RESUMO
OBJECTIVE: Although off-label use of rituximab is a common alternative to disease-modifying therapies (DMTs) approved for multiple sclerosis (MS) in several countries, the impact of this on treatment cost-effectiveness is not well known. METHODS: We evaluated the relative cost-effectiveness of rituximab and MS-approved DMTs in a register-based cohort study of Swedish residents with relapsing-remitting MS, aged 18-65 years, starting treatment with rituximab, natalizumab, fingolimod, or dimethyl fumarate between January 2010 and July 2016, and followed through July 2021 (n = 5,924). By linking the population-based Swedish MS register to several Swedish health care and demographic registers, we estimated health care costs in relation to number of relapses, over 5 years from treatment start. Differences between treatments were estimated in inverse probability of treatment-weighted regression models, adjusting for a broad range of potential confounders covering demographics, medical history, and MS-related clinical characteristics. RESULTS: Off-label rituximab was associated with both lower total health care costs (mean cost savings ranged $35,000-$66,000 vs. each approved DMT), and fewer relapses (mean number of prevented relapses ranged 0.12-0.22), per started therapy over 5 years. Results were robust to variations in discounting and pricing of health care visits, with the main driver of cost-savings being the price of the index drug itself. INTERPRETATION: The cost-effectiveness of rituximab dominated the MS-approved alternatives. Off-label, low-dose rituximab should be considered for persons with MS and could reduce barriers to treatment, especially in resource-limited settings. ANN NEUROL 2024;95:1099-1111.
Assuntos
Redução de Custos , Análise Custo-Benefício , Custos de Cuidados de Saúde , Esclerose Múltipla Recidivante-Remitente , Uso Off-Label , Sistema de Registros , Rituximab , Humanos , Rituximab/uso terapêutico , Rituximab/economia , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Adulto , Uso Off-Label/economia , Pessoa de Meia-Idade , Feminino , Masculino , Suécia , Adulto Jovem , Adolescente , Custos de Cuidados de Saúde/estatística & dados numéricos , Fatores Imunológicos/uso terapêutico , Fatores Imunológicos/economia , Idoso , Estudos de Coortes , RecidivaRESUMO
BACKGROUND: Uncertainty about disproportionate impact on health care budgets limits implementation of early highly effective treatment (EHT) in multiple sclerosis (MS). OBJECTIVE: To estimate cost-effectiveness of escalation versus EHT disease-modifying treatment (DMT) sequences. METHODS: Using a health-economic approach, we analysed health benefits (relapse rate reduction, disability prevention), direct/indirect DMT and societal costs of escalation versus EHT DMT sequences. In scenario analyses, we allowed (1) earlier use of alemtuzumab (ALE) and (2) a single retreatment with cladribine (CLA). RESULTS: In our model, we showed that the ratio between costs and quality-adjusted life years (QALYs) for the most cost-effective EHT and escalation sequence results into a similar net health benefit with higher costs and also higher QALYs associated with an EHT versus escalation strategy. Earlier use of ALE is more cost-effective than in later lines, even when aggravating the impact of its side-effects tenfold. Retreatment with CLA was more cost-effective in both escalation and EHT sequences. CONCLUSIONS: Certain EHT sequences are equally cost-effective to escalation sequences and are likely to result in more health at uncertain additional costs. The favourable cost-benefit ratio of CLA and ALE suggests that a wider application of affordable highly effective therapies could promote the cost-effectiveness both EHT and escalation approaches.
Assuntos
Alemtuzumab , Análise Custo-Benefício , Esclerose Múltipla Recidivante-Remitente , Anos de Vida Ajustados por Qualidade de Vida , Humanos , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Alemtuzumab/administração & dosagem , Alemtuzumab/economia , Cladribina/administração & dosagem , Cladribina/economia , Fatores Imunológicos/economia , Fatores Imunológicos/administração & dosagem , Modelos Econômicos , Imunossupressores/economia , Imunossupressores/administração & dosagem , Imunossupressores/uso terapêuticoRESUMO
BACKGROUND: Autologous haematopoietic stem cell transplantation (AHSCT) is a highly effective one-off treatment for relapsing-remitting multiple sclerosis (RR-MS), potentially representing an optimal front-loading strategy for costs. OBJECTIVE: Exploring cost/effectiveness of AHSCT and high-efficacy disease-modifying treatments (HE-DMTs) in RR-MS, estimating costs at our centre in Italy, where National Health Service (NHS) provides universal health coverage. METHODS: Costs (including drugs, inpatient/outpatient management) for treatment with AHSCT and HE-DMTs were calculated as NHS expenditures over 2- and 5-year periods. Cost-effectiveness for each treatment was estimated as "cost needed to treat" (CNT), i.e. expense to prevent relapses, progression, or disease activity (NEDA) in one patient over n-years, retrieving outcomes from published studies. RESULTS: Costs of AHSCT and HE-DMTs were similar over 2 years, whereas AHSCT was cheaper than most HE-DMTs over 5 years (46 600 vs 93 800, respectively). When estimating cost-effectiveness of treatments, over 2 years, mean CNT of HE-DMTs for NEDA was twofold that of AHSCT, whereas it was similar for relapses and disability. Differences in CNT were remarkable over 5 years, especially for NEDA, being mean CNT of HE-DMTs 382 800 vs 74 900 for AHSCT. CONCLUSIONS: AHSCT may be highly cost-effective in selected aggressive RR-MS. Besides priceless benefits for treated individuals, cost-savings generated by AHSCT may contribute to improving healthcare assistance at a population level.
