Off-label Use: What It Is, Why It Happens, and What You Need to Know

When a doctor prescribes a medication for something off-label use, the practice of using a drug for a purpose not approved by the FDA. Also known as unapproved use, it happens every day in clinics across the country. The FDA approves drugs for specific conditions based on clinical trials, but that doesn’t mean those are the only ways they can help. Many drugs work on multiple systems in the body — and smart doctors use them that way.

Think of prescription drugs, medications legally dispensed by a doctor to treat or manage a health condition. They’re tested for safety and effectiveness in one setting — say, depression or high blood pressure — but once they’re on the market, doctors can use them however they see fit. That’s because the FDA regulates drug approval, the official process that determines what a drug can be marketed for, not how doctors practice medicine. It’s legal. It’s common. And in many cases, it’s the best option.

For example, amitriptyline is approved for depression, but it’s often used for nerve pain. Gabapentin? Approved for seizures, but widely prescribed for chronic pain and anxiety. Even Viagra was originally developed for angina before its real use was discovered. These aren’t outliers — they’re routine. In fact, studies show that one in five prescriptions written in the U.S. is for off-label use, and it’s even higher in cancer, pediatrics, and psychiatry.

Why does this happen? Sometimes, there’s no approved drug for a condition. Other times, approved options are too expensive, too risky, or don’t work for a patient. Off-label use fills those gaps. It’s not guesswork — it’s often based on peer-reviewed studies, clinical experience, and guidelines from medical societies. But it’s not risk-free. Without formal approval, side effects might not be fully documented for that use. Insurance might not cover it. And patients need to be clear on why their doctor chose this path.

What you’ll find in the posts below are real-world examples of how off-label use shows up in everyday care. From using a blood pressure pill to treat migraine, to giving a cancer drug for a rare autoimmune condition, these aren’t theoretical scenarios. They’re decisions made by doctors, backed by evidence, and lived by patients. You’ll see how medication safety, the practice of using drugs in a way that minimizes harm and maximizes benefit plays out when the label doesn’t match the need. You’ll learn when it’s smart, when it’s risky, and how to ask the right questions if it’s offered to you.