Keywords: rare disease, autologous, gene therapy
Gene therapy products have been recently in rapid development for many diseases including rare diseases which have been challenging in product approval from many aspects including small patient population size and assessment tools (or endpoints) for clinical benefits. Many drugs currently in the market for rare diseases are at the best for controlling or/and delaying symptoms. They are however not the remedies for root cause of diseases. As many rare diseases have identified genes responsible, there has been a high hope and belief that gene therapies with gene editing, replacement or deliver of a new gene can effectively treat and even cure diseases. Depending on how gene therapy products are manufactured, the features of product manufacturing can affect how clinical trials are designed and analyzed. In this talk, I will present regulatory experiences and statistical considerations in the development and approval of gene therapies as well as guidance in development followed by several examples to illustrate our experiences and considerations.