A “breakthrough therapy” user fee might help deter product sponsors from submitting frivolous designation requests with impunity, a cancer expert suggested at the Brookings meeting on the expedited regulatory program April 24.

George Demetri, a professor of medicine at Harvard and senior VP for experimental therapeutics at the Dana-Farber Cancer Institute, said that companies with questionable data might be less inclined to seek designation in the face of a big user fee or a required public disclosure that they sought the designation but were denied.

“In many of these cases, you have to wonder whether it’s just, ‘Let’s do it. There’s no barrier to doing it. It’s secret if we fail,’” Demetri said.

Sponsor “self-control” might be better than a mandatory fee to limit frivolous submissions, CDER’s Moscicki said.

“I think that’s a potential problem,” he said, suggesting that the large volume of requests that FDA denies are potentially getting in the way of its review and approval of other drugs.

Office of New Drugs Director John Jenkins agreed that the current submission process presents little risk for sponsors.

“There’s no cost for submitting a breakthrough therapy request to the FDA. There’s a lot of upside and probably very little downside to submitting and being denied, because it’s confidential,” Jenkins said. “I think that’s led to a lot of the … applications that we receive that in the end are denied.”

A Discussion For PDUFA VI?

The breakthrough program was created in the FDA Safety and Innovation Act, but was not negotiated between the agency and industry as part of the 2012 Prescription Drug User Fee Act V reauthorization. Consequently, the agency did not receive additional, dedicated resources to implement the program.

The program’s enthusiastic reception by industry has led to a volume of requests far exceeding what was anticipated at the time FDASIA was enacted.

Although the Center for Drug Evaluation and Research is denying roughly two out of every three requests, each submission must be vetted by the review division and the center’s Medical Policy Council.

The program’s workload demands have generated the possibility that PDUFA VI talks could include discussion of a fee to fund the agency’s work on breakthrough-related activities.

However, industry and other stakeholders want to better understand whether the program has meant more work for FDA overall, or just more front-loaded work in the development and regulatory process for a given drug, before considering a new fee Also see "Lifting the ‘Breakthrough’ Veil: FDA Increases Transparency To Reduce Futile Requests" - Pink Sheet, 5 January, 2015..

In addition, industry is not likely to embrace the “fee as a deterrent” concept.

One CDER official suggested voluntary action on the part of sponsors, rather than a mandatory fee, might be a more prudent approach.

“We have close to 300 requests I think by now. We know that there’s nothing to lose by asking,” said Richard Moscicki, deputy center director for science operations. “I don’t know that the right answer should be an entry price of some kind. … Perhaps it’s best if there’s some self-control in what is submitted [to] us.”

Moscicki’s comment drew a rebuttal from Janssen Pharmaceuticals Inc. Head of Global Regulatory Affairs Karin Van Baelen. Janssen has had several designation requests granted and one denied.

“I want to assure that we do have a diligent process also in our company because we want to use our own resources effectively, too, and denial would not be success in our case either,” she said.

“What I’ve heard today, which I think is very good, is that we should maybe have some of those pre-brief … conversations at the time of the IND so that we don’t have submissions that are not proving valuable” (see related story,Also see "Unsure About ‘Breakthrough’-Worthiness? Talk To FDA First" - Pink Sheet, 4 May, 2015.).

‘Breakthrough’ Fee Eyed As Deterrent To Frivolous Requests

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