Modeling Approaches in Cost-Effectiveness Analysis of Disease-Modifying Therapies for Relapsing–Remitting Multiple Sclerosis: An Updated Systematic Review and Recommendations for Future Economic Evaluations

Hernández, Luis; O'Donnell, Malinda; Postma, Maarten

doi:10.1007/s40273-018-0683-9

Cited by 18 publications

(31 citation statements)

References 105 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The occurrence of relapse(s) within the previous 6 months was also considered as it has been shown to be [64,65], relapse rate is the most frequently primary endpoint in RRMS clinical trials [21,22], and utility decrements due to relapses are included in economic evaluations of DMTs to calculate QALYs [12]. The occurrence of relapse(s) within the last 6 months was modeled as a categorical variable with two levels: yes and no.…”

Section: Potential Predictors Of Utilitymentioning

confidence: 99%

“…Over the past three decades, numerous economic evaluations have assessed the cost effectiveness of DMTs for the treatment of people with RRMS to provide decision makers, payers, and stakeholders with the information needed to determine whether those treatments should be adopted and reimbursed [12][13][14][15][16][17][18][19]. The structure of the decision-analytic models used in these economic evaluations has converged over time [12,14]. The course of disease progression is characterized by changes in a person's disability, as measured by the Expanded Disability Status Scale (EDSS), and the occurrence of relapses during the relapsing-remitting phase and after progression to SPMS [12].…”

Section: Introductionmentioning

confidence: 99%

“…The structure of the decision-analytic models used in these economic evaluations has converged over time [12,14]. The course of disease progression is characterized by changes in a person's disability, as measured by the Expanded Disability Status Scale (EDSS), and the occurrence of relapses during the relapsing-remitting phase and after progression to SPMS [12]. Quality-adjusted life-years (QALYs) are the primary health outcome in these models, calculated using utility weights based on the EDSS during RRMS and utility decrements due to relapses and progression to SPMS [12].…”

Section: Introductionmentioning

confidence: 99%

“…Furthermore, with the growing interest in evaluating the efficacy of DMTs using multiple disability measures in clinical trials of MS, it may follow that additional disability measures could be more commonly included to supplement the EDSS in future economic evaluations of DMTs. However, as described by Hernandez et al, the use of multiple disability measures can pose additional challenges and will inevitably increase the complexity and data demands of these models [12].…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Predictors of Health Utility in Relapsing–Remitting and Secondary-Progressive Multiple Sclerosis: Implications for Future Economic Models of Disease-Modifying Therapies

et al. 2020

Self Cite

View full text Add to dashboard Cite

Background Decision-analytic models used in economic evaluations of disease-modifying therapies for relapsing-remitting multiple sclerosis (RRMS) have characterized disease progression and accrue quality-adjusted life-years from utility values based on the Expanded Disability Status Scale (EDSS), the occurrence of relapses, and progression to secondary-progressive multiple sclerosis (SPMS). The EDSS, used to characterize disability progression, has several limitations. If the EDSS is the only disability measure used in economic evaluations, the long-term clinical and economic implications of disease-modifying therapies may not be properly assessed. Objective The objective of this study was to explore if supplementary disability measures including the Timed 25-Foot Walk (T25FW), 9-Hole Peg Test (9HPT), and Paced Auditory Serial Addition Test (PASAT) significantly contribute additional information on health utility in RRMS and SPMS otherwise not captured by the EDSS and relapses and, therefore, should be considered in future economic evaluations of disease-modifying therapies. Methods Short-Form Six-Dimension utility scores were derived from the RAND 36-Item Health Survey 1.0 individual-level data available in the Multiple Sclerosis Outcome Assessment Consortium (MSOAC) Placebo Database. Repeated-measures mixed-effects models were conducted to estimate the effects of EDSS, T25FW, 9HPT (dominant and non-dominant hand), PASAT, and relapses on changes in utility over time, controlling for demographics. Results A higher level of EDSS, longer time to complete the T25FW test, and a recent relapse were significant predictors of lower utility in people with RRMS and SPMS. 9HPT and PASAT were not significant predictors. Conclusions This study suggests that in addition to EDSS and recent relapses, T25FW significantly predicts utility in RRMS and SPMS. These findings support the use of T25FW to supplement the EDSS and the occurrence of relapses to characterize the course of disease progression and to more accurately accrue quality-adjusted life-years in future economic evaluations of disease-modifying therapies for the treatment of RRMS. Data used in the preparation of this article were obtained from the Multiple Sclerosis Outcome Assessment Consortium (MSOAC). As such, the investigators within MSOAC contributed to the design and implementation of the MSOAC Placebo Database and/ or provided placebo data but did not participate in the analysis of the data or the writing of this report.

show abstract

Section: Potential Predictors Of Utilitymentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Predictors of Health Utility in Relapsing–Remitting and Secondary-Progressive Multiple Sclerosis: Implications for Future Economic Models of Disease-Modifying Therapies

et al. 2020

Self Cite

View full text Add to dashboard Cite

show abstract

“…While RMS transition probabilities and health preference data were used as referenced from Western and high-income countries, the data are from well-known and utilized sources. A recent systematic literature review of 23 publications from 10 countries concludes that the model structure has converged over time [ 44 ] and other international studies, such as our work, rely on the abovementioned model inputs [ 45 – 48 ].…”

