In the United States, the majority of medications and legal drugs must gain approval from the ]U.S. Food and Drug Administration (FDA) before they can be prescribed to the public. The FDA approval process often takes years and can cost millions, or even hundreds of millions, for the drug's producer. Once the FDA approves a drug, it's granted approval status only for specific conditions, or indications. Any use beyond these indications is considered off-label use [source: American Cancer Society].
While it's perfectly legal for doctors to prescribe drugs for off-label conditions, the makers of these drugs must take extreme caution when it comes to selling and marketing these medications in accordance with FDA standards. Companies who market drugs for off-label uses are subject to criminal and civil penalties from the FDA, the U.S. Department of Justice and any number of other organizations.
Despite these rigid standards, as many as 20 percent of prescriptions are for off-label uses. This figure can be even higher for cancer and psychosis medications [source: Surrey]. Read on to learn about some of the most popular drugs prescribed for unique purposes in the U.S.
Montelukast (Singulair) serves as an effective treatment method for patients suffering from asthma or a variety of seasonal allergies. This drug has undergone the rigorous testing required by the FDA for both of these conditions, as well as for the treatment of allergic rhinitis. Despite the fact that the FDA only granted its approval for these specific treatments, the drug is widely used to treat a number of additional illnesses, including lung and skin disorders.
Many doctors prescribe Montelukast for patients suffering from chronic obstructive pulmonary disease (COPD), which includes emphysema and other breathing difficulties. Not only has this drug not received FDA approval for treating COPD, but there's also little scientific research available to suggest that Montelukast even relieves COPD symptoms [source: NIH]. The FDA also warns of significant psychosis risks, including an increased incidence of suicide, among patients taking this drug [source: FDA].
Montelukast is also prescribed off-label for atopic dermatitis in children. While research shows potential for this type of treatment, further studies are needed in order to seek FDA approval [source: Correale].
While many patients are aware of the anti-aging effects of Botox (OnabotulinumtoxinA) injections, few know the facts about Botox indications and off-label use. Through rigorous testing and trials, Botox earned FDA approval to treat wrinkles and excessive underarm sweating, and in 2010, it gained approval to treat stroke-induced muscle spasms in the arms and hands. The makers of Botox also market the drug heavily for the treatment of headaches, muscle stiffness and juvenile cerebral palsy.
Limited scientific studies have shown Botox to be effective at treating these conditions, but more work is needed to earn FDA approval [source: Chan]. In 2010, Botox makers agreed to a $600 million settlement with the U.S. Department of Justice in response to claims of off-label marketing, kickbacks and unethical medical billing practices [source: U.S. Department of Justice].
The blood-thinning drug Warfarin (Coumadin) earned FDA approval to treat blood clots, significant heart defects and pulmonary embolisms. While this medication hasn't undergone FDA testing for high blood pressure, it's frequently prescribed off-label for patients struggling with hypertension. Preliminary studies show that Warfarin may help lower blood pressure in some patients, but much more work is needed to gain FDA approval for this condition [source: Krishnan].
The millions of people who take Warfarin face serious health risks in terms of uncontrolled bleeding. In fact, Warfarin represents one of the most common causes of drug-related emergency room visits among seniors, and the FDA warns of life-threatening bleeding if this drug is taken incorrectly. One of the biggest concerns involves dosing, which must be calculated carefully based on the needs of each individual. Those taking this drug for off-label uses are at particularly high risk for dosing-related side effects [source: NIH].
7. Avastin and Lucentis
Avastin is a cancer drug that's been shown to be effective at treating macular degeneration, a disease that leads to reduced visibility and, eventually, blindness. This drug costs roughly $50 per dose, and while it's not FDA-approved for macular degeneration, it provides an effective and low-cost solution for patients.
Genentech, the company that produces Avastin, also manufactures the drug Lucentis. Lucentis comes with a price tag of nearly $2,000 per dose and has been FDA-approved for macular degeneration [source: ABC]. To encourage doctors to prescribe the more expensive Lucentis over its off-label alternative, Genentech offers secret rebates to doctors. While this practice isn't exactly illegal, it does cause ethical concerns for patients and those in the medical profession [source: Pollack].
Topiramate(Topamax) earned FDA approval for the treatment of epileptic seizures and certain types of headaches through extensive testing and clinical trials [source: NYU Langone Medical Center]. Despite the fact that this drug was only approved for these specific uses, it's widely prescribed for a variety of unrelated conditions, including weight loss, pain management and even alcoholism. Though there's little evidence to suggest Topiramate is effective at treating these conditions, limited research suggests it may help with mood stabilization and Tourette's syndrome [source: Jancin].
