Thursday, February 26, 2009

Comparative Effectiveness vs. Innovation

Peter Orszag is President Obama's Director of the Office of Management and Budget (OMB) and he has emerged as the architect of the fiscal aspects of healthcare reform. He is known as an expert on the concept of Comparative Effectiveness Research. This is simply a wonkish term for using best available evidence to guide decision making; in particular, using evidence based medicine to determine the most cost effective treatment guidelines (without sacrificing efficacy). For example, if the science demonstrates that generic pills are just as good as newer, more expensive combo-pills at controlling high blood pressure, then it would be reasonable to implement restrictions that would preclude physicians from prescribing the more expensive medication.

The Dartmouth Atlas Project, very much the hot new thing in the medical blogosphere this year, is a long term study of health care markets in the US measuring variations in health care resources and their utilization by geographic area. Basically, it showed that clinical outcomes are not necessarily related to the amount or cost of care provided, and that there is an incredible variation in practice patterns depending on what part of the country you examine. The message is clear: standardize the delivery of health care according to Comparative Effectiveness Research protocols and we can save boatloads of money in the health care sector. And this sort of thinking is Peter Orszag's baby. This is the underlying theory (along with redistribution of wealth) behind President Obama's health reform agenda. And it seems to have substantial validity.

But medicine doesn't like to be categorized. Although it would seem intuitive that we ought to always practice according to guidelines established by the "evidence", the reality is that we often go off the reservation in medicine. The history of medicine is full of doctors who went against the grain, despite available evidence, and ultimately ushered in new eras of innovation. The history of organ transplantation is absolutely fascinating. (Check out Joseph Murray's Nobel speech.) A small cadre of surgeons in the 1960's basically hammered out the principles of immunology and, through trial and error (and what was in essence human experimentation) were able to hone and standardize the transplant process over just a decade. Dr. Murray's first three kidney transplant patients (not including the initial transplants performed between identical twins) all died. By 1965, his group had achieved a 1 year allograft survival rate of 65%. Nowadays, there are people walking the streets with 30 and 40 year old donor kidneys. We are transplanting livers and pancreases and cataracts and lungs and even hearts. These pioneers were not guided by "best available evidence". It was completely ad libbed and improvised on the fly, until some modicum of success was attained and could be systematized.

Laparoscopy developed in a similar vein. There were no randomized controlled trials comparing open with laparoscopic cholecystectomy when the minimally invasive approach swept across this country in the early nineties. The data came later. The actual practice of surgery, however, developed independently of CER. Likewise, we don't currently have any level one evidence supporting laparoscopic appendectomy as being superior to open appy. But ask any general surgeon today and more and more are opting to treat appendicitis laparoscopically because of the superior wound infection rates, better cosmesis, faster recovery, and an increasing comfort level with performing the procedure itself. Eventually the "evidence" will confirm what is already apparent.

You see, medicine is a constantly evolving and developing field of science. Evidence based decision making is an excellent method to assess what has been done in the past, and help guide us through the complex process of deciding which treatment option is most prudent in terms of both cost and efficacy. But it doesn't help us figure out new paradigms for unforeseen challenges in health care. Innovation and risk-taking and aggressiveness are fundamental to advances in medical knowledge. We don't have a transplant program in this country if CER determined the allocation of funding in the 1960's. Laparoscopy was significantly more expensive in the early days of minimally invasive surgery because the equipment costs and length of OR time outweighed the benefits of a shorter hospital stay. Things have changed.

Let's just hope the brainy Peter Orszag has made allowances for innovation somewhere in his complex mathematical manipulations. The next quantum leap in medicine is always just around the corner; let's make sure that leap isn't hamstrung by too much government red tape and bureaucracy....


Tyro said...

Likewise EMS and resuscitation practices are seldom purely 'evidence-based' due to the inability to randomize well. ACLS admits there's no evidence that using epi in a code situation improves outcomes but notes it is 'reasonable' to continue given it is standard practice. I wonder how attempts to control costs will deal with this, and I'm not encouraged by how CMS and JCAHO jumped on, for example, blood cultures for pneumonia when they never change therapy and are probably an association with mortality rather than a causative factor.

Most recent Annals of EM has interesting articles on forward field hospitals in Iraq; sounds like a lot of innovation is coming on the battlefield.

Joseph Sucher, MD FACS said...


I agree with you to a point. But I can't go with your shoot from the hip approach to medicine. We are in an era that allows us to be better than our forefathers, not-withstanding that the days of just being able to do something because you're a doctor just don't exist anymore.

Let's take a few of your examples. Lap chole; the introduction and expansion of lap chole happened outside the academic world because the white coats in the ivory towers couldn't get themselves to even look in the laparoscopic direction. Their failure resulted in an operation that remained with a significantly higher morbidity with respect to biliary injury than its open counterpart, leaving many patients to suffer with major complications. This may have been attenuated if given a proper review and implementation strategy. I believe that we are seeing this with NOTES.

As far as laparoscopic appendectomy is concerned. You know that I personally agree with your opinion on its superiority. However, we certainly can't see from the current literature that it is or isn't better, probably because no-one has had the ability to compare apples to apples. True and honest research to answer questions not only takes integrity, but it takes money, time and resources. This is the piece of the equation that is lacking in the discussion of Comparative Effectiveness.

We can't just cut off the ad-hoc innovation pipeline without supplying the rigorous scientific analysis pipline.

So, I agree. Innovation in medicine isn't just about the literature alone. Currently we know that it takes 15 years to implement strategies that we already KNOW are good. Cut off the ad-hoc innovation without investing in the structured approach will result in the death of medical advancement.

Finally, the one thing that does concern me about Comparative Effectiveness, is that it completely disregards what tools help you and me do our job faster and easier. For instance, I very much like to use a LigaSure device to divide the mesoappendix, and an EndoGIA to divide the appendix. Together, those devices make it my operation easier and faster. Unfortunately, it makes it more expensive, than using just a LigaSure and endoloops. I like my method. It is safe. It is effective. It works great. I can move fast. But will Comparative Effectiveness see that? The answer is NO.

So even I am concerned with the ultimate direction that I see we are heading. That is, I see that the tools will be used with a single minded implementation that simply counts the beans and doesn't care one bit about the taste.

Anonymous said...

Writing's on the wall Buckeye, your Laparoscopies/Laparotomies are gonna get reimbursed same as a 15 minute office visit...worse when you consider you gotta take care of them afterwards...
Eff "Plastics", Tatoo Removal's the future of Medicine...

walt dandy said...

In response to Dr. Sucher: When you think about it, it is very common to hear the phrase "I like..." when surgeons are talking shop. It seems that the freedom to use and do exactly what you like is going by the wayside. It can be frustrating, I know, but I think we're all going to have to get used to it. There are a lot of really expensive things that are used in the OR mostly because the surgeon in question "likes" them, and not so much because they make any difference for the patient. Of course it goes without saying that I'm including my own specialty here. One example that comes to mind is that of Dermabond and some knock off competitor. At the hospital I was at at the time a little tube of Dermabond was a $300 charge to the patient, but there was a $100 price tag for the knock off. The knock off was definitely not as nice as the Dermabond (it was really runny until it dried, and when it dried it was really thick and grainy). While it's probably arguable, I honestly doubt that the knock off was worse for the patient. Everyone hated it, though, and continued to use the Dermabond simply because the liked it more. Is that a good reason? No. Is an extra $200 on the $60,000 bill a big deal? No. But the same thing happens every day with equipment and instruments that cost a lot more than $300.