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 voce dei malati rari in EuropaEURORDIS Rare Diseases International, la voce internazionale dei malati rariRare Disease International RareConnect è un'iniziativa di EURORDIS che mette in contatto malati, famiglie ed esperti per condividere esperienze in un forum multilingua e moderato RareConnect Il Programma Rare Barometer è un’iniziativa di EURORDIS volta a condurre indagini con l’obiettivo di trasformare le esperienze delle persone affette da malattie rare in cifre e fatti che possono essere condivisi con i decisori politici.Rare Barometer La Giornata delle Malattie Rare, una campagna di sensibilizzazione internazionale che si svolge l'ultimo giorno di febbraio di ogni anno.Rare Disease Day La Conferenza Europea biennale sulle Malattie Rare è il più grande incontro di soggetti interessati al tema delle Malattie Rare in Europa. L'ECRD è un'iniziativa di EURORDISEuropean Conference on Rare Diseases