Economic evaluation of percutaneous cryoablation vs conventional surgery in extra-abdominal desmoid tumours in the Spanish healthcare system.

BACKGROUND: Desmoid tumours (DTs) or deep fibromatosis are benign soft-tissue tumours, sometimes locally aggressive, requiring intervention on some cases. Surgery has been the gold standard, but new less invasive techniques such as percutaneous cryoablation have proved their effectiveness, reducing health resources and complications. The study aimed to compare the total cost of percutaneous cryoablation and conventional surgery for patients with extra-abdominal and/or abdominal wall DTs, candidates for local ablative treatment in Spain. METHODS: A cost-analysis model was developed. An expert panel provided data about resource consumption for the percutaneous cryoablation technique and validated the epidemiology used for target population estimation. Unitary resources cost (€ 2022) derived from local cost databases. A retrospective analysis of 54 surgical cases in 3 Spanish hospitals was performed to estimate the cost of conventional surgery based on the cost of the Diagnosis-Related group (DRG) codes identified on this patient sample, weighted by each DRG proportion. The total cost for each alternative included intervention cost and complications cost, considering debridement required in 4.5% of cases with percutaneous cryoablation and minor surgery for surgical site infection in 18.0% for conventional surgery. RESULTS: The total cost for percutaneous cryoablation (€ 5774.78/patient-year) was lower than the total cost for conventional surgery (€ 6780.98/patient-year), yielding cost savings up to € 80,002 in 1 year for the entire cohort of 80 patients with DTs eligible for intervention estimated in Spain. One-way sensitivity analyses confirmed the results' robustness. CONCLUSION: Percutaneous cryoablation versus conventional surgery would yield cost savings for the management of DT patients in Spain. CRITICAL RELEVANCE STATEMENT: This manuscript provides insight into the economic impact derived from the savings related to the use of percutaneous cryoablation for desmoid-type tumours from the perspective of the Spanish National Healthcare System, providing useful information for the health decision-making process. KEY POINTS: • Desmoid tumours are locally aggressive and may require local therapy. • Percutaneous cryoablation procedure is less invasive than the conventional surgery. • Cost comparison shows savings associated to percutaneous cryoablation use.

Preemptive TPMT Genotyping and Adherence to Genotype-Based Therapeutic Recommendations Reduces the Healthcare Cost in Patients Receiving Azathioprine or 6-Mercaptopurine for Autoimmune Diseases.

A cost analysis of thiopurine treatment was carried out in 257 patients, with 153 preemptively genotyped for TPMT and 104 retrospectively genotyped in a Spanish setting. The healthcare cost was significantly higher in patients retrospectively genotyped compared to those who were preemptively genotyped (p < 0.001). TPMT intermediate metabolizers (IMs) (n = 23) showed a 3.3-fold higher healthcare cost when compared to normal metabolizers (NMs) (p < 0.001). The healthcare cost in patients with a TPMT IM phenotype whose physician adhered to the genotype-informed recommendation was similar than the cost in TPMT NMs and was significantly lower than IMs whose physician did not adhere to the therapeutic recommendation (3.8-fold, p = 0.016). Myelotoxicity occurrence was significantly lower in patients preemptively vs. retrospectively genotyped (2.0% and 21.2%, respectively, p < 0.001). Patients who developed myelotoxicity showed a significantly higher healthcare cost than those who did not (4.10-fold, p < 0.001). Overall, 87% of patients whose dose was not adjusted despite being TPMT IMs suffered myelotoxicity, while only one of the eight patients (13%) whose dose was adjusted suffered myelotoxicity (p < 0.001). In conclusion, TPMT preemptive genotyping and physician adherence to genotype-informed therapeutic recommendations prevents myelotoxicity and significantly reduces the healthcare cost, and it is therefore essential for the sustainability of the Spanish healthcare system.

Budget impact analysis of transurethral water vapor therapy for treatment of lower urinary tract symptoms associated with benign prostatic hyperplasia in the Spanish national healthcare system.

BACKGROUND: Several surgical treatments are available for managing lower urinary tract symptoms secondary to benign prostatic hyperplasia (LUTS/BPH). Water vapor thermal therapy (WVTT) is a new minimally invasive therapy. This study estimates the budget impact of introducing WVTT for LUTS/BPH into the Spanish health care system. METHODS: A model simulated the evolution of men over 45 years of age with moderate-severe LUTS/BPH after surgical treatment, over a 4-year time horizon, from the Spanish public health care service´s perspective. The technologies in scope included those most used in Spain: WVTT, transurethral resection (TURP), photoselective laser vapourization (PVP) and holmium laser enucleation (HoLEP). Transition probabilities, adverse events and costs were identified from the scientific literature and validated by a panel of experts. Sensitivity analyses were performed by varying the most uncertain parameters. RESULTS: Per intervention, WVTT resulted in savings of €3,317, €1,933 and €2,661 compared to TURP, PVP and HoLEP. Over a 4-year time horizon, when performed in 10% of the cohort of 109,603 Spanish males with LUTS/BPH, WVTT saved €28,770,125 against the scenario without WVTT availability. CONCLUSIONS: WVTT could reduce the cost of managing LUTS/BPH, increase the quality of health care and reduce the length of procedure and hospital stay.

