All Times EDT
Keywords: gene therapy, rare disease, data visualization
The patient population affected by a rare disease is typically small and heterogeneous. Clinical trials on a rare disease have their unique features in terms of study design, subject enrollment, as well as data analyses and interpretation of study results. And the demonstration of efficacy and safety in a reasonable degree of assurance in trials for rare disease could be challenging. In this talk, we share our experiences in the statistical review of two gene therapy products that were approved by FDA CBER in recent years.