Sooooo if you read the title and thought, “Who’s MacGyver?” then I’m sorry but you need to Google it. It may show my age, but it’s the perfect analogy for this blog. Basically, MacGyver was a famous TV show, where the main character, aka MacGyver, was the ultimate improvisor who turned ordinary tools into extraordinary solutions to get out of a dangerous or sticky situation. Got a stick of gum or a paperclip? He could make a mean solution out of the most random items.
Dermatologists are medical MacGyvers and often have to creatively repurpose existing medications to solve clinical problems when no perfect FDA-approved option exists. We call this off-label prescribing. With over 3000 dermatologic conditions, some of which are uncommon or rare, we don’t have the luxury of having an FDA-approved medication for everything we treat. So, we put on our thinking caps and see what tools or medications might help our patients even if they aren’t FDA approved for that particular skin condition.
Off-label is defined as the practice of prescribing a medication that is not used for its approved indication, dosage or frequency. For example, if you are taking a blood pressure pill only once a day yet it is FDA approved for twice a day, then you are technically taking it off-label. Or if you take a drug like spironolactone for acne, it is considered off-label as well since it does not have an FDA indication for acne.
The term off-label might sound a little “off-putting” but it’s common practice. Off-label does not mean experimental or illegal. The FDA allows clinicians to prescribe approved drugs for medically appropriate unapproved uses. The dilemma may come with insurance coverage of off-label medications. A prescription plan may deny coverage for a drug if it is not written on-label. Ironically, they may also decline coverage for an on-label drug if a cheaper option that is off-label could be used.
Now are you wondering why wouldn’t a drug get FDA approval for a particular medical condition? Well, in large part due to cost. Running a clinical trial, the process by which drugs receive FDA approval, is an expensive process, with the median cost of a clinical trial at a whopping $19 million per study. This cost makes drug manufacturers weigh the investment to seek FDA approval. In addition, some off-label medications are generic so the profit margin of seeking another approval isn’t worth the cost of the process.
And there is risk to off-label prescribing as well. Sometimes the evidence is weak or there is a publication bias. A patient’s positive result or anecdotal experience might bring overconfidence in a medication’s ability to perform. Unexpected side effects, drug interactions and lack of standardized monitoring can also be challenges when prescribing off-label.
Dermatologists have always been a little creative. We see a medication designed for one job and sometimes think, “But what else can this do?” That’s the basic idea behind off-label prescribing. But there’s a big difference between creative medicine and throwing spaghetti at the wall—and that difference is evidence. The evidence can range from well-designed trials to previous patient accounts or be more formal like published case reports or case series. Sometimes the evidence rests solely in understanding the mechanism behind the off-label medication and the skin condition.
That’s the spirit of off label-medicine: understanding the mechanism, recognizing clinical signs, examining the evidence and using our judgement when the label doesn’t tell the whole story. Off-label can be innovative. It can also be irresponsible. The difference is the evidence, transparency with patients and a pledge to uphold our promise to “Do no harm.”
Trotter’s Take: Off-label prescribing is a necessary practice that can benefit patients and truly personifies the art of medicine.
Thinking of going rogue yourself? Then you need to check out my podcast with Dr. Victoria Farley, where she discusses the art of off-label prescribing in dermatology.
