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What Does Effective PWS Treatment Advocacy Look Like?

The FDA is calling for PWS patient experience data. FPWR is ready with that data because of its work since 2008 with parents and other groups.

Last fall, the PWS community was excited to see two drugs complete Phase III clinical trials: both DCCR and Carbetocin hold promise in addressing hyperphagia, a top priority for PWS families. Both studies had positive results, but neither reached statistical significance for their primary endpoint: change in hyperphagia. While we are optimistic that these drugs could bring meaningful benefit to our loved ones with PWS, their path to approval will likely require many conversations with the FDA before they can submit their New Drug Applications.

With your support, we have been laying the groundwork to facilitate new drug approvals. For more than 10 years, FPWR has been conducting surveys, establishing a robust registry, and funding research into patient preferences so that we can effectively advocate for new treatments for PWS and bring the caregiver and patient voice to the FDA.

Below, we'll describe what we've been doing to engage in effective PWS treatment advocacy.

How We've Been Preparing for Today's PWS Advocacy Needs

Over the past decade, FPWR has partnered with families and other PWS groups (PWSA|USA, IPWSO) and worked diligently to build resources that address the FDA’s call for patient experience data, demonstrating high disease burden and the need for effective therapies addressing hyperphagia. We've conducted surveys, established a robust online patient registry, and funded research into patient preferences so that we can effectively advocate for new treatments for PWS and bring the caregiver and patient voice to the FDA.

PWS Advocacy Activities (5)

While FPWR doesn't highlight its advocacy efforts as often our research programs, it’s important to note that it is a part of FPWR's ongoing strategy to bring treatments to our loved ones with PWS. In order to achieve optimal effectiveness, advocacy for new treatments for PWS must happen at every stage of the drug development and approval pathway. This means working with pharmaceutical companies to help them understand the medical needs of the PWS community and the aspects of PWS that families want to see treated. It also requires establishing a strong relationship with the Food & Drug Administration (FDA), whose job it is to determine if a drug is safe and effective for the PWS. 

The Right Tools for the Job of PWS Treatment Advocacy

This past spring, in a community response letter to the FDA, our library of nearly one dozen resources was shared with the FDA to support regulatory flexibility when reviewing new drugs for PWS. These resources, which your donations have directly funded, include:

  1. PWS Across the Lifespan Video: This 20-minute film available in English, Spanish, and French illustrates the complexity and challenges of PWS on the individual with PWS and their families.
  2. Impact of PWS on Individuals and Their Families and Views on Treatments: This survey of more than 750 parents and caregivers of individuals with PWS captured the symptoms associated with PWS that affect day-to-day living, the impact of PWS, the effectiveness of current medications, and attitudes towards clinical trials.
  3. Caregiver Priorities for Endpoints to Evaluate Treatments for Prader-Willi Syndrome: A Best-Worst Scaling: Findings from this report indicate hyperphagia is “the” aspect of PWS that families want addressed through new therapies, followed by other critical behavioral issues such as anxiety and aggression.
  4. High Levels of Caregiver Burden in Prader-Willi Syndrome: Results from this survey show caregiver burden in PWS exceeds that of caregivers for stroke and Alzheimer's disease.
  5. Characteristics and Relationship Between Hyperphagia, Anxiety, Behavioral Challenges, and Caregiver Burden in Prader-Willi Syndrome: This paper demonstrated that higher levels of hyperphagia in individuals with PWS are associated with increased caregiver burden.
  6. PWS Caregiver Risk Tolerance: Academic Perspective on Patient Preference Research: This publication establishes that caregivers are willing to accept considerable risk in exchange for even modest improvements in hyperphagia.
  7. Measuring Meaningful Benefit-Risk Tradeoffs: Coming soon
  8. Patient Voice: Coming soon
  9. Natural History Study of Serious Medical Events in PWS (PATH for PWS): A four-year, ongoing, natural history study to help us better understand serious medical events in people with PWS ages 5 and up.
  10. Behavioral Features in Prader-Willi Syndrome (PWS): A consensus paper from the International PWS Clinical Trial Consortium: Submitted/in revision
  11. Impact of COVID on the PWS Community: The Global PWS Registry launched a survey in summer 2020 to capture the impact of COVID-19 on PWS families. We found COVID-19 had a profound impact on people with PWS which likely impacted the conduct and outcome of PWS clinical trials taking place during that time.

Your support makes a direct impact on our ability to support research that discovers new treatment approaches, advances clinical trials, and advocates for new treatments for PWS. We thank all of you who have made this work possible!

Learn more about advocating for new treatments for PWS as well as steps you can take to get involved in our advocacy efforts >>

Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.