Assuntos
Análise Custo-Benefício , Transplante de Células-Tronco Hematopoéticas , Esclerose Múltipla Recidivante-Remitente , Transplante Autólogo , Humanos , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/terapia , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Transplante Autólogo/economia , Masculino , Feminino , Adulto , Itália , Resultado do Tratamento , Pessoa de Meia-IdadeRESUMO
OBJECTIVES: Disease-modifying therapies (DMTs) reduce relapse rates and disability progression for relapsing multiple sclerosis (MS). Although 25% to 30% of all US patients with MS are Medicare beneficiaries, limited information exists on this population. This is the first study using national Medicare data to (1) describe characteristics of patients with MS using DMTs, (2) estimate adherence to DMTs over a 1-year and 3-year follow-up, and (3) examine factors associated with DMT adherence. METHODS: This retrospective claims analysis used 2011-2014 100% Medicare files. Monthly adherence to MS DMTs was defined as the proportion of days covered ≥0.80 with any DMT in each month for 1-year (n = 36 593) and 3-year (n = 17 599) follow-up samples of MS DMT users. Generalized estimating equation logistic regressions were used to estimate factors associated with adherence to DMTs. RESULTS: Over 90% of patients were eligible for Medicare owing to disability, and about three-quarters qualified for low-income subsidies. A downward trend in DMT adherence was observed over time in both samples. Monthly adherence dropped significantly between December of the prior year to January of the following year (from 76% to 65% in the 1-year follow-up sample and similar drops seen across all years in the 3-year follow-up sample). Multivariable regressions indicated characteristics such as being low-income, having a disability, and having high patient out-of-pocket DMT costs associated with poor adherence to DMTs. CONCLUSION: Our study provides important insights into the characteristics and DMT adherence of Medicare patients with MS and highlights the need for interventions and policies mitigating barriers to adherence in this population.
Assuntos
Acessibilidade aos Serviços de Saúde , Fatores Imunológicos/uso terapêutico , Medicare , Adesão à Medicação , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Demandas Administrativas em Assistência à Saúde , Adulto , Idoso , Data Warehousing , Bases de Dados Factuais , Avaliação da Deficiência , Custos de Medicamentos , Definição da Elegibilidade , Feminino , Gastos em Saúde , Acessibilidade aos Serviços de Saúde/economia , Humanos , Fatores Imunológicos/efeitos adversos , Fatores Imunológicos/economia , Renda , Masculino , Pessoa de Meia-Idade , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/epidemiologia , Estudos Retrospectivos , Fatores de Tempo , Estados Unidos/epidemiologiaRESUMO
OBJECTIVE: To evaluate the cost-effectiveness of alemtuzumab compared with fingolimod, natalizumab, ocrelizumab, and generic glatiramer acetate 20 mg among patients with relapsing multiple sclerosis (RMS) in the United States. STUDY DESIGN: Markov model with annual periods from payer perspective. METHODS: The modeled population represented pooled patients from the CARE-MS I and II trials. Therapies' comparative efficacy at reducing relapses and slowing disability worsening was obtained from network meta-analyses. Safety information was extracted from package inserts. Withdrawal rates, treatment waning, resource use, cost, and utility inputs were derived from published studies and clinical expert opinion. To project the natural history of disease worsening, data from the British Columbia cohort was used. RESULTS: Alemtuzumab dominated comparators by accumulating higher total quality-adjusted life-years (QALYs) (8.977) and lower total costs ($421 996) compared with fingolimod (7.955; $1 085 814), natalizumab (8.456; $1 048 599), ocrelizumab (8.478; $908 365), and generic glatiramer acetate (7.845; $895 661) over a 20-year time horizon. Alemtuzumab's dominance was primarily driven by savings in treatment costs because alemtuzumab has long-term duration of response and is initially administered as 2 annual courses, with 36.1% of patients requiring retreatment over 5 years, whereas comparators are used chronically. In model scenarios where alemtuzumab's long-term duration of response was assumed not to hold and therapy had to be administered annually, probabilistic sensitivity analyses showed that alemtuzumab remained cost-effective versus ocrelizumab at a willingness-to-pay threshold of $100 000/QALY in 74% to 100% of model runs. CONCLUSIONS: Alemtuzumab was a cost-effective therapy. Model results should be used to optimize clinical and managed care decisions for effective RMS treatment.
