Understanding and Applying Comparative Effectiveness Research

Atlanta—Driven by the variation in medical care and the increasing cost of healthcare, the US healthcare system is undergoing a transformation in the way healthcare is accessed and delivered. Central to this transformation is the push for comparative effectiveness research (CER) that relies on collaboration with providers to improve quality outcomes and affordability as well as engagement of consumers to enable more informed healthcare decisions. To help pharmacists and other healthcare providers understand and apply CER, Daniel C. Malone, RPh, PhD, professor at the University of Arizona Colleges of Pharmacy and Public Health, and Lisa Hines, PharmD, clinical pharmacist, Center for Health Outcomes & PharmacoEconomic Research, University of Arizona, Tucson, provided an overview of key concepts of CER in a Contemporary Issues session at the AMCP meeting titled Comparative Effectiveness Research: From Concept to Practical Application. Citing the Federal Coordinating Council for Comparative Effectiveness Research, the group formed under the American Recovery and Reinvestment Act of 2009 to coordinate CER, the speakers highlighted that the goal of CER is to improve health outcomes by conducting research in “real world” settings that compare the benefits and harms of different interventions (eg, medications, surgical procedures, medical devices) to improve healthcare. Central to the goal of CER is to disseminate evidence-based information to patients, clinicians, and other healthcare decision-makers. Currently, the top 10 research areas of CER, as identified in an Institute of Medicine report on CER, are in healthcare delivery systems, racial and ethnic disparities, functional limitations and disabilities, cardiovascular and peripheral vascular disease, geriatrics, psychiatric disorders, neurologic disorders, pediatrics, endocrinology and metabolism disorders, and musculoskeletal disorders. The federal government has developed an Effective Health Care (EHC) program administered by the Agency for Healthcare Research and Quality through the US Department of Health and Human Services to conduct CER with the goal to inform policymakers, healthcare providers, and patients about the best healthcare decisions available by providing data from CER studies. Key components of these studies that inform decision-making include innovative research, a comparison of medical options, and patient-focused research. After identifying a topic to research, such as an assessment of which hypertensive medication is best, the EHC program uses varying types of evidence from which to generate CER evidence. For topics that have insufficient current evidence, CER is conducted based on randomized controlled trials or observational studies. For topics on which existing evidence is sufficient, systematic reviews of the literature are generated. These reviews are free and available on the EHC program site at www.effectivehealthcare.ahrq.gov/. Evaluating CER Applicability, Quality, and Implications To help pharmacists and other healthcare providers apply CER in their practices, the speakers highlighted several important considerations when reviewing CER studies. The first is to consider the applicability of the CER data, which the speakers emphasized depends on context. One useful tool for assessing context is to use a framework called PICOS (population, intervention, comparison, outcome, timing of outcome assessment, and setting/or study design). Key questions to ask about applicability include: (1) Does the study sample resemble the population of interest? (2) Are intervention and comparators reasonable? (3) Are outcomes relevant to health decision needs? (4) Are results clinically significant, and effect sizes sufficient? (5) Does the clinical setting resemble yours? The presenters also spoke on the need to assess the quality of CER data by looking at the internal validity of a study based on whether the study design and conduct of the study protects against bias and inferential error. Good quality studies, they said, will address bias, include appropriate outcomes measurement and statistical and analytical methods, will have low dropout rates, and include adequate reporting. Key questions to ask about quality include: (1) What was the purpose of the study? (2) How were the groups selected? (3) How were the subjects allocated to treatment? (4) How were outcomes measured? (5) Is the statistical analysis appropriate? (6) What happened to the subjects?. In conclusion, the speakers highlighted key questions to understand the practice implications of a CER study: (1) Is this research area rapidly changing? (2) Will results impact clinical guidelines or policy? (3) Is future work in this area likely? (4) Could subgroup analysis clarify findings?