Cost-effectiveness of weekly gastro-resistant risedronate 35 mg, compared with weekly alendronate 70 mg tablets, in the treatment of postmenopausal osteoporosis in Spain.

Aim: To estimate the cost-effectiveness of treating postmenopausal osteoporosis (PMO) with weekly gastro-resistant risedronate 35 mg gastro-resistant tablets (RIS-GR), compared with weekly alendronate 70 mg tablets (ALN) in Spain. Methods: A probabilistic analysis (second-order Monte Carlo simulation) was performed with a time horizon of 5 years, from the perspective of the Spanish National Health System. The bone fracture probabilities were obtained from a cohort study of 3614 women from USA with PMO treated with RIS-GR (1807) or ALN (1807) (Thomasius, 2022). The pharmacological cost and the cost of fractures were obtained from Spanish sources (€ 2022). The utilities of patients with and without fracture (quality-adjusted life years [QALYs]) were obtained from the medical literature. Results: Compared with ALN, treatment with RIS-GR can avoid 79 fractures (between 75 and 82) every 1000 patients treated, and 0.0119 QALYs would be gained (between 0.0098 and 0.0140) per patient. Additionally, GR-RIS would generate a cost saving per patient of €1994 (€1437-2904) with a probability of 99.7%. The scenario analyses confirmed the stability of the base case results. Conclusion: According to this study, RIS-GR would be the dominant treatment (lower costs with QALY gain) compared with ALN.

Cost-effectiveness of apixaban and rivaroxaban in thromboprophylaxis of cancer patients treated with chemotherapy in Spain.

BACKGROUND: Apixaban and rivaroxaban are two direct-acting oral anticoagulants (DOACs) recommended for thromboprophylaxis in cancer patients treated with chemotherapy in an ambulatory setting. We aimed to assess the cost-utility of thromboprophylaxis with apixaban and rivaroxaban vs no thromboprophylaxis in ambulatory cancer patients starting chemotherapy with an intermediate-to-high risk of venous thromboembolism (VTE), Khorana score ≥ 2 points. METHODS: A cost-effectiveness analysis was performed from the perspective of Spain's National Health System (NHS) using an analytical decision model in the short-term (180 days) and a Markov model in the long-term (5 years). Transition probabilities were obtained from randomized, double-blind, placebo-controlled clinical trials of apixaban and rivaroxaban in adult ambulatory patients with cancer at risk for VTE, treated with chemotherapy (AVERT and CASSINI trials). The costs (€2,021) were taken from Spanish sources. The utilities of the model were obtained through the EQ-5D questionnaire. Deterministic (base case) and probabilistic (second-order Monte Carlo simulation) analyses were conducted. RESULTS: In the probabilistic sensitivity analysis, apixaban generated a cost per patient of €1,082 ± 187, with a 95% confidence interval (CI) of €713-1,442, while no prophylaxis produced a cost per patient of €1,146 ± 218, with a 95% CI of €700-1,491, with a saving of €64 per patient and a gain of 0.008 QALYs. Likewise, rivaroxaban provided a cost per patient of €993 ± 133, with a 95% CI of €748-1,310, while no prophylaxis produced a cost per patient of €872 ± 152, with a 95% CI of €602-1,250, with an additional expense of €121 per patient and a gain of 0.008 QALYs. CONCLUSIONS: In thromboprophylaxis of cancer patients, the use of apixaban and rivaroxaban generated similar costs compared to non-prophylaxis, without the difference found being statistically significant, with a clinically insignificant QALY gain.

Economic Impact Associated with Patients with Overactive Bladder on Drug Treatment with Mirabegron or Antimuscarinics in Spain

Objective: To assess the economic impact associated with overactive bladder (OAB) patients, treated with mirabegron or antimuscarinics (AM) in Spain, over a 12-month period. Methods: A probabilistic model (second-order Monte Carlo simulation) was used in a hypothetical cohort of 1000 patients with OAB and a time horizon of 12 months. The use of resources was obtained from the retrospective observational study MIRACAT that included 3330 patients with OAB. The analysis was carried out from the perspective of the National Health System (NHS) including that of society with the indirect cost of abseenteism in a sensitivity analysis. Unit costs were obtained from Spanish public healthcare prices (€ 2021) and from previously published Spanish studies. Results: The annual average savings for the NHS for each patient with OAB treated with mirabegron would be € 1135 (95%confidence interval (CI) € 390; 2421) compared with a patient treated with AM. Annual average savings were maintained in all the sensitivity analyses carried out, ranging from a minimum of € 299 to a maximum of € 3381 per patient. The substitution of 25% of the AM treatments (for 81534 patients) to mirabegron would generate, within 1 year, savings for the NHS of € 92 million (95% CI € 31; 197 million). Conclusions: According to the present model, the treatment of OAB with mirabegron would generate savings compared with treatment with AM in all scenarios and sensitivity analysis performed, and for the NHS and for society perspectives (AU)

Cost-effectiveness of cladribine tablets and dimethyl fumarate in the treatment of relapsing remitting multiple sclerosis in Spain.

