RESUMEN

AIM: Our study aimed to evaluate the cost-effectiveness of the chimeric antigen receptor (CAR) T-cell therapy, axicabtagene ciloleucel (axi-cel), compared to standard of care (SOC) in Sweden for second-line (2L) treatment of adult transplant-intended diffuse large B-cell lymphoma (DLBCL) patients who relapse within 12 months from completion of, or are refractory to (early r/r), first-line (1L) chemoimmunotherapy. METHODS: Cost-effectiveness was assessed using a three-state partitioned survival model. Mixture cure models were used to extrapolate time-to-event data from the ZUMA-7 trial (NCT03391466) beyond the observational period. Sensitivity and scenario analyses were performed to test the robustness of the base case results, including an analysis that assumed no switching to off-protocol CAR T-cell therapy in subsequent lines in the SOC arm. RESULTS: The model estimated an incremental cost-effectiveness ratio (ICER) of SEK 534,704 (EUR 50,303) per quality-adjusted life year (QALY) gained over a lifetime horizon of 50 years, with an incremental cost of SEK 812,944 (EUR 76,479) and incremental QALY of 1.52 for axi-cel compared with SOC. The probabilistic sensitivity analysis showed that axi-cel was cost-effective in 73% of the simulations when assuming a willingness-to-pay threshold of SEK 1,000,000 (EUR 94,077) per QALY. The ICER was SEK 694,351 (EUR 65,313) in the scenario analysis where the costs and effects of treatment switching were not included. CONCLUSION: 2L treatment with axi-cel in transplant-intended DLBCL patients with early r/r after completing 1L chemoimmunotherapy was cost-effective compared to SOC in a Swedish setting. Administering axi-cel in 2L is cost-effective as it enhances the possibility of curing more patients, resulting in not just a survival advantage, but also a reduction in the burden on quality of life and cost of subsequent therapy. This will be advantageous to both patients and society.

Asunto(s)

Productos Biológicos , Linfoma de Células B Grandes Difuso , Adulto , Humanos , Análisis de Costo-Efectividad , Inmunoterapia Adoptiva , Linfoma de Células B Grandes Difuso/tratamiento farmacológico , Recurrencia Local de Neoplasia , Calidad de Vida , Nivel de Atención , Suecia , Ensayos Clínicos como Asunto

RESUMEN

BACKGROUND: There is limited real-world data on the economic burden of patients with autosomal dominant polycystic kidney disease (ADPKD). The objective of this study was to estimate the annual direct and indirect costs of patients with ADPKD by severity of the disease: chronic kidney disease (CKD) stages 1-3; CKD stages 4-5; transplant recipients; and maintenance dialysis patients. METHODS: A retrospective study of ADPKD patients was undertaken April-December 2014 in Denmark, Finland, Norway and Sweden. Data on medical resource utilisation were extracted from medical charts and patients were asked to complete a self-administered questionnaire. RESULTS: A total of 266 patients were contacted, 243 (91%) of whom provided consent to participate in the study. Results showed that the economic burden of ADPKD was substantial at all levels of the disease. Lost wages due to reduced productivity were large in absolute terms across all disease strata. Mean total annual costs were highest in dialysis patients, driven by maintenance dialysis care, while the use of immunosuppressants was the main cost component for transplant care. Costs were twice as high in patients with CKD stages 4-5 compared to CKD stages 1-3. CONCLUSIONS: Costs associated with ADPKD are significant and the progression of the disease is associated with an increased frequency and intensity of medical resource utilisation. Interventions that can slow the progression of the disease have the potential to lead to substantial reductions in costs for the treatment of ADPKD.

Asunto(s)

Trasplante de Riñón/economía , Riñón Poliquístico Autosómico Dominante/economía , Diálisis Renal/economía , Insuficiencia Renal Crónica/economía , Costo de Enfermedad , Costos y Análisis de Costo , Estudios Transversales , Dinamarca/etnología , Progresión de la Enfermedad , Femenino , Finlandia/etnología , Gastos en Salud , Recursos en Salud/economía , Humanos , Masculino , Persona de Mediana Edad , Noruega/etnología , Riñón Poliquístico Autosómico Dominante/etnología , Insuficiencia Renal Crónica/etnología , Estudios Retrospectivos , Suecia/etnología , Receptores de Trasplantes

RESUMEN

BACKGROUND: A limited number of studies have assessed health-related quality of life (HRQoL) in autosomal dominant polycystic kidney disease (ADPKD). Results to date have been conflicting and studies have generally focused on patients with later stages of the disease. This study aimed to assess HRQoL in ADPKD across all stages of the disease, from patients with early chronic kidney disease (CKD) to patients with end-stage renal disease. METHODS: A study involving cross-sectional patient-reported outcomes and retrospective clinical data was undertaken April-December 2014 in Denmark, Finland, Norway and Sweden. Patients were enrolled into four mutually exclusive stages of the disease: CKD stages 1-3; CKD stages 4-5; transplant recipients; and dialysis patients. RESULTS: Overall HRQoL was generally highest in patients with CKD stages 1-3, followed by transplant recipients, patients with CKD stages 4-5 and patients on dialysis. Progressive disease predominately had an impact on physical health, whereas mental health showed less variation between stages of the disease. A substantial loss in quality of life was observed as patients progressed to CKD stages 4-5. CONCLUSIONS: Later stages of ADPKD are associated with reduced physical health. The value of early treatment interventions that can delay progression of the disease should be considered.

