The Food and Drug Administration (FDA) approve drugs for certain health conditions. When a doctor prescribes a drug off-label, they are prescribing it for a different condition or at a different dosage than the FDA have approved.
Off-label prescribing is common. According to the Agency for Healthcare Research and Quality, around 20% of all prescriptions in the United States are for off-label use.
Taking a drug for an off-label use can be safe and effective. However, there may also be certain risks involved.
This article describes why doctors prescribe drugs off-label and lists some examples of drugs that have off-label uses. It also outlines some of the benefits and risks associated with off-label drug use.
The FDA are the agency responsible for approving and legalizing drugs in the U.S.
For a drug to gain FDA approval, the company that make it must send certain information to the FDA. This includes clinical trial information on the following:
- health conditions that the drug can effectively treat
- the dosage that people can safely take
- the populations that they have tested the drug in, such as adults, females, or children
- evidence showing that the drug is both safe and effective, including the adverse events and side effects it can cause
If the FDA approve the drug, doctors may then prescribe it to their patients. However, a doctor does not have to prescribe the drug only for the uses the FDA approve. Doctors have the ability to prescribe drugs for other uses if they feel that it will benefit the patient.
Below are some reasons a doctor may prescribe a drug for off-label use:
- There is no FDA-approved drug available to treat a person’s condition.
- The drugs that the FDA have approved for a particular condition have not been effective for the person.
- The drugs that the FDA have approved for a particular condition are too expensive or otherwise inaccessible.
- A person is unable to take the FDA-approved drug due to side effects or possible interactions with other drugs they are taking.
- The doctor has seen evidence that a certain drug works well for a particular off-label use.
Doctors prescribe many different drugs for off-label use. Historically, some off-label uses have proven ineffective or harmful. However, others have proven effective and even life changing.
The table below lists some FDA-approved drugs alongside their FDA-approved uses and their common off-label uses.
|FDA-approved drug||FDA-approved use||Off-label use|
|tamoxifen||treating breast cancer||treating certain causes of infertility in females|
|spironolactone||treating high blood pressure, heart failure, edema, and Conn’s syndrome||treating acne vulgaris in females|
|modafinil||improving wakefulness in people with narcolepsy, obstructive sleep apnea, or shift work sleep disorder||treating depression|
|escitalopram||treating depression||treating bipolar disorder|
|amitriptyline||treating depression||treating fibromyalgia|
|fluoxetine||treating major depressive disorder (MDD), panic disorder, obsessive-compulsive disorder, and bulimia nervosa||treating fibromyalgia|
|venlafaxine||treating MDD, panic disorder, generalized anxiety disorder, and social anxiety disorder||treating fibromyalgia|
|beta-blockers||treating high blood pressure, abnormal heart rhythms, angina, coronary heart disease, heart attack, migraine, and glaucoma||treating anxiety|
Even without FDA approval, there may be clinical trials and other evidence demonstrating that a drug works well for an off-label use.
This can happen if the drug company decides not to pursue additional drug use approvals due to cost or lack of resources, or if the drug is in the process of receiving FDA approval but they have not yet finalized it.
However, off-label prescribing is not always safe. This is especially true if there is a lack of evidence about a drug’s off-label use.
When discussing off-label drug use with a doctor, a person should keep the following points in mind:
- The FDA have not investigated how well the drug works for their particular health condition.
- There may not be strong medical evidence that supports using the drug for their particular health condition.
- People with their condition may experience unique side effects while taking the drug.
People should also be aware that doctors are not obligated to tell a patient when they are prescribing a drug off-label.
Because of this, a person may want to ask their doctor the following questions when receiving a prescription drug for the first time:
- What does this drug have approval for?
- What are the benefits of taking this drug over a drug that has approval for my condition?
- What scientific evidence is there to show that this drug could work for my health condition?
- What kind of results should I expect to see while taking this drug?
- What are the side effects of this drug?
- What dosage should I take?
- Will this drug interact with other drugs, supplements, or herbs that I am taking?
- What are the other options for treating my condition?
Off-label prescribing is legal in the U.S. Doctors can prescribe FDA-approved medications for purposes that are not on the label if they feel that it is appropriate and beneficial for the patient.
The practice of prescribing drugs off-label is neither ethical nor unethical. It depends on the doctor’s reasons for off-label prescribing.
For example, people may consider it to be ethical if they are unable to take the FDA-approved treatment options for their condition due to side effects or possible interactions with other medications, or if they have exhausted all other FDA-approved treatment options for their condition.
The doctor should discuss any potential side effects with the patient. Both parties should agree that the potential benefits of taking the drug off-label outweigh the risks. The doctor should also be willing to change the dosage or change the drug entirely if it proves ineffective or harmful.
There are many reasons a doctor may prescribe a drug off-label. For example, a person may not have access to a particular FDA-approved drug, or they may have exhausted all other FDA-approved treatment options for their condition.
Taking a drug off-label can be beneficial, but there may also be certain risks involved. A person should discuss the potential benefits and risks with their doctor before starting a new drug off-label.
A person should also tell their doctor if they experience side effects while taking a drug off-label. The doctor may recommend changing the dosage or changing the drug altogether.