Janet Woodock, MD, Director of the Center for Drug Evaluation and Research at the FDA, recently highlighted how the FDA is placing a high priority on patient input and perspective in the drug development process. The agency recognizes that patients, families, and caregivers are true experts in their disease or syndrome. They have valuable knowledge and information that is being incorporated into the drug review and approval process.
Insights into biggest burdens, priorities for improving quality of life, and risk tolerance are important for accurately representing the the patients perspective when evaluating risk versus benefit of a potential therapy. Members of the FPWR research team were able to see Dr. Woodcock speak at the recent NORD Rare Disease and Orphan Drug Breakthrough Summit in Arlington Virginia.
FPWR is committed to incorporating the patient/family voice into the development of therapies for PWS. There are several recent and ongoing initiatives in this vein. In the summer of 2014, a Patient Voices Survey was conducted to begin quantifying unmet medical needs and preference for drug development. A summary of over 700 responses from the PWS community weighing in on our greatest challenges can be found here. More recently, a "Caregiver Burden" survey aims to capture information about the financial and emotional burden on families caring for a loved one with PWS and a summary of results is in progress. Both of these were conducted in collaboration with PWSA-USA.
The FPWR family conference this fall included a workshop on the use of "Real World Data". This was part of a PCORI award supporting an in-person workshop as well as a webinar on, “Real World Data Research Readiness: Engaging the Prader-Willi Syndrome Community.” The workshop and webinar brings together patient and stakeholder partners in a co-learning environment. Families that participated contributed tremendous insidhe and suggestions to inform the development of real world data research in PWS. Keep an eye out for the webinar version which will be taking place in the Spring!
How else can you make your voice heard? Be sure to participate in the Global PWS Registry! Every story is a key piece to our learning about PWS. The Registry is comprehensive database of individuals with PWS aimed at better understanding the full spectrum of PWS characteristics, expediting the completion of clinical trials, determining areas of needed research and treatments to improve the lives of those affected by PWS.