Disease-Modifying Therapies for Relapsing–Remitting and Primary Progressive Multiple Sclerosis: A Cost-Utility Analysis

Zimmermann, Marita; Brouwer, Elizabeth; Tice, Jeffrey A.; Seidner, M.; Loos, Anne M.; Liu, Shanshan; Chapman, Richard H.; Kumar, Varun; Carlson, Josh J.

doi:10.1007/s40263-018-0566-9

Cited by 36 publications

(54 citation statements)

References 33 publications

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“…Os resultados obtidos nesta análise são qualitativamente similares aos descritos anteriormente, demonstrando a dominância de alentuzumabe comparado a natalizumabe em outras análises de custo-efetividade 25,26,27 . Apesar dos estudos terem analisado o uso de alentuzambe como tratamento em primeira linha, o natalizumabe também foi feito considerado nesta linha na análise.…”

Section: Discussionunclassified

Custo-efetividade de Alentuzumabe vs. Natalizumabe no tratamento de terceira linha da esclerose múltipla remitente recorrente para a saúde suplementar no Brasil

Nacazume¹,

Cavalcanti²,

Julian³

et al. 2022

JAFF

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Resumo: Objetivo: A esclerose múltipla é uma doença crônica, inflamatória e degenerativa do sistema nervoso central que afeta aproximadamente 2,5 milhões de pessoas no mundo. Cerca de 80% dos casos são classificados no fenótipo 'remitente recorrente' (EMRR). Objetivo: Avaliou-se, sob a perspectiva de pagador na saúde suplementar no Brasil, a relação de custo-efetividade do tratamento com alentuzumabe em pacientes de EMRR em 3ª linha de tratamento versus natalizumabe, único tratamento disponibilizado na saúde suplementar atualmente. Método: Com auxílio de modelo de Markov, simulou-se, no horizonte temporal de 30 anos, uma coorte de mil pacientes, com parâmetros demográficos baseados em dados do sistema público de saúde do Brasil. Em cada ciclo anual, os pacientes poderiam transitar entre diferentes estados de saúde, mensurados através do escore na Escala Expandida de Estado de Incapacidade (EDSS), além de experienciar surtos e progressão para o tipo secundariamente progressivo. A distribuição da população inicial da coorte por nível de EDSS baseou-se no estudo clínico CARE MS II. Os parâmetros de eficácia relacionados à progressão de incapacidade e taxa de surtos foram selecionados de metanálise de rede, assim como taxas de descontinuação e retratamento, parâmetros de utilidade, segurança e custos da doença foram obtidos de ensaios clínicos e estudos publicados. Custos por dose e administração foram retirados de tabelas de preço oficiais. Resultados: Demonstra-se que alentuzumabe é dominante, acumulando mais QALYs (6.752) por um custo total de tratamento menor (R$ 377,9 milhões) em relação ao natalizumabe (5.652; R$ 432,9 milhões). O tratamento com alentuzumabe apresenta vantagens quanto à eficácia no longo-prazo e efetividade na redução da ocorrência de surtos e hospitalizações. A incerteza paramétrica do modelo foi analisada através de análises de sensibilidade determinística e probabilística. A análise de sensibilidade corrobora os resultados pois, mesmo com a variação de parâmetros-alvo de incerteza, o alentuzumabe ainda possui 97,8% de probabilidade de ser custo-efetivo versus o natalizumabe, considerando o limiar de 3 PIB per capita. Conclusão: Alentuzumabe é uma nova terapia custo-efetiva, provando ser alternativa economicamente viável. Análises de custo-efetividade como esta são importantes para otimizar a tomada de decisões de gestores do sistema de saúde, tanto públicos como privados.

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Section: Discussionunclassified

Custo-efetividade de Alentuzumabe vs. Natalizumabe no tratamento de terceira linha da esclerose múltipla remitente recorrente para a saúde suplementar no Brasil

Nacazume¹,

Cavalcanti²,

Julian³

et al. 2022

JAFF

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“…Three effects of shared decision making were assumed and modeled ( Figure 2 and further specified in the supplementary material): 1) shared decision making changes the DMD mix chosen, 2) shared decision making increases the proportion of optimally adherent patients, and 3) shared decision making increases persistence with the chosen treatment. Key assumptions for modeling shared decision making in comparison with usual care Figure 1 Health state transition model structure for multiple sclerosis course, adapted from Zimmermann et al 40 are presented in Table 1. Effect 1 was based on the fact that patients' preferences are elicited and included in the treatment choice during a shared decision making process, and these preferences might differ from physicians' preferences.…”

Section: Intervention Effectsmentioning

confidence: 99%

“…[15][16][17] No data were available for ocrelizumab and alemtuzumab. Discontinuation rates were therefore determined based on the rate of natalizumab, proportionately according to differences found in trial discontinuation rates between 28,40 Mortality risk within each health state was the same for RRMS and SPMS. Patients progressing to SPMS continued treatment with DMD.…”