Aim: To analyze the cost-effectiveness of treatment of relapsing remitting multiple sclerosis (RRMS) with cladribine tablets (CladT) and dimethyl fumarate (DMF) from the perspective of the Spanish National Health System (NHS). Methods: A probabilistic Markov model (second-order Monte Carlo simulation) with a 10-year time horizon and annual Markov cycles was performed. Results: CladT was the dominant treatment, with lower costs (-74,741 € [95% CI: -67,247; -85,661 €]) and greater effectiveness (0.1920 [95% CI: -0.1659; 0.2173] QALY) per patient, compared with DMF. CladT had a 95.1% probability of being cost-effective and a 94.1% chance of being dominant compared with DMF. Conclusion: CladT is the dominant treatment (lower costs, with more QALYs) compared with DMF in the treatment of RRMS in Spain.

Comparison of the design and methodology of Phase 3 clinical trials of bictegravir/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF) and dolutegravir-based dual therapy (DTG) in HIV: a systematic review of the literature.

INTRODUCTION: Current recommended antiretroviral regimens include a combination of two (dual; DT) or three (triple; TT) antiretroviral drugs. This study aims to determine whether the quality of evidence from clinical trials of dolutegravir (dolutegravir/lamivudine [DTG/3TC] or dolutegravir/rilpivirine [DTG/RPV]) is methodologically comparable to that of clinical trials conducted with bictegravir/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF). AREAS COVERED: A systematic review of the medical literature was carried out in PubMed without date or language restrictions, following the PRISMA guidelines. All aspects of the methodological design of phase 3 randomized clinical trials (RCTs) of DT and TT, evaluated by the European Medicines Agency (registration trials), were reviewed. The quality of clinical trials was assessed using the Jadad scale. EXPERT OPINION: The search identified 5, 3 and 2 phase 3 RCTs with BIC/FTC/TAF, DTG/3TC and DTG/RPV, respectively, that met the inclusion criteria. The designs would not be comparable due to differences in pre-randomization losses, blinding, patient recruitment, as well as differences in methodological quality, with the average score of the RCTs conducted with BIC/FTC/TAF, DTG/3TC and DTG/RPV being 4.2 (high quality), 3.0 (medium quality) and 3.0 (medium quality), respectively. Due to methodological differences between the BIC/FTC/TAF, DTG/3TC and DTG/RPV RCTs, the results of these are not comparable.

Beneficio clínico y económico de 32 años de tratamiento antirretroviral de personas que viven con VIH en España: ¿ha sido una intervención eficiente? / Clinical and economic benefit of 32 years of antiretroviral treatment for people living with HIV in Spain: Has it been an efficient intervention?

Introducción: Aunque el tratamiento antirretroviral (TAR) del VIH/sida se introdujo en 1987, la mejora en la progresión de la enfermedad y reducción de la mortalidad poblacional no se observó sino hasta 1996, con la combinación de tres o más fármacos. El objetivo fue estimar el beneficio clínico y económico del TAR en España en el periodo de 32 años, comprendido entre 1987 y 2018. Métodos: Se realizó un análisis de coste-beneficio mediante la simulación de Monte Carlo de segundo orden, desde las perspectivas de la sociedad (caso base) y el Sistema Nacional de Salud (SNS). Los nuevos casos de VIH, sida y muertes relacionadas se obtuvieron de los registros SINIVIH y ONUSIDA, con proyecciones poblacionales sin TAR mediante suavizamiento exponencial triple. El gasto en TAR se obtuvo de informes del Plan Nacional del SIDA y estudios de mercado. Resultados: El SNS invirtió 6.185 millones de euros en 32 años. Durante este periodo se evitaron 323.651 muertes por sida, 500.129 casos de sida y 161.417 casos de VIH, con un ahorro total de 41.997 millones de euros. El beneficio neto (ahorros netos) se estima en 35.812 millones de euros (sociedad) y 1.032 millones de euros (SNS). Por cada euro invertido en TAR, se obtuvo un retorno de la inversión de 6,79 € y 1,16 €, respectivamente. Conclusión: La utilización de TAR durante 32 años ha evitado gran número de muertes y casos de sida y VIH, generando significativos ahorros económicos para el SNS. El TAR es una intervención eficiente para el SNS.(AU)

