Pharmaceutical Litigation – Are Off Label Drugs Off Limits?

The term “off label drug use (OLDU)” refers to the practice of doctors prescribing medications for a condition, use or category of patients, that has not been approved by the FDA. All prescription medications sold in the United States must be approved by the Federal Drug Administration (FDA). The approval process can be long, complicated and expensive, but basically involves the manufacturer of the drug subjecting the medication to clinical trials and then reporting the results of this testing to the FDA.   The drug manufacturer must prove to the FDA that the drug is 1) Safe, and 2) Effective.  The drug manufacturer also has an ongoing obligation to report to the FDA any adverse incidents of which it becomes aware related to the use of its drug. A doctor has the right to prescribe any FDA approved medication for any purpose, even if it is not the original purpose approved by the FDA, so long as in her independent medical judgment it is in the best interest of the patient.

Off Label Drug Use is common in areas of medicine where the patient population is less likely to be included in clinical trials (i.e. pregnant, psychiatric and pediatric). It is also seen often when a life-threatening or terminal condition is present, motivating a health care provider to try any treatment that might work or bring relief to the patient. Finally, physicians commonly prescribe a medication that does not have FDA approval if others from its class do have the approval. An example of an Off Label drug use is morphine for children; morphine has never received an FDA indication for pain treatment in children, but it is extensively used for this in hospitalized pediatric patients.

It is estimated that 1 in 5 prescriptions are Off Label.

Often times, pharmaceutical companies choose not to have an Off Label drug approved for a new usage, due to the expense and time it takes. However, if a specific use is considered off label, the pharmaceutical companies are not allowed to promote or market their medications for this unapproved off-label use, which has lead to several large settlements for illegal marketing. One recent example is with Zofran, a prescription medication made by GlaxoSmithKline, used to treat nausea and vomiting. The FDA approved Zofran to treat cancer patients who were suffering with the side effects of chemotherapy or surgery.  Glaxo convinced doctors that Zofran would be an effective and safe drug to treat their pregnant patients suffering from morning sickness. We now know that Zofran crosses the placental barrier exposing the developing baby to the drug. Making matters worse is the fact that most women experience morning sickness during their first trimester exactly when the fetus is undergoing rapid and critical development.   Thousands of lawsuits have been filed alleging that the use of Zofran during pregnancy is unsafe and can result in significant birth defects to the developing baby including cleft palate and lip, mouth deformities, heart defects and club foot.

Back in 2012, GlaxoSmithKline agreed to pay a $3 billion fine (the largest health-care fraud settlement in U.S. history at the time) for what the Government considered fraudulent and illegal marketing practices related to several of its drugs, including Zofran and the antidepressant Paxil. Glaxo had been promoting the use of Paxil for children and adolescents even though it was never approved for this age group and studies had found it was ineffective and potentially dangerous for kids. GSK was also charged with marketing the antidepressant Wellbutrin for weight loss and the treatment of sexual dysfunction, substance addictions, and other unapproved uses. Some experts argue that pharmaceutical companies might view such fines, although hefty, as merely a necessary cost of doing business.

One of the strongest criticisms levied against the makers of Zofran is that they promoted the use of their drug to doctors for an unapproved “off label” use without warning the medical community or the public about potential side effects that they should have been aware of. When a doctor is considering an off label use for one of her patients we want that physician to have as much good information as possible.   The prescribing of drugs by doctors for off label uses is complicated by the fact that drug manufacturers market and promote their use for such purposes by paying doctors to be spokespersons for the company or attempt to educate doctors by putting on medical conferences where they “educate” physicians about other potential uses for their products. The decision of whether to prescribe a certain drug for an off label use should hinge on one thing: what is best for the patient.  When large drug manufacturers start paying doctors for their opinions there is the danger that this decision could be tainted by the undue influence of the drug manufacturer rather than the doctor’s independent medical judgment.

The United States Department of Justice filed criminal and civil charges against GlaxoSmithKline related to how it was marketing and selling various drugs as well as paying doctors for their participation in this scheme.

So How Can We Protect Ourselves?

Physicians are not mandated to disclose the fact that they are prescribing a medication for an Off Label to their patients. Experts feel that it could confuse and frighten patients, plus it would place an extensive burden on the physician to stay current on current risks and benefits. Most people assume that if a doctor prescribes a medication, then the FDA has approved it for that specific ailment. While legal (and common!), drugs prescribed off label come with both benefits and challenges for consumers as well as doctors. Off label prescribing can be helpful if the medical community finds that a drug is effective in treating an unapproved condition or patient population. Off label use allows doctors and patients to benefit from this new use immediately without the delay of more clinical trials and approval by the FDA. However, doctors and patients need to understand that when a drug is used in such a manner it has not been subjected to the same testing and rigorous scrutiny as an approved use drug.

So how can we stay informed?

  • When your doctor prescribes medication, ask if it’s an approved use or off label. If he or she doesn’t know, ask your pharmacist.Check for yourself. Regardless of what your doctor says, do your own homework. Studies show that often times doctors didn’t realize they were prescribing a drug off label. Read the label, package insert or go to a web site such as to learn about what you are putting into your body.
  • If you find that you are taking a medication for an off-label use, ask your doctor if it’s supported by well-designed trials showing significant improvement for people with your condition.
  • Ask your doctor why she thinks the drug will work better than approved drugs for your illness.
  • Finally, find out if your insurance covers payment for the off-label use. Some may require evidence of effectiveness or failure with conventional treatments, especially if the drug is expensive.