Off-Label Drug Use

Although the FDA approves all prescription drugs sold in the United States, the agency can’t limit how doctors prescribe drugs after they’re on the market. Doctors often direct patients to take medications for conditions that have not been approved by the FDA. This is called off-label drug use.

Fast Facts
  • Off-label drug use is when doctors legally prescribe drugs for unapproved uses.
  • The FDA has no control over how doctors prescribe drugs.
  • Off-label drug use is not without risks.

Off-label drug use is when drugs are prescribed for a condition or a type of patient or a dosage not officially approved by the U.S. Food and Drug Administration. The FDA does not regulate the medical profession. The agency has no control over how doctors prescribed approve drugs to patients. So once a medication is on the market, it is up to physicians to determine the medically appropriate use of the drug.

Consequently, it’s legal and common for doctors to prescribe drugs for uses other than what is listed on the label, commonly known as off label. One fifth of all prescriptions are off label.

Fact
20 percent of all prescriptions are written for uses not approved by the FDA.

The fact that a condition or a dosage or a category of patient is not included on a label doesn’t mean that the drug is not appropriate for that use. It means only that the FDA has not approved that particular use of the drug.

off-label safety icons

Off-Label Drug Use Safety

Off-label drug use is generally not backed by the level of testing and data that allows FDA approvals. Patients don’t have the guides and warnings that come from FDA-approved labels. Doctors and patients therefore don’t always have enough information about the effects and dangers of the off-label use of the drug to make informed decisions.

Off-label drug use risks
This can create a situation where patients are unknowingly at risk for dangerous, unexpected side effects.

Researchers examined drugs approved as antipsychotics for schizophrenia and bipolar disorder. Those drugs were used for anxiety, agitation and obsessive behaviors. But there was no evidence that the medications were effective for the off-label conditions. So these patients risked the dangerous side effects of these drugs, including sudden death from heart disease, when the use of the drugs was questionable at best.

Evidence lacking

“Patients do benefit from off-label prescribing that is supported by sound scientific and medical evidence,” wrote two medical ethics professor in the fall 2009 issue of the Journal of Law, Medicine & Ethics. “In the absence of such evidence, however, off-label prescribing can expose patients to risky and ineffective treatments.”

Opioids
Authorities say powerful opioids have been prescribed off-label for patients whose pain does not justify their use. This raises the risk of addiction and overdose deaths. One example of this was the potent fentanyl drug Actiq, a lollipop approved for cancer patients who had developed a tolerance for other pain medications.

But the drug maker promoted the drug to physicians who treated other conditions. The subsequent increase in sales was linked to more than 120 deaths, including two children.
Antipsychotics
Nursing home patients were given powerful antipsychotic drugs, including Risperdal, for symptoms of dementia, even though the drugs were considered by many in the medical establishment to be dangerous and inappropriate for such patients.
Fen-Phen
A notorious example of how off-label drug prescribing can be dangerous is the use in the 1990s of Fen-Phen. Two drugs, phentermine hydrochloride and fenfluramine, were approved separately. But doctors prescribed them together for weight loss. The combination also caused severe heart-valve problems. They were taken off the market.
medication icons

Why Doctors Prescribe Drugs Off Label

Physicians prescribe drugs off-label for a variety of reasons. Advances in medicine can outpace the FDA’s ability to approve drugs for new uses. It can take six years to get a new drug approved. The cost to get a drug approved for a different use may not be worth it for the manufacturer.

Then there is the problem that clinical trials don’t always include certain patients, such as children, pregnant women and the elderly, as well as some psychiatric patients.

Off-Label Awareness

Often, doctors don’t even realize the prescriptions they’re writing are for conditions or patients not approved by the FDA for the particular drugs. A 2009 survey found that many doctors thought some common off-label drug treatments were approved by the FDA. In other words, sometimes they didn’t even realize that they were prescribing the drugs off label.

Patient Responsibility

So the responsibility falls on the patient, and in the case of children, the parent, to ask questions when receiving a prescription.

The FDA recommends asking the following questions about your off-label prescription:
  1. What is the drug approved for?
  2. Are there other drugs or therapies that are approved to treat my disease or medical condition?
  3. What scientific studies are available to support the use of this drug to treat my disease or medical condition?
psychiatric disorder icons

Psychiatric Disorders

Off-label drug prescribing is common in treating psychiatric disorders. One analysis found that 88.5 percent of all defined psychiatric disorders have no FDA-approved medication for their treatment. According to that analysis, for two common classes of psychiatric medications — mood stabilizers and atypical antipsychotics — there were more off-label uses than FDA-approved uses.

