Purpose: Tisagenlecleucel, a chimeric antigen receptor T-cell (CAR-T) therapy, is a promising alternative for the management of children and young adults with relapsed and refractory B-cell acute lymphoblastic leukemia (r/r ALL). The aim of this study was to determine whether treatment with tisagenlecleucel is a cost-effective intervention compared with salvage chemotherapy in paediatric and young adult patients with r/r ALL in Spain. Materials and Methods: A partitioned survival model of monthly cycles with three health states was used (event-free survival, progressive/relapsed disease and death). A lifetime time horizon and the Spanish National Health System perspective were adopted. During the first 5 years, permanence in the different health states was determined according to the results in the clinical studies. In successive years, mortality tables of the Spanish general population adjusted by standardized mortality rate for survivors of childhood cancer were used. Clinical, economic, and quality of life parameters were drawn from clinical trials and the literature. Only direct health costs (pharmacological costs and the costs derived from health resource use) were included. The robustness of the results was evaluated in a sensitivity analysis. Results: This cost-effectiveness analysis showed a greater benefit (10.10 and 8.97 life-years gained [LYGs] and quality-adjusted life-years [QALYs] gained, respectively) and a higher cost (€ 258,378.40) for tisagenlecleucel compared to salvage chemotherapy. The resulting incremental cost-effectiveness and cost-utility ratios were € 25,576.80 per LYG and € 28,818.52 per QALY gained, respectively. In the sensitivity analysis, all the results were below € 50,000/QALY. Conclusion: Tisagenlecleucel would represent a cost-effective intervention for the treatment of children and young adults with r/r ALL in Spain.

Malnutrition has been related to prolonged hospital stays, and to increases in readmission and mortality rates. In the NOURISH (Nutrition effect On Unplanned Readmissions and Survival in Hospitalized patients) study, administering a high protein oral nutritional supplement (ONS) containing beta-hydroxy-beta-methylbutyrate (HP-HMB) to hospitalised older adult patients led to a significant improvement in survival compared with a placebo treatment. The aim of this study was to determine whether HP-HMB would be cost-effective in Spain. We performed a cost-effectiveness analysis from the perspective of the Spanish National Health System using time horizons of 90 days, 180 days, 1 year, 2 years, 5 years and lifetime. The difference in cost between patients treated with HP-HMB and placebo was €332.75. With the 90 days time horizon, the difference in life years gained (LYG) between both groups was 0.0096, resulting in an incremental cost-effectiveness ratio (ICER) of €34,700.62/LYG. With time horizons of 180 days, 1 year, 2 years, 5 years and lifetime, the respective ICERs were €13,711.68, €3377.96, €2253.32, €1127.34 and €563.84/LYG. This analysis suggests that administering HP-HMB to older adult patients admitted to Spanish hospitals during hospitalisation and after discharge could be a cost-effective intervention that would improve survival with a reduced marginal cost.

The addition of midostaurin to standard chemotherapy (cytarabine and daunorubicin) has shown significant improvements in the survival of patients with acute myeloid leukemia with the FLT3 mutation (FLT3-AML). The objective of this study was to determine whether this intervention would be cost-effective in Spain. Methods: A partitioned survival model with five health states was developed (diagnosis and induction, complete remission, no complete remission, transplantation and death). A lifetime time horizon and the Spanish National Health System perspective were adopted. During the first three years, permanence in the different health states was determined according to the results of the RATIFY study. In successive years, the death rates of the Spanish population adjusted by a factor to reflect long-term disease-related mortality were used. Utilities were obtained from the literature. Pharmacological costs (first and second line) and the costs of other health resources (hospitalizations, visits and tests) were included. The robustness of the model was evaluated by deterministic and probabilistic sensitivity analyses. Results: The addition of midostaurin resulted in 1.46 life years gained (LYG) and 1.23 quality-adjusted life years (QALY) gained and implied an additional cost of € 47,955, resulting in an incremental cost-effectiveness ratio (ICER) of € 32,854/LYG and an incremental cost-utility ratio of € 38,985/QALY. In the univariate sensitivity analysis, the threshold of € 50,000/QALY was not exceeded in any case; taking into consideration potential discounts of 20-40% in the PVL of midostaurin the ICER would be below € 30,000/QALY, a commonly accepted threshold in Spain. In the probabilistic analysis, when the threshold was € 50,000/QALY, midostaurin was cost-effective in 82.3% of simulations. Conclusion:According to our modeling, midostaurin, in combination with standard chemotherapy, could be an efficient alternative for the treatment of FLT3-AML in Spain.

objeCtives: To conduct a cost-effectiveness analysis to evaluate whether the addition of a fecal occult blood test (FOBT) kit to a mailed invitation for recall compared to a mailed invitation for recall alone increases participation among patients who had responded previously to a mailed invitation in screening for colorectal cancer (CRC). Methods: The perspective of the analysis will be that of the cancer agency in Ontario, Canada. Eligible patients (responders to the initial pilot study invitation, FOBT negative, and now due for repeat screening) from 61 physicians were randomly allocated to one of the two interventions: (1) Mailed FOBT kit and mailed invitation for recall from their family physician (intervention group) OR (2) mailed invitation alone for recall (control group). Health system and program resources and costs associated with each group will be identified and quantified. Resources will be stratified into fixed costs (initial set-up costs including document development, programming for ongoing maintenance, etc.), variable or recurrent costs (costs of the kit, administrative costs, physician visits etc.) and staff costs (call centre support personnel and business analyst). Results: There are a total of 431 patients in the intervention group and 452 patients in the control group. Overall costs for each group will be determined, and the cost per patient will be reported. Cost drivers will be identified by conducting a series of univariate sensitivity analyses, including reducing the cost of the kit and using different assumptions for kit wastage. The results will show whether the addition of the FOBT kit to the mailed invitation is cost effective when compared to a mailed invitation only. ConClusions: This cost effectiveness analysis will help in determining effective strategies for screening programs that are needed to reduce CRC mortality at a population level.objeCtives: To assess the clinical and cost effectiveness of mammography for breast cancer screening in India Methods: A systematic literature search was conducted in all the available scientific databases -Cochrane library, MEDLINE, PUBMED Science Direct, EMBASE, SCOPUS and Google Scholar for relevant studies. We identified 31 studies and literature filter started by scanning titles; abstracts as well as the content of the articles according to Inclusion criteria; finally 12 studies were included in quantitative synthesis (Meta analysis). We estimated risk of bias using Cochrane collaborating guidelines. Results: Review Manager 5.2 was used to do the data analysis and results are expressed in legible diagrams, considering all 12 studies with data from 4047721 participants, Risk Ratio was calculated (RR): 0.71 (95% confidence Interval, CI: 0.67, 0.75). ConClusions: Annual screening of female population above 30 years of age could reduce breast cancer associated mortality by 29% mainly due to early detection of breast cancer detection and subsequent early treatment pathways. The cost effectiveness is about Rs. 19520/-per life year gained which ...