Cost-effectiveness analysis of haploidentical vs matched unrelated allogeneic hematopoietic stem cells transplantation in patients older than 55 years.

Due to limited donor availability, high comorbidities, and cost issues, allogeneic hematopoietic stem cell transplant is not universally accessible. The aim of this study was to conduct a cost-effectiveness analysis of haploidentical vs matched unrelated transplant. This retrospective study included patients with hematological malignancies older than 55 years who underwent haploidentical or matched unrelated transplant between 2011 and 2013 in Marseille. The incremental cost-effectiveness ratio has been calculated using the mean overall survival and the mean transplant costs. Costs were calculated using a micro-costing strategy from the hospital perspective and a time horizon at 2 years. Haploidentical transplant was considered an innovative procedure and matched unrelated transplant as the reference. Probabilistic and sensitivity analyses were performed on the incremental cost-effectiveness ratio. During inclusion, 29 patients underwent haploidentical transplant and 63 matched unrelated transplant. In haploidentical and matched unrelated transplant, the mean overall survival was 19.4 (1.6) months and 15.1 (1.2) months (p = 0.06), respectively, and the mean cost was 98,304 (40,872) € and 151,373 (65,742) € (p < 0.01), respectively. The incremental cost-effectiveness ratio was assessed to -148,485 (-1,265,550; -64,368) € per life year gained. Among older patients suffering from hematological malignancies, haploidentical transplant seemed in our analysis to be cost-effective compared with matched unrelated transplant.

Cost effectiveness of pegfilgrastim versus filgrastim after high-dose chemotherapy and autologous stem cell transplantation in patients with lymphoma and myeloma: an economic evaluation of the PALM Trial.

BACKGROUND: Use of the recombinant human granulocyte colony-stimulating factor (rhG-CSF) filgrastim accelerates neutrophil recovery following myelosuppressive chemotherapy. Since filgrastim requires multiple daily administrations, forms of rhG-CSF with a longer half life, including pegfilgrastim, have been developed. Pegfilgrastim is safe and effective in supporting neutrophil recovery and reducing febrile neutropenia after conventional chemotherapy. Pegfilgrastim has also been successfully used to support patients undergoing peripheral blood stem cell (PBSC) transplantation for haematological malignancies. To our knowledge, no cost-effectiveness analysis (CEA) of pegfilgrastim in this setting has been published yet. OBJECTIVE: We undertook a CEA to compare a single injection of pegfilgrastim versus repeated administrations of filgrastim in patients who had undergone PBSC transplantation for lymphoma or myeloma. The CEA was set in France and covered a period of 100 ± 10 days from transplant. METHODS: The CEA was designed as part of an open-label, multicentre, randomized phase II trial. Costs were assessed from the hospital's point of view and are expressed in 2009 euros. Costs computation focused on inpatient, outpatient, and home care. Costs in the two arms of the study were compared using the Mann-Whitney test. When differences were statistically significant, multiple regression analyses were performed in order to identify cost drivers. Incremental cost-effectiveness ratios (ICER) were calculated for the major endpoints of the trial; i.e., duration of febrile neutropenia (absolute neutrophil count [ANC] <0.5 × 10(9)/L and temperature ≥38 °C), duration of neutropenia (ANC <1.0 × 10(9)/L and ANC <0.5 × 10(9)/L), duration of thrombopenia (platelets <50 × 10(9)/L and <20 × 10(9)/L), and days with a temperature ≥38 °C). Uncertainty around the ICER was captured by a probabilistic analysis using a non-parametric bootstrap method. RESULTS: 151 patients were enrolled at ten French centres from October 2008 to September 2009. The mean total cost in the pegfilgrastim arm of the study (n = 74) was 25,024 (SD 9,945). That in the filgrastim arm (n = 76) was 28,700 (SD 20,597). Pegfilgrastim strictly dominated filgrastim for days of febrile neutropenia avoided, days of neutropenia (ANC <1.0 × 10(9)/L) avoided, days of thrombopenia (platelets <20 × 10(9)/L) avoided, and days with temperature ≥38 °C) avoided. Pegfilgrastim was less costly and less effective than filgrastim for the number of days with ANC <0.5 × 10(9)/L avoided and the number of days with platelets <50.0 × 10(9)/L avoided. Taking uncertainty into account, the probabilities that pegfilgrastim strictly dominated filgrastim were 67 % for febrile neutropenia, 86 % for neutropenia (ANC <1.0 × 10(9)/L), 59 % for thrombopenia (platelets <20 × 10(9)/L), 86 % for temperature ≥38 °C, 32 % for neutropenia (ANC <0.5 × 10(9)/L), and 43 % for thrombopenia (platelets <50 × 10(9)/L). Conversely, the probability that filgrastim strictly dominated pegfilgrastim for neutropenia (ANC <0.5 × 10(9)/L) is 5 %. CONCLUSION: This study found no evidence that the use of pegfilgrastim is associated with greater cost in lymphoma and myeloma patients after high-dose chemotherapy and PBSC transplantation.

