For any of you who have been told that the FDA (Food and Drug Administration) does not approve a therapy and therefore that doctor won’t prescribe it, you are likely missing a patch of hair from where you pulled it out in frustration. You or a loved one were ill and hurting, but the FDA’s approval stood in the way of getting something that might help. You had already tried the approved meds and had minimal benefits or adverse effects, and just wanted other options. Nope, they can’t go off their script nor show some compassion. Not approved by FDA means they are not going to write it.
But wait, does the FDA really control what doctors are allowed to prescribe or test? If so, then a lot of providers are in big trouble looking at the following statistics. Some would estimate that 20-30% of prescriptions are written for off-label uses. In 1 review noted by the article, 46% of cardiovascular meds are for off label uses. In fact, as the article states “Off-label use does not imply a contraindicated or illegal use.” It actually just means that the producing company cannot market that drug or device for an unapproved use.
To get FDA approval, a company must run studies concerning safety and efficacy for a given condition. Once approval is given in that case, prescribers can choose to use the medication for other conditions. Some of these off-label uses eventually get studied and approved later. In the meantime, ethically and to comply with common sense, these off-label uses should have scientific support rather than being haphazard guesses at therapy. Very often health care providers discover other uses beyond the approved indications as better understanding of the medication’s effects and side effects develop with repeated widespread use. We have a number of diabetes medications now being touted as weight loss drugs thanks to such recognition.
In today’s study, I appreciate the author’s emphases on criteria for a provider to prescribe an off-label medication, but I would say they set the bar a little too high with some of their criteria. I agree with having reasonable studies to support the efficacy and safety of the therapy in a given condition. I agree that approved alternatives should be considered first if possible. I agree with asking if the potential benefits are worth the uncertainty and risk? On the other hand, I do not agree that one needs agreement from published clinical guidelines or reference handbooks. The political nature of medical organizations can hinder advances while the delay from content finalization of a reference book till the actual distribution means that the recommendations may already be out of date by the time it prints.
Again, the FDA does not control medicine or providers; it just influences the practice. Approved drugs will mostly be the first line therapies for common conditions. However, when first line therapies fail, eventually the continued suffering of the patient begs for further help that may necessitate off-label therapies. Also, when conditions tend on the rare side of prevalence, there may be a limited number of approved therapies available, as no drug company is willing to develop a medicine without a significant market.
Helping our patients who have exhausted the options offered by mainstream medicine means utilizing the best therapies that will most likely help them restore health, whether FDA approved or not. The next time your provider refuses to prescribe a therapy because it is not FDA approved, you can ask if they have prescribed other off-label therapies before. Then ask for a discussion of the risk benefits of the therapy for you rather than a debate over the FDA’s stranglehold on their practice of medicine.
Article in focus:
Van Norman GA. Off-Label Use vs Off-Label Marketing of Drugs: Part 1: Off-Label Use-Patient Harms and Prescriber Responsibilities. JACC Basic Transl Sci. 2023 Feb 27;8(2):224-233. doi: 10.1016/j.jacbts.2022.12.011. PMID: 36908673; PMCID: PMC9998554.
Medical Disclaimer:
This article is intended for educational purposes only. I have attempted to confirm the accuracy of these statements but with time, some currently true statements may be proven incorrect. Before making any medical decisions, you should consult your medical provider. I am not a lawyer nor am I a remediator and cannot provide definitive answers in either category. Discuss legal and remediation decisions with appropriate professionals. Sanctuary is not responsible for decisions you make based on this short video.
Sanctuary Functional Medicine, under the direction of Dr Eric Potter, IFMCP MD, provides functional medicine services to Nashville, Middle Tennessee and beyond. We frequently treat patients from Kentucky, Alabama, Mississippi, Georgia, Ohio, Indiana, and more... offering the hope of healthier more abundant lives to those with chronic illness.