Budget impact analysis of bevacizumab biosimilars for cancer treatment in adult patients in Spain.

OBJECTIVE: To assess the economic impact of introducing biosimilars of bevacizumab for the management of cancer patients receiving systemic bevacizumab in the National Health System (SNHS) of Spain. METHODS: A 3-year budget impact analysis model was adapted to estimate the cost of introducing biosimilars of bevacizumab in the SNHS for the adult population who were candidates to receive treatment with bevacizumab. Values for the estimation of the population were obtained from the literature and were validated by an expert panel. In this analysis only pharmaceutical costs (€, year 2021) obtained from official databases were considered. A sensitivity analysis was performed to examine the robustness of the model. RESULTS: The introduction of bevacizumab biosimilars would generate an annual cost saving of €11 558 268 (-5.1%) for the first year with a penetration share of biosimilars from 30.0%, €29 126 373 (-8.5%) for the second year with a share of 50.0% and €52 361 778 (-13.6%) for the third year with a share of 80.0%. The total pharmaceutical costs of the scenario without biosimilars are €227 033 352 for the first year, €342 663 209 for the second year and €385 013 076 for the third year. In contrast, the pharmaceutical costs of the scenario with bevacizumab biosimilars are €215 475 084, €313 536 836 and €332 651 297 for years 1, 2 and 3, respectively. CONCLUSIONS: The introduction of biosimilars in the Spanish Health System would generate saving costs in the pharmacological budget to boost biological drugs from the first year.

Epidemiologic and Economic Analysis of Rapid Antiretroviral Therapy Initiation with Bictegravir/Emtricitabine/Tenofovir Alafenamide in Spain.

OBJECTIVE: This study aimed to assess the potential epidemiological and economic impact of rapid initiation of human immunodeficiency virus (HIV) treatment with bictegravir/emtricitabine/tenofovir alafenamide (B/F/TAF) on HIV transmission compared with the current initiation observed in clinical practice in Spain. METHODS: A transmission model was adapted to estimate the cumulative HIV infection incidence and potential cost savings based on the number of HIV infections prevented among men who have sex with men, heterosexual males and females, and people who inject drugs (PWID) over a 20-year time horizon. The analysis compared rapid antiretroviral therapy (ART) initiation with B/F/TAF (9 days from diagnosis until treatment initiation) versus current ART initiation practice (with an average of 35 days from diagnosis to treatment). People living with HIV were distributed according to their treatment status. Risk for transmission was assigned to undiagnosed, diagnosed in care and not receiving ART, and receiving ART but virally unsuppressed, which was estimated by sexual contact, needles and syringes shared among PWID, state of HIV infection, and ART use. RESULTS: In the base-case analysis, rapid ART initiation with B/F/TAF is expected to prevent 992 new HIV infections over the next 20 years compared with current ART initiation practices. Considering the lifetime costs of treating HIV infection, the reduction in HIV incidence could result in potential cost savings of €323 million. CONCLUSIONS: These results suggest that rapid ART initiation with B/F/TAF in newly diagnosed patients with HIV is a high-value strategy for the Spanish National Health System and society, reducing HIV incidence and thereby reducing future related direct and indirect costs of care.

Targeted screening for human immunodeficiency virus infection in Spanish emergency departments: an analysis of epidemiologic and economic impact. / Cribado dirigido del virus de la inmunodeficiencia humana en los servicios de urgencias en España: análisis de las consecuencias epidemiológicas y económicas.

OBJECTIVES: To evaluate the potential epidemiologic and economic impact of applying an HIV screening protocol in hospital emergency departments (ED) and compare it to current clinical practice in Spain. MATERIAL AND METHODS: We estimated the cumulative incidence of human immunodeficiency virus (HIV) infections and associated costs in Spain for a 20-year time horizon based on a model comprised of various health states with different risks for HIV transmission. The impact of current clinical practices in Spain, in which there is no established protocol for HIV screening, was compared to the potential impact of applying a targeted screening protocol in persons who come to the ED with certain conditions suggestive of HIV infection (diagnosis of a sexually transmitted infection, mononucleosis, herpes zoster infection, community-acquired pneumonia; practice of chemsex, and need for postexposure prophylaxis). RESULTS: Screening all persons with a condition suggestive of HIV infection in hospital EDs would require an investment of €20 million over 20 years, but it would prevent 13 615 new infections (reducing the incidence by 20.6%, down from 66 265 to 52 650 cases) in comparison with the current diagnostic approaches. Such a reduction in the incidence of HIV infection would potentially save €4411 million over 20 years, giving a return of €224 per euro invested. CONCLUSION: A protocol for targeted screening of persons in circumstances suggestive of risk for HIV infection in Spain would increase diagnoses, avert new infections, and generate savings in comparison with screening practices currently in effect.

