Some reflections on the use of inappropriate comparators in CEA.

Although the choice of the comparator is one of the aspects with a highest effect on the results of cost-effectiveness analyses, it is one of the less debated issues in international methodological guidelines. The inclusion of an inappropriate comparator may introduce biases on the outcomes and the recommendations of an economic analysis. Although the rules for cost-effectiveness analyses of sets of mutually exclusive alternatives have been widely described in the literature, in practice, they are hardly ever applied. In addition, there are many cases where the efficiency of the standard of care has never been assessed; or where the standard of care has demonstrated to be cost-effective with respect to a non-efficient option. In all these cases the comparator may lie outside the efficiency frontier, so the result of the CEA may be biased. Through some hypothetical examples, the paper shows how the complementary use of an independent reference may help to identify potential inappropriate comparators and inefficient use of resources.

Cost-effectiveness analysis of ixekizumab versus secukinumab in patients with psoriatic arthritis and concomitant moderate-to-severe psoriasis in Spain.

OBJECTIVE: To conduct a cost-effectiveness analysis from the perspective of the Spanish National Health System (NHS) comparing ixekizumab versus secukinumab. DESIGN: A Markov model with a lifetime horizon and monthly cycles was developed based on the York model. Four health states were included: a biological disease-modifying antirheumatic drug (bDMARD) induction period of 12 or 16 weeks, maintenance therapy, best supportive care (BSC) and death. Treatment response was assessed based on both Psoriatic Arthritis Response Criteria (PsARC) and ≥90% improvement in the Psoriasis Area Severity Index score (PASI90). At the end of the induction period, responders transitioned to maintenance therapy. Non-responders and patients who discontinued maintenance therapy transitioned to BSC. Clinical efficacy data were derived from a network meta-analysis. Health utilities were generated by applying a regression analysis to Psoriasis Area Severity Index and Health Assessment Questionnaire‒Disability Index scores collected in the ixekizumab SPIRIT studies. Results were subject to extensive sensitivity and scenario analysis. SETTING: Spanish NHS. PARTICIPANTS: A hypothetical cohort of bDMARD-naïve patients with psoriatic arthritis and concomitant moderate-to-severe psoriasis was modelled. INTERVENTIONS: Ixekizumab and secukinumab. RESULTS: Ixekizumab performed favourably over secukinumab in the base-case analysis, although cost savings and quality-adjusted life-year (QALY) gains were modest. Total costs were €153 901 compared with €156 559 for secukinumab (difference -€2658). Total QALYs were 9.175 vs 9.082 (difference 0.093). Base-case results were most sensitive to the annual bDMARD discontinuation rate and the modification of PsARC and PASI90 response to ixekizumab or secukinumab. CONCLUSION: Ixekizumab provided more QALYs at a lower cost than secukinumab, with differences being on a relatively small scale. Sensitivity analysis showed that base-case results were generally robust to changes in most input parameters. TRIAL REGISTRATION NUMBER: SPIRIT-P1: NCT01695239; Post-results, SPIRIT-P2: NCT02349295; Post-results.

Resource use and costs in patients with poorly controlled type 2 diabetes mellitus and obesity in routine clinical practice in Spain.

OBJECTIVE: To compare healthcare resource use (HRU) and annual costs in type 2 diabetes mellitus (T2DM) patients with poor glycaemic control and obesity versus good glycaemic control without obesity. METHODS: Observational retrospective study based on the analysis of electronic medical records from the BIG-PAC database, with one year of follow-up. T2DM patients aged ≥30 years who requested medical care during 2013 were included. Annual HRU and costs per patient were compared between a reference group (HbA1c ≥ 8%, BMI ≥30 kg/m2, receiving ≥2 oral antidiabetic drugs [OADs]) and a control group (HbA1c < 7% and BMI <30 kg/m2). Direct and indirect costs (lost productivity) were analysed. Cost comparisons across groups were made using the analysis of covariance (ANCOVA) for each cost component, with age, sex, time from diagnosis, Charlson comorbidity index, OAD number and sex by group interaction as covariates. RESULTS: During the follow-up, patients in the reference group (N = 2709) had a greater HRU than those in the control group (N = 5266), especially in the number of primary care (PC) visits (11.8 vs. 9.8; 95%CI: 11.5-12.1 vs. 9.6-10.0) and days of hospitalization (1.1 vs 0.6; 95%CI: 1.0-1.2 vs. 0.5-0.7). The main components of the total cost were hospital admissions (24.5%), productivity losses (16.3%), complementary tests (14.4%), PC visits (14.2%) and medication (13.6%) in the reference group and medication (19.6%), hospital admissions (18.7%) and PC visits (18.2%) in the control group. The corrected mean annual cost per patient was higher in the reference than in the control group: €1804 vs. €1309; p < .001. CONCLUSIONS: Poor glycaemic control and obesity in T2DM patients were associated with increased HRU and costs in routine clinical practice.

