PLX4032 inspires fierce debate over cancer trial designs

Plexxikon's PLX4032 has been the subject of tremendous buzz after the biotech unveiled startling efficacy data from a small trial of lethal melanoma cases. Now its pivotal study is triggering a loud debate inside cancer circles over the ethics of designating a control group when chemo plus placebo amounts to nothing much better than a particularly painful death sentence. And the discussion has hit the front of the New York Times, which chronicles the lives of two cousins who are fighting the disease while taking their places in two different arms of the study.

Inside drug development and regulatory circles, of course, it's well understood that the classic study design requires a comparison of an experimental therapy with standard of care. But doctors who handle these melanoma cases know all too well that they're condemning patients who could be helped by PLX4032, and the knowledge has inspired an anguished response among patients and family members who have had a chance to size up the odds.

"With chemotherapy, you're subjecting patients to a toxic treatment, and the response rates are much lower, so it's important to answer ‘Are you really helping the patient?'" Dr. Charles Sawyers from Sloan-Kettering tells the Times. "But with these drugs that have minimal side effects and dramatic response rates, where we understand the biology, I wonder, why do we have to be so rigorous? This could be one of those defining cases that says, ‘Look, our system has to change.'"

Richard Pazdur, who runs the FDA's cancer drug office, has signaled his willingness to be flexible on the subject. But for dying patients, a flexible long-term approach isn't likely to help.

- here's the story from the New York Times

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