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Frequently Asked Questions
The most common form of OLDU involves prescribing currently available and marketed medications but for an indication (eg, a disease or a symptom) that has never received Food and Drug Administration (FDA) approval.
Off-label drug uses can become widely entrenched in clinical practice and become predominant treatments for a given clinical condition. For example, tricyclic antidepressants do not have FDA approval as a treatment for neuropathic pain, yet this class of drugs is considered a first-line treatment option. The use of aspirin provides another interesting example of OLDU. Aspirin was widely used before the introduction of the Food, Drug, and Cosmetic Act of 1938. Therefore, aspirin was grandfathered and approved as an existing drug without the rigorous testing that modern medications undergo. Currently, aspirin is FDA approved for use in patients with pain, fever, rheumatic diseases, cardiovascular diseases (eg, acute myocardial infarction, previous myocardial infarction, angina pectoris, and previous cerebrovascular disease), and a history of a revascularization procedure (eg, coronary artery bypass grafting and carotid endarterectomy). However, aspirin does not have an indication for coronary disease prophylaxis in diabetic patients, yet guidelines recommend its use in these patients. Therefore, aspirin prophylaxis for coronary disease in high-risk patients is an off-label use.
Elsewhere, medications are often prescribed for OLDU with poor or absent clinical evidence. Radley et al reported that 73% of medications prescribed for an off-label use had poor or no scientific support. In critical care patients, OLDU was without adequate evidence 48.3% of the time. Because OLDU is typically less critically evaluated than is on-label drug use, OLDU may be associated with an increase in medication errors. Rinke et al studied pediatric antidepressant drug use in a national error-reporting database and found that 77% involved off-label prescribing.
There are examples of widely practiced OLDUs in every specialty of medicine. Since the patient population in pediatrics is often excluded from clinical drug studies, examples of OLDU are especially abundant. For example, morphine has never received an FDA indication for pain treatment in children, but it is extensively used for this indication in hospitalized pediatric patients. In another example, researchers discovered in the 1970s that the nonsteroidal anti-inflammatory agent indomethacin was efficacious as a medical therapy for closing a persistent, symptomatic patent ductus arteriosus in newborns. Thus, a trial of indomethacin became the treatment of choice for many affected newborns in an attempt to avoid curative surgery. Indomethacin has never been approved for this indication and, as such, this use remains an OLDU. In addition, many inhaled bronchodilators, antimicrobials, anticonvulsants, and proton pump inhibitors are often used in the pediatric population without formal FDA approval.
The FDA has attempted to lessen the gap between FDA approval and contemporary drug-prescribing practices in pediatrics through the FDA Modernization Act of 1997. This Act created incentives, including exclusive marketing and patent extension, for pharmaceutical companies to test medications on children. Medications for psychiatric disorders are also frequently used for unapproved indications. Patients with psychiatric disorders are often excluded from clinical trials, and these disorders are inherently difficult to study. Moreover, there is often crossover in symptoms from disease state to disease state, which has lead physicians to use psychiatric medications approved for one psychiatric condition for additional unapproved indications. For example, selective serotonin reuptake inhibitors have been used off-label for rare or difficult-to-study disorders, such as borderline personality disorder, stuttering, pathologic gambling, and alcoholism. Moreover, selective serotonin reuptake inhibitors (eg, paroxetine, sertraline, and fluoxetine) are considered first-line treatments for premature ejaculation, another off-label use. In recent years, antipsychotic drug use for unapproved FDA indications has increased. Alexander et al estimated that the cost of off-label antipsychotic drug use in 2008 was $6.0 billion.
During the 1970s and 1980s, there was a proliferation of cardiac surgery to repair or replace diseased heart valves. Disease in many of these patients was the result of rheumatic abnormalities in patient populations with inadequate or no antibiotic drug treatment of infections earlier in their lives. In these patient populations, hemodynamic stability was of utmost concern during anesthesia, surgery, and the immediate postoperative course. Lowenstein reported that high-dose morphine, combined with amnestic agents, could provide the type of stable anesthetic required for these patients and that the beneficial effects of the anesthetic would continue into the postoperative intensive care period. With the later introduction of the short-acting opioid fentanyl, it was infused in doses much greater than approved by the FDA, thus converting a short-acting drug into a long-acting drug. High-dose morphine- and fentanyl-based anesthetics, highly favored therapy for valve replacement surgery, were retained as core anesthetics with the introduction of coronary artery bypass graft surgery. Today, patients are typically brought to surgery much earlier in the disease course (hence, they tend to be more stable hemodynamically), and there is a focus on shortening stays in the intensive care unit after cardiac surgery. In addition, improvements in surgical technique have shortened operation times. For these reasons, high-dose opioid anesthesia is less common than in the past, although it is still used. These high doses of morphine and fentanyl have never been approved by the FDA, and, therefore, their use has always been off-label.
Physicians have been involved in legal claims due to an adverse reaction related to a medication prescribed for an off-label use. The legal theories used in these lawsuits include unregulated use of a research drug, failure to provide adequate informed consent for an OLDU, and medical negligence. In developing legal precedents for off-label therapies, the courts have typically treated drugs and devices as coequals. As such, many of the courts’ views on OLDU have evolved from decisions regarding off-label uses of medical devices.
Research vs practice
Medical malpractice: Informed consent
Medical malpractice: Informed consent
Research vs practice
Medical malpractice: Informed consent
Medical malpractice: Negligencea
Reports to evaluate new drug therapies seeking FDA approval
Reports to evaluate off-label uses, or describe adverse effects, of drugs approved for other indications
The 1938 Food, Drug, and Cosmetic Act gave the FDA the power to regulate promotional materials on medications. Two provisions from the FDA prohibit most promotion of off-label uses of medications by pharmaceutical manufacturers and marketers. First, the FDA requires approval before distribution into interstate commerce of all medication labeling (including the package insert, print and broadcast advertisements, brochures, and patient education materials). Second, the FDA prohibits “misbranding” of medications. Misbranding includes labeling a medication with misleading information, including off-label uses.
Although pharmaceutical manufacturers are not allowed to promote off-label uses of medications, they are allowed to respond to unsolicited questions from health care professionals about off-label use and to distribute peer-reviewed publications regarding off-label use. Responses to questions regarding off-label use must be completed by the manufacturer’s medical affairs office and not their sales representatives, and interactions with the questioner must be documented.
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