By ALAINA BUSCH

FDA granted another breakthrough drug designation, bringing the total to two,

while also denying one of the seven designation requests received since the new program was enacted in the FDA Safety and Innovation Act, agency officials said this week. FDA officials emphasized the need for designation requests to include clinical data, a requirement that distinguishes the pathway from the fast track program, they said.

The drug center’s medical policy council is reviewing the incoming requests for the new designation to apply consistency across review divisions, said John Jenkins, director of the drug center’s Office of New Drugs. So far, the agency has received seven designation requests, granted two, and four are pending. It is unclear which companies have received the designation as FDA officials cannot disclose the information and the applications have not been publicly announced.

Jenkins said the two granted designations had phase two clinical trial data, but the agency is still working through trial requirements for the designations. “You could imagine it being as early as very impressive phase one data,” he said.

While not getting into specifics about the one denial, Bob Temple, the drug center’s deputy director for clinical science, emphasized that breakthrough candidates must have clinical data.

“The test for breakthrough there has to be…clinical evidence, early clinical evidence, some clinical evidence,” he said at the recent FDA-CMS Summit. It is distinguished from fast track where companies can “get in just if you’ve got a good idea.”

With breakthrough there should be a clinical marker, he said. “And if we didn’t agree, it would be because we didn’t think the clinical marker was valid or we didn’t think it showed it,” Temple said.

Because breakthrough is determined further on in the development process, the designations could hold up better than fast track, Jenkins said. “So maybe those will hold up a little better than fast track because fast track is almost based on a promise that you can meet an unmet medical need,” he said.

The agency is also working on guidance to harmonize terminology and distinguish among the varying expedited pathways such as breakthrough, fast track and accelerated approval. Jenkins said the document is in the works and the agency is aiming to meet statutory time frame for issuing the breakthrough guidance, within 18 months of enactment.