Asunto(s)

Riñón Poliquístico Autosómico Dominante/terapia , Calidad de Vida , Diálisis Renal , Anciano , Estudios Transversales , Progresión de la Enfermedad , Femenino , Humanos , Masculino , Persona de Mediana Edad , Estudios Retrospectivos

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OBJECTIVES: Tiotropium (TIO), Spiriva® Handihaler®, is a well-established bronchodilator, LAMA (long acting muscarinic antagonist), for the treatment of moderate to very severe chronic obstructive pulmonary disease (COPD). Clinical evidence from the SPARK trial suggests that TIO is superior to glycopyrronium (GLY), Seebri® Breezhaler®, in terms of severe exacerbations. This modeling study assessed the cost-effectiveness of TIO versus GLY for Canada (CAN), Spain (ESP), Sweden (SWE), and the UK, making use of this new clinical evidence. METHODS: A Markov cohort model, with moderate to very severe (Global Initiative for Chronic Obstructive Lung Disease II-IV) COPD patients, was populated with efficacy data from the Understanding Potential Long-term Impacts on Function with Tiotropium (UPLIFT) and SPARK trials as well as costs, utilities, and epidemiological data relevant for each country. Treatment efficacy was modeled as improvements in lung function, quality-adjusted life years (QALYs), and as a lowering of the risk of exacerbations (rate of exacerbations). Risks of exacerbations differed between cohorts based on data from SPARK. Health and cost outcomes were simulated over an approximate lifetime horizon, starting from the age of 65 years. Robustness of results was validated in deterministic sensitivity analyses. RESULTS: Over the lifetime horizon, patients treated with TIO accumulated -623 (CAN), 1,066 (ESP), 1,137 (SWE), and -169 (UK), respectively, in incremental costs (€2014). TIO generated better health outcomes compared to GLY in all countries, 0.21 (CAN), 0.25 (ESP), 0.23 (SWE), and 0.23 (UK) in incremental QALYs. The cost per QALY gained was found to be €4,281 and €1,137 for ESP and SWE, respectively, while TIO was found to be cost saving in CAN and the UK. The results were mainly driven by the relative risk of severe exacerbations found in SPARK (GLY/TIO relative risk: 1.43, 95% confidence interval: 1.05-1.97, P=0.025). CONCLUSION: The results from this study show that TIO is a cost-effective treatment compared to GLY in moderate to very severe COPD. The cost per QALY is well below the existing implicit and explicit willingness-to-pay thresholds.

RESUMEN

BACKGROUND: Tiotropium (TIO) is a well-established bronchodilator, LAMA (long-acting anticholinergic), for the treatment of moderate to very severe chronic obstructive pulmonary disease (COPD). Clinical evidence suggests that tiotropium is superior to usual non-LAMA care (UC) but may also have benefits compared to other LAMAs in preventing and limiting the effects of severe exacerbations. The primary objective of this study was to undertake a cost-effectiveness analysis of adding tiotropium to usual care versus usual care alone. A secondary objective was to assess the cost-effectiveness of tiotropium compared to glycopyrronium (GLY), another LAMA. The study was conducted with a Swedish setting in mind. METHODS: A Markov cohort model, incorporating the effects of exacerbations, was populated with efficacy data from the UPLIFT and SPARK trials and epidemiological data relevant for a Swedish patient population. Treatment efficacy of tiotropium was modelled as a lowering of the risk of exacerbations and as a slow-down of overall disease progression. The model followed patients over their remaining life-time. RESULTS: The base case analysis showed that patients treated with tiotropium gained 0.07 quality-adjusted life years (QALYs) compared to usual care alone at an incremental cost of SEK 15,041, resulting in a cost per QALY gained of SEK 224,850. Compared to glycopyrronium the QALY gained was estimated to 0.23 QALYs in favour of tiotropium at an incremental cost of SEK 2423, yielding a cost per QALY gained of SEK 10,456. The results were mainly driven by differences in the risk of severe exacerbations. CONCLUSION: At the current implicit willingness-to-pay (WTP) per QALY threshold in Sweden, the results from this study indicate that tiotropium is a highly cost-effective intervention when added to usual non-LAMA care in the treatment of moderate to very severe COPD in Sweden. In addition, tiotropium is a highly cost-effective intervention when compared to glycopyrronium monotherapy.