Cost-effectiveness of somatrogon in the Spanish pediatric population with growth hormone deficiency.

OBJECTIVE: To analyze the cost-effectiveness of weekly somatrogon compared to daily growth hormones (GH-d) in the pediatric population of Spain with growth hormone deficiency (GHD). METHODS: Markov model with two states (patients with or without GH-d or somatrogon treatment) in prepubertal children (3 to 11 years and 3 to 12 years in girls and boys, respectively) with GHD in isolation or as part of multiple pituitary hormone deficiency and without previous treatment, from the perspective of the National Health System. The simulation of the economic model ends at the age of 18. The costs of hormones and monitoring were obtained from Spanish sources. The utilities were obtained from the literature. Spanish clinical experts validated the assumptions of the model. RESULTS: In the deterministic analysis, somatrogon would be cost-effective, compared to GH-d, with a cost per QALY (quality-adjusted life year) gained of €19,259 and a clinically relevant QALY gain (0.336). This result was confirmed in deterministic sensitivity analyses. According to the probabilistic analysis, somatrogon would be the dominant treatment, with a 61% probability of a willingness to pay of €25,000 per QALY gained. CONCLUSION: Compared to GH-d, somatrogon is cost-effective in the Spanish pediatric population with GHD.

Prospective, multicenter study on the economic and clinical impact of gene-expression assays in early-stage breast cancer from a single region: the PREGECAM registry experience.

INTRODUCTION: The aim of this study is to evaluate the cost-effectiveness and impact of gene-expression assays (GEAs) on treatment decisions in a real-world setting of early-stage breast cancer (ESBC) patients. METHODS: This is a regional, prospective study promoted by the Council Health Authorities in Madrid. Enrolment was offered to women with estrogen receptor-positive, human epidermal growth factor receptor 2-negative, node-negative or micrometastatic, stage I or II breast cancer from 21 hospitals in Madrid. Treatment recommendations were recorded before and after knowledge of tests results. An economic model compared the cost-effectiveness of treatment, guided by GEAs or by common prognostic factors. RESULTS: 907 tests (440 Oncotype DX® and 467 MammaPrint®) were performed between February 2012 and November 2014. Treatment recommendation changed in 42.6% of patients. The shift was predominantly from chemohormonal (CHT) to hormonal therapy (HT) alone, in 30.5% of patients. GEAs increased patients' confidence in treatment decision making. Tumor grade, progesterone receptor positivity and Ki67 expression were associated with the likelihood of change from CHT to HT (P < 0.001) and from HT to CHT (P < 0.001). Compared with current clinical practice genomic testing increased quality-adjusted life years by 0.00787 per patient and was cost-saving from a national health care system (by 13.867€ per patient) and from a societal perspective (by 32.678€ per patient). CONCLUSION: Using GEAs to guide adjuvant therapy in ESBC is cost-effective in Spain and has a significant impact on treatment decisions.

Cost-effectiveness of Cladribine Tablets and fingolimod in the treatment of relapsing multiple sclerosis with high disease activity in Spain.

OBJECTIVE: To estimate the cost-effectiveness of Cladribine Tablets in the treatment of relapsing multiple sclerosis (RMS) with high disease activity compared with fingolimod, from the perspective of the National Health System (NHS) in Spain. METHODS: A Markov model was developed. The annual transition probabilities, were adjusted to patients with RMS with high disease activity. The effect of the treatments compared on the Expanded Disability Status Scale (EDSS) was modeled by hazard ratios for the confirmed progression of disability. The annual relapse rate and the probability of suffering adverse reactions were obtained from a meta-analysis and the literature. The derived costs were calculated from Spanish unit costs. The utilities were obtained from the CLARITY clinical trial and the literature. Deterministic and probabilistic sensitivity analyzes were performed. RESULTS: Cladribine tablets was the dominant treatment: lower costs (-86,536 €) and more effective (+1.11 quality-adjusted life years - QALYs) compared to fingolimod. The probability that Cladribine Tablets was cost-effective compared to fingolimod ranged between 94.6% and 96.1% for willingness to pay from € 20,000 to € 30,000 per QALY gained. CONCLUSIONS: Cladribine Tablets is a cost-effective treatment, compared to fingolimod, for the treatment of RMS with high disease activity. EXPERT OPINION: According to the present study, compared to fingolimod, treatment with Cladribine Tablets of relapsing multiple sclerosis with high disease activity is an option that could generate savings for the Spanish National Health System, with a considerable gain in QALYs. Cladribine Tablets is considered cost-effective and dominant (less costs and more effectiveness) than fingolimod treatment option in this population.