Clinicians' adherence versus non adherence to practice guidelines in the management of patients with sarcoma: a cost-effectiveness assessment in two European regions.

BACKGROUND: Although the management of sarcoma is improving, non adherence to clinical practice guidelines (CPGs) remains high, mainly because of the low incidence of the disease and the variety of histological subtypes. Since little is known about the health economics of sarcoma, we undertook a cost-effectiveness analysis (within the CONnective TIssue CAncer NETwork, CONTICANET) comparing costs and outcomes when clinicians adhered to CPGs and when they did not. METHODS: Patients studied had a histological diagnosis of sarcoma, were older than 15 years, and had been treated in the Rhône-Alpes region of France (in 2005/2006) or in the Veneto region of Italy (in 2007). Data collected retrospectively for the three years after diagnosis were used to determine relapse free survival and health costs (adopting the hospital's perspective and a microcosting approach). All costs were expressed in euros (€) at their 2009 value. A 4% annual discount rate was applied to both costs and effects. The incremental cost-effectiveness ratio (ICER) was expressed as cost per relapse-free year gained when management was compliant with CPGs compared with when it was not. To capture uncertainty surrounding ICER, a probabilistic sensitivity analysis was performed based on a non-parametric bootstrap method. RESULTS: A total of 219 patients were included in the study. Compliance with CPGs was observed for 118 patients (54%). Average total costs reached 23,571 euros when treatment was in accordance with CPGs and 27,313 euros when it was not. In relation to relapse-free survival, compliance with CPGs strictly dominates non compliance, i.e. it is both less costly and more effective. Taking uncertainty into account, the probability that compliance with CPGs still strictly dominates was 75%. CONCLUSIONS: Our findings should encourage physicians to increase their compliance with CPGs and healthcare administrators to invest in the implementation of CPGs in the management of sarcoma.

A workplace physical activity program at a public university in Mexico can reduce medical costs associated with type 2 diabetes and hypertension / Un programa de actividad física en el lugar de trabajo en una universidad pública de México puede reducir los costos médicos asociados a la diabetes tipo 2 e hipertensión

OBJECTIVE: To assess the impact of a workplace leisure physical activity program on healthcare expenditures for type 2 diabetes and hypertension treatment. MATERIAL AND METHODS: We assessed a workplace program's potential to reduce costs by multiplying the annual healthcare costs of patients with type 2 diabetes and hypertension by the population attributable risk fraction of non-recommended physical activity levels. Feasibility of a physical activity program was assessed among 425 employees of a public university in Mexico. RESULTS: If 400 sedentary employees engaged in a physical activity program to decrease their risk of diabetes and hypertension, the potential annual healthcare cost reduction would be 138 880 US dollars. Each dollar invested in physical activity could reduce treatment costs of both diseases by 5.3 dollars. CONCLUSIONS: This research meets the call to use health economics methods to re-appraise health priorities, and devise strategies for optimal allocation of financial resources in the health sector.

