Off-label use is the practice of prescribing drugs for a purpose outside the scope of the drug's approved label. The FDA requires numerous clinical trials to prove a drug's safety and efficacy in treating a specific symptom. Once deemed safe and effective, physicians exercise discretion for the use of the drug. It is entirely legal in the United States and in many other countries to use drugs off-label.
More physicians are discovering that many drugs are effective for off-label uses and apply to the needs of their patients. Off-label prescription drugs have become so popular that, today, 1 out of every 4 prescriptions written is off-label. The antiseizure drug gabapentin (Neurontin) is used off-label to treat people with diabetic neuropathy. Another drug used off-label is Lucentis, manufactured by Genentech. It was originally approved for age related macular degeneration, but is now in FDA trials for the treatment of diabetic macular edema. If Lucentis is approved, it would be the first drug to treat this debilitating complication of diabetes.
There are advantages and disadvantages to off-label uses. First (and foremost) is the risk versus reward. You could be the first to experience a breakthrough treatment or you could suffer irreparable consequences from the off-label use. Another consideration is feasibility - often times an off-label treatment may cost you substantially more than the other treatments approved for use. For example, without enrolling in an FDA trial to receive intravitreal injections of Lucentis - it would cost me $2,000 a pop. My peepers are precious, but my pockets aren't that deep! What off-label drug would you consider for use?(With your doctor's blessing, of course).
1. I am currently taking Topamax for an "off-label" usage, namely, weight-loss. It is typically used for migraine headaches and seizures. It also seems to be effective for people who have problems with binge eating. I have been morbidly obese for most of my life, and have tried just about every diet plan under the sun. I did not want to have gastric bypass surgery. My internist referred me to the local hospital's weight management clinic and I've been taking Topamax since November. The only side effect I've experienced is occasional "word-retrieval" issues - about once a day or so I might not be able to come up with a word during a conversation that otherwise I normally wouldn't have any trouble coming up with. As time goes on, I'm noticing this less and less. I'm also unable to drink carbonated beverages because it's affected their taste, but that's a good side effect in my opinion. So far I've lost 60 lbs. In my opinion, this is a miracle drug....it's finally put me in the right frame of mind to make the right choices about my diet and my eating, something that I couldn't do on my own.
Posted at 2:22PM on Mar 2nd 2007 by Rassi