Child Study Center Grand Rounds: "Improving Cognitive and Behavioral Endpoints in Pediatric and Rare Disease Clinical Trials"

Current prevalence estimates indicate that there are over 6,000 rare diseases with over half affecting children. The vast majority of rare diseases have no effective treatment. To combat this global health crisis and accelerate the process of drug development for the 350 million people living with rare diseases worldwide, regulatory agencies have expedited approval processes for drugs targeting rare indications. As such, clinical trials in rare diseases, as well as in pediatrics as a whole, are growing rapidly. For these trials to succeed, they require objective endpoints, that are sensitive to detecting therapy-related change, relevant to the indication and appropriate for use in children