More than 90% of the 7,000 rare diseases known to science don’t have a single FDA approved treatment. We believe there’s no time to waste in bringing meaningful treatments to lives affected by rare disease.
We also believe in empowering every stakeholder to make rare disease clinical research as efficient, effective and successful as possible.
We know from experience, that patient-centric rare disease clinical trials that reduce barriers to participation create efficiencies.
This toolkit was developed to support sponsors, patient communities, and other stakeholders to operationalize true patient-centricity in clinical development.
Finding a Cure for Myasthenia Gravis
The Rare Disease Shuffle: Living With a Health Mystery
RARE DISEASES NEWSLETTER Volume 10, July 2018
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.