June 22, 2009 -- For a number of years I have enjoyed an invitation to submit opinion essays for consideration for posting in the Health Section on ABCnews.com. Gratifyingly, many have been deemed worthy and dozens are archived.
I am not paid by ABC. For that matter, I am not a paid consultant to any component of the health care industry except by the State of North Carolina for my roles as a professor of medicine on the faculty of the School of Medicine of the University of North Carolina. I have a vested interest in the care of the patient and in the teaching of that caring, and I refuse to compromise that ministry.
In several recent opinion pieces on ABCnews.com I broached the hot-button topic of effectiveness. I asserted in no uncertain terms that many of the things we offer American patients, and many of the things we do to American patients afford them no personal advantage. I based these arguments on a comprehensive and compelling science.
Much to my surprise, I learned that my name and my arguments were bandied about in segments of the media as advocating the rationing of health care. Out of curiosity, I agreed to a "talk radio" interview on this topic on May 27.
The host wasted no time in confronting me as an agent of the White House preparing the country for a health care reform agenda that entailed rationing of health care services. I made it clear that I have no formal consultative relationship with any federal agency. I always speak on behalf of my patients, my students and colleagues, and my personal ethic. Once that was established, we were able to dispassionately address the notion of rationing. I hope we can do so again, here.
Firstly, it is interesting that "rationing" has become a toxic buzzword in the context of health care. Rationing is the act of apportioning a fixed amount of a finite resource. The "finite resource" we are apportioning annually for "health care" in the United States is $3 trillion. That's much greater per capita than the apportionment of any other resource-advantaged country.
Furthermore, our current apportionment manages to exclude some 30 percent of the workforce, and many others. No other resource-advantaged country apportions so unevenly, if they apportion unevenly at all. I hear very little advocacy for decreasing our $3 trillion finite resource. Bizarrely, there is advocacy for further increasing this finite resource. The arguments I hear relate to the fashion in which our finite resource is apportioned.
I and many others have strong thoughts about this apportionment. No one advocates wasting money. To the contrary, we are swimming in plans to decrease the waste that is consequent to inefficiencies in delivery and in the quality of performance. There is no argument.
There is also no argument about ineffectiveness. If some medical or surgical act does not advantage me or my family or my patients, it shouldn't be done. I don't care how well it is done, how cheaply it is done, how efficiently it is done; if it doesn't work, don't do it.
Evidence for Benefit
America takes pride in the availability of the seemingly miraculous advances of medicine and surgery that we have come to consider our entitlement. America is less aware of the most important advance of the past 50 years, which is the ability to test whether the advances are as miraculous as they seem.
Some advances do not require testing because they are obviously wonderful: antibiotics for infections that are otherwise fatal, some forms of trauma surgery, kidney dialysis and transplantation, etc. Other advances seem miraculous because they make sense but how do we know we're not being fooled as we often were in the recent past:
At mid-century we removed the inflamed tonsils from most young children who had persistent upper respiratory infections in the winter and thought we were heroes because they were spared tonsillitis come next winter. Now we know that they outgrow such with their tonsils in place. And remember the tubes in the ears? The antibiotics for every inflamed ear drum? Gone with the data.
At mid-century we were removing wombs and teeth and gall bladders from patients with rheumatoid arthritis to get rid of the evil focus of infection. Now we know we only offered them the possibility of surgical complications.
Many of the drugs that made sense to treat heart failure and asthma in 1970 turned out to be useless or harmful by 1990.
And much more...
Today we know that our best guess and our best theories can lead us astray. Today we educate every medical student to know to ask, and to know how to ask for the evidence that interventions actually work. Today, an enormous investment is made is doing the studies that define the evidentiary basis for all new pharmaceuticals before FDA licensure. Today, an enormous investment is made by industry to provide the evidence that their pill or device is better than the competition's.
Thousands and thousands of papers appear each year, varying in scientific quality, varying in the patient groups studied, varying in the intervention studied. Many of us who practice medicine feel compelled to analyze this information as it pertains to the problems of our patient.
The task of defining the evidentiary basis of medical practice has such scope that my profession has fostered not-for-profit, independent organizations devoted to the exercise. Some of the most influential are the U.S. Preventative Services Task Force, the British National Institute for Health and Clinical Excellence, and the ACP Journal Club.
The granddaddy is the Cochrane Collaboration. The Cochrane Collaboration was founded in 1993. Based in Oxford, England, it is a highly structured, international collaboration of thousands of volunteer scientists organized into review groups based on particular topics. These groups produce and disseminate systematic reviews of the literature on the efficacy of healthcare interventions; thousands are already available, more come every year, and older reviews are updated.
