Staying on Track When Prescribing Off-Label

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Am Fam Physician. 2014 Jan 1;89(1):4-5.

Using medications or medical devices for patient populations, symptoms, or diseases not officially approved by the U.S. Food and Drug Administration (FDA) is a practice commonly called off-label use. Off-label prescribing is legal in the United States under the premise that regulatory agencies do not have authority to control the practice of medicine. It occurs for many reasons, including advances in medicine that outpace the FDA's and manufacturers' ability to approve or relabel medications and devices (e.g., aspirin use for acute coronary syndromes prior to FDA approval), limited availability of study data in certain populations (e.g., children, pregnant women), limited FDA-approved alternatives (e.g., fibromyalgia symptoms), and documented effectiveness without formal approval (e.g., beta blockers for congestive heart failure). For these and other reasons, off-label use is common, accounting for approximately 10% to 20% of prescriptions.1,2

Most physicians would likely agree that off-label prescribing is an acceptable choice when there is published scientific evidence supporting it, and when the medication has a low likelihood of adverse effects and a moderate to strong likelihood of benefit. For example, the only beta blocker approved for migraine prophylaxis is propranolol, but metoprolol has been found to be equally effective and

Address correspondence to April S. Fitzgerald, MD, at afitzg10@jhmi.edu. Reprints are not available from the authors.

Author disclosure: No relevant financial affiliations.


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2. Radley DC, Finkelstein SN, Stafford RS. Off-label prescribing among office-based physicians. Arch Intern Med. 2006;166(9):1021–1026.

3. Silberstein SD, Holland S, Freitag F, Dodick DW, Argoff C, Ashman E. Evidence-based guideline update: pharmacologic treatment for episodic migraine prevention in adults: report of the Quality Standards Subcommittee of the American Academy of Neurology and the American Headache Society [published correction appears in Neurology. 2013;80(9):871]. Neurology. 2012;78(17):1337–1345.

4. Vahedi K, Taupin P, Djomby R, et al.; DIAMIG investigators. Efficacy and tolerability of acetazolamide in migraine prophylaxis: a randomized placebo-controlled trial. J Neurol. 2002;249(2):206–211.

5. Largent EA, Miller FG, Pearson SD. Going off-label without venturing off-course: evidence and ethical off-label prescribing. Arch Intern Med. 2009;169(19):1745–1747.

6. Maggioni F, Bruno M, Mainardi F, Lisotto C, Zanchin G. Migraine responsive to warfarin: an update on anticoagulant possible role in migraine prophylaxis. Neurol Sci. 2012;33(6):1447–1449.

7. Asherson RA, Giampaulo D, Singh S, Sulman L. Dramatic response of severe headaches to anticoagulation in a patient with antiphospholipid syndrome. J Clin Rheumatol. 2007;13(3):173–174.

8. O'Malley PG. What does off-label prescribing really mean? Arch Intern Med. 2012;172(10):759–760.

9. Wittich CM, Burkle CM, Lanier WL. Ten common questions (and their answers) about off-label drug use. Mayo Clin Proc. 2012;87(10):982–990.

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