Why Doctors Prescribe Off-Label Drugs
- When a drug is used in a way that is different from that described in the FDA-approved drug label, it's said to be an "off-label" use.
- Off-label prescribing is legal and very common.
- The biggest problem is getting insurance plans to pay for off-label drug use.
Every prescription drug available in the U.S. has been approved by the Food and Drug Administration (FDA) via a lengthy, expensive process of clinical trials. These trials show that the drug is safe and effective at treating a particular condition. When the FDA is satisfied that the drug works and is safe, they create the drug label. This is not an actual label that sticks to a bottle, but a report of very specific information about the drug. This label gives information about the drug, such as:
- What the drug is made of
- How it works in the body
- The research studies that led to its approval
- Side effects it may cause
- The specific medical condition(s) it's approved for (called the indication(s) for use).
- Dosages
- The route it should be given.
What does off-label mean? Is it safe?
Read More- Used for a different disease or medical condition
- Given in a different way (such as by a different route)
- Given in a different dose than in the approved label
However, Dr. Mikkael Sekeres, Chief of the Hematology Division at University of Miami says that we already have the ability to obtain drugs that aren’t yet FDA approved for our patients. “One mechanism is clinical trials. Clinical trials are something I prefer because they are rigorously monitored including for whether or not a drug works. And for the toxicities that a patient may experience on that clinical trial.”
It is estimated that up to 20% of all medications prescribed are for indications that are not approved by the FDA. In the study, which was published in JAMA Internal Medicine, the rate of side effects for off-label drugs was 44% higher than on-label ones.
Off-Label Drug Use in Cancer Treatment
The use of off-label is very common in cancer treatment. Often, usual care for a specific type or stage of cancer includes the off-label use of one or more drugs.
When a chemotherapy drug is approved for treating one type of cancer but is used to treat a different cancer, it's off-label use. The same is true if a drug is approved to treat a type of cancer at a specific stage (extent of spread), but it is used to treat a cancer at a different stage.
Off-label prescribing may be advantageous in oncology as it presents evidence-based treatment options to patients who have no alternative options, for example in indications where there were no approved drugs or for patients who have exhausted standard lines of treatment. While off-label drug use may sometimes be clinically justified, it is associated with a number of safety and ethical issues. Off-label prescribing can jeopardize patient safety in certain clinical scenarios where a positive benefitrisk ratio is not fully established. This is mainly due to the fact that off-label drug uses are not systematically appraised by regulators, guidelines formulators or even healthcare policymakers.
“Off-label prescribing can expose patients to risky and ineffective treatments,” medical ethics professor Rebecca Dresser and Joel Frader, MD, write in the fall 2009 issue of The Journal of Law, Medicine & Ethics.
Is it legal?
The practice, called “off-label” prescribing, is entirely legal and very common: at least one in five outpatient prescriptions written in the United States are for off-label therapies. While it's legal for doctors to use drugs off-label, it's not legal for drug companies to market, advertise or promote their drugs for off-label uses. Off-label marketing is very different from off-label use.
Who pays the bill?
The biggest problem is getting insurance plans to pay for off-label drug use. Many insurance plans decline to reimburse the cost of off-label cancer treatments on the ground that these uses are "experimental" or "investigational." Health insurance coverage laws and regulations are complex. For years, rules have been changing to cover more off-label uses of cancer treatment drugs.
Before starting a treatment with off-label drugs, make sure to check if your insurance covers the drug that is prescribed. If coverage is denied, it may be helpful for the doctor to provide the insurance company with copies of documents that support the suggested off-label use, such as copies of peer-reviewed journal articles or other respected sources that support the off-label use.
Questions to ask your doctor
- Is the medication used on- or off-label?
- What is the drug approved for?
- Why do you think the off-label use of this drug will help the type of cancer that I have?
- Is the off-label drug likely to work better than an approved drug?
- Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in?
- What are the risks and benefits of treatment with this drug?
- Will my health insurance cover my treatment with this drug?
Article with contributions from Dr. James Taylor
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