New Cancer Drug Approved, But Does It Prolong Life?

The New York Times reports this morning on the FDA's approval of a new cancer drug based on data showing it shrinks kidney tumors. This is one of the first fruit's of the new regime at the FDA, which is now headed by National Cancer Institute director and Bush family friend Andrew von Eschenbach.

As the story points out, the FDA does not know if Bayer's Nexavar will prolong patients' lives. European regulators have taken a tougher stance, requiring that there be actual medical benefit from a new drug before giving manufacturers the right to sell it.

The evidence offered in the story that Nexavar will prove beneficial was encouraging, and God knows, we need new and better cancer drugs. I just learned last night that a dear friend is dying of pancreatic cancer.

But reporter Gardiner Harris failed to ask a number of critical questions. Will the Center for Medicare and Medicaid Services (CMS) require that every patient who gets Nexavar be enrolled in a clinical trial so that we'll get confirmatory data that the drug actually benefited patients (and didn't just give them encouraging x-rays with shrunken tumors)? CMS director Mark McClellan has said that the agency will demand that patients enroll in such trials as the price for the government paying for experimental cancer drugs. Even if it has been approved by the FDA, any drug that has not yet shown that it prolongs life should still be considered experimental.

Also, did the FDA require the company to complete its own study to see if the drug actually prolongs life? A recent FDA Oncology Drugs Advisory Committee heard testimony from at least six drug manufacturers who failed to complete promised trials after their drugs had been approved on an accelerated basis. The drug industry has been notorious for failing to follow through on promises made during the accelerated approval process.

Finally, will the FDA pull the drug off the market if the data from trials -- assuming they are required -- show no life-prolonging benefits from Nexavar? Harris should have gotten the FDA on the record on that critical question. If the agency is going to start rushing drugs to market based on surrogate markers of health benefit, then the least it can do is promise to remove them if they later prove useless.