Edging into the Center: Health Prevention, Improving the Research Process, and the Placebo Effect

It is often difficult to discern what is happening on the fringe of things and then decide whether any of it matters. In the last year, the center of health care has bulged with inevitably growing and demoralizing statistics of illness and costs, predictably warmed by the heat of presidential campaign rhetoric. But at its edge, centrifugal forces have been at work, tilting some usually obscure elements into the light.

When I last wrote for Altarum in the spring of 2011, the National Prevention and Health Promotion Council, its advisory group and the National Prevention Strategy were emerging from the directives of the Affordable Care Act. The ACA’s $15 billion prevention fund was floating precariously in the recessionary winds. The comparative effectiveness research regime and its Patient-Centered Outcomes Research Institute were organizing and preparing to identify first research projects. And the placebo effect remained well out in its usual orbit at the far, far edge of discussion.

A year later, the prevention fund has had a third of its $15 billion stripped away and the balance is still hovering precariously over the chopping block. Its early expenditures appear to have been redirected to replace budgetary reductions at CDC and other programs instead of to objectives identified in the National Prevention Strategy, although some intended initiatives, including Community Transformation Grants, have started to roll out resources to local communities. The NPC and its advisory group are proceeding to define wellness, prevention and integrative health for the nation and to help federal agencies establish their own wellness cultures. As for the placebo effect, if it had any more media attention since the first of the year, it would be planting its hands in wet cement on Hollywood’s Walk of Fame.

In the meantime, in the same yearlong period, the public health community advocated strenuously for protection of the prevention fund and continued to reinforce the importance of prevention and health promotion. For example, in an October 2011 review of the research into the return on investment of prevention, a Robert Wood Johnson Foundation report summarized several studies that projected overwhelming cost savings that could be realized from more substantial investments in prevention. (1) The numbers are compelling, but I was more drawn to the language of ideas that seemed to have tumbled in from the edge. A study cited in Health Affairs, for example, introduced a new modifier for prevention: “protection,” as in “a specific prevention strategy defined as enabling healthier behavior and safer environments.” The study’s recommendation is “efforts to protect health and encourage healthy behavior are a core element of disease prevention.” Another study by the New York Academy of Medicine strongly recommended “focus on community-based disease prevention programs that do not require medical care.”

Protect health by preventing disease without medical care? There is an ethos from the edge. Now we’re getting somewhere. Into any school’s gymnasium, for instance.

Requiring 120 minutes a week of physical education would surely not be thought of as “medicine.” Yet by now we know that not only does the science show the preventive qualities of exercise, it has also shown its salutary effects on cognition. The February issue of The Atlantic Monthly published the article “The Case for Recess,” which described a review of 14 scientific studies of the effects of exercise (studies involved 53–12,000 students ages 6–18). The researchers concluded, “Evidence from the studies included in the present systematic review suggests that there is a significant positive relationship between physical activity and academic performance.” (2) The studies cited in The Atlantic Monthly mirror the work of its Cambridge neighbor, John Ratey, M.D., a clinical psychiatrist at Harvard and author of SPARK: The Revolutionary New Science of Exercise and the Brain, who has been the leading researcher into academic performance boosted by exercise during the last decade.

This work was reflected oddly in an offhand remark made by American Cancer Society chief medical officer Otis Brawley, M.D., at an event that would be thought of as in the center of health care: “The National Public and Stakeholder Dialogue for Patient-Centered Outcome Research,” held in Washington in late February by PCORI. Discussing the factors that PCORI-funded research should take into account, Brawley noted the firestorm created when he was quoted by the New York Times observing that many people believe mammography to be better than it actually is. The experience illustrated for him the serious disconnect between the true capacities of medicine and what the public believes they are, which is that it should all just work. With a hint of frustration, Brawley suggested that it might be better to “increase funding for second grade.” And then what? Prevent disease without medical care?

PCORI was established to create better research knowledge by bringing the patient deeper into the research process, an approach that inevitably leads out to the edge. If the execution of its research agenda actually reflects the aspirations expressed by governing board member Harlan Krumholz, M.D. in his introduction to the day’s dialogue, PCORI may spend quite a bit of time out there. A cardiologist and professor of medicine and epidemiology at Yale’s School of Medicine, Krumholz said that among PCORI’s primary objectives is “to be transformational. Not just about funding, but as a movement that put patients firmly in the center.”