Patient-physician discrepancy in the perception of immune-mediated inflammatory diseases: rheumatoid arthritis, psoriatic arthritis and psoriasis. A qualitative systematic review of the literature.

INTRODUCTION: Recommendations on chronic diseases management emphasise the need to consider patient perspectives and shared decision-making. Discrepancies between patients and physicians' perspectives on treatment objectives, disease activity, preferences and treatment have been described for immune-mediate inflammatory diseases. These differences could result on patient dissatisfaction and negatively affect outcomes. OBJECTIVE: To describe the degree of patient-physician discrepancy in three chronic immune-mediated inflammatory diseases (rheumatoid arthritis [RA], psoriatic arthritis [PsA] and psoriasis [Ps]), identifying the main areas of discrepancy and possible predictor factors. METHODS: Qualitative systematic review of the available literature on patient and physician discrepancies in the management of RA, PsA and Ps. The search was performed in international (Medline/PubMed, Cochrane Library, ISI-WOK) and Spanish electronic databases (MEDES, IBECS), including papers published from April 1, 2008 to April 1, 2018, in English or Spanish, and conducted in European or North American populations. Study quality was assessed by the Oxford Centre for Evidence-Based Medicine criteria. RESULTS: A total of 21 studies were included (13 RA; 3 PsA; 4 Ps; 1 RA, Ps, and Axial Spondyloarthritis). A significant and heterogeneous degree of discrepancy between patients and physicians was found, regarding disease activity, treatment, clinical expectations, remission concept, and patient-physician relationship. In RA and PsA, studies were mainly focused on the evaluation of disease activity, which is perceived as higher from the patient's than the physician's perspective, with the discrepancy determined by factors such as patient's perception of pain and fatigue. In Ps, studies were focused on treatment satisfaction and patient-physician relationship, showing a lower degree of discrepancy in the satisfaction regarding these aspects. CONCLUSIONS: There is a significant degree of patient-physician discrepancy regarding the management of RA, PA, and Ps, what can have a major impact on shared decision-making. Future research may help to show whether interventions considering discrepancy improve shared decision-making.

¿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

What Matters Most to Patients and Rheumatologists? A Discrete Choice Experiment in Rheumatoid Arthritis.

INTRODUCTION: To determine patient and rheumatologist preferences for rheumatoid arthritis (RA) treatment attributes in Spain and to evaluate their attitude towards shared decision-making (SDM). METHODS: Observational, descriptive, exploratory and cross-sectional study based on a discrete choice experiment (DCE). To identify the attributes and their levels, a literature review and two focus groups (patients [P] = 5; rheumatologists [R] = 4) were undertaken. Seven attributes with 2-4 levels were presented in eight scenarios. Attribute utility and relative importance (RI) were assessed using a conditional logit model. Patient preferences for SDM were assessed using an ad hoc questionnaire. RESULTS: Ninety rheumatologists [52.2% women; mean years of experience 18.1 (SD: 9.0); seeing an average of 24.4 RA patients/week (SD: 15.3)] and 137 RA patients [mean age: 47.5 years (SD: 10.7); 84.0% women; mean time since diagnosis of RA: 14.2 years (SD: 11.8) and time in treatment: 13.2 years (SD: 11.2), mean HAQ score 1.2 (SD: 0.7)] participated in the study. In terms of RI, rheumatologists and RA patients viewed: time with optimal QoL: R: 23.41%/P: 35.05%; substantial symptom improvement: R: 13.15%/P: 3.62%; time to onset of treatment action: R: 16.24%/P: 13.56%; severe adverse events: R: 10.89%/P: 11.20%; mild adverse events: R: 4.16%/P: 0.91%; mode of administration: R: 25.23%/P: 25.00%; and added cost: R: 6.93%/P: 10.66%. Nearly 73% of RA patients were involved in treatment decision-making to a greater or lesser extent; however, 27.4% did not participate at all. CONCLUSION: Both for rheumatologists and patients, the top three decision-making drivers are time with optimal quality, treatment mode of administration and time to onset of action, although in different ranking order. Patients were willing to be more involved in the treatment decision-making process.

