Cost-Effectiveness of Disease-Modifying Therapies in the Management of Multiple Sclerosis for the Medicare Population
ABSTRACT To evaluate the cost-effectiveness of disease-modifying therapies (DMTs) for the management of multiple sclerosis (MS) compared to best supportive care in the United States.
Cost-effectiveness analysis was undertaken using a state transition model of disease natural history and the impact of DMTs for the representative Medicare beneficiary with MS. Costs and outcomes were evaluated from the health-care payer perspective using a 50-year time horizon. Natural history data were drawn from a longitudinal cohort study. The effectiveness of the DMTs was evaluated through a systematic review. Utility data were taken from a study of patients with clinically definite MS in Nova Scotia. Resource use and cost data were derived from the Sonya Slifka database and associated literature.
When based on placebo-controlled evidence, the marginal cost-effectiveness of interferon beta (IFNβ) and glatiramer acetate compared to best supportive care is expected to be in excess of $100,000 per quality-adjusted life-year gained. When evidence from head-to-head trials is incorporated into the model, the cost-effectiveness of 6 MIU IFNβ-1a is expected to be considerably less favorable. Treatment discontinuation upon progression to Expanded Disability Status Scale 7.0 is expected to improve the cost-effectiveness of all DMTs.
Further research is required to examine the long-term clinical effectiveness and cost-effectiveness of these therapies. There is no definitive guidance in the United States concerning discontinuation of DMTs; this study suggests that the prudent use of a treatment discontinuation rule may considerably improve the cost-effectiveness of DMTs.
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ABSTRACT: Glatiramer acetate (Copaxone®) is a synthetic analogue of the multiple sclerosis (MS)-associated antigen, myelin basic protein. Although its exact mechanisms of action in MS remain to be fully elucidated, the key mechanisms of action of glatiramer acetate appear to be modulation of the inflammatory response and neuroprotective and/or neuroregenerative effects. Subcutaneous glatiramer acetate is indicated for the treatment of adult patients with relapsing-remitting MS (RRMS) and the treatment of patients who have experienced a well-defined first clinical episode and have magnetic resonance imaging (MRI) features consistent with MS or have been determined to be at high risk of developing clinically definite MS (CDMS). In clinical trials in patients with RRMS, glatiramer acetate reduced the frequency of relapses and reduced the burden and activity of disease on MRI, was more effective than placebo and showed generally similar efficacy to subcutaneous interferon (IFN) β-1a and IFNβ-1b. Furthermore, the beneficial effects of glatiramer acetate were sustained during up to 15 years of treatment in an extension study. In patients with clinically isolated syndrome (CIS), glatiramer acetate significantly delayed the onset of CDMS compared with placebo. The drug was generally well tolerated in these patient populations, with injection-site reactions being the most commonly occurring adverse events. Therefore, glatiramer acetate remains a valuable first-line option in the treatment of RRMS and is an option for delaying the onset of CDMS in patients with CIS.CNS Drugs 11/2013; 27(11). DOI:10.1007/s40263-013-0117-3 · 4.38 Impact Factor
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ABSTRACT: The growing number of disease-modifying treatments (DMTs) for patients with multiple sclerosis (MS) and the high acquisition costs of these DMTs are likely to increase the demand for information on their cost effectiveness. To improve the comparability and applicability of the findings from future cost-effectiveness analyses, it would be useful to have a clear understanding of the methodological challenges of modelling the cost effectiveness of DMTs in MS and the different approaches taken by such studies to date. In contrast to previous review studies, this review focuses on long-term time horizon (≥10 years) simulation-based cost-effectiveness analyses with homogeneous contexts of analysis (i.e. those with similar study objectives, comparators, and target populations) published over the past decade. By doing so, it provides a clearer picture of how modelling approaches taken in the existing studies truly differ across studies, and reveals major areas for improvement in conducting future cost-effectiveness analyses of DMTs for patients with MS.PharmacoEconomics 03/2014; 32(6). DOI:10.1007/s40273-014-0150-1 · 3.34 Impact Factor
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ABSTRACT: Background Pregnant women who gain excess weight are at risk of complications during pregnancy and in the long term. Interventions based on diet and physical activity minimise gestational weight gain with varied effect on clinical outcomes. The effect of interventions on varied groups of women based on body mass index, age, ethnicity, socioeconomic status, parity, and underlying medical conditions is not clear. Our individual patient data (IPD) meta-analysis of randomised trials will assess the differential effect of diet- and physical activity-based interventions on maternal weight gain and pregnancy outcomes in clinically relevant subgroups of women. Methods/design Randomised trials on diet and physical activity in pregnancy will be identified by searching the following databases: MEDLINE, EMBASE, BIOSIS, LILACS, Pascal, Science Citation Index, Cochrane Database of Systematic Reviews, Cochrane Central Register of Controlled Trials, Database of Abstracts of Reviews of Effects, and Health Technology Assessment Database. Primary researchers of the identified trials are invited to join the International Weight Management in Pregnancy Collaborative Network and share their individual patient data. We will reanalyse each study separately and confirm the findings with the original authors. Then, for each intervention type and outcome, we will perform as appropriate either a one-step or a two-step IPD meta-analysis to obtain summary estimates of effects and 95% confidence intervals, for all women combined and for each subgroup of interest. The primary outcomes are gestational weight gain and composite adverse maternal and fetal outcomes. The difference in effects between subgroups will be estimated and between-study heterogeneity suitably quantified and explored. The potential for publication bias and availability bias in the IPD obtained will be investigated. We will conduct a model-based economic evaluation to assess the cost effectiveness of the interventions to manage weight gain in pregnancy and undertake a value of information analysis to inform future research. Systematic review registration PROSPERO 2013: CRD4201300380411/2014; 3(1):131. DOI:10.1186/2046-4053-3-131