Assuntos
Alemtuzumab/economia , Antineoplásicos Imunológicos/economia , Análise Custo-Benefício , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Adulto , Idoso , Idoso de 80 Anos ou mais , Alemtuzumab/uso terapêutico , Anticorpos Monoclonais Humanizados/economia , Anticorpos Monoclonais Humanizados/uso terapêutico , Antineoplásicos Imunológicos/uso terapêutico , Análise Custo-Benefício/métodos , Feminino , Cloridrato de Fingolimode/economia , Cloridrato de Fingolimode/uso terapêutico , Acetato de Glatiramer/economia , Acetato de Glatiramer/uso terapêutico , Humanos , Imunossupressores/economia , Imunossupressores/uso terapêutico , Masculino , Cadeias de Markov , Pessoa de Meia-Idade , Esclerose Múltipla Recidivante-Remitente/epidemiologia , Natalizumab/economia , Natalizumab/uso terapêutico , Resultado do Tratamento , Estados Unidos/epidemiologia , Adulto JovemRESUMO
BACKGROUND: Published literature suggests that early treatment with natalizumab ("escalation strategy") is more effective than switch within the same class of immunomodulators (interferons/glatiramer acetate, "switching strategy") in relapsing-remitting multiple sclerosis (RRMS) patients who failed first-line self-injectable disease-modifying treatment (DMT). The present analysis aims to evaluate the cost-effectiveness profile of escalation strategy vs. switching strategy, adopting the Italian societal perspective. METHODS: A lifetime horizon Markov model was developed to compare early escalation to natalizumab vs. switching among immunomodulators, followed by subsequent escalation to natalizumab. The two compared treatment algorithms were: a) early escalation until progression to Expanded Disability Status Scale (EDSS) = 7.0 vs. b) switching until EDSS = 4.0, followed by escalation until EDSS = 7.0. The model analyzed social costs, quality-adjusted survival and effects of therapies in prolonging time without disability progression and burden of relapses. Clinical data were mainly extracted from a published observational study. RESULTS: Lifetime costs of early escalation to natalizumab and switching among immunomodulators amounted to 699,700 and 718,600 per patient, respectively. Early escalation was associated with prolonged quality-adjusted survival (11.19 vs. 9.67 QALYs, + 15.8%). A slight overall survival increase was also observed (20.10 vs. 19.67 life years). Both deterministic and probabilistic sensitivity analyses confirmed the robustness of findings. CONCLUSIONS: Adopting the Italian social perspective, early escalation to natalizumab is dominant vs. switching among immunomodulators, in RRMS patients who do not respond adequately to conventional immunomodulators.
Assuntos
Fatores Imunológicos/economia , Fatores Imunológicos/uso terapêutico , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Natalizumab/economia , Natalizumab/uso terapêutico , Adulto , Análise Custo-Benefício , Progressão da Doença , Feminino , Humanos , Itália , Masculino , Cadeias de Markov , Esclerose Múltipla Recidivante-Remitente/fisiopatologia , RecidivaRESUMO
OBJECTIVES: Several disease-modifying therapies have marketing authorizations for the treatment of relapsing-remitting multiple sclerosis (RRMS). Given their appraisal by the National Institute for Health and Care Excellence, the objective was to systematically identify and critically evaluate the structures and assumptions used in health economic models of disease-modifying therapies for RRMS in the United Kingdom. METHODS: Embase, MEDLINE, The Cochrane Library, and the National Institute for Health and Care Excellence Web site were searched systematically on March 3, 2014, to identify articles relating to health economic models in RRMS with a UK perspective. Data sources, techniques, and assumptions of the included models were extracted, compared, and critically evaluated. RESULTS: Of 386 results, 26 full texts were evaluated, leading to the inclusion of 18 articles (relating to 12 models). Early models varied considerably in method and structure, but convergence over time toward a Markov model with states based on disability score, a 1-year cycle length, and a lifetime time horizon was apparent. Recent models also allowed for disability improvement within the natural history of the condition. Considerable variety remains, with increasing numbers of comparators, the need for treatment sequencing, and different assumptions around efficacy waning and treatment withdrawal. CONCLUSIONS: Despite convergence over time to a similar Markov structure, there are still significant discrepancies between health economic models of RRMS in the United Kingdom. Differing methods, assumptions, and data sources render the comparison of model implementation and results problematic. The commonly used Markov structure leads to problems such as incapability to deal with heterogeneous populations and multiplying complexity with the addition of treatment sequences; these would best be solved by using alternative models such as discrete event simulations.
