Cost-effectiveness of omalizumab for the treatment of chronic spontaneous urticaria.

BACKGROUND: Chronic spontaneous urticaria (CSU) is a skin disease with itchy hives and/or angio-oedema that last for at least 6 weeks without an obvious external trigger. OBJECTIVES: To determine the cost-effectiveness of omalizumab relative to standard of care (SoC; up to four times the daily dose of H1 -antihistamines) in the Netherlands from a societal perspective. METHODS: The Markov model used consisted of five health states based on Urticaria Activity Score over 7 days. Model settings and characteristics of the Dutch patient population were based on an online survey among clinical experts and were validated during an expert committee meeting. Transition probabilities were derived from the GLACIAL trial. Healthcare consumption, quality of life (using EuroQol-5D) and productivity losses were derived from a burden-of-illness study (ASSURE-CSU) among 93 Dutch patients. Healthcare consumption and productivity losses were evaluated using the Dutch costing manual. The comparator treatment was SoC, consisting of (updosed) antihistamines. A 10-year time horizon was used. RESULTS: The incremental cost-effectiveness ratio (ICER) of omalizumab vs. SoC was €17 502 per quality-adjusted life-year (QALY) gained. Productivity costs played an important role in the value of the ICER; discarding productivity costs resulted in an ICER of €85 310 per QALY. CONCLUSIONS: Omalizumab is cost-effective compared with SoC. The outcomes of this study were used to establish omalizumab as third-line therapy in the Dutch treatment guidelines for CSU.

The search for relevant outcome measures for cost-utility analysis of systemic family interventions in adolescents with substance use disorder and delinquent behavior: a systematic literature review.

PURPOSE: Systemic family interventions have shown to be effective in adolescents with substance use disorder and delinquent behavior. The interventions target interactions between the adolescent and involved systems (i.e. youth, family, peers, neighbors, school, work, and society). Next to effectiveness considerations, economic aspects have gained attention. However, conventional generic quality of life measures used in health economic evaluations may not be able to capture the broad effects of systemic interventions. This study aims to identify existing outcome measures, which capture the broad effects of systemic family interventions, and allow use in a health economic framework. METHODS: We based our systematic review on clinical studies in the field. Our goal was to identify effectiveness studies of psychosocial interventions for adolescents with substance use disorder and delinquent behavior and to distill the instruments used in these studies to measure effects. Searched databases were PubMed, Education Resource Information Center (ERIC), Cochrane and Psychnet (PsycBOOKSc, PsycCRITIQUES, print). Identified instruments were ranked according to the number of systems covered (comprehensiveness). In addition, their use for health economic analyses was evaluated according to suitability characteristics such as brevity, accessibility, psychometric properties, etc. RESULTS: One thousand three hundred seventy-eight articles were found and screened for eligibility. Eighty articles were selected, 8 instruments were identified covering 5 or more systems. CONCLUSIONS: The systematic review identified instruments from the clinical field suitable to evaluate systemic family interventions in a health economic framework. None of them had preference-weights available. Hence, a next step could be to attach preference-weights to one of the identified instruments to allow health economic evaluations of systemic family interventions.

Burden of illness and health care resource utilization in adult psychiatric outpatients with attention-deficit/hyperactivity disorder in Europe.

