Presenters: Chenxuan Zang, Shihua Wen, Gerry Downey
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.