Saturday, March 27, 2010

Comparative effectiveness research

I’m a doctor, so maybe I have a different take on this than other people, but somehow I don’t think so. I would imagine most folks would want to know, before being put on a new, expensive medication (and in medications, “new” virtually always = “expensive”) a little more about it than that it worked better than nothing, or a placebo (which is not always the same thing, given the “placebo effect” – folks sometimes improve somewhat if they think a drug is going to work). I would imagine they’d want to know that it works better than, or as well as, or at least almost as well as, the drug that they are taking, or another drug, or especially a long-established, effective, we-know-how-well-it-works-and-the-side-effects-too, and frequently generic and cheaper, drug.

Studies that do this are called “comparative effectiveness research” (they can also be used for devices and other treatments). You would think, then, that there would be consensus that a new drug should be tested against existing drugs for the same condition and shown to be superior, or at least (in the scientific parlance) “non-inferior”. Not true; the FDA just requires studies that show it is better than placebo. This issue has been getting a lot of attention in the medical and scientific communities recently. A major contribution to this discussion was the recent study published by Michael Hochman and Danny McCormick in JAMA, March 10, 2010, “Characteristics of published comparative effectiveness studies of medications” (JAMA. 2010 Mar 10;303(10):951-8). This study looked at 328 previous studies evaluating medications and found that less than a third (104) had compared the drug against something other than placebo. Only 11 compared the drug against non-drug (“non-pharmacologic”) treatments; among the others only 45 compared two or more drugs, 32 with different pharmacologic “strategies”, and 16 different dosing schedules. Most of these, 90 of the 104, were financed by non-commercial sources (e.g., the government); the placebo studies were almost all funded by pharmaceutical manufacturers, who fund, it should be noted, more than half the medical research done in this country. This would be a good and noble thing, were it not actually a self-serving and thus often bad thing. Since many people cannot access the JAMA article (and might find it harder to understand it anyway), Hochman and McCormick also published an op-ed piece in the Los Angeles Times, “Medicine in the dark” which is clear and persuasive. In the same JAMA issue, two HHS physicians, Patrick Conway and Carolyn Clancy (who is head of the Agency for Healthcare Research and Quality, AHRQ, that supports much comparative effectiveness research), comment on the Hochman and McCormick article and call for increased comparative effectiveness research (“Charting a path from comparative effectiveness funding to improved patient-centered health care”, JAMA. 2010 Mar 10;303(10):985-6). They do not comment on the pharmaceutical industry funded studies.

Most practicing physicians are not active clinical researchers, and even those who are usually have a very limited research focus. Therefore, physicians need access to accurate and unbiased research, and need to spend the time keeping up with important new information that may impact your care. (By “important”, here I mean something that actually may benefit your health or that of another patient, not something that may benefit the bank accounts of the drug company shareholders – which I am sure is important to them.) Before beginning you on a new drug for your condition, especially when it is more expensive – often very much more expensive – and newer (please note that with drugs “newer” does not always – in fact, uncommonly, mean better. It does, however, virtually always mean “used and tested less so we are not necessarily aware of all adverse effects”, so you would hope that your doctor had reason to believe it was going to work better (or at least not worse) for you than your old treatment, and that this belief was based on independent reliable sources. That is, if not the original research, at least on independent reviews and summaries (like that of Hochman and McCormick, or The Medical Letter), and not simply information provided to them by drug salesmen. If you see lots of pens, pads, clocks, and other doodads with pharmaceutical company logos in your doctor’s office – not to mention if you get those neat “free samples” (which are never free; you and others buying the drug eventually pay for them) of the newest, most expensive drugs being pushed by the drug companies, you might begin to get suspicious. You might even ask your doctor how much time s/he spends with drug company reps and how much of his/her information comes from them, and has s/he ever audited his/her charts to actually find out (whatever s/he believes) if s/he is prescribing more of drugs pushed by drug reps? You might even complain. Doctors are sensitive to their patients’ complaints.

But what about when the doctor does look at the original research, but the research itself is largely funded by drug companies, who frequently suppress negative results? And if, as Hochman and McCormick point out, most of them just compare the new drug to placebo, not other established treatments? What value is there in that? Well, Peter Pitts, interviewed on NPR’s Morning Edition in their story on this issue on March 10, 2010, thinks there is value. He says doctors need to know what options are available. Of course, Mr. Pitts is a spokesman for the Center for Medicines in the Public Interest, a group whose name is absolutely contradictory to its function, since it functions in the interest of the pharmaceutical industry, which funds it. (And, really importantly, should never be confused with the absolutely wonderful Center for Science in the Public Interest, a truly great public interest organization that interprets research and advocates for food safety, the environment, and other issues that we need real scientists to help us to understand.)

As a physician, I agree with Mr. Pitts that we need to know what options are available. And, apparently unlike Mr. Pitts and the industry he represents, I think we need to know the whole story and how those options compare to one another, not a placebo. And I think you would want your doctor to know that also.

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