Introduction: Although antiretroviral therapy (ART) for HIV / AIDS was introduced in 1987, improvement in disease progression and reduction in mortality at a population level was not observed until 1996, with the combination of three or more drugs. The objective was to estimate the clinical and economic benefit of ART in Spain in the 32-year period between 1987 and 2018. Methods: A cost-benefit analysis was performed, using a second-order Monte Carlo simulation, from the societal (base case) and the National Health System (NHS) perspectives. New cases of HIV, AIDS and related deaths were obtained from the SINIVIH and UNAIDS registries, with population projections without ART using triple exponential smoothing. Expenditure on ART was obtained from the National AIDS Plan reports and market studies. Results: The NHS invested 6,185 million euros in 32 years. In that period, 323,651 AIDS-related deaths, 500,129 AIDS cases and 161,417 HIV cases were averted, with total savings of 41,997 million euros. The net benefit (net savings) is estimated at 35,812 million euros (societal) and 1,032 million euros (NHS). For every euro invested in ART, a return on investment of € 6.79 and € 1.16 was obtained, respectively. Conclusion: The use of ART over 32 years prevented a large number of deaths and cases of AIDS and HIV, providing significant economic savings for the NHS. ART is an efficient intervention for the NHS.(AU)

Clinical and economic benefit of 32 years of antiretroviral treatment for people living with HIV in Spain: Has it been an efficient intervention?

INTRODUCTION: Although antiretroviral therapy (ART) for HIV/AIDS was introduced in 1987, improvement in disease progression and reduction in mortality at a population level was not observed until 1996, with the combination of three or more drugs. The objective was to estimate the clinical and economic benefit of ART in Spain in the 32-year period between 1987 and 2018. METHODS: A cost-benefit analysis was performed, using a second-order Monte Carlo simulation, from the societal (base case) and the National Health System (NHS) perspectives. New cases of HIV, AIDS and related deaths were obtained from the SINIVIH and UNAIDS registries, with population projections without ART using triple exponential smoothing. Expenditure on ART was obtained from the National AIDS Plan reports and market studies. RESULTS: The NHS invested 6185 million euros in 32 years. In that period, 323,651 AIDS-related deaths, 500,129 AIDS cases and 161,417 HIV cases were averted, with total savings of 41,997 million euros. The net benefit (net savings) is estimated at 35,812 million euros (societal) and 1032 million euros (NHS). For every euro invested in ART, a return on investment of € 6.79 and € 1.16 was obtained, respectively. CONCLUSIONS: The use of ART over 32 years prevented a large number of deaths and cases of AIDS and HIV, providing significant economic savings for the NHS. ART is an efficient intervention for the NHS.

Clinical and economic benefit of 32 years of antiretroviral treatment for people living with HIV in Spain: Has it been an efficient intervention? / Beneficio clínico y económico de 32 años de tratamiento antirretroviral de personas que viven con VIH en España: ¿ha sido una intervención eficiente?

INTRODUCTION: Although antiretroviral therapy (ART) for HIV / AIDS was introduced in 1987, improvement in disease progression and reduction in mortality at a population level was not observed until 1996, with the combination of three or more drugs. The objective was to estimate the clinical and economic benefit of ART in Spain in the 32-year period between 1987 and 2018. METHODS: A cost-benefit analysis was performed, using a second-order Monte Carlo simulation, from the societal (base case) and the National Health System (NHS) perspectives. New cases of HIV, AIDS and related deaths were obtained from the SINIVIH and UNAIDS registries, with population projections without ART using triple exponential smoothing. Expenditure on ART was obtained from the National AIDS Plan reports and market studies. RESULTS: The NHS invested 6,185 million euros in 32 years. In that period, 323,651 AIDS-related deaths, 500,129 AIDS cases and 161,417 HIV cases were averted, with total savings of 41,997 million euros. The net benefit (net savings) is estimated at 35,812 million euros (societal) and 1,032 million euros (NHS). For every euro invested in ART, a return on investment of € 6.79 and € 1.16 was obtained, respectively. CONCLUSION: The use of ART over 32 years prevented a large number of deaths and cases of AIDS and HIV, providing significant economic savings for the NHS. ART is an efficient intervention for the NHS.

Health and economic impact of the correct diagnosis of transthyretin cardiac amyloidosis in Spain.

Objective: To estimate the health and economic impact of the reduction in mortality and cardiovascular hospitalizations, associated with correct diagnosis of cardiac transthyretin amyloidosis (ATTR-CM), from the Spanish National Health System (NHS) perspective.Methods: A costs and effects analysis were performed (probabilistic Markov model) with time horizons between 1 and 15 years, comparing the correct diagnosis of ATTR-CM versus the non-diagnosis. Transition probabilities were obtained from the ATTR-ACT study (placebo arm) and from the literature. Costs and healthcare resources were obtained from Spanish sources (€ 2019) and from a panel of Spanish clinical experts.Results: After 1, 5, 10 and 15 years, the diagnosis of ATTR-CM would generate a gain of 0.031 (95%CI 0.025; 0.038); 0.387 (95%CI 0.329; 0.435); 0.754 (95%CI 0.678; 0.781) and 0.944 (95%CI 0.905; 0.983) life years per patient, respectively, with savings of € 212 (95%CI € -632; 633), € 2,289 (95%CI € 2,250; 2,517), € 2,859 (95%CI € 2,584; 3,149) and € 2,906 (95%CI € 2,669; 3,450) per patient, respectively, versus the non-diagnosis.Conclusions: Just by correctly diagnosing ATTR-CM, years of life would be gained, cardiovascular hospitalizations would be avoided, and savings would be generated for the NHS, compared to the non-diagnosis of the disease.