OBJETIVO: Evaluar el impacto de un programa de actividad física en el lugar de trabajo sobre la reducción de costos médicos directos relacionados con la diabetes tipo 2 y la hipertensión. MATERIAL Y MÉTODOS: Calculamos el potencial de la actividad física para reducir costos médicos, multiplicando los gastos médicos anuales que realizan diabéticos e hipertensos, multiplicados por la fracción atribuible poblacional asociada a un nivel de actividad física insuficiente. La factibilidad de ejecutar el programa fue evaluada en 425 trabajadores de una universidad pública en México. RESULTADOS: Si 400 trabajadores sedentarios participaran en un programa de actividad física recreativa para reducir su riesgo de diabetes e hipertensión, los costos médicos anuales reducirían en 138880 dólares. Cada dólar invertido en actividad física podría reducir 5.3 dólares en el tratamiento de ambas enfermedades. CONCLUSIONES: Esta investigación evidencia la utilidad de los estudios costoeconómicos en salud para optimizar los recursos financieros en este sector.

Economic issues involved in integrating genomic testing into clinical care: the case of genomic testing to guide decision-making about chemotherapy for breast cancer patients.

The use of taxanes to treat node-positive (N+) breast cancer patients is associated with heterogeneous benefits as well as with morbidity and financial costs. This study aimed to assess the economic impact of using gene expression profiling to guide decision-making about chemotherapy, and to discuss the coverage/reimbursement issues involved. Retrospective data on 246 patients included in a randomised trial (PACS01) were analyzed. Tumours were genotyped using DNA microarrays (189-gene signature), and patients were classified depending on whether or not they were likely to benefit from chemotherapy regimens without taxanes. Standard anthracyclines plus taxane chemotherapy (strategy AT) was compared with the innovative strategy based on genomic testing (GEN). Statistical analyses involved bootstrap methods and sensitivity analyses. The AT and GEN strategies yielded similar 5-year metastasis-free survival rates. In comparison with AT, GEN was cost-effective when genomic testing costs were less than 2,090€. With genomic testing costs higher than 2,919€, AT was cost-effective. Considering a 30% decrease in the price of docetaxel (the patent rights being about to expire), GEN was cost-effective if the cost of genomic testing was in the 0€-1,139€, range; whereas AT was cost-effective if genomic testing costs were higher than 1,891€. The use of gene expression profiling to guide decision-making about chemotherapy for N+ breast cancer patients is potentially cost-effective. Since genomic testing and the drugs targeted in these tests yield greater well-being than the sum of those resulting from separate use, questions arise about how to deal with extra well-being in decision-making about coverage/reimbursement.

Impact of uncertainty on cost-effectiveness analysis of medical strategies: the case of high-dose chemotherapy for breast cancer patients.

OBJECTIVES: The object of this study was to determine, taking into account uncertainty on cost and outcome parameters, the cost-effectiveness of high-dose chemotherapy (HDC) compared with conventional chemotherapy for advanced breast cancer patients. METHODS: An analysis was conducted for 300 patients included in a randomized clinical trial designed to evaluate the benefits, in terms of disease-free survival and overall survival, of adding a single course of HDC to a four-cycle conventional-dose chemotherapy for breast cancer patients with axillary lymph node invasion. Costs were estimated from a detailed observation of physical quantities consumed, and the Kaplan-Meier method was used to evaluate mean survival times. Incremental cost-effectiveness ratios were evaluated successively considering disease-free survival and overall survival outcomes. Handling of uncertainty consisted in construction of confidence intervals for these ratios, using the truncated Fieller method. RESULTS: The cost per disease-free life year gained was evaluated at 13,074 Euros, a value that seems to be acceptable to society. However, handling uncertainty shows that the upper bound of the confidence interval is around 38,000 Euros, which is nearly three times higher. Moreover, as no difference was demonstrated in overall survival between treatments, cost-effectiveness analysis, that is a cost minimization, indicated that the intensive treatment is a dominated strategy involving an extra cost of 7,400 Euros, for no added benefit. CONCLUSIONS: Adding a single course of HDC led to a clinical benefit in terms of disease-free survival for an additional cost that seems to be acceptable, considering the point estimate of the ratio. However, handling uncertainty indicates a maximum ratio for which conclusions have to be discussed.