Adult-onset Still's disease (AOSD) poses a not well estimated burden on patients and healthcare systems. To assess this burden, a systematic review (SR) was undertaken to identify health-related quality of life (HRQoL), utilities, costs and healthcare resource use data. Searches of twelve databases, four conferences, and three key technology assessment and regulatory agency websites were conducted in August 2022. Reference lists of retrieved SRs published since 2017 were also checked. Overall, 16 studies were eligible for inclusion. Eight studies reported HRQoL outcomes, one of which also reported utilities data. Two studies reported direct costs outcomes, and seven reported healthcare resource use data. No indirect costs were identified. A range of outcomes were reported, thus limiting the comparability of results across studies. SF-36 data were impaired in AOSD on most scales, especially those concerning physical activity. Mean SF-36 data were lower across all subscales in patients with active AOSD compared with inactive AOSD. Biologic therapy showed improvements in the SF-36 physical health summary. Utility scores (one study) were significantly lower for AOSD than for healthy controls. Limited direct economic costs data were identified but were substantial where reported. Hospital length of stay ranged from 6.1 to 23.5 days. The SR showed there is a paucity of research reporting the HRQoL and cost burden of AOSD.

Background Mitochondrial disease is a degenerative, progressive, heterogeneous group of genetic disorders affecting children and adults. Mitochondrial disease is associated with morbidity and mortality, with predominantly neurological and neuromuscular symptoms including dystonia, weakness, encephalopathy, developmental delay and seizures. Seizures are one of the most common and severe manifestations of mitochondrial disease. These seizures are typically refractory to common anti-seizure therapies. There are no approved disease-modifying treatments for mitochondrial disease. Our objective was to conduct two systematic literature reviews to identify health-related quality of life (HRQoL), utilities, costs and healthcare resource use data in mitochondrial disease with associated seizures. Methods A range of databases and information sources were searched up to July 2022 to identify eligible studies. Search strategies included a range of variant terms for mitochondrial disease and HRQoL, utilities, cost and healthcare resource use outcomes. Two reviewers independently assessed articles against the eligibility criteria; studies were extracted by one reviewer and checked by a second. Risk of bias was assessed for studies reporting HRQoL data. Results were narratively assessed. Results Seven studies were eligible for the HRQoL and utilities review. The studies used different tools to report data, and despite the variability in methods, HRQoL scores across the studies showed moderate/severe disease in patients with mitochondrial disease with associated seizures. Parents of patients with mitochondrial disease with associated seizures were characterised by high total parenting stress. No studies reported utilities data. Two case reports and one retrospective review of medical records of children who died in hospital were eligible for the costs and resource use review. These provided limited information on the duration of hospital stay, in an intensive care unit (ICU), on mechanical ventilation. No studies reported costs data. Conclusion These reviews highlight the limited HRQoL, utilities, costs and resource use data and the variability of instruments used in mitochondrial disease with associated seizures. However, the data available indicate that mitochondrial disease with associated seizures affects patients’ and caregivers’ HRQoL alike. No robust conclusion can be drawn on the impact of mitochondrial disease with associated seizures on hospital or ICU length of stay. Trial registration PROSPERO: CRD42022345005.

There has been no extensive synthesis of studies evaluating the cost of chronic hand eczema (CHE). This review evaluated the societal costs, healthcare resource utilisation, missed work time and job loss due to CHE. MEDLINE and 16 other databases and websites were searched in October 2020 for studies meeting prespecified inclusion criteria. Studies conducted in Europe, Australia, New Zealand or the Americas were included. Two reviewers independently assessed titles and abstracts, and full-text papers published in English between 2000 and 2020, for relevance. Data extraction was carried out by one reviewer and checked by a second reviewer. All data were based on costs between 2001 and 2013 but have been inflated to 2020 prices and converted to US dollars and Euros. A total of 30 studies (reported in 33 publications) were included in the synthesis. Mean total societal costs per year per patient ranged from $2549 (€1813) to $10,883 (€7738). Pharmacological therapy was, on average, $28.34 (€20.15) per month in Italy and $36.49 (€25.94) per month for emollients in Switzerland. Yearly treatment costs were $599.05 (€425.92) for drugs, including topical corticosteroids, topical calcineurin inhibitors, other topical treatments and oral treatments, and $178.40 for emollients, in Germany. CHE was associated with hospitalisation costs ranging from $81.86 (€58.20) per patient per month (US) to $105.04 (€74.68) per patient per month (Italy) and $639.59 (€454.75) per year (Germany). Up to 57% of patients took sick leave and up to 25% reported job loss/job change due to CHE. This review confirms the significant cost burden of CHE. Given the paucity of studies estimating the monetary costs of absenteeism, presenteeism and job loss associated with CHE, current mean societal costs are likely underestimated. Uncontrolled disease may also lead to increased costs to patients and society.

OBJECTIVE:Congenital cytomegalovirus (cCMV) is the most common infectious cause of congenital disability. It can disrupt neurodevelopment, causing lifelong impairments including sensorineural hearing loss and developmental delay. This study aimed, for the first time, to estimate the annual economic burden of managing cCMV and its sequelae in the UK.DESIGN:The study collated available secondary data to develop a static cost model.SETTING:The model aimed to estimate costs of cCMV in the UK for the year 2016.PATIENTS:Individuals of all ages with cCMV.MAIN OUTCOME MEASURES:Direct (incurred by the public sector) and indirect (incurred personally or by society) costs associated with management of cCMV and its sequelae.RESULTS:The model estimated that the total cost of cCMV to the UK in 2016 was £732 million (lower and upper estimates were between £495 and £942 million). Approximately 40% of the costs were directly incurred by the public sector, with the remaining 60% being indirect costs, including lost productivity. Long-term impairments caused by the virus had a higher financial burden than the acute management of cCMV.CONCLUSIONS:The cost of cCMV is substantial, predominantly stemming from long-term impairments. Costs should be compared against investment in educational strategies and vaccine development programmes that aim to prevent virus transmission, as well as the value of introducing universal screening for cCMV to both increase detection of children who would benefit from treatment, and to build a more robust evidence base for future research.

PURPOSE OF REVIEW:To provide a brief review of literature published since January 2012 on the subject of the cost-effectiveness of follow-up services for childhood cancer survivors. A pragmatic literature review was carried out to identify relevant literature. RECENT FINDINGS:There is very little literature or evidence around the cost-effectiveness of follow-up services for the survivors of childhood cancer. The literature that is partially relevant focuses on the need for follow-up services to change to allow a more risk-based, personalized approach for survivors. There are implications in these changes for the costs and effectiveness of services. Some current literature also focuses on the economic impact of childhood cancer and treatment on the individual. SUMMARY:There is considerable interest in understanding whether innovative approaches to follow-up care are cost-effective. There is little evidence currently but new models of care imply a potential shift in the resources required from the hospital setting to care provided outside hospitals. The rapid growth in numbers of survivors means that traditional hospital-based follow-up services will continue to experience high demand so it is important that new models of care are cost-effective.