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Advancing Methods for Comparative Effectiveness Research

Smith, Scott R. MSPH, PhD

doi: 10.1097/MLR.0b013e3181e1cde5

From the Pharmaceutical Outcomes Research & the DEcIDE Program; AHRQ, Rockville, MD.

The author of this article is responsible for its content. Statements should not be construed as official endorsement by the Agency for Healthcare Research and Quality or by the US Department of Health and Human Services.

Reprints: Scott R. Smith, MSPH, PhD, Pharmaceutical Outcomes Research, Agency for Healthcare Research and Quality, US Department of Health and Human Services, 540 Gaither Rd, Rockville, MD 20850. E-mail:

Since the Effective Health Care program was established in 2005 through the Medicare Modernization Act, the Agency for Healthcare Research and Quality (AHRQ) has regularly convened researchers from different disciplines to propose, discuss, and deliberate new scientific approaches for conducting studies on comparative effectiveness and safety. These forums are intended to advance the science and strengthen the methods of comparative effectiveness and patient-centered outcomes research, so that studies produce valid scientific evidence that helps to inform the decisions of patients, providers, and policymakers. With the American Recovery and Reinvestment Act of 2009 and subsequent health care reform legislation, the national interest in comparative effectiveness research and the infrastructure for conducting it has been substantially expanded. This expansion has renewed interest by scientists and consumers of research to find new and better approaches to design studies so that high quality evidence about the effectiveness of health care interventions is generated. Although all research has limitations, the development of new methods for conducting comparative effectiveness research is intended to reduce threats to the validity of research findings, improve the transparency and reproducibility of these investigations, and facilitate the generation of new knowledge about the balance of harms and benefits of therapies.

In June 2009 AHRQ sponsored a symposium on research methods for comparative effectiveness studies and this supplement to Medical Care represents the proceedings of the symposium as revised by the authors and independently reviewed by peers and the journal. The major themes of the symposium were developed with substantial input from external stakeholders, researchers, and federal scientists who have expertise in conducting comparative effectiveness studies. With this input, a Planning Committee of approximately 10 experts from academia, the private sector, and government determined the 2 main emphases of the symposium: (a) examination of ways to enhance the inclusion of clinically heterogeneous populations in comparative and clinical effectiveness studies and (b) ways to implement longitudinal investigations that capture longer term health outcomes, including patient-reported outcomes. Cutting across these themes were 4 major content areas for which abstracts were solicited from scientists across the United States: (1) study design, (2) data collection, (3) statistics and analytic methods, and (4) policy issues and applications.

The 2009 symposium was a direct follow-up to the 2006 AHRQ conference on Emerging Methods in Comparative Effectiveness and Safety; papers presented at that conference appeared in a 2007 Medical Care supplement. As I wrote in 2007, this supplement is meant to serve as a resource to scientists investigating the safety and effectiveness of treatments, particularly the effects of therapies whose efficacy has been previously established through randomized controlled trials. This supplement is not intended to endorse particular research methods or address all the challenges of conducting observational and quasi-experimental research. Instead, it brings together the current thinking of experts in the field to publicly discuss ways science can be appropriately used to provide valid and useful information for improving health outcomes and take advantage of the growing sources of electronic health information for improving public health.

As access to insurance coverage for health care is expanded in the United States, a new national focus has emerged on understanding the outcomes, effectiveness, and comparative effectiveness of treatments in clinical practice. This includes improving understanding about the balance of individual harms and benefits for all forms of therapies and diagnostic tools as they used in clinical care. An overarching goal of comparative effectiveness research is to improve patient health outcomes by providing unbiased scientific evidence that informs the health care decisions of patients, their caregivers, clinicians, and policy-makers. Hopefully, the symposium and this journal supplement helps to advance the science of comparative effectiveness and improve patient-centered outcomes research.

I extend my sincere thanks to the many people who contributed to the symposium and the production of this journal supplement. I am particularly grateful to Dr. Kathleen Lohr and her colleagues at the RTI DEcIDE center who lead this project with AHRQ and helped guide its development. The symposium and this supplement would not have been completed without Kathy's leadership, experience, and dedication. I also acknowledge and thank members of the Planning Committee who were Wade Aubry, MD; Jean Paul Gagnon, PhD; Eric Johnson, PhD; Malcolm Maclure, PhD; Sharon-Lise Normand, PhD; Mitchell Sugarman, MBA; Jean Slutsky, MSPH, PA; and Thomas Trikalinos, MD, PhD. The project is also indebted to several staff at AHRQ, particularly Dr. Carolyn Clancy who serves as AHRQ's Director. I am also immensely thankful to each of the authors for their contributions, particularly in meeting a compressed publication timeline.

© 2010 Lippincott Williams & Wilkins, Inc.