FDA’s Sham Regulations Impede Medical Innovation

In the Wall Street Journal this week, Dr. Scott Gottlieb discussed (gated, by subscription only) a worrying trend in the Food and Drug Administration’s regulation of medical devices. Increasingly, the FDA is demanding that device makers conduct trials of new devices by randomly assigning patients to the new device…or to a sham surgery without the new device.

In the example cited by Dr. Gottlieb, patients were assigned to sham surgery instead of to a real surgery that inserted a device that ablates (destroys) small nerves in arteries, a procedure that reduces high blood pressure. At great expense, these patients’ arteries were cut open, poked, and prodded, but no functioning device was inserted. The point was to determine whether the device actually worked or whether a placebo effect caused a positive outcome.

What is especially unfortunate about this development is that the traditional clinical trial of such medical devices is a “non-inferiority study,” a study in which the new device is tested against an already approveddevice. If the new device is at least as good as the incumbent, it is approved.

It is hard to see how the emerging approach is ethical. Patients who would have received an older, proven device are now increasingly subjected to sham surgeries instead. I have heard anecdotes of surgeons refusing to participate in such procedures.

Patients, payers, and regulators should be thrilled that device makers are content to conduct studies that compare effective devices against each other. In contrast, pharmaceutical companies are very reluctant to conduct trials that compare safe and effective drugs against each other. In the pharmaceutical context, such trials are used not to demonstrate non-inferiority, but to show whether the new drug is superior to the old drug.

In these cases, they are called head-to-head trials, and many self-styled patient advocates have lobbied to demand that drug makers conduct such trials. A few years ago, I wrote an article explaining how head-to-head trials for the purpose of demonstrating superiority are much more expensive and difficult to interpret than placebo trials for drugs. (In the latter, patients in the control group are given a sugar pill.)

How strange then that the FDA seeks to prevent device makers from conducting trials of two competing, effective technologies, in favor of sham surgeries. It appears to be another case of regulatory overreach that puts many patients in harm’s way.

Comments (11)

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  1. Martha B. says:

    What rank insanity to risk surgery with only the potential improvement from a placebo effect. Such practice should be outlawed, not endorsed as fair medical research. Whatever happened to “First do no harm.”

    • jimbino says:

      As a nuclear engineer, I’d love to have the opportunity to test new weapons by dropping real ones on one country and placebo weapons on another, just to see how many people are killed.

      If I get enough discouraged by the medical-drug-hospital-doc complex in this country, I could consider skipping the placebo test.

      • Matthew says:

        This is precisely the same thinking that was involved when deciding to have these “sham surgeries.”

    • Jay says:

      Now its “first make money, then try to do no harm.”

  2. Jim says:

    Random assignment to real surgery or sham surgery sounds kind of unethical, if you ask me.

  3. Thomas says:

    “At great expense, these patients’ arteries were cut open, poked, and prodded, but no functioning device was inserted.”

    This sounds as a very unethical approach to test a device. Surgeries can be very risky, and to give the patient a placebo after a surgery seems to put them in harms way.

    • James M. says:

      The patient is in harms way. I understand that they have to elect to be in these studies and they have the chance of receiving the placebo, but it sounds high risk.

  4. Buddy says:

    “I have heard anecdotes of surgeons refusing to participate in such procedures.”

    Well it is nice to see that at least the surgeons are still ethically sound.

  5. Apple says:

    Randomization is definitely an ideal research design for many sciences. However, applying such experiment to humans seems to be unethical.