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Rare Diseases Q1 2023

Patient-led advocacy can lead to better rare disease policies

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Yann Le Cam

Chief Executive Officer, EURORDIS-Rare Diseases Europe

Julia Jenkins

Executive Director, EveryLife Foundation for Rare Diseases

Broad patient advocacy fosters change that translates the newest research into practice and nurtures partnerships within the rare disease sector.


Meeting a policymaker and presenting a case for why they should take action is not only a form of direct patient advocacy. It is also a way to ensure policymakers see each rare disease patient as a person, rather than just another number.  

Creating more informed rare disease policies  

EURORDIS-Rare Diseases Europe has launched Brussels Rare Disease Week, February 6–9, during the lead-up to Rare Disease Day. Its purpose is to strengthen advocacy knowledge and skills so that more rare disease patients and their families can effectively influence policies by sharing their stories and articulating their needs directly to policymakers. The engagement ensures a growing cohort of informed and action-oriented rare disease champions among policymakers themselves. 

After the first virtual event two years ago, EURORDIS — with support from the Washington, DC-based EveryLife Foundation for Rare Diseases — gathered over 40 rare disease patient champions in Brussels for a week-long learning and development experience. Throughout the week, the participants in Brussels had a chance to speak to representatives of the Swedish and Spanish EU Presidencies, the European Commission and Members of the European Parliament. 

Patient advocacy has been a driving force in adopting policies that improve individual and community health.

Rare disease policies that leave no patient behind 

Proper patient advocacy helps policymakers make informed, evidence-based decisions on legislation. They can improve regulatory policy and expedite the development of safe and effective drugs and provide them to patients who can benefit at the earliest moment. Well-informed decisions help clinicians and patients by unravelling the landscape of today’s healthcare and ensuring that rare disease patients are not left behind. 

Patient advocacy has been a driving force in adopting policies that improve individual and community health.  

People-centred policymaking is more effective 

With the active involvement of the patient community in the development of milestone legislation — from the EU Regulation on Orphan Medicinal Products to the Paediatric Regulation, to the Directive on patients’ rights in cross-border healthcare — an integrated EU strategy on issues including diagnosis, treatment and care became a reality for the 30 million Europeans living with a rare disease. 

These successes are never taken for granted by our community’s ceaselessly dedicated patient advocates. Far too many improvements in policymaking are desperately needed across Europe, so rare disease patient advocates are continuing their efforts to bring about policymaking that is more effective and, crucially, more people-centred. 

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