The Breakthrough Therapy Designation (BTD) program, launched nearly 18 months ago by the FDA and aimed at expediting development and review of drugs intended to treat a serious condition, has the potential to shorten development time considerably, according to the Tufts Center for the Study of Drug Development (Tufts CSDD), which recently completed an assessment of the BTD program.
The FDA approved only 30% of the first 113 BTD requests (with 60% denied/withdrawn and 10% pending) in the year and a half following the program's 2012 launch. Christopher-Paul Milne, director of research at Tufts CSDD, said early intensive guidance from senior FDA managers, a key element of the program, suggests BTD-designated development programs will likely learn, sooner rather than later, whether BTD products will be successful.
"A key success factor for the program will be whether it serves the goal of helping drug sponsors and the FDA work together to cut development time, while encouraging the utilization of new development tools and methodologies, such as targeted diagnostics and adaptive clinical trial designs," said Milne.
The FDA already has tools to speed drug development—Priority Review, Fast Track and Accelerated Approval—but these programs are limited in their ability to address scientific, regulatory and economic factors dramatically shifting the R&D landscape, he said.
The assessment, reported in the January/February Tufts CSDD Impact Report, found orphan and oncology drugs dominated the first group of BTDs awarded, but Central Nervous Systems (CNS) drugs and diagnostics—which currently represent only 3% and 5%, respectively, of BTD approvals—are likely to benefit from the designation going forward. Also, although Orphan Designation technically is not an FDA expedited program for serious conditions, it often applies to the same investigational compounds as BTD.
By numbers, neurology compounds currently are fourth behind oncology, antiviral and hematology candidates seeking the BTD designation, according to Milne.
A major challenge for the BTD program is whether the FDA can sustain the early momentum. Milne said the application is not “terribly onerous,” so it makes sense for sponsors to apply for drug candidates that might meet the criteria. “The FDA is a little overwhelmed with the degree to which sponsors have responded to this. We may want to look at how well FDA is able to keep up the pace and whether they’re going to put some additional resources into the program,” said Milne, as told to BioWorld Today.
The study, based on Tufts CSDD proprietary databases, FDA publications, Thompson Reuters Cortellis and professional literature, examined BTD determinations made between July 9, 2012 and Dec. 17, 2013.