Patient Decision Aid (PDA) for Patients with Rheumatoid Arthritis Reduces Decisional Conflict and Improves Readiness for Treatment Decision Making.

OBJECTIVES: The aim of this study was to develop and assess the effectiveness of a patient decision aid (PDA) to support treatment decision making in Spanish patients with moderate-to-severe rheumatoid arthritis (RA) who fail to achieve the therapeutic goal with the current disease-modifying antirheumatic treatment strategy. METHODS: The PDA was developed in accordance with the International Patient Decision Aids Standards recommendations. A steering group led the project. Three literature reviews and two focus groups were performed to develop the PDA prototype. To check its comprehensibility, acceptability, and feasibility, alpha-testing was performed using the Decision Support Acceptability Scale (DSAS). Beta-testing was conducted to assess preliminary evidence of PDA efficacy using the Decisional Conflict Scale (DCS) before and after PDA use. Readiness was evaluated using the Preparation for Decision Making Scale (PDMS). RESULTS: The PDA included (1) a brief description of RA, (2) treatment information, and (3) a values clarification section. Alpha-testing revealed that most patients considered that the information was presented in a good or excellent way and it could help clarify their values and facilitate treatment decision making. Most rheumatologists agreed that the PDA was easy to understand, to use, and allowed them to reach a shared decision. Beta-testing showed that PDA significantly reduced overall patients' decisional conflict [33.2 (DE: 21.4) vs 24.6 (23.5); p < 0.001] and prepared the patient for decision making [PDMS: 67.5 (21.0)]. CONCLUSIONS: We developed a PDA for Spanish patients with moderate-to-severe RA that reduces patients' decisional conflict and increases their readiness for decision making. The use of this PDA in routine clinical practice may improve the quality of the decision-making process and the quality of the choices made.

Clinical Inertia in Poorly Controlled Type 2 Diabetes Mellitus Patients with Obesity: An Observational Retrospective Study.

INTRODUCTION: To evaluate clinical inertia in patients with type 2 diabetes mellitus (T2DM), obesity and poor glycaemic control in routine clinical practice. METHODS: This was a retrospective, observational study based on the analysis of medical records from the BIG-PAC® database. Subjects who required medical care in 2013 with the following characteristics were enrolled in the study: age ≥ 30 years, diagnosis of T2DM, glycosylated haemoglobin (HbA1c) ≥ 8%, obesity (body mass index [BMI] ≥ 30 kg/m2) and treatment with ≥ 2 oral antidiabetic drugs (OADs). Inertia was evaluated by time (days) to the first intensification during the period while HbA1c levels were ≥ 8% and percentage of patients whose treatment was not intensified at 6 months, 1, 2 and 3 years and the end of follow-up. The minimum length of follow-up was 4 years. Descriptive analyses and Kaplan-Meier survival curves were performed. RESULTS: A total of 13,824 patients with T2DM receiving ≥ 2 OADs were identified; of these 2709 (19.6%) had HbA1c ≥ 8% and BMI ≥ 30 kg/m2, thus fulfilling the inclusion criteria. Of these 2709 patients, the mean age was 65.5 (standard deviation [SD] 12.0) years; 54.9% were male, mean HbA1c level was 9.2% (SD 1.3%); mean BMI was 32.1 (SD 0.9) kg/m2; and mean time from diagnosis was 8.2 (SD 3.0) years. HbA1c remained ≥ 8% for a median of 440 (95% confidence interval [CI] 421-459) days. The median time to first intensification was 456 (95% CI 429-483) days. No intensification had occurred in 77.8, 59.5, 41.5, 28.1 and 22.4% of patients at 6 months, 1, 2, 3 years and the end of follow-up, respectively. CONCLUSIONS: The patients with T2DM analysed in this study had a mean HbA1c of 9.2% at baseline, and this remained at ≥ 8% for > 1 year. The time to the first treatment intensification was longer than that recommended by guidelines. Treatment was not intensified in a large percentage of patients, with almost 60% of patients not receiving intensification at 1 year of follow-up.

