FDA — VOICES for REASON

A lawsuit between the FDA and a medical technology company, Regenerative Sciences, has been getting attention recently.  The company has pioneered a procedure (Regenexx-C) whereby a person’s own stem cells can be extracted and reinjected into his or her body to help repair damaged joints. The FDA argues that they have jurisdiction in this case because the stem cells are being extracted and manipulated via a technological process. They claim that as a result the stem cells themselves need to be regulated the way other medications are.

People are reacting with outrage, and rightly so, that this is a new expansion of governmental power that puts the FDA in control of what individuals do with parts of their own bodies.

While it’s encouraging to know that some Americans find the FDA’s arguments in this case to be ludicrous and to be a particularly offensive increase in the government’s intrusion into our lives, it’s dismaying to observe that the direction in cases like these always seems to be one of increasing regulatory power, never decreasing.

In the current case, the discussion is focused on whether and where the lines of FDA’s authority should be expanded (not shrunk). Should it be able to regulate drugs but not stem cells used in medical therapies? Should a drug be defined as something chemical and manufactured as opposed to something chemical and natural (such as stem cells)? How much manipulation is required for something to be considered a drug? And so on and so forth.

The answers always seem to push us in the direction of increasing levels of regulation. Once a precedent is set for regulating a sphere of human activity, the default becomes a growth in regulation. The pattern seems to be: if you’re not sure where the line should be drawn, better err on the side of regulation . . . just in case.

How does such a pattern become established? That’ll be the topic of my next post.

image: wiki commons

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.

Image: WikiMedia Commons

Over at the Wall Street Journal online, Gregory Conko has an interesting article about the Food and Drug Administration’s upcoming hearing on Avastin. That’s the cancer-fighting drug that was recommended for disapproval by the FDA’s oncology committee almost a year ago.

Conko, a senior fellow at the Competitive Enterprise Institute, has focused on the really important theme here. Choices about whether to undertake risky, expensive treatments are properly made by the individual patient, in consultation with her doctor, not by compulsion from a government bureau. He writes:

When well-known scientist Stephen Jay Gould was diagnosed with a rare form of lung cancer in July 1982, he was told the diagnosis meant a median survival time of just eight months. His doctor gave up on him. But he lived another 20 years.

“Means and medians are the abstractions,” he wrote in Discover magazine in 1985. “Therefore, I looked at the mesothelioma statistics quite differently—and not only because I am an optimist . . . but primarily because I know that variation itself is the reality.”

Like Gould’s doctor, the FDA and its technocratic supporters are giving up on breast cancer patients because of their slavish obsession with median response rates. Everyone can agree that, on average, Avastin does not extend most patients’ life expectancy. But some patients have responded incredibly well, living years longer than expected. The medical community calls them “super responders.” Statisticians might describe them as “outliers.” But they’re real people, alive because of Avastin.

I had something similar to say in an op-ed a while back, responding to the oncology committee’s finding that Avastin does not “represent a favorable risk/benefit analysis.”

Does that mean the drug fails to help any woman more than it hurts her? Not at all—many individual women benefit from the drug. But the FDA regards such facts as sentimental distractions, to be deliberately ignored when deciding the fate of a drug like Avastin. The FDA’s idea of a risk/benefit analysis deals with health in the aggregate, as revealed in statistics involving large populations, not with the health of individuals.

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?

I’m glad Gregory Conko and CEI are speaking up on this issue. Everyone interested in making sure pharmaceutical companies have the freedom to sell life-saving drugs should follow this controversy closely.

Image: Wikimedia Commons

Richard Pazdur, the FDA’s top regulator of cancer drugs, is busy defending his agency’s decision to rescind approval of Avastin for treating breast cancer. In this Wall Street Journal article, he’s quoted as saying “the drug has a marginal effect on tumor growth.” As I indicate in my Pajamas Media article, he’s talking about the drug’s effect on average.

But as we know, averages are drawn from the experiences of actual individuals, some of whom have a better experience than the average. (And even the average experience, he admits, is positive.) Yet the FDA regards it as its professional duty to ignore every particular individual’s benefit, in favor of a collective decision. (The image accompanying this post is an actual slide from an FDA PowerPoint presentation, showing graphically how the agency conceives its risk-benefit analysis as collective rather than individual.)

