The jury's still out on the FDA's 'breakthrough' designation

The FDA's much-heralded breakthrough therapy designation (BTD) program has been up and running for more than a year, and while the industry's excitement has yet to wear off, it remains to be seen whether the initiative will be the boon drug developers expect, researchers say.

According to a report by the Tufts Center for the Study of Drug Development (CSDD), the FDA approved just 30% of the 113 BTD applications it has received, with 60% denied or withdrawn and another 10% still pending. Three of the smiled-upon therapies have since been approved--Roche's ($RHHBY) Gazyva, Johnson & Johnson ($JNJ) and Pharmacyclics' ($PCYC) Imbruvica, and Gilead's ($GILD) Sovaldi--winning nods between one and three months ahead of schedule.

But despite the still-fresh optimism over the program's promise, no one's quite sure exactly what effect the BTD will have on development timelines. Those expecting a string of regulatory green lights out of the program are likely mistaken, as the FDA's main promise is early intensive guidance from decisionmakers, access to key agency personnel and a collaborative spirit, Tufts CSDD Research Director Christopher-Paul Milne said.

"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," Milne said in a statement.

Tufts researchers figure it'll take years of data before anyone can make an authoritative ruling on the program's benefits, but the promise of earlier access to lifesaving treatments was enough to spur rumblings of a similar effort in the U.K. Under a "promising innovation medicine" proposal making its way through the British government, U.K. regulators would consider licensing drugs for marketing in the country and making them available to National Health Service patients before they're approved by the European Medicines Agency.

To date, the chief beneficiaries of the BTD have been orphan drugs, oncology treatments and anti-infectives, but Tufts expects central nervous system therapies and diagnostics to eventually find their way into the FDA's good graces.

- read Tufts' release
- check out the report (sub. req.)

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