Abstract
Purpose. The purpose of this study was to demonstrate feasibility of use of outcomes and economic variables to help guide health policy for expensive breakthrough therapies that only benefit a small percentage of patients who are treated. In the United States, use of these therapies is fragmented and significant health care dollars are spent on patients who ultimately do not benefit. Presently, inputs to health policy seldom address balancing cost with patient benefit. If treatment policies were based on clinical outcomes and economic analysis, it might be possible to improve processes for health policy formation. Theoretical framework. This study will examine use of clinical information, economic analysis and stakeholder relevance as it applies to health care policy formation. Several factors have been identified which influence health policy including: (1) physician desire to give quality care; (2) economic incentives that reward maximization or minimization of care; (3) special interest groups; (4) confusion as to roles of federal and state governments; (5) administrative interpretation of legislation; and (6) Incrementalism. Methodology. Initially, outcomes data from a clinical trial involving chronic HCV patients were analyzed by univariate analysis. To account for interaction among variables, statistically significant variables found by univariate analysis were subjected to multivariate analysis. Significant variables by multivariate analysis were used to determine subsets of patients who were likely to benefit from treatment. Finally, economic analysis was completed to determine cost effectiveness of patient selection. Findings. Several baseline characteristics were shown to be significant predictors of outcomes. Using these variables to select sub-sets of patients allowed for an improvement in response ranging from 14.48 to 18.76 percent in the unselected population to 40.10 to 43.47 percent in the selected population. Patient selection resulted in a net present value savings over a twenty-year period of approximately $5 million. Conclusions and recommendations. This study demonstrates feasibility of use of outcomes and economic variables as inputs to health policy. Since this study examined only one disease, the findings should be applied to other clinical settings with caution. It is recommended that the methodology developed in this study be applied to addition diseases to demonstrate utility further.