Cost of patients with hemophilia A treated with standard half-life or extended half-life FVIII in Spain.

BACKGROUND: In a real-world analysis (RWA) conducted in the United States (US), median international units (IUs) of extended half-life (EHL) recombinant coagulation factor VIII (rFVIII) dispensed were 10% to 45% greater than standard half-life (SHL) rFVIII. The mean IUs of each rFVIII dispensed quarterly were obtained from two databases (N = 776). METHODS: A probabilistic model in a 1-year time horizon was used in order to analyze the cost comparison of SHL and EHL rFVIII products in Spain. In this analysis, mean IUs were those of the RWA, and frequency of use and prices for each rFVIII were obtained from sales estimates based on Spanish sources (IQVIA; €, 2019). RESULTS: Data showed an average annual savings per patient of €11,227 for SHL rFVIII versus EHL rFVIII products, with a savings probability of 75.5%. The results were stable in the sensitivity analyses. Not switching treatment from SHL to EHL rFVIII resulted in greater savings per patient (€53,078), with a savings probability of 99.9%. Considering the frequency of rFVIII dispensation in the US, annual savings per patient would increase to €16,350 in Spain, with a savings probability of 79.9%. CONCLUSIONS: According to this model, use of SHL rFVIII versus EHL rFVIII products could lead to savings for the Spanish National Health System.

Cost of fremanezumab, erenumab, galcanezumab and onabotulinumtoxinA associated adverse events, for migraine prophylaxis in Spain.

Objective: To compare the cost of adverse events (AEs) associated with preventive treatment of migraine with fremanezumab, versus erenumab, galcanezumab, and onabotulinumtoxinA.Methods: A probabilistic modeling analysis was performed, using second-order Monte Carlo simulations, with 1,000 simulations, in patients with at least 4 days of migraine per month, from the perspective of the National Health System and a time horizon of 12 weeks. The frequency of AEs described in the clinical trials was analyzed with 12 weeks of treatment. Unit costs (€) of management of AEs were obtained from public health prices, expert panels, and published Spanish studies.Results: Fremanezumab would generate average savings of -€469 (95% CI -€303; -€674) versus erenumab, -€268 (95% CI -€171; -€391) versus galcanezumab, -€1,100 (95% CI -€704; -€1,608) or -€1,295 (95% CI -€835; -€1,893) versus onabotulinumtoxinA using real-life or clinical trial data, respectively.Conclusions: The different safety profile of treatment with fremanezumab, compared to erenumab, galcanezumab, and onabotulinumtoxinA, would generate savings in health-care resources in all the scenarios considered.

Analysis of the Health and Budgetary Impact of Chondroitin Sulfate Prescription in the Treatment of Knee Osteoarthritis Compared to NSAIDs and COXIBs.

BACKGROUND: Chondroitin sulfate, alone or associated with glucosamine (CS), is an effective treatment of osteoarthritis, better tolerated than non-steroidal anti-inflammatory drugs (NSAIDs) and cyclooxygenase 2 inhibitors (COXIBs) at gastrointestinal, cardiovascular and renal levels. OBJECTIVE: To estimate the health impact (toxicity by NSAIDs/COXIBs avoided with CS with or without glucosamine) and economic impact (savings due to avoided toxicities) of treatment of knee osteoarthritis with CS compared to NSAIDs/COXIBs, as a consequence of the avoidance of mild-moderate or severe gastrointestinal adverse effects (GIAE), ischaemic heart disease (IHD), acute kidney insufficiency (AKI) and chronic kidney failure (CKF). METHODS: We compared the current situation (available reimbursed prescription with CS) with a hypothetical situation without CS (treatment only with NSAIDs/COXIBs). The frequency of GIAE, IHD, AKI and CKF with CS and NSAIDs/COXIBs was obtained from published ad hoc studies. The cost of AE management and of the drugs (180 days of treatment) was obtained from Spanish sources. A probabilistic economic model was made for a 3-year period, both at national (NHS) and regional levels. Sensitivity analyses were performed for different durations of treatment (90 and 240 days). RESULTS: In Spain, it is estimated that 519,130, 513,616 and 507,377 patients with knee osteoarthritis will be treated with NSAIDs/COXIBs and 112,775, 114,963 and 117,262 with CS in 2020, 2021 and 2022, respectively. Due to better CS tolerability, 55,098 mild-moderate GIAE, 3060 severe GIAE, 204 IHD, 1089 AKI and 733 CKF would be avoided in 3 years. Discounting the cost of the drugs, the three-year savings for the NHS would be 21.8 (12.7-29.5) million euros. CONCLUSION: Due to its better tolerability profile, CS treatment is expected to prevent thousands of AEs over the next 3 years, some of which may be life-threatening for patients, while generating considerable savings for the NHS.

