Over the years the criteria for a response to treatment has
eroded changed. We once considered a decrease in size of a tumor of 50% with prolongation of survival a partial response. Then we considered any response as long as the patient lived longer as evidence of a treatment’s efficacy. Now, in the new world of money driven medicine, the benchmark has eroded even further and seems to be anything the manufacturer decides is a response, whether or not the patient lives longer. Medicyically I’d add anything that boosts a profit.
The NEJM (July 14,2011) discusses the recent removal of breast cancer by the Food and Drug Administration (FDA) from the indications for use of Avastin. The article focuses on whether a slowing of progression is adequate for recommending use of a drug. It should also be understood that this drug has been shown to improve survival in breast cancer patients by just 1 or 2 months; and that as new studies accrue the length of the delay in progression (and survival benefit) appears to be less and less; and that this drug costs between $80,000 and $100,000/year, for the drug alone.
Genentech argues that progression-free survival is an acceptable measure of direct clinical benefit in metastatic breast cancer and that confirmatory trials demonstrate that bevacizumab prolongs progression-free survival. Yet the progression-free–survival benefit in confirmatory trials, while statistically significant, was considerably smaller than that seen in E2100. In addition, safety concerns — both new and previously described — have arisen from the recent clinical trials. Genentech further claims that the FDA has switched approval standards for bevacizumab. The agency, however, has consistently maintained that progression-free survival is “not statistically validated as surrogate for survival in all settings” and is “not precisely measured.” Therefore, the FDA has full authority to respond to adverse safety and efficacy data by changing medication labeling.
Medicynical Note: This company obviously doesn’t give a damn whether this drug offers value, or whether the drug has significant benefit for the amount charged for it. Under the guise of offering desperate patients a choice, albeit and expensive ineffective one with significant side-effects, the company’s management is keeping true to its fiduciary responsibility to simply make money for stockholders.
It’s hard to conceive of a worse basis for health care.