OBJETIVO: El objetivo del análisis fue evaluar el impacto epidemiológico y económico de protocolizar el cribado dirigido del virus de la inmunodeficiencia humana (VIH) en los servicios de urgencias hospitalarios (SUH) comparado con la actual práctica clínica en España. METODO: Mediante un modelo formado por varios estados de salud con diferentes riesgos de transmisión se estimó la incidencia acumulada de infecciones por VIH y los costes asociados, en 20 años, en España. El análisis comparó la protocolización del cribado dirigido a personas que presentan alguna condición indicadora (CI) de infección por VIH (diagnóstico de enfermedad de transmisión sexual, síndrome mononucleósido, herpes zóster, neumonía adquirida en la comunidad, práctica del chemsex y profilaxis postexposición) que acuden a los SUH frente a la actual práctica clínica en España en la que el cribado del VIH no está protocolizado. RESULTADOS: El cribado dirigido a personas con alguna CI de VIH en los servicios de urgencias requeriría una inversión de 20 millones de euros en 20 años, pero evitaría 13.615 nuevas infecciones (de 66.265 a 52.650 casos; ­20,6%) comparado con la actual estrategia de diagnóstico. La reducción de la incidencia de VIH supondría unos ahorros potenciales de 4.411 millones de euros en 2 décadas, con un retorno económico de 224 € por euro invertido. CONCLUSIONES: Protocolizar el cribado dirigido a personas con alguna CI de VIH en los SUH en España podría incrementar el diagnóstico, evitar nuevas infecciones de VIH y generar ahorros versus el cribado no protocolizado realizado en la práctica clínica actual.

Cribado dirigido del virus de la inmunodeficiencia humana en los servicios de urgencias en España: análisis de las consecuencias epidemiológicas y económicas / Targeted screening for human immunodeficiency virus infection in Spanish emergency departments: an analysis of epidemiologic and economic impact

Objetivo. El objetivo del análisis fue evaluar el impacto epidemiológico y económico de protocolizar el cribado dirigi- do del virus de la inmunodeficiencia humana (VIH) en los servicios de urgencias hospitalarios (SUH) comparado con la actual práctica clínica en España. Método. Mediante un modelo formado por varios estados de salud con diferentes riesgos de transmisión se estimó la incidencia acumulada de infecciones por VIH y los costes asociados, en 20 años, en España. El análisis comparó la protocolización del cribado dirigido a personas que presentan alguna condición indicadora (CI) de infección por VIH (diagnóstico de enfermedad de transmisión sexual, síndrome mononucleósido, herpes zóster, neumonía adquirida en la comunidad, práctica del chemsex y profilaxis postexposición) que acuden a los SUH frente a la actual práctica clínica en España en la que el cribado del VIH no está protocolizado. Resultados. El cribado dirigido a personas con alguna CI de VIH en los servicios de urgencias requeriría una inversión de 20 millones de euros en 20 años, pero evitaría 13.615 nuevas infecciones (de 66.265 a 52.650 casos; –20,6%) comparado con la actual estrategia de diagnóstico. La reducción de la incidencia de VIH supondría unos ahorros potenciales de 4.411 millones de euros en 2 décadas, con un retorno económico de 224 € por euro invertido. Conclusiones. Protocolizar el cribado dirigido a personas con alguna CI de VIH en los SUH en España podría incrementar el diagnóstico, evitar nuevas infecciones de VIH y generar ahorros versus el cribado no protocolizado realizado en la práctica clínica actual.

Objective. To evaluate the potential epidemiologic and economic impact of applying an HIV screening protocol in hospital emergency departments (ED) and compare it to current clinical practice in Spain. Methods. We estimated the cumulative incidence of human immunodeficiency virus (HIV) infections and associated costs in Spain for a 20-year time horizon based on a model comprised of various health states with different risks for HIV transmission. The impact of current clinical practices in Spain, in which there is no established protocol for HIV screening, was compared to the potential impact of applying a targeted screening protocol in persons who come to the ED with certain conditions suggestive of HIV infection (diagnosis of a sexually transmitted infection, mononucleosis, herpes zoster infection, community-acquired pneumonia; practice of chemsex, and need for postexposure prophylaxis). Results. Screening all persons with a condition suggestive of HIV infection in hospital EDs would require an investment of €20 million over 20 years, but it would prevent 13615 new infections (reducing the incidence by 20.6%, down from 66265 to 52650 cases) in comparison with the current diagnostic approaches. Such a reduction in the incidence of HIV infection would potentially save €4411 million over 20 years, giving a return of €224 per euro invested. Conclusion. A protocol for targeted screening of persons in circumstances suggestive of risk for HIV infection in Spain would increase diagnoses, avert new infections, and generate savings in comparison with screening practices currently in effect.

Cost-minimization analysis of immunoglobulin treatment of primary immunodeficiency diseases in Spain.

