Two-Horse Kentucky Derby

Currently there are only two drugs licensed for long-term obesity treatment in Canada and the US: orlistat and sibutramine.

In a talk given yesterday by Louis Aronne, (NewYork-Presbyterian/Weill Cornell) at the Obesity Meeting in Phoenix, Arizona, on the off-label use of drugs for the treatment of obesity, he likened our current, often self-imposed restrictions to using only these two prescription anti-obesity drugs in practice, to watching a Kentucky Derby with only two horses – not very exciting!

According to Aronne, fact is that using medications for the treatment of conditions for which there is no official labeling (e.g. low-dose aspirin in patients with type 2 diabetes) is widespread and has been specifically declared by the American Medical Association as an important and legitimate part of medical care.

This is particularly true in areas like pediatrics, where most drugs used in kids are not specifically “licensed” for use in infants or children.

There may in fact be ethical reasons that dictate the off-label use of medications – i.e. when there is scientific support (e.g. randomized controlled trials) but the makers of the drug, for whatever reasons, have not “officially” pursued an expansion of their label. Furthermore, as regulatory expansion of indications is a complicated and often lengthy process, it may well be very much in the interest of patients, who need immediate help (especially for “orphan” conditions), to use drugs that are clinically reasonable and/or for which there is some evidence of efficacy.

Obviously, as with any “off-label” use, this does not absolve clinicians from using their best clinical judgment and discussing the potential risks and benefits of drugs that may not be specifically licensed for that indication.

In the case of obesity, drugs that may be useful for obesity management in “off-label” use include topiramate, metformin, and fluoxitine.

Given the litigious climate in the US, Aronne was very careful to recommend that physicians should discuss the use with their lawyer, malpractice carrier and institution, check with their medical college or association and get written consent from the patient.

A word of caution: limit “off-label” use to treatments that have been well studied; only use in appropriate situations (e.g. patients who would qualify for surgery, but for whatever reason refuse or are found high risk); use good clinical judgment; present in a neutral to negative discussion of risk and benefits – as risk-aversion varies amongst patients; use low doses and go slow.

Phoenix, Arizona