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Off-Label Drug Use

By April 25, 2011Commentary

Two articles in the Annals of Internal Medicine discuss the off-label use of Recombinant Factor VIIa, which is intended for patients with hemophilia.  Once a product is approved for any use by the Food & Drug Administration, doctors have a fair amount of flexibility in being able to prescribe it for purposes other than those for which it was tested and approved.  In the case of Factor VIIa, one of the studies found that from 2000 to 2008, off-label use in hospitals increased by 140 times, and 97% of the prescriptions were off-label.  It was being used for body and brain trauma, cardiovascular surgery and intracranial hemorrhage, among other things, indications for which there was little if any clinical trial evidence.  Mortality in all cases where the drug was prescribed in the hospital was 27%.  (Annals Study I)

The second study on the same drug examined outcomes in greater detail.  It surveyed the evidence for five off-label uses of Factor VIIa–intracranial hemorrhage, cardiac surgery, trauma, liver transplants and prostatectomy.  A total of about 64 studies were found.  Across the indications minimal additional benefit was found from using Factor VIIa in the treatment and for some indications there was a heightened risk of adverse events such as thromboembolism.  Overall the amount and quality of research on these off-label uses was poor.  (Annals Study II)

The misuse of Recombinant Factor VIIa may be an extreme, but is not an isolated, case of prescription drugs being used extensively for indications other than those they are approved for.  The drug industry attempts to claim that it has no responsibility for how doctors prescribe their products, but that is not believable and the FDA has begun cracking down on inappropriate promotion.  If drug companies think their product has a new use, there are a variety of trial structures which can be used to both test the new use and provide patients with a new treatment option which may benefit them.  Stronger regulation may be needed, however, to prevent doctors from willy-nilly prescribing products before they have been adequately tested.  Otherwise, health care spending is being raised with no gain in patient outcomes.

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