Informed Clinical Decision Making
Robert McNutt, one of the editors of the Journal of the American Medical Association, wrote a brilliant essay on how we as patients should advocate for our own health care. He, and I, believe we are each the captain of our own ship. Our physician is the navigator. Our physician owes us more than clinical judgment, which is wisdom derived from the accumulation of clinical experiences. We are owed a statement as to whether there is scientific evidence for benefit, and if so how much benefit? Enough so that it is meaningful to me? If there is no evidence for benefit, was the intervention studied so adequately to support a statement of "there is no benefit"?
Often the answer lies between certainty as to meaningful benefit and certainty as to no benefit. Our values and clinical judgment conspire to inform the decision. That is the 21st century medical treatment act.
In my last book, I take the reader through this exercise for a number of commonly offered and commonly accepted clinical interventions. I define the clinical issue, examine the science for benefit in some detail, and contrast our analysis with that from the Cochrane Collaboration or the like. Surprises abound that have great implications for personal decision making and for policy. In the next book, low back pain provides the object lesson. Let me demonstrate how this works.
Coronary Artery Bypass Grafts, Angioplasties and Stents
Let's examine the example of interventional cardiology. In the United States, well over a million people undergo these procedures each year for various manifestations of coronary artery disease. They are technological advances designed to overcome blocked arteries. They all have technical limitations in that the unblocking or bypassing tends to reverse itself. So there is much invested in the attempt to more permanently overcome the blockage.
There are four stents licensed for this purpose and many in the "pipeline" -- and there are many surgical approaches to bypassing. There are also thousands of trials comparing one technique with another to determine the relative fate of the unblocking.
However, I don't care about the fate of the unblocking. I care about the fate of the patient. Is the patient better off for all this technology?
That central question gets lost in the race for technological achievement. There have been strong reasons for doubt for years. Doubt is now dispelled thanks to four recent randomized controlled trials comparing such interventions with medical care without the interventions.
Three of the trials were American -- OAT, COURAGE and BARI 2D -- and one -- RITA-2 -- was European (these are all acronyms based on the type of heart disease studied). Each recruited thousands of patients and followed them for years. Each recruited a somewhat distinctive population in terms of the manifestations of their coronary artery disease (active angina, after a heart attack, angina and diabetes, etc.), but all recruited patients for whom interventional cardiology was thought to be a reasonable option.
No one, I'll say it again, NO ONE was advantaged by submitting to the intervention. They did not live longer, or have less chest pain, or suffer fewer heart attacks than they would have without the procedure.
When Clinical Science and Clinical Judgment are Dissonant
Not long ago, I attended "Grand Rounds" at a prominent medical school where the professor and director of the Cardiac Catheterization Laboratory was holding forth on the mechanisms by which newer stents can stay open longer. I asked him how he explains the stent option to a patient with chest pain in view of the results of OAT, COURAGE, BARI 2D and RITA-2.
He, of course, was aware of all these trials. Even though the trials recruited patients with four very typical patterns of disease, he suggested many patients do not quite fit these categories. He was willing to rely on his clinical judgment to infer that a particular patient would be advantaged because he is endowed with a special level of expertise.
I asked him if he told the patient that his judgment was superseding a compelling science and that his judgment had not been subjected to testing. He ducked the question. As far as I'm concerned, when the precedent is not even encouraging, such an inference demands scientific validation before it is put into clinical practice.
The Lottery Mentality
For me, this is irrational behavior on the part of the patient and worse on the part of the navigator. It survives in America because of what I call the "lottery" mentality: "Doc, if you can get me through this and it only works one time in a thousand, I want to be that one. Go for it!"
True, someone wins the lottery, even if it's exceedingly improbable. But the science we're considering is not a lottery. The argument that pertains is that the patient is as likely to do as well without the procedure as with it. It would be as if you could win the lottery without buying a ticket.
Furthermore, would the American lottery mentality drive clinical decision making if the patient had to buy the "ticket" instead of all of us sharing its cost. And if we knew the science, would we be willing to share the cost. If not, is that rationing, or rational?
Interventional cardiology for coronary artery disease consumes about a third of the finite health care resource and advantages no patient. If we couple that with common practices where studies lead to a conclusion of "no discernible benefit," we can free up about half of the finite resource and easily provide rational care and caring for all.
Then we can tackle the thorny issues of interventions that work sometimes or just a little, and of interventions yet to be discovered. If we hold the care of the patient as the entire reason for the enterprise, even the thorniest of issues are surmountable.
Dr. Nortin Hadler is professor of medicine and microbiology/immunology at the University of North Carolina at Chapel Hill, and an attending rheumatologist at University of North Carolina Hospitals. He is the author of "Worried Sick: A Prescription for Health in an Overtreated America" and "The Last Well Person."