ABSTRACT
ABSTRACT This chapter discusses a range of issues that might help frame how evidence of comparative effectiveness (CE) is judged in the regulatory setting, taking into account the experiences gained from evaluating the efficacy of medical products, especially pharmaceuticals. We propose a
hierarchy of standards of evidence that are aligned with the questions and issues of importance to each constituency that will use the evidence. The Food and Drug Administration regulatory experience for pharmaceuticals is based upon the evaluation of substantial evidence of the efficacy and safety of pharmaceutical products. The regulatory standard is derived from randomized-controlled clinical trials and can inform about the types of studies, including randomized studies and observational studies that could be conducted to support CE conclusions. There are several government bodies that have sponsored work on guidances and methodologies relevant to comparative effectiveness research and some of this material will be briefly covered.
