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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[Show abstract][Hide abstract] ABSTRACT: Objective. To provide a current and comprehensive understanding of the cost-effectiveness of DMTs for the treatment of MS by quantitatively evaluating the quality of recent cost-effectiveness studies and exploring how the field has progressed from past recommendations. Methods. We assessed the quality of studies that met our systematic literature search criteria using the Quality of Health Economic Studies validated instrument. Results. Of the 82 studies that met our initial search criteria, we included 22 in this review. Four studies (18%) achieved quality category 2, three studies (14%) achieved quality category 3, and 15 studies (68%) achieved the highest quality category 4. 91% of studies were simulation models. 13 studies (59%) had quality-adjusted life years (QALYs) as the primary outcome measure, included a societal perspective in the analysis, and utilized time horizons of 10 years to lifetime. Conclusions. To continue to improve the cost-effectiveness evidence of DMTs, we recommend: lifetime horizons, societal perspectives, and QALYs; supplemental evidence with shorter horizons, payer perspectives, and clinical outcomes to inform multiple decision makers; development of modeling and input standards for comparability; head-to-head RCTs between DMTs and long-term prospective studies; and comprehensive cost-effectiveness studies that compare all appropriate DMTs.
"The efficacy of the disease modifying drug (DMD) glatiramer acetate (GA) in reducing relapses in relapsing remitting multiple sclerosis (RRMS) has been demonstrated in randomized placebo-controlled trials [1,2]. Studies on the effectiveness of GA treatment in daily neurological practice have concentrated on relapses, disability, fatigue [3-5], work absenteeism  and cost-effectiveness [6,7]. "
[Show abstract][Hide abstract] ABSTRACT: Glatiramer acetate (GA) and interferon-beta (INFb) are first-line disease modifying drugs for relapsing remitting multiple sclerosis (RRMS). Treatment with INFb is associated with a significant increase in health-related quality of life (HR-QoL) in the first 12 months. It is not known whether HR-QoL increases during treatment with GA.
197 RRMS patients, 106 without and 91 with prior immunomodulation/immunosuppression, were studied for HR-QoL (Leeds Multiple Sclerosis-QoL [LMS-QoL] scale, score range 0 - 32), fatigue (Fatigue Impact Scale [FIS]) and depressed mood (Beck Depression Inventory-Short Form [BDI-SF]) at baseline and 6 and 12 months after start of GA treatment.
At 6 and 12 months mean LMS-QoL scores were significantly increased in the treatment-naive patient group (p < 0.001), not in the pre-treated group. At month 12 43% of treatment-naïve patients had improved HR-QoL (increase LMS-QoL score 3 or more points) (p < 0.001). Likewise, mean FIS scores were decreased at months 6 and 12 in the treatment-naïve group (p < 0.01), not in the pre-treated group. In both groups mean BDI-SF scores did not change. No demographic or clinical baseline factor was predictive of HR-QoL increase. HR-QoL changes were zero to negative for patients who had discontinued GA before month 12 (28.4% of patients).
In RRMS patients without prior immunomodulation/immunosuppression treatment with GA was associated with an increase in HR-QoL in the first 6 months, that was sustained at 12 months. In 4 out of 10 patients HR-QoL improved. Increase in HR-QoL was associated with decrease in fatigue.
Health and Quality of Life Outcomes 11/2010; 8(1):133. DOI:10.1186/1477-7525-8-133 · 2.12 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Multiple sclerosis (MS) is a chronic, disabling, and progressive illness, representing one of the most common causes of neurological disability in young and middle-aged adults. There is not a definitive treatment for MS yet. However, disease-modifying drugs (DMDs) for MS, which include interferon-beta and copolymer-1 have shown to be effective in reducing the frequency and severity of relapses and the progression of disability. The clinical efficacy of such therapies has been well documented in the medical literature. Instead, the factors underlying the decision to start the pharmacological treatment, to continue it or to drop out, have not been studied so far. Adverse drug effects, as well as patients’ emotional states, therapeutic expectations, the need to assume the medicines very often, and lack of communication with medical staff, are some of the elements affecting patients’ adherence to the therapy. Data from medical records of 567 MS patients referred to the MS Centre of the IRCCS Centro Studi Neurolesi (Messina) between the years 2001-2008 have been retrospectively analyzed in a first phase. Factors influencing patient decision to start a pharmacological treatment with DMDs, in agreement with the neurologist suggestion, have been evaluated by applying a multinomial logit model. The second phase of the study was cross-sectional and analyzed the data obtained through a questionnaire administered to consecutive outpatients referred to Centro Studi Neurolesi within March and May 2009 (n = 143). The probability to proceed in the treatment or to drop out was estimated through a probit model. The present research constitutes a novelty among the existing economic and medical literature: in fact, there are no, so far, studies evaluating factors underlying MS patients’ decision to undergo a pharmacological treatment and to proceed it according to medical protocols. Moreover, a significant expenditure for health care systems is associated to MS treatment, both for patients who undergo the treatment (cost of medicines, productivity losses for patients who experience severe side effects, etc.) and for those who do not take the medicine or take it discontinuously. Given the documented evidence of augmenting costs (direct and indirect) with increasing disease severity, the ability of the DMDs to reduce relapse rates and slow the progression of MS may help to offset the cost of these therapies. Conversely, delayed treatment or poor compliance can dramatically increase costs and reduce benefits.