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What does "off-label" mean and what you can do about it ?

Updated: 6 days ago

By Martine G. Brousse (still not AI!)

"The Medical Bill Whisperer"

Patient Advocate, Certified Mediator


April 4, 2024

It may happen to you or your loved one that your Dr orders a treatment or Rx deemed "off-label".

Let's explore:

  • What does it mean clinically?

  • What does it mean financially?

  • What can you do about it?

A. The FDA "label"

Let's start with what "on-label" means first.

From the FDA website, the agency that regulates drugs, this is the process for approval for a drug manufacturer to bring their product to market:

"Before a drug can be approved, a company must submit clinical data and other information to FDA for review. The company must show that the drug is safe and effective for its intended uses. “Safe” does not mean that the drug has no side effects. Instead, it means the FDA has determined the benefits of using the drug for a particular use outweigh the potential risks".

Once a "label" has been appro

ved, it will show:

  • The specific diseases and conditions that the drug is approved to treat.

  • How to use it: dosage, frequency, way to administrate

  • Information about the risks and potential side effects

  • Information for healthcare providers to discuss with patients before they take a drug

Of importance:

"From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient". This is "Off-Label"

B. What is "Off Label"?

  1. Use in your case is NOT approved by the FDA (see reasons below)

  2. Not being used for the diagnosis, condition, medical need or clinical purpose it is meant and approved for

  3. not used in the frequency, way of administration or dosage indicated on the label, or as usually prescribed

  4. 2 (or more) on-label drugs added to each other to create an off-label protocol  


  • As of this writing, Ozempic is in the news. It is only approved by the FDA to treat patients with an established Diabetes Type 2 diagnosis, for improved glucose control and/or reduction of cardiovascular events.

  • But off-label use has exploded: for general weight loss and to treat food (and possibly other ) addictions.

  • If you don't have Diabetes type 2 and use Ozempic for weight loss, it is "off-label". If you are under 18, or have Diabetes type 1 and use Ozempic, it is "off-label".

  • Once newer studies or trials come to demonstrate safety and efficacy for other diagnoses, the FDA could/would then expand the scope of their approval, but as of this writing, the widespread use of Ozempic is "off-label".

C. Why use off-label drugs or treatments?

  1. When there is no other or left treatment to treat your condition. This happens with cancer stage 4 patients for example, when all conventional protocols have failed

  2. When there is no treatment at all for your (rare) disease. Rare or 'newer" diseases may not have the public attention, funding, trials, or even will to promote the costly development of new treatments. Using drugs currently on the market "off-label" once proven, even in just a few patients, to be safe and effective, may be the one option

  3. When studies and trials have been undertaken but the FDA has not yet given a positive decision

  4. The prescribing physician may be the leading pioneer in this field, establishing a case for trials, or "standard of care"

D. Insurance POV

  1. Insurance companies can usually be compelled to cover all FDA-approved drugs (though they may prefer generic options), but they routinely reject "off-label" requests as "experimental", "unproven" or "experimental".

  2. While a few widely accepted off label uses can be automatically covered, the majority would require a special exception authorization.

  3. If you are involved in an off-label trial, meaning you are part of a study run by a manufacturer and medical entity to "test" a new drug or protocol, then the drug would be provided by the study sponsors.

  4. If you are not in a trial, an off-label Rx or treatment could be at your full charge.

E. What to do if your Dr advises or prescribes an "off-label" treatment?

  1. Ask questions to your physician:

  • Are there other approved drugs or therapies to treat my disease at this stage?

  • What is the scientific data supporting this off-label use for my condition?

  • What are the odds of this off-label drug/treatment working better than other potential conventional alternatives, or at all?

  • What are potential side effects and risks to my health?

  • Are there any trials I could enroll in?

  • Does the manufacturer offer a drug donation or financial assistance program? Would I be eligible (some insurance members may not be eligible, and off label may be excluded)

  • What would be my cost if insurance does not cover?

2. Get insurance to pay

  • Your physician should request an exception authorization, based on medical necessity and potential benefits

  • History of tried but failed previous conventional treatments must be included

  • Findings of studies, trials and research must be provided

  • Inclusion of protocol or use in one of the Reference Compendia will be useful

  • Documentation from medical peer-reviewed articles or from governmental health agencies can help

  • I have won cases based on State laws overriding insurance medical policies and FDA-approvals

  • Ordering physician must be ready to discuss rationale for ordering the RX/treatment, and provide the medical justification as evidence.

In Conclusion:

"Off label" can be complicated but many instances are more mundane, and merely insurance companies' efforts at directing you to use Rx cheaper generic versions and receive conventional rather than brand-new treatment protocols.

Your physician should be able to build a case explaining and supporting any "off-label" use, and should be encouraged to place efforts into obtaining insurance coverage rather than placing the financial burden solely on your and your family's shoulders.

Martine Brousse was a long-time Billing Manager for Physicians before switching to the side of patients in 2013. The move has allowed her to apply her deep expertise and vast experience of the intricacies of resolving all types of medical bill and claim payment issues in ways that directly and positively impact her clientsʻ finances.

(424) 999 4705 - F (424) 226 1330

@martine brousse 2024 @ the medical bill whisperer 2024

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