Monday, 2 February 2009

“Off Label” Medication Use: What does this mean? (Part II)

Medication regulatory agencies (such as Health Canada, the FDA, etc.) have no authority in deciding on how medicine is practiced. That is the role of professional and licensing bodies. Thus, while a medication may be “off label” as far as the medication regulatory agency is concerned, that medication may be a useful part of accepted medical practice as far as professional and licensing bodies are concerned.Therefore, medicines may be frequently used in a medically appropriate manner, even if they are “off label”.

Fundamentally, the lack of a “label” for use does not mean that the medicine is not appropriate for use in any particular medical condition (that is a professional and licensing oversight decision). What this means is that the medication has not received medicine regulator approval for treatment of the condition in which the physician is using the medication, frequently because the company making the 
medication has not submitted for regulatory approval.

There are many useful criticisms that can be made of this state of affairs, not the least of which is the confusion that the concept of “labeling” has created in the minds of the public and health providers alike. Another criticism is the concern that the standard of proof for safety and efficacy required by medicine regulatory agencies prior to the awarding of a “label” for use may be less for those uses not labeled. Other criticisms may relate to concerns that the public may be less protected from “off label” use than it is from “on label” use. Some commentators point out however that all “over the counter” medications and “health supplements” (including herbal remedies) and all psychological treatments never receive regulatory approval – despite their widespread use.

So what is the patient or the family to do in such a confusing situation?

Just because a treatment is “off-label” does not mean that it is not appropriate. If that were the case then many of our medication treatments would be deemed inappropriate and all of our psychological treatments would be deemed inappropriate.

First, is to understand what the tem “off label” use means, and what it does not mean.Hopefully, the information in part one of this post helps with that. Unlike what the mass media suggests, “off label” use does NOT mean dangerous, ineffective or inappropriate use. So gaining clarity and understanding is a good start.

Second, is to understand what questions you should ask your health provider about ANY treatments that you are receiving. All treatments should be based on best available evidence of effectiveness and safety. This is called “evidence-based medicine”.

All treatments should be subjected to the same degree of rigorous scientific scrutiny and judged by the same high standards. All patients and their families have the right to know the evidence upon which any treatment is based and it is the ethical duty of their health provider to provide them with that information.
So, here are the questions to ask:
  • Why are you recommending this treatment?
  • What is the evidence (effectiveness and safety) supporting the use of this treatment for people who are like me and who have the same diagnosis (or problem) that I have?
  • What regulatory or professional bodies support the use of this treatment?
  • What are other treatments that could be used?
  • What is the evidence for those treatments? Is it stronger or weaker than the evidence for the treatment you have recommended?
There are many other questions you should ask about your treatments. Reading the evidence-based medicine information guide for patients and seeking more information about medications at should help with that.

~ Dr. Stan Kutcher

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