Bad News On December 14, 2012, the US Food and Drug Administration granted an accelerated approval to the drug Ponatinib, which is used to treat patients with serious and life threatening forms of leukemia. Unfortunately, the risks associated with the use of the drug have proved to be far greater than anticipated. Thus on October 31, 2013, the FDA switched course and asked the manufacturer of ponatinib, Ariad Pharmaceuticals of Cambridge Massachusetts, to suspend marketing and sales of the drug pending the FDA’s further evaluation of the potential costs and benefits of ponatinib’s use. The FDA wants to conduct that study so that it can better determine which patients are the ideal targets for the drug, and which patients are most likely to succumb to its grave negative side effects, “the risk of life-threatening blood clots and severe narrowing of blood vessels.”
Accordingly, the ever-prudent FDA immediately recommends the adoption of a three part program. First, no new patients should begin treatment with the drug. Second, those who are using the drug who are not responding to it should immediately cease use. And third, those patients who are responding favorably to the drug should be allowed to continue its use if their health care professionals believe that the potential benefits from the drug use justifies their filing for a “a single-patient Investigational New Drug (IND) application or expanded access registry program while FDA’s safety investigation continues.” Not surprisingly a recent New York Times Editorial takes the view that the FDA has set up the right process for dealing with this problem by allowing physicians and other health care professionals to go through a “streamlined” process to continue drug use where they think that the anticipated benefits exceed the anticipated costs.
Whose Cost Benefit Analysis? Both the FDA and the Times have done the wrong cost benefit analysis. For starters, all the information about the adverse effects of ponatinib are in the public domain, available to all professionals who prescribe the drug. If the FDA had done nothing other than republish the conclusion of the recent studies, we can be confident that the serious risks associated with its use would quickly work themselves through the system. The amount of the drug prescribed for established and new patients would drop, given the increased revelation of the risk. Higher cost means lower use.
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