Cost-effectiveness of three echinocandins and fluconazole in the treatment of candidemia and/or invasive candidiasis in nonneutropenic adult patients.

OBJECTIVE: To estimate the cost-effectiveness of three echinocandins (anidulafungin, caspofungin, and micafungin) and generic fluconazole in the treatment of nonneutropenic adult patients with candidemia and/or invasive candidiasis in intensive care units in Spain. MATERIALS AND METHODS: A decision-tree model was applied. The success and safety (hepatic and renal adverse effects) of first-line treatments were obtained from meta-analyses and systematic reviews of clinical trials. In the case of failure, a second-line treatment (liposomal amphotericin B after the echinocandins, or one of the echinocandins after fluconazole) was administered. The duration of the treatments (14 days total) was established by a panel of clinical experts using the Delphi method and according to Infectious Diseases Society of America guidelines. The cost of the medications and renal toxicity were considered. Deterministic and probabilistic sensitivity analysis using Monte Carlo simulations were carried out. RESULTS: The total cost of the treatment of candidemia and/or invasive candidiasis with anidulafungin, caspofungin, micafungin, and fluconazole was €5,483, €5,968, €6,231, and €2,088, respectively. Anidulafungin was the dominant treatment (more effective, less expensive) compared to micafungin and caspofungin. The cost of achieving one more patient successfully treated with anidulafungin, caspofungin, and micafungin compared to fluconazole was €17,199, €23,962, and €27,339, respectively. The result remained stable, despite modification of the duration of the first-line and second-line treatments, as well as most of the dosing regimens. The probabilistic analysis also remained stable. CONCLUSION: In accordance with this economic study, anidulafungin would produce savings and would be the dominant treatment compared with micafungin and caspofungin in nonneutropenic adult patients with candidemia and/or invasive candidiasis in intensive care units in Spain.

Economic assessment of fidaxomicin for the treatment of Clostridium difficile infection (CDI) in special populations (patients with cancer, concomitant antibiotic treatment or renal impairment) in Spain.

The objective of this paper was to assess the cost-utility of fidaxomicin versus vancomycin in the treatment of Clostridium difficile infection (CDI) in three specific CDI patient subgroups: those with cancer, treated with concomitant antibiotic therapy or with renal impairment. A Markov model with six health states was developed to assess the cost-utility of fidaxomicin versus vancomycin in the patient subgroups over a period of 1 year from initial infection. Cost and outcome data used to parameterise the model were taken from Spanish sources and published literature. The costs were from the Spanish hospital perspective, in Euros (€) and for 2013. For CDI patients with cancer, fidaxomicin was dominant versus vancomycin [gain of 0.016 quality-adjusted life-years (QALYs) and savings of €2,397 per patient]. At a cost-effectiveness threshold of €30,000 per QALY gained, the probability that fidaxomicin was cost-effective was 96 %. For CDI patients treated with concomitant antibiotic therapy, fidaxomicin was the dominant treatment versus vancomycin (gain of 0.014 QALYs and savings of €1,452 per patient), with a probability that fidaxomicin was cost-effective of 94 %. For CDI patients with renal impairment, fidaxomicin was also dominant versus vancomycin (gain of 0.013 QALYs and savings of €1,432 per patient), with a probability that fidaxomicin was cost-effective of 96 %. Over a 1-year time horizon, when fidaxomicin is compared to vancomycin in CDI patients with cancer, treated with concomitant antibiotic therapy or with renal impairment, the use of fidaxomicin would be expected to result in increased QALYs for patients and reduced overall costs.

Economic analysis of thrombo inCode, a clinical-genetic function for assessing the risk of venous thromboembolism.

