Comparative effectiveness research: improving patient care or drug rationing?

Faced with runaway healthcare costs, evidence of haphazard and wasteful healthcare practices, and little evidence on the relative merits of competing healthcare interventions, the US has become heavily invested in comparative effectiveness research (CER). In addition to the one-time $US1.1 billion allocated to CER in the American Recovery and Reinvestment Act of 2009, the subsequent Patient Protection and Affordable Care Act (PPACA) created the Patient-Centered Outcomes Research Institute (PCORI), an ongoing public-private partnership to support and coordinate CER. By 2014, PCORI funding is estimated at $US500million annually. CER has been defined by the Institute of Medicine as ‘‘the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.’’ Unlike traditional efficacy trials, which focus on demonstrating the efficacy of medical interventions versus placebo in highly selected populations, often to gain regulatory approval or to enhance market share, key aspects of CER are its focus on comparisons of benefits and harms between interventions in populations such as those encountered in everyday practice. In other words, the question shifts from ‘‘Can this intervention work?’’ to ‘‘Does this intervention work better than the next best alternative?’’ Another sometimes overlooked aspect of CER is the evaluation of differential effects of interventions in patient subgroups defined by age, sex, race, comorbidities, genomic differences or other factors. CER encompasses a broad range of research, including head-to-head randomized trials, effectiveness trials and observational research, as well as systematic reviews that synthesize the primary research. As with any research, the usefulness and reliability of a CER study will depend on the study design used, its methodological rigour and its applicability to clinical practice. For example, with pharmacoepidemiological studies conducted on large administrative databases, it would be critical to determine how accurately exposures, outcomes and confounders were ascertained. Proponents of CER view it as a way to provide the valuable comparative information that is needed, but often lacking, to help guide many clinical decisions. One example of a comparative effectiveness trial with important clinical implicationswasALLHAT (Antihypertensive andLipidLowering Treatment to Prevent Heart Attack Trial), which showed that older (and often less expensive) antihypertensives were as effective as newer drugs at reducing important cardiovascular outcomes. Nonetheless, critics have raised concerns about the potential effects of CER on public policy and patient care, charging that it could lead to reduced physician autonomy and rationing. Rationing is a highly loaded term with predominantly negative connotations, but its real meaning is often not well understood. In general EDITORIAL Drugs 2011; 71 (4): 383-386 0012-6667/11/0004-0383/$55.55/0