Studies Raise Concern Over FDA’s Increasing Use of Expedited Development Pathways

Two new studies point to concerns over the FDA’s increasing use of expedited development and approval pathways — programs that rely on early-stage trials that produce less robust data.

The studies, published last week in the British Medical Journal, raise questions about whether most new drugs are any more effective than existing products or whether they have been adequately assessed before approval.

One study, which evaluated FDA approvals from 1987 to 2014, shows an annual increase of 2.6 percent in the number of drugs qualifying for expedited development and approval programs. The trend is being driven by drugs that are not first-in-class and are potentially less innovative, the researchers say.

The second study shows wide variations in the quality of efficacy evidence supporting FDA approval of 295 supplemental indications for 164 unique drugs between 2005 and 2014.

Nearly all of the supplemental indications that expanded the patient population were for pediatric patients. Thirty-four percent of approvals for expanded populations were based on uncontrolled trials, while 14 percent had no clinical efficacy trials, the study says.

Both studies were conducted by researchers at Harvard’s Brigham and Women’s Hospital. Read them at www.fdanews.com/09-25-15-fdatrends.pdf and www.fdanews.com/09-25-15-fdaefficacy.pdf. — Jonathon Shacat