Despite their moniker, Rare Diseases are a common health issue and affect over 30 million Europeans.

Twenty years ago, rare diseases were truly orphaned, as only a handful of authorised medicines were available to treat them. This situation has changed in large part thanks to the European Orphan Medicinal Product (OMP) Regulation that put in place an incentive framework to attract more development into rare diseases.

This has led to a significant improvement of 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, to a lack of transformative treatments where authorised treatments exist, to insufficient diagnosis and unequal patient access.

In our new report “Innovating in Rare Diseases”, 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 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 addressing the barriers along the OMP lifecycle.
• Partnerships are a key tool to overcoming those barriers.

Building on existing initiatives and structures and inspired from 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.