Is Off-Label Medication Safe?

colorful pills spread on table - is off-label medication safeMillions of Americans take prescription medications to treat their diseases and improve their health. It’s easy to assume that the medication your doctor prescribes for you has been approved for your particular ailment. But sometimes, doctors prescribe medications that have not been approved for your ailment, a practice referred to as off-label medications. Is off-label medication safe?

The FDA approval process.

The FDA approves medications based on clinical data and other information provided by the drug manufacturer. In order to win approval, the drug company has to demonstrate that the drug is safe and effective for its intended uses. By the way, you should know that “safe” doesn’t mean no side effects. Rather, FDA approval indicates that after careful evaluation, they determined the benefits of a medication for a particular use outweigh the potential risks. In other words, the FDA feels there is strong scientific data supporting the use of the drug for its intended purpose.

Additionally, the FDA approves drug labeling for each medication that provides healthcare providers with important information about how to safely and effectively use each medication. Information on the labels includes:

  • The specific diseases and conditions for which the FDA approves the drug.
  • How to use the medication to treat patients with those specific diseases and conditions.
  • The risks of the drug.
  • Information that healthcare providers should discuss with patients before they use the medication.

Off-label use.

It’s legal and scary.  But, after the FDA approves a medication for its intended use, doctors can prescribe the drug for any other disease or ailment. Furthermore, doctors can prescribe a medication in different dosages and/or to other age groups than those approved by the FDA. Doctors decide how and when to use any medication. And your doctor doesn’t even have to tell you he/she is recommending a medication outside of FDA guidelines.

Why do doctors prescribe off-label medications?

Doctors may prescribe off-label medications when they think it’s medically appropriate for their patients. In what circumstances? Usually this practice occurs when:

  • Patients have a disease or medical condition for which there is no approved medication.
  • Doctors have tried all approved treatments without seeing any benefits.

How do doctors decide to use an off-label medication?

There are several ways in which doctors determine that a medication might help patients with ailments other than the FDA approved purpose, including:

  • Some doctors thoroughly understand the science of pharmacology, helping them determine which medications to use off-label.
  • Doctors notice unexpected effects of a medication and realize it could help other patients.
  • Doctors learn about off-label uses by talking to colleagues, listening to patients, attending medical conferences and reading medical journals.

What types of patients are likely to receive off-label medications?

photo child in hospital with doctorThere are several categories of patients for which doctors are more likely to prescribe off-label medications. Since there are limited clinical trials for patients under 18 or over 65, doctors often prescribe off-label medications for pediatric and geriatric patients. Additionally, end-stage cancer patients with few options often try riskier, off-label drugs. And, mental health patients frequently try off-label drugs because there is a lack of official treatments for many mental health disorders. Finally, the thousands of rare diseases which have few, if any, FDA approved options often lead to the use of off-label drugs.

How common is off-label use?

Research conducted in 2001 estimated that in the US, 21% of prescriptions written in outpatient offices were for off-label uses. Unfortunately, there is little research on this topic, which should concern us all due to the risks involved.

Is off-label medication safe?

Using off-label drugs is risky, potentially leading to patient harm. Experts worry about the lack of rigorous scientific study for off-label use. Unfortunately, research is rarely conducted to test the effectiveness and safety of drugs used for purposes other than their intended uses. Importantly, one study found that 73% of off-label drug use had little or no scientific evidence to support the choice.

David Cavalla wrote about his concerns of the practice in his 2014 book Off-Label Prescribing: Justifying Unapproved Medicine. He writes that “while the regulatory authorities are very strict about the manufacturing standards and documentation supporting the medicines we take, 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.”

However, in some cases, off-label use is supported by scientific evidence. For instance, a clinical trial may demonstrate secondary benefits. Or, a drug manufacturer may conduct (or fund) additional studies, past the original scope, that show benefits for other conditions.

What about the drug companies?

photo of grey and yellow pills on top of money - is off-label medication safeThe FDA has a process for approving a medication for additional uses. However, the process, called the supplemental New Drug Application (sNDA), can take a year and cost tens of millions of dollars. Therefore, drug companies are reluctant to take this costly and time-consuming step if doctors already prescribe their medication for off-label.

Importantly, off-label use is highly lucrative for the pharmaceutical industry. It makes perfect sense – the more patients using a drug, the more the pharmaceutical company earns. And, although the FDA does not generally allow drug companies to promote off-label uses, they continue to do so. In fact, a 2018 report showed that from 2004 to 2017, drug companies paid over $11 billion in criminal and civil penalties for unlawful product promotion, including the off-label uses of drugs.

How serious and potentially dangerous is this problem? In August 2019, Johnson & Johnson was fined $572 million, in part related to the intense marketing of opioids for off-label use.

Do drug companies influence doctors?

The enormous fines have not stopped the marketing of drugs for off-label uses. Because the potential for profit is huge, some companies see the FDA fines as a cost of doing business. Although it appears that drug companies are less aggressively marketing drugs for off-label use, it still occurs. Simply put, drug companies influence the prescription habits of doctors.

As mentioned above, the FDA restricts off-label marketing. But the FDA does allow drug companies to respond to unsolicited questions about off-label use from doctors. Additionally, the FDA allows drug companies to distribute peer-reviewed article reprints discussing off-label uses, and to sponsor impartial continuing medical education courses.

Furthermore, some drug companies skirt the rules. Drug sales reps cue doctors to ask off-label questions, opening the door to conversation. Drug companies organize seminars and conferences featuring their products. And sometimes they provide free samples, free meals and direct payments to doctors for speaking appearances or other services.

Is your doctor influenced by drug companies? Read this blog post to learn more: Pharmaceutical Companies Influence our Doctors’ Medication Recommendations

What should you do?

photo of doctor showing pregnant patient bottle of pills - is off-label medication safeAlthough not all uses of off-label drugs are dangerous, many are unproven and the potential for harm is real. Therefore, when your doctor prescribes a new medication, ask your doctor if the medication is FDA approved for your diagnosis. If your doctor tells you that the medication is not approved, ask these questions suggested by the FDA:

  • What is the drug approved for?
  • Are there other drugs or therapies approved to treat my medical condition?
  • What scientific studies support the use of this drug to treat my medical condition?
  • Is it likely that this drug will work better to treat my medical condition than using an approved treatment?
  • What are the potential benefits and risks of treating my medical condition with this drug?
  • Will my health insurance cover treatment of my medical condition with this drug?
  • Are there any clinical trials studying the use of this drug for my medical condition that I could enroll in?
Want to look up medications you currently take?

Use to learn if the medications you’re taking are FDA approved for your condition. Type in the name of the medication (be specific regarding dosage and type). Then scroll down and click on “Indications and Usages” to see if your diagnosis is listed. Beware – this site contains a lot of medical jargon. If you have any questions, ask your doctor or pharmacist.

Learn more….

Because medication issues are serious and potentially deadly, I encourage you to read these posts:


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