Emond
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While expanding access to healthcare has been the major focus of healthcare reform until now, efforts to improve patient care while confronting rising costs are rapidly coming to the fore. One effort sure to be closely watched will be an expanded program of comparative clinical effectiveness research led by the new, independent, nongovernmental Patient-Centered Outcomes Research Institute.
The institute's mission is sweeping, if daunting: to help patients, providers, payers and policymakers make better healthcare decisions based on scientific evidence, through rigorous comparative effectiveness research and thorough dissemination of the findings. The institute will be governed by a 21-member board to be appointed by the end of September by the U.S. comptroller general and representing various sectors of the healthcare system: seven physicians, four from government, three consumer representatives, three private payers, three manufacturers and one health services researcher.
Fleishman
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The diverse membership of the board should help ensure that research findings are diffused widely through the healthcare system, but it could also make consensus more difficult to reach. For the institute to be truly effective, we consider the following to be essential:
Mission-driven leadership. A key component of the institute's success will be creating an infrastructure for sponsoring, conducting and disseminating studies in new and innovative ways. To do this, the leadership of the institute will need to be composed of well-respected, independent experts with experience navigating multistakeholder organizations and building consensus. The board needs to act quickly to turn its diversity into strength and to establish procedures, protocols and processes that demonstrate its independence and transparency in decisionmaking.
Thoughtful dissemination. The institute will build on existing programs of the Agency for Healthcare Research and Quality in disseminating comparative research findings, yet successful dissemination will require action that goes well beyond AHRQ's capabilities. From the outset, the institute must commission research that is truly actionable—research that decisionmakers can quickly and seamlessly adapt into practice at the point of care.
Research methodologies will have a critical influence not only on whether research findings are clinically useful, but also on whether patients and clinicians consider them valid in the first place. Advanced analytical methods are available to derive important clinical findings from information that would otherwise be deemed insufficient, but the use of these methods must be transparent, and findings must be communicated in ways that convey both clinical effectiveness and the strength of underlying evidence.
As we learned from the U.S. Preventive Services Task Force's mammography controversy last year, rigorous research can prove ineffective and even counterproductive if patients do not accept the logic or the validity of findings. The institute needs to embrace a sophisticated use of methods that will produce new evidence, and it needs to fully involve patients and clinicians in communicating the findings.
Preserving innovation. Most healthcare technologies (drugs, devices and procedures) must meet regulatory thresholds of efficacy (Can it work in ideal clinical settings?) to enter the market. Comparative effectiveness research will place a new emphasis on demonstrations of effectiveness (Does it work in real-world settings?).
This shift could have a profound effect on innovation throughout the healthcare system. Many innovations require years of market use to accumulate enough data to be subject to robust comparative effectiveness evaluation. With its inclusive membership, the institute has an opportunity to develop guidance for the technology community that will establish reliable standards of evidence for the demonstration of effectiveness. Medicare's “coverage with evidence development” policy may provide a rough blueprint for more expansive policies that will create clear rules of the road for technologists and spur the early development of technologies designed to achieve demonstrable effectiveness. The institute should explicitly address issues of innovation throughout its emerging program.
Addressing value. Lawmakers worded the institute's charter so that issues of cost and value are handled carefully in the research. Specifically, the legislation prohibits the use of certain cost-effectiveness measures, such as quality-adjusted life years, in determining if interventions are cost-effective or recommended. The legislation also sets parameters on the government's ability to use information from the institute to make coverage decisions.
Nevertheless, issues of value are implicit in a wide range of real-life medical decisions, from decisions on expensive life-saving interventions to decisions on the daily management of chronic disease that affect out-of-pocket costs borne by patients. The contentious political environment around comparative effectiveness research suggests that new cost-effectiveness studies are likely to be generated outside of the institute.
Yet in the priorities it sets for research on clinical effectiveness, the institute has much to add to the knowledge base on value in medical decisionmaking. Many stakeholders will need to address issues of value in their decisions; the relevance and usefulness of the institute's research may hinge on its ability to navigate the issue of value.
The Patient-Centered Outcomes Research Institute has a broad and important mission to produce, support and disseminate comparative effectiveness information. For it to be successful, the institute must remain transparent and inclusive in its actions so that it can engage decisionmakers in the important process of improving the quality and value of the services delivered across the healthcare system.
Sarah Emond is chief operating officer of the Institute for Clinical and Economic Review, Boston. Valerie Fleishman is executive director of the New England Healthcare Institute, Cambridge, Mass.
