Authored: Scott Schliebner, SVP, Centers for Rare Diseases & Digital Therapeutics,
PRA Health Sciences
According to Global Genes, the 7,000 distinct types of rare and genetic diseases impact more people than cancer and aids combined. PRA Health Sciences understands that rare disease clinical development programs are unique, and to be successful, should be approached from an equally innovative perspective. PRA Health Sciences’ Center for Rare Diseases believes that by working directly with patient advocates offers us the opportunity to better understand the needs of the rare disease community and how we can transform clinical trials to be more effective and accessible for patients. We are passionate about the role that patients can play to help better design, enhance, and optimize rare disease clinical trials.
The Center for Rare Diseases believes that by partnering with patients, clinical trials are designed and executed more efficiently and with a focus on the items that are most important to patients. To help achieve this, we formed the Rare Disease Advisory Committee (RDAC), an independent, autonomous group of rare disease patient advocates who are committed to advising and working with PRA to improve the way patients are integrated into the clinical development lifecycle. The RDAC will examine and explore ways to optimize the drug development process by involving patients, thereby accelerating clinical development and bringing new therapies to patients faster. Central to this effort will be a focus on how patients and patient advocacy organizations can be impactful partners throughout the entire drug development lifecycle.
The Committee’s initial two focus areas are on (1) developing a Rare Disease Drug Development Patient Engagement Roadmap, and (2) developing a Rare Disease Clinical Trial Engagement Burden Assessment. We will also be focusing on how patients, and patient advocacy organizations can be made partners throughout this entire process. We are excited about the efforts of the RDAC and are committed to bringing new therapies to patients faster.
To learn more about the Rare Disease Advisory Committee (RDAC), please visit https://prahs.com/raretogether
Members have an opportunity to share educational information and new initiatives being introduced about clinical research to patients, caregivers, advocacy organizations and industry. To submit a brief article for consideration, please contact Joan Chambers, jchambers@ciscrp.org
