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Bloomberg Law – FDA Cancer Head Defends Path Used for Biogen Alzheimer Approval

Bloomberg Law – FDA Cancer Head Defends Path Used for Biogen Alzheimer Approval

The FDA’s cancer chief pushed back against criticisms over the agency’s expedited approval mechanisms, calling them one-sided arguments that fail to recognize the benefits of making potentially lifesaving treatments available more quickly.


“Do you think accelerated approval is under attack? I do,” Richard Pazdur, director of the Food and Drug Administration’s Oncology Center of Excellence, said Thursday at a Friends of Cancer Research online event.


Pazdur’s remarks follow the FDA’s controversial decision to grant accelerated approval to Biogen’s Alzheimer’s drug. It prompted the resignations of several scientists on the agency’s advisory panel, which said there was incomplete data on the drug’s effectiveness. The decision also sparked requests from congressional committees to turn over documents and other backlash about the use of the pathway.


Most of the drugs processed under accelerated approval programs, around 85%, are in oncology. and Pazdur has long defended them against criticisms that they rely on less rigorous evidence.


“There is room for improvement, but we really have to take a look at a comprehensive viewpoint of the accelerated approval program here. And we’ve had over 155 accelerated approvals over the past 20 years, but only about 10 of these indications have been removed from the market,” he said.


Programs such as accelerated approval and breakthrough therapy aim to speed up access to treatments for unmet medical needs. Both Pazdur and Ned Sharpless, director of the National Cancer Institute and former acting FDA commissioner, acknowledged the need for changes to the accelerated approval program but said they’ve offered lifesaving cancer treatments to patients.


“Trust me for cancer care, it has really been transformative,” Sharpless said. “Rick and I are old enough to remember the battle days when patients couldn’t get access to drugs like Herceptin. Women literally chained themselves to Genentech campus” to get that therapy.


“We don’t want to go back to those periods where we have these active agents that we can’t get the cancer patients in a timely manner because of regulatory hurdles,” he said.


Improving Expedited Review

Cancer deaths have declined for decades according to the NCI’s annual cancer statistics program, and that pace has picked up significantly in the past few years-in part due to programs like accelerated approval that have led to widespread dissemination of cancer drugs, Sharpless said.


Rep. Diana DeGette (D-Colo.) indicated ways to improve accelerated approval may appear in Cures 2.0, the follow-up to the landmark 21st Century Cures Act biomedical innovation law (Public Law 114-255). She and Rep. Fred Upton (R-Mich.) released a draft Cures 2.0 bill in June and are hoping to pass legislation this year.


“We need to make sure that we preserve all of our gold standards in this country with the expedited review, the safety and efficacy,” DeGette said at the Friends event on Cures 2.0. “That’s some of the debate we’re having right now. But people need to remember” that patients have really benefited from lung cancer drugs and other treatments made available through the program.


Potential improvements include addressing some of the difficulties the FDA has faced when a company doesn’t meet the primary outcome in clinical trials for why someone would want to take its drug, the standards of medicine have changed, or there are other drugs that address the unmet medical need, Pazdur said.


Another approach is to develop a plan earlier in the drug development process for what the accelerated approval studies should look like, he said.


Friends plans to hold a virtual event next month on breakthrough therapy designation, a faster approval arm that sprouted from one of the advocacy group’s events a decade ago.