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This article discusses the challenges of conducting head-to-head clinical trials for evaluating the efficacy of treatments for haemophilia A, a rare lifelong condition. The standard-of-care for haemophilia A is prophylaxis with recombinant FVIII replacement therapy. Since head-to-head trials for all available FVIII products have not been conducted, indirect approaches have been proposed to compare these products. The article presents an updated analysis using a decision analytical model to compare FVIII products based on the area under the plasma level curve (AUC). The results show that extended half-life (EHL) products have a larger AUC compared to standard half-life (SHL) products. However, further investigation is needed to understand the clinical implications of these findings. [Extracted from the article]

The aim of this pilot study was to evaluate the implementation of a universal school-based cognitive behavioral program whose target is to prevent depressive symptoms in adolescents. The study had a quasi-experimental design with pretest, posttest, and a 1-year follow-up and provides an illustrative calculation for the implementation costs of the intervention. Sixty-two students (aged 14) and seven tutors participated. A majority of the students and all of the tutors were satisfied with the intervention. The students, both females and males, rated their depressed symptoms as significantly lower after the course; and for the females, this was maintained 1-year postintervention. The implementation costs for the initial 2 years were about US$300 per student. Positive effects of a universal school-based cognitive behavioral intervention aiming at preventing depressive symptoms in adolescents were found, especially among females.

Introduction: In factor VIII (FVIII) prophylaxis for haemophilia A, cost comparisons have used price per international unit (IU) based on the once reasonable assumption of equivalent outcome per IU. Now, with several extended half‐life (EHL) products available, new outcome‐oriented ways to compare products are needed. Area under the curve (AUC) quantifies FVIII levels over time after infusion providing comparable data. Aim: To develop a decision analytical model for making indirect comparisons of FVIII replacement products based on AUC. Methods: A literature search identified 11 crossover studies with relevant pharmacokinetic data. A common comparator FVIII level curve was calculated using pooled data from selected studies. Absolute curves for other products were estimated based on relative differences to the common comparator (% difference vs the anchor). Three scenarios were investigated: (1) Kogenate® versus Kovaltry® and Jivi®; (2) Advate® versus Elocta®, NovoEight®, Kovaltry, Adynovate®, Afstyla®, and ReFacto®; and (3) Jivi versus Elocta, Adynovate, and Kogenate. Sensitivity analyses investigated effects of assay type and dose. Results: In scenario 1, Jivi (+50%) and Kovaltry (+14%) showed larger AUCs versus Kogenate. In scenario 2, EHL products, Elocta and Adynovate, had the largest AUC (+64% and +58%, respectively) versus Advate. Compared with all other products in scenario 3, Jivi had the largest AUC by +13%–28%. Conclusion: This analysis concludes that EHL products differ in relative AUC, have a larger AUC compared with standard half‐life, and thus, different FVIII levels over time after infusion. This model may aid decision makers in the absence of head‐to‐head data. [ABSTRACT FROM AUTHOR]

Objective: To investigate real-world costs of continuous insulin pump therapy compared with multiple daily injection (MDI) therapy for type 1 diabetes.Research Design and Methods: Individuals with type 1 diabetes and pump therapy in the Swedish National Diabetes Register (NDR) since 2002 were eligible. Control subjects on MDI were matched 2:1 using time-varying propensity scores. Longitudinal data on health care resource use, antidiabetes treatment, sickness absence, and early retirement were taken from national registers for 2005-2013. Mean annual costs were analyzed using univariate analysis. Regression analyses explored the role of sociodemographic factors. Subgroup and sensitivity analyses were performed.Results: A total of 14,238 individuals with type 1 diabetes entered in the NDR between 2005 and 2013 (insulin pump n = 4,991, MDI n = 9,247, with switches allowed during the study) were included. Mean age at baseline was 34 years, with 21 years of diabetes duration and a mean HbA1c of 8.1% (65 mmol/mol). We had 73,920 person-years of observation with a mean follow-up of 5 years per participant. Mean annual costs were higher for pump therapy than for MDI therapy ($12,928 vs. $9,005, respectively; P < 0.001; mean difference $3,923 [95% CI $3,703-$4,143]). Health care costs, including medications and disposables, accounted for 73% of the costs for pump therapy and 63% of the costs for MDI therapy. Regression analyses showed higher costs for low education, low disposable income, women, and older age.Conclusions: Nine years of real-world data on all measurable diabetes-related resource use show robust results for additional costs of insulin pump therapy in adults by subgroup and alternative propensity score specifications. Identification of tangible and intangible benefits of pump therapy over time remain important to support resource allocation decisions. [ABSTRACT FROM AUTHOR]

