L Scalone

Development of the EQ-5D-Y: a child-friendly version of the EQ-5D

PurposeTo develop a self-report version of the EQ-5D for younger respondents, named the EQ-5D-Y (Youth); to test its comprehensibility for children and adolescents and to compare results obtained using the standard adult EQ-5D and the EQ-5D-Y.MethodsAn international task force revised the content of EQ-5D and wording to ensure relevance and clarity for young respondents. Children’s and adolescents’ understanding of the EQ-5D-Y was tested in cognitive interviews after the instrument was translated into German, Italian, Spanish and Swedish. Differences between the EQ-5D and the EQ-5D-Y regarding frequencies of reported problems were investigated in Germany, Spain and South Africa.ResultsThe content of the EQ-5D dimensions proved to be appropriate for the measurement of HRQOL in young respondents. The wording of the questionnaire had to be adapted which led to small changes in the meaning of some items and answer options. The adapted EQ-5D-Y was satisfactorily understood by children and adolescents in different countries. It was better accepted and proved more feasible than the EQ-5D. The administration of the EQ-5D and of the EQ-5D-Y causes differences in frequencies of reported problems.ConclusionsThe newly developed EQ-5D-Y is a useful tool to measure HRQOL in young people in an age-appropriate manner.

Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors

Summary.  Inhibitors represent one major complication of haemophilia treatment, as they increase the risk of bleeding, physical disability and mortality. The Cost Of Care Inhibitors Study (COCIS) showed that modern strategies applied to manage patients with inhibitors adsorb high amounts of resources but provide satisfactory levels of Health‐Related Quality‐of‐Life (HR‐QoL). This paper focuses on determinants of HR‐QoL in inhibitory patients. Fifty adult patients, enrolled by 11 Italian Haemophilia Centres, were clinically assessed and filled in two HR‐QoL generic questionnaires: the EuroQol instrument (EQ‐5D) and the Short Form‐36 (SF‐36). According to our results, bleeding frequency and inhibitor titres were not found associated with HR‐QoL. Global HR‐QoL, and in particular the physical component of wellbeing in these patients was found negatively associated with their orthopaedic condition: the EQ‐5D Visual Analogue Scale (P < 0.001) scores, the SF‐36 domain ‘physical functioning’ and ‘physical component summary’ (P < 0.01) scores were found significantly correlated with the orthopaedic joint score, even after adjusting for patients’ age. These results were confirmed by those from the EQ‐5D profile. To conclude, the COCIS study is the first study showing that HR‐QoL in inhibitory patients is impaired by their orthopedic status, while other aspects do not seem to influence patients’ global wellbeing. Our results suggest that while the management of this complication is satisfactory, the attention has now to be focused on the prevention of the orthopaedic problems in these patients, which nowadays constitute one of the most important aspects to be considered in the haemophilia care.

Discrete choice modeling for the quantification of health states: The case of the EQ-5D

OBJECTIVES Probabilistic models have been developed to establish the relative merit of subjective phenomena by means of specific judgmental tasks involving discrete choices (DCs). The attractiveness of these DC models is that they are embedded in a strong theoretical measurement framework and are based on relatively simple judgmental tasks. The aim of our study was to determine whether the values derived from a DC experiment are comparable to those obtained using other valuation techniques, in particular the time trade-off (TTO). METHODS Two hundred nine students completed several tasks in which we collected DC, rank, visual analog scale, and TTO responses. DC data were also collected in a general population sample (N=444). The DC experiment was designed using a Bayesian approach, and involved 60 choices between two health states and a comparison of all health states to being dead. The DC data were analyzed using a conditional logit and a rank-ordered logit model, relying, respectively, on TTO values and the value for being dead to anchor the DC-derived values to the 0 to 1 quality-adjusted life-year (QALY) scale. RESULTS Although modeled DC data broadly replicated the pattern found in TTO responses, the DC consistently produced higher values. The two methods for anchoring DC-derived values on the QALY scale produced similar results. CONCLUSIONS On the basis of the high level of comparability between DC-derived values and TTO values, future valuation studies based on a combination of these two techniques may be considered. The results further suggest that DC can potentially be used as a substitute for TTO.

Differences between patients’, physicians’ and pharmacists’ preferences for treatment products in haemophilia: a discrete choice experiment

Summary.  The provision of health care to patients with haemophilia through replacement of the deficient coagulation factor is the result of a complex interaction between patients, physicians and policy makers, each carrying their individual sets of preferences. Preferences of patients, physicians and pharmacists towards perceived viral safety, risk of inhibitor development, infusion frequency during prophylaxis, pharmaceutical dosage form, distribution modes and price were evaluated by conjoint analysis, using a discrete choice experiment. Overall 178 patients’, 69 physicians and 58 pharmacists completed the study. Patients, physicians and pharmacists displayed preferences: (i) similar in direction and strength for risk of inhibitors and frequency of prophylaxis, (ii) similar in direction, but not in strength for perceived viral safety and price, with patients showing lower strength compared with physicians and pharmacists, and (iii) dissimilar in direction and/or strength for: (i) dosage form, which tested important only for pharmacists and (ii) distribution mode, which tested important for patients and physicians only. Our study provides evidence of the differences between different stakeholders in the preferences towards haemophilia replacement therapy, indicating that different opinions should be taken into account when planning optimal care.