OBJECTIVE: To assess the burden of illness and health care resource utilization of adult nonpsychotic psychiatric outpatients with attention-deficit/hyperactivity disorder (ADHD) in Europe. METHODS: This was a multicountry, cross-sectional, observational study where unselected routine patients from clinical psychiatric outpatient settings were screened and assessed for ADHD. Patients were evaluated using the Clinical Global Impressions of Severity (CGI-S) scale, the Sheehan Disability Scale (SDS), and the EuroQol-5 Dimensions questionnaire. Data on comorbidities, functional impairment, and health care resource utilization were captured. RESULTS: The study enrolled 2284 patients, of whom 1986 completed the study. The prevalence of ADHD was 17.4%, of whom 46.0% had a previous ADHD diagnosis. Patients with ADHD had a high clinical burden with psychiatric comorbidities, especially depression (43.0%) and anxiety disorders (36.4%). Substance abuse (9.2% vs. 3.4%) and alcohol abuse (10.3% vs. 5.2%) were more common in the ADHD cohort vs. the non-ADHD cohort. Only 11.5% of the patients with ADHD had no other psychiatric disorder. Various measures indicated a significantly poorer level of functioning for patients with ADHD than without ADHD, as indicated by higher scores for CGI-S (3.8 vs. 3.3) and SDS (18.9 vs. 11.6) and higher percentages of debt (35.5% vs. 24.3%) and criminality (13.8% vs. 6.1%). Lastly, the health care resource utilization was considerable and similar between adult psychiatric outpatients diagnosed and not diagnosed with ADHD. CONCLUSIONS: Although care was taken when choosing the sites for this study, to make it representative of the general outpatient adult psychiatric population, caution should be advised in generalizing the findings of our study to the general ADHD or psychiatric outpatient population. This was an observational study, thus no inference on causality can be drawn. Having ADHD imposes a considerable health and social burden on patient and health care resource utilization comparable to other chronic psychiatric disorders.

Comparison of EQ-5D and SF-6D utilities in Pompe disease.

PURPOSE: Comparative studies between Euroqol-5D (EQ-5D) and ShortForm 6D (SF-6D) utilities have been performed for a number of diseases, but not yet for orphan diseases. Pompe disease is an orphan disease with a prevalence of <5/10,000, characterized by impaired ambulatory and pulmonary functioning. We compared the psychometric properties of EQ-5D and SF-6D in patients with this disease and assessed their convergent validity, discriminative ability and sensitivity to change. METHODS: EQ-5D utilities and SF-6D utilities were computed using the UK value set. Dimensions and utilities of the two instruments were compared by correlation coefficients and descriptive statistics. We assessed whether EQ-5D and SF-6D were able to discriminate between different levels of severity and examined sensitivity to change for patients with multiple observations. RESULTS: Correlations between theoretically related dimensions of the EQ-5D and SF-6D were highly significant and were moderate to strong (range rho = 0.409-0.564). Utility values derived from the two instruments were similar (mean EQ-5D = 0.670; mean SF-6D = 0.699) and correlated strongly (rho = 0.591). Discriminative properties were somewhat better for EQ-5D; mean changes and effect sizes were better for SF-6D. CONCLUSIONS: Overall, we conclude that both instruments appear to be equally appropriate with respect to assessing utilities in Pompe disease, but neither of them performed excellently. The descriptive system of the SF-6D describes health states for Pompe disease more accurately. EQ-5D showed better discriminative properties. The SF-6D performed better with respect to sensitivity to change.

Pregnancy chances on an IVF/ICSI waiting list: a national prospective cohort study.

BACKGROUND: The effectiveness of IVF over expectant management has been proven only for bilateral tubal occlusion. We aimed to estimate the chance of pregnancy without treatment for IVF patients, using data on the waiting period before the start of IVF. METHODS: A prospective cohort study included all couples eligible for IVF or ICSI treatment, registered in a national waiting list in The Netherlands. The cumulative probability of treatment-free ongoing pregnancy on the IVF waiting list was assessed and the predictive effect of female age, duration of infertility, primary or secondary infertility and diagnostic category was estimated using Cox regression. RESULTS: We included 5962 couples on the waiting list. The cumulative probability of treatment-free ongoing pregnancy was 9% at 12 months. In multivariable Cox regression, hazard ratios were: 0.95 (P < 0.001) per year of the woman's age, 0.85 (P < 0.001) per year of duration of infertility, 0.71 (P = 0.005) for primary versus secondary infertility. Diagnostic category showed hazard ratios of 0.7, 1.6, 1.2, 1.7 and 2.6 for endometriosis, male factor, hormonal, immunological and unexplained infertility, respectively, compared with 'tubal infertility' (P < 0.001). The 12-months predicted probabilities ranged from 0% to 25%. CONCLUSIONS: The chance of an ongoing pregnancy without treatment while waiting for an IVF or ICSI is below 10% but may be as high as 25% within 1 year for selected patient groups. Timing of IVF should take predictive factors into consideration.

