Abstract
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Linde's plenary stirred the audience toward a research-oriented action agenda. For those peering downstream at the human costs of this fealty to efficacy, Linde's presentation posed a larger, but not fully articulated question. Is it time for the integrative health and medicine research community to find ways to march for effectiveness science?
A reference point for Linde's explorations was recent guideline development by the United Kingdom's National Institute for Clinical Excellence (NICE). The April 2017 JACM issue spoke directly to these issues. A paper by Birch et al. 2 and an invited commentary by MacPherson 3 challenged NICE recommendations on osteoarthritis of the knee and on back pain, respectively. In each, the decision of the government panel came through elevating efficacy and kicking effectiveness out of the room. (Notably, the scientific judgment reflected a decidedly non-interprofessional political one: no acupuncture or complementary medicine expert was appointed to the panel that made these calls.) NICE's final determination, based on its reliance on effect sizes of acupuncture over sham, was “Do not offer acupuncture ….” 4
Group Health Research Institute's Daniel Cherkin wrote a retirement post last year that echoed Linde's, Birch et al.'s, and MacPherson's perspective. Looking back over a career of examining various integrative practices, Cherkin saw the pattern with acupuncture research outcomes replicated many times over. Sham research on massage, yoga, mindfulness, and chiropractic also often finds little or no effect size differentiating the verum from the decidedly not-inert placebos in those studies. Yet sham plus verum typically reach significance. From the end-user's perspective in particular, it is notable that each arm, alone, is typically more effective than a third arm of usual care.
The paper and commentary each point out that, if one turns over the tables at NICE and prioritizes the effect sizes from effectiveness trials, NICE's recommendation would be turned on its head: “Offer acupuncture.” In fact, acupuncture's effect size perches near the top of recommended practices.
MacPherson suggests the human toll: “… misleading guidance related to chronic pain may inadvertently drive up the utilization of opioids, which is already emerging as an epidemic in the United States.” Recently, in JAMA Network, a prescriber described how physicians who lack access to reimbursed nonpharmacologic tools are “backed into a corner.” 5 Given the high levels of morbidity and frank mortality associated with opioids, how far from the truth is it to suggest that the ultimate “effect size” of the efficacy-worship at NICE includes murderous outcomes?
Linde put a series of questions to the audience. Why, as mentioned at the top of this column, is most of the scientific community thinking efficacy is more important than effectiveness? Is the primary goal of health care effectiveness or efficacy? Why are specific effects so important? Is functional specificity fundamental or is effectiveness fundamental?
For those taking the task seriously, Linde offered a cautionary reminder that “empirical evidence is not interpreted in an empty space.” One need not be on the frontlines of these inclusion debates to respect the wisdom in such guidance. One reaches a certain age, particularly in this era of precision and personalized models of problem solving, when it becomes exceedingly clear that life is all apples and oranges and rutabagas and parsnips and you name it. These pattern into groups with unique lenses to form not-at-all empty spaces from which they interpret the data and stories at hand.
To answer Linde's core question of differential importance is to begin to color in the not-so empty space that shapes interpretations of the effectiveness deniers in the scientific community. The subject merits serious probing with the finest qualitative and quantitative methods. Does anxiety rise for veteran efficacy champions when confronted with the ambiguity inherent in the real world? What happens to their heart rates if asked to imagine working in a context where effectiveness rules and efficacy is relegated to subordinate, precision-determining roles? What fight or flight responses?
Linde suggested some possible factors shaping the preferences. He spoke to the powerful, restrictive influence of the present culture of medical science. Think of inculcated hierarchies, habitual practices, and known funding channels. I found myself musing wildly. Is favoring efficacy's dream of perfection over effectiveness' grimy ambiguities a vestige of religious beliefs in a life beyond that is better and purer than the one in the mirror? Linde offered a nod in a less speculative direction. He alluded to the aspersive charge that polarizes the integration debate: protection, or enhancement, of one's personal and financial interests. Efficacy stacks the deck for those associated with drug practices measured well through relatively inert placebos.
Policy, Linde announced, is not his forte. Then began in a matter-of-fact way to offer the roughly 800 researchers, clinicians, and administrators from 59 counties a set of action steps. He urged the audience to accept as fact that different groups will interpret the evidence on topics very differently. He gave climate change as an example. “It is unlikely,” he said, “that further meta-analyses will change the outcomes.” More of the same won't do it.
He suggested course corrections. He would not typically “favor” placebo-controlled trials as a method for examining the value of integrative services. Birch et al. and MacPherson were with him on this. He took it global. The “concept of the placebo, even though engraved in our minds, needs rethinking.” What would such a revolution be without some Thomas Paine-type common sense: “One can't separate the nonspecific from the specific effects in human behavior, so why try to separate them in research?”
Linde hedged his bets, however. In closing, he bowed to the dominant view in biomedical science and among those researchers who are invited to form policy-making panels. He urged the room of researchers and clinicians to not abandon efforts to understand specific effects. In a closing slide, he wrote: “Functional specificity is central to legitimizing a therapy. Believing in functional specificity is crucial for a provider! Explaining and showing this remains important for complementary and alternative medicine therapies! If you fail this is a problem!”
The choice of efficacy over effectiveness is not a controversy that involves only scientists, or policy makers. The effect size of scientific decisions to favor efficacy reaches through coverage decisions to clinical practice choices and thus to human health outcomes.
This empowerment of efficacy and concurrent diminution of effectiveness shapes health potentials well beyond acupuncture and pain and NICE, and the United Kingdom. It influences multiple interventions, for scores of conditions, in every nation. What are the human health outcomes of the dominant school of science's preferential devotion to efficacy and the isolation of specific effects over the effectiveness that may be more meaningful to both patients and practitioners?
The Berlin meeting at which Linde spoke followed less than 2 weeks after than the international March for Science. 6 The image that lifted off Linde's musings was of integrative research and practice communities marching, in dialogues, in their own departments, before policy makers, and elsewhere for a new era of respect and encouragement for effectiveness science. It can comfortably be framed as a research adjunct to the patient-centered care movement. Such engagement could have a positive effect size far beyond this present dialogue.