Italian population-based values of EQ-5D health states

OBJECTIVE To estimate a value set for the calculation of Italian-specific quality-adjusted life years (QALYs), based on preferences elicited on EuroQol five-dimensional (EQ-5D) questionnaire health states using the time trade-off technique. METHODS The revised standard Measurement and Valuation of Health protocol was followed. Twenty-five health states, divided into three groups and given to 450 subjects, were selected to obtain 300 observations per state. Subjects aged 18 to 75 years were recruited to be representative of the Italian general adult population for age, sex, and geographical distribution. To improve efficiency, face-to-face interviews were conducted by using the Computer Assisted Personal Interviewing approach. Several random effects regression models were tested to predict the full set of EQ-5D questionnaire health states. Model selection was based on logical consistency of the estimates, sign and magnitude of the regression coefficients, goodness of fit, and parsimony. RESULTS The model that satisfied the criteria of logical consistency and was more efficient includes 10 main effect dummy variables for the EQ-5D questionnaire domain levels and the D1 interaction term, which accounts for the number of dimensions at levels 2 or 3 beyond the first. This model has an R(2) of 0.389 and a mean absolute error of 0.03, which are comparable to or better than those of models used in other countries. The utility estimates after state 11111 range from 0.92 (21111) to -0.38 (33333). Italian utility estimates are higher than those estimated in the United Kingdom and Spain and used so far to assess QALYs and conduct cost-utility evaluations in Italy. CONCLUSIONS A specific value set is now available to calculate QALYs for the conduction of health economic studies targeted at the Italian health care system.

Cost-benefit analysis of supplemented very low-protein diet versus dialysis in elderly CKD5 patients

BACKGROUND Dialysis increases patient life expectancy but is associated with clinically severe and costly complications. Health and economic benefits could derive from postponing dialysis with a supplemented very low-protein diet (sVLPD). METHODS An economic evaluation was conducted to compare benefits and costs of sVLPD versus dialysis in elderly CKD5 patients. Data from 57 patients aged >or=70 years, with glomerular filtration rate (GFR) 5-7 mL/min, previously participating in a clinical trial demonstrating non-inferior mortality and morbidity of starting sVLPD compared to dialysis treatment, were analysed: 30 patients were randomized to dialysis and 27 to sVLPD. A cost-benefit analysis was conducted, in the perspective of the National Health Service (NHS). Direct medical and non-medical benefits and costs occurring in 3.2 mean years of follow-up were quantified: time free from dialysis, cost of dialysis treatment, hospitalization, drugs, laboratory/instrumental tests, medical visits and travel and energy consumption to receive dialysis. Prices/tariffs valid in 2007 were used, with an annual discount rate of 5% applied to benefits and costs occurring after the first year. Sensitivity analyses were conducted to identify how estimates could vary in different contexts of applications. Results are reported as net benefit, expressed as mean euro/patient (patient-year). RESULTS The opportunity to safely postpone initiation of dialysis of 1 year/patient on average translated into an economic benefit to the NHS, corresponding to 21 180 euro/patient in the first, 6500 euro/patient in the second and 682 euro/patient in the third year of treatment, with a significant net benefit in favour of sVLPD even in a worst-case hypothesis. CONCLUSION The initiation of sVLPD in elderly CKD5 subjects is a safe and beneficial strategy for these patients and allows them to gain economic resources that can be allocated to further health care investments.

Cost and quality of life in patients with severe chronic hand eczema refractory to standard therapy with topical potent corticosteroids

Little is known about the socio‐economic burden of severe chronic hand eczema in patients refractory to treatment with potent corticosteroids.

Cost-Effectiveness and Cost-Utility Analysis of Spinal Cord Stimulation in Patients With Failed Back Surgery Syndrome: Results From the PRECISE Study

To assess the cost‐effectiveness and cost‐utility of Spinal Cord Stimulation (SCS) in patients with failed back surgery syndrome (FBSS) refractory to conventional medical management (CMM).

Immune tolerance induction in patients with haemophilia a and inhibitors: effectiveness and cost analysis in an European Cohort (The ITER Study)

Although immune tolerance induction (ITI) is considered the first choice treatment to eradicate inhibitors in haemophilia A patients, little is known about outcomes determinants and cost magnitude.

The societal burden of chronic liver diseases: results from the COME study

Objective Chronic liver diseases (CLDs) impose a significant socioeconomic burden on patients and the healthcare system, but to what extent remains underexplored. We estimated costs and health-related-quality-of-life (HRQoL) among patients with CLDs at different stages and with different aetiologies. Design A cost-of-illness study was conducted. Direct costs, productivity loss and HRQoL were estimated in patients with chronic hepatitis, cirrhosis hepatocellular carcinoma (HCC) or where orthotopic liver transplantation (OLT) had been performed, for hepatitis C virus (HCV) infection, hepatitis B virus (HBV) infection, or in those with liver disease from other causes. Patients were retrospectively observed for 6 months. The societal perspective was adopted to calculate costs. Results In total, 1088 valid patients (median age=59.5 years, 60% men) were enrolled. 61% had chronic hepatitis, 20% cirrhosis, 8% HCC and 12% underwent OLT. HCV infection was identified in 52% and HBV infection in 29% of the patients. Adjusted mean direct costs increased from <€200/patient-month in HCV-infected patients with hepatitis to >€3000/patient-month in HBV infected patients with OLT. Antiviral treatment was the cost driver in patients with hepatitis, while hospital costs were the driver in the other subgroups. Absenteeism increased from HBV-infected patients with hepatitis (0.7 day/patient-month) to patients with OLT with other aetiologies (3.7 days/patient-month). HRQoL was on average more compromised in cirrhosis and patients with HCC, than in hepatitis and patients with OLT. HBV-infected patients generated higher direct costs, patients with other aetiologies generated the highest productivity loss and HCV-infected patients reported the worst HRQoL levels. Conclusions The present study can be considered a benchmark for future research and to guide policies aimed at maximising the cost-effective of the interventions.