting the development of new medicines and medical devices. 

The rare disease community has been advocating for a European action plan on rare diseases, which entails a co-ordinated, objective-driven strategy

Sharing knowledge, experience and data helps develop practical solutions. The EHDS also enables the development of innovative approaches to rare disease registration, allowing for timelier, more efficient information collection on various types of rare diseases.  

The Network of Parliamentary Advocates for Rare Diseases advocates for the improvement of the lives of rare disease patients. It is co-ordinated by the European Organisation for Rare Diseases (EURORDIS). With this network, MEPs commit to work on EU-wide action in rare diseases.  

The rare disease community has also been advocating for a European action plan, which entails a co-ordinated, objective-driven strategy.  

We need a multifaceted approach to address the unique needs of affected individuals. By fostering collaboration, supporting research and empowering patient organisations, we can improve the lives of rare disease patients. 

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

Read the most recent articles written by Tomislav Sokol - Time for a necessary revision of cross-border healthcare rules


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