Ronquest N, Paret K, Mladsi DM, Lee LJ. Exploring the value of country-specific cost-effectiveness models in early stages of drug development. Poster presented at the ISPOR Europe 2023; November 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 supplement):S94. doi: 10.1016/j.jval.2023.09.493


OBJECTIVES: Despite increasing use of cost-effectiveness analyses by healthcare systems in countries that historically have not utilized them, the United Kingdom (UK) is often the first country for which an early cost-effectiveness model (eCEM) is developed to guide early health economic and outcomes research strategies across global regions. The aim of this study was to explore the value of country adaptations of eCEMs by assessing the influence of country-specific differences in key model inputs on estimated economically justifiable prices (EJPs), a proxy for assessing the opportunity for a new drug to provide value to patients and the healthcare system across a variety of potential development options using a hypothetical eCEM in oncology.

METHODS: We developed a United Kingdom–based eCEM evaluating a hypothetical product for advanced cancer and adapted it for the United States (US) using two approaches. First, a simple US adaptation was conducted by updating the willingness-to-pay threshold, discount rates, and costs using the hospital price purchasing power parity. Subsequently, each model input was varied to reflect the US-specific plausible ranges informed by the literature. The model inputs were then ranked by their influence on the change in estimated EJP between the two methods of country adaptation. Potential reasons for substantial differences between countries were summarized for scenarios with the highest impacts on EJPs.

RESULTS: US-specific inputs with the highest influence on EJPs were the price of current standard of care (up to 19%), subsequent treatment costs (17%), health state utility values (12%), and background treatment costs (8%). Costs of adverse events did not contribute to significant changes in EJPs (0.1%).

CONCLUSIONS: Our case study highlighted the value of developing additional country-specific eCEMs when there are substantial differences in treatment patterns, timing of generic drug availability, costs of background treatments, and availability of utility data across countries.

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