Análisis de costes y consecuencias relacionados con la persistencia del tratamiento con Mirabegrón y los fármacos antimuscarínicos y su impacto sobre la calidad de vida en los pacientes con vejiga hiperactiva en España: resultados de un modelo probabilístico / Analysis of costs and consequences related to the persistence of Mirabegron and antimuscarinic treatments and their impact on quality of life in patients with overactive bladder in Spain: Results of a probabilistic model

OBJETIVO: Evaluar si la menor tasa de abandonos del tratamiento de la vejiga hiperactiva (VH) con mirabegrón podría generar ahorros para el Sistema Nacional de Salud (SNS) y ganancia de años de vida ajustados por calidad (AVACs), en comparación con los fármacos antimuscarínicos (AM) (tolterodina, fesoterodina, oxibutinina, solifenacina). MÉTODOS: Modelo probabilístico (simulación de Monte Carlo de segundo orden) en una cohorte hipotética de 1.000 pacientes con VH y un horizonte temporal de 1 año. Las tasas de abandono/persistencia del tratamiento con mirabegrón y AM se obtuvieron de un estudio observacional español en 1.798 pacientes. Los costes unitarios (€ 2018) y la pérdida de utilidades ligada al abandono del tratamiento se obtuvieron de precios públicos españoles y de la literatura, respectivamente. RESULTADOS: En cada paciente tratado con mirabegrón se duplica la tasa de persistencia en comparación con los AM, ganándose anualmente 0,0151 ±0,0007 AVACs, frente a AM. Con mirabegrón se generaría un ahorro anual por paciente de 80,74 ± 4,61 € en comparación con los AM, con una probabilidad de ahorro del 100%. La sustitución hipotética de los AM por mirabegrón, generaría en el plazo de 1 año un ahorro para el SNS de 6,6 millones de euros (IC 95%3,9-10,1 millones de euros) y se ganarían 1.238 AVAC (IC95% 731; 1.885 AVAC). CONCLUSIONES: El modelo probabilístico muestra una mayor persistencia en pacientes tratados con mirabegrón en comparación con los AM, generando un impacto positivo sobre la calidad de vida de los pacientes así como ahorros para el SNS

OBJECTIVE: To evaluate whether the lower dropout rate of the treatment of overactive bladder (OAB) with mirabegron could generate cost savings to the National Health System (NHS) and lead to quality-adjusted life years (QALYs) gains, compared to the most commonly prescribed antimuscarinics (AM) in Spain (tolterodine, fesoterodine, oxybutynin, solifenacin). METHODS: A probabilistic model (second order Monte Carlo simulation) in a hypothetical cohort of 1,000 patients with OAB and a time horizon of 1 year was carried out. Discontinuation and persistence rates for both mirabegron and AM were obtained from a Spanish observational study in 1798 patients. Unit costs (€ 2018) and utility loss associated with treatment discontinuation were obtained from Spanish public prices and literature, respectively. RESULTS: Persistence rates in patients treated with mirabegron were twice as high compared to AM, leading to a QALY gain of 0.0151 ± 0.0007 per year. Treatment with mirabegron could generate savings of 80.74 ± 4.61 € per patient per year compared to AM, assuming 100% probability of saving. The hypothetical substitution of AM treatment for mirabegron could potentially generate savings of 6.6 million euros (95% CI 3.9-10.1 million euros) to the NHS and 1,238 QALYs gains (CI 95% 731; 1,885 QALYs) within a period of 1 year. CONCLUSIONS: The probabilistic model presented showed a greater persistence in patients treated with mirabegron compared to AM, leading to a positive impact in patients quality of life, as well cost savings to the NHS in Spain

[Analysis of costs and consequences related to the persistence of Mirabegron and antimuscarinic treatments and their impact on quality of life in patients with overactive bladder in Spain: Results of a probabilistic model.] / Análisis de costes y consecuencias relacionados con la persistencia del tratamiento con Mirabegrón y los fármacos antimuscarínicos y su impacto sobre la calidad de vida en los pacientes con vejiga hiperactiva en España: resultados de un modelo probabilístico.

