Innovating for people living with a rare disease

Rare diseases are a common health issue and affect up to 30 million Europeans. Twenty years ago, the European Orphan Medicinal Product (OMP) Regulation was put in place as an incentive framework to attract more development into rare diseases.

This has led to a significant improvement in the situation of rare disease patients, with many more authorised treatments being available. However, unmet needs still exist ranging from a lack of authorised treatments, over lack of transformative treatments to insufficient diagnosis and unequal patient access. In this report, we assess how the barriers to addressing those unmet needs can be tackled.

We find that

• the current incentive framework should be evolved by maintaining current orphan designation thresholds, but allowing for a recalibration of incentives.
• addressing the 95% and delivering on further unmet needs requires Europe to go beyond the incentives of the current OMP regulation to systematically address the barriers along the OMP lifecycle
• partnerships are a key tool for overcoming those barriers

Building on existing initiatives and structures and inspired by partnership models in and beyond the rare disease space, we, therefore, propose six partnership-based solutions for the European OMP space.

The study is commissioned by Takeda.

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