BACKGROUND: Patients with venous thromboembolism (VTE) commonly have an underlying genetic predisposition. However, genetic tests nowadays in use have very low sensitivity for identifying subjects at risk of VTE. Thrombo inCode(®) is a new genetic tool that has demonstrated very good sensitivity, thanks to very good coverage of the genetic variants that modify the function of the coagulation pathway. OBJECTIVE: To conduct an economic analysis of risk assessment of VTE from the perspective of the Spanish National Health System with Thrombo inCode(®) (a clinical-genetic function for assessing the risk of VTE) versus the conventional/standard method used to date (factor V Leiden and prothrombin G20210A). METHODS: An economic model was created from the National Health System perspective, using a decision tree in patients aged 45 years with a life expectancy of 81 years. The predictive capacity of VTE, based on identification of thrombophilia using Thrombo inCode(®) and using the standard method, was obtained from two case-control studies conducted in two different populations (S. PAU and MARTHA; 1,451 patients in all). Although this is not always the case, patients who were identified as suffering from thrombophilia were subject to preventive treatment of VTE with warfarin, leading to a reduction in the number of VTE events and an increased risk of severe bleeding. The health state utilities (quality-adjusted life-years [QALYs]) and costs (in 2013 EUR values) were obtained from the literature and Spanish sources. RESULTS: On the basis of a price of EUR 180 for Thrombo inCode(®), this would be the dominant option (more effective and with lower costs than the standard method) in both populations. The Monte Carlo probabilistic analyses indicate that the dominance would occur in 100 % of the simulations in both populations. The threshold price of Thrombo inCode(®) needed to reach the incremental cost-effectiveness ratio (ICER) generally accepted in Spain (EUR 30,000 per QALY gained) would be between EUR 3,950 (in the MARTHA population) and EUR 11,993 (in the S. PAU population). CONCLUSION: According to the economic model, Thrombo inCode(®) is the dominant option in assessing the risk of VTE, compared with the standard method currently used.

Economic evaluation of Cardio inCode®, a clinical-genetic function for coronary heart disease risk assessment.

BACKGROUND: A clinical­genetic function (Cardio inCode®) was generated using genetic variants associated with coronary heart disease (CHD), but not with classical CHD risk factors, to achieve a more precise estimation of the CHD risk of individuals by incorporating genetics into risk equations [Framingham and REGICOR (Registre Gironí del Cor)]. OBJECTIVE: The objective of this study was to conduct an economic analysis of the CHD risk assessment with Cardio inCode®, which incorporates the patient's genetic risk into the functions of REGICOR and Framingham, compared with the standard method (using only the functions). METHODS: A Markov model was developed with seven states of health (low CHD risk, moderate CHD risk, high CHD risk, CHD event, recurrent CHD, chronic CHD, and death). The reclassification of CHD risk derived from genetic information and transition probabilities between states was obtained from a validation study conducted in cohorts of REGICOR (Spain) and Framingham (USA). It was assumed that patients classified as at moderate risk by the standard method were the best candidates to test the risk reclassification with Cardio inCode®. The utilities and costs (€; year 2011 values) of Markov states were obtained from the literature and Spanish sources. The analysis was performed from the perspective of the Spanish National Health System, for a life expectancy of 82 years in Spain. An annual discount rate of 3.5 % for costs and benefits was applied. RESULTS: For a Cardio inCode® price of €400, the cost per QALY gained compared with the standard method [incremental cost-effectiveness ratio (ICER)] would be €12,969 and €21,385 in REGICOR and Framingham cohorts, respectively. The threshold price of Cardio inCode® to reach the ICER threshold generally accepted in Spain (€30,000/QALY) would range between €668 and €836. The greatest benefit occurred in the subgroup of patients with moderate­high risk, with a high-risk reclassification of 22.8 % and 12 % of patients and an ICER of €1,652/QALY and €5,884/QALY in the REGICOR and Framingham cohorts, respectively. Sensitivity analyses confirmed the stability of the study results. CONCLUSIONS: Cardio inCode® is a cost-effective risk score option in CHD risk assessment compared with the standard method.

Análisis económico de la promoción intensiva y especializada de la lactancia materna en las unidades neonatales en España / Economic analysis of specialised and intensive promotion of breastfeeding in neonatal units in Spain