Predicting ongoing pregnancy chances after IVF and ICSI: a national prospective study.

BACKGROUND: The Dutch IVF guideline suggests triage of patients for IVF based on diagnostic category, duration of infertility and female age. There is no evidence for the effectiveness of these criteria. We evaluated the predictive value of patient characteristics that are used in the Dutch IVF guideline and developed a model that predicts the IVF ongoing pregnancy chance within 12 months. METHODS: In a national prospective cohort study, pregnancy chances after IVF and ICSI treatment were assessed. Couples eligible for IVF or ICSI were followed during 12 months, using the databases of 11 IVF centres and 20 transport IVF clinics. Kaplan-Meier analysis was performed to estimate the cumulative probability of an ongoing pregnancy, and Cox regression was used for assessing the effects of predictors of pregnancy. RESULTS: 4928 couples starting IVF/ICSI treatment were prospectively followed. On average, couples had 1.8 cycles in 12 months for both IVF and ICSI. The 1-year probability of ongoing pregnancy was 44.8% (95% CI 42.1-47.5%). ICSI for severe oligospermia had a significantly higher ongoing pregnancy rate than IVF indicated treatments, with a multivariate Hazard ratio (HR) of 1.22 (95% CI 1.07-1.39). The success rates were comparable for all diagnostic categories of IVF. The highest success rate is at age 30, with a slight decline towards younger women and women up to 35 and a sharp drop after 35. Primary subfertility with a HR of 0.90 (95% CI 0.83-0.99) and duration of subfertility with a HR of 0.97 (95% CI 0.95-0.99) per year significantly affected the pregnancy chance. CONCLUSIONS: The most important predictors of the pregnancy chance after IVF and ICSI are women's age and ICSI. The diagnostic category is of no consequence. Duration of subfertility and pregnancy history are of limited prognostic value.

Stepped care vs. matched care for mood and anxiety disorders: a randomized trial in routine practice.

OBJECTIVE: The effectiveness of two versions of stepped care [with either brief therapy (BT) or cognitive behavioural therapy (CBT) as a first step] is studied in comparison with the traditional matched care approach (CAU) for patients with mood and anxiety disorders. METHOD: A randomized trial was performed in routine mental health care in 12 settings, including 702 patients. Patients were interviewed once in 3 months for 18-24 months (response rate 69%). RESULTS: Overall, patients' health improved significantly over time: 51% had achieved recovery from the DSM-IV disorder(s) after 1 year and 66% at the end of the study. Respectively, 50% and 60% had 'normal' SCL90 and SF36 scores. Cognitive behavioural therapy and BT patients achieved recovery more often than CAU patients (ORs between 1.26 and 1.48), although these results were not statistically significant. CONCLUSION: Stepped care, with BT or CBT as a first step, is at least as effective as matched care.

Comparison of EQ-5D and SF-6D utilities in mental health patients.

Various preference-based measures of health are available for use as an outcome measure in cost-utility analysis. The aim of this study is to compare two such measures EQ-5D and SF-6D in mental health patients. Baseline data from a Dutch multi-centre randomised trial of 616 patients with mood and/or anxiety disorders were used. Mean and median EQ-5D and SF-6D utilities were compared, both in the total sample and between severity subgroups based on quartiles of SCL-90 scores. Utilities were expected to decline with increased severity. Both EQ-5D and SF-6D utilities differed significantly between patients of adjacent severity groups. Mean utilities increased from 0.51 at baseline to 0.68 at 1.5 years follow-up for EQ-5D and from 0.58 to 0.70 for SF-6D. For all severity subgroups, the mean change in EQ-5D utilities as well as in SF-6D utilities was statistically significant. Standardised response means were higher for SF-6D utilities. We concluded that both EQ-5D and SF-6D discriminated between severity subgroups and captured improvements in health over time. However, the use of EQ-5D resulted in larger health gains and consequent lower cost-utility ratios, especially for the subgroup with the highest severity of mental health problems.