Guillermo Villa

Generic utilities in Chronic Obstructive Pulmonary disease patients stratified according to different staging systems

BackgroundTo determine generic utilities for Spanish chronic obstructive pulmonary disease (COPD) patients stratified by different classifications: GOLD 2007, GOLD 2013, GesEPOC 2012 and BODEx index.MethodsMulticentre, observational, cross-sectional study. Patients were aged ≥40 years, with spirometrically confirmed COPD. Utility values were derived from EQ-5D-3 L. Means, standard deviations (SD), medians and interquartile ranges (IQR) were computed based on the different classifications. Differences in median utilities between groups were assessed by non-parametric tests.Results346 patients were included, of which 85.5% were male with a mean age of 67.9 (SD = 9.7) years and a mean duration of COPD of 7.6 (SD = 5.8) years; 80.3% were ex-smokers and the mean smoking history was 54.2 (SD = 33.2) pack-years. Median utilities (IQR) by GOLD 2007 were 0.87 (0.22) for moderate; 0.80 (0.26) for severe and 0.67 (0.42) for very-severe patients (p < 0.001 for all comparisons). Median utilities by GOLD 2013 were group A: 1.0 (0.09); group B: 0.87 (0.13); group C: 1.0 (0.16); group D: 0.74 (0.29); comparisons were statistically significant (p < 0.001) except A vs C. Median utilities by GesEPOC phenotypes were 0.84 (0.33) for non exacerbator; 0.80 (0.26) for COPD-asthma overlap; 0.71 (0.62) for exacerbator with emphysema; 0.72 (0.57) for exacerbator with chronic bronchitis (p < 0.001). Comparisons between patients with or without exacerbations and between patients with COPD-asthma overlap and exacerbator with chronic bronchitis were statistically-significant (p < 0.001). Median utilities by BODEx index were: group 0–2: 0.89 (0.20); group 3–4: 0.80 (0.27); group 5–6: 0.67 (0.29); group 7–9: 0.41 (0.31). All comparisons were significant (p < 0.001) except between groups 3–4 and 5–6.ConclusionIrrespective of the classification used utilities were associated to disease severity. Some clinical phenotypes were associated with worse utilities, probably related to a higher frequency of exacerbations. GOLD 2007 guidelines and BODEx index better discriminated patients with a worse health status than GOLD 2013 guidelines, while GOLD 2013 guidelines were better able to identify a smaller group of patients with the best health.

Cost-Effectiveness Analysis of the Spanish Renal Replacement Therapy Program

♦ Background: We undertook a cost-effectiveness analysis of the Spanish Renal Replacement Therapy (RRT) program for end-stage renal disease patients from a societal perspective. The current Spanish situation was compared with several hypothetical scenarios. ♦ Methods: A Markov chain model was used as a foundation for simulations of the Spanish RRT program in three temporal horizons (5, 10, and 15 years). The current situation (scenario 1) was compared with three different scenarios: increased proportion of overall scheduled (planned) incident patients (scenario 2); constant proportion of overall scheduled incident patients, but increased proportion of scheduled incident patients on peritoneal dialysis (PD), resulting in a lower proportion of scheduled incident patients on hemodialysis (HD) (scenario 3); and increased overall proportion of scheduled incident patients together with increased scheduled incidence of patients on PD (scenario 4). ♦ Results: The incremental cost-effectiveness ratios (ICERs) of scenarios 2, 3, and 4, when compared with scenario 1, were estimated to be, respectively, –€83 150, –€354 977, and –€235 886 per incremental quality-adjusted life year (ΔQALY), evidencing both moderate cost savings and slight effectiveness gains. The net health benefits that would accrue to society were estimated to be, respectively, 0.0045, 0.0211, and 0.0219 ΔQALYs considering a willingness-to-pay threshold of €35 000/ΔQALY. ♦ Conclusions: Scenario 1, the current Spanish situation, was dominated by all the proposed scenarios. Interestingly, scenarios 3 and 4 showed the best results in terms of cost-effectiveness. From a cost-effectiveness perspective, an increase in the overall scheduled incidence of RRT, and particularly that of PD, should be promoted.

Validation of a computer-adaptive test to evaluate generic health-related quality of life.

BackgroundHealth Related Quality of Life (HRQoL) is a relevant variable in the evaluation of health outcomes. Questionnaires based on Classical Test Theory typically require a large number of items to evaluate HRQoL. Computer Adaptive Testing (CAT) can be used to reduce tests length while maintaining and, in some cases, improving accuracy. This study aimed at validating a CAT based on Item Response Theory (IRT) for evaluation of generic HRQoL: the CAT-Health instrument.MethodsCross-sectional study of subjects aged over 18 attending Primary Care Centres for any reason. CAT-Health was administered along with the SF-12 Health Survey. Age, gender and a checklist of chronic conditions were also collected. CAT-Health was evaluated considering: 1) feasibility: completion time and test length; 2) content range coverage, Item Exposure Rate (IER) and test precision; and 3) construct validity: differences in the CAT-Health scores according to clinical variables and correlations between both questionnaires.Results396 subjects answered CAT-Health and SF-12, 67.2% females, mean age (SD) 48.6 (17.7) years. 36.9% did not report any chronic condition. Median completion time for CAT-Health was 81 seconds (IQ range = 59-118) and it increased with age (p < 0.001). The median number of items administered was 8 (IQ range = 6-10). Neither ceiling nor floor effects were found for the score. None of the items in the pool had an IER of 100% and it was over 5% for 27.1% of the items. Test Information Function (TIF) peaked between levels -1 and 0 of HRQoL. Statistically significant differences were observed in the CAT-Health scores according to the number and type of conditions.ConclusionsAlthough domain-specific CATs exist for various areas of HRQoL, CAT-Health is one of the first IRT-based CATs designed to evaluate generic HRQoL and it has proven feasible, valid and efficient, when administered to a broad sample of individuals attending primary care settings.

