What matters is what we know, not what we think
In the late 1980’s I cared for a pregnant woman with breast cancer. Breast cancer is the most common cancer in pregnancy, but uncommon in number, occurring in about 1 in 3000 pregnancies. It is a compounded emotional treating experience for sure, and at that time uncertainty in how to treat was the norm. The woman had a mastectomy but did not take chemotherapy based on concern for her baby.
Three months after her delivery, now getting chemotherapy for her aggressive breast cancer, the woman asked me to consider treating her newborn child with “mild” chemotherapy, a clear contrarian idea given her reluctance to expose her child while in her uterus. Her reasoning, she said, after giving it “lots” of thought, was that it made sense to her; she had cancer at a young age and reasoned her child would also. In her mind it was rational and reasonable to give her child treatment.
Fear and depression fueled her concern, for sure, and universally we would deny the request. The woman would not live to see her child’s second birthday and wanted to do what she could for her. But, there was no evidence of benefit to the baby, making her request irrational. So, I did not comply. In fact, what would you have thought of me if I had complied with this woman’s concerns?
In 1882 a surgeon reasoned that removing a woman’s cancerous breast, nodes, and muscles was the way to get rid of breast cancer. There was no proof of benefit, but the idea spurred action. That idea, and the radical surgery, persisted even after the publication of a randomized trial (NSAPB-04) in 1974 showing that more surgery was not better than less; yet, about 5000 radical mastectomies were still performed in 1983, about 100 years after the first. What should we think of physicians who acted without proof of benefit, and, even, after proof of no benefit?
In the 1990’s over 40,000 women had high-dose chemotherapy and bone marrow transplant if they had breast cancer. Some of those women died of treatment; I know so because I knew some. The intervening years between the ideas of doing more and more to a woman with breast cancer and the publication of randomized trials showing that more and more was actually less was marred by politics and legal threats for not performing the “best”, and most. All this wrangling occurred without knowing if the treatment plan was better than others. What should we think of those physicians, lawyers, and insurers who forced compliance and then complied with an idea rather than knowing what was best?
In a previous blog, I showed data on bilateral mastectomy for DCIS; there is data, but no information, as comparative studies have not been done [link]. I was consulted by several women who were considering complying with the bilateral procedure based on ideas that getting rid of everything might be good for them. Their physicians had proposed the procedure. What should we think of these physicians for proposing an unproven procedure based on an idea?
Acting without knowing, in my view, should be considered an abdication of professional responsibility. A professional obligation includes informing patients that there is no evidence of benefit for some treatment plans, and then not proposing the plan.
The only way a decision can/should occur is if there is a balance between compared options. The compared options must be examined in studies that give the best chance of determining the independent contribution of one intervention versus another (randomized). The balance that emanates from these studies is that some treatment will be shown to independently contribute to better disease related outcomes at the expense of more treatment related complications. The differences in outcomes of disease and treatment compete for a patient’s attention, and choice.
Without such comparisons, no choice should be made. Ideas don’t suffice; it doesn’t matter whether the ideas are the physician’s or the patient’s.
How many 100, 000’s of women underwent radical mastectomy before it took only 1700 in a randomized trial to show it offered no benefit despite increased harm? What about high dose chemotherapy and bone marrow transplant? Six randomized trials, reviewed in 2005, including only a cumulative total of 850 women, showed high-dose treatment offered nothing, but studies kept trying to prove the value until finally 14 randomized trials, now including 5600 women came to the same conclusion. Far more women got the procedures before knowing what was best than the number required to find out what is best in the first place. This should be a lesson to us all. Knowing is better than not knowing, and doing without knowing may be the worst thing a profession can do.
I am writing this blog with a purpose. That purpose is to make physicians and patients uncomfortable about acting on beliefs without evidence from comparative research trials. I believe patients are the best decision makers, but that will be shown to be true only if they are informed and allowed to decide.
But, physicians need to be on board, as well. They should not allow treatments of unproven benefit and, instead, they should demand study over action. There are too many examples of the failure of “good ideas” to subsequently help patients. What matters is what we know, not what we think. Informing patients that no evidence exists, and promoting a dutiful process of scientific inquiry could be a powerful way to change medical care for the better.
Great comments; thanks. The key word, Barry, in your comment is “experimental”. I am an oncologist and some of my patients tried experimental treatments, but only under the stringency of evaluation. Also, phase 1 trials should not be confused with trials to evaluate marginal benefits to many. We should not confuse treatments aimed to show any effect in desperate situations for individuals when outcomes will be measured in days to weeks with outcomes measured over many years in many people. There is much to say about how science should proceed (we can do so much better), and there will be exeptioins, but the exceptions do not disprove the rule; study over hunches.
I find this contribution to THCB very thought provoking in light of a recent contribution by By Niran AL-Agba, MD (Will CVS-Aetna Merger Lead to “Separate But Unequal” Healthcare?) which questioned patient safety by using pharmacists and nurses in CVS clinics instead of MDs.
I have been the recipient of a more than a few “diagnosis” by MDs in my lifetime of 65 years and many have been useless and wrong. I’ve done better by my own trial and error. In the last few years, especially when I was uninsured, I used a local urgent care with a very competent PA, and have continued to go there on the rare occasions I need “diagnosis”.
This has led me to having adopted a protective rule that says trust no one with a degree. This doesn’t mean I disregard what I am told, but it means to dig further before taking anything at face value and blindly trusting “experts”.
What about experimental treatments for patients in end of life situations? If the patient is fully informed of risks and benefits and still wants to give it a try, isn’t that OK from a medical ethics standpoint? At the very least, doing the procedure should have some research value. Cost and who pays are separate issues.
Eventually, the concept of universal health insurance will need a means to protect itself from “ad hoc” trials of novel healthcare strategies. The opportunity to fund this healthcare, fully, with unique philanthropy or research studies should continue but not effect the over-all funding of healthcare related to the COMMON GOOD of each community. Currently, we lack a tool to assess the fundamental level of stability for a person’s HEALTH at any one point in time. It likely represents a means to use the frequency domain for assessing non-linear control systems for characterizing a person’s fundamental thermodynamic resiliency, clearly a very hypothetical concept.
For Basic Healthcare Needs, it is almost always better to do nothing when there is no well defined basis for intervention. As for Complex Healthcare Needs, it is acceptable especially for rapidly Emergent Health Conditions to act with less well defined analysis for intervention. In the absence of enhanced Primary Healthcare that is equitably available to each citizen, excessive healthcare for Complex Healthcare Needs is often associated with a preceding level of inconsistent health care for Basic Healthcare Needs. Overly aggressive health care for Complex Healthcare frequently occurs in the absence of a preceding level of Primary Healthcare devoid of trust, cooperation (patient responsibility) and reciprocity (supported by responsive medical Triage). I propose that the level of Social Capital governing the medical triage offered to each person by their Primary Physician would very closely predict its global effectiveness and efficiency, not by 2-3% but by 15-20%.
Enhanced Primary Healthcare depends principally on the caring relationships that are offered by the Registered Nurses who directly answer the Triage phone line during office hours. During our weekly staff meeting, at least 10-15 minutes was spent on the scheduled review of medical Triage algorithms. One of the most important algorithms was a call from someone with a chief complaint of a new breast lump (NOTE: during 40 years of practice, there were three men in our practice who developed malignant breast cancer!). The next comment for the nurse was discussed at length: “can you come to the office right now.!” A good nurse knows how to be supportive in the midst of this interchange to make it happen in the midst of a person’s busy civil life.