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Pink Sheet – Accelerated Approval Is For Patients, Not Sponsors – US FDA’s Pazdur

Pink Sheet – Accelerated Approval Is For Patients, Not Sponsors – US FDA’s Pazdur

The US Food & Drug Administration oncology drug review team is getting a lot of attention for cleaning up accelerate approval indications after confirmatory studies fail.

But the agency is also drawing some lines in the sand when it comes to granting Accelerated Approval in the first place. In 2021 – in the midst of a wide-ranging review of so-called “dangling” AA indications for checkpoint inhibitors – FDA used an advisory committee review of Incyte Corporation’s retifanlimab to deliver a broader message that single-arm trials are not generally going to be acceptable for accelerated approvals in the class going forward. (Also see “Incyte’s Retifanlimab Tests US FDA’s Tolerance For Low Response Rates In Accelerated Approval” – Pink Sheet, 24 Jun, 2021.)

More recently, FDA used an attempted Accelerated Approval application from Spectrum Pharmaceuticals Inc. for the lung cancer therapy poziotinib to highlight its efforts to encourage better dose optimization work and its expectation for confirmatory trials to be well under way prior to approval. (Also see “Poziotinib Fails To Get FDA Panel Support Though Members Not As Dubious As Agency” – Pink Sheet, 22 Sep, 2022.)

FDA followed up both reviews with “Complete Response” letters to the sponsors. In the latter case, the letter also prompted Spectrum to announce it would “deprioritize” the further development of poziotinib and lay off 75% of its staff. (Also see “Spectrum Sees Potential Path For Poziotinib In NSCLC Despite Expected CRL” – Scrip, 28 Nov, 2022.)

That prompted headlines about FDA “blowing up” Spectrum.

Don’t expect those headlines to change anything: The leader of the agency’s oncology team, Oncology Center of Excellence Director Richard Pazdur, is eager to remind sponsors that accelerated approval is not supposed to be a reward or incentive for them – but rather a pathway to serve patients with unmet medical needs.

Pazdur reiterated his views during the recent Friends of Cancer Research annual meeting, after a panelist, Mass General Cancer Center’s Keith Flaherty, commented from the podium about the challenges of smaller biotechs in raising funding needed to conduct confirmatory trials. Flaherty argued that undercapitalized sponsors may need to “pause” development to get the funding to continue. “That’s not the end of the world, if that were to happen, but it’s not a fast process.”

Pazdur took issue with that characterization.

“I’d like to remind people that the whole idea behind accelerated approval was a patient-centric approach. It was not, not, not – can I make this any clearer? – an incentive program for the pharmaceutical industry,” Pazdur said.

“Somehow this got perverted along the years as an incentive program to do less. No. What we are asking for here is a randomized study somewhere down the line.”

Speaking to smaller, “undercapitalized” sponsors, Pazdur said: “If you don’t have the ability to do a randomized study, you should be really questioning whether you should be in the game. This is not a big hurdle to overcome to do a randomized trial.” (Also see “US FDA Says Confirmatory Trials Should Start Before Accelerated Approval But Is Not ‘Dogmatic’” – Pink Sheet, 30 Nov, 2022.)–US-FDAs-Pazdur