How Many is Too Many? Off-Label Uses and RX Drugs

J0321092 There was a lot of coverage as well as sturm and drang about a study published by the Archives of Internal Medicine regarding the off-label use by prescribers of medications.  According to the report, there should be further study to see if off-label use of drugs doesn’t present a safety issue.  That seems logical and straight forward.

But the media coverage of this study tended to be a little more sensational.  One article stated "that 1 in 7 prescriptions were written without good medical evidence that they would work."  That seems very much like a conclusion reached prior to any further study to see if off-label use doesn’t present a safety issue. 

I contest that.  In my experience, physicians don’t write off-label prescriptions for the hell of it, they write them because they have, through their own study and experience, determined that there is a good, sound reason for doing so.   It is that e3xperience which would seem to be "good medical evidence" in my mind. 

I have sat through more advisory committee meetings than I care to count.  I have watched advisory committees allow drugs to the market, only to judge later that they should be taken off, only to judge later that they should be placed back on the market (Lotronex, e.g.).

I have seen advisory committees refuse an OTC switch for Zovirax because they believed that physicians examined patients with herpes to determine the infection before prescribing (they don’t). 

I have seen advisory committees have hours of discussion based on the belief that physicians follow treatment guidelines to the letter (they don’t). 

I have seen the FDA put a Black Box Warning on a label because of a demonstration of cancer in rats, only to remove it later when it appeared to be species specific and when the drug became the world’s most prescribed drug (omeprazole).

So frankly, I don’t see why I should judge the decision of my physician any less.  Actually, the "1 in 7 " was a figure I thought low, but the media attention seemed to think was terribly high, glom onto it and make some conclusions with their reporting I don’t think were justified by the nature of the study

After all, look how many uses aspirin has.  I can imagine that there are many other drugs that have multiple benefits.  In fact, there are many drugs out there that are found to be good for another ailment, even though they were originally investigated for another condition (Rogaine). 

My bottom line is this:  I’m not alarmed by the statistic that 1 in 7 drugs prescribed are for off-label use.  In fact, I think it is good.  It shows that there is innovation out there that is beyond the single-minded system of the FDA – one based on real, rather than theoretical experience.  Many of the articles stated that "good science" is lacking in these off-label uses.  I would say it differently – I would say that "different science" applies to off-label use.  It is the science of the physician’s experience and practice.  I’m not sure I trust it less or more than the FDA’s method.  I think we need them both. 

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1 Response to How Many is Too Many? Off-Label Uses and RX Drugs

  1. Off label uses of presciption drugs is a topic I could elaborate for pages. In the interest of time, I want to make three (3) points.
    First, when a physician writes a Rx for a use that has NOT met “FDA clinical trial standards” does not mean it could not meet the burden. Often, these uses are discovered and published in medical journals by university physicians. The next step is for the drug mfr to file an abbreviated PMA and supporting clinical studies. If the mfr cannot justify sufficient economic return on the additional filing – for a drug already producing good revenues for its primary use – they won’t seek new FDA uses due to economic return. In some instances, the drugs could offer less effectiveness than the approved counterparts, but also less risks and costs.
    Secondly, in my experience with Rx drugs, you can’t compare like medicines as you would “apples to apples.” One drug can offer certain benefits to certain variants of a disorder, while another drug might have its value with other patient types.
    Lastly, the best kept secret in all of medical science – is finding those little published studies in the literature that never made it into mainstream journals and use due to economics. The most expensive treatment is not always the most efficacious. For eg., I recall a 1994 Swiss study that touted using high doses of Vitamins B12 and Folic Acid to treat vitillago. No side effects, no promised efficacy, yet it proved to be well worth a trial use.
    In closing, my experiences as to when a physician tries an off-label use, he/she is merely trying a more simple treatment. Physicians tend to prescribe downwards in risks/costs with off label use, not upwards. The risks with off-label usage is often less than their approved counterparts, and could be less effective – a good trade-off if the patient is informed. Off-label use CAN result in not being reimbursed by insurance, a sad commentary when one proves to be “the right medicine.”

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