FDA Padzur Is Not the Problem, The Randomized Trial IS

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  • 06/24/2013
Over at Forbes, Matt Herper's excellent article on cancer drug approvals and the FDA sums has a revealing quote from Richard Pazdur, the director of the Food and Drug Administration’s Office of Oncology and Hematology Products.   Herper writes that Padzur " personifies the tension between the need to get new drugs to patients fast and the competing desire to make sure they are safe and effective first."  

Actually Padzur personifies the problems inherent in what Eric Topol has called the "contrived clinical trial environment."  Here's Padzur's punchline: 

“I am extremely disappointed in the sponsor’s proposed labeling for this drug,” Pazdur said during a long aside quoted in The Cancer Letter. “There is no survival curve in the proposed labeling. There is no hazard ratio— there’s difference in the means and the explanation in one or two sentences confounding by crossover.

“But if this drug is approved, one would have to have a very careful conversation with the patient about this potential negative impact on overall survival. And how would you do this? I’ve been playing this in my mind in several scenarios, and any logical patient that I could think of would say, ‘Doc, if you are so uncertain about this most important endpoint, don’t we have any other drug to use here?’”

If my doctor used terms like survival curves, endpoints and hazard ratios in discussing my cancer treatment, I wouldn't stick around for an explanation.   Why waste months debating such crap when my life is at stake?

The fact that Richard Padzur or any regulator is doing the thinking on behalf of patients underscores just how dysfunctional FDA review has become.  It’s how drugs work in the real world that matters. Yet the most recent data shows that it actually takes cancer drugs more time to get to patients than any other area of medicine. And orphan designation is simply a way to get more patent life, not speed treatments to patients. An orphan designation is associated with more time in development, not less. Why are we still using phase 2 and 3 randomized trials to test medicines where a driver mutation is known? Why are we allowing insurance companies to force advanced stage patients to ‘fail first’ on cookbook treatments when we have can match mutation to treatment? The call for ‘more evidence’ is a deceitful dodge because the ‘evidence’ people like Padzur and others call for is randomized trials where survival curves mean more than real survival.

Padzur is not the problem.  He is obviously a well-intentioned -- and caring -- public servant. Rather he is part of broken system where most patients die in less time than it takes to organize and conduct clinical trials.

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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