PB, thanks for the invite on the Idec thread to jump in on this one. Funny thing, I was just about to, before I saw your post. The JAMA article you found was very interesting. But except in one spot, which may have been just imprecise wording, the author (Dr. Eddy) does not seem to be equating "investigational" with "off-label." For those who have not yet read the linked article, Dr. Eddy is with Kaiser, which I believe is the largest HMO in the U.S. The article appeared to be a discusssion of Dr. Eddy's view of the best criteria and process an HMO could use to decide whether or not a particular treatment is "investigational", and therfore is not covered by their typical plan. It was not stated that this is the way that Kaiser, or any other HMO, actually makes the pay-no pay decision. But for discussion purposes, I'll assume that this is Kaiser's process.
Eddy states that a drug is properly not covered if it is "investigational". That is decided, he asserts, by the query " Is the available evidence sufficient to enable appropriately trained, motivated, and impartial people to draw conclusions about the magnitudes of the effects of the treatment, compared with no treatment, on all the health outcomes they consider important? " ( He readily admits in the followup Q&A letters that the HMO decision-makers are not "impartial.") Note: the criterion is not whether or not the particular us is approved. He discusses at some length how to determine whether or not the available evidence is "sufficient", and discusses the requirements for clinical trials which would provide such evidence. Nowhere does he talk about any need for these clinical trials to be in the context of FDA approval. (Of course, for a drug to be available at all it must have at least one FDA-approved use.) It seems clear to me that his criteria for "non-investigational" drugs, which he states decisively that HMO's should cover, includes any off-label use for which there is sufficient clainical trial evidence that the drug is adequately safe and reasonably effective. His explicit criteria would readily allow a drug to be considered "non-investigational" after one or more controlled studies had been done and published by reputable investigators. This kind of study fills the medical journals every month, and includes many studies of off-label uses. Next time you walk through a hospital, or even glance through a newspaper, look at all the postings for patiets willing to participate in just this kind of trial, which in most cases are not being run by drug companies as part of an approval process.
The one spot I referred to above was one where Dr. Eddy asserts that "denial of coverage is one of the few barriers that delays the dissemination of procedures, tests, and off-label uses of drugs before their effectiveness can be determined." Lumping all off-label drugs into the investigational, non-reimbursible category is inconsistent with the reasoning and definitions in the rest of the article. And note that he does not say that denial of coverage is a needed barrier to dissem. of treatments before their effectiveness can be determined AS EVIDENCED BY FDA APPROVAL AS A LABEL USE.
Conclusion: an apparent representative of Kaiser seems to be saying that off-label uses of drugs are reimbursable if controlled studies are available to show efficacy. While it seems clear that reimbursement would be available for all approved uses, it may well be readily available for many off-label uses once the drug has been out long enough that academic docs have had a chance to do some clinical studies. |
