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Off-Label Drug Use: What You Need to Know

Prescription drugs are often prescribed for uses other than what the FDA has approved. Find out why.

Who Is in Control?

Off-label drug use can sometimes evoke negative connotations and may diminish public expectations that drugs will go through the rigorous testing required by the FDA, experts say. Murphy says she'd be more comfortable knowing the medicine she takes was specifically tested for her particular condition.  

The big question, Alexander says, is how to best regulate off-label drug use without unduly stifling clinical innovation. Some have called for a greater emphasis on evidence-based prescribing, with stricter rules governing off-label drug use. But others worry that tightening the reins could hinder patients' early access to novel therapies.

Off-Label, Higher Costs?

Greater promotion of off-label drug uses could have patients reaching deeper into their pockets to cover prescription costs. Health insurance companies closely scrutinize drugs that are used off-label. Although Medicare recently changed its rules to allow for wider coverage of off-label uses of cancer drugs, insurers do not always pay for an unapproved, or unproven, product.

"This trend is likely to continue as coverage decisions target wasteful spending," Emily Largent, BSN, Frank Miller, PhD, and Steven Pearson, MSc, tell WebMD by email. Largent, Miller, and Pearson work for the department of bioethics at the NIH Clinical Center.

Questions to Ask Your Doctor

Doctors aren't required to disclose off-label drug use to patients, and doctors, including Alexander, say doing so would be impractical. So it's important for patients to understand the difference between on- and off-label prescribing. Largent, Miller, and Pearson recommend asking your doctor the following questions whenever you are given a prescription:

  • Is the medication used on- or off-label?
  • What is the evidence behind your decision to prescribe this to me?
  • How certain are you that I am likely to benefit from this medication?

If the drug is being used off-label, you may want to check with your insurance company to make sure it's covered. You might also consider asking your doctor if there are any clinical trials studying the off-label use in which you could enroll. A number of clinical trials are being done to test the safety and effectiveness of off-label drug uses.


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