Have you ever heard of off-label drugs? No? Well, the FDA describes them as an “FDA-approved drug for an unapproved use to treat your disease or medical condition.” Sounds harmless at first pass but it is anything but risk-free. Currently, 21% of all medications prescribed are for uses not approved by the FDA. And, according to the January 2016 JAMA Network journal by Drs. Eguale, Buckeridge, Verma, et al, the side effects for off-label drugs is 44 percent higher than on-label drugs.
Avastin is just one example of an off-label drug. The FDA first approved this drug in 2004 for the treatment of various cancers. At the time it was thought the drug’s benefits outweighed its side effects. (Remember when a drug is declared safe by the FDA during its careful evaluation process the word “safe” does not mean free from side effects.) Yet a few years later the FDA decided the drug was not safe for all its approved uses and the risks outweighed its benefit. Avastin was then pulled from the market for breast cancer treatment but remained on the market for other cancer treatments and for use as an off-label drug eye injection to slow vision loss in people with wet age-related macular degeneration. Since it was off-label there was no need to submit paperwork for review by the FDA. It could just be simply used in a way other than what was originally submitted to the FDA. And the results have caused law firms to set up lawsuits. Of note, from 2014 – 2022, the FDA reports that 43,652 serious cases were reported including 14,292 deaths. Is this risk as demonstrated by these numbers of serious cases and death worth having an injection in the eye to slow down the loss of vision? Perhaps to some and definitely not to others. After all, you must look at the total number of injections being given each year for this drug to decide if these numbers are too high a risk. There are those who might say even the loss of one person is too high a number to pay. And there are those who will claim that with over seven billion dollars in sales of this solution each year in the United States the drug is considered “safe”. Although Avastin is used as an example you must remember that more than one-fifth of all drugs are off-label drugs as well. Other examples include allergy medicine diphenhydramine. It is used off-label for chemotherapy-related emesis and insomnia and has listed 11,848 serious cases with the FDA and 8,212 deaths from 2008-2019. And even Bactrum and Amoxicillin are off-label drugs. Do their benefits outweigh their risks? And are off-label drugs necessarily bad? The answer is no off-label drugs are not necessarily bad but it can be argued they should be drugs that are converted back to “on-label” status with the same scrutiny process that the drug originally had to undergo to become approved. This would create a greater calm in people when taking it or deciding whether or not to take it. Yet despite the need, according to nih.gov obtaining a new FDA approval for medication can be costly and time-consuming, it can be a burden for physicians to have to disclose to each patient and it may “unduly frighten patients”. Knowledge is not frightening. It is the unknown we need to be scared of in our lives. This is why the FDA created black box warnings on drugs to let the patient know the drug poses a significant risk. But unfortunately, patients usually are not privy to this medication box information. They must rely on the information they are given by their doctor. But wait there is help. If your doctor recommends you take an injection or any type of medicine go to the Internet first and check on the FDA site https://www.fda.gov/drugs/questions-and-answersfdas-adverse-event-reporting-system-faers/fda-adverse-eventreporting-system-faers-public-dashboard. This site was created and is maintained to give you all the information on adverse effects of a drug so you can open up an informed dialog with your doctor to make sure your off-label drug’s benefits outweigh the risks.