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PWS Clinical Trials Consortium Meets with FDA for a Critical Path Innovation Meeting

The PWS-Clinical Trial Consortium recently met with FDA, to discuss the progress made to overcome clinical trial challenges in PWS. With an increasing number of trials in PWS, it is a critical time for the FDA to fully understand the unmet medical needs of people with PWS.

CPIM_Group2The PWS-Clinical Trial Consortium (PWS-CTC) met with FDA on November 19th for a 90-min session, to discuss the progress made in the last three years to overcome clinical trial challenges in PWS. With an increasing number of clinical trials in PWS, it is a critical time for the FDA and industry partners to fully understand the unmet medical needs and the impact that PWS has on those with the disorder and their families.

The Critical Path Innovation Meeting (CPIM) allows for those involved in drug development (pharma company representatives, patient groups, and university researchers) to meet with the FDA to discuss general issues such as methodology, technology or strategies in drug development, outside of the context of review for any particular medical product. These meetings are a venue to get FDA`s perspective as well as advice on opportunities or issues.

The PWS-CTC CPIM was attended by more than ten FDA personnel, including leaders in the Center for Drug Evaluation and Research, Office of Translational Sciences, Office of Biostatistics, Office of New Drugs, Clinical Outcome Assessment Staff, and the Office of Drug Evaluation II, and twenty-two consortium members including representatives from FPWR, PWSA(USA), IPWSO, industry and academia. Members of the consortium presented an overview of PWS, behavioral complexities, and challenges measuring these complexities in clinical trials.

Major topics of discussion during this CPIM-FDA meeting included the manifestations characteristic of PWS and the understanding of concepts such as hyperphagia, anxiety and rigidity that are commonly used by the PWS community to describe PWS behaviors. The FDA suggested the need for the PWS community to come to consensus definitions of these concepts as they may impact how drugs will be considered by the regulatory agency.

The ability of individuals with PWS to self-report on how PWS impacts their lives was another major topic of discussion. Despite intellectual challenges faced by this population in general, along with limited self-awareness, the consortium felt that the work that it has initiated last year, thanks to the leadership of E. Roof, to document the perspectives of individuals with PWS will help the Agency to have a better grasp on the needs of those with PWS, as well as the capabilities and limitations of individuals with PWS to self-report.

The level of commitment that the FDA showed during the meeting was impressively clear in our interactions with them and their willingness to interact with the Consortium on a regular basis. PWS is on the FDA agenda thanks to the Consortium and the multiple ongoing clinical trials. The core activities of the PWS-CTC, which have centered on the development of measures to best assess the efficacy of a treatment in clinical trials (what we call endpoints) and on studies to measure the views of patients and caregivers, are aligned with the FDA's patient-focused drug development mandate. Three years after its creation, the Consortium is delivering results as a unique collaboration between experts from industry, academia and patient representatives. The meeting with the FDA was also a great opportunity for the different stakeholders of the consortium to come together as a unified voice in the dialog with FDA to explore ways to leverage its work and ultimately benefit the entire PWS community. 

About the Prader-Willi Syndrome Consortium (PWS-CTC)

The PWS-CTC was formed in July 2015 to encourage collaboration and leverage the expertise and perspective of stakeholders from industry, academia, and patient organizations. The consortium aims to address the unmet scientific, technical, clinical and regulatory needs of clinical trials for PWS.

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Topics: News

Nathalie Kayadjanian


Nathalie Kayadjanian, Ph.D is an expert in translational biomedical research. A neuroscientist by training, she has extensive R&D experience in academia, biotech, and the pharmaceutical industry in Europe and the USA. Nathalie has occupied top management positions in patient-driven non-profit organizations, developing and implementing strategies to accelerate the development of innovative therapies for rare diseases.