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The study characterised differences in costs associated with raising a child between four rare disorders and examined the associations between these costs with clinical severity. Caregivers of 108 individuals with Prader-Willi, Angelman (AS), Chromosome 15q Duplication and fragile X (FXS) syndromes completed a modified Client Services Receipt Inventory and participants completed intellectual/developmental functioning and autism assessments. AS incurred the highest yearly costs per individual ($AUD96,994), while FXS had the lowest costs ($AUD33,221). Intellectual functioning negatively predicted total costs, after controlling for diagnosis. The effect of intellectual functioning on total costs for those with AS was significantly different to the other syndromes. The study highlights the significant costs associated with these syndromes, particularly AS, linked with severity of intellectual functioning.

Objectives: The objective of this review was to identify sources of variability in cost-effectiveness analyses of chimeric antigen receptor T-cell (CAR-T) therapies, tisagenlecleucel and axicabtagene ciloleucel, evaluated by health technology assessment (HTA) agencies, focusing on young compared with older patients. Methods: HTA evaluations in pediatric acute lymphoblastic leukemia (ALL) and adult diffuse large B-cell lymphoma (DLBCL) were included from Australia, Canada, England, Norway, and the United States. Key clinical evidence, economic approach, and outcomes (costs, quality-adjusted life-years [QALYs] and incremental cost-effectiveness ratios) were summarized. Results: Fourteen HTA evaluations were identified (5 ALL, 9 DLBCL [4 tisagenlecleucel, 5 axicabtagene]). Analyses were naive comparisons of prospective single-arm studies for the CAR-Ts with retrospective cohort studies for the comparators. Key clinical evidence and economic model approaches were generally consistent by CAR-T and indication, although outcomes varied. Notably, incremental QALYs varied substantially in ALL (3.67-10.6 QALYs gained), whereas variation in DLBCL was less (1.21-1.97 [tisagenlecleucel], 1.97-3.40 [axicabtagene]). Discounting of costs and outcomes varied, with the highest QALYs generated for tisagenlecleucel in ALL (10.95) associated with the lowest discount rate (1.5%) and vice versa (4.97 QALYs; 5% discount rate). The approach to extrapolation of overall survival data varied, even where the same empirical data were used. Conclusion: Modeled, long-term treatment benefit in young patients may be associated with greater uncertainty compared with adults because of potential life-long benefits with cell and gene therapies. This reflects the methodological challenges identified by HTA agencies associated with single-arm, short-term studies.

PurposeHealth related quality of life (HRQoL) is rarely routinely measured in the clinical setting. In the absence of patient reported data, clinicians rely on proxy and informal estimates to support clinical decisions. This study compares clinician estimates (proxy) with patient reported HRQoL in patients with advanced heart failure and examines factors influencing discrepancies.MethodsSeventy-five patients with heart failure, (22 females, 53 males) completed the EQ-5D-5L questionnaire. Thirty-nine clinicians (11 medical, 23 nursing, 5 allied health) completed the proxy version (V1) producing 194 dyads. Correlation was assessed using Spearman's rank tests, systematic bias was examined with Bland-Altman analyses. Inter-rater agreement at the domain level, was investigated using linear weighted Kappa statistics while factors influencing the IRG were explored using independent student t-tests, analysis of variance and regression.ResultsThere was a moderate positive correlation between clinician HRQoL estimates and patient reported utility (r = 0.38; p ConclusionClinicians in this sample overestimated HRQoL. Factors affecting the inter-rater gap, including sex and depression, support formal HRQoL screening to enhance clinical conversations and decision making. The discrepancy also supports regulatory restriction on the use of expert opinion in the development of QALYs in health economic analysis.

Health technology assessment (HTA) was introduced in Australia for the reimbursement of pharmaceuticals in 1992 and in the following years for procedures, diagnostic tests, and devices. The Australian health system is largely funded by the government. The Pharmaceutical Benefits Scheme is a national list of prescription pharmaceuticals for which the patient pays a small copayment. HTA submissions to the Pharmaceutical Benefits Scheme are assessed by the Pharmaceutical Benefits Advisory Committee. The Medical Benefits Scheme provides ambulatory medical services and HTA submissions are assessed by the Medical Services Advisory Committee. This article describes the processes of reimbursement in Australia as well as the special case of codependent technologies (eg, diagnostic test and a therapeutic drug) where a combined Medical Services Advisory Committee and Pharmaceutical Benefits Advisory Committee application is required. There are many future challenges for HTA in Australia, with growing pressure to provide early access to promising treatments and high cost personalized medicines looming on the horizon. However, Australia is well placed to deal with these issues as the early adoption of HTA and coexistence between industry, academia and the payer has proven to be a fertile environment for developing capacity to undertake and evaluate HTA.

© 2019 John Wiley & Sons Ltd Background: Behaviour and emotional problems are highly prevalent in children with autism spectrum disorder (ASD). In typically developing children, attachment quality acts as a risk/protective factor for behavioural outcomes and adjustment, warranting investigation in children with ASD. Method: We investigated the relationship between attachment and child behaviour and emotional problems in children with ASD and comorbid intellectual disability. Data were collected from parent–child dyads where children were diagnosed with ASD and ID (n = 28) or other developmental disabilities (n = 20). Results: Children with ASD had higher levels of behaviour and emotional problems and more attachment difficulties than children with other developmental disabilities. Poorer attachment quality contributed uniquely to the variance in child behaviour and emotional problems. Conclusions: Interventions targeting behaviour and emotional problems in children with ASD may benefit from an attachment model which addresses the child's difficulty in using caregivers as a coregulatory agent of emotions.

BACKGROUND:Children with intellectual disability (ID) frequently have significant educational, social and health care needs, resulting in caregivers often experiencing a wide range of negative effects. This paper aims to determine the impact of childhood ID on caregivers' health-related quality of life (HRQoL) across co-morbid diagnostic groups. The second aim of this study is to determine the risk factors associated with lower HRQoL in this population. METHODS:Caregivers of a child with ID aged between 2 and 12 years old completed an online survey to determine their HRQoL using the EQ-5D-5L measure. They were also asked demographic questions and about their dependent child's level of behavioural and emotional difficulties. RESULTS:Of the total sample of 634 caregivers, 604 caregivers completed all five questions of the EQ-5D-5L. The mean age of caregivers was 39.1 years and 91% were women. Caregivers spent on average 66.6 h per week caring for their child related to their child's disability. The mean EQ-5D-5L score of caregivers was 0.80 (95% confidence interval: 0.79, 0.82), which is below the estimated Australian population norms (mean utility score of 0.92) for the age-equivalent population. Caregivers of children with autism spectrum disorders reported the lowest HRQoL (0.77, 95% confidence interval: 0.74, 0.79) of the five included co-morbid diagnostic groups. Caregivers with a lower income, a perceived low level of social support and children with higher degree of behavioural and emotional problems were likely to have a statistically lower HRQoL. CONCLUSIONS:This is the first study to produce utility values for caregivers of children with ID. The utility values can be used to compare health states and can be used to inform comparative cost-effectiveness analyses. Demonstrating that caregivers of children with ID have reduced HRQoL and that this is associated with the degree of behavioural and emotional problems has important policy implications, highlighting the potential for policy interventions that target behavioural and emotional problems to improve outcomes for caregivers.