Primary immunodeficiency diseases (PID), which are comprised of over 400 genetic disorders, occur when a component of the immune system is diminished or dysfunctional. Patients with PID who require immunoglobulin (IG) replacement therapy receive intravenous IG (IVIG) or subcutaneous IG (SCIG), each of which provides equivalent efficacy. We developed a cost-minimization model to evaluate costs of IVIG versus SCIG from the Spanish National Healthcare System perspective. The base case modeled the annual cost per patient of IVIG and SCIG for the mean doses (per current expert clinical practice) over 1 year in terms of direct (drug and administration) and indirect (lost productivity for adults and parents/guardians of pediatric patients) costs. It was assumed that all IVIG infusions were administered in a day hospital, and 95% of SCIG infusions were administered at home. Drug costs were calculated from ex-factory prices obtained from local databases minus the mandatory deduction. Costs were valued on 2018 euros. The annual modeled costs were €4,266 lower for patients with PID who received SCIG (total €14,466) compared with those who received IVIG (total €18,732). The two largest contributors were differences in annual IG costs as a function of dosage (- €1,927) and hospital administration costs (- €2,688). However, SCIG incurred training costs for home administration (€695). Sensitivity analyses for two dose-rounding scenarios were consistent with the base case. Our model suggests that SCIG may be a cost-saving alternative to IVIG for patients with PID in Spain.

Cost Analysis of FreeStyle Libre® 2 System in Type 2 Diabetes Mellitus Population.

INTRODUCTION: FreeStyle Libre® 2 system is a sensor-based flash-monitoring system that measures interstitial fluid glucose. The study aimed to compare cost of FreeStyle Libre 2 system and self-monitoring of blood glucose (SMBG) in the type 2 diabetes mellitus (T2DM) population from the Spanish Health System perspective. METHODS: On the basis of data collected from a literature review, the cost of glucose monitoring was modelled for patients with T2DM on a basal-bolus insulin regimen. The cost estimate included annual consumption for glucose monitoring (strips, lancets and sensors) and severe hypoglycaemic events (SHE) management. A published rate of SHE (2.5 episodes/patient-year) was considered. A reduction of SHE (- 48.8%) associated with FreeStyle Libre 2 system, derived from the REPLACE trial, was applied. Hospital attendance for 20.5% of SHEs (with subsequent hospitalization in 16.0%) was applied. Consumption of strips and lancets was set at 6/day for SMBG (derived from national monitoring recommendations), and 0.2/day for FreeStyle Libre 2 system users, with 26 FreeStyle Libre 2 sensors/year. Unitary costs (€, year 2020 excluding VAT) were derived from literature (€0.28/strip; €0.09/lancet; €3.09/daily FM sensor; €3804/hospitalized SHE; €1794/hospital-attended non-admitted SHE; €389/community-attended SHE). RESULTS: Costs were €2700 and €2120/year/patient using SMBG or FreeStyle Libre 2 system, respectively. For 1000 patients with T2DM using basal-bolus insulin, 1220 SHEs/year (with 48 hospitalizations) could be prevented and FreeSytle Libre 2 system could generate cost savings of up to €580,953/year versus SMBG (- 21.5%). CONCLUSION: FreeStyle Libre 2 system is a potential cost-saving strategy in patients with T2DM in Spain on a basal-bolus insulin regimen.

Cost-Effectiveness Analysis of Gemtuzumab Ozogamicin for First-Line Treatment of Patients with Cd-33 Positive Acute Myeloid Leukaemia in Spain.

OBJECTIVE: To assess the incremental cost-utility ratio (ICUR) of gemtuzumab ozogamicin (GO) + standard of care (SOC) vs SOC alone for treatment of patients with de novo AML from a Spanish Health Service perspective. METHODS: A cohort state-transition model, with 12 health-states, was used to estimate the lifetime accumulated cost and benefits in terms of quality-adjusted-life-years (QALYs) in AML patients with favourable, intermediate, and unknown cytogenetic profiles. Patient profile was defined based on the ALFA-0701 trial. Therapeutic regimens were defined by 5 haematologists. SOC was assumed to be idarubicin and cytarabine, the combination most used in Spain. QALYs were estimated by applying utilities for the time spent by the cohort in each health-state and utility decrements associated with adverse events (AE). Total cost (€,2020) included drug-acquisition, hematologic stem-cell transplantation, disease management, AE management and end-of-life costs. Unit costs were derived from local databases. All parameters were validated by haematologist. Costs and outcomes were discounted (3%/year). RESULTS: Higher cost/patient (€177,618 vs €151,434) and greater QALYs (5,70 vs 4,62) were obtained with GO+SOC vs SOC. The ICUR was €24,203/QALY gained. CONCLUSION: This simulation suggests that GO + SOC could be a cost-effective option for treatment of patients with de novo AML in first line.

Cost analysis of the flash monitoring system (FreeStyle Libre 2) in adults with type 1 diabetes mellitus.

