According to Peter Marks, an official of the U.S. Food and Drug Administration, the agency has to begin employing rapid approval for the advancement of gene therapies for rare diseases.
According to the report, which cited Marks, director of the agency’s Center for Biologics Evaluation and Research, accelerated approval would be especially critical for ultra-rare disorders, for which there aren’t enough patients to conduct placebo-controlled studies.
The story quotes Marks as saying, “When you’re creating a gene therapy for 10, 20 people a year, the assumption that you’re going to undertake a randomised clinical study comes apart very rapidly.