Building the Evidence Base for Decision-making in Cancer Genomic Medicine Using Comparative Effectiveness Research ; Genet Med

Background ; The clinical utility is uncertain for many cancer genomic applications. Comparative effectiveness research (CER) can provide evidence to clarify this uncertainty. ; Objectives ; To identify approaches to help stakeholders make evidence-based decisions, and to describe potential challenges and opportunities using CER to produce evidence-based guidance. ; Methods ; We identified general CER approaches for genomic applications through literature review, the authors’ experiences, and lessons learned from a recent, seven-site CER initiative in cancer genomic medicine. Case studies illustrate the use of CER approaches. ; Results ; Evidence generation and synthesis approaches include comparative observational and randomized trials, patient reported outcomes, decision modeling, and economic analysis. We identified significant challenges to conducting CER in cancer genomics: the rapid pace of innovation, the lack of regulation, the limited evidence for clinical utility, and the beliefs that genomic tests could have personal utility without having clinical utility. Opportunities to capitalize on CER methods in cancer genomics include improvements in the conduct of evidence synthesis, stakeholder engagement, increasing the number of comparative studies, and developing approaches to inform clinical guidelines and research prioritization. ; Conclusions ; CER offers a variety of methodological approaches to address stakeholders’ needs. Innovative approaches are needed to ensure an effective translation of genomic discoveries. ; 5U18-GD000005-02/GD/OGDP CDC HHS/United States ; P50HG003374/HG/NHGRI NIH HHS/United States ; RC2 CA148570-01/CA/NCI NIH HHS/United States ; RC2CA148041-01/CA/NCI NIH HHS/United States ; RC2CA148570/CA/NCI NIH HHS/United States ; U01GM092676/GM/NIGMS NIH HHS/United States ; U18GD000005/GD/OGDP CDC HHS/United States ; UC2 CA148471/CA/NCI NIH HHS/United States ; UC2 CA148471/CA/NCI NIH HHS/United States ; UC2CA150911/CA/NCI NIH HHS/United States ; 2013-04-22T00:00:00Z ; 22516979 ...