Antidepressants for alcoholism, oral contraceptives for menstrual disorders, stimulants for weight loss; all of these are examples of off-label drug prescription use. It's a common practice, but is it safe or is it misuse or even abuse? The answer is, that depends.
According to the FDA, when a drug is prescribed for its intended use, patients can be sure that the FDA has conducted a careful evaluation of its benefits and risks for that use. The doctor's decision to prescribe the drug is supported by strong scientific data, and there is approved labeling for health care providers, including pharmacists, with which to inform patients about the drug, its proper use and possible side effects.
That's not the case with off-label drug use. Studies show 73 percent of medications prescribed for an off-label use have little or no scientific support1. The FDA has not evaluated it for that type of usage, so there's no determination of safety or effectiveness. And, there may be health policy, regulatory, ethical, medicolegal, and economic implications associated with off-label prescribing2.
Yet, off-label prescribing is legal and extremely widespread; and, it contributes to ever-growing drug expenditures. Why? Increased marketing of the drug to both patients and physicians, patient demand for the drug, and rapidly changing standards of practice.
Some of the most common areas of off-label uses include:
- Seroquel (quetiapine) is FDA indicated for the treatment of schizophrenia, depression, and bipolar disorder, but is often prescribed off-label for the treatment of insomnia because it is a generic drug and not a controlled substance.
- Provigil/Nuvigil (modafinil/armodafinil) is FDA indicated for use in patients with narcolepsy, obstructive sleep apnea (OSA), or shift work disorder (SWD). It is sometimes prescribed off-label for treatment of age-related memory decline, ADD, depression, post-anesthesia fatigue, and many other lifestyle issues like work-induced stress and jet lag.
- Victoza is FDA indicated only for the management of Type 2 Diabetes. It is prescribed off-label for weight loss management, particularly in plans where weight loss agents are excluded. Saxenda—the same medication with a different name and slightly altered dosing—is indicated for weight loss.
- Oral Fentanyl Products (Abstral, Actiq, Subsys, etc) are FDA indicated specifically for the control of breakthrough pain in patients with cancer. These medications are prescribed off-label for pain management in patients without cancer which is not only an off-label use but also a violation of FDA's REMS program guidelines.
- Naltrexone is FDA-indicated for treatment of alcohol and opioid dependence. It is prescribed off-label for management of eating disorders and pathological gambling.
- Factor Xa Inhibitors (Eliquis, Savaysa, Arixtra, Xarelto) are FDA indicated for the treatment of deep vein thrombosis (DVT) and prevention of DVT in patients with atrial fibrillation. Given these medications' potent inhibition of coagulation, they have grown in use for management of strokes and other vascular disorders.
Pharmacists come across off-label use every day, and sometimes they're faced with judgment calls as they are the final safety check before the prescription is dispensed. It is important to be familiar with off-label prescribing, especially for pain medications, in order to promote patient safety through proper monitoring and individual assessment. This is where utilization management protocols, such as prior authorization (PA) and retrospective drug utilization review (RetroDUR), bring value to stakeholders across the care continuum. The whole point of prior authorization is to make sure the prescribed medication is appropriate and necessary. Meanwhile, RetroDUR provides an opportunity for prescriber engagement to identify patients at risk based on diagnosis and medication data. But, how does off-label use fit in with these programs? Is it even allowed?
Oftentimes, it depends on the health benefit plan. Off-label prescribing by physicians is legal3. However, some health plans limit coverage of drugs to FDA-approved uses, or off-label uses that have solid scientific data behind them. If you're an organization responsible for the expense of prescription drugs, limiting your expense to FDA-approved medications and indications seems like a reasonable course of action.
Bottom line: Off-label prescribing is here to stay. Evidence-based prior authorization criteria leveraged within an objective and independent prior authorization solution and prescriber engagement through RetroDUR programming are the best ways to make sure the right patients are getting the right drugs at the right time for the right price. At Health Information Designs, we're committed to making that happen.
Clinical support for this piece was provided by Adam Syfrett, PharmD, MBA.
- Radley D.C., Finkelstein S.N., Stafford R.S. Off-label prescribing among office-based physicians. Arch Intern Med. 2006;166(9):1021–1026.
- Demonaco HJ, Ali A, Hippel E. The major role of clinicians in the discovery of off-label drug therapies. Pharmacotherapy. 2006;26:323-332.
- Blum RS. Legal considerations in off-label medication prescribing. Arch Intern Med. 2002;162:1777-1779.