Williams Cancer Institute

What is “Off-Label” Cancer Treatment and Does It Have Advantages?

cancer treatment (1)

What is “Off-Label” Cancer Treatment and Does It Have Advantages?

You have a lot to process when you receive a cancer diagnosis. Part of that discussion can include a decision about off-label treatment options.
You’re probably familiar with the U.S. Food and Drug Administration (FDA). Their research determines a drug therapy’s safety and its designated use.
But a particular drug’s effectiveness doesn’t stop there


Simply put, an off-label treatment would involve the use of a specific (FDA-approved) drug for a purpose other than the FDA designation. Therefore, a drug’s secondary use is not listed on its “label”.
For example, you’ll notice a few things when examining a drug’s label.
How it’s designed to work
Relevant research data that supports its approval
Side effects
The FDA takes seriously its responsibility for assuring a medication is safe and effective for its specific use. Their control does not extend to your doctor’s decision about what drugs to prescribe you.
FDA approval is valid and essential. But your doctor has the latitude to prescribe it for a purpose in your best interest following additional tests.
Off-label use can vary greatly from one doctor to another, depending on doctors’ preferences, knowledge, and past patient experiences. A 2008 study found 8 out of 10 cancer doctors surveyed had prescribed off-label drugs. 1


An FDA approved drug therapy can be used off-label for a specific health condition such as
Cancer that’s different than one the drug is specifically designed to treat
A different prescribed dosage or use frequency
Treating a child instead of an adult
Keep in mind that off-label treatments still require approval from the FDA at the manufacturing company’s request. Additional approvals also require research that shows the treatment to be safe and effective for new or innovative uses. This research requires time and money from the manufacturer.


Off-label use of drugs is common for treating cancer. The stages and types of cancer can be treated with the off-label use of one or more drug therapies. Certainly, advanced cancers that are considered incurable may have more options as they can fall under the “right to try laws.”
A cancer drug can be effective against more than one type of cancer. For example, immunotherapy was initially approved only for melanoma, so use in lung cancer was “off-label” at the time. Since it has gained FDA approval because it was effective. Often, a drug in cancer may get approval for one type first but is later shown effective in other types of cancer as well.
Research is trusted to discover new uses for approved drugs. These studies are accessible by your doctor and will be used to evaluate a drug’s effectiveness for treating your type of cancer.
At the same time, off-label drug use might not be safe. Circumstances can include:
No proof of effectiveness for a certain type of cancer or reason to believe it would be
The potential risks of usage are greater than the possible benefits
Again, your doctor will prescribe off-label use of a particular drug based on their knowledge and experience with it and the drug’s effectiveness, as revealed by research.


We’re prepared to help you understand the reason off-label treatment could be a viable solution for treating your type of cancer. We encourage you to ask and discuss these questions with us:
Why will my specific cancer respond to the off-label use of this drug?
What difference in results will I experience between the off-label drug and the one specifically approved for treatment?
What risks and benefits can I expect from the off-label use of this drug?
Does my health insurance cover my treatment with this drug?
Will my health insurance cover combination chemotherapy and the use of an off-label drug?
These questions and more are common and important to ask prior to beginning cancer treatment with off-label drug therapies. Our goal is to give you the best opportunity to treat cancer and restore your health.

Contact us about cancer treatment solutions. Schedule a consultation and ask us about off-label drug therapies for treating and eliminating cancer.

Reference: National Cancer Institute, Off-Label Drug Use in Cancer Treatment, https://www.cancer.gov/about-cancer/treatment/drugs/off-label

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