INTRODUCTION: Compare cost of the interstitial liquid glucose flash monitoring (FM) system (FreeStyle Libre 2) versus self-monitoring of blood glucose (SMBG) in adults with type 1 diabetes mellitus (T1DM) in Spain. RESEARCH DESIGN AND METHODS: A model was developed to estimate, with the perspective of the Spanish health system, the annual costs associated with glucose monitoring and hypoglycemic events management in T1DM population, with multiple insulin daily doses (MDI). According to published evidence, rate of severe hypoglycemia (SHE) of 4.90 episodes per patient-year was applied. Reduction of SHE (58.6%) was modeled associated with FM use. Published rates of hospital care (20.2%) and subsequent admission (16%) were assumed for SHE. The daily consumption of strips and lancets was 9 in patients with SMBG (before and after 4 daily intakes and at bedtime) and 0.5 for FM users (according to IMPACT trial findings). Annual consumption of 26 FM sensors was considered (1 every 14 days). Unit costs (in € of 2019, excluding VAT) were obtained from literature and national databases. Sensitivity analyses (SA) were carried out to evaluate the model robustness. RESULTS: The total annual cost/patient was €4437 for SMBG and €2526 for FM. The use of FM would be associated with an annual savings in the costs of monitoring and managing hypoglycemic events of €1911 per patient-year. In a hypothetical cohort of 1000 patients with T1DM MDI, FM could avoid in 1 year 4900 SHE, 93 hospitalizations for SHE. In addition, the use of FM would generate total savings of up to €1 910 000 per year. In the SA with alternative hypoglycemia events rates and use of strips and lancets, and including non-SHE episodes, savings from €370 000 to €1 760 000 were observed with FM. CONCLUSIONS: The use of the FM system to monitor glucose in adults with T1DM treated with MDI, would reduce hypoglycemic events and would result in cost savings for the health system.

Cost-effectiveness analysis of treatment sequences containing tofacitinib for the treatment of rheumatoid arthritis in Spain.

OBJECTIVE: To assess the cost-effectiveness of tofacitinib-containing treatment sequences versus sequences containing only standard biological therapies in patients with moderate-to-severe rheumatoid arthritis (RA) after the failure of conventional synthetic disease-modifying antirheumatic drugs (csDMARD-IR population) and in patients previously treated with methotrexate (MTX) who show an inadequate response to second-line therapy with any tumour necrosis factor inhibitor (TNFi-IR population). METHODS: A patient-level microsimulation model estimated, from the perspective of the Spanish Public NHS, lifetime costs and quality-adjusted life years (QALY) for treatment sequences starting with tofacitinib (5 mg twice daily) followed by biological therapies versus sequences of biological treatments only. Concomitant treatment with MTX was considered. Model's parameters comprised demographic and clinical inputs (initial Health Assessment Questionnaire [HAQ] score and clinical response to short- and long-term treatment). Efficacy was measured by means of HAQ score changes using mixed treatment comparisons and data from long-term extension (LTE) trials. Serious adverse events (SAEs) data were derived from the literature. Total cost estimation (€, 2018) included drug acquisition, parenteral administration, disease progression and SAE management. RESULTS: In the csDMARD-IR population, sequences starting with tofacitinib proved dominant options (more QALYs and lower costs) versus the corresponding sequences without tofacitinib. In the TNFi-IR population, first-line treatment with tofacitinib+MTX followed by scAbatacept+MTX➔rituximab+MTX➔certolizumab+MTX proved dominant versus scTocilizumab+MTX➔scAbatacept+MTX➔rituximab+MTX➔certolizumab+MTX; and tofacitinib+MTX➔scTocilizumab+MTX➔scAbatacept+MTX➔rituximab+MTX versus scTocilizumab+MTX➔scAbatacept+MTX➔rituximab+MTX➔certolizumab+MTX was less effective but remained a cost-saving option. CONCLUSIONS: Inclusion of tofacitinib seems a dominant strategy in moderate-to-severe RA patients after csDMARDs failure. Tofacitinib, as initial third-line therapy, proved a cost-saving strategy (€- 337,489/QALY foregone) in moderate-to-severe TNFi-IR RA patients. Key points • Therapeutical approach in rheumatoid arthritis (RA) consisted in sequences of several therapies during patient lifetime. • Treatment sequences initiating with tofacitinib followed by biological drugs provided higher health effects in csDMARDs-IR population, compared with sequences containing only biological drugs. • In both csDMARD-IR and TNFi-IR RA populations, initiating treatment with tofacitinib was associated to lower treatment costs for the Spanish National Health System.

Cost analysis of the management of brain metastases in patients with advanced ALK+ NSCLC: alectinib versus crizotinib.

AIM: To estimate management cost of NSCLC ALK+ patients with and without brain metastasis (BM), and to compare annual costs in patients treated with alectinib or crizotinib. METHODS: Management cost/year (€ 2018) in patients with and without BM was estimated with disaggregated resource consumption provided by local oncologists, including medical visits, hospitalizations, diagnostic/laboratory tests, imaging techniques and surgical procedures. The comparison of costs/year with alectinib and crizotinib, considered the cumulative 12-month incidence of BM in ALEX trial (9.4 and 41.4%, respectively). RESULTS: Management cost was €6173.42/patient-year without BM and €21,637.50/patient-year with BM. With alectinib, average cost/patient was lower than crizotinib (€4948.51/patient-year). CONCLUSION: Prevention of BM with alectinib may result in reductions of cost/year in the management of advanced ALK+ NSCLC.

Prophylaxis therapy with bypassing agents in patients with haemophilia A and inhibitors undergoing surgery: A cost analysis in Spain.