Assuntos
Custos de Medicamentos , Fatores Imunológicos/economia , Fatores Imunológicos/uso terapêutico , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Medicina Estatal/economia , Simulação por Computador , Análise Custo-Benefício , Avaliação da Deficiência , Gastos em Saúde , Pesquisa sobre Serviços de Saúde , Humanos , Fatores Imunológicos/efeitos adversos , Cadeias de Markov , Modelos Econômicos , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Esclerose Múltipla Recidivante-Remitente/fisiopatologia , Valor Preditivo dos Testes , Recuperação de Função Fisiológica , Reprodutibilidade dos Testes , Fatores de Tempo , Resultado do Tratamento , Reino UnidoRESUMO
BACKGROUND: Exercise is a safe, non-pharmacological adjunctive treatment for people with multiple sclerosis but cost-effective approaches to implementing exercise within health care settings are needed. OBJECTIVE: The objective of this paper is to assess the cost effectiveness of a pragmatic exercise intervention in conjunction with usual care compared to usual care only in people with mild to moderate multiple sclerosis. METHODS: A cost-utility analysis of a pragmatic randomised controlled trial over nine months of follow-up was conducted. A total of 120 people with multiple sclerosis were randomised (1:1) to the intervention or usual care. Exercising participants received 18 supervised and 18 home exercise sessions over 12 weeks. The primary outcome for the cost utility analysis was the incremental cost per quality-adjusted life year (QALY) gained, calculated using utilities measured by the EQ-5D questionnaire. RESULTS: The incremental cost per QALY of the intervention was £10,137 per QALY gained compared to usual care. The probability of being cost effective at a £20,000 per QALY threshold was 0.75, rising to 0.78 at a £30,000 per QALY threshold. CONCLUSION: The pragmatic exercise intervention is highly likely to be cost effective at current established thresholds, and there is scope for it to be tailored to particular sub-groups of patients or services to reduce its cost impact.
Assuntos
Terapia por Exercício/economia , Custos de Cuidados de Saúde , Esclerose Múltipla Crônica Progressiva/economia , Esclerose Múltipla Crônica Progressiva/terapia , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/terapia , Adulto , Análise Custo-Benefício , Inglaterra , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Modelos Econômicos , Esclerose Múltipla Crônica Progressiva/diagnóstico , Esclerose Múltipla Crônica Progressiva/fisiopatologia , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Esclerose Múltipla Recidivante-Remitente/fisiopatologia , Anos de Vida Ajustados por Qualidade de Vida , Índice de Gravidade de Doença , Inquéritos e Questionários , Fatores de Tempo , Resultado do TratamentoRESUMO
Interferon beta (IFNß) was the first specific disease-modifying treatment licensed for relapsing-remitting multiple sclerosis, and is still one of the most commonly prescribed treatments. A strong body of evidence supports the effectiveness of IFNß preparations in reducing the annual relapse rate, magnetic resonance (MRI) disease activity and disease progression. However, the development of binding/neutralizing antibodies (BAbs/NAbs) during treatment negatively affects clinical and MRI outcomes. Therefore, guidelines for the clinical use for the detection of NAbs in MS may result in better treatment of these patients. In October 2012, a panel of Italian neurologists from 17 MS clinics convened in Milan to review and discuss data on NAbs and their clinical relevance in the treatment of MS. In this paper, we report the panel's recommendations for the use of IFNß Nabs detection in the early identification of IFNß non-responsiveness and the management of patients on IFNß treatment in Italy, according to a model of therapeutically appropriate care.
Assuntos
Anticorpos Neutralizantes/sangue , Fatores Imunológicos/uso terapêutico , Interferon beta/imunologia , Interferon beta/uso terapêutico , Esclerose Múltipla/tratamento farmacológico , Esclerose Múltipla/imunologia , Diagnóstico Precoce , Humanos , Fatores Imunológicos/imunologia , Itália , Imageamento por Ressonância Magnética , Esclerose Múltipla/diagnóstico , Esclerose Múltipla/economia , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/imunologia , Proteínas de Resistência a Myxovirus/metabolismo , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
INTRODUCTION: At present, there is a lack of economic assessments of second-line treatments for relapsing-recurring multiple sclerosis. The aim of this study was to compare the efficiency between fingolimod and natalizumab in Spain. METHODS: A cost minimisation analysis model was developed for a 2-year horizon. The same relapse rate was applied to both treatment arms and the cost of resources was calculated using Spain's stipulated rates for 2012 in euros. The analysis was conducted from the perspective of Spain's national health system and an annual discount rate of 3% was applied to future costs. A sensitivity analysis was performed to validate the robustness of the model. RESULTS: Indirect comparison of fingolimod with natalizumab revealed no significant differences (hazard ratio between 0.82 and 1.07). The total direct cost, considering a 2-year analytical horizon, a 7.5% discount stipulated by Royal Decree, and a mean annual relapse rate of 0.22, was 40914.72 for fingolimod and 45890.53 for natalizumab. Of the total direct costs that were analysed, the maximum cost savings derived from prescribing fingolimod prescription was 4363.63, corresponding to lower administration and treatment maintenance costs. Based on the sensitivity analysis performed, fingolimod use was associated with average savings of 11% (range 3.1%-18.7%). CONCLUSIONS: Fingolimod is more efficient than natalizumab as a second-line treatment option for relapsing-remitting multiple sclerosis and it generates savings for the Spanish national health system.
