Cost Effectiveness of Sucroferric Oxyhydroxide Compared with Sevelamer Carbonate in the Treatment of Hyperphosphataemia in Patients Receiving Dialysis, from the Perspective of the National Health Service in Scotland.

BACKGROUND: Hyperphosphataemia is common and harmful in patients receiving dialysis. Treatment options include noncalcium-based phosphate binders such as sevelamer carbonate (SC) and sucroferric oxyhydroxide (PA21). OBJECTIVE: The aim of this study was to determine the health economic impact of PA21-based strategies compared with SC-based strategies, from the perspective of the Scottish National Health Service (NHS). METHODS: A Markov model was constructed based on data from a randomised clinical trial comparing PA21 and SC. Model input parameters were derived from published literature, national statistics and unpublished sources. Costs (price year 2012) and effects were discounted at 3.5 %. Analysis with a lifelong time horizon yielded the incremental cost-effectiveness ratio (ICER), expressed as cost or savings per quality-adjusted life-year (QALY) gained or forgone. Deterministic and probabilistic sensitivity analysis was performed to explore uncertainties around assumptions and model input parameters. RESULTS: In the base-case analysis, phosphorus reductions for PA21 and SC were 1.93 and 1.95 mg/dL. Average undiscounted survival was estimated to be 7.61 years per patient in both strategies. PA21 patients accrued less QALYs (2.826) than SC patients (2.835), partially due to differential occurrence of side effects. Total costs were £ 13,119 and £ 14,728 for PA21 and SC, respectively (difference per patient of £ 1609). By using PA21 versus SC, one would save £ 174,999 (or £ 123,463 when including dialysis and transplantation costs) for one QALY forgone. A scenario modelling the nonsignificant reduction in mortality (relative risk 0.714) observed in the trial yielded an ICER for PA21 of £ 22,621 per QALY gained. In probabilistic sensitivity analysis of the base-case, PA21 was dominant in 11 %, and at least cost-effective in 53 %, of iterations, using a threshold of £ 20,000 per QALY gained. CONCLUSIONS: The use of PA21 versus SC in hyperphosphataemic patients being intolerant of calcium-based phosphate binders may be cost saving and yields only very limited disadvantages in terms of quality-adjusted survival. PA21 appears to be cost-effective from the perspective of the Scottish NHS.

Determinants of quality of life of patients with heart failure and iron deficiency treated with ferric carboxymaltose: FAIR-HF sub-analysis.

BACKGROUND: Heart failure (HF) is a burden to patients and health care systems. The objectives of HF treatment are to improve health related quality of life (HRQoL) and reduce mortality and morbidity. We aimed to evaluate determinants of health-related quality of life (HRQoL) in patients with iron deficiency and HF treated with intravenous (i.v.) iron substitution or placebo. METHODS: A randomised, double-blind, placebo-controlled trial (n = 459) in iron-deficient chronic heart failure (CHF) patients with or without anaemia studied clinical and HRQoL benefits of i.v. iron substitution using ferric carboxymaltose (FCM) over a 24-week trial period. Multivariate analysis was carried out with various clinical variables as independent variables and HRQoL measures as dependent variables. RESULTS: Mean change from baseline of European Quality of Life - 5 Dimensions (EQ-5D) (value set-based) utilities (on a 0 to 100 scale) at week 24 was 8.91 (i.v. iron) and 0.68 (placebo; p < 0.01). In a multivariate analysis excluding baseline HRQoL, a higher exercise tolerance and i.v. iron substitution positively influenced HRQoL, whereas impaired renal function and a history of stroke had a negative effect. The level of HRQoL was also influenced by country of residence. When baseline HRQoL was factored in, the multivariate model remained stable. CONCLUSION: In this study, i.v. iron substitution, exercise tolerance, stroke, country of residence and renal function influenced measures of HRQoL in patients with heart failure and iron deficiency.

Health economic assessment of ferric carboxymaltose in patients with iron deficiency and chronic heart failure based on the FAIR-HF trial: an analysis for the UK.

AIMS: The purpose of this study was to evaluate the cost-effectiveness of iron repletion using intravenous (i.v.) ferric carboxymaltose (FCM) in chronic heart failure (CHF) patients with iron deficiency with or without anaemia. Cost-effectiveness was studied from the perspective of the National Health Service in the UK. METHODS AND RESULTS: A model-based cost-effectiveness analysis was used to compare iron repletion with FCM with no iron treatment. Using data from the FAIR-HF trial and publicly available sources and publications, per patient costs and clinical effectiveness of FCM were estimated compared with placebo. Cost assessment was based on study drug and administration costs, cost of CHF treatment, and hospital length of stay. The incremental cost-effectiveness ratio (ICER) of FCM use was expressed as cost per quality-adjusted life year (QALY) gained, and sensitivity analyses were performed on the base case. The time horizon of the analysis was 24 weeks. Mean QALYs were higher in the FCM arm (difference 0.037 QALYs; bootstrap-based 95% confidence interval 0.017-0.060). The ICER of FCM compared with placebo was €4414 per QALY gained for the FAIR-HF dosing regimen. Sensitivity analyses confirmed the base case result to be robust. CONCLUSION: From the UK payers' perspective, managing iron deficiency in CHF patients using i.v. FCM was cost-effective in this analysis. The base case ICER was clearly below the threshold of €22 200-€33 300/QALY gained (£20 000-£30 000) typically used by the UK National Institute for Health and Clinical Excellence and proved to be robust in sensitivity analysis. Improved symptoms and better quality of life contributed to this result.