[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 baricitinib versus adalimumab for the treatment of moderate-to-severe rheumatoid arthritis in Spain.

Background: Baricitinib is an oral janus kinase inhibitor for the treatment of rheumatoid arthritis (RA) and is approved in Europe for use in adults with moderately-to-severely active RA and an inadequate response or intolerance to conventional synthetic disease-modifying antirheumatic drug (csDMARD) therapy. To date, no economic evaluations have assessed the cost-effectiveness of baricitinib in the Spanish setting. Objectives: To evaluate the cost-effectiveness of baricitinib versus adalimumab for the treatment of moderately-to-severely active RA in the Spanish setting. Methods: A discrete event simulation model was developed in Microsoft Excel. Costs and outcomes were estimated over a lifetime horizon using the Spanish national payer perspective. The model compared baricitinib 4 mg once daily in combination with methotrexate with adalimumab 40 mg every other week in combination with methotrexate. Effectiveness and physical function were captured using the American College of Rheumatology criteria and the Health Assessment Questionnaire-Disability Index, input values of which were derived from a phase 3, double-blind, placebo- and active-controlled trial (RA-BEAM; funded by Eli Lilly and Incyte; ClinicalTrials.gov number, NCT01710358). Costs are presented in Euros, 2018 values. Results: In the base case analysis, baricitinib was associated with a quality-adjusted life year gain of 0.09 years over a lifetime horizon, at an incremental cost of -€558 versus adalimumab. Results of various scenario analyses and probabilistic sensitivity analysis generally were consistent with the base case analysis. Conclusion: This analysis suggests that baricitinib is a cost-effective treatment option compared to adalimumab for Spanish patients with moderately-to-severely active RA and a previous inadequate response or intolerance to csDMARD therapy.

Observational studies with type 2 diabetes mellitus treatments in Spain: A systematic literature review / Estudios observacionales con tratamientos para la dibetes mellitus tipo 2 en España: una revision sistemática de la literatura

The aim of this systematic literature review (SLR) was to provide an overview of the Spanish research landscape of observational studies conducted with antidiabetic drugs in T2DM patients, published in the last five years, with special focus on the objectives, methodology and main research areas. Twenty-two articles, corresponding to 20 studies, were included in the analysis. Around 82% of the studies employed a longitudinal study design, collected data retrospectively (72.7%), and were based on secondary data use (63.6%). Pharmacotherapeutical groups most frequently studied were insulin (31.8%) and DPP4i (13.6%). Analytic design was employed most in the studies (68.2%), followed by descriptive analysis (22.7%). In the top five of the most studied variables are those related to effectiveness assessed according to glycaemic control (91%), treatment patterns (82%), safety (hypoglycaemia) (59%), the identification of effectiveness predictive factors (45%) and effectiveness according to other control measures such as anthropometric control or cardiovascular risk factors (36%)

El objetivo de esta revisión sistemática de la literatura (SLR) fue proporcionar una visión general de los estudios observacionales realizados en España con fármacos antidiabéticos en pacientes con DM2, publicados en los últimos cinco años, con especial atención a los objetivos, metodología y principales áreas de investigación. Veintidós artículos, correspondientes a 20 estudios, se incluyeron en el análisis. Alrededor del 82% de los estudios tenían un diseño longitudinal, recogieron datos retrospectivamente (72.7%) y se basaron en datos secundarios (63.6%). Los grupos farmacoterapéuticos más frecuentemente estudiados fueron las insulinas (31.8%) y los DPP4i (13.6%). Los diseños analíticos fueron los más utilizados (68,2%), seguidos de los análisis descriptivos (22,7%). Entre las variables más estudiadas se encontraron las relacionadas con efectividad según control glucémico (91%), patrones de tratamiento (82%), seguridad (hipoglucemias) (59%), identificación de factores predictores de efectividad (45%) y efectividad según otras medidas, como control antropométrico o factores de riesgo cardiovascular (36%)

Health-related quality of life in type 1 diabetes mellitus pediatric patients and their caregivers in Spain: an observational cross-sectional study.

