Comparative Effectiveness Research in Cancer Genomics and Precision Medicine: Current Landscape and Future Prospects

Comparative Effectiveness Research in Cancer Genomics and Precision Medicine: Current Landscape and Future Prospects

1. Naoko I. Simonds,
2. Muin J. Khoury,
3. Sheri D. Schully,
4. Katrina Armstrong,
5. Wendy F. Cohn,
6. David A. Fenstermacher,
7. Geoffrey S. Ginsburg,
8. Katrina A.B. Goddard,
9. William A. Knaus,
10. Gary H. Lyman,
11. Scott D. Ramsey,
12. Jianfeng Xu and
13. Andrew N. Freedman

+ Author Affiliations

1. Affiliations of authors: Division of Cancer Control and Population Science (DCCPS), National Cancer Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, MD (NIS, MJK, SDS, ANF); Office of Public Health Genomics, Centers for Disease Control and Prevention, Atlanta, GA (MJK); Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA (KA); Division of Biomedical Informatics, Public Health Sciences, University of Virginia School of Medicine, Charlottesville, VA (WK); Biomedical Informatics Department, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL (DF); Institute for Genome Sciences and Policy, Duke University Medical Center, Durham, NC (GSG); Center for Health Research, Kaiser Permanente Northwest, Portland, OR (KABG); Applied Genomics Research Institute, Center for Clinical and Research Informatics, North Shore University Health System, Evanston, IL (WK); Department of Medicine, Division of Medical Oncology, Duke University School of Medicine and the Duke Cancer Institute, Durham, NC (GHL); Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA (SDR); Center for Cancer Genomics, Wake Forest School of Medicine, Winston-Salem, NC (JX).

1. Correspondence to: Naoko I. Simonds, Clinical and Translational Epidemiology Branch, DCCPS, 9609 Medical Center Dr, Rm 4E228, Bethesda, MD 20892 (e-mail: naoko.simonds@nih.gov).

• Received January 8, 2013.
• Revision received March 20, 2013.
• Accepted March 21, 2013.

Abstract

A major promise of genomic research is information that can transform health care and public health through earlier diagnosis, more effective prevention and treatment of disease, and avoidance of drug side effects. Although there is interest in the early adoption of emerging genomic applications in cancer prevention and treatment, there are substantial evidence gaps that are further compounded by the difficulties of designing adequately powered studies to generate this evidence, thus limiting the uptake of these tools into clinical practice. Comparative effectiveness research (CER) is intended to generate evidence on the “real-world” effectiveness compared with existing standards of care so informed decisions can be made to improve health care. Capitalizing on funding opportunities from the American Recovery and Reinvestment Act of 2009, the National Cancer Institute funded seven research teams to conduct CER in genomic and precision medicine and sponsored a workshop on CER on May 30, 2012, in Bethesda, Maryland. This report highlights research findings from those research teams, challenges to conducting CER, the barriers to implementation in clinical practice, and research priorities and opportunities in CER in genomic and precision medicine. Workshop participants strongly emphasized the need for conducting CER for promising molecularly targeted therapies, developing and supporting an integrated clinical network for open-access resources, supporting bioinformatics and computer science research, providing training and education programs in CER, and conducting research in economic and decision modeling.

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