The Rise of Comparative Effectiveness Research

Comparative effectiveness research (CER) refers to research aimed at determining which of multiple health care services – including diagnostic tests, treatments, public health programs, or other health care delivery strategies – is best. In 2009 and 2010, the American Recovery and Reinvestment Act and the Patient Protection and Affordable Care Act (ACA) expanded governmental funding for CER in the USA by creating the Patient-Centered Outcomes Research Institute (PCORI). The PCORI plans to prioritize research aimed at helping patients, clinicians, and other stakeholders make more informed decisions in “real-world” situations, a goal that will likely require an emphasis on less traditional research methods that are more representative of typical clinical practice such as pragmatic trials and observational research. The new funds should provide a much-needed boost to CER, however several challenges loom. In addition to promoting research that is both valid and relevant in “real-world” situations, the PCORI will need to find more effective ways to ensure that CER findings are disseminated and implemented in clinical practice. The latter task may be particularly challenging since the ACA places limits on the use of CER findings for decisions related to “payment, coverage, or treatment.” In addition, the ACA limits the PCORI from supporting cost-effectiveness research, which stakeholders might use to allocate health care resources most efficiently.