How a unique provision in the American Recovery and Reinvestment Act set a foundation for the Patient-Centered Outcomes Research Institute.

Abstract

In 2003, Tunis, Stryer and Clancy promoted the need for pragmatic clinical studies targeted to answer important clinical questions so that healthcare decision-makers could make informed choices among treatment options [1]. The call for greater emphasis on research that examines real-world clinical questions resulted in large part from frustration that traditional clinical research often excluded patients who have multiple conditions, are either young or old, live in hard-to-reach areas or are gender or racially diverse. The concept of ‘stakeholder-driven’ pragmatic research began to take hold as a rational approach to expand a research enterprise that sometimes seemed at odds with the needs of patients, their families, clinicians and payers. The Congressional Budget Office in 2007 issued a report that discussed the benefits of research comparing medical treatments, or comparative effectiveness research (CER) [2]. This report, along with the 2009 American Recovery and Reinvestment Act (ARRA), provided an extraordinary platform for conducting and building infrastructure for research that was explicitly designed to help healthcare decision-makers. ARRA provided an opportunity, never before realized, to develop research and a research platform that was stakeholder driven, pragmatically focused and targeted toward practical questions. ARRA dedicated an unprecedented US$1.1 billion to this effort, collectively involving the Agency for Healthcare Research and Quality (AHRQ), the NIH and the Office of the Secretary, Department of Health and Human Services. Many aspects of the legislation served as important foundations for the efforts of the future Patient-Centered Outcomes Research Institute (PCORI). One particular investment made by ARRA was to support the convening of an IOM committee to prioritize topics for CER. With the input of thousands of patients and other stakeholders, this committee issued a report, identifying and prioritizing 100 key CER topics, that continues to be a touchstone for identifying major topics for c omparative research. The legislation also created the Federal Coordinating Council for Comparative Effectiveness Research (FCCCER), which defined CER and developed a framework to guide the federal ARRA investment. One of FCCCER’s most notable outcomes was a broadening of CER to include prevention, delivery systems and behavioral change. This broadening was also apparent in the IOM 100 priority topics. CER is not, however, an entirely new concept. Well before ARRA, both NIH and AHRQ could point to important examples of comparative effectiveness studies. AHRQ in particular had begun implementing its mandate to fund CER by developing a stakeholder-driven, transparent research program. These efforts helped spark a movement for greater engagement of patients, caregivers, clinicians and other healthcare stakeholders in research. They not only formed a baseline for PCORI, but also an expectation that future clinical research would engage SPECIAL FOCUS y The ARRA investment in CER