The past twenty years have seen an impressive array of new treatments for rare diseases approved in the European Union – a success we can largely attribute to the Orphan Medicinal Products Regulation introduced in 2000.
This reality should not hide a hard truth: only 5% of people living with a rare disease have received a transformative treatment approved for the entire EU, according to data gathered by patient organisation EURORDIS-Rare Disease Europe.
The vast majority of patients – seven out of ten – only receive symptomatic treatments for their condition. Additionally, one in five patients are unable to get the treatments they need because they are not available where they live, reflecting the fragmentation of the market across the Member States. We must change that.
This article was commissioned as part of a series focused on Rare Disease Day 2023, in partnership with Takeda, Eurordis, Efpia and Eucope. Click here to read the full report
The upcoming revision of the Orphan Medicinal Products Regulation is the EU’s chance to build upon the current model and update it. This must start with addressing the needs of those living with the very rarest diseases and those currently without therapeutic options.
Among the tools at our disposal, the use of more targeted incentives is an option that could boost the development of treatments across the whole spectrum of rare diseases and specifically in the therapeutic areas most in need. Such new regime would need to guarantee that the European Union remains attractive in a highly competitive international environment. The future of Europe’s world-class health and research systems is at stake.
“We need to work towards a smooth and coherent European pathway, from development to access, and ensuring equity amongst Europeans”
The Regulation must also encourage early dialogue and guarantee that there are mechanisms to actively involve all stakeholders at an early stage in the development of a treatment. We need to be working towards a smooth and coherent European pathway, from development to access, and ensuring equity amongst Europeans.
It goes without saying that this Regulation must be fully aligned with current and evolving EU legislation, such as the other proposals in the Pharmaceutical Package, the European Health Data Space and the Health Technology Assessment Regulation.
Needs-led innovation should be the driving force of a healthy rare disease ecosystem. Any revision to the Orphan Medicinal Products Regulation must safeguard and leverage the rich European ecosystem of research, development and access to treatments against rare diseases. We owe it to the 20 million people living with a rare disease in the European Union.