Presentation to Outline Innovative Methodology for Developing Indication-Specific Outcome Measures in Rare Disease Clinical Trials
New Haven, CT – October 11, 2018: The cognitive science company, Cogstate Ltd (ASX.CGS) today announced a presentation entitled, “Methodology for Development of Indication-Specific Outcome Measures in Rare Disease Trials: An Innovative Research Approach” will be delivered at the National Organization for Rare Diseases (NORD) Rare Disease Summit in Washington, D.C. The poster will be presented on October 15, 2018 by co-author, Pam Ventola, Ph.D., who leads the Rare Disease and Pediatric Center of Excellence at Cogstate.
The poster will outline the development process for a novel visual analog scale (VAS) for Fragile X syndrome clinical trials. Fragile X syndrome (FXS) is a rare neurodevelopmental disorder caused by a genetic mutation and is associated with intellectual disability, anxiety disorders, behavioral and learning challenges, and various physical disabilities. As in many rare diseases, FXS is heterogeneous in symptomology, which presents significant challenges when developing a clinical outcome assessment strategy to measure treatment benefit and to support drug development. There is a pressing need to create novel outcomes that have improved specificity, assess constructs proximal to the targeted mechanism and include input from key stakeholders.
“In rare disease trials like FXS, input from caregivers, clinicians, and patients is particularly critical in order to better measure the full range of symptoms. We’re thrilled to present this important work for a FXS-specific visual analog scale, and look forward to highlighting how the methodological approach employed can be used as a model for future work in rare disease trials to continue to improve and refine existing outcome assessments and rating scales,” commented Dr. Pam Ventola.
The Rare Diseases and Orphan Products Breakthrough Summit is a multi-stakeholder event co-sponsored by the National Organinzation for Rare Disorders (NORD) and the FDA. It draws together more than 700 participants from across the rare disease community to discuss the most current and urgent topics related to rare diseases and orphan products.
About Pam Ventola
Pam Ventola, PhD, is Senior Science Director at Cogstate and Assistant Professor at the Yale Child Study Center. Dr. Ventola leads Cogstate’s pediatric and rare diseases portfolio, providing Cogstate customers with strategic oversight and expert guidance throughout all stages of their study planning and execution – from endpoint selection, rater training and strategic monitoring to final statistical analysis. As a licensed clinical psychologist, Dr. Ventola’s primary clinical expertise is in pediatric neuropsychology and specifically developmental and genetic disorders. Her research interests involve developmental disabilities, and among her recent research is a study on brain-based mechanisms of treatment response in autism spectrum disorder (ASD). She is also utilizing eye-tracking paradigms as a novel outcome of clinical trials in ASD and is involved in studies on novel pharmacological therapeutics in individuals with ASD. Dr. Ventola received her Doctor of Philosophy in Clinical Psychology from University of Connecticut and completed her clinical training and Postdoctoral Fellowship at the Yale University School of Medicine, Child Study Center. She serves on the editorial review board of multiple academic journals. Specifically, she serves as a reviewer for the Journal of Autism and Developmental Disorders, Journal of the American Academy Child and Adolescent Psychiatry, Clinical Psychology Review, and the Autism International Journal of Research and Practice. Dr. Ventola has authored numerous peer-reviewed manuscripts, book chapters, and scientific presentations.