The Cost Effectiveness of Rufinamide in the Treatment of Lennox-Gastaut Syndrome in the UK

Background: Lennox-Gastaut syndrome (LGS) is a catastrophic childhood form of epilepsy. The syndrome is characterized by mental impairment, frequent seizures of multiple types that are particularly resistant to treatment, and high rates of seizure-related injury. With the introduction of newer, but more costly, antiepileptic drugs (AEDs), it is important that decision makers are able to assess their value in the management of this rare and difficult-to-treat condition. Objective: To evaluate the cost effectiveness, from the UK NHS perspective, of rufinamide in patients with LGS. Methods: An individual patient-simulation model was developed to estimate the total treatment-related costs and clinical benefits of rufinamide compared with topiramate and lamotrigine over a 3-year time horizon. The model examines the treatment scenarios of adding rufinamide, lamotrigine or topiramate to older AEDs (standard therapy), or standard therapy alone within a primary-care or community setting. Three placebo-controlled clinical trials of adjunctive AED treatment for children with LGS were analysed. There are no head-to-head comparator studies. Between 98 and 139 patients were randomized in each study and the mean age in each study was 10, 11 and 14 years. A mixed-treatment comparison using a random-effects model was carried out on the number of patients in each response category, using the placebo arms of the respective trials. The primary outcome measure was the percentage of successfully treated patients, defined as >50% reduction in the frequency of total seizures and drop attacks. The hypothesis being tested was formulated after data collection. Costs (£, year 2006/07 values) of patient monitoring, switching treatments, hospitalization due to seizure, treatment of adverse effects, and personal and social services were included in the analysis. Results of 10 000 Monte Carlo simulations were bootstrapped to conduct probabilistic sensitivity analysis. Results: Over 3 years, adjunctive rufinamide resulted in higher total costs than topiramate and lamotrigine; however, with more patients being treated successfully, this leads to acceptable incremental cost-effectiveness ratios. If society is prepared to pay at least £250 for a 1% increase in the number of successfully treated LGS patients, in terms of a 50% reduction in the frequency of drop attacks, the probability of the treatment with rufinamide being cost effective is >80%. Conclusion: This cost-effectiveness analysis suggests that rufinamide results in more LGS patients being treated successfully at a reasonable cost from a UK NHS perspective.Antiepileptic-drugs, therapeutic use, Children, Cost-effectiveness, Lamotrigine, therapeutic use, Lennox-Gastaut-syndrome, treatment, Rufinamide, therapeutic use, Topiramate, therapeutic use

The efficacy and tolerability of perampanel and other recently approved anti-epileptic drugs for the treatment of refractory partial onset seizure: a systematic review and Bayesian network meta-analysis.

OBJECTIVES: This paper compares the efficacy and tolerability of perampanel (PER) relative to other recently approved anti-epileptic drug (AEDs) - lacosamide (LCS), retigabine (RTG), and eslicarbazepine (ESL) for the adjunctive treatment of partial onset seizures with or without secondary generalization and specifically in the secondary generalization subgroup. MATERIALS AND METHODS: A systematic literature review of all RCTs of PER and selected AEDs in EMBASE, Medline, and the Cochrane Central from 1998 to January 2011 with an update in PubMed in March 2013 was performed. A network meta-analysis was conducted for 50% responder rate for overall seizures; withdrawal due to adverse events; seizure freedom; and 50% responder rate for secondary generalized seizures. RESULTS: Twelve RCTs (three PER, three LCS, three RTG and three ESL) were included. PER performed significantly better than placebo for 'responder rate' (OR 2.151, 95% CrI 1.348-3.472) and 'seizure freedom' (OR 2.507, 95% CrI 1.067-7.429). When compared to other agents, PER was found to be equally effective. For 'withdrawal due to adverse events', PER had the lowest odds ratio vs. placebo compared with other AEDs. In the analysis for the subgroup of patients with secondary generalization, only four RCTs (three PER and one LCS) met the inclusion criteria for one outcome (responder rate) for LCS. In this subgroup, PER was statistically significantly better than placebo (OR 2.448, 95% CrI 1.088-5.828). CONCLUSION: PER was statistically significantly superior to placebo in responder rate, seizure freedom, and responder rate in the secondary generalization population. Though PER had statistically significant greater withdrawal compared to placebo, it had the lowest ORs vs. placebo, suggesting a superior safety profile among the comparators included in this analysis. In patients with partial onset seizure with secondary generalization, PER had a statistically significant effect on responder rate compared to placebo

