Don't Be Afraid To Talk With Your Doctor About Any Medication You Are Prescribed -- Especially If The Drug Is Being Used As A Treatment NOT Approved By FDA
(Posted by Tom Lamb at DrugInjuryWatch.com)
Let's start with an informal and short definition of so-called "off-label" prescribing: A drug is being prescribed "off-label" when it has not been approved for treatment of the patient's medical condition and, instead, is being used experimentally.
To put this practice into context, we draw from a provocative article, "Informed Consent and Shared Decision-Making: A Requirement to Disclose to Patients Off-Label Prescriptions", written by Michael Wilkes and Margaret Johns, which was published November 11, 2008 by the Public Library of Science (PLoS):
Because a basic premise of the US Federal Food, Drug, and Cosmetic Act is that manufacturers are prohibited from marketing drugs or devices without FDA approval, the public commonly assumes that all uses of prescription drugs have been approved by the FDA. However, after a drug is approved for one set of indications, researchers and doctors often discover new applications for it. Even when the FDA approves a drug for a single, specific use, doctors may legally prescribe the drug to any patient for any use. Physicians are not restricted to prescriptions that comply with the FDA approval. The FDA considers such treatments “off-label” because substantial evidence regarding their safety and efficacy has not been presented or evaluated. But such uses are perfectly legal. In fact, FDA policy explicitly states that “once a [pharmaceutical] product has been approved for marketing, a physician may prescribe it for uses in treatment regimes of patient populations that are not included in the approved labeling”. Indeed, as the Supreme Court has recognized, off-label prescribing “is an accepted and necessary corollary of the FDA's mission to regulate in this area without directly interfering with the practice of medicine”. [footnotes omitted]
This PLoS article by Wilkes and Johns points out that, in fact, there are many examples of responsible off-label prescribing -- one is aspirin being prescribed to reduce the risk of heart attack long before it was FDA-approved for this purpose -- but cautions that other off-label uses can be dangerous -- a prime example is fenfluramine for weight loss, which caused heart valve damage to thousands of people (it was onepart of the notorious "fen-phen" combination).
So what should a patient do when it seems that their doctor is suggesting, or prescribing, a drug for an "indication", or treatment use, other than what that drug was approved for by the FDA?
The Agency for Healthcare Research and Quality (AHRQ) publishes online an advice column from Carolyn M. Clancy, M.D., who wrote "Off-Label Drugs: What You Need to Know". Here is some guidance for patients offered by Dr. Clancy in that April 21, 2009 article:
Talk with your doctor if you have concerns about any medicine or treatment, particularly if it may be off label. Here are several questions to ask:
- Is this the approved use of the medicine? You may not know if the use is off label. This question can help you start the conversation with your doctor about your medicines.
- Is the off-label use of this drug likely to be more effective than one approved to treat my illness? This is important because the off-label drug may not be as well tested for your condition.
- What evidence shows that this off-label drug can treat my condition?
- What are the risks and benefits of off-label treatment with this drug? Will my health insurance cover off-label treatment with this drug?
As we like to say at AHRQ, "Questions are the answer," meaning that it's important to ask your doctor plenty of questions about any medicines he or she prescribes. When you understand why you are taking certain medicines—including off-label drugs—you are more likely to take them correctly.
The bottom line is that you should not be afraid to talk with your doctor if you know or suspect that a drug is being prescribed off-label, i.e., as a treatment which has not been approved by the FDA.