Comparative Effectiveness Research in Cancer: What Has Been Funded and What Knowledge Gaps Remain?
Authors: R. E. Glasgow, V. P. Doria-Rose, M. J. Khoury, M. Elzarrad, M. L. Brown, K. C. Stange
Journal: JNCI Journal of the National Cancer Institute (2013)

A recent explosion of interest in comparative effectiveness research (CER) has been accompanied by diverse attempts to define CER and specify CER research methods. We explore how CER is relevant across the cancer control continuum, including prevention, screening, diagnosis, treatment, and survivorship. We review cancer CER research funded by the National Cancer Institute by analyzing project characteristics along the dimensions of cancer type, stage of the cancer continuum, position on the T0 to T4 translational continuum, and the size and representativeness of both the settings and populations studied. We also provide an assessment of cost and resources considerations in CER. One hundred three relevant projects on CER were funded by the National Cancer Institute’s Division of Cancer Control and Population Science between 2009 and 2011. Prevention studies were most frequent (38.8%), and survivorship grants were least frequent (13.5%). Many projects included economic (35.0%) or simulation modeling (10.7%) approaches as well as multilevel behavioral (53.4%) and/or organizational change (54.4%) interventions. Most studies used convenience sampling (54.3%) and studied two or less settings (50.0%). Cancer CER is active and diverse but could be enhanced by a greater focus on knowledge integration, context, relevance to stakeholders, transparency, and population impact.

Processes for generating and evaluating evidence in cancer and other areas of health care are often slow, costly, or too unrepresentative to provide useful evidence to decision makers (1). Given cancer burden, increasing concerns about cancer treatment costs, and projections about the anticipated number of cancer survivors, there is a clear need for cancer comparative effectiveness research (CER) (2). Recently, CER has been recommended as a practical approach to determining what works in health care (3). As defined by the Institute of Medicine, CER is “the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care” (3).

Concerns about the effectiveness of health care have promoted interest in CER, culminating in the recently established Patient Centered Outcomes Research Institute (PCORI). PCORI responds to concerns that patients, providers, and caregivers do not have the types of comparative information they need to make choices aligned with their desired health outcomes. Thus, the concepts of personalization and patient-centeredness are embedded in the PCORI (4–6). For example, one of the tenets of this research includes answers to these questions: “Given my personal characteristics, conditions, and preferences, what should I expect will happen to me?” and “What are my options, and what are the benefits and harms of those options?” (7). These same questions apply to personalized or precision medicine that seeks to integrate “-omic” information into health care (7). Personalizing health care according to patient preferences will require incorporating social and behavioral information from the outset, not just when a new application is ready for clinical use. There has been an outpouring of recent articles on CER, including special issues of JAMA (April 18, 2012) and the Journal of Clinical Oncology (October 15, 2012) that included both conceptual papers and a series of large-scale observational studies, three of which addressed cancer. Given the vast amount of emerging information, it remains to be seen how much evidence will have to come from comparative randomized clinical trials, observational studies, natural experiments, adaptive trials, pragmatic intervention studies, and evidence synthesis and modeling, all of which are tools of CER (7).

Regardless, three things are clear: 1) CER research should be relevant and broadly applicable; 2) it should include information on costs, resources required, and efficiency issues; and 3) it should address public health impact (8,9).