Introducción: A pesar de que la lactancia materna (LM) es el alimento idóneo para el neonato porque cubre sus necesidades de crecimiento y le proporciona inmunidad activa frente a diversas enfermedades; esta es sustituida frecuentemente por la lactancia artificial. Se estimó el coste-efectividad de una mayor promoción de la LM en las unidades neonatales mediante la intervención intensiva y especializada de enfermeras o matronas dedicadas a ese propósito. Métodos: Se hizo un análisis de coste-efectividad, mediante un modelo de análisis de decisiones en el que se incluyeron los datos disponibles sobre una intervención especializada para la promoción de la LM, su efectividad, las consecuencias a corto plazo (sepsis, enterocolitis necrosante) y largo plazo (discapacidad por daño neurológico) de las diferentes formas de lactancia, así como la mortalidad, los costes y las utilidades que conllevan, obtenidos de la literatura médica y de los estudios y fuentes españolas disponibles. El análisis se limitó a 3 subgrupos de neonatos de bajo peso (500-999g, 1.000-1.749g y 1.750-2.500g). Resultados: En el análisis determinístico la intervención fue «dominante» para los 3 subgrupos de neonatos, lo que significa que fue más eficaz, con menores costes, que la no intervención. Los análisis probabilísticos de Monte Carlo confirmaron la estabilidad del resultado, con probabilidades de dominancia del 100, el 100 y el 99,9% para los 3 subgrupos de pesos, respectivamente. La intervención dejaría de ser coste-efectiva con costes de la intervención por puérpera de 70.504 €, 14.742 € y 7.106 €, respectivamente. Estos costes estarían muy por encima del máximo estimado (12,51 €). Conclusiones: Según el presente modelo, la promoción de la lactancia materna mediante una intervención intensiva y especializada de enfermeras o matronas dedicadas a ese propósito puede ser coste-efectiva en el caso de las puérperas con neonatos de bajo peso (500-2.500g)(AU)

Introduction: Although breastfeeding (BF) is the ideal food for newborns because it covers their growing needs and provides active immunity against various diseases, it is often replaced by artificial feeding. We estimated the cost-effectiveness of increased promotion of BF in neonatal units with intensive intervention with specialised nurses or midwives dedicated to this purpose. Methods: An analysis of cost-effectiveness, using a decision analysis model which included data on a specialised intervention for the promotion of BF, its effectiveness in the short-term (sepsis, necrotizing enterocolitis) and long-term (disabled due to neurodevelopmental impairment) of the different types of milk consumed, as well as the mortality, costs and benefits of these. Data was also obtained from current medical literature, studies and Spanish sources. The analysis was limited to three subgroups of low birthweights (500-999g, 1000-1749g, 1750-2500g). Results: In the deterministic analysis, the intervention was "dominant" for the three subgroups of infants, which meant it was more effective and with lower costs, than no intervention. The Monte Carlo probabilistic analysis confirmed the robustness of the result with probabilities of dominance of 100%, 100% and 99.9% for the three subgroups of weights, respectively. The intervention would be cost-effective with costs of puerperal intervention of € 70,504, € 14,742 and € 7,106, respectively. These costs would be well above the estimated maximum (€ 12.51). Conclusions: According to this model, the promotion of breastfeeding through an intensive promotion and with specialised nurses or midwives dedicated to this purpose, may be cost-effective in the case of puerperal women with low birth weight neonates (500-2500g)(AU)

[Economic analysis of specialised and intensive promotion of breastfeeding in neonatal units in Spain]. / Análisis económico de la promoción intensiva y especializada de la lactancia materna en las unidades neonatales en España.

INTRODUCTION: Although breastfeeding (BF) is the ideal food for newborns because it covers their growing needs and provides active immunity against various diseases, it is often replaced by artificial feeding. We estimated the cost-effectiveness of increased promotion of BF in neonatal units with intensive intervention with specialised nurses or midwives dedicated to this purpose. METHODS: An analysis of cost-effectiveness, using a decision analysis model which included data on a specialised intervention for the promotion of BF, its effectiveness in the short-term (sepsis, necrotizing enterocolitis) and long-term (disabled due to neurodevelopmental impairment) of the different types of milk consumed, as well as the mortality, costs and benefits of these. Data was also obtained from current medical literature, studies and Spanish sources. The analysis was limited to three subgroups of low birthweights (500-999g, 1000-1749g, 1750-2500g). RESULTS: In the deterministic analysis, the intervention was "dominant" for the three subgroups of infants, which meant it was more effective and with lower costs, than no intervention. The Monte Carlo probabilistic analysis confirmed the robustness of the result with probabilities of dominance of 100%, 100% and 99.9% for the three subgroups of weights, respectively. The intervention would be cost-effective with costs of puerperal intervention of € 70,504, € 14,742 and € 7,106, respectively. These costs would be well above the estimated maximum (€ 12.51). CONCLUSIONS: According to this model, the promotion of breastfeeding through an intensive promotion and with specialised nurses or midwives dedicated to this purpose, may be cost-effective in the case of puerperal women with low birth weight neonates (500-2500g).