Sunday, August 22, 2010

In Defense of the FDA

Cross-posted from The Agenda on National Review Online.

The Wall Street Journal opinion section, edited by Paul Gigot, is a tremendous resource. For economically sophisticated commentary on every area of public policy, one can hardly do better. Throughout the Obamacare debate, the Journal was particularly strong on health care policy, calling attention to PPACA’s deficiencies as well as to ideas for genuine reform.

Once in a blue moon, however, the Journal strikes a false chord, and such was the case with its recent editorial entitled “The Avastin Mugging.” The subject of the editorial is the recent recommendation, by an external advisory committee of the Food and Drug Administration, to revoke approval of Avastin, a biotech cancer drug, for advanced breast cancer.

The panel’s new recommendation came on the heels of new data showing that Avastin did not extend the survival of patients with advanced breast cancer. (In cancer trials, the length of a patient’s life on a given treatment is the accepted gold standard for measuring a treatment’s effectiveness.) In the Avastin trial, called AVADO, patients treated with placebo and Taxotere, another cancer drug, lived for 31.9 months on average; whereas patients treated with Avastin and Taxotere lived for 30.8 months at a low Avastin dose and 30.2 months at a high Avastin dose. Understandably, the FDA’s advisory committee saw this data as evidence that Avastin didn’t offer a real benefit to breast cancer patients.

The Wall Street Journal saw things differently. Their editorial’s opening paragraph is, unfortunately, a medley of hyperbole:
If there’s an American precedent for the medical central planning of ObamaCare, it’s the Food and Drug Administration. Witness a looming FDA ruling that may deplete the drug arsenal for terminally ill cancer patients.
The FDA has many problems, and I have written about many of them: but to call the agency a “precedent for central planning” is unfair. Every developed country, from the most market-oriented to the most socialized, has a government agency charged with approving new drugs for sale. I realize that hard-core libertarians believe that the FDA’s role could be adequately assumed by privately-sponsored third parties, but it is unfair to say that a free market for pharmaceuticals cannot coexist with a FDA-like regulatory agency.

It is also inaccurate to say that action by the FDA would “deplete the drug arsenal” for cancer patients. Avastin is approved for other diseases besides breast cancer. This is an important detail, despite the Journal’s assertions to the contrary. In the U.S., as in most countries, if a drug is approved for one disease, it can be prescribed by doctors for any purpose whatsoever: lung cancer, breast cancer, headaches, facial rashes, etc. Hence, if the FDA revokes Avastin’s approval for breast cancer, physicians are still free to use the drug for that purpose. No one’s arsenal has been depleted.

The real impact of any such decision by the FDA is on payors: on Medicare and Medicaid, and on private insurers, who will look at the FDA’s decision as a reason not to reimburse for the usage of Avastin in breast cancer. This appalls the Journal’s editors:
So here we have government-anointed medical patriarchs substituting their own subjective view of Avastin’s risks and costs for the value that doctors and patients recognize. If Avastin is rescinded, thousands of dying women will lose more than proverbial false hope in the time they have left. They will lose a genuinely useful medicine.
This, again, is an unfair argument. The people who pay for medicines have a responsibility to ensure that they are spending money on treatments that actually work. We can argue that these decisions are best devolved to individuals rather than governments: but it is irresponsible to claim that these decisions should never be made at all. And to say that “medical patriarchs” are imposing their “subjective view” is a bit rough. Overall survival, whatever its faults, is pretty objective: either the patient lived, or the patient died. There is no room for subjectivity in survival analyses, which is precisely why overall survival is the gold standard in clinical trials.

Ironically, is the Journal that is arguing for a somewhat more subjective standard: that Avastin is “genuinely useful” because it showed an improvement in “progression-free survival.” PFS is a commonly used test that asks not only if patients lived, but also if their tumors enlarged while under treatment, or if their disease worsened in some other way. Here, Avastin performed better: median PFS for the placebo group in the AVADO trial was 8.1 months, compared to 9.0 months for low-dose Avastin and 10.0 months for high-dose Avastin.

Some clinical trial experts believe that PFS is a more useful measurement than overall survival (OS), because the overall survival data can be influenced by treatments a patient gets after she has progressed on the tested therapy (in this case, Avastin). For example, a patient who received placebo and Taxotere in the AVADO trial, and later progressed, could have gotten Avastin as a second-line treatment; whereas patients receiving Avastin and Taxotere in the trial wouldn’t get Avastin a second time. But such an explanation, at this point, is hypothetical. It doesn’t take into account the meaningful side effects that Avastin can cause. And we can’t forget that one of Avastin’s principal side effects is financial: a year’s worth of the drug costs between $80,000 and $100,000.

The Journal, in criticizing the FDA, has misdirected its fire. The real problem is this: if the government is responsible for paying for our health care, the government is entitled, if not obligated, to decide how to spend its money most effectively. If we don’t want the government to be making these decisions for us, then we need to pay for our own medicines. We can’t have it both ways.


  1. Thanks much for this. Just out of curiosity, is there any first world country that does not have drugs approved by some governmental agency before general release? I have always thought this a theoretical libertarian argument, popular amongst the anarcho-capitalists, but not actually practiced anywhere.


  2. Hi Steve, as I write above, no there is not an industrialized country that doesn't have drugs approved by a governmental agency. I have heard cogent arguments (especially from the guys at Cato) as to why we could do without an FDA, but personally I do believe that the FDA has a legitimate role to play.

  3. I couldn't imagine a industrialized or non-industrial country without a governmental agency to oversee the safety of medical drugs created by the pharmaceutical industry. I agree with Roy concerning the FDA, that is, if the FDA was free of corporate influences, although recent history indicates it isn't.

  4. Hi Anonymous,

    If your greatest concern is that the FDA is too influenced by corporations, you have nothing to worry about. If anything, the FDA is too insulated from the real-world costs of its decisions.