JAMA- Charting a Path From Comparative Effectiveness Funding to Improved Patient-Centered Health Care | Friends of Cancer Research

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JAMA- Charting a Path From Comparative Effectiveness Funding to Improved Patient-Centered Health Care

by Carolyn Clancy and Patrick Conway, PATIENTS AND PHYSICIANS ARE FORTUNATE TO Confront expanded options for diagnosis, treatment, and monitoring.

While physicians strive to tailor recommendations to the unique characteristics and circumstances of each patient, many decisions today must be made without reliable comparative information. That information gap is frustrating for patients, who too often undergo trial and error medicine, as well as for physicians. Moreover, few health care organizations are designed to harness the best clinical insights for widespread dissemination and learning. It is precisely this chasm that a focus on comparative effectiveness (CE) research, also known as patient centered health research, is intended to address.

The concept of CE research is not new; indeed the establishment of the Agency for Healthcare Research and Quality (AHRQ) 20 years ago represented an important investment in supporting science that helps identify which options are most effective for which patients. In addition, the more applied aspects of clinical research often address comparative benefits and harms of therapeutic choices. What is new is the recognition of and substantial public support for research that is essential for delivering care that is consistently patient centered and an important accelerator for achieving the promise of personalized medicine.1

However, the dimensions of the scientific foundation from which to advance this enterprise are not clearly defined. For example, the Institute of Medicine (IOM) committee commissioned by the US Congress to identify priorities for CE research relied on broad stakeholder input and expert judgment regarding important clinical topics, but had no clear inventory of published studies or research in progress to inform their deliberations.2 Moreover, the context for increasing interest in comparative information implicitly includes assessment of whether the resulting information is useful to clinicians and patients—and is used.

In this issue of JAMA, the article by Hochman and McCormick3 represents an important step in articulating criteria for published studies that assess the CE of medications. The authors conducted an analysis of CE research studies from the 6 general and internal medicine journals with the highest impact factors. Their study highlights several issues that warrant further discussion.

First, the authors report that 87% (90 of 104) of the CE research studies published in these journals relied on noncommercial funding. The American Recovery and Reinvestment Act appropriated $1.1 billion for CE research and the US Department of Health and Human Services is now awarding grants and contracts based on these funds.4 The Federal Coordinating Council for Comparative Effectiveness Research report outlined a vision for how this funding could lay the foundation for an enterprise that improves the nation’s health and the performance of the US health care system. 5 The budget for fiscal year 2011 issued by President Obama recommends $286 million for the AHRQ portfolio dedicated to this type of research—a $261 million increase from the pre–American Recovery and Reinvestment Act baseline. 6 Given the importance of noncommercial funding to CE research, funding from AHRQ, the National Institutes of Health, the US Department of Veterans Affairs, and others will be essential to continue to the momentum of American Recovery and Reinvestment Act funding.

Second, the authors identified only 11 studies (11%) that compared medications with non pharmacologic interventions and 32 studies (31%) that compared different pharmacologic strategies (31%); both are essential to helping clinicians and patients make fundamental therapeutic decisions.

Similarly, both the IOM and the Federal Coordinating Council for Comparative Effectiveness Research included a broad definition of interventions within the spectrum of CE research including procedures, medical and assistive devices, diagnostic testing, behavioral change, and delivery system interventions. 2,5 Future CE research inventories should capture and categorize the full spectrum of these interventions.

Third, the authors note that only 32% (n=104) of medication studies published in these 6 journals met their criteria for CE research. Since many clinical trials, including those supported by the National Institutes of Health and the US Department of Veterans Affairs address benefits and harms of treatments, this finding raises important questions related to where these studies are published, the completeness of ascertainment, and possible publication bias. It is likely that major medical journals will publish an increasing number of CE research studies, given the focus on the real-world choices faced by clinicians and patients.

Fourth, the authors found that only 20 CE studies (19%) focused on safety. Both the Federal Coordinating Council for Comparative Effectiveness Research and IOM definitions of CE research highlighted that assessment of benefits and harms was an important component of CER research. The authors’ call for inclusion of safety outcomes raises questions regarding the often unclear boundary between post marketing surveillance to detect product deficiencies and comparative assessment of harms due to medications or other interventions (ie, CE research). The substantial but incomplete overlap between these 2 endeavors, both requiring similar data resources, methods development, and training, suggests fruitful opportunities for collaboration.

