Rare diseases: Ensuring Europe remains at the forefront of innovation


In the past two decades, a combination of scientific breakthroughs coupled with strong incentive systems has seen considerable progress for rare disease patients, but this innovation has tapered off in recent years.

Given that 95% of rare diseases conditions remain without treatment, the need for science and innovation to tackle rare diseases is far from decreasing.

The Orphan Medicinal Products (OMPs) regulation, introduced in 2000, has been instrumental in stimulating research and development, bringing new treatments to patients and incentivising companies to invest in OMPs.

In the lead up to the publication of the OMP and Paediatric Regulations evaluation, EUCOPE, Europe’s trade body for small to medium-sized innovative companies working in the field of pharmaceuticals and medical technologies, brought together a multi-stakeholder dialogue to explore the ways in which Europe can remain at the forefront of innovation to the benefit of patients with rare diseases.

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