Why Doctors Prescribe Medicines for Non-Approved Uses

Non-Approved Use Can Represent the Standard of Care

05.16.13 | By Stephanie Fischer

A recent article in Scientific American which explains “Why Doctors Prescribe Off-Label Drugs” caught my attention since non-approved use, also known as off-label use, of prescription medicines is often mentioned in the news without the context of why doctors choose to recommend it.

As the Scientific American article explains, prescription medicines go through a lengthy clinical trial process to demonstrate safety and efficacy before the U.S. Food and Drug Administration (FDA) reviews and can approve them for certain indications. 

Once a medicine has been reviewed by the FDA and found to be safe and effective for use for one or more indications, physicians may prescribe a medicine for something other than the approved indication(s).  In some cases, non-approved use of a medication is the standard of care, well-validated in practice but not by the clinical trials required for FDA approval of new indications. 

In fact, prescribing a medicine for non-approved uses is common. WebMD estimates that more than 20% of outpatient prescriptions are for non-approved uses of an approved medicine.

The four main reasons for non-approved use cited in the Scientific American article include:

  1. Evidence is good and consistent:  Independent studies may support the use of a medicine to treat a certain condition, but the large clinical trials required for FDA approval may not be feasible.
  2. FDA-approved options are lacking:  Many conditions, such as most rare diseases, do not have FDA-approved treatment options so physicians may try medicines approved for other conditions.
  3. No drug is 100 percent effective: We are moving towards personalized medicine which will allow physicians can better find the right drug at the right dose at the right time for each patient.  In the meantime, physicians may need to try medicines approved for one condition on patients with another condition who haven’t responded to any FDA-approved treatment options.
  4. Most drugs are not approved for children:  The Best Pharmaceuticals for Children Act and Pediatric Research Equity Act have been successful in driving research on use of innovative medicines in pediatric patients (and their recent permanent reauthorization provides certainty to biopharmaceutical companies pursuing pediatric drug development).  Since their passage, there has been a significant increase in useful information for administering drugs in children including information on dosing, safety and efficacy. According to the FDA, as of 2008, an estimated 50-60 percent of prescription drugs used to treat children have now been studied in some part of the pediatric population.  Physicians may determine that medicines which were not approved for use in children are an appropriate therapeutic option for their pediatric patients.

All of the medicines I have taken for my rare disease have been prescribed off-label since there are no therapies approved to treat it (which is the case for the vast majority of rare diseases).  It is unlikely that a drug company would recruit enough participants for a clinical trial to generate the data necessary for FDA approval for use in a rare disease (affecting fewer than 200,000 patients in the U.S.).




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