Infusion of statistical science in comparative effectiveness research

Purpose We present a general introduction to comparative effectiveness research (CER) from a statistician’s viewpoint and focus on how statisticians can contribute to the methodology of CER.

Conclusions The statistical science community needs to determine the priorities for methodological research in CER, in collaboration with our colleagues in the aligned medical fields. CER requires that we apply a new paradigm – a focus on the patient. The emphasis on the patient is driven by patients themselves, the federal government, and private payers, in addition to the fact that there is a rising chronic disease burden that is making patient populations more heterogeneous. The availability of new technology and data sources introduces not only complexity but also opportunity. Statistical scientists should rise to meet these new demands and develop optimal, statistically valid approaches to CER, just as they have in agriculture and clinical trials.