If you bought a new toaster, carried it home, plugged it in, and it didn’t work, what would you do? Suppose you purchased a pineapple at the grocery store, sliced it open, and found it was inedible. In both cases you’d seek the same remedy: you’d return the item and ask for your money back. With reputable merchants, the chances are extremely high that you would receive both an apology and your refund. If the merchant wouldn’t return your money, you could likely get the charges reversed anyway if you paid by credit card.
Now let’s look at one more variation on this theme. Imagine that you bought (or had someone buy for you) a product that cost a whopping $88,000. You used it according to the manufacturer’s directions, but not only did this product fail to work, it significantly raised your blood pressure or caused a hemorrhage. You’d want to get your money back for sure. But in this final scenario, you are a breast cancer patient, and the thing you (or more likely your insurance company, or Medicare) paid all that money for is a cancer drug called bevacizumab (Avastin). Genentech, the medicine’s manufacturer, follows a general convention in the industry and offers no refunds if this drug doesn’t work. My question is: why not? Wouldn’t it make sense for manufacturers to refund the cost of uber-expensive drugs that provide no demonstrable benefit to a majority of patients?
The use of Avastin in breast cancer patients has been mired in controversy. The FDA created an “accelerated approval” program in 1992 to speed the arrival of critical drugs onto the market for those with life-threatening diseases, like AIDS or cancer. Avastin, Genentech’s best selling drug, won FDA approval in 2004 for treating metastatic colon cancer and in 2006 for non-small cell lung. It received conditional approval in 2008 for use in breast cancer patients, even though it failed to extend their survival in a clinical trial. The preliminary approval was given with the concomitant requirement that there be a later review to see if the drug conferred either a survival benefit or improved quality of life for patients. The subsequent analysis showed that in this particular disease indication bevacizumab provided neither benefit, and its approval for use in breast cancer patients was rescinded in December 2010. Genentech appealed the decision, but a just completed review by an FDA advisory panel led to the same conclusion. The panel voted unanimously that breast cancer patients should no longer use the drug.
This decision does not prevent doctors from still prescribing the drug “off label” for their breast cancer patients. However, it reduces the likelihood that your insurance company will pay for the drug to near zero, meaning that you will have to pay for it out of pocket. This medicine is not cheap, with doses for the typical breast cancer patient costing $88,000 a year. The practical result is that only the wealthiest patients will be able to afford this (and other) expensive medication for “off-label” treatments.
A recent Wall Street Journal editorial opined that it was bad medicine for the FDA to rescind the drug’s approval for breast cancer patients. This was based on the fact that while bevacizumab demonstrated no overall statistical survival benefits to breast cancer patients, anecdotal reports suggest that a small (but unknown) percentage of patients responded favorably to the drug. I can readily sympathize with patients who may feel that a potential lifeline is being taken away from them. However, the advocacy group Breast Cancer Action agreed with the FDA decision to withdraw the treatment’s approval for this specific disease indication. As their Executive Director, Karuna Jaggar, succinctly stated, “…anecdotes are not science….”. Christine Brunswick of the National Breast Cancer Coalition echoed these comments “The FDA’s decision on Avastin must be based on scientific evidence from well-done trials and cannot be based on any one individual story, no matter how compelling.” The FDA itself was unable to find any evidence for the existence of a population of “super-responders” to the drug.
The problem with the WSJ argument, of course, is that it could just as easily be applied to nearly all drugs. It’s possible (maybe even likely) that you could find a tiny fraction of patients tucked away in the tail end of the bell-shaped curve that might somehow benefit from any rejected medicine if you looked