Budget impact analysis of first-line treatment with pazopanib for advanced renal cell carcinoma in Spain

BackgroundDue to economic constraints, cancer therapies are under close scrutiny by clinicians, pharmacists and payers alike. There is no published pharmacoeconomic evidence guiding the choice of first-line therapy for advanced renal cell carcinoma (RCC) in the Spanish setting. We aimed to develop a model describing the natural history of RCC that can be used in healthcare decision-making. We particularly analyzed the budget impact associated with the introduction of pazopanib compared to sunitinib under the Spanish National Healthcare System (NHS) perspective.MethodsWe developed a Markov model to estimate the future number of cases of advanced RCC (patients with favorable or intermediate risk) resulting either from initial diagnosis or disease progression after surgery. The model parameters were obtained from the literature. We assumed that patients would receive either pazopanib or sunitinib as first-line therapy until disease progression. Pharmacological costs and costs associated with the management of adverse events (AE) were considered. A univariate sensitivity analysis was undertaken in order to test the robustness of the results.ResultsThe model predicted an adult RCC prevalence of 7.5/100,000 (1-year), 20.7/100,000 (3-year) and 32.5/100,000 (5-year). These figures are very close to GLOBOCAN reported RCC prevalence estimates of 7.6/100,000, 20.2/100,000 and 31.1/100,000, respectively. The model predicts 1,591 advanced RCC patients with favorable or intermediate risk in Spain in 2013. Annual per patient pharmacological costs were €32,365 and €39,232 with pazopanib and sunitinib, respectively. Annual costs associated with the management of AE were €662 and €974, respectively. Overall annual per patient costs were €7,179 (18%) lower with pazopanib compared to sunitinib. For every point increase in the percentage of patients treated with pazopanib, the NHS would save €67,236. If all the 1,591 patients predicted were treated with pazopanib, the NHS would save €6,723,622 in 2013. Results were robust according to the sensitivity analysis.ConclusionsWe developed a model that accurately reproduces the natural history of RCC and can be thus used in healthcare decision-making. When applied to the Spanish case, the introduction of pazopanib results in savings for the NHS, as a consequence of both reduced pharmacological costs and lower costs associated with the management of AE compared to sunitinib.

Desarrollo y validación de un cuestionario genérico para la evaluación de la satisfacción de los pacientes crónicos: Cuestionario SAT-Q

OBJECTIVES To develop and validate a brief general questionnaire to assess satisfaction in patients with chronic disease. DESIGN epidemiological, observational, multicentre cross-sectional study. LOCATION 4 Primary Health Care Centers from the IV Health Area in the Principality of Asturias. SUBJECTS A total of 202 patients diagnosed with chronic illness who were following a pharmacological regimen for at least one year. METHODS An extensive literature review was conducted to create an initial item pool of 61 questions regarding patient satisfaction. Then, a forward-backward translation into Spanish was carried out. Next, both the expert (n=8) and patient (n=30) panels led to a new version of 34 items (concerning satisfaction with medication and satisfaction with health service). Finally, scale item reduction (item-total correlation and exploratory factorial analysis -EFA-) and psychometrical validation (feasibility, reliability and criterion validity) of the SAT-Q- were evaluated. Both the SAT-Q and the SF-12 (to assess patient health related quality of life) were applied. RESULTS Item reduction analysis resulted in 18 items: general satisfaction with medication (3), adverse-events (3), oversights (2), effectiveness (3), convenience (4) and Health services (3). Internal consistency (Cronbach α) and Intraclass Correlation Coefficients were moderate-high. Moreover, significant positive correlations between SAT-Q scores and SF-12 Physical and Mental Summary Components were found (with the exception of oversights). CONCLUSIONS A brief questionnaire for measuring satisfaction in chronic patients has been developed and preliminary validated.

