Rare diseases are thought to affect up to 446 million people worldwide. With more than 7,000 rare diseases, most of which are genetic, it’s vital all patients can access new and effective treatments.
We know patient recruitment can be challenging even for non-complex trials, but this issue is exacerbated for rare disease studies due to the much smaller patient populations involved. This can lead to lengthy delays in bringing what can be cutting-edge and potentially life-changing therapies to market.
With the global rare disease market expected to be worth more than US$547 billion within the next 10 years overcoming these challenges is critical. This webinar will:
explore some of the patient recruitment challenges in rare disease studies, particularly relating to sickle cell trials
hear from a patient and family member about what it means to live with a rare disease and the barriers they must often overcome to join a trial, and
highlight strategies to accelerate patient recruitment for these specialized trials.
Learning Objective
Patient recruitment challenges in rare disease studies
Challenges in sickle cells studies
Family/Patient perspective
Accelerating patient recruitment in rare disease studies
Speaker 1 – Whitney Taynton – Business Unit Head, Site Enrollment Optimization, Innovative Trials
Speaker 2 – Donna Dail – Director, Patient Recruitment Programs (Rare Disease)
Speaker 3 – Margaret Alege – Patient
