Off-label prescribing is a fairly common practice in the United States. As many as 20% of prescriptions in the U.S. are off-label uses. So, what is off-label prescribing?
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Off-label use is when medicines are prescribed for different purposes than they are approved for, or in different dosages or forms. This kind of prescribing is legal for many drugs. It’s currently illegal to prescribe opioid drugs like fentanyl for purposes other than their approved use, however.
The FDA explains that there are multiple reasons a doctor might prescribe an approved drug for an unapproved use. These include when:
- Drugs are used to treat different but similar conditions, such as types of cancer
- The drug is administered or taken differently, such as a pill versus a liquid
- A drug is given in a different dose than it is approved for.
Prescriptions for off-label use may be provided to children, who can safely consume certain medications but only in smaller doses, or people who require different forms of medicine.
The Agency for Healthcare Research and Quality, a branch of the U.S. Department for Health and Human Services, explains there may be safe and effective drugs that are technically off-label as they await approval or more research. With cancer treatment, for example, these drugs may be the only treatment option left. In other situations, these drugs may have extensive anecdotal evidence of effectiveness to treat conditions outside of their approved use.
Some of the common health conditions for which off-label drugs are prescribed include:
- Mental health
- Heart conditions
There is some controversy about off-label prescribing and use, as it is at the physician’s discretion. In some cases, off-label drugs have been prescribed and greatly benefitted patients. In other cases, off-label drug use has adverse effects. There are both risks and benefits.
The Risks of Off-Label Drug Use
The American Cancer Society details what is perhaps the biggest objection to off-label drug use, which is reimbursement. Many insurance companies won’t pay for off-label drugs. Medicaid will sometimes pay for them. Also, while off-label prescribing is not technically illegal, there are multiple points at which providers could be susceptible to malpractice suits, or the practice of this prescribing could strengthen a malpractice suit from a different point of a patient’s care.
There can be risks when the companies who make certain drugs market them for uses beyond their approved scope. A landmark case was a 2012 ruling requiring the drug company GlaxoSmithKline pay $3 billion for allegations of fraud. The company was allegedly using marketing messaging for their drug Paxil that suggested it be used in adolescents and children. This was not an approved use of this drug. Research began to show significant dangers in giving anti-depressants to teens and kids, indicating it may enhance depressive symptoms and even lead to suicide. The dramatic consequences in this scenario led to attention and prosecution.
The Benefits of Off-Label Drug Use
The process for FDA approval can take a long time, with multiple steps and some barriers. While strict regulation may ensure the greatest safety, it may also limit access for people who are very ill and could benefit from unapproved drugs. In this case, it is often up to the physician to perform medical trials with unapproved drugs as an effort to keep someone alive.
Drug regulations play a vital role in public health and the FDA’s systems of review for new drugs is an important step prior to their release to the general public. If a drug has not yet been approved, there are other bodies of review that physicians can use to learn about new drugs. These are outside of manufacturer oversight, reasonably ensuring that there are no biases. For doctors who are seeking alternative treatment, these sources can offer insight into what unapproved drugs are available for treatment.
Off-label prescribing is a practice that can be immensely helpful to people who have found no effective treatment with approved drugs. It is something that healthcare providers have to approach with research and care. The goal should always be that patients receive the safest and most effective treatment for their conditions.
American Cancer Society. “Off-Label Drug Use.” Updated March 17, 2015. Accessed August 3, 2019.
Consumerreports.com. “Off-label drug prescribing: What does it mean for you?” Updated July 2015. Accessed August 3, 2019.
Food and Drug Administration. “Understanding Unapproved Use of Approved Drugs ‘Off Label.’” Updated February 2, 2018. Accessed August 3, 2019.
National Institutes of Health. “Off-Label Drug Information.” August 2009. Accessed August 3, 2019.
The United States Department of Justice Office of Public Affairs. “GlaxoSmithKline to Plead Guilty and Pay $3 Billion to Resolve Fraud Allegations and Failure to Report Safety Data.”July 2, 2012. Accessed August 3, 2019.
Wittich, Christopher M. et al. “Ten Common Questions (and Their Answers) About Off-label Drug Use.” October 2012. Accessed August 3, 2019.