A Focus on the Patient

 

The Prader-Willi Syndrome Clinical Trials Consortium is committed to engaging the PWS community and incorporating the patient perspective into the development of new therapies for PWS.  We believe the voice of the patient community is critical to understanding the unmet medical needs of this population, establishing priorities and value assessments, and framing the benefit-risk analysis of new drugs. A dynamic dialog between all stakeholders is essential to this process.

 

Active Projects

 

The Impact of PWS Symptoms on Caregivers

This survey of more than 200 parents and caregivers of individuals with PWS is a longitudinal study (0 and 6 months) to examine whether the caregiver`s burden levels can be incorporated in clinical trials as a relevant proxy measure indicative of the treatment outcomes of their child. Several questionnaires are administered to caregivers to assess the degree of hyperphagia of their child, the level of caregiver burden, the severity of symptoms and tasks caregivers are performing, and the impact of PWS on their quality of life.

Completed Projects

Measuring Caregiver Perspective of Unmet Clinical Needs for PWS​

A national online survey of caregivers that helped identify caregiver priorities for potential treatment and quantified caregiver preferences for the benefits and risks of treating PWS. An analysis will be published soon.

PWS Impact Film

The PWS-CTC has collaborated with Partner-Films to produce a short 20-min film to illustrate the complexity and challenges of PWS on the individual with PWS and their families.  Watch the film.

 

PWS Patient Voices Survey 

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 effectiveness of current medications, and attitudes towards clinical trials. 

 

© 2016 PWS Clinical Trial Consortium