Eficiencia del inicio con ambrisentan frente a bosentan en el tratamiento de la hipertensión arterial pulmonar

OBJECTIVE To evaluate the efficiency of initiation with endothelin receptor antagonists, ambrisentan or bosentan, followed by sequential combination with phosphodiesterase-5 inhibitors and prostanoids in the treatment of pulmonary arterial hypertension, from the Spanish National Health System perspective. METHODS A Markov model was developed based on the four New York Heart Association functional classes. A panel of three experts reached a consensus on patient management based on clinical practice. Patients revised their treatment every 12 weeks, based on their health status and previous medication records. Pharmacological treatment costs and costs associated with very frequent adverse events (AE) were considered in a horizon of 60 weeks. Outcomes were measured in qualityadjusted life years (QALY). A probabilistic sensitivity analysis was performed. RESULTS No clinically relevant differences in QALY per-patient and year were found for initiation with ambrisentan and bosentan: 0.6853 and 0.6902, respectively. Initiation with ambrisentan resulted in lower pharmacological treatment and AE management costs: ?35,550 and ?117 versus ?40,224 and ?171. In the sensitivity analysis, initiation with ambrisentan resulted in a negative significant cost difference: ?-4,982; CI95%[?- 8,014; ?-2,500]; while no significant differences in QALY were found: -0.0044; CI95%[-0.0189; 0.0101]. CONCLUSIONS Initiation with ambrisentan followed by sequential combination with phosphodiesterase-5 inhibitors and prostanoids yields comparable outcomes at lower costs than initiation with bosentan.Objective: To evaluate the efficiency of initiation with endothelin receptor antagonists, ambrisentan or bosentan, followed by sequential combination with phosphodiesterase-5 inhibitors and prostanoids in the treatment of pulmonary arterial hypertension, from the Spanish National Health System perspective. Methods: A Markov model was developed based on the four New York Heart Association functional classes. A panel of three experts reached a consensus on patient management based on clinical practice. Patients revised their treatment every 12 weeks, based on their health status and previous medication records. Pharmacological treatment costs and costs associated with very frequent adverse events (AE) were considered in a horizon of 60 weeks. Outcomes were measured in qualityadjusted life years (QALY). A probabilistic sensitivity analysis was performed. Results: No clinically relevant differences in QALY per-patient and year were found for initiation with ambrisentan and bosentan: 0.6853 and 0.6902, respectively. Initiation with ambrisentan resulted in lower pharmacological treatment and AE management costs: 35,550 and 117 versus 40,224 and 171. In the sensitivity analysis, initiation with ambrisentan resulted in a negative significant cost difference: -4,982; CI95%[- 8,014; -2,500]; while no significant differences in QALY were found: -0.0044; CI95%[-0.0189; 0.0101]. Conclusions: Initiation with ambrisentan followed by sequential combination with phosphodiesterase-5 inhibitors and prostanoids yields comparable outcomes at lower costs than initiation with bosentan.

Evaluación económica del tratamiento de la trombocitopenia inmune primaria crónica refractaria con agonistas del receptor de la trombopoyetina

Purpose: To develop a tool to assist the decision-making for selection of Thrombopoyetin Receptor Agonists of adult patients with chronic immune primary thrombocytopenia (PTI). Methods: Stochastic cost-effectiveness analysis with a 6-Health- States Markov model: stable, bleeding type 2, 3 or 4, post-type 4 bleeding and death. Each simulation analyzes a randomly generated scenario that describes patients characteristics, results measured in quality adjusted life years (QALYs) and costs (in 2011). Distributions were obtained from the Spanish data of the European health survey of 2009, the INE estimate of population for 2011 and the 6-months clinical studies for Eltrombopag and Romiplostim. Utility values were obtained from the literature and the costs from Spanish official rates lists. A set of 10.000 random scenarios were generated and the patients evolution of each scenario was simulated during a time horizon of five years (in 2-weeks cycles). National Health System Perspective was used and the annual discount rate was set at 3%. Results: Eltrombopag showed more effectiveness in 9.983 scenarios and there was no difference in 17. In 7.048 scenarios the alternative Eltombopag was dominant. It was cost-effective in another 19 (threshold 30,000 /AVAC). Conclusions: Eltrombopag was the most cost-effective alternative in 70,67% of the simulated scenarios and its use could produce lower costs to the NHS.

PUK18 COST-EFFECTIVENESS ANALYSIS OF TIMELY VERSUS LATE DIALYSIS REFERRAL AFTER RENAL TRANSPLANT FAILURE IN SPAIN

vs. 4.6 ± 8.9; P = 0.0029) and interventions (5.8 ± 11.1 vs. 0.7 ± 1.1; P = 0.0042). Only 11.6% of PD patients used an ambulance or transport organized by the dialysis center/sickness fund, compared with 67.8% for HD. The estimated annual cost for the public payer (PP) was c72,350 per HD and c55,343 per PD patient (i.e., 31% more). As in 2006 there were approximately 6400 patients on dialysis (90% on HD, 10% on PD), the PP total cost is estimated to be around c452 million (2.45% of 2006 health care budget). The dialysis procedure was the main cost driver (66% of costs) being 27% more expensive for HD. Hospital and ambulatory services were respectively 28% and 45% more expensive for HD. CONCLUSIONS: The economic burden of dialysis is important in Belgium. Considering that survival of PD patients is at least as good as that of HD patients and that home-treatment reduces exposure to hospital pathogens, PD represents good value for money and should be considered in more patients.