Excellence in Rare Diseases and Orphan Drugs

Summary of work

RAREfest is the first of its kind; an open to the public rare disease-inspired festival and campaign bringing passionate and inspirational people together to change the rare disease landscape for the better. This heart-warming, informative and action-inspiring campaign connected scientists, technology companies, charities, social enterprises, artists, performers, schools and the public.

The campaign successfully created a community united behind the goal of addressing the mismatch between public perception, knowledge and the reality of rare diseases. That community became the platform for rare disease expertise to be disseminated, connections to be established, and ideas readily exchanged and discussed.

The result was more than 20 new working collaborations with, and between, pharma, biotech, academia and patient groups meaning additional research, events, discovery projects and materials that add up to a significant change for the better.

Judges' comments

The judges liked that RAREfest created a community of people that otherwise would feel isolated. It is laudable and making a real difference to patients’ lives.