Prescription drugs go through rigorous testing before they are approved by the Federal Drug Administration (FDA) in order to ensure they are safe and effective for patients. However, once the drugs are FDA-approved, doctors can prescribe them for any reason they deem beneficial for the patient–and they can do so without telling you. This legal practice is called off-label prescribing, and it’s equal parts common and controversial.
What is off-label drug prescribing?
Off-label drug prescribing is when a doctor may prescribe a medication beyond FDA approval if the drug has been shown to work for conditions other than the one for which it was made. This includes prescribing the drug for a different age group, for a different condition, in different dosages, or for different lengths of time than the FDA has formally approved.
How common is this practice?
A study on off-label drug prescribing done in 2006 found that an estimated 21 percent of all medications prescribed in the United States are done so for off-label usage.
The following are medications commonly prescribed for off-label use.
- Metformin. Metformin is a first-defense drug used to treat high blood sugar and type 2 diabetes. A common side effect is weight loss, so many doctors prescribe it to treat conditions like obesity, polycystic ovarian syndrome (PCOS), and early stages of heart disease.
- Anticonvulsants. Medications that are used to prevent and lessen the severity of seizures are often used to treat bipolar disorder as well. Additionally, anticonvulsants may also be used to prevent migraines and treat other brain disorders.
- Gabapentin. This drug was designed to treat seizures and postherpetic neuralgia. However, it has been prescribed to treat many health conditions, including bipolar disorder, migraines, peripheral neuropathy, tremors, and restless leg syndrome.
This practice is most common in treating children, seniors, cancer patients, and people with mental illness. Medications made to treat children, seniors, and mentally ill patients often have fewer clinical studies to provide evidence that a drug works.
One reason for so many drugs being used for off-label reasons can be boiled down to saving time and money. Once a drug is approved by the FDA, having a supplemental New Drug Application (sNDA) approved could take up to a decade and millions of dollars.
Is it dangerous?
According to David Cavalla, author of the book Off-Label Prescribing: Justifying Unapproved Medicine, the practice is dangerous and unethical. “While the regulatory authorities are very strict about manufacturing standards and documentation supporting the medicines we take,” he said, “they do not regulate the way they are prescribed. This is a grave scandal at the heart of our health care system, an offense to the principle of evidence-based medicine.”
An illegal push for off-label prescribing from big pharma
Another controversial aspect to this practice is that it benefits drug companies. Although drug companies are prohibited from encouraging off-label use of drugs, many still do.
Between 2004 and 2017, several drug companies were fined $11 billion in financial criminal and civil penalties for promoting off-label uses for their drugs. One high-profile case was that of the landmark Oklahoma lawsuit against Johnson & Johnson, which was in part related to their off-label marketing of opioids.
According to a 2016 analysis, drug companies spent $20.3 billion in marketing to healthcare providers.
Trust your doctor, check with your pharmacist
Scott Gottlieb, former head of the FDA, recommends directly asking your doctor for information about the intended use of drugs prescribed to you. “Consumers should always ask their doctors for the information for the particular use of the drug,” he said. “It’s a very straightforward question: Is the drug approved for that use?”
Always check with your pharmacist before you begin using an off-label medication, even if your doctor is aware of your current medications. Your pharmacist may catch a drug interaction your doctor is unaware of, or they can tell you when and how to take the drug to make it more effective.