Expediting Drug Development — The FDA's New “Breakthrough Therapy” Designation

The FDA's new “breakthrough therapy” designation for investigational drugs adds to the agency's portfolio of expedited programs for serious conditions. The designation requires preliminary clinical evidence demonstrating substantial improvement over existing therapies. Many people with serious or life-threatening illnesses for which there are no satisfactory treatments are understandably eager to gain access to new therapies and are willing to trade off greater certainty about a drug's performance for speed of access. Because the typical clinical drug-development program takes about 7 years, during which a substantial body of safety and efficacy data is generated, the Food and Drug Administration (FDA) has long-standing expedited pathways available for drugs being studied for such illnesses. However, many patients and their advocates continue to believe that clinical development is sometimes prolonged beyond what is necessary. During the . . .