“We have a chance to take some risks now with PCORI resources,” Krumholz said. “Maybe we can invert the traditional power structure just a bit, so that people are ensured that the final decisions are in alignment with patient values and goals.”

PCORI’s first inversion is evident in its proposed research agenda, which does not focus on specific conditions or populations. The panelists from the participating stakeholder associations included several charter members of the power structure, and they did not appear to welcome the prospect of inversion. Several expressed serious skepticism about the idea that nonspecific approaches to health research would yield any appreciable benefit.

Will the framework described by Krumholz truly open research paths that lead out to the edge? Several strong recommendations reflected emerging new patient-centered realties. Stuart Spielman of Autism Speaks advised, “Use patient groups as intermediaries. Science should look at patient groups as allies.”

 In the last couple of years, social media have been used to create new models based on that precept. The Army of Women, formed by the Susan Love Foundation, and the patient community site PatientsLikeMe.com, have been forming and organizing patient communities to play more direct roles in research and data collection. Models like these would fit one of PCORI’s five primary proposed research priorities: “Accelerating Patient-Centered Methodological Research.” (PCORI is scheduled to announce first grant awards in mid-May. See http://www.pcori.org for details.)

And the placebo? Until recently it rested in its usual place, far out on the edge. But for reasons still not entirely clear, The New Yorker, The Wall Street Journal, and “60 Minutes” each undertook penetrating reports in the first 60 days of the new year. A high-level conference cosponsored by the Samueli Institute and the National Institutes of Health’s National Center for Complementary and Alternative Medicine in January was the first of several that will focus on the topic this year. (5)

As with other elements that have moved out from the edge (meditation, the nutrition-cognition connection), the placebo effect has been a subject for recent imaging-based research that shows observable neuromodulations that occur when a beneficial therapeutic result takes place.

At the NIH-Samueli conference, these findings raised a question: If such effects can be made predictable, can they become part of a regime of therapy (e.g., pain reduction) that yields the same or better results for the patient than standard treatment alone? If so, should techniques for activating this healing effect become part of clinician toolkits?

But surely considering such questions would require dancing far out on the edge. Perhaps only a funder truly interested in “inverting the power structure” might even consider them.

A debilitated Prevention Fund. Prevention without medical care. Cognition-enhancing PE. Inverting power structures. Patient-trial-participant communities. Placebos. An odd continuum, to be sure. But whatever their indirect relationship, each reflects concerted efforts to contribute to rebuilding a new center of health for the nation.


1. “Return on Investments in Public Health and Prevention: A Summary of Groundbreaking Research Studies,” Policy Highlight Brief, October 2011, www.rwjf.org
2. http://www.theatlantic.com/health/archive/2012/02/the-case-for-recess/253549/
3. http://www.johnratey.com/newsite/index.html
4. For an eye-opening account of Ioannidis’s role in research transformation, see “Lies, Damned Lies, and Medical Science,” David Freedman, The Atlantic Monthly, December 2010, http://bit.ly/atl-dh
5. I attended this conference as a consultant to the Samueli Institute


SHAM DESIGNS.. Another miss application of the power of placebo response comes from the evaluation of neuro-surgery treatments for Parkinson's disease in North America. The vast majority of Neurologists in this country view the extremely strong and long lasting placebo response to brain surgery as bias to be eliminated by experimental designs that include sham surgery control groups. In Europe this is not an issue, because of the risks for volunteer research participants in sham control groups (not a sugar pill) are considered to be un ethical. After a series of failed pivotal clinical trials for promising treatments (50% improvement lasting 5 years or more in early phase studies), patient advocates questioned the validity of the sham design. Patients view the placebo response to surgery to be part of the treatment. Blinding patients and doctors create an uncertainty that distort and undermine the treatment. Assumptions about independence of treatment and controls are not verified, and the door to false negatives is left open by sham designs. A single blind (for raters of clinical outcomes) design plus time for placebo response to wear off provides more information and greater validity of results. see Miller-McCune Jan/Feb 2012.. http://www.miller-mccune.com/health/placebo-effect-stronger-than-we-thou..., and Nature Aug, 2011.. http://www.nature.com/news/2011/110810/full/476142a.html?s=news_rss

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