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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Data sharing critical to close ‘innovation blackhole’ for rare diseases
Access to data and 'real world evidence' obtained from observational data is needed to help push research and innovation on rare diseases, with support needed to encourage and harmonise this across Europe, stakeholders have said.
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Early stakeholder dialogues crucial for success of rare orphan medicines
Early dialogue between stakeholders is crucial for the effective development and success of rare orphan drugs, used to treat patients with rare diseases, Anna Bucsics, project adviser at the mechanism of coordinated access to orphan medicinal products (MoCA), told EURACTIV in an interview.
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Europe at the crossroad of Rare Diseases innovation: lessons from the past and future outlooks
In shaping the future of rare diseases policies all stakeholders should cooperate to create an environment that actively supports research and clinical development of orphan medicinal products (OMPs). This entails building synergies all across the innovation ecosystem to address the …
