Advancing Effectiveness Science

Abstract

Effectiveness science is essential to improving quality and cost of U.S. health care. It incorporates testing health interventions in real world settings, advancing use of research findings by diverse populations, and training and career development for next generation of investigators in field. Effectiveness science uses comparisons, subgroups, and context to determine what health treatments work for whom and in what setting. This includes comparisons of treatments as well as models of delivery (Simpson et al., 2010). OVERVIEW OF EFFECTIVENESS SCIENCE Effectiveness science has received increasing attention over past 3 years. This is evidenced from large comparative effectiveness studies awarded under American Recovery and Reinvestment Act of 2009 (about $1.1 billion), Institute of Medicine (IOM; 2009) report submitted to Congress that outlines 100 priority research topics and 10 recommendations to support a robust effectiveness research program, a report submitted to Congress by Federal Coordinating Council to guide effectiveness research efforts, and Patient Protection and Affordable Care Act of 2010, section 6301, which mandates creation of a Patient-Centered Outcomes Research Institute (PCORI; Sox, 2010). The IOM (2009) defines comparative effectiveness research as the generation and synthesis of evidence that compares benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical condition or to improve delivery of care (p. 41). The purpose of comparative effectiveness research is to help consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health at individual and population level (IOM, 2009). Findings from effectiveness research are designed to strengthen our health system by ensuring that that is delivered is based on best possible evidence and informed decisions. Defining characteristics of comparative effectiveness research include following (IOM, 2009): * It is conducted in settings similar to those in which intervention will be used in practice. * It employs methods and data sources appropriate for decision of interest. * It includes measures of outcomes that are important to patients, both benefits and harms. * It directly informs a specific clinical decision from patient perspective or a policy decision from population perspective. * It compares at least two alternative interventions. * It describes results at population and subgroup level. EFFECTIVENESS SCIENCE METHODS Results from full range of high-quality effectiveness studies as well as research that meet highest possible standards in its conduct and reporting are needed to make decisions about patient care. Methods commonly used in comparative effectiveness research include experimental and observational studies, research synthesis, and meta-analysis (IOM, 2009). There is ongoing controversy about using observational studies to fill evidence gaps in comparative effectiveness research. Although a thorough discussion of this controversy is beyond scope of this editorial, Dreyer et al. (2010) as well as Fleurence, Naci, and Jansen (2010) discuss advantages and shortcomings of observational studies as well as other research designs for effectiveness studies. Of particular note, large observational studies often provide insight and empirical understanding of how different interventions work in real world settings of practice where patient populations are likely to be more heterogeneous than those in original randomized clinical trials. The Methods Committee of newly formed U.S. Government Accountability Office's (GAO) PCORI has charge to develop and improve science and methods of comparative clinical effectiveness research (GAO, 2011). PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE The PCORI is a nonprofit organization that assists patients, clinicians, purchasers, and policy makers in making informed health decisions by conducting research that provides quality and relevant evidence on how diseases, disorders, and other health conditions can be effectively and appropriately prevented, diagnosed, treated, monitored, or managed. …