Engaging Rare Disease Patients & Accelerating Clinical Development

Rare disease trials differ from those in most other therapeutic areas because the indications are more complex and involve smaller patient populations. But rare disease trials have one distinct advantage: these patients are very vocal, with caretakers, family members, and advocates eager to share their experiences. It is essential to understand the current disease landscape, the prior research, and the competitive clinical trial environment when posi­tioning a new clinical trial within the rare disease space. Drug developers must be aware of the status of the indication and the location of open trials, their phases, and the patients they seek.

In a series of white papers, PRA experts discuss the barriers drug developers face when positioning a new trial in the rare disease space and how to minimize these while engaging with the right patients.