Section: Discussionmentioning

confidence: 99%

Cost-effectiveness of Teriflunomide Compared to Interferon Beta-1b for Relapsing Multiple Sclerosis Patients in China

Ningying

Chirikov

et al. 2019

Clin Drug Investig

View full text Add to dashboard Cite

Background and Objective Teriflunomide is a once-daily oral immunomodulatory agent approved in 80 countries for the treatment of patients with relapsing multiple sclerosis (RMS). The study objective was to estimate the cost effectiveness of teriflunomide (14 mg tablet, daily) versus interferon beta-1b (250 mcg subcutaneous injection, every other day) among RMS patients from the Chinese healthcare system perspective. Methods A Markov model with annual cycles and a lifetime horizon was utilized to assess cost-effectiveness of teriflunomide in comparison with interferon beta-1b in RMS patients. Treatment effects, including 3-month confirmed disability worsening and annualized relapse rate, were derived from a network meta-analysis. Cost inputs included costs related to treatment acquisition, administration, monitoring, natural disease management through Expanded Disability Status Scale states, relapse treatment, and adverse event management. These costs were calculated as the product between unit costs from published sources and healthcare resource utilization patterns identified in a survey conducted among 11 neurologists across different areas in China. Health effects were expressed as quality-adjusted life years (QALYs) with costs in local currency (¥) and US dollars (US$), 2018. Results Teriflunomide dominated interferon beta-1b and was associated with lower total costs (teriflunomide ¥1,887,144 vs interferon beta-1b ¥2,061,393) and higher QALYs (teriflunomide 9.60 QALYs vs interferon beta-1b 8.88 QALYs). In probabilistic sensitivity analysis, teriflunomide was dominant in 62.2% of model runs. Conclusion Teriflunomide is a cost-effective therapy over a lifetime time horizon compared to interferon beta-1b in the treatment of RMS patients in China. Results should be interpreted with caution as head-to-head comparisons are not available. Electronic supplementary material The online version of this article (10.1007/s40261-019-00750-3) contains supplementary material, which is available to authorized users.

show abstract

An Innovative Approach to Modelling the Optimal Treatment Sequence for Patients with Relapsing–Remitting Multiple Sclerosis: Implementation, Validation, and Impact of the Decision-Making Approach

Piena

Kroep

Simons³

et al. 2021

Adv Ther

View full text Add to dashboard Cite

Introduction An innovative computational model was developed to address challenges regarding the evaluation of treatment sequences in patients with relapsing–remitting multiple sclerosis (RRMS) through the concept of a ‘virtual’ physician who observes and assesses patients over time. We describe the implementation and validation of the model, then apply this framework as a case study to determine the impact of different decision-making approaches on the optimal sequence of disease-modifying therapies (DMTs) and associated outcomes. Methods A patient-level discrete event simulation (DES) was used to model heterogeneity in disease trajectories and outcomes. The evaluation of DMT options was implemented through a Markov model representing the patient’s disease; outcomes included lifetime costs and quality of life. The DES and Markov models underwent internal and external validation. Analyses of the optimal treatment sequence for each patient were based on several decision-making criteria. These treatment sequences were compared to current treatment guidelines. Results Internal validation indicated that model outcomes for natural history were consistent with the input parameters used to inform the model. Costs and quality of life outcomes were successfully validated against published reference models. Whereas each decision-making criterion generated a different optimal treatment sequence, cladribine tablets were the only DMT common to all treatment sequences. By choosing treatments on the basis of minimising disease progression or number of relapses, it was possible to improve on current treatment guidelines; however, these treatment sequences were more costly. Maximising cost-effectiveness resulted in the lowest costs but was also associated with the worst outcomes. Conclusions The model was robust in generating outcomes consistent with published models and studies. It was also able to identify optimal treatment sequences based on different decision criteria. This innovative modelling framework has the potential to simulate individual patient trajectories in the current treatment landscape and may be useful for treatment switching and treatment positioning decisions in RRMS. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01975-5.

show abstract

Modeling Approaches in Cost-Effectiveness Analysis of Disease-Modifying Therapies for Relapsing–Remitting Multiple Sclerosis: An Updated Systematic Review and Recommendations for Future Economic Evaluations

Abstract: The structure of economic models used in CEAs of DMTs for RRMS has converged over time. However, variation remains in terms of model approach, inputs, and assumptions. Though some recommendations from previous reviews have been incorporated in later models, areas for improvement remain.

Cited by 18 publications

References 105 publications

Predictors of Health Utility in Relapsing–Remitting and Secondary-Progressive Multiple Sclerosis: Implications for Future Economic Models of Disease-Modifying Therapies

Predictors of Health Utility in Relapsing–Remitting and Secondary-Progressive Multiple Sclerosis: Implications for Future Economic Models of Disease-Modifying Therapies

Cost-effectiveness of Teriflunomide Compared to Interferon Beta-1b for Relapsing Multiple Sclerosis Patients in China

An Innovative Approach to Modelling the Optimal Treatment Sequence for Patients with Relapsing–Remitting Multiple Sclerosis: Implementation, Validation, and Impact of the Decision-Making Approach

Contact Info

Product

Resources

About