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 voz de los pacientes de enfermedades raras en EuropaEURORDIS La voz internacional de las personas que tienen enfermedades rarasRare Disease International Reuniendo a pacientes, familiares y expertos para compartir experiencias en un foro moderado en distintos idiomas. RareConnect El Programa Rare Barometer es una iniciativa de EURORDIS que realiza encuestas para transformar las experiencias de los pacientes con enfermedades raras en cifras que puedan compartirse con los responsables políticos.Rare Barometer Una campaña internacional de sensibilización que se celebra todos los años el último día de febrero, El Día de las Enfermedades Raras es una iniciativa de EURORDISRare Disease Day Únete al mayor grupo de partes interesadas en toda Europa, en la Conferencia Europea sobre Enfermedades Raras y Medicamentos Huérfanos que se celebra cada dos años. ECRD es una iniciativa de EURORDISEuropean Conference on Rare Diseases