OBJECTIVE: To evaluate whether the lower dropout rate of the treatment of overactive bladde r(OAB) with mirabegron could generate cost savings to the National Health System (NHS) and lead to quality-adjusted life years (QALYs) gains, compared to the most commonly prescribed antimuscarinics (AM) in Spain (tolterodine, fesoterodine, oxybutynin, solifenacin). METHODS: A probabilistic model (second order Monte Carlo simulation) in a hypothetical cohort of 1,000 patients with OAB and a time horizon of 1 year was carried out. Discontinuation and persistence rates for both mirabegron and AM were obtained from a Spanish observational study in 1798 patients. Unit costs (€ 2018) and utility loss associated with treatment discontinuation were obtained from Spanish public prices and literature, respectively. RESULTS: Persistence rates in patients treated with mirabegron were twice as high compared to AM, leading to a QALY gain of 0.0151 ± 0.0007 per year. Treatment with mirabegron could generate savings of 80.74 ±4.61 € per patient per year compared to AM, assuming 100% probability of saving. The hypothetical substitution of AM treatment for mirabegron could potentially generate savings of 6.6 million euros (95% CI 3.9-10.1 million euros) to the NHS and 1,238 QALYs gains (CI95%731; 1,885 QALYs) within a period of 1 year. CONCLUSIONS: The probabilistic model presented showed a greater persistence in patients treated with mirabegron compared to AM, leading to a positive impactin patients quality of life, as well cost savings to the NHS in Spain.

OBJETIVO: Evaluar si la menor tasa de abandonos del tratamiento de la vejiga hiperactiva (VH) con mirabegrón podría generar ahorros para el Sistema Nacional de Salud (SNS) y ganancia de años de vida ajustados por calidad (AVACs), en comparación con los fármacos antimuscarínicos (AM) (tolterodina, fesoterodina, oxibutinina, solifenacina).MÉTODOS: Modelo probabilístico (simulación de Monte Carlo de segundo orden) en una cohorte hipotética de 1.000 pacientes con VH y un horizonte temporal  de 1 año. Las tasas de abandono/persistencia del tratamiento con mirabegrón y AM se obtuvieron de un estudio observacional español en 1.798 pacientes. Los costes unitarios (€ 2018) y la pérdida de utilidades ligada al abandono del tratamiento se obtuvieron de precios públicos españoles y de la literatura, respectivamente. RESULTADOS: En cada paciente tratado con mirabegrón se duplica la tasa de persistencia en comparación con los AM, ganándose anualmente 0,0151 ±0,0007 AVACs, frente a AM. Con mirabegrón se generaría un ahorro anual por paciente de 80,74 ± 4,61 € en comparación con los AM, con una probabilidad de ahorro del 100%. La sustitución hipotética de los AM por mirabegrón, generaría en el plazo de 1 año un ahorro para el SNS de 6,6 millones de euros (IC 95%3,9-10,1 millones de euros) y se ganarían 1.238 AVAC (IC95% 731; 1.885 AVAC). CONCLUSIONES: El modelo probabilístico muestra una mayor persistencia en pacientes tratados con mirabegrón en comparación con los AM, generando un impacto positivo sobre la calidad de vida de los pacientes así como ahorros para el SNS.

Análisis económico del tratamiento ambulatorio intermitente con levosimendán de la insuficiencia cardiaca avanzada en España / Economic analysis of intermittent intravenous outpatient treatment with levosimendan in advanced heart failure in Spain

INTRODUCCIÓN Y OBJETIVOS: La insuficiencia cardiaca (IC) avanzada conlleva altas tasas de hospitalización y mortalidad. El estudio LION-HEART fue un ensayo clínico aleatorizado y controlado con placebo que evaluó la eficacia y la seguridad de la administración intravenosa de dosis intermitentes de levosimendán en pacientes ambulatorios con IC avanzada. El objetivo del presente estudio es realizar un análisis de costes para determinar si la menor tasa de hospitalizaciones por IC observada en pacientes tratados con levosimendán en el estudio LION-HEART puede generar ahorros para el Sistema Nacional de Salud, en comparación con la opción de no tratar a los pacientes con IC avanzada. MÉTODOS: Se realizó un modelo económico que incluyó las tasas de hospitalización por IC del estudio LION-HEART y los costes de hospitalización por IC y de adquisición y administración intravenosa de levosimendán. El horizonte temporal del análisis fue de 12 meses. Se realizaron 2 análisis, uno determinístico y otro probabilístico (simulación de Monte Carlo de segundo orden). RESULTADOS: Según el análisis determinístico, el ahorro total por cada paciente tratado con levosimendán ascendería a -698,48 euros. En el análisis probabilístico, el ahorro por paciente tratado con levosimendán sería de -849,94 (IC95%, 133,12 a -2.255,31) euros. La probabilidad de que se produzcan ahorros con levosimendán en comparación con la opción de no tratar sería del 94,8%. CONCLUSIONES: El tratamiento ambulatorio intermitente con levosimendán puede generar ahorros para el Sistema Nacional de Salud, en comparación con la opción de no tratar a los pacientes con IC avanzada

