There are two new buzz words that could have significant implications for our health care going forward.  Those words are “comparative effectiveness.”

Why is comparative effectiveness so important?  Because depending on your interpretation and definition of the concept, this could be anything from a very much needed way to address some nagging questions about how we treat different diseases or the forerunner of a more aggressive effort to control medical costs.

It is no secret that many of us think we spend too much money on health care.  We have a difficult time in this country demonstrating that we get true value for the money we spend.  Our technology in health care is state-of-the-art, unlike any place else on earth.  However, our outcomes are far down the list when we compare ourselves to other countries when we look at some measures of health, such as our average life expectancy.

One of the things we have learned over the past couple of decades is that we don’t know as much about the effectiveness of our treatments as we should. 

For example, are newer medicines for the treatment of common chronic diseases like diabetes and high blood pressure really better than the older drugs that are substantially less expensive?  In cancer, the question would be whether drugs that doctors use in treating patients—especially at the end of life with third or fourth line therapy for metastatic disease— are really appropriate if there is no evidence that they provide significant benefit under such circumstances?

Just look back on our experience with hormone replacement therapy, which took years to develop a sufficient body of evidence to help us understand the true benefits and risks of these commonly used medicines.

I could go on, but in my world comparative effectiveness means trying to get to the heart of these issues.  There simply has been very little money available to organize the type of research that needs to be done to answer these fundamental questions.  It would be—in my opinion—a good thing to be able to conduct such studies and get the evidence we need.

But there is another side to the comparative effectiveness debate that is beginning to raise some concerns, especially in the context of health care reform.

I have personally been troubled about how easily some think we can easily reduce health care costs in the United States. 

I have been around for a while, and if someone had the answer to that question (think managed care and Medicare, the former which is supposed to organize networks of care and the latter for the most part intent on driving down payments to the lowest fee) I think we would have seen it already.  We haven’t, and we aren’t going to get there quickly with our current “piece work” payment system.

Enter comparative effectiveness definition #2: we should carefully study the evidence supporting our treatments, and if we don’t have evidence, we shouldn’t pay for the treatment.

In cancer, it goes to the question of off-label therapy which I addressed in a recent blog discussing articles that appeared in the Annals of Internal Medicine.  No evidence, no treatment was the conclusion of the journal’s articles and editorial.  The problem is that the majority of cancer treatments in this country are in fact off-label based on the opinions of experts who have reviewed the evidence, none of which may be sufficient for a company to go to the FDA and get approval for a specific cancer drug or combination of drugs to treat a specific cancer.

Another example might be CT colonography.  The American Cancer Society thinks the evidence is sufficient to recommend this test as a reasonable one to prevent colorectal cancer.  The US Preventive Services Task Force does not.  The Centers for Medicare and Medicaid Services (CMS)—which administers the Medicare program—has issued a preliminary decision not to cover the test.  Their final recommendation is currently under review.  And this is a test where there is published literature to support its value and its use.

On the other hand, when I sat on a panel to evaluate the evidence supporting the use of PET scans in cancer, the quality and amount of credible information to support PET scanning in the diagnosis and treatment of cancer was actually very limited.  It was only because we had data from a PET scan registry that had followed some patients on Medicare that we got anywhere near concluding that the science in any way supported the use of PET scans.  And this is for a test that is used routinely and accepted without question by cancer specialists in the United States.

In a comparative effectiveness world as envisioned by some, they would use this process to deny payment for “unapproved” treatments under government programs.  That, to me, is not comparative effectiveness.  It may be something else, but it isn’t comparative effectiveness.

 In the United Kingdom, they have a group called NICE that determines what medical services are paid for by the National Health Service.  I have been interviewed fairly often on the topic of how cancer treatment differs in the United States and Britain, and how our systems differ in advancing new technologies.  Inevitably, the questions come down to which approach to providing health care is best: basically open access to everything, or strict limits which allow more people to be covered?

I am not a fan of the concept that every new test, every new drug and every new gadget necessarily improves our health care here in the United States.  But there have been some major examples where a new treatment is available quickly here, such as the use of Herceptin as an adjuvant therapy in HER-2 positive breast cancer, long before Britain decides that  they can pay for it.

My concern is that if we adopt a “strict evidence” standard here in the United States, we are going to become much more like Britain.  Maybe that’s a good thing, maybe it’s not.  I can’t make that determination.   That’s something that needs to be discussed and debated.  But it has to be an open and honest discussion, not couched in words that have different meanings to different people.

We can’t go on spending the way we have on health care, without expecting some limits of some kind.  We simply can’t afford it as a nation, while millions go without insurance and adequate medical care.  We are going to have to make some decisions at some point on how we can more effectively make our system more rational.  Right care for the right person at the right time is a responsibility for all of us, doctors, medical professionals, patients and families alike.

How we address the question of “comparative effectiveness” and what it means and what it is expected to accomplish may seem like a small point that is flying under almost everyone’s radar as we tackle larger issues, but I suspect if we don’t get it right we will live with the consequences for years to come.