Off-Label Drugs

Gregory Conko and Henry I. Miller explain the issues surrounding off-label prescriptions.

Why You Should Care about Off-Label Drugs:

Before a drug can be sold in the U.S., it must be certified by the FDA as safe and effective for a specific, or “on-label,” use; but traditionally, once it has been approved, physicians may legally prescribe it for any other purpose. On the basis of articles in medical journals and information gleaned from conferences, doctors prescribe this way more than 100 million times a year. By some estimates, at least 20% of all prescriptions written are off-label, and those uses often constitute the accepted standard of care. The practice is ubiquitous in cancer and cardiac treatment, where as many as half of all prescriptions are for off-label uses. In the absence of off-label prescribing, physicians would have fewer treatment options and many patients would die or suffer needlessly.

Why You Should Care about What the Government Thinks about Off-Label Drugs:

The FDA, with support from many in Congress, has sought repeatedly to limit physicians’ and patients’ discretion about treatment decisions, including restricting physicians from prescribing drugs off-label and interfering with drug companies’ ability to disseminate information about new uses of approved drugs.

Attacks on Drug Companies:

The FDA and federal prosecutors take these restrictions very seriously, charging violators with both civil and criminal sanctions. In 2009 drug manufacturer Pfizer pleaded guilty to criminal charges and paid a record $2.3 billion to settle allegations of promoting 14 of its products for off-label uses, and Eli Lilly was forced to pay $1.4 billion for promoting its schizophrenia drug Zyprexa for off-label use. On April 27, 2010, the Department of Justice announced a $520 million settlement with pharmaceutical manufacturer AstraZeneca that resolved allegations that the drug maker had illegally promoted off-label uses of the company’s antipsychotic drug Seroquel.

Attacks on Doctors:

It appears that no possible violation is too minor to attract regulators’ attention. Earlier this year the FDA even sent a warning letter to a Florida dermatologist for mentioning in interviews with Elle and Allure magazines and on NBC’s Today show that an anti-wrinkle drug she was testing had shown positive results and that “early data shows it may last longer and kick in faster than Botox.”

Comments (8)

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  1. Devon Herrick says:

    An important part of drug approval is verifying the compound has a beneficial effect on a disease condition and that the drug is not toxic at typical doses. Once a drug is approved and the toxic effects are known, doctors who observe other benefits are free to experiment. Requiring additional human drug trials for every additional use would be hugely expensive and result in many beneficial drugs never being tests for numerous conditions.

    The calculus of drug research means that many conditions to not offer sufficient market potential to justify the expense of a drug trial because the investment in time and money would not pay for itself.

  2. Virginia says:

    I’m a little torn on this one. Of course, it would be better if the FDA would not even be in the picture to begin with. But, I wonder why there are so many “off label” uses for various drugs. If the drug is so good at treating other ailments, then why aren’t those ailments listed as general treatments?

    I suppose the answer has to do with the regulation process. Perhaps it’s cheaper to get a drug approved for one thing and then hope the doctors figure out that it has other uses. But, still. Wouldn’t it be better just to get approval for all uses?

  3. Virginia says:

    Ah. Now I see Devon’s discussion. Point taken.

  4. Joe S. says:

    This is a very worrisome development. The Obama Administration will be tempted to control costs by limiting off label uses. Patients will be the losers.

  5. artk says:

    Before all you uber free market folks get all bent out of shape about off label drugs, you should remember that phen-fen was off label. That didn’t turn out so well.

  6. Bart Ingles says:

    artk: Can you name other examples?

    There have also been a number of on-label uses that haven’t turned out so well. I’m thinking of the arthritis and diabetes medications that appear to have caused heart attacks…

  7. Devon Herrick says:

    Artk and Bart, there is no such thing as a completely risk free drug. They are all bound to have some toxic or unpleasant side effects. The trade-off is always going to be whether the benefits outweigh the risks – which will vary from one person to the next. Some people get liver damage or a rare muscle atrophy from statins, yet by most accounts statins are so beneficial that the British Medical Journal once whimsically asked whether statins should be injected into the water supply.

    Vioxx has been linked to heart attack and stroke, but not everyone on Vioxx was at risk for heart problems and many people really benefited from Vioxx. Not taking daily aspirin is a heart risk – yet people on Vioxx would not be on aspirin (why take a $3 Vioxx pill if you could tolerate a $0.03 aspirin tablet?). Aspirin causes an estimated 16,500 death annually from bleeding ulcers. Many of these people possibly could have benefits from Bextra, Vioxx or Celebrex (Bextra and Vioxx are both off the market due to cardiac risks). When Phen-fen was yanked from the market, I read several experts claim health risks from heart valve problems was less than the risk of obesity in many people.

  8. Linda Gorman says:

    Off-label uses abound. They do a lot of good.

    Amoxicillin to cure stomach ulcers caused by H. pylori is an example of an off-label use which is now the standard of care. Amoxicillin will never be approved for on-label use in killing H. pylori because it is off patent and there is no reason to spend gigabucks running clinical trials when the drug is known to be clinically safe and effective in an off-label use.

    Many chemotherapy drugs are off-label. A drug might be approved for the treatment of pancreatic cancer and then be shown to be effective against other kinds of tumors. Thalidomide, which was approved for use against leprosy, is also used in cancer treatment and HIV-related mouth and throat ulcers.

    Arguments about off-label use will increase as eliminating off-label is an effective way to reduce government health care spending.