INTRODUCTION AND OBJECTIVES: Advanced heart failure (HF) leads to high hospitalization and mortality rates. The LION-HEART study was a randomized, placebo-controlled clinical trial that evaluated the safety and efficacy of intravenous administration of intermittent doses of levosimendan in outpatients with advanced HF. The aim of the present study was to perform a cost analysis to determine whether the lower rate of hospitalizations for HF, observed in patients treated with levosimendan in the LION-HEART study, can generate savings for the Spanish national health system compared with the option of not treating patients with advanced HF. METHODS: An economic model was used that included IC hospitalization rates from the LION-HEART study, the costs of hospitalization due to HF and those of the acquisition and intravenous administration of levosimendan. The time horizon of the analysis was 12 months. Two analyses were carried out, one deterministic and the other probabilistic (second-order Monte Carlo simulation). RESULTS: In the deterministic analysis, the total saving for each patient treated with levosimendan would amount to−€698.48. In the probabilistic analysis, the saving per patient treated with levosimendan would be−€849.94 (95%CI, €133.12 to−€2,255.31). The probability of savings with levosimendan compared with the no treatment option would be 94.8%. CONCLUSIONS: Intermittent ambulatory treatment with levosimendan can generate savings for the Spanish national health system compared with the option of not treating patients with advanced HF

¿Qué es una intervención sanitaria eficiente en España en 2020? / What is an efficient health intervention in Spain in 2020?

Hace más de 15 años que en Gaceta Sanitaria se publicó el artículo titulado «¿Qué es una tecnología sanitaria eficiente en España?». El creciente interés por fijar el precio de las nuevas tecnologías en función del valor que estas proporcionan a los sistemas de salud y la experiencia acumulada por los países de nuestro entorno hacen oportuno revisar qué es una intervención sanitaria eficiente en España en el año 2020. El análisis de coste-efectividad sigue siendo el método de referencia para maximizar los resultados en salud de la sociedad con los recursos disponibles. La interpretación de sus resultados requiere establecer unos valores de referencia que sirvan de guía sobre lo que constituye un valor razonable para el sistema sanitario. Los umbrales de eficiencia deben ser flexibles y dinámicos, y actualizarse periódicamente. Su aplicación debe estar basada en la gradualidad y la transparencia, considerando, además, otros factores que reflejen las preferencias sociales. Aunque la fijación de los umbrales corresponde a los decisores políticos, en España puede ser razonable utilizar unos valores de referencia como punto de partida que podrían estar comprendidos entre los 25.000 y los 60.000 euros por año de vida ajustado por calidad. No obstante, en la actualidad, más que la determinación de las cifras exactas de dicho umbral, la cuestión clave es si el Sistema Nacional de Salud está preparado y dispuesto a implantar un modelo de pago basado en el valor, que contribuya a lograr la gradualidad en las decisiones de financiación y, sobre todo, a mejorar la previsibilidad, la consistencia y la transparencia del proceso

Fifteen years ago, Gaceta Sanitaria published the article entitled "What is an efficient health technology in Spain?" The growing interest in setting the price of new technologies based on the value they provide to health systems and the experience accumulated by the countries in our environment make it opportune to review what constitutes an efficient health intervention in Spain in 2020. Cost-effectiveness analysis continues to be the reference method to maximize social health outcomes with the available resources. The interpretation of its results requires establishing reference values that serve as a guide on what constitutes a reasonable value for the health care system. Efficiency thresholds must be flexible and dynamic, and they need to be updated periodically. Its application should be based on and transparency, and consider other factors that reflect social preferences. Although setting thresholds is down to political decision-makers, in Spain it could be reasonable to use thresholds of 25,000 and 60,000 Euros per QALY. However, currently, in addition to determining exact figures for the threshold, the key question is whether the Spanish National Health System is able and willing to implement a payment model based on value, towards achieving gradual financing decisions and, above all, to improve the predictability, consistency and transparency of the process

Economic analysis of intermittent intravenous outpatient treatment with levosimendan in advanced heart failure in Spain.

INTRODUCTION AND OBJECTIVES: Advanced heart failure (HF) leads to high hospitalization and mortality rates. The LION-HEART study was a randomized, placebo-controlled clinical trial that evaluated the safety and efficacy of intravenous administration of intermittent doses of levosimendan in outpatients with advanced HF. The aim of the present study was to perform a cost analysis to determine whether the lower rate of hospitalizations for HF, observed in patients treated with levosimendan in the LION-HEART study, can generate savings for the Spanish national health system compared with the option of not treating patients with advanced HF. METHODS: An economic model was used that included IC hospitalization rates from the LION-HEART study, the costs of hospitalization due to HF and those of the acquisition and intravenous administration of levosimendan. The time horizon of the analysis was 12 months. Two analyses were carried out, one deterministic and the other probabilistic (second-order Monte Carlo simulation). RESULTS: In the deterministic analysis, the total saving for each patient treated with levosimendan would amount to-€698.48. In the probabilistic analysis, the saving per patient treated with levosimendan would be-€849.94 (95%CI, €133.12 to-€2,255.31). The probability of savings with levosimendan compared with the no treatment option would be 94.8%. CONCLUSIONS: Intermittent ambulatory treatment with levosimendan can generate savings for the Spanish national health system compared with the option of not treating patients with advanced HF.