Introduction Comprehensive geriatric assessment (CGA) is a multidimensional, interdisciplinary diagnostic process used to determine the medical, psychological and functional capabilities of frail older people. The primary aim of our current study is to confirm whether CGA-based outpatient care is superior than usual care in terms of health-related outcomes, resource use and costs. Methods and analysis The Geriatric Mobile Team trial is designed as a single-centre randomised, controlled, assessor-blinded (at baseline) trial. All participants will be identified via local healthcare registries with the following inclusion criteria: age =75 years, =3 different diagnoses and =3 visits to the emergency care unit (with or without admittance to hospital) during the past 18 months. Nursing home residency will be an exclusion criterion. Baseline assessments will be done before the 1:1 randomisation. Participants in the intervention group will, after an initial CGA, have access to care given by a geriatric team in addition to usual care. The control group receives usual care only. The primary outcome is the total number of inpatient days during the follow-up period. Assessments of the outcomes: mortality, quality of life, health care use, physical functional level, frailty, dependence and cognition will be performed 12 and 24 months after inclusion. Both descriptive and analytical statistics will be used, in order to compare groups and for analyses of outcomes over time including changes therein. The primary outcome will be analysed using analysis of variance, including in-transformed values if needed to achieve normal distribution of the residuals. Ethics and dissemination Ethical approval has been obtained and the results will be disseminated in national and international journals and to health care leaders and stakeholders. Protocol amendments will be published in ClinicalTrials. gov as amendments to the initial registration NCT02923843. In case of success, the study will promote the implementation of CGA in outpatient care settings and thereby contribute to an improved care of older people with multimorbidity through dissemination of the results through scientific articles, information to politicians and to the public. [ABSTRACT FROM AUTHOR]

Background: Multidisciplinary team meetings (MDTMs) have developed into standard of care to provide expert opinion and to grant evidence-based recommendations on diagnostics and treatment of cancer. Though MDTMs are associated with a range of benefits, a growing number of cases, complex case discussion and an increasing number of participants raise questions on cost versus benefit. We aimed to determine cost of MDTMs and to define determinants hereof based on observations in Swedish cancer care. Methods: Data were collected through observations of 50 MDTMs and from questionnaire data from 206 health professionals that participated in these meetings. Results: The MDTMs lasted mean 0.88 h and managed mean 12.6 cases with mean 4.2 min per case. Participants were mean 8.2 physicians and 2.9 nurses/other health professionals. Besides the number of cases discussed, meeting duration was also influenced by cancer diagnosis, hospital type and use of video facilities. When preparatory work, participation and post-MDTM work were considered, physicians spent mean 4.1 h per meeting. The cost per case discussion was mean 212 (range 91-595) EUR and the cost per MDTM was mean 2675 (range 1439-4070) EUR. Conclusions: We identify considerable variability in resource use for MDTMs in cancer care and demonstrate that 84% of the total cost is derived from physician time. The variability demonstrated underscores the need for regular and structured evaluations to ensure cost effective MDTM services. [ABSTRACT FROM AUTHOR]

Background: Randomised controlled trials have demonstrated substantial clinical benefit for thrombectomy in patients with acute ischaemic stroke and proximal anterior circulation arterial occlusion. Aim: We investigated the long-term cost-effectiveness of thrombectomy after thrombolysis versus thrombolysis alone using real-world outcome data on need for health care, home help and nursing home care. Methods:We used real-life resource use and survival data from the Swedish Stroke Register and pooled outcomes from five randomised controlled trials published in 2015 in a newly constructed Markov cost-effectiveness model with a societal perspective. Data were stratified by age (18-64; 65-74; 75-84 years) and modified Rankin scale at three months for patients with an index ischaemic stroke in 2014 fulfilling inclusion criteria NIHSS≥8 before treatment and treated with thrombolysis (n=710). Univariate sensitivity analyses explored robustness of results. A life-time perspective and 3% discount rate were applied. Results: Thrombectomy increases the health care cost per patient (þGBP 9000) mainly because of intervention costs, but the reduced burden on the social services (home help services ~GBP 13,000; nursing home care ~GBP 26,000) implies overall cost savings. The average patient gain was 1.0 quality-adjusted life year (QALY) with higher gains for younger age groups. Thrombectomy was a dominant strategy in the base case and all sensitivity analyses where social services were considered. Conclusion: Thrombectomy has a small effect on hospital costs except for the direct intervention cost. However, thrombectomy is highly likely to lead to substantial cost savings in the social service sector, up to four times the increase in health-care costs. [ABSTRACT FROM AUTHOR]

Background: To analyse 5-year cost-effectiveness of early versus optional delayed acute anterior cruciate ligament (ACL) reconstruction. Methods: 121 young, active adults with acute ACL injury to a previously uninjured knee were randomised to early ACL reconstruction (n=62, within 10 weeks of injury) or optional delayed ACL reconstruction (n=59; 30 with ACL reconstruction within 6-55 months); all patients received similar structured rehabilitation. Real life data on health care utilisation and sick leave were obtained from regional and national registers. Costs and quality-adjusted life years (QALYs) were discounted at 3%. Full-analysis set (based on study randomisation) and as-treated analysis (according to actual treatment over 5 years) principles were applied. Results: Mean cost of early ACL reconstruction was €4695 higher than optional delayed ACL reconstruction (p=0.19) and provided an additional 0.13 QALYs (p=0.11). Full-analysis set showed incremental net benefit of early versus optional delayed ACL reconstruction was not statistically significantly different from zero at any level. As-treated analysis showed that costs for rehabilitation alone were €13 650 less than early ACL reconstruction (p<0.001). Results were robust to sensitivity analyses. Conclusions: In young active adults with acute ACL injury, a strategy of early ACL reconstruction did not provide extra economic value over a strategy of optional delayed ACL reconstruction over a 5-year period. Results from this and previous reports of the KANON-trial imply that early identification of individuals who would benefit from either early ACL reconstruction or rehabilitation alone might reduce resource consumption and decrease risk of unnecessary overtreatment. Trial Registration: ISRCTN84752559. [ABSTRACT FROM AUTHOR]