OBJECTIVES: This study estimated the cost of prophylaxis with activated prothrombin complex concentrate (aPCC) and recombinant activated factor VIIa (rFVIIa) in surgical patients with haemophilia A and inhibitors in Spain. METHODS: A decision-analytic model was developed to estimate the cost to the Spanish National Health System of providing haemostatic coverage in this haemophilia population, with age distribution and average weight derived from the literature, and the annual number of surgeries (0.33 per patient) from local data. Drug costs were calculated from official ex-factory prices with a 7.5% mandatory deduction and recommended dosing regimens. RESULTS: The estimated average costs per patient were €10 100.73 (aPCC) and €14 265.89 (rFVIIa) for dental extraction, €24 043.88 (aPCC) and €62 301.08 (rFVIIa) for minor surgery and €126 595.81 (aPCC) and €347 731.09 (rFVIIa) for major surgery. Assuming an estimated 23 annual surgeries in this population (N = 69), distributed as 19% dental extraction, 50% minor surgery and 31% major surgery, the total annual cost of prophylaxis was €1 209 682.35 with aPCC and €3 221 929.28 with rFVIIa. CONCLUSIONS: aPCC costs were 62.5% lower than rFVIIa. Assuming potential clinical equivalence, aPCC is a potentially cost-saving option for surgical patients with haemophilia A and inhibitors.

Cost-Effectiveness Analysis of Exenatide versus GLP-1 Receptor Agonists in Patients with Type 2 Diabetes Mellitus.

OBJECTIVE: The aim of this study was to assess the efficiency of exenatide 2 mg/week compared with other glucagon-like peptide-1 (GLP-1) receptor agonists (dulaglutide 1.5 mg/week, liraglutide 1.2 mg/day, liraglutide 1.8 mg/day and lixisenatide 20 µg/day) in adult patients with type 2 diabetes mellitus (T2DM) not adequately controlled on metformin alone from the perspective of the Spanish National Health System (NHS). METHODS: Quality-adjusted life-years (QALYs) gained and total costs of each assessed drug combined with metformin (2 g/day) were estimated over a 40-year time horizon using the Cardiff Diabetes Model (based on UK Prospective Diabetes Study [UKPDS] 68 equations), which simulates disease progression considering the T2DM-related micro- and macrovascular complications, hypoglycaemia, nausea, body mass index (BMI) changes and treatment discontinuation due to adverse effects (AEs). Drug efficacy derived from an indirect comparison performed in a network meta-analysis. Patient characteristics were obtained from the literature. The baseline utility value (0.80) was derived from the PANORAMA study, applying utility decrements to micro- and macrovascular complications, hypoglycaemia episodes and changes in BMI. Treatment discontinuation due to AEs or poorly controlled diabetes (HbA1c > 7.5%) involved switching to second-line (basal insulin) or third-line (basal-bolus insulin) treatment. Total cost (€, 2018) included the costs of drug acquisition, hypoglycaemia, weight gain, micro- and macrovascular complications, nausea and treatment discontinuation due to AEs. An annual discount rate of 3% was applied to costs and outcomes. Deterministic and probabilistic sensitivity analyses (SA) were performed. RESULTS: In base-case, exenatide 2 mg/week resulted in more QALYs (8.26) than dulaglutide 1.5 mg/week (8.19 QALYs), liraglutide 1.2 mg/day (8.10 QALYs), liraglutide 1.8 mg/day (8.20 QALYs) and lixisenatide 20 µg/day (8.13 QALYs). Total cost/patient was €20,423.27 (exenatide 2 mg/week), €22,611.94 (dulaglutide 1.5 mg/week), €21,065.97 (liraglutide 1.2 mg/day), €24,865.69 (liraglutide 1.8 mg/day) and €21,334.58 (lixisenatide 20 µg/day). Deterministic SA confirmed the robustness of the model. In the probabilistic SA, 95-99% of the 1000 Monte Carlo iterations performed were under a hypothetical willingness-to-pay threshold of €20,000/QALY gained. CONCLUSIONS: Exenatide 2 mg/week would be a dominant strategy (more effective and less costly) versus the other GLP-1 receptor agonists assessed for the treatment of T2DM patients who are not adequately controlled on metformin alone.

Cost-Effectiveness Analysis of Apixaban Versus Edoxaban in Patients with Atrial Fibrillation for Stroke Prevention.

OBJECTIVE: Our objective was to assess the cost effectiveness of apixaban versus edoxaban in the prevention of stroke and systemic embolism (SE) in patients with atrial fibrillation (AF) in Spain. METHODS: We customized a Markov model with ten health states to estimate the lifetime economic and clinical outcomes in 6-week cycles. The efficacy (clinical event rates per 100 patient-years) and safety data were derived from a pairwise indirect treatment comparison. The analysis was conducted from both the national health service (NHS) and societal perspectives, and included pharmaceutical costs (retail price plus value-added tax (VAT) and applicable national deductions) according to daily dosages (apixaban 10 mg (5 mg twice daily (bid)) and edoxaban 60 or 30 mg) and complications and disease-management costs, obtained from national databases. Utilities for quality-adjusted life-year (QALY) calculations reflected EuroQoL 5-Dimension scores in patients with AF. An annual discount rate of 3% was applied for costs (€, year 2019 values) and outcomes. RESULTS: In a 1000-patient cohort, apixaban 5 mg bid versus edoxaban 60 mg could avoid five strokes, six major bleedings and 29 clinically relevant non-major bleedings (CRNMBs). Compared with edoxaban 30 mg, apixaban could avoid 21 strokes and two SEs. An increase in bleedings was observed with apixaban (seven haemorrhagic strokes, 48 major bleedings and 17 CRNMBs). Apixaban yielded 0.04 additional QALYs compared with edoxaban 60 mg or 30 mg. Incremental costs/QALY were €9639.33 and €354.22 for apixaban versus edoxaban 60 mg and edoxaban 30 mg, respectively, from the NHS perspective and €7756.62 for apixaban versus edoxaban 60 mg from the societal perspective. Apixaban was dominant versus edoxaban 30 mg from the societal perspective. Sensitivity analyses confirmed the robustness of the model. CONCLUSIONS: This study suggests that apixaban 5 mg bid is a cost-effective alternative to edoxaban for stroke prevention in the AF population in Spain.