Objectives: This study assessed the health-related quality of life (HRQOL) of pediatric patients with type 1 diabetes mellitus (T1DM) and their caregivers.Methods: CHRYSTAL was an observational cross-sectional study conducted in Spain in 2014 on 275 patients under 18 years old diagnosed with T1DM. Patient/caregiver pairs were stratified by patients' HbA1c level (≥7.5% versus <7.5%) and by presence or absence of T1DM complications and/or comorbidities. EQ-5D and PedsQL questionnaires were administered to patients and caregivers.Results: On the EQ-5D, according to caregivers' perception, 17.7% of children experienced moderate pain or discomfort, 9.7% suffered problems performing usual activities, and 13.2% demonstrated moderate anxiety or depression. Mean EQ-5D index score was 0.95 and mean visual analog scale (VAS) score was 86.1. By HbA1c level (≥7.5% versus <7.5%), mean index scores were 0.94 and 0.95, and mean VAS scores were 82.8 and 89.2, respectively. Mean index scores were 0.91 for children with complications and/or comorbidities and 0.96 for children without. Mean VAS scores were 83.7 and 87.2, respectively. HRQOL per the PedsQL tool ranged from 68.1 (ages 2-4) to 73.1 (ages 13-18). EQ-5D index and VAS scores were significantly correlated (rho = 0.29-0.43) with several age groups of the PedsQL. EQ-5D scales showed significant moderate correlation between EQ-5D-Y and EQ-5D-3L proxy VAS score (rho = 0.45; p < .001).Conclusions: Patients with few complications and controlled HbA1c reported a relatively high HRQOL. The results suggest that parent-proxy EQ-5D ratings are valid for use as part of an overall health outcomes assessment in clinical studies of T1DM in pediatric patients.

Epidemiology, Quality of Life, and Costs Associated with Hypoglycemia in Patients with Diabetes in Spain: A Systematic Literature Review.

OBJECTIVE: To assess the burden of hypoglycemia in patients with diabetes mellitus (DM) in Spain, including epidemiological data and information relating to healthcare resource utilization (HRU) and costs, and patients' quality of life (QoL). METHODS: A systematic literature review (SLR) was conducted to identify studies that included original information on epidemiology, HRU and costs, and/or QoL associated with hypoglycemia in patients with DM in Spain, published in either Spanish or English, between January 2007 and April 2017. RESULTS: Fifteen articles, involving 14 studies, were identified in the SLR and included in the analysis. The estimated rate of severe hypoglycemia (SH) events per patient per year ranged from 0.90 to 1.50 in patients with type 1 DM (T1DM) and from 0.30 to 0.63 in patients with type 2 DM (T2DM). The data on HRU differed extensively between studies, making it difficult to draw a conclusion. Total costs per SH event ranged from €409.97 in patients with T1DM to €713.10 in patients with DM. Work absence was reported in 11.80-18% of the working patients. Further, patients who experienced hypoglycemic events expressed a higher fear and had a poorer QoL than those who did not report these events. CONCLUSION: Although the data included in the SLR were difficult to synthesize due to heterogeneity of the study designs and patient characteristics in the 14 studies, our search identified a high burden associated with hypoglycemic events in terms of HRU and costs, and patients' QoL. Further research is recommended to reach a consensus on hypoglycemia definition and study design to provide robust evidence on the burden of hypoglycemia and to accurately weigh the impact of this acute complication in Spain. FUNDING: Eli Lilly and Company.

Observational studies with type 2 diabetes mellitus treatments in Spain: A systematic literature review.

