Conferences

The inaugural Rare Disease Clinical Trials Conference took place on World Rare Disease Day 2024. 

World leading researchers and patient advocates shared experiences across topics including rare disease clinical trial methodology, patient relevant outcomes, patient perspective in clinical trial design and the journey from discovery to drug delivery. The importance of public and patient involvement and the power of collaboration  were the overriding themes across the programme.

Thanks to our sponsors Pfizer, Savara, PTx, Takeda, Theravance, Roche, Boehringer Ingelheim and Kyowa Kirin for their support.

Check out our video to get a flavour of the day and join our mailing list for updates on future events!