Califf Favors Pragmatic Trials Over Traditional RCTs To Tackle Gene Editing

FDA Commissioner Robert Califf says pragmatic clinical research within real-world settings is a better alternative to traditional randomized clinical trials in some cases, saying it can address challenges posed by gene editing technologies and biases in clinical trials. According to Califf, pragmatic evidence is necessary for evaluating new technologies like gene editing for rare diseases. With the long-term effects of such interventions, including off-target genetic changes, still unknown, he says cost-effective methods are needed to monitor patient progress well after...