Off-Label: Why Drugs Like Ivermectin Spark Debate

Off-label use refers to the practice of prescribing a medication for a purpose, dosage, patient group, or form of administration that has not been approved by the FDA.

Healthcare providers may consider off-label prescribing when they believe it is medically appropriate for a patient’s condition, even though the specific use is not included in the FDA-approved labeling. For example, a drug approved to treat one type of cancer might be used off-label to treat a different type of cancer.

There continues to be interest in ivermectin for the prevention or treatment of COVID-19 in humans. The FDA has not authorized or approved ivermectin for use in preventing or treating COVID-19 in humans or animals. 

For humans, ivermectin tablets are approved at specific doses to treat some parasitic worms, and there are topical formulations for head lice and skin conditions like rosacea (see DailyMed for Indication and Usage section).

Off-label use carries both potential benefits and risks. While it can provide access to “potentially” effective treatments not otherwise available, it also means that the medication has not undergone the same level of scrutiny by the FDA for the specific use, which may lead to unforeseen adverse effects. Therefore, healthcare providers must weigh the available scientific evidence and consider the patient’s specific circumstances before prescribing medications off-label.

Questions you may want to consider:

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If the provider is thinking about using an approved drug for an unapproved use, you may want to discuss the following:

  • Are there other drugs or therapies that are approved to treat the disease or medical condition?
  • What scientific studies are available to support the use of this drug to treat the disease or medical condition?
  • Is it likely that this drug will work better to treat the disease or medical condition than using an approved treatment?
  • What are the potential benefits and risks of treating the disease or medical condition with this drug?
  • Will health insurance cover treatment of the disease or medical condition with this drug? 
  • Are there any clinical trials studying the use of this drug for the disease or medical condition that the patient could enroll in?

PAAS National® analysts have seen PBMs recoup medications being used off-label before. Medicare and Medicaid do not knowingly pay for off-label medication use. They employ a “pay and chase” model meaning that they assume the medication is being used for a medically accepted indication, so they pay the claim to ensure the patient is not inconvenienced – then they may audit the claim for appropriateness. Please see March 2022’s Newsline article, Off-Label Use Not Covered Under Medicare Part D, for more information.

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Kristen Sterzinger, CPhT
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