Assuntos
Anticorpos Monoclonais Humanizados/economia , Anticorpos Monoclonais Humanizados/uso terapêutico , Controle de Custos/métodos , Imunossupressores/economia , Imunossupressores/uso terapêutico , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Propilenoglicóis/economia , Propilenoglicóis/uso terapêutico , Esfingosina/análogos & derivados , Custos de Medicamentos , Cloridrato de Fingolimode , Humanos , Natalizumab , Espanha , Esfingosina/economia , Esfingosina/uso terapêuticoRESUMO
OBJECTIVES: Biosimilars provide an opportunity for a more sustainable and cost-effective treatment for multiple sclerosis (MS). This study evaluated the potential financial impact of implementing a formulary change from reference to biosimilar natalizumab (NTZ) from the US commercial payer perspective. STUDY DESIGN: The budget impact of transitioning to biosimilar NTZ for the treatment of relapsing-remitting MS (RRMS) was estimated over a 3-year time horizon based on real-world dosing. Additional scenario analyses were conducted by varying the price differential of biosimilar NTZ. METHODS: The target population was estimated from a 1-million-member hypothetical commercial health plan. Model inputs were drug acquisition costs and treatment-related and patient coinsurance costs. Budget impact and cost savings per member per year were calculated by assuming a biosimilar uptake of 10% in year 1 to 20% in year 3. RESULTS: Over 3 years, 255 patients were estimated to be treated with high-efficacy disease-modifying therapies for RRMS. The inclusion of biosimilar NTZ onto a formulary would result in cumulative cost savings to payers of $452,611 over 3 years, with mean savings per treated member per year of $1179, $1769, and $2359 in years 1, 2, and 3, respectively. One-way sensitivity analyses indicated that budget impact results were most sensitive to drug acquisition costs of both reference and biosimilar NTZ. CONCLUSION: Adoption of biosimilar NTZ can yield considerable cost savings to US health plans that could result in increased treatment access for patients with RRMS.
Assuntos
Medicamentos Biossimilares , Orçamentos , Natalizumab , Humanos , Natalizumab/uso terapêutico , Natalizumab/economia , Estados Unidos , Medicamentos Biossimilares/economia , Medicamentos Biossimilares/uso terapêutico , Anticorpos Monoclonais Humanizados/uso terapêutico , Anticorpos Monoclonais Humanizados/economia , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Esclerose Múltipla Recidivante-Remitente/economia , Análise Custo-Benefício , Redução de Custos , Custos de Medicamentos/estatística & dados numéricosRESUMO
BACKGROUND: Fatigue is a common and troubling symptom for people with multiple sclerosis (MS). AIM: To evaluate the effectiveness and cost-effectiveness of a six-session group-based programme for managing MS-fatigue (Fatigue: Applying Cognitive behavioural and Energy effectiveness Techniques to lifeStyle (FACETS)). METHODS: Three-centre parallel arm randomised controlled trial with economic evaluation. Patients with MS and significant fatigue were randomised to FACETS plus current local practice (FACETS) or current local practice alone (CLP), using concealed computer-generated randomisation. Participant blinding was not possible. Primary outcomes were fatigue severity (Fatigue Assessment Instrument), self-efficacy (Multiple Sclerosis-Fatigue Self-Efficacy) and disease-specific quality of life (Multiple Sclerosis Impact Scale (MSIS-29)) at 1 and 4 months postintervention (follow-up 1 and 2). Quality adjusted life years (QALYs) were calculated (EuroQoL 5-Dimensions questionnaire and the Short-form 6-Dimensions questionnaire). RESULTS: Between May 2008 and November 2009, 164 patients were randomised; primary outcome data were available for 146 (89%). Statistically significant differences favour the intervention group on fatigue self-efficacy at follow-up 1 (mean difference (MD) 9, 95% CI (4 to 14), standardised effect size (SES) 0.54, p=0.001) and follow-up 2 (MD 6, 95% CI (0 to 12), SES 0.36, p=0.05) and fatigue severity at follow-up 2 (MD -0.36, 95% CI (-0.63 to -0.08), SES -0.35, p=0.01) but no differences for MSIS-29 or QALYs. No adverse events reported. Estimated cost per person for FACETS is £453; findings suggest an incremental cost-effectiveness ratio of £2157 per additional person with a clinically significant improvement in fatigue. CONCLUSIONS: FACETS is effective in reducing fatigue severity and increasing fatigue self-efficacy. However, it is difficult to assess the additional cost in terms of cost-effectiveness (ie, cost per QALY) as improvements in fatigue are not reflected in the QALY outcomes, with no significant differences between FACETS and CLP. The strengths of this trial are its pragmatic nature and high external validity. TRIAL REGISTRATION: Current Controlled Trials ISRCTN76517470.
Assuntos
Terapia Cognitivo-Comportamental/economia , Terapia Cognitivo-Comportamental/métodos , Fadiga/economia , Fadiga/reabilitação , Estilo de Vida , Esclerose Múltipla Crônica Progressiva/economia , Esclerose Múltipla Crônica Progressiva/reabilitação , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/reabilitação , Psicoterapia de Grupo/economia , Psicoterapia de Grupo/métodos , Adulto , Idoso , Terapia Combinada , Análise Custo-Benefício , Avaliação da Deficiência , Fadiga/diagnóstico , Feminino , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Esclerose Múltipla Crônica Progressiva/diagnóstico , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Autoeficácia , Medicina Estatal/economia , Inquéritos e QuestionáriosRESUMO
PURPOSE OF REVIEW: The aim of this review is to summarize unmet needs for patients with multiple sclerosis (MS). It is important to understand the current status of these patients and both the benefits and limitations of the most commonly used MS treatments as new medications with the potential to simplify therapy and improve outcomes may soon be available. RECENT FINDINGS: Current treatments for MS decrease the frequency of relapses and slow progressive disability. However, nearly all of these medications require frequent administration, and some patients also experience side effects. In some patients, adherence to MS treatment may be less than optimal. This may be associated with increased risk for relapses and hospitalizations and higher cost of care. SUMMARY: Healthcare providers involved in the treatment of MS must be aware of the unmet needs of their patients and intervene as needed to improve adherence and/or modify treatment regimens to optimize outcomes.