The aim of this systematic literature review (SLR) was to provide an overview of the Spanish research landscape of observational studies conducted with antidiabetic drugs in T2DM patients, published in the last five years, with special focus on the objectives, methodology and main research areas. Twenty-two articles, corresponding to 20 studies, were included in the analysis. Around 82% of the studies employed a longitudinal study design, collected data retrospectively (72.7%), and were based on secondary data use (63.6%). Pharmacotherapeutical groups most frequently studied were insulin (31.8%) and DPP4i (13.6%). Analytic design was employed most in the studies (68.2%), followed by descriptive analysis (22.7%). In the top five of the most studied variables are those related to effectiveness assessed according to glycaemic control (91%), treatment patterns (82%), safety (hypoglycaemia) (59%), the identification of effectiveness predictive factors (45%) and effectiveness according to other control measures such as anthropometric control or cardiovascular risk factors (36%).

Cost effectiveness of ixekizumab versus secukinumab in the treatment of moderate-to-severe plaque psoriasis in Spain.

BACKGROUND: Currently, several biologic agents are available for the treatment of moderate-to-severe plaque psoriasis, including newer agents with similar mechanisms of action and efficacy; therefore, there is a need to evaluate their efficiency in terms of cost effectiveness. OBJECTIVE: This study evaluates the cost effectiveness of recently approved interleukin (IL)-17A antagonists, ixekizumab and secukinumab, for the treatment of moderate-to-severe plaque psoriasis from the perspective of the Spanish National Health System (NHS). MATERIALS AND METHODS: A Markov model with a lifetime horizon was developed to compare the cost effectiveness of ixekizumab vs. secukinumab in a hypothetical cohort of patients with moderate-to-severe plaque psoriasis. The model used monthly cycles and included four health states: a 12-week induction period, treatment maintenance, best supportive care (BSC), and death. Patients meeting response criteria at the end of the induction period transitioned to maintenance therapy, whereas non-responders transitioned to BSC. It was assumed that, each year, 20% of patients receiving maintenance therapy would discontinue treatment. The model incorporated data from various sources, including published literature, a network meta-analysis, and expert opinion for some variables. RESULTS: Ixekizumab was dominant over secukinumab in that it gained 0.037 more quality-adjusted life years (QALYs) and saved €1951 in total costs over the lifetime horizon. Probabilistic sensitivity analysis showed a 96.6% likelihood that ixekizumab would be cost effective at a threshold of €30,000 per QALY gained. CONCLUSION: For the treatment of moderate-to-severe plaque psoriasis in Spain, ixekizumab provided additional QALYs and potential savings for the Spanish NHS compared with secukinumab. Since the magnitude of the differences in costs and QALYs was modest, other factors such as patient preferences (eg, for number of injections) and long-term safety (eg, related to time on the market) may also be important for guiding clinical decisions.

Should clinically meaningful outcomes in cancer be based on individual survival rather than median overall survival?

AIM: To assess whether the use of median overall survival to define clinically meaningful outcomes in the area of oncology could yield different decisions compared with those obtained with a more realistic measure such as individual survival. METHODS: Two scenarios that offered equivalent health gains/money spent were presented: 'median overall survival' scenario (new treatment provided small clinical benefits for the average population) and 'individual survival'scenario (new treatment provided substantial clinical benefits for a small percentage of the patients and no benefits for the rest). Responses from both scenarios were compared. RESULTS: Responses between the two scenarios were different for oncologists, healthcare policy makers and patients (p < 0.05). 'Individual survival' scenario obtained higher percentage of positive answers compared with 'median overall survival'. CONCLUSION: Expressing the benefits of new oncologic treatments in terms of 'individual survival' may yield to different healthcare decisions compared with the widely used median overall survival.

Costs and quality of life in community-dwelling patients with Alzheimer's disease in Spain: results from the GERAS II observational study.

BACKGROUND: Country-specific data on resource use and costs associated with Alzheimer's disease (AD) help inform governments about the increasing need for medical and financial support as the disease increases in prevalence. METHODS: GERAS II, a prospective observational study, assessed resource use, costs, and health-related quality of life (HRQoL) among patients with AD and their caregivers in Spain. Community-dwelling patients aged ≥55 years with probable AD, and their primary caregivers, were recruited by study investigators during routine clinical practice and assessed as having mild, moderate, or moderately severe/severe (MS/S) AD dementia based on patient Mini-Mental State Examination scores. Costs of AD were calculated by applying costs to resource-use data obtained in caregiver interviews using the Resource Utilization in Dementia instrument. Total societal costs included patients' health and social care costs and caregiver informal care costs. Baseline results are presented. RESULTS: Total mean monthly societal costs/patient (2013 values) were €1514 for mild (n = 116), €2082 for moderate (n = 118), and €2818 for MS/S AD dementia (n = 146) (p value <0.001 between groups). Caregiver informal care costs comprised most of the total societal costs and differed significantly between groups (€1050, €1239, €1580, respectively; p value = 0.013), whereas patient healthcare costs did not. Across AD dementia severity groups, patient HRQoL (measured by proxy) decreased significantly (p value <0.001), caregiver subjective burden significantly increased (p value <0.001) and caregiver HRQoL was similar. CONCLUSIONS: Societal costs associated with AD in Spain were largely attributable to caregiver informal care costs and increased with increasing AD dementia severity.