Section: Intervention Effectsmentioning

confidence: 99%

Exploring the Cost Effectiveness of Shared Decision Making for Choosing between Disease-Modifying Drugs for Relapsing-Remitting Multiple Sclerosis in the Netherlands: A State Transition Model

et al. 2020

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Background Up to 31% of patients with relapsing-remitting multiple sclerosis (RRMS) discontinue treatment with disease-modifying drug (DMD) within the first year, and of the patients who do continue, about 40% are nonadherent. Shared decision making may decrease nonadherence and discontinuation rates, but evidence in the context of RRMS is limited. Shared decision making may, however, come at additional costs. This study aimed to explore the potential cost-effectiveness of shared decision making for RRMS in comparison with usual care, from a (limited) societal perspective over a lifetime. Methods An exploratory economic evaluation was conducted by adapting a previously developed state transition model that evaluates the cost-effectiveness of a range of DMDs for RRMS in comparison with the best supportive care. Three potential effects of shared decision making were explored: 1) a change in the initial DMD chosen, 2) a decrease in the patient’s discontinuation in using the DMD, and 3) an increase in adherence to the DMD. One-way and probabilistic sensitivity analyses of a scenario that combined the 3 effects were conducted. Results Each effect separately and the 3 effects combined resulted in higher quality-adjusted life years (QALYs) and costs due to the increased utilization of DMD. A decrease in discontinuation of DMDs influenced the incremental cost-effectiveness ratio (ICER) most. The combined scenario resulted in an ICER of €17,875 per QALY gained. The ICER was sensitive to changes in several parameters. Conclusion This study suggests that shared decision making for DMDs could potentially be cost-effective, especially if shared decision making would help to decrease treatment discontinuation. Our results, however, may depend on the assumed effects on treatment choice, persistence, and adherence, which are actually largely unknown.

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“…Comparison of the DMT AOMS studies has inherent limitations; multiple comparisons have been reported including a value based comparison from the Institute for Clinical and Economic review, ICER, looking at the strength of the evidence, patient goals, and costs for each FDA approved DMT products [47]. A standardized ICER evaluation of all MS DMT concluded the newest FDA AOMS approved product, ocrelizumab dominating the other DMTs when cost was compared with supportive care for the AOMS patient, showing it as the best value in the US market or as cost effective as a first line treatment for RRMS [27]. There is no similar ICER evaluation for DMT use in POMS.…”

Section: Escalation Of Therapymentioning

confidence: 99%

Long-Term Treatment Strategies of Pediatric Multiple Sclerosis, Including the use of Disease Modifying Therapies

Rensel

2019

Children

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Multiple sclerosis (MS) presenting in the pediatric years can lead to landmark disability levels younger in life than adult onset MS and so therefore early and effective treatment remains paramount for long-term outcomes. The goals of MS therapeutics in adults have widened to address multiple mechanisms: anti-inflammatory, neuroprotective, and myelin repair, yet the optimal paradigm for MS therapies in the pediatric population is not known. Pediatric onset MS add complexities due to the ongoing development of the central nervous system and the immune system. Clinical trials have led to an increasing number of pharmaceutical therapies for adult onset MS (AOMS), one POMS randomized controlled trial is completed and other trials are ongoing, yet due to the low prevalence of POMS, the dynamic landscape and risk management of the MS disease modifying therapies (DMT) it remains more difficult to complete trials in POMS. There is consensus that controlled clinical trials leading to appropriate and safe therapies for POMS are important for a multitude of reasons that include unique pediatric pharmacokinetics, short and long-term safety, developmental issues, clinical benefits, and regulatory approval. This review will focus on new treatment goals, paradigm, strategies, monitoring, compliance, and products in the long-term treatment of POMS. The discussion will focus on these new concepts and the published data related to DMT use in POMS. This review provides significant insight into new concepts of treatment goals and current approaches to enhance the lives of the POMS patients now and in the future.

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Disease-Modifying Therapies for Relapsing–Remitting and Primary Progressive Multiple Sclerosis: A Cost-Utility Analysis

Cited by 36 publications

References 33 publications

Custo-efetividade de Alentuzumabe vs. Natalizumabe no tratamento de terceira linha da esclerose múltipla remitente recorrente para a saúde suplementar no Brasil

Custo-efetividade de Alentuzumabe vs. Natalizumabe no tratamento de terceira linha da esclerose múltipla remitente recorrente para a saúde suplementar no Brasil

Exploring the Cost Effectiveness of Shared Decision Making for Choosing between Disease-Modifying Drugs for Relapsing-Remitting Multiple Sclerosis in the Netherlands: A State Transition Model

Long-Term Treatment Strategies of Pediatric Multiple Sclerosis, Including the use of Disease Modifying Therapies

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