ISTOCK, NIKADADue to advances in rare-disease research and individualized cell and gene therapies, there has been a recent crop of treatments approved by regulatory agencies not based on the “gold standard” randomized, controlled, Phase 3 clinical trial. Rather, drug companies are getting products on the market from small, Phase 2 trials with a single arm—those that treat all patients with the experimental therapy rather than having some on a placebo or standard-of-care treatment.
For instance, the FDA recently approved the first two chimeric antigen receptor (CAR) T-cell therapies for patients with certain kinds of blood cancers based on data from Phase 2, single-arm experiments. And data on just 18 patients was enough for the European equivalent of the FDA to approve the second-ever marketed gene therapy for those with a rare and severe form of combined immunodeficiency.
But with small studies come small datasets—and accurate assessments of how well a treatment will work out in the real world can be a challenge to conduct with only a few patients’ results to go off.
“As we are going to be using more therapies that don’t lend themselves to the traditional randomized, controlled trials, we need to have a mechanism ...