Second-line treatment with lesinurad and allopurinol versus febuxostat for management of hyperuricemia: a cost-effectiveness analysis for Spanish patients.

INTRODUCTION/OBJECTIVES: Lesinurad, in combination with allopurinol, has been approved for treatment of patients with gout which do not reach therapeutic serum urate target with xanthine oxidase inhibitors monotherapy. The study aimed to assess the incremental cost-effectiveness ratio of adding lesinurad to allopurinol as second-line therapy, compared to febuxostat for patients with gout in Spain. METHOD: A Markov model representing disease evolution was used to estimate the lifetime accumulated cost and benefits in terms of quality-adjusted-life-year (QALY). Patients could either continue with second-line treatment with lesinurad (200 mg/daily) plus allopurinol (400 mg/daily) or febuxostat (80 mg/daily) switch to allopurinol monotherapy (271 mg/daily) in case of intolerance or discontinue treatment. The treatment's efficacy captured in the transition probabilities between health states were derived from CLEAR and EXCEL trials. Quality of life related to gout severity and flare frequency was considered by means of utilities. The total cost estimation (€, 2019) included drug acquisition cost, disease monitoring, and flare management cost. Unitary local costs derived from databases and literature. A 3% annual discount rate was applied for cost and outcomes. RESULTS: Lesinurad plus allopurinol provided higher QALYs (14.79) than febuxostat (14.69). Total accrued cost/patient was lower with lesinurad and allopurinol (€50,631.51) versus febuxostat (€56,698.64). Lesinurad plus allopurinol resulted more effective and less costly (dominant option) versus febuxostat. CONCLUSIONS: Lesinurad plus allopurinol therapy compared with febuxostat seems an effective option for the management of hyperuricemia in patients who did not reach serum urate target to previous allopurinol monotherapy, associated to cost-savings for the Spanish Health System.Key Points• Lesinurad, in combination with allopurinol, has been recently authorized as second-line treatment of hyperuricemia in gout patients.• Lesinurad plus allopurinol provided higher effectiveness in terms of quality-adjusted-life-years (14.79) than febuxostat (14.69).• Lesinurad plus allopurinol resulted less costly (total cost/per patient) compared with febuxostat.• Lesinurad plus allopurinol resulted a dominant option compared with febuxostat.

Análisis de impacto presupuestario del implante intravítreo de dexametasona para el tratamiento del edema macular diabético / Budget impact analysis of dexamethasone intravitreal implant for the treatment of diabetic macular oedema

Objetivo: Determinar el impacto económico tras la inclusión del implante intravítreo de dexametasona para el tratamiento del edema macular diabético en un área sanitaria en España. Método: Se diseñó un modelo de impacto presupuestario a tres años para estimar los costes directos en pacientes adultos con edema macular diabético, desde la perspectiva del Sistema Nacional de Salud, considerando terapias intravítreas actualmente utilizadas (aflibercept/ ranibizumab/dexametasona). La población diana se obtuvo a partir de la prevalencia (6,41%) e incidencia (0,82%) del edema macular diabético publicadas para una población de 25.000 pacientes adultos. Se asumió un 20%, 30% y 40% anual de pacientes tratados con dexametasona, respectivamente. El coste total incluyó: coste farmacológico (precio de venta del laboratorio con deducción obligatoria y fraccionamiento de viales, según frecuencia de inyecciones necesarias cada año de tratamiento), administración intravítrea, seguimiento de pacientes y manejo de eventos oculares (cataratas, hipertensión ocular, endoftalmitis, hemorragia intravítrea y desprendimiento de retina) y cardiovasculares. El consumo de recursos según la práctica habitual fue estimado por expertos en retina y vítreo. Los costes unitarios (Euros 2016) se obtuvieron de la literatura y de bases de datos nacionales. Los análisis de sensibilidad evaluaron la robustez del modelo. Resultados: La inclusión del implante intravítreo de dexametasona supondría reducciones de 35.030 Euros (-4,2%), 10.743 Euros (-1,8%) y 5.051 Euros (-0,9%) cada año, respectivamente, disminuyendo principalmente por el menor número anual de inyecciones requeridas con dexametasona. La reducción anual promedio supondría 350 Euros, 96 Euros y 41 Euros por paciente

