Off Label: Like Giving Change To The Homeless

Anyone who has made a visit to downtown Cleveland recently may have seen the "Change Where it Counts" signs. This public education campaign is meant to discourage people from giving money to panhandlers and instead donate funds to organizations that will use them to truly benefit the homeless. While those that give money to a panhandler have good intentions, doing so actually has adverse consequences and undermines the systems in place that really make your change count.

These signs come to mind when I think of physicians who perform off label procedures and prescribe drugs for off label use outside of the setting of a clinical trial. It does seem well intended and in many cases does offer some immediate benefit. How much benefit? Is the benefit real or perceived? Is it safer than other alternatives? Is the risk I am exposing my patient to really worth it?

These are exactly the kinds of questions that clinical trials are intended to answer and exactly the type of information the FDA is seeking when they evaluate a new drug or device or a novel use for either. It is not the FDA's responsibility to seek out new drugs, devices or new uses for same; that responsibility falls on the shoulders of the drug or device manufacturer. When a new use is thought to be of benefit to patients the drug/device company sends an application for approval to the FDA. Before that novel use can be added to a package insert the benefit, safety and effectiveness are reviewed by the FDA and either accepted or denied.

Collecting information costs money, a lot of money. It also takes time and the commitment of the prescriber to recognize and report adverse outcomes. What incentive does a company have to do this when they know that physicians will use their product without demanding that the safety and effectiveness are validated? None. Further, what incentive is there to test the safety and effectiveness of these drugs on populations that do not generate profits (pregnant women, children, minorities) or generic drugs that offer very little profit potential?

More important than the FDA approval or the practice of off label use by physicians is the patient's right to make an informed choice. Without the information that provides key answers to the risk, benefit and alternatives of therapies the fact remains that the patients who decide to proceed with these therapies cannot make an informed choice. Patients are left with only the faith that they have in their provider, a faith that is in many cases misplaced. The fact remains that without data no one really knows what the widespread clinical implications are and no one is seeking the answer.

As health care providers adminstering drugs, performing surgeries and the reality of bad outcomes has become a fact of life. We hand out drugs and administer therapies, literally, by the thousands. We often do this without even a second thought. The same is not true for our patient. For many, the decision to have surgery or take a new drug is one that is a serious life decision. We owe it to the individuals that we care for to take the risk of an adverse outcome seriously and we should demand that we are fully aware of those risks. We should demand that the companies who profit from our decisions fully disclose the information they have. Finally, as providers we should be honest with our patients when we do not know the answers - these disclosures lie at the very heart of the meaning of informed consent. While the quest for innovation is laudable, the true human costs should not be ignored.

In short,while well intended, prescribing off label is much like giving change to the homeless undermining the systems in place to ensure that the therapies we use are in fact helpful and beneficial to all people who may be subject to them. The short term fix may look appealing but really does not promote change where it counts.