While further studies are needed to win FDA approval for using the drug to treat these or other illnesses, the potential risks of Topiramate have been well documented. This drug comes with some serious side effects, including vision changes, which plague nearly 13 percent of patients taking Topiramate [source: Cereza].
The drug Zyprexa maintains FDA approval for treating schizophrenia and bipolar disorder, yet it has been widely marketed to treat everything from depression to Alzheimer's. Even more troublesome is the fact that this drug is regularly prescribed for dementia patients, despite an FDA warning that Zyprexa could increase the risk of stroke or even death in those suffering from dementia [source: NCBI].
While some of these off-label uses may be beneficial to patients, the off-label marketing by the drug's manufacturer goes against FDA standards. In 2009, the U.S. Department of Justice announced a $1.4 billion settlement with Zyprexa manufacturer Eli Lilly. As part of the settlement, the company admitted to marketing the drug for off-label uses based on symptoms, not on approved conditions [source: U.S. Department of Justice].
In 1993, the FDA approved the drug Gabapentin (Neurontin) specifically to treat seizures, yet in 2010, more than 90 percent of use was off-label [source: Washington State Office of the Attorney General]. In addition, this drug is now out of patent, so the manufacturer has little motivation to invest in FDA testing for further approval.
Throughout the '90s, manufacturers marketed the drug for everything from nerve pain to bipolar disorders, despite little evidence that it was helpful for these types of conditions. They also used company-sponsored research and anecdotal reports to market the drug for off-label uses, resulting in a $430 million settlement in 2004 [source: Connecticut Office of the Attorney General].
3. Psychosis Medications
Psychosis medications, including drugs like Quetiapine, Seroquel, Sertaline and Risperidone, represent some of the most common drugs for off-label use in the U.S. Each of these drugs received FDA approval for schizophrenia and/or bipolar disorder, yet a staggering 86 percent of prescriptions for these drugs are for off-label conditions like dementia [source: Center for Medicare Advocacy]. In fact, nearly one in five doctors surveyed erroneously believed Seroquel to be FDA-approved to treat dementia [source: McKnight's Long-Term Care News and Assisted Living]. This widespread off-label use continues despite FDA warnings on the risk of death in dementia patients who take these drugs.
Even more alarming is the fact that nearly 26 percent of nursing home patients receive one or more of these medications, many for symptoms of dementia or general conditions that have nothing to do with the approved indications for these drugs. The Center for Medicare Advocacy estimates that 15,000 elderly people die each year from off-label use of psychosis medications [source: Center for Medicare Advocacy].
The drug Adderall earned FDA approval for the treatment of attention-deficit or hyperactivity disorders in children only. While this drug has been highly effective at treating these types of conditions, it also comes with some major side effects, including weight loss. Because of the high incidence of childhood obesity in the U.S., some doctors have taken to prescribing Adderall off-label to help overweight children shed pounds [source: Cohen].
While this type of off-label use may help change the number on the scale, it does little to help the child address the causes of obesity. When considering Adderall for obesity-related conditions, doctors and patients must balance the health risks of obesity with the potential side effects of taking the drug for off-label conditions.
Risperidone (Risperdal) is a powerful anti-psychotic drug with FDA approval for the treatment of schizophrenia. Despite the fact that the FDA hasn't granted approval for any other conditions, it's commonly used to treat illnesses unrelated to schizophrenia, including bipolar disorder, depression, dementia and even simple sleep disorders. While some studies show that Risperidone may be effective at treating obsessive-compulsive or sleep disorders, there's little scientific research to suggest that the drug is effective for dementia or other mental health patients [source: Medical News Today].
In 1994, and again in 1999, the FDA warned Risperidone manufacturers about marketing the drug for these types of off-label conditions. Despite the warnings, employees at a major Risperidone maker have admitted to marketing the drug for conditions other than schizophrenia. In 2010, the Department of Justice began an investigation into illegal kickbacks to nursing homes that used this drug on patients. At least 10 states have filed lawsuits against Risperidone makers due to these unethical or illegal practices [source: Fisk].
To learn more about these drugs and their unique uses, check out the sources on the next page.
