Tuesday, August 27, 2013

What is “Best” Care and How is it Determined

In medicine, randomized controlled studies have long been the gold standard in defining the “best” care. In these types of studies, usually two large groups of patients, the larger the better, receive the same care except for one intervention that is different for each group and the results are compared. This defines the best care medical science has to offer. No one disputes the usefulness of these types of studies, however, are they enough? Do they really tell us all we need to know about the care of an individual? 

A recent article in the journal “Medical Decision Making” by Bruce Barrett M.D. entitled Sufficiently Important Difference: Concepts, Caveats, and Challenges questions whether our approach to using and interpreting these studies is adequate. The article looks at randomized controlled studies and the facts that they define in light of a new reality in health care. The new reality is that the value from the patient’s point of view is the key factor that must be taken into consideration when doctors and patients make decisions about care. That patient point of view has rarely been used as an end point in randomized controlled studies and the evidence based medicine that results. Dr. Barrett defines this focus on value from the patient’s point of view for clinical research design purposes as the “sufficiently important difference” (SID) or the “smallest worthwhile effect” which he defines as “the smallest amount of patient-valued benefit that an intervention would require to justify associated costs, risks and other harms.” He then adds, “SID is understood in the context of existing evidence and is measured at the individual level.” Measuring at the individual level is earthshaking for clinical researchers and for those who apply clinical research to the practice of medicine because when randomized controlled studies are done, the gold standard for results is always population based. While individual decisions must be informed by population statistics, the real impact of a particular intervention on an individual may not perfectly fit a population fact found by study. By telling us that the way to do clinical research is by measuring at the individual level and that it be “patient-valued” Dr. Barrett is telling us that we have to look anew at the outcomes of studies and as a result, many of our favorite “facts” and “goals” in population health and in the diagnosis and treatment of disease. A recent case in the news illustrates this point. As I write this an 11 year old girl is going home from the hospital after receiving a double lung transplant. The standard of care based on population evidence and expert consensus is that this lung transplant should not have been allowed to happen since this child was considered too young to receive adult lungs. A persistent legal and public relations fight by her parents forced the circumvention of this rule and this child is going home now with new lungs. The population facts were not disproved and remain helpful; they just did not perfectly apply to this young girl.

Dr. Barrett in this article points out that evidence based decisions, which are usually based on randomized controlled studies are not perfect in their design and suffer from their own limitations including “1) difficulties in forecasting individual outcomes from observed group effects; 2) the fact that negative outcomes are underassessed and underrepresented; and 3) the high degree of variability in how individuals value and weigh various positive and negative outcomes.” 

He does not say this in a policy journal or in a popular magazine. He makes these statements in a journal that only a statistician can really love. The science of medical decision making and the articles written in this journal are more geared to statistical researchers than to clinicians. In this same issue, there is an article entitled, “Development of a Framework for Cohort Simulation in Cost-Effectiveness Analyses Using a Multistep Ordinary Differential Equation Solver Algorithm in R”. For the journal Medical Decision Making, that is a relatively common type of title. I mention that only to make the point that Dr. Barrett is making these pronouncements as a way to improve analytics and as someone who believes in scientific and even mathematical approaches rather than as a moral argument. 

However he does realize that there is a moral dimension to his argument. After making a convincing case that “Patient-oriented evidence that matters (POEM) is superior to disease-oriented evidence (DOE), such as biomarkers or surrogate markers” on analytic grounds he goes on to say,

“Given this background, I would hazard the contention that the current system may to some extent be both unethical and irrational. Difficult questions must be asked: Is it rational to implicitly value benefits more than comparable harms? Should we continue to design and interpret trials based on benefit effect size only, ignoring harms? Is it ethical to take decision making away from individuals (guided by their clinicians) and to instead give that power to medical scientists, insurance companies, technocrats, and policy makers who set guidelines and formularies that determine care? I don’t believe that any of these questions should be answered in the affirmative.”

Thus he defines the challenge to the good, ethical clinical researcher and to the practitioner trying to follow evidence based medicine and best medical care. The challenge is at the least, to interpret randomized controlled studies with outcomes that are defined by patient values not only population metrics. It may mean moving to a whole new paradigm of clinical study that builds upon randomized controlled studies in new ways to measure these important patient-oriented outcomes that matter. He argues that until we understand how individuals value various benefits and harms we cannot really say what the best course is for a patient who is ill. For the practitioner that means knowing who your patient is as a person and not only knowing the biology of their disease. Ultimately, his scholarly analytic approach supports the contention that each individual is unique and autonomous with his or her own values that must be supported and respected.

