Abstract

ObjectiveTo determine the cost effectiveness of molecular monitoring in patients with chronic myeloid leukemia in the chronic phase (CML-CP) compared to no molecular monitoring from a Chinese payer perspective.MethodsAnalyses were conducted using a semi-Markov model with a 50-year time horizon. Population data from multicenter registry-based studies of Chinese patients with CML-CP informed the model. Transition probabilities were based on time-to-event data from the literature. Utility values were obtained from published studies and were assumed to be the same for patients with and without molecular monitoring. Costs were based on values commonly used in the Chinese healthcare system, including drug acquisition, drug administration, follow-up, treatment for disease progression, molecular monitoring, and terminal care costs, and were in the local currency (2020 Chinese Yuan RMB [¥]). Outcomes were total life-years (LYs) and quality-adjusted life years (QALYs), lifetime costs, and incremental cost-effectiveness ratio.ResultsMolecular monitoring was dominant to no molecular monitoring, with increased LYs (1.52) and QALYs (1.90) and costs savings (¥93,840) over a lifetime compared to no monitoring in discounted analyses. The opportunity of patients that receive molecular monitoring to discontinue treatment during treatment-free remission, an opportunity not afforded to those without molecular monitoring, was the principle driver of this result. Results were similar across multiple clinical scenarios. Particularly, molecular monitoring remained dominant even if the proportion of patients achieving deep molecular response (DMR) was reduced by 10%-30%, or the proportion of patients maintaining DMR for 1 year was reduced by 10%-30% or increased by 10%. Cost savings in these scenarios ranged from ¥62,230 to ¥103,964.ConclusionsOverall, this analysis demonstrates that adherence to guideline recommendations of regular molecular monitoring of patients with CML-CP treated with TKIs provides significant clinical benefit that leads to substantial cost savings compared to no molecular monitoring from the perspective of a Chinese payer. In a time where healthcare systems have limited resources to allocate to optimal patient care, investment in molecular monitoring is an ideal choice for improving patient benefits at a reduced cost.

Highlights

  • Chronic myeloid leukemia (CML), a myeloproliferative blood cancer, has an incidence of 0.39–0.55 cases per 100,000 adults in China [1]

  • Molecular monitoring was dominant to no molecular monitoring, with increased life year (LY) (1.52) and quality-adjusted life year (QALY) (1.90) and costs savings (¥93,840) over a lifetime compared to no monitoring in discounted analyses

  • By 10%-30%, or the proportion of patients maintaining deep molecular response (DMR) for 1 year was reduced by 10%30% or increased by 10%. Cost savings in these scenarios ranged from ¥62,230 to ¥103,964. This analysis demonstrates that adherence to guideline recommendations of regular molecular monitoring of patients with chronic myeloid leukemia in the chronic phase (CML-CP) treated with tyrosine kinase inhibitors (TKIs) provides significant clinical benefit that leads to substantial cost savings compared to no molecular monitoring from the perspective of a Chinese payer

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Summary

Introduction

Chronic myeloid leukemia (CML), a myeloproliferative blood cancer, has an incidence of 0.39–0.55 cases per 100,000 adults in China [1]. The standard of care for CML-CP patients in China is treatment with tyrosine kinase inhibitors (TKIs) such as imatinib, nilotinib, dasatinib and flumatinib. In further support of this notion, accumulating evidence demonstrates that patients with CML-CP that achieve a complete cytogenetic response have a similar overall survival as that of the general population [11]. It is well-established that patients on TKI therapy can go on to achieve a deep molecular response (DMR), typically defined as BCR-ABL1 transcript levels of 0.01% (MR4) or

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