Assuntos
Necessidades e Demandas de Serviços de Saúde , Esclerose Múltipla Recidivante-Remitente/terapia , Pessoas com Deficiência , Progressão da Doença , Humanos , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/epidemiologia , Esclerose Múltipla Recidivante-Remitente/psicologia , Cooperação do PacienteRESUMO
BACKGROUND: Walking impairment has a major influence on the quality of life of people with multiple sclerosis (MS). The Multiple Sclerosis Walking Scale (MSWS-12) assesses the impact of MS on walking ability from the patient's perspective, but in its current form, is not amenable for use in many policy decision-making settings. OBJECTIVES: Statistical 'mapping' methods were used to convert MSWS-12 scores to EQ-5D health state values. METHODS: The relationship between the measures was estimated using cohort data from people with MS in South West England. Regression analyses were conducted, estimation errors assessed, and predictive performance of the best models tested using longitudinal data. RESULTS: Model performance was in line with that of other mapping studies, with the best-performing models being an ordinary least squares (OLS) model using MSWS-12 item scores, and an OLS model using the total MSWS-12 score and its squared term. CONCLUSIONS: A process has been described whereby data from a patient-reported outcome measure (MSWS-12) can be converted to (EQ-5D) health state values. These values may be used to consider the health-related quality of life of people with MS, to estimate quality adjusted life-years for use in effectiveness and cost-effectiveness analyses, and to inform health policy decisions.
Assuntos
Avaliação da Deficiência , Política de Saúde , Nível de Saúde , Esclerose Múltipla Crônica Progressiva/diagnóstico , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Formulação de Políticas , Medicina Estatal/legislação & jurisprudência , Caminhada , Adolescente , Adulto , Idoso , Algoritmos , Análise Custo-Benefício , Inglaterra , Feminino , Custos de Cuidados de Saúde , Política de Saúde/economia , Pesquisa sobre Serviços de Saúde , Humanos , Análise dos Mínimos Quadrados , Masculino , Pessoa de Meia-Idade , Modelos Estatísticos , Esclerose Múltipla Crônica Progressiva/economia , Esclerose Múltipla Crônica Progressiva/fisiopatologia , Esclerose Múltipla Crônica Progressiva/psicologia , Esclerose Múltipla Crônica Progressiva/terapia , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/fisiopatologia , Esclerose Múltipla Recidivante-Remitente/psicologia , Esclerose Múltipla Recidivante-Remitente/terapia , Valor Preditivo dos Testes , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Índice de Gravidade de Doença , Medicina Estatal/economia , Fatores de Tempo , Adulto JovemRESUMO
BACKGROUND AND PURPOSE: Data on disease burden of multiple sclerosis from Eastern-Central Europe are very limited. Our aim was to explore the quality of life, resource utilisation and costs of ambulating patients with multiple sclerosis in Hungary. METHODS: Cross-sectional questionnaire survey was performed in two outpatient neurology centres in 2009. Clinical history, health care utilisation in the past 12 months were surveyed, the Expanded Disability Status Scale and the EQ-5D questionnaires were applied. Cost calculation was conducted from the societal perspective. RESULTS: Sixty-eight patients (female 70.6%) aged 38.0 (SD 9.1) with disease duration of 7.8 (SD 6.7) years were involved. Fifty-five (80.9%) had relapsing-remitting form and 52 (76.5%) were taking immunomodulatory drug. The average scores were: Expanded Disability Status Scale 1.9 (SD 1.7), EQ-5D 0.67 (SD 0.28). Mean total cost amounted to 10 902 Euros/patient/year (direct medical 67%, direct nonmedical 13%, indirect costs 20%). Drugs, disability pension and informal care were the highest cost items. Costs of mild (Expanded Disability Status Scale 0-3.5) and moderate (Expanded Disability Status Scale 4.0-6.5) disease were 9 218 and 17 634 Euros/patient/year respectively (p<0.01), that is lower than results from Western European countries. CONCLUSION: Our study provides current inputs for policy making and contributes to understanding variation of cost-of-illness of multiple sclerosis in Europe.
