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.
Good Clinical Practice: A PRA Perspective
Improving Treatment for Rare Cancers
In recognition of World Cancer Day on February 4th, we explore questions around the latest rare cancer research, treatments, and how to improve…