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- ABC. "Eye doctor claims company's using unethical tactics." Nov. 8, 2010. (Nov. 15, 2010) http://abclocal.go.com/wtvg/story?section=news/local&id=7772160
- American Cancer Society. "Off-label Drug Use." Feb. 23, 2010. (Nov. 15, 2010) http://www.cancer.org/Treatment/TreatmentsandSideEffects/TreatmentTypes/Chemotherapy/off-label-drug-use
- Carlson, Bob. "Declaring War on Warfarin Misdosing." Biotechnology Healthcare. Vol. 5, No. 2. July 2008. (Nov. 15, 2010) http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2706162/
- Center for Medicare Advocacy Inc. "Off-Label Drug Use is Common and Hurts Nursing Home Residents." 2009. (Nov. 15, 2010) http://www.medicareadvocacy.org/InfoByTopic/SkilledNursingFacility/10_03.25.OffLabelDrugUse.htm
- Cereza, Gloria et al. "Topiramate in non-approved indications and acute myopia or angle closure glaucoma." Journal of Clinical Pharmacology. Vol. 60, No. 5. November 2005. (Nov. 15, 2010) http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1884934/
- Chan, V.W. et al. "Botox treatment for migraine and chronic daily headache in adolescents." Journal ofNeuroscience Nursing. Vol. 41, No. 5. October 2009. (Nov. 15, 2010) http://www.ncbi.nlm.nih.gov/pubmed/19835236
- Childs, Dan. "Adderall: Weight Loss Fix of the Stars?" ABC News. March 25, 2008. (Nov. 15, 2010) http://abcnews.go.com/Health/Diet/story?id=4515712&page=1
- Cohen, Elizabeth. "ADHD drug use for youth obesity raises ethical questions." CNN. March 21, 2007. (Nov. 15, 2010) http://articles.cnn.com/2007-03-21/health/vs.adderall_1_adhd-drugs-adhd-medications-adderall?_s=PM:HEALTH
- Connecticut Office of the Attorney General. "Attorney General Joins in 50-State Settlement With Warner-Lambert On Deceptive Marketing Of Neurontin." May 13, 2004. (Nov. 15, 2010) http://www.ct.gov/ag/cwp/view.asp?a=1779&q=284306
- Connecticut State Government. "SustiNet Childhood & Adult Obesity Task Force Regular Meeting." Meeting Minutes. June 4, 2010. (Nov. 15, 2010) http://www.ct.gov/sustinet/cwp/view.asp?a=3845&q=461820
- Correale, Christine E. et al. "Atopic Dermatitis: A Review of Diagnosis and Treatment." American Academy of Family Physicians. Sept. 15, 2009. (Nov. 15, 2010)
- Edelman, Toby S. "The War on Drugs Meets the War on Pain: Nursing Home Residents Caught in the Crossfire." Center for Medicare Advocacy Inc. March 24, 2010. (Nov. 15, 2010) http://www.canhr.org/newsroom/newdev_archive/PDF/SenateAging-nh-residents-pain03-24-10.pdf
- Federal Bureau of Investigation. "Health Care Fraud: $600 Million Settlement in Botox Case." Sept. 1, 2010. (Nov. 15, 2010) http://www.fbi.gov/news/stories/2010/september/settlement-in-botox-case/settlement-in-botox-case
- Fisk, Margaret Cronin et al. "Did J&J Plan to Break the Rules?" Bloomberg Businessweek. March 11, 2010. (Nov. 15, 2010) http://www.businessweek.com/magazine/content/10_12/b4171068582130.htm
- Jancin, Bruce. "Off-label uses of Topiramate gather supporting evidence." Clinical Psychiatry News. June 1, 2003. (Nov. 15, 2010) http://www.thefreelibrary.com/Off-label+uses+of+Topiramate+gather+supporting+evidence.+(Tourette's...-a0105368199
- Krishnan, Subramanian et al. "Warfarin Therapy and Systolic Hypertension in Men With Atrial Fibrillation." American Journal of Hypertension. Vol. 12. June 1, 2005. (Nov. 15, 2010) http://www.nature.com/ajh/journal/v18/n12/abs/ajh2005269a.html
- McKnight's Long-Term Care News and Assisted Living. "Many doctors unknowingly prescribe drugs 'off-label,' study finds." Aug. 26, 2009. (Nov. 15, 2010) http://www.mcknights.com/many-doctors-unknowingly-prescribe-drugs-off-label-study-finds/article/147284/
- Medical News Today. "No Evidence To Support Many Off-Label Uses Of Atypical Antipsychotics." Jan. 21, 2007. (Nov. 15, 2010) http://www.medicalnewstoday.com/articles/61241.php
- National Center for Biotechnology Information (NCBI). "Off-Label Use of Atypical Antipsychochotic Drugs." July 12, 2007. (Nov. 15, 2010) http://www.ncbi.nlm.nih.gov/bookshelf/br.fcgi?book=cerclin