Assuntos
Assistência Ambulatorial/economia , Efeitos Psicossociais da Doença , Custos de Cuidados de Saúde , Recursos em Saúde/economia , Nível de Saúde , Esclerose Múltipla/economia , Pacientes Ambulatoriais , Qualidade de Vida , Adulto , Idoso , Estudos Transversais , Pessoas com Deficiência , Custos de Medicamentos , Feminino , Recursos em Saúde/estatística & dados numéricos , Humanos , Hungria , Imunomodulação , Masculino , Pessoa de Meia-Idade , Esclerose Múltipla Recidivante-Remitente/economia , Pacientes Ambulatoriais/estatística & dados numéricos , Pensões , Índice de Gravidade de Doença , Inquéritos e QuestionáriosRESUMO
OBJECTIVE: Although the global rate of multiple sclerosis (MS) is low, a few studies have documented high costs. Costs are highly variable depending on MS stage. This study was designed to assess the economic burden of Medicare-eligible patients by MS type in the United States using a claims-based classification algorithm to examine cost variation by disease stage. METHODS: A sample of 2003 to 2006 Medicare patients was selected. Cases were classified as pre-existing progressive MS or pre-existing relapsing-remitting MS (RRMS); the latter were further subdivided into relapsing, remitting, or stable. RESULTS: The sample had 5044 MS subjects, of whom 34.4% had prevalent progressive MS and 65.6% had prevalent RRMS. There were many chronic, comorbid conditions. The mean all-cause Medicare expenditures (not including self-administered medications) per person-year for MS in 2006 were $23,630 for prevalent progressive patients and $5887 for prevalent RRMS patients. Within the RRMS type, Medicare expenditures per person per month in 2006 were $1418 for relapsing patients, $608 for remitting patients, and $331 for stable patients. CONCLUSIONS: There are substantial cost advantages to Medicare for keeping RRMS patients in a stable health state and in keeping them from advancing in disability severity. The overall cost advantage would be diminished by the large cost burden of comorbidity, which would likely remain fixed with improved MS therapies.
Assuntos
Custos de Cuidados de Saúde , Medicare/economia , Esclerose Múltipla Crônica Progressiva/economia , Esclerose Múltipla Recidivante-Remitente/economia , Adulto , Idoso , Feminino , Gastos em Saúde , Nível de Saúde , Humanos , Masculino , Medicare/estatística & dados numéricos , Pessoa de Meia-Idade , Modelos Econométricos , Análise Multivariada , Análise de Regressão , Estados UnidosRESUMO
Importance: Pediatric-onset multiple sclerosis (PoMS) is associated with significant cognitive and physical disability. Whether this disability translates into differences in educational achievements and earnings is unknown. Objective: To evaluate the association between PoMS and educational level and income throughout adulthood. Design, Setting, and Participants: A prospective register-based cohort study of individuals with PoMS and a population-based matched reference cohort was conducted using nationwide microdata from linked registers in Sweden from January 1, 1990, to December 31, 2016; analyses were completed from May 1, 2019, to September 1, 2020. Of 772 persons with PoMS identified in the Swedish MS registry, 485 had an onset during the period from 1980 to 2014 and had socioeconomic data available. The general population reference cohort without multiple sclerosis (MS) (n = 4850) was randomly selected from the full Swedish population, matched 10:1 on age, sex, and country of birth. Exposure: Pediatric-onset MS, diagnosed by a neurologist, with onset before 18 years of age. Main Outcomes and Measures: Highest educational level (elementary school, high school, or university) was assessed using logistic regression. Income, measured as the mean annual earnings from paid work in US dollars, was compared using Tobit models, and net annual sickness absence and disability pension days were compared using zero-inflated negative binomial regression. Earnings and days receiving disability benefits were compared within 4 age periods (19-24, 25-34, 35-44, and 45-54 years). Results: The median age of the cohort with PoMS (n = 485) and the matched reference cohort (n = 4850) in 2016 was 32 years (interquartile range, 26-40 years), and most participants were women (348 [71.8%] in the PoMS cohort and 3480 [71.8%] in the matched reference cohort). Persons with PoMS were less likely than persons in the matched reference cohort to attend university (odds ratio, 0.80 [95% CI, 0.66-0.97]) and had significantly lower annual earnings than the reference cohort, ranging from -$1618 (95% CI, -$2558 to -$678) in the youngest age period to -$10â¯683 (95% CI, -$18â¯187 to -$3178) in the eldest. Persons with PoMS received higher rates of disability benefits, as sickness absence days in the youngest age period (rate ratio, 3.06 [95% CI, 2.08-4.52]) and disability pension days in the oldest age period (rate ratio, 1.43 [95% CI, 1.11-1.85]). Conclusions and Relevance: This study suggests that having PoMS is associated with less educational achievement, lower earnings, and greater use of disability benefits throughout the working-age life span. As adults, persons with PoMS never earned as much as their counterparts without MS, and they exhibited a heavier reliance on disability benefits.
