Any confusion over the recent news of cholesterol guidelines in the U.S. is perfectly understandable. On the one hand, the guidelines suggest that nearly half the population should use statins to stave off heart attacks and strokes. On the other, use of the drugs is not with potential side effects and, to many, will offer no substantive benefits. The controversy highlights a problem mired in an outdated way of thinking about health care and the doctor-patient relationship.
Guidelines came about after generations of physicians wanted to bring something more than “opinion and experience” to the patient’s bedside. In the late 1960s legislation for the U.S. Food and Drug Administration was amended to call for a demonstration of efficacy and an assessment of benefits and risk as prerequisite to the licensing of any pharmaceutical. Modern clinical science resulted, first slowly and now with an avalanche of clinical trials, each pouring forth outcome data galore.
The Burden of Clinical Data
Clinicians are expected to stay current with this wealth of information. The modern medical curriculum instructs all budding physicians on how to evaluate the quality and the clinical relevance of all such contributions to the body of clinical science. Because some (or perhaps many) find this exercise overwhelming, there are organizations—many academic and some without any discernible relationships with purveyors that could pose a conflict of interest—that attempt to bundle the information in a fashion that might be relevant to particular physicians or physicians in particular specialties. Some of this bundling is quite systematic, some quite helter-skelter.
Occasionally there is a contribution to the literature that offers an unequivocal advantage for a particular patient group. More often, the bundlers are faced with a heterogeneous literature that often demonstrates little, if any, efficacy. Faced with these circumstances, biostatistics has offered up many a method to impute more value to the literature than is apparent at first blush. The result is that all this bundling adds to an enormous and ever-expanding secondary literature. What is the clinician to do?
Some clinicians suck it up and even suck it up in discussions with patients about the level of certainty that pertains to clinical options. Many take refuge in another trend that starts about the same time, the promulgation of “clinical practice guidelines.” There are nearly 3,000 such exercises waiting to extricate the clinician who is faced with responding to a patient’s complaint. These Guidelines are the work product of “thought leaders” who have undertaken the same sort of analysis of science and are charged with declaring the implications of the state of the science. This seems a reasonable compromise between the difficulties any clinician might face in dissecting the literature relevant to a particular patient and the difficulty clinical epidemiologists have in separating the wheat from the chaff in any particular literature. The “Guideline” on the topic unburdens all; it is today’s “truth.”
Guidelines as a Disservice
But for us it is not “truth” but an erosion of the patient-physician contract for an empathic trusting relationship and more often than not, a sophism. We will use the guidelines for treating cholesterol recently issued by the American College of Cardiology (ACC) and the American Heart Association (AHA) as an object lesson. This guideline has been met with considerable controversy regarding its validity. We will not engage that controversy. Rather, we will examine the way this sort of guideline facilitates patient-physician dialogues.
There are many guidelines that could provide similar object lessons: the recent British guideline for stents for coronary artery disease was worthy of a treatment similar to that we are applying here. We will argue that guidelines and the principles that underlie their promulgation serve many “stakeholders” quite well, but they do a great disservice to patients and the 21st-century ethic that demands a patient-physician dialogue that promotes informed decision-making.
Progress in ethically driven medical care has ground to a standstill because physicians, insurers, economists, governments, pharmaceutical companies, medical device companies, hospital executives and other stakeholders currently determine the menu of diagnostic and therapeutic options by more mechanisms than just Clinical Practice Guidelines. By virtue of various indemnity schemes, including Medicare, the choice of menu items is largely predetermined for any individual. Much of this occurs in committees and advisory panels that are tucked into the process of each stakeholder. Since these menus are produced by august bodies, they have the effect of defining the standard of care. Some of these guidelines consider how to do things to patients; these are not the object lesson for this essay. Our focus is on the guidelines that consider whether to do things to patients—guidelines like those from the American College of Cardiology and the American Heart Association (ACC/AHA) for treating cholesterol levels in the blood.
At best, these guidelines are recommendations based on scientific studies with results that pertain to the average among us. They do not adequately incorporate the personal differences and preferences of each of us as individuals. Furthermore, while these recommendations are based on clinical science, rarely is the science complete or incontrovertible. Hence, the recommendations are consensus statements reflecting the perspectives of those charged with the production of the guideline. Of the thousands of clinical practice guidelines that have been produced, the majority is based on inadequate science and therefore reflects the conjecture of the “thought leaders” recruited to the task. Many of the thought leaders admit to relationships with industry that are potentially problematic and all have the human foible of preconception.
