Makary’s plausible mechanism pathway praised as positive step, criticized for vagueness

FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research Director Vinay Prasad have outlined approval criteria that are intended to make development of ultrarare disease therapies faster and more predictable. To support bespoke, personalized medicines, FDA, they wrote in The New England Journal of Medicine, is “committed to providing regulatory guidance and encouragement, outlining a path to market entry for products where a randomized trial is not feasible.”

Rare disease advocates welcomed the “plausible mechanism” pathway,  even as they noted its lack of specificity...