Columnist Taylor Walsh

The June 2009 Institute of Medicine Report to guide federal expenditures on Comparative Effectiveness Research (CER) elevated "complementary and alternative medicine" as a subject for which a CER approach would be useful. IOM's inclusion of CAM would seem to create a venue for addressing assertions of countless integrative practitioners that it's a damn shame that more of what they do is not covered as their experience is that health care would be more effective (and cost effective) compared to conventional treatment.

The Integrator has since followed CER developments, including NIH National Center for Complementary and Alternative Medicine (NCCAM) director Josephine Briggs, MD taking a lead role on an NIH-wide CER committee, the appointment of Christine Goertz, DC, PhD to the Board of Governors of the huge new multi-year CER-focused Patient Centered Outcomes Research Institute, and the elevation of "real world research" inside of NCCAM's 2011-2015 strategic plan.

Integrator columnist Taylor Walsh, a consultant and writer on integrative health and digital media in Washington, DC, most recently covered CER at a meeting hosted by the Institute for Integrative Health (TIIH) that brought together leading integrative practice researchers with leaders in the CER field. The co-leader of the TIIH gathering, Sean Tunis, MD, was also convenor for this "National Leadership Summit on CER Priorities, Methods and Policy: Transforming Oncology through Comparative Effectiveness Research,

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Patient Centered Outcomes Research and Oncology Transformation

- Taylor Walsh
Getting to Integrative Health & Wellness
[email protected]
Twitter:  @taylorw

The "National Leadership Summit on CER Priorities, Methods and Policy: Transforming Oncology through Comparative Effectiveness Research," held Nov 1-2, 2010 in Baltimore was a high-level gathering of several stakeholder communities with strong interests in the work of the Patient Centered Outcomes Research Institute, PCORI, established by the CER legislation earlier this year.

Tunis: Convened the exploration

The PCORI council was formally organized and its members appointed in September and is now in the process of establishing its committees, notably on CER methodology. The convener of the summit, Sean Tunis of the Center for Medical Technology Policy (CMPT), said that the purpose of the working conference was to "help PCORI create a CER framework for oncology."  Tunis and conference keynoter, former CMS director Mark McClellan, noted that cancer provides a good foundation as a model for examining the many questions that the CER program is intended to address, in particular for patient involvement in the process.

"The preferred approach is to design studies that look like the real world that don't look like NCI-sponsored RCTs."

CMTP has convened a number of CER and PCORI-focused workshops and meetings in the last 18 months, including co-hosting the "Complementary and Integrative Medicine Stakeholder Symposium" with the Institute for Integrative Health in November of 2009, which was a first occasion for integrative researchers to work with CER experts.

As he has done introducing other meetings, Tunis reiterated the over-riding purpose of the CER initiative itself, to address the inconsistencies that plague medical research.  "The gaps in evidence," Tunis said, "reflect insufficient information for decision makers."  Later, responding to a question, he boiled down the preferred approach that is evolving from such CER discussions to "design studies that look like the real world...that don't look like NCI-sponsored RCTs."

McClellan added, "There are many unanswered questions related to lifestyle, screenings, targeted treatment - where we can identify those who are most likely to respond, and delivery."

"More patient-centric approaches," he said, will enable researchers to "do work that is relevant to funders."

	As a practical matter for integrative practice and research, PCORI creates an intersection with the research agenda now being prepared by NCCAM as part of its strategic plan.

While the full conference included examinations of genomics, clinical evidence and the value and costs of oncology research, this report primarily covers the session entitled "Engaging Patients in CER."   This is perhaps the most problematic point of focus for CER, since patients reside in that "real world."  Much of the CER initiative's establishing language encourages inclusion in the evidence base what happens during the clinical encounter.   which explicitly includes the need to address the every day reality of CAM and integrative approaches throughout the population.

The potential for patient engagement in CER was presented by speakers from the nation's most prominent breast cancer advocacy and research organizations: The Susan Love Foundation; Susan B. Komen for the Cure; National Breast Cancer Coalition; the Research Advocacy Network (Otis Brawley, MD, chief medical officer of the America Cancer Society, delivered a keynote).

