Objective: Burosumab is an orphan medicinal product (OMP) approved in Europe for the treatment of X-linked hypophosphatemia (XLH). The aim of this study was to assess the value of burosumab versus conventional therapy for the treatment of paediatric XLH, through a multi-criteria decision analysis (MCDA) framework for health technology assessment (HTA) of OMPs in Portugal. Methods: The MCDA framework considered 14 criteria related to disease burden, therapeutic value and economic burden. A multidisciplinary panel of national stakeholders participated in a two-phase exercise. In the first phase, relative weights and part-worth utilities for the criteria and their levels were elicited and a reimbursement likelihood function was calibrated through adaptive conjoint analysis. In the second phase, burosumab and conventional therapy were assessed against the criteria, providing a global value score (0-100) and reimbursement likelihood (0-100%) for both. Results: Of the 14 criteria, disease burden, therapeutic value and economic burden criteria represented 27.29%, 57.17% and 15.53% of the total weight in the decision, respectively. All disease burden and some therapeutic value criteria, typically not included in traditional HTA, represented 47.88% of the total weight. Burosumab was unanimously considered superior to conventional therapy, with an average (range) global value score of 84.96 (82.48-86.54) against 48.06 (43.37-57.68), and reimbursement likelihood of 97.50% (96.78%-98.32%) against 43.66% (31.48%-68.73%), respectively. Conclusions: MCDA represents a powerful tool in HTA decision-making for OMPs. The results of this MCDA acknowledge burosumab as a disease-modifying drug, deemed superior to conventional therapy for the treatment of paediatric XLH.

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INTRODUCTION:Anemia is a major problem, frequently resulting from iron deficiency (1). Guidelines recommend the administration of intravenous (IV) iron, leaving blood transfusions for critical patients due to the potential impact in length of stay (LOS) and mortality (2,3). We aimed to characterize IV iron sucrose utilization and health resource utilization in anemic patients.METHODS:This is a retrospective ongoing cohort study. Patient records from a general Portuguese Hospital with an administration of iron sucrose in 2014–2015 were reviewed. Adult anemic patients with at least one hemoglobin (Hb) evaluation before and after the administration of IV iron were included. Endpoints assessed were: Hb level (baseline, 4 and 8 weeks after), anemia correction rate at weeks 4 and 8, blood transfusions, length of stay (LOS), rate of readmissions (<30 days) and inpatient mortality. Statistical analysis included non-parametric and chi-square tests to assess differences between groups and a logistic regression model, using a 5 percent significance level.RESULTS:Data was collected for 401 patients (63.1 percent female; mean age Standard Deviation, SD: 62.6 (21.7) years) and 431 IV iron sucrose administration episodes. Mean cumulative iron dose was 679.5 mg. Baseline Hb level was 84.5 g/l and increased to 94.3 g/l (week 4) and to 103.0 g/l (week 8). Blood transfusions were performed in 53.8 percent of the episodes. Overall 157 (36.4 percent) episodes had a >20 g/l increase in Hb level. Blood transfusions were associated with a higher proportion of Hb level increase >20 g/l (44.0 percent versus 27.9 percent, p<.001). The overall mean LOS was 15.3 days, although episodes with transfusions had a significantly longer duration (17.5 days versus 12.7 days; p<.001). Overall readmission rate was 25.8 percent, with a higher proportion in episodes with blood transfusions (29.3 percent versus 21.6 percent). A total of 36 patients (9.0 percent) died at the hospital before discharge. Transfusions performed during or after IV iron administration increased 3.1 times the risk of in-hospital death (95 percent Confidence Interval, CI: 1.3-7.0; p = .008), after adjusting for age and sex.CONCLUSIONS:We observed a high rate of blood transfusions in this cohort treated with intravenous iron sucrose for anemia. Transfusions were associated with substantial burden of resource consumption and in-hospital mortality.