Model-Based Cost-Effectiveness Analyses for the Treatment of Chronic Lymphocytic Leukaemia: A Review of Methods to Model Disease Outcomes and Estimate Utility

Abstract

Assessing the economic value of treatments for chronic lymphocytic leukaemia (CLL) is necessary to support healthcare decision makers; however, it poses a number of challenges. This paper reviews economic models of CLL treatment to learn the lessons from this experience and support ongoing model efforts. A search of databases and submissions to key health technology assessment agencies identified nine models. The modelling approaches adopted across these studies were fairly similar, with most models adopting a cohort Markov structure, though one example of a discrete event simulation was identified. While the cohort Markov approach has been acceptable to the National Institute for Health and Care Excellence, the review identifies a number of key uncertainties with these models, including the extrapolation of survival outcomes beyond the period observed by the trial, the effectiveness of second-line therapies, and estimates of health state utility. Further work is required to overcome these uncertainties, including comprehensive sensitivity analysis, systematic review of the evidence on the natural progression of CLL, and the collection of longer-term trial and registry data.

Análise de custo-efetividade de obinutuzumabe associado à quimioterapia para o tratamento de pacientes adultos com leucemia linfoide crônica não elegíveis ao tratamento baseado em fludarabina na perspectiva do Sistema Único de Saúde (SUS)

“…Em adição a isso, a metodologia simples de modelagem com três estados de saúde pode representar uma forma limitante de refletir uma doença crônica, uma vez que, em um período de lifetime, o paciente pode apresentar outros estados de saúde, além de sofrer influências de linhas posteriores de tratamento. A ausência de dados que apresentem o acompanhamento de pacientes em todas as fases da doença também é um fator que dificulta as estimativas de desfechos clínicos para avaliações econômicas (Marsh, et al, 2014).…”

“…Mesmo na ausência de informações a longo prazo, a maioria dos estudos com modelos econômicos para LLC adota o mesmo formato de modelagem do presente estudo, e foi e é aceita por agências de tecnologia de saúde internacionais (Marsh et al, 2014). A utilização de análise de custo-efetividade auxilia na comparação tanto de desfechos clínicos quanto de desfechos econômicos de duas intervenções, além de serem ferramentas importantes e úteis durante a tomada de decisão de fontes pagadoras, principalmente em um contexto de recursos econômicos limitados.…”

Cost-Effectiveness Model for Chemoimmunotherapy Options in Patients with Previously Untreated Chronic Lymphocytic Leukemia Unsuitable for Full-Dose Fludarabine-Based Therapy

“…Although we used a cohort Markov model for analyzing costeffectiveness-as endorsed by NICE in the United Kingdomsimilar previous analyses in patients with CLL have shown that there are some uncertainties associated with this approach [31]. In this respect, patients in the CLL11 trial that we included in our analysis continue to be followed up.…”

Economic evaluation of obinutuzumab in combination with chlorambucil in first-line treatment of patients with chronic lymphocytic leukemia in Spain

“…The model utilized a Markov process, which is the standard method used in cost-effectiveness studies to represent the natural history in various diseases 33 , 34 and the most widely used model for analyzing cost-effectiveness in CLL. 35 …”