Ten Common Questions (and Their Answers) About Off-label Drug Use

Abstract

The term off-label drug use (OLDU) is used extensively in the medical literature, continuing medical education exercises, and the media. Yet, we propose that many health care professionals have an underappreciation of its definition, prevalence, and implications. This article introduces and answers 10 questions regarding OLDU in an effort to clarify the practice's meaning, breadth of application, acceptance, and liabilities. Off-label drug use involves prescribing medications for indications, or using a dosage or dosage form, that have not been approved by the US Food and Drug Administration. Since the Food and Drug Administration does not regulate the practice of medicine, OLDU has become common. It occurs in every specialty of medicine, but it may be more common in areas of medicine in which the patient population is less likely to be included in clinical trials (eg, pediatric, pregnant, or psychiatric patients). Pharmaceutical companies are not allowed to promote their medications for an off-label use, which has lead to several large settlements for illegal marketing. To limit liability, physicians should prescribe medications only for indications that they believe are in the best interest of the patient. In addition, health care professionals should educate themselves about OLDU to weigh the risks and benefits and provide the best possible care for their patients.

Prescribing “Off-Label”: What Should a Physician Disclose?

“…Approval by the Food and Drug Administration (FDA) implies that available evidence shows that a drug is safe and effective for the specific indication (disease or symptom) for which it was tested [1]. "Off-label" drug use commonly refers to prescribing currently available medication for an indication (disease or symptom) for which it has not received FDA approval [1,2].…”

“…"Off-label" drug use commonly refers to prescribing currently available medication for an indication (disease or symptom) for which it has not received FDA approval [1,2]. Off-label use also includes prescribing a drug for a different population or age range than that in which it was clinically tested and using a different dosage or dosage form [1,2].…”

“…Once a drug is FDA-approved for a specific indication, legally it can be used for any indication [3,4]. Off-label prescribing is common; it accounts for 10 to 20 percent of all prescriptions written [5], although the practice is more common in specific patient populations like children and the elderly [1,2,5]. Physicians also might be more likely to prescribe off-label medications for patients facing life-threatening or terminal medical conditions for which there are limited or no FDA-approved alternatives [1,5].…”

“…Advances in clinical medical practice often outpace the FDA's ability to approve new drugs or relabel previously approved drugs with new indications [1,2,5]. The FDA approves only 40 to 60 percent of all drugs submitted for review, and it can take six to eight years and approximately $1.7 billion to get a new drug approved [4].…”

“…The FDA approves only 40 to 60 percent of all drugs submitted for review, and it can take six to eight years and approximately $1.7 billion to get a new drug approved [4]. Moreover, the revenue associated with relabeling a drug with additional indications might not offset the expense required to conduct the necessary clinical trials, which discourages most pharmaceutical companies from relabeling drugs once they have already been FDAapproved for one indication [1].…”