Asking the Right Questions: How the Patient Perspective Impacts the Success of Rare Disease Studies

June 26, 2024
11 a.m. EDT

Rare diseases affect a small percentage of the population but have a profound impact on the lives of both patients and their loved ones. For most patients with rare diseases, the journey to diagnosis is long and the options for treatment are limited. While clinical trials offer hope for addressing the significant unmet need in rare diseases, these studies must be developed with the patient perspective in mind. Asking patients and caregivers about the challenges they face and the expectations they have of study participation is critical for designing and executing successful clinical trials.

This webinar will explore effective strategies and tactics for integrating patient perspectives and priorities into rare disease studies to increase engagement, accelerate enrollment, and optimize study success.   

Topics will include:

  • Unique challenges of rare disease clinical research
  • Methods for building relationships with patients early in development
  • Approaches for seeking and incorporating patient feedback to design relevant and feasible study designs
  • Options for enhancing the experience of study participation


Kris O’Brien

Executive Director, Program Strategy, Rare Disease