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The second author's name should be "Maria De Francesco" rather than "Maria de Fransesco".
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BACKGROUND: Cladribine tablets have recently become available in The Netherlands for patients with relapsing-remitting multiple sclerosis (RRMS) as a disease-modifying agent that reduces the frequency and severity of relapses and delays disability progression. OBJECTIVE: The aim of this study was to evaluate the cost effectiveness of cladribine tablets, compared with alternative options, in the treatment of RRMS patients with high disease activity (HDA) and patients with rapidly evolving severe (RES) MS in The Netherlands. METHODS: A Markov model was developed simulating the costs and effects of RRMS treatment. For HDA, alemtuzumab and fingolimod were used as comparators; natalizumab was used for the RES subpopulation. The analysis included a societal perspective and a value-of-information (VOI) analysis. RESULTS: For the HDA subpopulation, treatment with cladribine tablets was the cost-effective (dominant) strategy compared with alemtuzumab and fingolimod, with 50.9% and 98.2%, respectively, probability of being cost effective at a threshold of 50,000/QALY gained and a net monetary benefit (NMB) of 10,866 and 151,115, respectively. For the RES subpopulation, treatment with cladribine tablets dominated treatment with natalizumab, with 94.1% probability of being cost effective at a threshold of 50,000/QALY gained and an NMB of 122,986. Note that these outcomes are driven by the lower costs of cladribine tablets. Efficacy differences were small, very uncertain, and likely not clinically meaningful. The probabilistic sensitivity analyses showed significant overlap in the credible intervals for total lifetime QALY outcomes and costs of cladribine tablets and all relevant comparators. The population-level VOI amounted to 19,295,441. CONCLUSIONS: The base-case analysis shows that treatment of RRMS with cladribine tablets is cost effective versus alemtuzumab and fingolimod in HDA patients, and cost effective versus natalizumab in RES patients, at a threshold of 50,000. Driven by the lower costs, cladribine tablets were cost effective (dominant) in all base-case analyses. However, given that outcomes are based on indirect comparisons and post hoc subgroup analysis, as well as the uncertainty surrounding the outcomes, the results presented in this paper should be interpreted with caution.
Assuntos
Cladribina/administração & dosagem , Cladribina/economia , Imunossupressores/administração & dosagem , Imunossupressores/economia , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Adulto , Análise Custo-Benefício , Feminino , Humanos , Masculino , Cadeias de Markov , Países Baixos , Anos de Vida Ajustados por Qualidade de VidaRESUMO
OBJECTIVES: To assess the level of comprehensiveness of health technology assessment (HTA) practices around the globe and to formulate recommendations for enhancing legitimacy and fairness of related decision-making processes. METHODS: To identify best practices, we developed an evaluation framework consisting of 13 criteria on the basis of the INTEGRATE-HTA model (integrative perspective on assessing health technologies) and the Accountability for Reasonableness framework (deliberative appraisal process). We examined different HTA systems in middle-income countries (Argentina, Brazil, and Thailand) and high-income countries (Australia, Canada, England, France, Germany, Scotland, and South Korea). For this purpose, desk research and structured interviews with relevant key stakeholders (N = 32) in the selected countries were conducted. RESULTS: HTA systems in Canada, England, and Scotland appear relatively well aligned with our framework, followed by Australia, Germany, and France. Argentina and South Korea are at an early stage, whereas Brazil and Thailand are at an intermediate level. Both desk research and interviews revealed that scoping is often not part of the HTA process. In contrast, providing evidence reports for assessment is well established. Indirect and unintended outcomes are increasingly considered, but there is room for improvement. Monitoring and evaluation of the HTA process is not well established across countries. Finally, adopting transparent and robust processes, including stakeholder consultation, takes time. CONCLUSIONS: This study presents a framework for assessing the level of comprehensiveness of the HTA process in a country. On the basis of applying the framework, we formulate recommendations on how the HTA community can move toward a more integrated decision-making process using HTA.