Tuesday, August 13, 2013

Review, Quality Control and Arrogance

In 2002, I had an 80% right coronary artery narrowing that resulted in my having an angioplasty and a stent placement.   I thought of my procedure when I first read of the coronary artery stent placement performed on former President Bush and when I subsequently read the Washington Post opinion piece entitled President Bush’s Unnecessary Heart Surgery” written by Drs. Vinod Prasad and Adam Cifu.   While I understand the authors concern that people who are asymptomatic will mistakenly interpret President Bush’s stress test and subsequent stent placement as meaning that this sort of approach should be taken for everyone, I am even more concerned about their strong statements that the former President’s care was wrong.   In their zeal to prevent unnecessary and potentially dangerous care, the article crossed a line by proclaiming judgments on medical care based on limited facts and incomplete information.    They presented as absolutes, decisions made daily by caring physicians and informed patients that are often more nuanced than they appear.  Medicine exists in the realm of uncertainty and consists of the constant balancing of risks and benefits for individuals.  Within this realm of uncertainty there still must be quality control to protect the public from dangerous and unnecessary care since doctors are human and make mistakes.  However, there is a difference between careful and thoughtful quality control and Monday morning quarterbacking.  There are four major factors that cause me to pause before I make the kinds of absolute statements that are made in this article.  These same factors also cause me to hesitate before endorsing many of the decisions that insurance carriers and regulators can be too quick to make about payment for services and quality of medical providers. These factors are:
Facts Change:
Back to my angioplasty and stent in 2002.  At that time, I had what was considered to be the correct standard of care.  The cardiology department at the Harvard teaching hospital where my procedure was done was steeped in the academic studies that rigorously followed the facts in the medical literature.  Today the facts are different as pointed out by Drs. Prasad and Cifu because as more information and different studies are done, facts change.  In an article published in the October 2012 issue of the American Journal of Medicine, Dr. Joseph Alpert points this out quite eloquently.  He states in the article entitled, What Is True Today Is Often Not True Tomorrow”,In fact, it is likely that many of our current medical beliefs will turn out to be untrue or only half true as a result of information obtained through subsequent scientific investigation.”
Populations Change:
When studies are done, the population is often defined in one way; however as more facts become available, certain subpopulations are defined that do not necessarily follow the lessons learned from the broader population.  Thus, as an example, women with breast cancer as a population do not benefit from removing the unaffected breast.  The smaller subpopulations of women with breast cancer who are positive for the BRCA gene however do benefit. 
Context Matters:
If someone cannot afford medication, then prescribing a medication that they will not take is not good care, even if the care pathway says it is.  Context is any factor that may make the “correct” approach or therapy not be applicable.  Every few months, I get the obligatory letter from my health plan saying that I should be on an ACE inhibitor due to my history of heart disease.  With my history, taking an ACE inhibitor every day is a good idea and is a part of many care guidelines and standards.  The health plan does not see my context which is that I don’t tolerate ACE inhibitors because they cause me to cough.   Context can be medical issues, social issues, even religious issues.  When I was in practice I cared for a number of people who were Jehovah’s Witnesses.  As part of their religious beliefs they often refused blood transfusions which, as a gastroenterologist treating them for bleeding from their GI tract caused me to modify my approach in order to respect their religious belief.  I found that they could be treated successfully even while being sensitive to their culture and their beliefs, however, the treatment often was not in keeping with the standard care approaches.  Context can be related to issues of culture or simply related to issues of communication and trust.  Dr. Saul Weiner and his group at the University of Illinois have studied context in medical care and have found that care quality is compromised and care outcomes are worse if doctors and nurses do not actively ask about contextual issues and change their approach based on the answers to those questions.
We don’t know the context of the former President’s procedure (although I suspect it was not a financial issue).
Real, Pertinent Information is Often Limited Outside of the Patient and Caregiver:
Perhaps the most intelligent statement made in the Washington Post piece by Prasad and Cifu is “Few facts are known about the case”.  In the case of the former President, we don’t have all the facts about what was said between doctor and patient.  We don’t know if President Bush had mild jaw pain that was considered to possibly be atypical cardiac pain during the long bike ride that was noted.  We don’t know if his ability to do other activities had been affected in small ways that his physician believed to be a warning sign.  The fact is that both media reports, and also the claims data that health plans depend on, are very limited and usually do not include the small clues that may lead a physician and patient in partnership to decide on a specific course of action.  Even electronic medical records may not have all the necessary facts.  It is the height of arrogance to think that through media reports we have enough information to make these types of statements.   We also can be misguided if we make payment decisions and judgments on the quality of care a physician is providing for an individual patient solely on claims data. 
I think there is another way.  I think there is a way to encourage good individualized medical care without the second guessing that is becoming even more prevalent in medicine, insurance and in public policy.  There is a way to include context in care pathways and guidelines; to develop quality systems that review patient physician interactions based both on outcomes (and right now the former President’s outcome looks pretty good) and on the process of individualized care.  That will require more communication and integration within medicine, that goes beyond system integration and back to physicians and nurses actually talking to one another and talking more with their patients.  It will also require that we acknowledge that medicine is really about uncertainty and that includes uncertainty about applying the ever evolving science of pathophysiology to the individual patient to maximize the value to that patient.  It will require a rethinking of our belief that every person fits neatly into a care guideline or insurance medical policy and that claims data can adjudicate every medical interaction easily.  It will require less arrogance and more cooperation and coordination in the pursuit of the best result for every patient.