The Accelerated Approval pathway at FDA is meant to support earlier approvals and access to treatments when there is a strong unmet need and an accepted proxy for FDA to evaluate a treatment’s safety and efficacy. In return for these earlier approvals, drug companies promise to pursue trials that will confirm the initial proxy used by FDA with a larger follow-up trial.
While FDA would like to see randomized clinical trials in applications for approval, CLL Society noted the challenges with such trials and urged the agency to be pragmatic in getting treatments to patients more quickly.
Specifically, our feedback to FDA noted the inherit difficulty in enrolling CLL patients in trials when approved drugs are already on the market, and the related problem of trial participants often being much sicker by the time they enroll (i.e., multiple relapses). We highlighted the significantly longer evaluation time needed in randomized trials for conditions like CLL with high variability in disease progression and existing treatments that can offer very durable responses, the need for equipoise in all trial arms, and the ethical considerations related to evaluation of clinical benefit with likely crossover considerations.
