“Large Enough to Be Meaningful” — Who Decides?

As expected (and dreaded, by some), the U.S. Food and Drug Administration has revoked its approval of Avastin for treatment of advanced breast cancer. The Wall Street Journal has reported and editorialized on the event, which came in the form of a 69-page decision by Food and Drug Commissioner Margaret A. Hamburg, M.D.

If you wade through the legalese and medical technicalities all the way to page 38, you will encounter Dr. Hamburg’s crucial conclusion about Avastin’s effects on “PFS.” What’s PFS? It’s an acronym for “progression free survival,” a period of time during which a patient’s disease fails to worsen. In other words, for a patient in a fatal stage of breast cancer, an extra day of PFS = an extra day of no tumor growth.

Back to page 38, where Dr. Hamburg concludes that “the evidence does not show that Avastin has had an effect on PFS large enough to constitute clinical benefit.” The italics are mine—to stress the kind of value judgment Dr. Hamburg is making. What does that mean, “large enough”? According to Dr. Hamburg, it means “large enough to be meaningful to a patient.”

Do you see what’s going on here? Dr. Hamburg, a federal bureaucrat, is claiming a right to decide whether a particular period of delayed tumor growth is “meaningful” to individual patients whom she will never meet, much less examine and treat. In the particular case of Avastin, the latest statistics show that median PFS ranges from 0.8 months to 2.9 months, depending on which study you look at. By putting “median” in italics, I’m stressing that half the women in these studies experienced even longer periods of delayed tumor growth than those deemed not “large enough” to matter. Yet our law permits an FDA bureaucrat to limit these women’s access to medication, simply because an equal number of other women are predicted to benefit less, or not at all.

This is the practical meaning of collectivized medicine, about which I wrote at greater length last year, in a PJMedia op-ed called “The Avastin Travesty”:

But can risks and benefits really be weighed at the level of society as a whole? A society is only a collection of individuals. A society doesn’t enjoy life, or suffer — only individuals do. Metaphors aside, a society doesn’t get sick and die — only individuals do. To appreciate the difference, consider how a rational patient with breast cancer decides whether to undergo drug treatment.

Such a patient weighs (among other things) the statistical likelihood of a favorable result against the statistical likelihood of painful side effects. At all times, her judgment is individual and personal: How will my life improve if these tumors temporarily stop growing? How might side-effects interfere with my enjoyment of life? How much better will I feel if the results are above average — or how much worse, if the results are below average? How much is an additional year, month, or week of relatively normal life worth to me?

The FDA’s experts take professional pride in refusing to allow such individual considerations to influence their decisions. Instead, they float among the statistical clouds, observing that Avastin delays tumor growth by only 3 to 12 weeks on average and that some patients actually get worse after taking the drug. From behind a veneer of scientific respectability supplied by charts and graphs that ignore the individual patient, these experts then ask a question to which no rational answer can be given: What is the meaning to society of one month in an individual’s life?

In the ongoing debate over American medicine, it is crucial to understand how often we are asked to sacrifice the individual’s welfare for the alleged needs of society.