Innovating for impact: Shaping the future of rare disease treatment [Video]

Millions of Europeans are living with rare diseases for which no treatment options exist.

Out of the 6,000 to 7,000 identified rare diseases, 95 per cent do not have an approved therapy, and research is not ongoing for most of them.

The Rare Disease Moonshot project was created to find solutions to accelerate this research and increase partnerships between the public and private sectors.

One of the ways to make it happen is to defragment the field of investment towards the treatments, which is an objective of the initiative.

Magda Chlebus, Executive Director of Scientific and Regulatory Affairs at the European Federation of Pharmaceutical Industries and Associations (EFPIA), listed the major challenges to achieve this goal: “science and translational research capabilities”, “regulatory and access barriers”, and …