“Off Label” Medication Use: What does this mean? (Part I)

Recently, there has been a plethora of media reports about the “off label” use of medicines for the treatment of child and adolescent mental illnesses. Invariably these reports note that such use is inappropriate if not downright dangerous and often counsel parents, patients and others to refrain from using medications prescribed “off label”.

Overall the impression is that such use is not part of usual medical practice and that for some reason it is being preferentially applied with psychotropic medications. The latent argument is that physicians may be acting irresponsibly or recklessly and putting the lives of their patients at risk. But, what actually is “off-label” use? Does it only apply to medications used to treat young people who have mental disorders? Is it an aberration in medical practice? Is it an attempt by physicians to control patients rather than treat them?

The answer is complicated and goes back to understanding the historical development of systems designed to regulate the sale and distribution of medications.

Over time, regulation of prescription medications has moved from providing a degree of certainty that these compounds were relatively safe, to providing a degree of certainty that these compounds were also likely to be effective. And, believe it or not, these developments are relatively recent and continue to be refined by all national regulators (such as: Health Canada - Canada; Food and Drug

Administration – USA).

As part of this process, companies that make products that they wish to be submitted for regulatory approval gear their drug discovery and development programs to meet the standards demanded by regulators. Once a compound has met these standards it receives regulatory approval, which is accompanied by detailed information about the medication issued under the direction of the regulator. This is what is meant by the medicine being “on label”.

Following introduction into the marketplace, the regulator conducts or oversees post-marketing surveillance that is designed to identify possible safety or effectiveness concerns that initial development research was not able to address. Regulators do not dictate how medications may be used by physicians for conditions outside those identified in the regulation approval process, however, companies that make or distribute medications are not allowed to advertise their use in areas outside those already approved by regulators.

What does this mean in real life? Once a medication has received regulatory approval it is said to be “on label” for use in a particular disorder or condition. For example, medication “A” has been approved for the treatment of medical condition “Z”. However, physicians and researchers may discover that medication “A” is also effective and safe in the treatment of conditions “X” and “Y”. However, for many different reasons (such as cost, business development plans, market issues, etc.) the company holding the patent on that medication (or the generic company making that medication) decides not to conduct the studies required for registration of that medication for conditions “X” and “Y”. This means that the medication does not receive regulatory approval for the treatment of conditions “X” and “Y”, even if it has been found by independent researchers and clinicians to be effective and safe in those conditions. Thus the medicine does not receive a “label” for use in conditions “X” and “Y”.

But, the studies on the use of the medicine have been published in medical journals and experts in the treatment of those conditions have decided that in their experience the medication is a useful addition to the treatment options for that condition. Therefore, many physicians begin using that medication in those conditions – even if it has not received a “label” for use in those conditions. They are using the medication “off label”.

~ Dr. Stan Kutcher