New US Breakthrough Therapy Designation has potential to shorten drug development time considerably

The recently launched Breakthrough Therapy Designation (BTD) program in the USA, 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, which recently completed an assessment of the BTD program.

While the US Food and Drug Administration approved only 30% of the first 113 BTD requests (with 60% denied/withdrawn and 10% pending) in the year and a half following the program's 2012 launch, Christopher-Paul Milne, director of research at Tufts CSDD, said early intensive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 that 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," Mr Milne said.