Atypical Antipsychotics

Examples of atypical antipsychotics include Seroquel, Zyprexa and Abilify. They are being used to treat an array of conditions off label, including anxiety, attention-deficit disorder, sleep problems, behavioral problems in toddlers and dementia.

“Antipsychotics are overused, overpriced and oversold,” said Allen Frances, former chair of psychiatry at Duke University School of Medicine, according to Kaiser Health News.

Serious Side Effects

Careful off-label use may be appropriate for some patients who haven’t been helped by other drugs. Frances told Kaiser that the medications, which are designed to calm patients and to moderate the hallucinations and delusions of psychosis, are being used “promiscuously, recklessly,” often to control behavior and with little regard for their serious side effects, which can range from rapid weight gain to death.

Cancer Treatment icons

Cancer Treatment

According to the National Cancer Institute, research has shown that off-label use of drugs in cancer treatments is very common. Chemotherapy and other cancer drugs are often prescribed off label because the drug approved for a certain kind of cancer may target different types of tumors or generally enhance chemotherapy.

According to the American Cancer Society, cancer patients often present problems that have few treatment options. These patients may be more willing to try unapproved drugs.

Please seek the advice of a medical professional before making health care decisions.

30 Cited Research Articles

  1. Agency for Healthcare Research and Quality. (2015, September) Off-Label Drugs: What You Need to Know. Retrieved from https://www.ahrq.gov/patients-consumers/patient-involvement/off-label-drug-usage.html
  2. Chen, D.T. et al. (2009, November). U.S. physician knowledge of the FDA-approved indications and evidence base for commonly prescribed drugs: Results of a national survey. Retrieved from https://www.researchgate.net/publication/26757858_US_physician_knowledge_of_the_FDA-approved_indications_and_evidence_base_for_commonly_prescribed_drugs_Results_of_a_national_survey
  3. Clancy, C.M. (2009, April 21). Off-Label Drugs: What You Need to Know. Retrieved from https://archive.ahrq.gov/news/columns/navigating-the-health-care-system/042109.html
  4. Agency for Healthcare Research and Quality. (2009, January). Use of Atypical Antipsychotic Drugs Increases Risk of Sudden Cardiac Death in Adults. Retrieved from https://archive.ahrq.gov/news/newsroom/press-releases/2009/antipsych.html
  5. U.S. Food and Drug Administration. (n.d.). Understanding Unapproved Use of Approved Drugs “Off Label.” Retrieved from: https://www.fda.gov/forpatients/other/offlabel/default.htm
  6. Consumer Reports. (2015, July). Off-label drug prescribing: What does it mean for you? Retrieved from https://www.consumerreports.org/cro/2012/05/off-label-drug-prescribing-what-does-it-mean-for-you/index.htm
  7. Thomas, K., (2016, March 8). FDA Deal Allows Amarin to Promote Drug for Off-Label Use. Retrieved from https://www.nytimes.com/2016/03/09/business/fda-deal-allows-amarin-to-promote-drug-for-off-label-use.html
  8. Ballentine, C. (1981, June). Taste of Raspberries, Taste of Death. The 1937 Elixir Sulfanilamide Incident. Retrieved from https://www.fda.gov/aboutfda/whatwedo/history/productregulation/sulfanilamidedisaster/
  9. U.S. Food and Drug Administration. (n.d.). Milestones in U.S. Food and Drug Law History. Retrieved from https://www.fda.gov/AboutFDA/WhatWeDo/History/Milestones/default.htm
  10. Wittich, C.M. et al. (2012, October). Ten Common Questions (and Their Answers) About Off-label Drug Use. Retrieved from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3538391/#bib1
  11. American Academy of Pediatrics. (2014, March). Off-Label Use of Drugs in Children. Retrieved from http://pediatrics.aappublications.org/content/133/3/563
  12. Haelle, T. (2014, March 10). 500 Drugs Updated with Directions for Child Use Since 2002. Retrieved from https://www.scientificamerican.com/article/500-drugs-updated-with-directions-for-child-use-since-2002/
