Development and Use of Disease-Specific (Reference) Models for Economic Evaluations of Health Technologies: An Overview of Key Issues and Potential Solutions

Decision-analytic models are increasingly used to inform decisions about whether or not to publicly fund new health technologies such as pharmaceuticals. They are large-scale models with different boundaries, providing a consistent framework for the economic evaluation of a wide range of health technologies for a specific condition. The proposed disease specific (reference) model in this article is not a system-level model, such as a whole disease model, simulating disease and treatment pathways. Rather, a disease-specific model focuses on the disease progression, capturing all key clinical states/events, and the transition between them, and patient attributes that influence disease and/or response to treatment. During a panel discussion at the recent Health Technology Assessment International (HTAi) conference in Washington, DC, USA, the value of such disease-specific reference models was discussed from different HTA stakeholder viewpoints: academia, industry, and HTA agencies. This commentary aims to briefly outline and share key points from our panel at HTAi, focusing on practical issues around the development and overcoming barriers prohibiting the use of standardized disease-specific models.