Susan Love, MD, MBA immediately expressed what many people believe. "The public is not invested in CER," she said.   Susan Brown, MS, RN of Komen concurred: "We have not heard patients talk about CER."  The corollary to this is the belief that patients and the public have a great deal to contribute.

Amy Bonhoff, MBA, of NBCC, noted that, "Consumers have a voice that has a powerful effect, (which is) acknowledged as insightful by specialists."  Deborah Collyar, president of Patient Advocates in Research said, "Patients are a rich source that is hardly ever used." 

On patient-centered research, Susan Love, MD, MBA: "Dis-intermediate the medical establishment entirely."

Love had the most to offer on an active role of the public, which she said, "is ready and willing to participate."  But "they don't create studies, they depend on the medical establishment to recruit and collect data."  As Love described it, this dependency is the flip side of the same problem: a flawed recruitment process that too often produces flawed research.  The providers and payers (and the electronic medical records and health claims data that are the heart of the patient record) she said, "are not set up for this purpose."  In fact, they're in the way.   Her strongly delivered recommendation: "Dis-intermediate the medical establishment entirely."  

Love's organization is already well down this path.   In partnership with the Avon Foundation, it has created The Army of Women, formed in 2008 as a matching service for researchers.  Prospective participants self-select by registering independently on the website (and are then screened and organized by the staff) thus forming an available trial participant resource for researchers. The site has more than 345,000 registrants (growing at some 4,000 a month).  Forty trials have been initiated, with 17 completed.  Of consequence to researchers studying other real world conditions, the model has drawn the interest of other chronic disease communities, including those that already have a significant online presence.  

	This paradigm of "social research," according to Love, is based on bringing the public in at the beginning.

This paradigm of "social research," according to Love, is based on bringing the public in at the beginning.  It is worth noting that 80% of her registered members are not patients or survivors. This is as good a model as any for PCORI to mull over, since it is built on the place patients and their advocates can mostly be found and the continuing hand off of health responsibility to individuals.

Less dramatic recommendations and observations from the advocacy community included these from Bonhoff of NBCC:

• The confusion and emotional upset that arises from conflicting results, such as the mammogram guidance reported earlier this year.
• The pressure to employ very expensive technologies, such as fMRIs
• Over-treatment ("Few trials look at reduction," she noted.)

Speakers also warned about perceptions, as CER starts to receive more public attention.  Bonhoff said it could be perceived as limiting choice.  Collyer said, "My biggest concern is that the ‘C' will stand for cost."

Next steps in PCORI/CER process

McClelland, now with the Brookings Institution, said that several cost-based reforms will be tried in the next two years under the CER program, and noted the formation of an independent payment advisory board that wants to reduce research spending per person by 3 -4%.  "We need a stronger foundation for what works," he said, emphasizing the need to strengthen the "infrastructure for care improvement." Including:

• Data infrastructure: with consistent patient information collection and exchange, registries and decision support tools
• Consistent measurement: collecting and aggregating outcomes and costs data
• Payment reforms
• CER strategy and support: to build randomization structure into trials that will help evaluation of policy benefits, and show how specific sub-groups are being treated

Iioannidis: 90% of usual studies problematic

Post-Election Prospects for CER?

As this conference was held the week before Election Day, Tunis felt compelled to speak to the looming political question that now hangs over CER and the entire new health law.  "The problems and principles of approaching research are firmly embedded," he said.  "They will persist whether or not PCORI exists (as a distinct program).  We've gone past the tipping point."

The creation of the CER program was a reflection of an awareness of problems in medical research first observed in the 1990s.

The creation of the CER program was a reflection of an awareness of problems in medical research first observed in the 1990s.  For an eye-opening report of how far past the tipping point the CER train has gone, see the November 2010 issue of Atlantic Monthly, which describes the work of John Iioannidis, MD, DSc (as of September 2010, the head of the Stanford Prevention Research Center), whose now much embraced studies on the process of medical research itself concluded in 2005 that "as much as 90% of the published medical information that doctors rely on is flawed."  Well known in the research community since its release, these disturbing findings will now reach coffee tables and web screens in the real world, on paper and here at Atlantic's web site. (Stanford release on Ioannidis appointment is here.)