Precise Measurements for Those Severely Affected by Epilepsy
What is an inchstone?
Drug development relies on clinical trials to show clinical efficacy and impact on the intended population. Currently available clinical outcome assessments measure the milestones - rolling over, walking, talking - leaving the smaller inchstones completely overlooked. For profoundly impacted individuals such as those living with Developmental and Epileptic Encephalopathy, we need sensitive, specific measurements beyond what is currently available.
Who are we?
An interdisciplinary, caregiver-led consortium of researchers, clinicians, and industry partners dedicated to removing measreument-related barriers limiting clinical trial inclusion and drug development.
Countless new targeted or gene-based therapies are nearing trials which could be jeopardized by the absence of tools to capture small but significant improvements in populations who routinely fall 3-5 standard deviations below the mean and are not currently measured. With the nearing of clinical trials for many rare epilepsy communities, we began to feel the urgency for outcome measures that would actually measure the smaller improvements our children make over time—the inchstones, not milestones.
The Inchstone Project is a multidisciplinary group of consumers, stakeholders, researchers and clinicians collaborating to accelerate outcome measures development. Our goal is to have FDA approved measures that have been adapted to work for those severely impacted DEEs within 5 years.
