EURORDIS welcomes European Commission’s evaluation of EU Regulation on orphan medicinal product but urges swift action to improve patients’ access to orphan medicines


14 August 2020, Brussels

EURORDIS-Rare Diseases Europe - an alliance of 929 rare disease patient organisations - welcomes the publication of the European Commission’s evaluation of the EU Regulation on orphan medical products and now urges the Commission to uphold the principle established by the Regulation that people with rare diseases deserve access to the same quality treatment as the general population.

The EU Regulation on orphan medicinal products has helped transform the lives of many people, with 181 new orphan treatments approved and 2,286 orphan designations.

However, the reality we are facing is one of increasing inequalities across Europe. Results from our latest survey of 7,500 rare disease patients and family members revealed that only 5% of respondents already accessed a centrally approved curative/ transformative treatment. We hear regularly of cases when a patient’s access to an approved treatment is obstructed because of the fragmented European market. And most of the 6,000+ rare diseases still do not have a treatment, resulting in a high unmet need.

Simone Boselli, EURORDIS Public Affairs Director, commented, “While an improved implementation of the orphan regulation will help to improve access to orphan medicines for the 30 million people living with a rare disease in Europe, our community simply does not have the time to wait for the finalisation of the revision of the Regulation for any action to be taken. Member States and industry could already move forward with joint assessment and negotiations without waiting for changes in the regulation. This could be done by creating a European joint table of negotiation to widen access to therapies in an affordable way – we just need the political willingness to drive such an initiative. The Beneluxa Initiative has shown this approach can work.” 

He added, “In the context of the overall Pharmaceutical Strategy, we believe Europe has the opportunity to develop a new ecosystem, a framework based on a global approach to innovation for unmet medical needs and on sustainability for healthcare systems, as well as financial attractiveness to developers and investors.

About EURORDIS-Rare Diseases Europe

EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over 900 rare disease patient organisations from 72 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services.

Press contact

Eva Bearryman, Senior Communications Manager, EURORDIS-Rare Diseases Europe

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Page last updated: 14/08/2020
La voix des patients atteints de maladies rares en EuropeEURORDIS Donnant la voix, à l'international, aux personnes vivant avec une maladie rareRare Disease International Pour rapprocher patients, familles et experts sur un forum modéré multilingue, où partager leurs expériences. RareConnect Le programme Rare Barometer est une initiative d’EURORDIS qui vise à réaliser des sondages pour transformer les expériences des patients de maladie rare en faits et chiffres à communiquer aux décideurs politiques.Rare Barometer Campagne internationale de sensibilisation organisée chaque année le dernier jour de février, la Journée Internationale des Maladies Rares est une initiative d'EURORDISRare Disease Day Participez au plus grand événement réunissant tous les acteurs des maladies rares en Europe lors de Conférence européenne bisannuelle sur les maladies rares et les médicaments orphelins (European Conference on Rare Diseases and Orphan Products, ou ECRD). L'ECRD est une initiative d'EURORDISEuropean Conference on Rare Diseases