Lockup Doc

The prescription of medications for diagnoses, to age groups, or in doses other than the FDA-approved ones, is a common, legal, and necessary practice in medicine today. This practice is known as off-label use or off-label prescribing. Those new to this issue may wonder why physicians and other prescribers would ever deviate from prescribing in territory where the FDA has not given their stamp of approval.

In the ideal world, clinical trials would be conducted for all patient populations and diagnoses for which various medications might be useful. In reality, though, this is neither practical nor possible. Consequently, many patient populations and many patients with particular diagnoses would have few treatment options available if the medical establishment avoided off-label prescribing.

For example, child psychiatry is a huge area where millions of mentally ill children and adolescents depend on off-label medications. The field of general pediatrics is no different. The fact is that most medications are not tested in children. But, does that mean that children should be deprived medical treatments that may be helpful or life-saving? What’s interesting is that a child psychiatrist may be practicing outside of the community standard of care by not treating a patient with one of several commonly-established treatments that happens not be be FDA-approved.

Patients who have developmental disabilities are another example. What if a person with autism is a danger to himself and others because of aggressive behavior?  He may be at risk for losing his community placement and end up spending months in a psychiatric hospital. Once all of the non-medication interventions such as applied behavior analysis have been tried and his diagnosis has been clarified as much as possible, medications should be considered. Currently risperidone and aripiprazole have FDA approval for “irritability” related to autism. After that, though, the patient would be out of luck if his psychiatrist did not consider off-label alternatives.

It would be easy to fill many pages with examples of patients who depend on off-label prescribing. At the same time, I realize that there are many critics of the practice. The main criticism I hear is that much off-label prescribing is not evidence-based.

I want to make it very clear that I do not oppose the practice of evidence-based medicine. We need to pay attention to clinical trial evidence as much as possible in daily practice. The practical problem from my personal viewpoint as a physician, and especially as a psychiatrist, is that the research-based evidence for what to do for the patient in front of me often is minimal or nonexistent.

What if your doctor said to you, “Well, I’m sorry, but you’ve already tried the medications that the FDA has approved for your condition, and they didn’t work. So, I can’t help you.” I’m guessing that most people would not like to hear that.

Or, would you rather hear, “Well, you’ve been through quite a few medications for your condition. Unfortunately they haven’t helped you. Although those were the only ones approved by the FDA for what you’ve got, there are some other ones that physicians in my specialty often use quite successfully in your situation. I’ll tell you more about your options, including the risks, benefits, and potential side effects. Would you like to discuss this?”

My hope right now is that we as a medical profession can appropriately scrutinize the common practice of off-label prescribing and ensure that we are being as evidence-based as possible. My fear, especially in the current context of what some see as the “cleaning up” of the practice of medicine as health care reform moves forward, is that naive ivory-tower-dwelling purists and bureaucrats will further damage the practice of medicine by making it impossible or impractical for off-label prescribing to continue. That, folks, will affect you and your families, just when you may desperately need whatever is in the medical toolbox.

As I learned in medical school, “always” and “never” are usually the wrong answers to test questions. Nor are they the right answer to most of life’s questions…