PHP142 Landmark CER Study Methods Review

Abstract

Comparative effectiveness research (CER), constitutes a ‘real-world' comparison of the risk-benefit profile of a new product with the existing standard of care. In the US, the federal government is supporting the expansion of CER through funding made available in the American Recovery and Reinvestment Act of 2009 (ARRA) and by establishing the Patient-Centered Outcomes Research Institute through the Patient Protection and Affordable Care Act of 2010. Similarly in Europe there is push for relative effectiveness studies. Industry and governments have deployed a variety of experimental research design methods to appraise real-world performance of products vis-à-vis competitors. An evaluation of five recent studies was analyzed to assess CER implications. We performed a structured review and assessment of five different therapeutic classes (antihyperlipidemics, antipsychotics, antiplatelets, anti-VEGF, and insulin analogues) investigated in CER related studies (AIM-HIGH, CATIE, GeCCO, CATT, and AHRQ CER Premixed Insulin Analogues). CER metrics included study population including comparative agent(s), relevance of conclusions and interpretations outside the study population. Study designs varied including head to head, study agent versus placebo, and systematic literature reviews as well as various types of metrics (safety, efficacy, and effectiveness). One study was stopped early due to lack of benefit, three studies determine equivalence of effectiveness (cost, clinical). Another study is still underway (GeCCO) with results expected towards the end of 2011. The number of CER focused studies is increasing with wide variability in study designs, comparators, populations, and endpoints. With the surge of new agencies dedicated to this evolving field of research (e.g., PCORI in the US), it will be important to evaluate the various types of CER studies and resulting information from a multi-stakeholder perspective.