New study of FDA cites the flaws behind rapid-fire cancer drug OKs

Richard Padzur

Over the past few years the FDA’s oncology group under Richard Pazdur has truly revolutionized the way cancer drugs are developed and reviewed for marketing approval. In order to ramp up the arrival of new drugs, regulators waived old rules on overall survival, flagging their willingness to carve years out of the R&D process in order to field new therapies.

It was a straight up trade off. The FDA was willing to accept preliminary evidence of efficacy and safety and delay confirmatory studies until after a drug was on the market. And patient groups have cheered the FDA on at every turn.

But there is another perspective on how this is all playing out, now that we’re a few years into the brand new game of cancer R&D.

A group of researchers, including some familiar FDA critics, took a look at 22 drugs that were given an accelerated approval. Three years after approval, only half of the 38 confirmatory studies required by the FDA had been completed, and most still used endpoints that had strayed far from the old gold standard on randomized survival results.

Huseyin Naci

The study, published by the Journal of the American Medical Association, was done by Huseyin Naci, Katelyn Smalley and Aaron Kesselheim, a policy expert at Harvard who’s been repeatedly critical of the FDA’s approval process.

“Drugs granted Accelerated Approval should be rigorously evaluated using convincing patient-centered clinical outcomes in rigorous studies,” Naci told Reuters. But, he added, “we have found numerous situations in which required confirmatory studies with rigorous designs and outcomes are not pursued or are not completed in a timely fashion, and in these cases, we are concerned that regulators appear to accept data that would not otherwise meet FDA standards.”

Don’t look for any sudden toughening of cancer R&D standards at the FDA. As Scott Gottlieb was under review for the top job at the agency, he heralded the cancer group’s work in finding new ways to approve oncology drugs faster than ever before, a game-changer in the field made possible for drugs that are typically initially used as a last resort among drug-resistant, dying patients.

The emphasis at the FDA is to make the rest of the agency more like the oncology organization.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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