Thanks to a combination of scientific advances and economic incentives, the development of therapeutics to treat rare or “orphan” diseases has grown dramatically in recent years. With the advent of FDA-approved gene therapies and the promise of gene editing, many experts believe we are at an...

In the midst of epidemics such as COVID-19, therapeutic candidates are unlikely to be able to complete the usual multiyear clinical trial and regulatory approval process within the course of an outbreak. We apply a Bayesian adaptive patient-centered model--which minimizes the expected harm of...