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Improving the PWS Clinical Trial Experience for Families and Clinical Study Site Staff

Learn how the PWS Clinical Trials Consortium is working to improve the clinical trial experience for families and study site staff.

It has been an exciting time for PWS research and clinical trials. The field has been working hard to find effective treatments for the symptoms of PWS to help our children lead more independent and fulfilling lives. In the last several years, there have been more than a dozen clinical trials evaluating potential drugs to treat PWS. 

improving-the-pws-clinical-trial-experience-for-families-and-clinical-study-site-staffTo better understand what is working with respect to how PWS clinical trials are run and where there is room for improvement, last year the PWS Clinical Trials Consortium sought feedback from PWS families who have participated in clinical trials and clinical study site staff regarding their experiences with PWS clinical trials.

For this project, Dr. Lauren Schwartz Roth conducted interviews with PWS study site clinicians and staff. Those who participated in the interviews worked in academic medical centers and had many years of experience conducting multiple PWS clinical trials. For PWS caregivers, an in-depth survey was created to capture the experience of those participating in one or more PWS clinical trials with their child. The goal of the survey was to identify: 1) factors that have the greatest influence on clinical trial participation; 2) what makes it easier for families to participate in trials; and 3) challenges that make participating in trials more difficult.

Data from the clinical trial site staff interviews and the PWS caregiver surveys have been summarized into two documents on improving the PWS clinical trial experience (see below). The interviews and survey revealed several key recommendations to improve PWS clinical trials and better ensure their quality and completion. Additionally, there was good consensus on several prominent issues to improve clinical trial experiences and efficiencies for both PWS families and trial site staff.

Improving the PWS Clinical Trial Experience: Results From a Caregiver Survey of PWS Clinical Trial Participant Experiences

Read the Results from Caregivers

Improving the PWS Clinical Trial Experience: Recommendations From Principal Investigators and Study Site Coordinators

Read the Results from Site Staff

These documents have been shared with pharmaceutical companies and other stakeholders who are currently working or interested in working in the PWS space.

Thank you to all the families and trial site staff who graciously donated their time and effort to participate in this important study!

Note that, with the generous support of Dr. Assumpta Caixàs Pedragós (Professor of Medicine, Endocrinology, and Nutrition, Autonomous University of Barcelona), these documents have also been translated into Spanish. Please contact us for a copy of the Spanish translation.

Topics: News, Clinical Issues, Resource Development