Prioritization in Comparative Effectiveness Research: The CANCERGEN Experience

• Published in: Medical care 2012-05, Vol.50 (5), p.388-393
• Main Authors: Thariani, Rahber, Wong, William, Carlson, Josh J., Garrison, Louis, Ramsey, Scott, Deverka, Patricia A., Esmail, Laura, Rangarao, Sneha, Hoban, Carolyn J., Baker, Laurence H., Veenstra, David L.
• Format: Article
• Language: English
• Subjects: Breast cancer; Brief Report; Clinical trials; Comparative Effectiveness Research - economics; Comparative Effectiveness Research - organization & administration; Genetic Testing; Genomics; Gray literature; Health Services Research; Humans; Lung neoplasms; Marketing of Health Services; Medical research; Neoplasms - genetics; Oncology; Practice Guidelines as Topic; Prioritization; Standards of care; Voting
• ISSN: 0025-7079; 1537-1948
• DOI: 10.1097/MLR.0b013e3182422a3b

Background: Systematic approaches to stakeholder-informed research prioritization are a central focus of comparative effectiveness research. Genomic testing in cancer is an ideal area to refine such approaches given rapid innovation and potentially significant impacts on patient outcomes. Objective: To develop and pilot test a stakeholder-informed approach to prioritizing genomic tests for future study in collaboration with the cancer clinical trials consortium SWOG. Methods: We conducted a landscape analysis to identify genomic tests in oncology using a systematic search of published and unpublished studies, and expert consultation. Clinically valid tests suitable for evaluation in a comparative study were presented to an external stakeholder group. Domains to guide the prioritization process were identified with stakeholder input, and stakeholders ranked tests using multiple voting rounds. Results: A stakeholder group was created including representatives from patient-advocacy groups, payers, test developers, regulators, policy makers, and community-based oncologists. We identified 9 domains for research prioritization with stakeholder feedback: population impact; current standard of care, strength of association; potential clinical benefits, potential clinical harms, economic impacts, evidence of need, trial feasibility, and market factors. The landscape analysis identified 635 studies; of 9 tests deemed to have sufficient clinical validity, 6 were presented to stakeholders. Two tests in lung cancer (ERCC1 and EGFR) and 1 test in breast cancer (CEA/CA15-3/CA27.29) were identified as top research priorities. Conclusions: Use of a diverse stakeholder group to inform research prioritization is feasible in a pragmatic and timely manner. Additional research is needed to optimize search strategies, stakeholder group composition, and integration with existing prioritization mechanisms.