Objective: To assess the economic impact following the inclusion of an intravitreal implant of dexamethasone for the treatment of diabetic macular oedema in a healthcare area in Spain. Method: A 3-year budget impact model was designed to estimate healthcare direct costs for adult patients with diabetic macular oedema from the National Health System perspective. The approved therapies in use (aflibercept/ranibizumab/dexamethasone) were considered. The target population was estimated from published diabetic macular oedema prevalence (6.41%) and incidence (0.82%) for a population of 25,000 adults. Dexamethasone was assumed to be used annually in 20%, 30% and 40% of patients, respectively. Annual total costs included: drug acquisition (based on frequency of injections per every year, considering exfactory prices with mandatory deduction and split of vials), intravitreal administration, patient monitoring, management of cardiovascular and ocular adverse events (cataracts, increased intraocular pressure, endophthalmitis, vitreous haemorrhage and retinal detachment). Detailed resource consumption reflecting clinical practice was provided from local experts in retina and vitreous. Unitary costs (Euros 2016) were obtained from national databases and literature. Sensitivity analyses were performed to assess model robustness. Results: The inclusion of intravitreal dexamethasone implant would lead to annual cost savings of Euros 35,030 (-4.2%), Euros 10,743 (-1.8%) and Euros 5,051 (-0.9%), years 1-3 respectively. Total costs were reduced mainly by the fewer annual injections required by dexamethasone. The average annual incremental costs were - Euros 350, -Euros 96 and -Euros 41 per patient

Budget impact analysis of dexamethasone intravitreal implant for the treatment of diabetic macular oedema.

OBJECTIVE: To assess the economic impact following the inclusion of an intravitreal implant of dexamethasone for the treatment of diabetic macular oedema in a healthcare area in Spain. METHOD: A 3-year budget impact model was designed to estimate healthcare  direct costs for adult patients with diabetic macular oedema from the National  Health System perspective. The approved therapies in use  (aflibercept/ranibizumab/dexamethasone) were considered. The target  population was estimated from published diabetic macular oedema prevalence  (6.41%) and incidence (0.82%) for a population of 25,000 adults.  Dexamethasone was assumed to be used annually in 20%, 30% and 40% of  patients, respectively. Annual total costs included: drug acquisition (based on  frequency of injections per every year, considering exfactory prices with  mandatory deduction and split of vials), intravitreal administration, patient  monitoring, management of cardiovascular and ocular adverse events  (cataracts, increased intraocular pressure, endophthalmitis, vitreous  haemorrhage and retinal detachment). Detailed resource consumption reflecting  clinical practice was provided from local experts in retina and vitreous. Unitary  costs (€, 2016) were obtained from national databases and literature. Sensitivity  analyses were performed to assess model robustness. RESULTS: The inclusion of intravitreal dexamethasone implant would lead to  annual cost savings of €35,030 (-4.2%), €10,743 (-1.8%) and €5,051 (-0.9%), years 1-3 respectively. Total costs were reduced mainly by the fewer annual  injections required by dexamethasone. The average annual incremental costs  were -€350, -€96 and -€41 per patient. CONCLUSIONS: The inclusion of an intravitreal dexamethasone implant for the  treatment of diabetic macular oedema would lead to cost-savings for the  considered health area, mainly by reducing the administration costs.

Objetivo: Determinar el impacto económico tras la inclusión del implante intravítreo de dexametasona para el tratamiento del edema macular diabético en un área sanitaria en España.Método: Se diseñó un modelo de impacto presupuestario a tres años para  estimar los costes directos en pacientes adultos con edema macular diabético,  desde la perspectiva del Sistema Nacional de Salud, considerando terapias  intravítreas actualmente utilizadas (aflibercept/ranibizumab/dexametasona). La  población diana se obtuvo a partir de la prevalencia (6,41%) e incidencia  (0,82%) del edema macular diabético publicadas para una población de 25.000  pacientes adultos. Se asumió un 20%, 30% y 40% anual de pacientes tratados  con dexametasona, respectivamente. El coste total incluyó: coste farmacológico  (precio de venta del laboratorio con deducción obligatoria y fraccionamiento de  viales, según frecuencia de inyecciones necesarias cada año de tratamiento),  administración intravítrea, seguimiento de pacientes y manejo de eventos  oculares (cataratas, hipertensión ocular, endoftalmitis, hemorragia intravítrea y  desprendimiento de retina) y cardiovasculares. El consumo de recursos según la  práctica habitual fue estimado por expertos en retina y vítreo. Los costes  unitarios (€, 2016) se obtuvieron de la literatura y de bases de datos nacionales.  Los análisis de sensibilidad evaluaron la robustez del modelo. Resultados: La inclusión del implante intravítreo de dexametasona supondría reducciones de 35.030 € (­4,2%), 10.743 € (­1,8%) y 5.051 € (­ 0,9%) cada año, respectivamente, disminuyendo principalmente por el menor  número anual de inyecciones requeridas con dexametasona. La reducción anual  promedio supondría 350 €, 96 € y 41 € por paciente.Conclusiones: La inclusión del implante intravítreo de dexametasona para el  tratamiento del edema macular diabético supone ahorros para el área sanitaria  considerada, fundamentalmente por la reducción de costes de administración.