PWS Across the Lifespan
This 20-minute video illustrates the complexity and challenges of PWS for the person with PWS and their family.
PWS Clinical Trial Consortium
Bringing patient and caregiver priorities together with research and industry expertise to improve PWS clinical trials and advance treatment development.
About the PWS Clinical Trial Consortium
The Prader-Willi Syndrome Clinical Trial Consortium (PWS-CTC) is a collaborative partnership of researchers, pharmaceutical companies, clinicians, and patient advocacy organizations working together to accelerate the development of safe and effective treatments for Prader-Willi syndrome. Established in 2015, the Consortium helps remove barriers to clinical research by addressing shared scientific, clinical, and regulatory challenges.
The PWS-CTC addresses unmet scientific, technical, clinical, and regulatory needs for PWS clinical trials. Primary objectives of the PWS-CTC include:
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Advancing the understanding of PWS natural history to support therapeutic development
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Developing patient-centric outcome measures to assess treatment efficacy
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Incorporating patient and caregiver perspectives into the benefit/risk assessment of new treatments.
The PWS-CTC is guided by a steering committee with equal representation from industry, academic researchers, and patient advocacy organizations. Members meet regularly to identify priorities, share knowledge, and collaborate on projects that strengthen the PWS clinical trial landscape.
The PWS-CTC meets regularly to discuss the ongoing needs of PWS clinical trials.
Strengthening the Path to Treatment Approval
In 2018, the PWS Clinical Trial Consortium convened a Critical Path Innovation Meeting with the U.S. Food and Drug Administration (FDA) to address the unique challenges of developing treatments for Prader-Willi syndrome. Discussions focused on issues that affect every PWS clinical trial, including meaningful clinical endpoints, patient-reported outcomes, caregiver perspectives, and the behavioral features that distinguish PWS from other disorders.
By working collaboratively with the FDA, the Consortium is helping advance the scientific and regulatory framework needed to evaluate future therapies—not for a single drug, but for the entire PWS field. This meeting exemplifies the Consortium's role in bringing stakeholders together to solve shared challenges, strengthen clinical trial design, and accelerate the development of safe and effective treatments for people with PWS.
Understanding PWS Natural History Through the Global PWS Registry
Completed in January 2024, PATH for PWS was a four-year prospective natural history study involving 700 participants aged 5 and older.
This substudy within the Global PWS Registry generated an unprecedented longitudinal dataset on serious medical events, providing researchers with essential natural history data that has already informed clinical trial design and supported regulatory submissions for new therapies.
Publications from this important study will be available soon.
Publications and Resources
The PWS-CTC has generated a number of publications and resources to support the advancement of PWS clinical trials.
Video: PWS Across the Lifespan
This 20-minute video illustrates the complexity and challenges of PWS for the person with PWS and their family. Complete this short form to access the video.
Understanding PWS
PWS Across the Lifespan (Brief)
This 5-minute video is a shortened version of the full-length PWS Across the Lifespan. It provides a brief description of PWS and the impact it has on the family and caregiver.
Behavioral Features of PWS
This paper presents consensus definitions and descriptions of key PWS behaviors, including hyperphagia, temper outbursts, anxiety, obsessive-compulsive behaviors, rigidity, and social cognition deficits.
Patient and Caregiver Experience
PWS Patient Voices Survey
This survey of more than 750 parents and caregivers of individuals with PWS captures the symptoms associated with PWS that affect day-to-day living, the effectiveness of current medications, and attitudes towards clinical trials.
Caregiver Perspective of Treatment Preferences for PWS
A national online survey of caregivers was conducted to identify caregiver priorities for potential treatment for PWS. The results were published in the Journal of Medical Economics.
High Levels of Caregiver Burden
Caregivers of children and adults with PWS were surveyed using the Zarit Burden Interview to measure the level of burden in caregivers of individuals with PWS, explore the impact of PWS on caregiver quality of life, and assess the ZBI as an indicator of that impact.
Characteristics of Caregiver Burden
This study found that high levels of hyperphagia are associated with increased caregiver burden in PWS and that caregiver burden and hyperphagia scores are stable over a 6-month period. The results were published in PLoS One.
Quantifying the Burden of Hyperphagia in Prader-Willi Syndrome Using Quality Adjusted Life Years
Time trade-off and visual analog scale were used to elicit PWS caregivers’ values for three fixed health states, demonstrating that reducing hyperphagia will reduce the burden of PWS.
'The Cure For Us is a Lot of Things': How Young People with PWS View Themselves and Future Clinical Trials
This study reports the perspective of twenty-one adolescents/young adults with PWS, sharing their thoughts about clinical trials and possible treatment for PWS.
Improving PWS Clinical Trials
Improving the PWS Clinical Trial Experience, Part 1
Results from a Caregiver Survey of PWS clinical trial participant experiences.
Improving the PWS Clinical Trial Experience, Part 2
Recommendations from principal investigators and study site coordinators.
Conducting a Successful Clinical Trial in PWS
This concise resource provides tips for clinical trial sites and study sponsors to help facilitate successful clinical trials for people with PWS.
Health Equity in PWS Clinical Trials
A comparison of PWS clinical trial recruitment with Global PWS Registry demographics, US Census data, and rare disease clinical trial enrollment data from Rett and Fragile X communities.
Development of Clinical Outcome Assessments
The Prader-Willi Syndrome Anxiousness and Distress Behaviors Questionnaire
The PADQ measures observable behaviors that are meaningful to patients and their families and provides a valid and reliable method to assess beneficial treatment effects for some of the most challenging behaviors associated with PWS.
Use of PROMIS and Glasgow Depression Scales
Mean PROMIS Global Health and Life Satisfaction scores in PWS are lower compared to the general population. These validated instruments may be useful for assessing mood and QOL in PWS.
Hyperphagia Scores in Typical Individuals and Those With PWS
This study summarizes HQ-CT Scores from more than 400 individuals with PWS and 600 typically developing individuals aged 5-26.
The Prader-Willi Syndrome Profile
The PWS Profile is a valid, psychometrically sound questionnaire that has already shown responsivity to treatment in a previous clinical trial.
Membership
The PWS-CTC is open to industry, academic researchers, and patient advocacy organizations who are interested in advancing PWS clinical trials.
To join the PWS-CTC or to learn more, email Theresa Strong, Ph.D.