Rare Disease Week 2023: Advocates call for a patient-driven approach in the evolution of the Orphan Medicinal Products Regulation
On 8 February, patient advocates and policymakers from across Europe gathered at the European Parliament for a discussion about the evolution of the Orphan Medicinal Products Regulation and the rare disease community’s unmet needs that it needs to address. Currently, about 95% of people living with rare diseases lack access to any form of transformative treatment. The conference, co-hosted by EURORDIS and Frédérique Ries MEP, sought to present the perspectives of a wide range of experts while fostering a space for honest conversation with participants.
The ongoing revision of the Regulation presents a unique opportunity to amend the existing legislation in a way that continues to drive innovation while improving the accessibility of treatment for people living with rare diseases. Speakers asserted the importance of promoting Europe as a global pioneer in drug development, while ensuring that medicines remain accessible to those who need them. They also discussed the need for intervention by regulators earlier on in the drug development process, in order to better support clinical trial design and ensure that a greater number of drugs in development actually make it to the market authorisation stage.
Additionally, EURORDIS took the opportunity to once more present their proposal for the revision. These focus on prioritising the transformation of Europe’s research and development landscape for rare diseases; positioning the EU as a competitive arena for drug development; and ultimately, ensuring that the needs and participation of people living with rare diseases are kept at the heart of the revised Regulation.
The event took place within the framework of Rare Disease Week 2023, a four-day program organised by EURORDIS meant to give patient advocates the tools they need to take action at the European level. In addition to the conference, participants had the opportunity to meet with Members of the European Parliament and representatives from the European Commission and the next Spanish Presidency of the EU Council.
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bartosb (24 mai 2023). Rare Disease Week 2023: Advocates call for a patient-driven approach in the evolution of the Orphan Medicinal Products Regulation. ELSIBI. Consulté le 16 mars 2026 à l’adresse https://doi.org/10.58079/o6br





