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...

A key driver in biopharmaceutical investment decisions is the probability of success of a drug development program. We estimate the probabilities of success (PoSs) of clinical trials for vaccines and other anti-infective therapeutics using 43,414 unique triplets of clinical trial, drug, and...