MAY 31 – JUNE 1, 2012

Comparative Effectiveness Research:
Controversies and Opportunities

Comparative-effectiveness research attempts to establish the relative health benefits of different drugs, medical devices, diagnostic and surgical procedures, diagnostic tests, and health care services in real world settings as a tool to improve health care outcomes and quality.  The focus of the 2012 HSPH-Merck Workshop is on statistical, analytical and design methods for comparative effectiveness research (CER) with an emphasis on evaluating the comparative effectiveness of therapeutic interventions.  CER must take into account variations in health care delivery, clinical practice patterns, socio-economic and demographic factors, and characteristics of human behavior that can modify effectiveness across large segments of the populations under investigation.  Of considerable interest is the way in which the design of clinical trials can be modified to provide such information, such as the use of multi-course clinical trials (dynamic treatment regimes), pragmatic clinical trials, genomic profile-based trials, and Bayesian clinical trials.  Use of observational databases, such as Medicare/Medicaid data, Surveillance, Epidemiology, End Results (SEER) data, and prospective clinical registries are also of interest with particular emphasis on identifying treatment heterogeneity.  During this workshop, faculty and participants will explore the challenges and opportunities associated with various CER methodologies.  Didactic and case study-based presentations will focus on controversial issues and topics within the context of indirect treatment comparisons, causal inference, personalized medicine, post-marketing drug surveillance, patient-reported outcomes, and cost-effectiveness.  Through interactive, small group discussions and feedback, the workshop will provide a forum to identify promising opportunities to develop innovative methods to achieve the national priorities for comparative effectiveness research as put forth by the Institute of Medicine of the National Academies.[1]

[1] Initial National Priorities for Comparative Effectiveness Research. Committee on Comparative Effectiveness Research Prioritization, Institute of Medicine of the National Academies, The National Academies Press, Washington DC. 2009 http://www.iom.edu/Reports/2009/ComparativeEffectivenessResearchPriorities.aspx