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.

Our presenters will be: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).

Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments

What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.