All Times EDT
Keywords: gene therapy, rare disease, drug development, clinical trial
In the development of cell and gene therapies, a small, uncontrolled, and single arm study has often been employed for registration purpose. Reasons include a highly targeted magnitude of treatment effect, often with cure intent, rarity and severity of the disease associated with unmet medical needs, and the practical challenge to randomize and blind. With the need to conduct frequent interim analyses, formal statistical inference becomes a challenge. In this talk, we will discuss a single arm, open label, confirmatory study design with a small sample size, a large targeted effect size, and multiple interim analyses that is amenable to development of a cell or gene therapy. Formal statistical inference is possible with exact tests and a prescriptive procedure to perform interim analyses.