  13. Spiesel, S. (2008, October 15). Prozac on the Playground. The dangers of off-label use of psychiatric medications in children. Retrieved from http://www.slate.com/articles/health_and_science/whats_up_doc/2008/10/prozac_on_the_playground.html
  14. Lai, L.L. et al. (2017, March). Off-Label Prescribing for Children with Migraines in U.S. Ambulatory Care Settings. Retrieved from http://www.jmcp.org/doi/pdf/10.18553/jmcp.2017.23.3.382
  15. Zito, J.M. et al. (2008, September 15). Off-label phsychopharmalogic prescribing for children: History supports close clinical monitoring. Retrieved from https://capmh.biomedcentral.com/track/pdf/10.1186/1753-2000-2-24?site=capmh.biomedcentral.com
  16. Caudet, J.V. (2012, August 21). Complexities of Off-label Prescribing in CV Pediatrics. Retrieved from http://www.cardiovascularbusiness.com/topics/practice-management/complexities-label-prescribing-cv-pediatrics
  17. Tweodros, E. et al. (2016, January). Association of Off-label Drug Use and Adverse Drug Events in an Adult Population. Retrieved from https://jamanetwork.com/journals/jamainternalmedicine/article-abstract/2467782
  18. Avitzur, O., (2017, June 21). Stay Safe When Your Drug is Off-Label. Retrieved from https://www.consumerreports.org/drugs/stay-safe-when-your-drug-is-off-label/
  19. Bang, V. et al. (2014). Awareness about and views of parents on the off-label drug use in children. Retrieved from https://www.ncbi.nlm.nih.gov/pubmed/24902503
  20. Consumer Reports. (2015, July). Off-label drug prescribing: What does it mean for you? Retrieved from https://www.consumerreports.org/cro/2012/05/off-label-drug-prescribing-what-does-it-mean-for-you/index.htm
  21. U.S. Food and Drug Administration. (n.d.). Understanding Unapproved Use of Approved Drugs “Off Label.” Retrieved from https://www.fda.gov/forpatients/other/offlabel/default.htm
  22. Gleckman, H. (2013, November 20). Dementia Patients Still Getting Dangerous Antipsychotic Drugs In Nursing Homes. Retrieved from https://www.forbes.com/sites/howardgleckman/2013/11/20/dementia-patients-still-getting-dangerous-antipsychotic-drugs-in-nursing-homes/#6229c3274dd7
  23. Christensen, J. FDA spotlights unapproved use of drugs, medical devices. Retrieved from http://www.cnn.com/2016/11/11/health/fda-hearings-off-label-drugs/index.html
  24. Devulapalli, K.K. et al. (2009, March). An analysis of the high psychotropic off-label use in psychiatric disorders. The majority of psychiatric diagnoses have no approved drug. Retrieved from https://www.ncbi.nlm.nih.gov/pubmed/23051015/
  25. Darshan, K. et al. (2015, March). Off-label drug use in Psychiatry Outpatient Department: A prospective study at a Tertiary Care Teaching Hospital. Retrieved from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4356999/
  26. Boodman, S. G. (2012, March 122). Off-Label Use of Risky Antipsychotic Drugs Raises Concerns. Retrieved from https://khn.org/news/off-label-use-of-risky-antipsychotic-drugs/
  27. Association for Medical Ethics. (n.d.). Off-Label Prescribing: Part 1. Retrieved from http://www.ethicaldoctor.org/medical-ethics/articles/off-label-prescribing/off-label-prescribing-1/
  28. National Cancer Institute. (n.d.). Off-Label Drug Use in Cancer Treatment. Retrieved from https://www.cancer.gov/about-cancer/treatment/drugs/off-label
  29. Dresser, R & Frader, J. (2009). Off-label prescribing: a call for heightened professional and government oversight. The Journal of Law, Medicine & Ethics. Retrieved from https://www.ncbi.nlm.nih.gov/pubmed/19723258
  30. American Cancer Society. (2015, March 17). Off-label Drug Use. Retrieved from https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/chemotherapy/off-label-drug-use.html
View All Sources
Who Am I Calling?

Calling this number connects you with Wilson and Peterson, LLP or one of its trusted legal partners. A law firm representative will review your case for free.

Wilson and Peterson, LLP funds Drugwatch because it supports the organization’s mission to keep people safe from dangerous drugs and medical devices.

(888) 645-1617

To contact Drugwatch Managing Editor Kevin Connolly, call (855) 839-9780.