Assuntos
Pessoas com Deficiência , Escolaridade , Renda/tendências , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/epidemiologia , Fatores Socioeconômicos , Adulto , Idade de Início , Criança , Estudos de Coortes , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Sistema de Registros , Suécia/epidemiologia , Adulto JovemRESUMO
OBJECTIVE: The prevailing approaches to selecting multiple sclerosis (MS) disease modifying therapies (DMTs) have contributed to exponential increases in societal expenditures and out-of-pocket expenses, without compelling evidence of improved outcomes. Guidance is lacking regarding when and in whom the benefits of preventing MS-related disability likely outweighs the risks of highly effective DMTs (HET) and when it is appropriate to consider DMT costs. Our objective was to develop a standardized approach to improve the quality, affordability and equity of MS care. METHODS: MS experts partnered with health plan pharmacists to develop an ethical, risk-stratified, cost-sensitive treatment algorithm. We developed a risk-stratification schema to classify patients with relapsing forms of MS as high, intermediate or low risk of disability based on the best available evidence and, when the evidence was poor or lacking, by consensus. DMTs are grouped as highly, modestly or low/uncertain effectiveness and preferentially ranked within groups by safety based on pre-specified criteria. We reviewed FDA documents and the published literature. When efficacy and safety are equivalent, the lower cost DMT is preferred. RESULTS: Assignment to the high-risk group prompts treatment with preferred HETs early in the disease course. For persons in the intermediate- or low-risk groups with cost or health care access barriers, we incorporated induction therapy with an affordable B-cell depleting agent. Based on more favorable safety profiles, our preferred approach prioritizes use of rituximab and natalizumab among HETs and interferon-betas or glatiramer acetate among modestly effective agents. INTERPRETATION: The risk-stratified treatment approach we recommend provides clear, measurable guidance in whom and when to prescribe HETs, when to prioritize lower cost DMTs and how to accommodate persons with MS with cost or other barriers to DMT use. It can be adapted to other cost structures and updated quickly as new information emerges. We recommend that physician groups partner with health insurance plans to adapt our approach to their settings, particularly in the United States. Future studies are needed to resolve the considerable uncertainty about how much variability in prognosis specific risk factors explain.
Assuntos
Acessibilidade aos Serviços de Saúde , Agentes de Imunomodulação/economia , Esclerose Múltipla Recidivante-Remitente , Melhoria de Qualidade , Acessibilidade aos Serviços de Saúde/economia , Acessibilidade aos Serviços de Saúde/normas , Humanos , Esclerose Múltipla Recidivante-Remitente/classificação , Esclerose Múltipla Recidivante-Remitente/diagnóstico , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/terapia , Prognóstico , Melhoria de Qualidade/economia , Melhoria de Qualidade/normas , Medição de RiscoRESUMO
BACKGROUND: As healthcare management of highly active-relapsing-remitting multiple sclerosis (HA-RRMS) patients is more complex than for the whole multiple sclerosis (MS) population, this study assessed the related economic burden from a National Health Insurance's (NHI's) perspective. RESEARCH DESIGN AND METHODS: Study based on French NHI databases, using individual data on billing and reimbursement of outpatient and hospital healthcare consumption, paid sick leave and disability pension, over 2010-2017. RESULTS: Of the 9,596 HA-RRMS adult patients, data from 7,960 patients were analyzed with at least 2 years of follow-up. Mean annual cost/patient was 29,813. Drugs represented 40% of the cost, hospital care 33%, disability pensions 9%, and all healthcare professionals' visits combined 8%. Among 3,024 patients under 60 years-old with disability pension, disability pension cost 7,168/patient/year. Among 3,807 patients with paid sick leave, sick leave cost 1,956/patient/year. Mean costs were 2,246/patient higher the first year and increased by 1,444 between 2010 and 2015, with a 5,188 increase in drug-related expenditures and a 634 increase in healthcare professionals' visits expenditures but a 4,529 decrease in hospital care expenditures. CONCLUSIONS: The cost of health care sick leaves, and disability pensions of HA-RRMS patients was about twice as high as previously reported cost of MS patients.
Assuntos
Efeitos Psicossociais da Doença , Hospitalização/estatística & dados numéricos , Esclerose Múltipla Recidivante-Remitente/terapia , Esclerose Múltipla/terapia , Adulto , Estudos de Coortes , Bases de Dados Factuais , Atenção à Saúde/economia , Atenção à Saúde/métodos , Pessoas com Deficiência , Feminino , Seguimentos , França , Custos de Cuidados de Saúde/estatística & dados numéricos , Hospitalização/economia , Humanos , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Esclerose Múltipla/economia , Esclerose Múltipla Recidivante-Remitente/economia , Programas Nacionais de Saúde/economia , Pensões/estatística & dados numéricos , Estudos Retrospectivos , Licença Médica/economiaRESUMO
Multiple sclerosis is a leading cause of disability in young adults, with the Scottish population suffering the highest prevalence in Europe. Disease-modifying therapies, including beta-interferon (IFN-ß), are increasingly used to minimise relapse frequency in the majority of patients who present with a relapsing-remitting disease pattern. Unfortunately, neutralising antibodies (NABs) may develop against IFN-ß and are associated with reduced efficacy. These antibodies may be detected using a serum sample. Despite the importance of this problem, from both a patient's perspective and a wider community and economic standpoint, there is no universally agreed protocol for the use of NAB testing. Authorities variously suggest routine 'screening' testing or, conversely, testing only in specific situations. In Scotland, routine testing is seldom used. We report our experience of routine NAB testing in 105 patients (of whom 35 were NAB-positive) over two years in NHS Tayside and comment on its cost and implications.