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Inside Health Policy – FDA Seeks To Standardize Breakthrough Pathway, Urges Sponsors To Talk Early With Payers

Inside Health Policy – FDA Seeks To Standardize Breakthrough Pathway, Urges Sponsors To Talk Early With Payers

FDA is looking to standardize how it chooses drugs for the breakthrough approval pathway, and an early analysis of the handful of drugs chosen shows the most important criteria is that the therapy show a “spectacular advance in the field” over the typical improvement in drug development, drug center chief Janet Woodcock recently told an industry audience. Drugs selected also tended to have a genetic component and had marginally more people enrolled in trials than those rejected, she said.


Woodcock also stressed that ensuring payer coverage of breakthrough drugs, which have tended to carry high pricetags, is the responsibility of the companies and urged sponsors to start negotiating with payers prior to their product’s approval.

Woodcock shared observations of an FDA evaluation of breakthrough drugs at a Friends of Cancer Research and Brookings Institution conference on clinical cancer research held Nov. 21.

“What we are working on is looking at some ways to make these things more standardized,” said Woodcock.

Another FDA official said earlier in the month that the agency might not be able to maintain its “all hands on deck” approach as the number of breakthrough designations increase. FDA is collaborating with the Brookings Institution to host a public workshop next year, which will include case studies of drugs that did and did not receive breakthrough designation.

Breakthrough designations are granted to drugs that show high treatment effect early in clinical trials and provide expedited developments and reviews.

Woodcock said that while it is hard to describe the threshold a drug has to cross in order to be considered a breakthrough, it has to be greater than the general level of improvement.

“We have had people send in requests where the magnitude of improvement is about 10 percent,” she said. “That is typical in drug development… If that’s the general level of improvement with most therapies in that disease, then we’re not going to say it’s a… potential breakthrough.”

Woodcock also said therapies with a genetic component in their indication were more likely to be granted breakthrough designation, but orphan and rare disease status did not make a difference on whether a therapy was granted or denied status.

Treatments that received the status also had marginally more trials and more patients than those that were denied, she said.

Woodcock noted that if a breakthrough request had a small sample, the agency would not know if the sample comprised a highly selective group of people that would react to the treatment and, as people were added, there would be a regression in drug development effectiveness.

She also said coverage of the treatments that go through the breakthrough pathway falls upon the companies to reach out to payers and begin negotiating.

“We don’t nor are we supposed to know what the company is going to price the drug for… It’s really up to companies to go to payers and say, ‘We expect approval of this [drug]’ and start negotiating,” said Woodcock.

This comes as patients and payers are trying to find ways to handle the high price of specialty drugs, including the hepatitis C drug Sovaldi.

Recently, the American Academy of Actuaries suggested policies to improve state Medicaid officials’ handling of the approaching slew of specialty drugs, including use of risk-mitigation strategies already used in Medicare Part D. Actuaries said they are struggling with setting capitated Medicaid rates because of the accelerated reviews of breakthrough drugs. Since these drugs tend to be expensive the approach of using historical experience to set rates does not work, they said.…