There are several next steps to disseminate and translate CE research into practice. The short-term goal should be that an Inventory and analysis similar to that completed in this article will no longer be necessary because a CE research inventory will be readily accessible by researchers, clinicians, patients, and others. The US Department of Health and Human Services will establish such an inventory and it will be essential for a CE research inventory to build on current mechanisms to capture studies (eg, tagging of published results via PubMed).The inventory also will need to be a dynamic system that allows for entities and individuals to contribute CER research findings and these studies should be vetted for inclusion and categorized. In addition, the inventory must include both private sector and non–private sector CE research and therefore incentives for participation will be critical. In addition, an effective inventory should include categorization of studies and searching mechanisms so clinicians and patients can locate summaries of evidence and studies of interest.

Perhaps most important is the question of how CE research will be disseminated to clinicians, patients, and others in a way that informs decisions, improves health, and enhances the performance of the health care system.7 A substantial portion of the Office of Secretary funds for the American Recovery and Reinvestment Act CE research was designated for the segment of the strategic framework termed “Dissemination, Translation, and Adoption.”4,5 These investments will include multiple modes of disseminating CE research information and testing which of these methods are more effective in changing behavior. This also includes investment in testing strategies to increase adoption of CE research by clinicians and patients.8 In addition, the best methods of delivering this information at the point of care must be determined to integrate the information into the normal workflow and decision processes of clinicians and patients.7 Moreover, peer-reviewed journals can further the CE research enterprise by publishing an increasing number of well-designed CE research studies and accepting studies that use established and novel methods to analyze research questions in real-world settings.

Both the Federal Coordinating Council for Comparative Effectiveness Research report and the IOM report highlighted the importance o fCE research being guided by the needs of clinicians and patients. This concept of research directly addressing the most important real-world decisions faced by clinicians and patients is paramount to CE research having maximal effects and benefits. The challenges and frustrations for clinicians and patients of daily clinical decisions necessarily made under conditions of uncertainty are self-evident. The United States leads the world in biomedical science and now must implement a framework for research that advances patient-centered care.9 This enterprise, both public and private, is by definition widely distributed so transparency and ongoing participation by all stakeholders—researchers, clinicians, patients, and health care leaders—is essential. Further, the challenges of increased longevity and prevalence of chronic conditions demand innovative approaches to longitudinal assessments, enhanced methods for learning from daily practice, and strategies for dynamic interactions between researchers and the front lines of care.

Both the Federal Coordinating Council for Comparative Effectiveness Research and IOM have provided highly congruent frameworks for priority setting, conduct and dissemination of research, and ongoing engagement of all stakeholders including the needs of diverse populations and patient subgroups. If the CE research or patient-centered health research enterprise maintains this focus, it will reach its full potential and transform care delivery by closing the gap between physicians’ and patients’ aspirations and the reality of current practice.


1. Garber AM, Tunis SR. Does comparative-effectiveness research threaten personalized medicine? N Engl J Med. 2009;360(19):1925-1927.

2. Institute of Medicine; Committee on Comparative Effectiveness Research Prioritization. Initial National Priorities for Comparative Effectiveness Research http://www.nap.edu/catalog/12648.html. Accessed February 8, 2010.

3. Hochman M, McCormick D. Characteristics of published comparative effectiveness studies of medications. JAMA. 2010;303(10):951-958.

4. US Department of Health and Human Services. ARRA comparative effectiveness research funding. http://www.hhs.gov/recovery/programs/cer/index.html.

Accessed February 8, 2010.

5. Federal Coordinating Council for Comparative Effectiveness Research. Report to the President and the Congress, June 30, 2009. http://www.hhs.gov/recovery/programs/cer/cerannualrpt.pdf. Accessed February 8, 2010.

6. US Department of Health and Human Services. Budget of the United States government browse fiscal year 2011. http://www.gpoaccess.gov/usbudget/fy11 /index.html. Accessed February 8, 2010.

7. Conway PH, Clancy C. Transformation of health care at the front line. JAMA. 2009;301(7):763-765.

8. US Department of Health and Human Services. ARRA OS: Recovery Act 2009: accelerating adoption of comparative effectiveness research results by providers and patients (R18). National Institutes of Health Web site. http://grants.nih.gov /grants/guide/rfa-files/RFA-AE-10-001.html. Accessed February 8, 2010.

9. Dougherty D, Conway PH. The “3T’s” road map to transform US health care: the “how” of high-quality care. JAMA. 2008;299(19):2319-2321.