Langley P C
School of Economics, La Trobe University, Melbourne, Australia.
Clin Ther. 1994 May-Jun;16(3):538-52.
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.
为使制药公司能够合理规划药物研发过程，并在药物审批过程中满足处方委员会的要求，必须在研发过程的早期阶段就关注治疗干预措施的经济分析。确定疾病组内的对照产品对治疗干预进行建模，是确定支持结果研究所需数据的关键步骤，而结果研究又能为比较治疗评估提供支持。本文旨在建立一个框架，以确定关键结果指标，探讨治疗干预模型的作用，同时强调成本效益框架的必要性。这里考虑的关键结果指标包括直接和间接成本、药物替代以及对处方预算影响的评估。还强调了成本效益和成本效用指标作为资源分配框架中福利结果指标的局限性。主要结论是，治疗干预模型的早期建立对于药物研发和评估过程以及确定支持经济评估所需的数据项至关重要。对治疗干预进行建模必须就干预点、治疗途径、途径概率和治疗终点达成一致。除非确定这些参数，否则制药公司向处方委员会提交的材料以及对药物市场潜力的评估在本质上仍将是临时的，在任何决策过程中的用途都将有限。
