The International Consortium to Advance Clinical Trials for Prader-Willi Syndrome (PWS-CTC) was established in 2015 to encourage collaboration and leverage the expertise and perspectives of stakeholders from industry, academia,  and patient organizations in a pre-competitive space in order to address the unmet scientific, technical, clinical and regulatory needs of clinical trials for PWS.

The PWS-CTC meets annually in person to address the ongoing needs of PWS clinical trials.The PWS-CTC is composed of a steering committee made of an equal representation of patient advocacy groups, academics and industry, and working groups composed of experts from academia and patient advocacy groups. The CTC meets in person annually to discuss the ongoing needs of PWS Clinical Trials.

The Consortium aims to support the development of outcome measures to assess treatment efficacy against hyperphagia and other behavioral challenges, as well as develop rigorous methods to incorporate the patient and caregiver perspectives into the benefit/risk assessment of new treatments.

Current Work of the PWS-CTC

    • Support/Develop clinical trial endpoints
    • Incorporate patient and caregiver perspectives and benefit/risk assessment
    • Supporting patient access of FDA approved therapies

Achievements

The PWS-CTC attends a CPIM with the FDA, November 2018

For more information please refer to the PWS-CTC website: https://www.pwsctc.org/ and/or contact Nathalie Kayadjanian, Ph.D., Executive Director of the PWS-CTC (nathalie.kayadjanian at FPWR.org)