How the Cholesterol Guideline Falls Short
The recently published ACC/AHA guideline for managing cholesterol levels was written by movers and shakers in the cardiology world who have been charged by the ACC and the AHA to recommend what might be best. The newest response to this charge results in a recommendation that suggests, to our reading, that nearly half of us should be swallowing one statin drug or another. The participants writing this guideline were faced with robust data that demonstrates a correlation between cholesterol level and clinical outcomes over time. But despite the substantive science behind the connection, the correlation is weak. The guideline neglects to emphasize this fact.
For example, the recommendation is based on the 38 percent reduction in adverse clinical outcomes—heart attack, stroke and other cardiovascular events—that result from treating cholesterol levels. But, this number alone offers no meaningful information for a patient faced with the decision to accept treatment; it is a relative, not an absolute value. It could mean that if my risk of a heart attack or death over the next decade is about 60 percent, I can reduce the risk to about 40 percent by swallowing statins every day for the next 10 years. This reduction in risk is substantial. The 38 percent reduction in risk, however, means less to me if my risk is 1 percent, not 60 percent, over the next decade. At that level of risk, taking statins daily reduces my risk a small amount, only about 0.5 percent— if you believe that this tiny effect is measurable. Each of us would value the popping of statins differently in these two circumstances: one circumstance provides a tangible improvement in outcomes at 10 years and the other a questionable one. An average amount of 7.5 percent, proposed in the ACA/AHA guideline, means nothing to me as an individual. That 7.5 percent risk is an averaged amalgam of many individual risks. Applying one to all is not advanced health care decision-making
The reality of the average benefit is closer to the second example, a small risk. The best we can hope for in treating 50 people, even 50 people with higher risk profiles, for their entire lives with a statin is to perhaps spare 1 of those 50 from a fatal heart attack. The other 49 will have been wasting their time, effort, co-pay and deductible. Are you willing to swallow this recommendation in the hope that you are not among the 49 out of every 50 who wasted time and money and risked a far greater likelihood of side effects?
Criticizing the reasoning of this guideline panel or their notion of patient education is not our main agenda. The point we want to make is that there should be no clinical “guideline” for reducing the risk from cholesterol at all. Guideline recommendations become standards of care and lead to treatments being applied in all people regardless of their personal appetites for taking risk, either of not swallowing the statin or of side effects if they did. The guideline developers are not the decision-makers; patients are. The only guideline needed in medical care is designed to allow, even require people to choose their medical interventions after being fully informed as to what is known about benefits, costs and risks. No one, “thought leader” or not, can say what is best for an individual. Making a recommendation that most should be treated denies patients their rightful place in the decision-making hierarchy.
Decisions Should Fall to the Patient
The idea of the patient as the sole decision-maker—and not governments, insurers or guideline producers—may seem like a radical shift in the philosophical premise of making medical choices. But it is this sort of radical shift in the goal of medical care that is needed and is feasible thanks to an informative science. The patient is not the partner in medical care; the patient is the leader, the pilot, the decision-maker. Patients don't need recommendations; they need information about the added benefit of one therapy versus another balanced against the added harm of the therapy. People are smart enough to make the trade-offs; they don't need to be told what to do. People are smart enough to understand it is as rational to forego statin treatment as it is to adhere to this guideline. In fact, people are smart enough to apply a similar razor to decisions regarding all sorts of items on the therapeutic menu from knee arthroscopy to cardiac stents.
Changing medicine from a "telling what to do" profession to a "telling the patient the information" profession will lead us to a better system of care, better in every way. The profession of medicine should have the goal to serve and inform, not recommend. (A series of essays recently posted on Scientific American discuss this mandate in detail—see below for the links). When uncertainty prevails, no longer should a patient ask, “What would you do, Doctor?” The question should be “What would you do, Doctor, if you were me?” That’s a question that demands a trusting, empathic treatment act between two people committed fully and only to serving the needs of the partner who has chosen to be a patient.
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