Exploration of an alternative reconstructed individual patient data-based approach for budget impact analysis of anticancer drugs

Abstract

Abstract Background The duration of treatment (DOT) of the initial intervention and subsequent treatment is the key to determining the accuracy of anticancer-drug budget impact analysis (BIA) calculations. However, existing studies only use simple assumptions as a proxy for DOT, resulting in a high degree of bias. Objectives To enhance the accuracy and reliability of anticancer-drug BIA and solve the problem regarding DOT, we propose an alternative individual patient data (IPD)-based approach that reconstructs IPD from the published Kaplan Meier survival curves to estimate DOT. Methods We developed a four-step methodological framework for this new approach, taking the use of pembrolizumab in treating microsatellite-instability–high (MSI-H) advanced colorectal cancer as an example: (1) reconstructing the IPD; (2) calculating the total DOT of the initial intervention and subsequent treatment for each patient; (3) assigning a randomized time and DOT; and (4) multiple replacement sampling and calculation of the mean value. Results Using this approach, the average DOT for the initial intervention and subsequent treatment in each year of the BIA time horizon can be calculated and used to calculate the resources consumed and costs in each year. In our example, the average DOT for the initial intervention with pembrolizumab from the first to the fourth year was 4.90, 6.60, 5.24, and 5.06 months, respectively, while the average DOT for subsequent treatment was 0.75, 2.84, 2.99, and 2.50 months, respectively. Conclusions The reconstructed IPD-based approach can improve the accuracy and reliability of anticancer-drug BIA compared with conventional methods, and can be widely used, especially for anticancer drugs with excellent efficacy.