[What is an efficient health intervention in Spain in 2020?] / ¿Qué es una intervención sanitaria eficiente en España en 2020?

Fifteen years ago, Gaceta Sanitaria published the article entitled "What is an efficient health technology in Spain?" The growing interest in setting the price of new technologies based on the value they provide to health systems and the experience accumulated by the countries in our environment make it opportune to review what constitutes an efficient health intervention in Spain in 2020. Cost-effectiveness analysis continues to be the reference method to maximize social health outcomes with the available resources. The interpretation of its results requires establishing reference values that serve as a guide on what constitutes a reasonable value for the health care system. Efficiency thresholds must be flexible and dynamic, and they need to be updated periodically. Its application should be based on and transparency, and consider other factors that reflect social preferences. Although setting thresholds is down to political decision-makers, in Spain it could be reasonable to use thresholds of 25,000 and 60,000 Euros per QALY. However, currently, in addition to determining exact figures for the threshold, the key question is whether the Spanish National Health System is able and willing to implement a payment model based on value, towards achieving gradual financing decisions and, above all, to improve the predictability, consistency and transparency of the process.

Cost-effectiveness analysis of second-line pharmacological treatment of acromegaly in Spain.

Objective: To estimate the cost-effectiveness of second-line pharmacological treatments in patients with acromegaly resistant to first-generation somatostatin analogues (FG SSA) from the Spanish National Health System (NHS) perspective.Methods: A Markov model was developed to analyze the cost-effectiveness of pegvisomant and pasireotide in FG SSA-resistant acromegaly, simulating a cohort of patients from the treatment beginning to death. Treatment with pegvisomant or pasireotide was compared to FG SSA retreatment. Efficacy data were obtained from clinical trials and utilities from the literature. Direct health costs were obtained from Spanish sources (€2018).Results: The Incremental Cost Effectiveness Ratio (ICER) of pegvisomant vs. FG SSA was €85,869/Quality-adjusted life years (QALY). The ICER of pasireotide vs. FG SSA was €551,405/QALY. The ICER was mainly driven by the incremental efficacy (4.41 QALY for pegvisomant vs. FG SSA and 0.71 QALY for pasireotide vs. FG SSA), with a slightly lower increase in costs with pegvisomant (€378,597 vs. FG SSA) than with pasireotide (€393,151 vs. FG SSA).Conclusion: The ICER of pasireotide compared to FG SSA was six times higher than the ICER of pegvisomant vs. FG SSA. Pegvisomant is a more cost-effective alternative for the treatment of acromegaly in FG SSA-resistant patients in the Spanish NHS.

Burden of diabetes mellitus in patients with acromegaly treated with second-line pharmacotherapy in Spain.

OBJECTIVE: To evaluate the burden of diabetes mellitus (DM) in adult patients with acromegaly treated with second-line pharmacotherapy, from the perspective of the Spanish National Health System (NHS). METHODS: A Markov model was developed including three states: normal glucose metabolism, DM and death. The evolution of a hypothetical cohort of acromegaly patients requiring second-line pharmacological treatment (pegvisomant or pasireotide) after first generation somatostatin analogues therapy was analyzed. Direct healthcare costs regarding acromegaly management, diabetes management and drugs costs were obtained from Spanish sources. Transition probabilities between health states were obtained from published studies. Deterministic and probabilistic sensitivity analyses were undertaken. RESULTS: Compared to pasireotide, pegvisomant increased the likelihood of glucose normalization and reduced the likelihood of DM. Consequently, in a cohort of 1,000 patients with acromegaly, treatment with pegvisomant compared to pasireotide would prevent 243, 413 and 453 cases of DM after 1, 2 and 5 years, respectively, and would reduce mortality by 0.1% after 5 years of treatment. This would result in 1 million euros savings for the NHS in 5 years. These health benefits would be obtained with savings of €1,512, €3,422 and €10,162 per patient treated with pegvisomant, after 1, 2 and 5 years, respectively. After 5 years of treatment, the probability that pegvisomant generated savings versus pasireotide would be 65.3%. CONCLUSION: The favorable effects of pegvisomant on glucose metabolism would allow a considerable number of cases of DM to be avoided compared to pasireotide, resulting in savings for the NHS in Spain.