Outcomes research and modeling therapeutic interventions for economic evaluations.

For pharmaceutical companies to plan properly the drug development process and meet the needs of formulary committees in the drug approval process, attention must be given to the economic analysis of therapeutic interventions at an early stage in the development process. Modeling therapeutic intervention, where comparator products within disease groups are identified, is a key step in identifying the data required to support outcomes research, which in turn, supports comparative therapy evaluations. The purpose of this paper is to develop a framework for identifying key outcome measures, exploring the role of therapy intervention models while emphasizing the need for a cost-benefit framework. The key outcome measures considered here include both direct and indirect costs, drug substitution, and the assessment of formulary budget impacts. The limitations of cost-effectiveness and cost-utility measures as indicators of welfare outcomes in a resource allocation framework are also emphasized. The principal conclusion is that the early development of a therapy intervention model is essential to the process of drug development and assessment and for the identification of data items needed to support economic evaluation. Modeling therapeutic interventions must involve agreement on intervention points, treatment pathways, pathway probabilities, and end points of therapy. Unless these parameters are determined, submissions by drug companies to formulary committees and assessments of the market potential for drugs will remain ad hoc in nature and of limited use in any decision process.