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PWS at the FDA

On Tuesday, June 17, key leaders and scientific staff members at the FDA took time out of their busy schedules to learn about PWS.

On Tuesday, June 17, key leaders and scientific staff members at the FDA took time out of their busy schedules to learn about the serious challenges of PWS, to hear about our loved ones’ unmet medical needs, and to discuss ways to work with the PWS community to bring the perspective of the PWS patients and caregivers into the FDA’s evaluation of new candidate drugs for PWS. With several drugs in, or soon to be in, clinical trials for PWS (see info from FPWR’s “Clinical Trials webinar”, stay on top of new clinical trials by signing up for our Clinical Trial Alert) it is critical that the FDA fully understand how PWS impacts those with the disorder and their families.  We are indebted to the patient advocates who worked long and hard to ensure that the patient’s perspective is incorporated into the drug review and approval process.  As part of the Prescription Drug User Fee Act V (2012) ,  the FDA has established new programs to engage stakeholders, including the Patient Focused Drug Development Initiative  and the Patient Network. The FDA is thus mandated to incorporate the viewpoint of the patient in considering whether a drug has an acceptable benefit/risk profile for approval.

The level of commitment that the FDA has to this directive was impressively clear in our interactions with them. On May 26, FPWR and PWSA(USA) sent a joint letter to the FDA requesting a meeting with FDA leaders and staff to educate them on PWS and the need for new therapies, and to seek guidance on how best to partner with the FDA to bring the patient’s voice to the agency.  Within one day (and it was Memorial Day!), we received a response and a meeting was quickly scheduled for June.  There was a flurry of activity to get ready on our end, and the National Organization of Rare Diseases (NORD) was a great help in preparation for the meeting,  also assisting with travel and logistics. The meeting at the FDA’s White Oak Campus was attended by approximately 15 FDA personnel, including leaders from the Center for Drug Evaluation and Research, Medical Officers, who are responsible for evaluating new drugs, members of the SEALD team (study endpoint and labeling development), and FDA staff members whose role it is to facilitate patient engagement.

In a one-hour meeting, we covered quite a bit of ground.  Representing the PWS community, Janalee Heinemann, Director of Research and Medical Affairs at PWSA(USA) started off with a succinct overview of PWS, covering PWS symptoms across the years, common medical problems, complications of obesity and the life threatening aspects of the disorder.  Rob Lutz (PWSA(USA) Board member and dad to Isabel, 14) next spoke about his family’s experience.  Isabel is a beautiful teenager who looks like any other child, but Rob spoke of the tremendous lengths to which his family, Isabel’s school and their healthcare providers have had to go to bring Isabel to this point, and the challenges they face every day.  Jim Kane (PWS Research Advocate and dad to Kate, 32) spoke about the limitations his daughter has experienced as a consequence of her overwhelming food drive.  Though bright, articulate and engaging, Kate’s life is consumed with finding food, and that prevents her from doing many things that she is otherwise capable of achieving.  

Finally, I discussed some of the ways we propose to work with the FDA moving forward, including approaches to collect information on the patient/family perspective.  There were insightful questions and discussions about the manifestations of PWS and what kinds of treatment effects would represent meaningful improvements for our kids.  Our ultimate goal is to help FDA personnel understand the unmet medical needs of our population and the potential benefits and impact of new drugs, to assist them in developing benefit-risk assessments that are based on the experience of those who live with PWS every day.

We left the attendees with a comprehensive packet of information on PWS for their reference (a special thank you to Kerry Headley for pulling it all together). Importantly for us, we left with specific ideas on how to engage the PWS community and bring valuable information back to the FDA.  With that in mind, stay tuned for opportunities to have your voice be heard!

Topics: Research

Theresa Strong


Theresa V. Strong, Ph.D., received a B.S. from Rutgers University and a Ph.D. in Medical Genetics from the University of Alabama at Birmingham (UAB). After postdoctoral studies with Dr. Francis Collins at the University of Michigan, she joined the UAB faculty, leading a research lab focused on gene therapy for cancer and directing UAB’s Vector Production Facility. Theresa is one of the founding members of FPWR and has directed FPWR’s grant program since its inception. In 2016, she transitioned to a full-time position as Director of Research Programs at FPWR. She remains an Adjunct Professor in the Department of Genetics at UAB. She and her husband Jim have four children, including a son with PWS.