La carga económica de las condiciones cardíacas en Venezuela

En Venezuela, las condiciones cardíacas imponen limitaciones físicas, sociales, financieras y de salud relacionadas con la calidad de vida de los individuos. Objetivos: Este estudio valoró la carga económica de cuatro condiciones cardíacas en Venezuela: hipertensión, insuficiencia cardíaca, infarto de miocardio y fibrilación auricular. Adicionalmente se evaluó el costo-efectividad de la telemedicina y el soporte telefónico estructurado para el manejo de la insuficiencia cardíaca. Métodos: Se utilizó un marco de costo de enfermedad estándar para valorar los costos asociados con las cuatro condiciones en 2015. El análisis evaluó la prevalencia e (en caso de infarto de miocardio) incidencia de las condiciones, los gastos asociados con el tratamiento médico, las pérdidas de productividad por reducción en el empleo, los costos de proveer cuidado formal e informal y la pérdida de bienestar. El análisis estuvo basado en información recolectada mediante una revisión de literatura y estadísticas, y modulación de datos. Todas las entradas de datos y métodos fueron validados mediante la consulta de quince clínicos y expertos en Venezuela. El análisis de costo-efectividad fue basado en un meta-análisis y en una evaluación económica de programas para pacientes con insuficiencia cardíaca dados de alta, valorado desde la perspectiva del Programa Nacional de Salud. Resultados: El infarto de miocardio impone el mayor costo financiero (3,5 millo- nes de bolívares/553 millones de USD), seguido por hipertensión arterial (3,4 millones de bolívares/539 millones de USD), la insuficiencia cardíaca (3,3 millones debolívares/522 millones de USD) y, finalmente, fibrilación auricular (0,4 miles de millones de bolívares/66 millones de USD). La telemedicina y el soporte telefónico estructurado son intervenciones costo-efectivas para alcanzar mejoras en el manejo de la insuficiencia cardíaca. Conclusiones: Las condiciones cardíacas imponen una pérdida sustancial de bienestar y de costos financieros en Venezuela y deberían ser una prioridad de salud pública.Heart conditions impose physi- cal, social, financial and health related quality of life limitations on individuals in Venezuela. Objectives: This study assessed the economic burden of four main heart conditions in Venezuela: hypertension, heart failure, myocardial infarction, and atrial fibrillation. In addition, the cost-effectiveness of telemedicine and structured telephone support for the management of heart failure was assessed. Methods: A standard cost of illness framework was used to assess the costs associated with the four conditions in 2015. The analysis was informed by a targeted literature review, data scan and modeling. All inputs and methods were validated by consulting fifteen clinicians and other stake-holders in Venezuela. The cost-effectiveness analysis was based on a meta-analysis and economic evaluation of post-discharge programs in patients with heart failure, assessed from the perspective of the National Health Fund. Results: Myocardial infarction imposes the greatest financial cost (3.5 million bolivares/553 million USD), followed by hypertension (3.4 million bolivares/539 million USD), heart failure (3.3 million bolivares/522 million USD) and, finally, atrial fibrillation (0.4 billion bolivares/66 million USD).Telemedicine and structured telephone support are cost effective interventions for achieving improvements in the management of heart failure. The analysis assessed the prevalence and (in the case of myocardial infarction) incidence of the conditions, the associated expenditures on health care treatment, productivity losses from reduced employment, costs of providing formal and informal care, and lost well- being. Conclusions: Heart conditions impose substantial loss of wellbeing and financial costs in Venezuela and should be a public health priority.&nbsp

The Economic Burden of Heart Conditions in Brazil

Abstract Background: Heart conditions impose physical, social, financial and health-related quality of life limitations on individuals in Brazil. Objectives: This study assessed the economic burden of four main heart conditions in Brazil: hypertension, heart failure, myocardial infarction, and atrial fibrillation. In addition, the cost-effectiveness of telemedicine and structured telephone support for the management of heart failure was assessed. Methods: A standard cost of illness framework was used to assess the costs associated with the four conditions in 2015. The analysis assessed the prevalence of the four conditions and, in the case of myocardial infarction, also its incidence. It further assessed the conditions’ associated expenditures on healthcare treatment, productivity losses from reduced employment, costs of providing formal and informal care, and lost wellbeing. The analysis was informed by a targeted literature review, data scan and modelling. All inputs and methods were validated by consulting 15 clinicians and other stakeholders in Brazil. The cost-effectiveness analysis was based on a meta-analysis and economic evaluation of post-discharge programs in patients with heart failure, assessed from the perspective of the Brazilian Unified Healthcare System (Sistema Unico de Saude). Results: Myocardial infarction imposes the greatest financial cost (22.4 billion reais/6.9 billion USD), followed by heart failure (22.1 billion reais/6.8 billion USD), hypertension (8 billion reais/2.5 billion USD) and, finally, atrial fibrillation (3.9 billion reais/1.2 billion USD). Telemedicine and structured telephone support are cost-effective interventions for achieving improvements in the management of heart failure. Conclusions: Heart conditions impose substantial loss of wellbeing and financial costs in Brazil and should be a public health priority

Lennox-Gastaut Syndrome (LGS): development of conceptual models of health-related quality of life (HRQL) for caregivers and children

LGS is a severe form of childhood epilepsy which is characterized by multiple seizures and cognitive impairment. Semi-structured interviews were conducted with 40 parents of children with LGS in the US, UK, and Italy. Parents were asked to report on their perceptions of the HRQL of their child and also to describe the impact on their own HRQL. Thematic analysis was conducted to develop themes relating to the impact on HRQL. The themes were organized into conceptual models of the impact of LGS on the HRQL of the parent and the child. The models demonstrate the complex relationships between the components of LGS and their impact on HRQL