Social economic costs of type 1 diabetes mellitus in pediatric patients in Spain: CHRYSTAL observational study.

AIMS: To estimate the social-economic costs of Type 1 Diabetes Mellitus (T1DM) in patients aged 0-17years in Spain from a social perspective. METHODS: We conducted a cross-sectional observational study in 2014 of 275 T1DM pediatric outpatients distributed across 12 public health centers in Spain. Data on demographic and clinical characteristics, healthcare utilization and informal care were collected from medical records and questionnaires completed by clinicians and patients' caregivers. RESULTS: A valid sample of 249 individuals was analyzed. The average annual cost for a T1DM patient was €27,274. Direct healthcare costs were €4070 and direct non-healthcare cost were €23,204. Informal (familial) care represented 83% of total cost, followed by medical material (8%), outpatient and primary care visits (3.1%) and insulin (2.1%). Direct healthcare cost per patient statistically differed by glycated haemoglobin (HbA1c) level [mean cost €4704 in HbA1c ≥7.5% (≥58mmol/mol) group vs. €3616 in HbA1c<7.5% (<58mmol/mol) group)]; and by the presence or absence of complications and comorbidities (mean cost €5713 in group with complications or comorbidities vs. €3636 in group without complications or comorbidities). CONCLUSIONS: T1DM amongst pediatric patients incurs in considerable societal costs. Informal care represents the largest cost category.

The cost-effectiveness of dulaglutide versus liraglutide for the treatment of type 2 diabetes mellitus in Spain in patients with BMI ≥30 kg/m2.

OBJECTIVE: Dulaglutide 1.5 mg once weekly is a novel glucagon-like peptide 1 (GLP-1) receptor agonist, for the treatment of type two diabetes mellitus (T2DM). The objective was to estimate the cost-effectiveness of dulaglutide once weekly vs liraglutide 1.8 mg once daily for the treatment of T2DM in Spain in patients with a BMI ≥30 kg/m2. METHODS: The IMS CORE Diabetes Model (CDM) was used to estimate costs and outcomes from the perspective of Spanish National Health System, capturing relevant direct medical costs over a lifetime time horizon. Comparative safety and efficacy data were derived from direct comparison of dulaglutide 1.5 mg vs liraglutide 1.8 mg from the AWARD-6 trial in patients with a body mass index (BMI) ≥30 kg/m2. All patients were assumed to remain on treatment for 2 years before switching treatment to basal insulin at a daily dose of 40 IU. One-way sensitivity analyses (OWSA) and probabilistic sensitivity analyses (PSA) were conducted to explore the sensitivity of the model to plausible variations in key parameters and uncertainty of model inputs. RESULTS: Under base case assumptions, dulaglutide 1.5 mg was less costly and more effective vs liraglutide 1.8 mg (total lifetime costs €108,489 vs €109,653; total QALYS 10.281 vs 10.259). OWSA demonstrated that dulaglutide 1.5 mg remained dominant given plausible variations in key input parameters. Results of the PSA were consistent with base case results. LIMITATIONS: Primary limitations of the analysis are common to other cost-effectiveness analyses of chronic diseases like T2DM and include the extrapolation of short-term clinical data to the lifetime time horizon and uncertainty around optimum treatment durations. CONCLUSION: The model found that dulaglutide 1.5 mg was more effective and less costly than liraglutide 1.8 mg for the treatment of T2DM in Spain. Findings were robust to plausible variations in inputs. Based on these results, dulaglutide may result in cost savings to the Spanish National Health System.