Modeling Cost-Effectiveness of a Biomarker-Based Approach to Neoadjuvant Chemotherapy for Muscle-Invasive Bladder Cancer.

Lotan Y1, Woldu SL1, Sanli O1,2, Black P3, Milowsky MI4.

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Abstract

OBJECTIVES:

To model the cost-effectiveness (CE) of a biomarker-based approach to select patients for neoadjuvant chemotherapy (NAC) prior to radical cystectomy (RC) in muscle-invasive bladder cancer (MIBC).

MATERIALS AND METHODS:

We obtained most recently clinical studies on locally-advanced bladder cancer treated by RC, including stage distributions, overall survival (OS) estimates, associated costs, and utilization/response to NAC. Additionally, we estimated the putative efficacy of 3 biomarkers to select patients for NAC: DNA repair gene panel [ATM, RB1, and FANCC], ERCC2, and RNA subtypes. A decision analysis model was developed to evaluate the CE of biomarker-based approaches to select patients with MIBC for NAC. Comparison of CE included RC alone, unselected NAC plus RC, and NAC based on the 3 aforementioned biomarkers.

RESULTS:

The DNA repair gene panel-based approach to NAC was the most CE strategy (mean OS of 3.14 years, $31,482/life year). Under this approach, 38% would undergo NAC, approximately twice the number of patients who are currently receiving NAC for MIBC. Such an approach would improve mean OS by 5.2 months, 1.6 months, and 4.4 months compared to RC alone, a hypothetical scenario where all patients received NAC, and compared to current estimates of NAC utilization, respectively.

CONCLUSIONS:

A biomarker-based strategy to identify MIBC patients who should undergo NAC was more cost-effective than unselected use of NAC or RC alone. As further data becomes available, such a model may serve as a basis for incorporating biomarkers into clinical decision making. This article is protected by copyright. All rights reserved.