Wait, did I say the FDA ignores the patients who benefit? That’s not quite true. Toward the end of the article, Pazdur makes a startling admission. “There may be a subset of women who could be helped by Avastin, but Genentech [Avastin’s manufacturer] needs to figure out who they are,” the article says.

Although the phrasing is cagey, Pazdur clearly knows what the rest of the world is shouting—that some women with breast cancer are benefiting from the drug. Nevertheless, he is perfectly comfortable with the FDA’s decision to withdraw approval of the drug. Why? Because some other women don’t benefit, and Genentech cannot yet scientifically separate the two groups.

It never occurs to Pazdur to question whether one individual woman’s right to undergo a successful treatment should hinge on whether some other women can’t share that benefit. To him and his FDA cohorts, sick people are dots on a statistical curve, and it’s only the curve that matters.

Image: Food and Drug Administration, “Overview and Introduction to Drug Regulation,” Slide No. 5

Last month, the FDA began withdrawing approval of Avastin for treating breast cancer. Over at Pajamas Media, I explained why that violates the rights of sick patients. Now USA Today is attempting to quell the “Outcry over FDA withdrawal of anti-cancer drug Avastin” by painting the drug’s advocates as emotionalist, unscientific crybabies. Here’s the article’s approach in a nutshell:

“The emotional appeal is so compelling,” Fran Visco, president of the non-profit National Breast Cancer Coalition, says of breast cancer patients doing well on Avastin. But, Visco says, “drug approval cannot be based on any individual story. It has to be based on the highest level of science.”

Let’s stop and think about this for a minute. Ms. Visco refers to “breast cancer patients doing well on Avastin.” In other words, she’s talking about actual, living women with a dreadful, fatal disease, who are actually benefiting from the drug. What is Ms. Visco’s attitude toward them? Well, she’s willing to concede emotional sympathy with their anger at losing access to Avastin.

But according to Ms. Vasco, these individual women need to put aside their petty, individual concerns in deference to the demands of science. As she puts it, any given woman’s “individual story” has nothing to do with FDA regulators’ quest to reach “the highest level of science.” What is that “highest level of science”? It consists of statistical studies on large aggregates of patients, studies that deliberately ignore the individual patient in favor of averages.

In my article, I talk about the FDA’s idea that Avastin does not “represent a favorable risk/benefit analysis”:

Does that mean the drug fails to help any woman more than it hurts her? Not at all—many individual women benefit from the drug. But the FDA regards such facts as sentimental distractions, to be deliberately ignored when deciding the fate of a drug like Avastin. The FDA’s idea of a risk/benefit analysis deals with health in the aggregate, as revealed in statistics involving large populations, not with the health of individuals.

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?

The bottom line is that statistical studies are useful tools for individual risk analysis—but when the FDA coercively substitutes that collective data for the individual’s rightful choice, the agency turns that science against those who should benefit from it.

Image: Wikimedia Commons

Early news reports indicate that the Food and Drug Administration is withdrawing its approval for the use of Avastin to combat advanced breast cancer. This, as I argue in my Pajamas Media article on the controversy, is a travesty. Here’s a video version of that article:

Over the weekend, Pajamas Media published my op-ed “The Avastin Travesty.” The timing is good, because the federal Food and Drug Administration is scheduled to vote this week on withdrawing approval of the cancer-fighting drug Avastin, for treatment of breast cancer.

Here’s some of what I said (click here for the full article, which includes an active comment section):

The FDA is slated to decide whether to follow the advice of its own Oncologic Drugs Advisory Committee, which back in July voted 12-1 that Avastin does not “represent a favorable risk/benefit analysis.” Does that mean the drug fails to help any woman more than it hurts her? Not at all — many individual women benefit from the drug. But the FDA regards such facts as sentimental distractions, to be deliberately ignored when deciding the fate of a drug like Avastin. The FDA’s idea of a risk/benefit analysis deals with health in the aggregate, as revealed in statistics involving large populations, not with the health of individuals.

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?

When choosing what medicines to take, each individual patient is entitled to regard his or her own life as the most precious thing in the world.  But the FDA regularly outlaws that kind of individualistic decision-making. Tell me again why we tolerate the FDA?

[Update: Thanks to Andrew Breitbart's BigGovernment.com for linking to my Pajamas Media article.]

Image: Wikimedia Commons