RESUMEN
The purpose of this work is to estimate the costs associated with managing patients with MS in Panama and evaluating the impact of the disease on their health-related quality of life (HRQoL). Multicentric observational, retrospective, cross-sectional study. The costs were estimated from societal and patient perspectives and expressed in USD, 2015. The focus of the study is based on prevalence and on a "bottom-up" approach. To estimate the total cost per patient, annual reported use for each resource was multiplied by its unit cost. To evaluate HRQoL, patients completed the EQ-5D-3L questionnaire. 108 patients took part in the study. 82.41% were women with 44.78 (SD: 12.27) years. 61.11% presented mild (EDSS = 0-3.5), 25.93% moderate (EDSS = 3.5-6) and 12.96%, severe disability (EDSS≥6.5). The mean annual cost from the patient's perspective was estimated at 777.99 USD (SD: 1,741.45) per patient. The mean cost from a societal perspective was estimated at 23,803.21 USD (SD: 13,331.83) per patient. Disease-modifying therapies (DMT) accounted for the main component of the cost. A deterioration in HRQoL was observed as the disease advances and as disability increases, with mobility and usual activities being the areas most affected by its progression. From both perspective, the cost per MS patient in Panama is high. In addition to the high economic impact, MS also exerts a negative impact on patient HRQoL, which increases as the disease advances.
Asunto(s)
Costo de Enfermedad , Esclerosis Múltiple/economía , Adulto , Estudios Transversales , Progresión de la Enfermedad , Femenino , Costos de la Atención en Salud , Humanos , Masculino , Persona de Mediana Edad , Esclerosis Múltiple/epidemiología , Esclerosis Múltiple/terapia , Panamá/epidemiología , Calidad de Vida , Estudios Retrospectivos , Encuestas y CuestionariosRESUMEN
BACKGROUND: Multiple myeloma is a hematologic malignancy affecting bone marrow derived plasma cells. Current therapies are not able to eradicate the disease and most patients become refractory to the treatment. Lenalidomide and bortezomib have proved effective in the second-line treatment of these patients. OBJECTIVE: To evaluate the cost-effectiveness of lenalidomide in combination with dexamethasone compared to bortezomib in combination with dexamethasone in patients with multiple myeloma previously treated with bortezomib, from the perspective of the Chilean National Health Service. METHODOLOGY: A four-state Markov model (preprogression on treatment; preprogression off treatment, progression and death) was used to simulate the evolution of a cohort of multiple myeloma patients over a 25-year time horizon. Efficacy data, resource use and frequency of adverse events were extracted from MM009/010 studies and a retrospective analysis of retreatment with bortezomib. All inputs were validated by experts. A 3% annual discount rate was used for costs and health outcomes. The robustness of the results was evaluated through univariate and probabilistic sensitivity analyses. RESULTS: Lenalidomide in combination with dexamethasone treatment provided 1.41 incremental life years and 0.83 incremental quality-adjusted life years in comparison with bortezomib in combination with dexamethasone, with an incremental cost of 11 864 597.86 CLP (19 589.86 US$). The incremental cost-effectiveness and cost-utility ratio were estimated at 8 410 266.92 CLP (13 886,35 US$) / incremental life year and 14 271 896.16 CLP (23 564,59 US$)/incremental quality-adjusted life years, respectively. CONCLUSIONS: Lenalidomide in combination with dexamethasone represents a potentially cost-effective alternative for the second-line treatment of patients with multiple myeloma who are not eligible for transplantation, from the perspective of the Chilean National Health Service.
CONTEXTO: El mieloma múltiple es una neoplasia de las células plasmáticas de la medula ósea. Las terapias disponibles no son curativas y la mayoría de los pacientes se vuelve refractario al tratamiento. Agentes como lenalidomida y bortezomib han demostrado su eficacia en el tratamien-to en segunda línea de estos pacientes. OBJETIVO: Evaluar el costo-efectividad de la combinación lenalidomida/dexametasona frente a bortezomib/dexametasona en pacientes con mieloma múltiple, no candidatos a trasplante, previamente tratados con bortezomib, desde la perspectiva del sistema nacional de salud chileno. METODOLOGÍA: Se empleó un modelo de Markov que simula la evolución de una cohorte de pacientes a través de cuatro estados de salud (preprogresión en tratamiento, preprogresión sin tratamiento, progresión o muerte) en un horizonte temporal de 25 años. Los datos de eficacia, uso de recursos y frecuencia de efectos adversos fueron extraídos de los ensayos sobre mieloma múltiple MM-009 y MM-010 y de un estudio retrospectivo de retratamiento con bortezomib. Todos los parámetros fueron validados por expertos. Se aplicó una tasa de descuento en costos y beneficios de 3%. La robustez de los resultados fue evaluada mediante un análisis de sensibilidad univariante y probabilístico. RESULTADOS: El tratamiento con lenalidomida/dexametasona proporciona 1,41 años de vida y 0,83 años de vida ajustados por calidad incrementales respecto a bortezomib/dexametasona, con un costo incremental de 11 864 597,86 pesos chilenos (19 589,86 dólares). La ratio de cos-to-efectividad y costo-utilidad incremental se cifró en 8 410 266,92 pesos chilenos (13 886,35 dólares) por año de vida ganado y 14 271 896,16 pesos chilenos (23 564,59 dólares) por año de vida ajustado por calidad respectivamente. CONCLUSIÓN: La lenalidomida/dexametasona representa una alternativa potencialmente costo-efectiva, desde la perspectiva del sistema nacional de salud chileno, para el tratamiento en segunda línea de pacientes con mieloma múltiple no candidatos a trasplante.