Health care resource use in preschool children with cerebral palsy.

AIM: To estimate the burden of disease and evaluate which factors affect health care resource use (HCRU) in young children with cerebral palsy (CP). METHOD: Data were collected as part of a prospective, longitudinal cohort study of children with CP born in Queensland, Australia between 2006 and 2009. HCRU questionnaires were administered at six time points. Data on resource use, socio-demographics, and disease severity were collected. Costs were sourced from Medicare, the Australian National Hospital Cost Data Collection, and market prices. A generalized linear model was used to identify factors influencing CP-related costs. RESULTS: A total of 794 questionnaires were completed by 222 participants (mean = 3.6 per participant). Physiotherapy (94%, n = 208) was the most widely accessed allied health care therapy; almost half of the participants (45%; 354 of 794) reported one or more hospital admissions. From the health care funder perspective, a child with CP costs on average A$24 950 per annum (A$12 475 per 6 months). Higher costs were associated with increased motor impairment (Gross Motor Function Classification System, p < 0.001) and increased comorbidities (p = 0.012). INTERPRETATION: HCRU in preschool children with CP can be analysed according to disease severity. Both increased motor impairments and increased comorbidities were associated with higher health care costs.

An overview of reproductive carrier screening panels for autosomal recessive and/or X-linked conditions: How much do we know?

BACKGROUND & AIM: Reproductive carrier screening seeks to identify couples at a high risk of having offspring affected by autosomal recessive and X-linked (XL) conditions. The aim of this paper is to provide a comprehensive overview of existing carrier screening panels by examining their gene content and characteristics, identifying the most common genes/conditions included in these panels, and analyzing their listed prices. METHODS: A comprehensive evaluation of existing carrier screening panels was conducted by searching for web-based content, reviewing information brochures, and establishing direct contact with the providers via email or phone. RESULTS: Twenty-two panels and their providers were identified with a cumulative total of 2205 unique genes. The number of genes included in these panels varied from 44 to 2054. Only 15 genes (0.7%) were included in all the panels. The carrier frequency of these 15 common genes and their associated conditions varied greatly, but the conditions associated with the genes are "severe". The price of these 22 panels ranged from $349 to $4320 per couple (USD in 2023). The correlation between the listed price and the number of selected genes among these panels was small and not statistically significant (r = 0.1023, p = 0.6959). CONCLUSION: Considerable discrepancies exist among carrier screening panels. Ongoing research and monitoring are necessary to capture the dynamic nature of the carrier screening landscape, providing up-to-date information for clinical practice and informed decision-making.

A cross-sectional study of symptoms and health-related quality of life in menopausal-aged women in China.

OBJECTIVE: To measure symptoms and health-related quality of life in a cross-sectional cohort of menopausal-aged women in China. METHOD: A cross-sectional survey was conducted in a general population cohort of 2,000 Chinese females over the age of 45 years. Patients completed the Chinese version of the EuroQol-5D five level (EQ5D5L) health-related quality of life instrument via Personal Digital Assistant. Raw scores were converted to utility tariffs using value sets for China. Statistical analysis included Pearson's chi-square test, z test for multiple comparisons with adjustment by the Bonferroni method, independent-sample t-test, ANOVA, and adjustment by the Tukey method for multiple comparison. Results were considered statistically significant when p < 0.05 and the study was reported according to the STROBE recommendations. RESULTS: In a cohort of 2000 women, 732 (37%) were premenopausal, 798 (40%) were perimenopausal and 470 (23%) were postmenopausal. Perimenopausal women reported significantly more symptoms (91%) compared to premenopausal (77%) and postmenopausal (81%) women. Health-related quality of life was significantly lower in symptomatic perimenopausal women compared to premenopausal (0.919, p < 0.05) and postmenopausal (0.877, p < 0.05) women. Within each group there was a statistically significant difference between the health-related quality of life of women with symptoms compared to without symptoms. CONCLUSION: The perimenopausal phase of menopause is associated with significantly more symptoms and significantly lower HRQoL compared to premenopausal and postmenopausal phases.

Why is this study needed?• It is important to understand the impact of menopausal symptoms on women's quality of life so that decisions can be made about what healthcare services to provide and what treatments to fund.• Currently there are only a few older studies investigating menopausal symptoms in the perimenopausal phase, which is characterised by hormone fluctuations and symptoms likely to impact quality of life, and lasts up to four years. What is the key problem this manuscript addresses?• This research measures symptoms and the impact on quality of life of menopausal women in China. What are your main results and what do they mean?• Women in the perimenopausal phase are disproportionately affected by symptoms compared to premenopausal and postmenopausal women.• Perimenopausal women have significantly lower HRQoL compared to premenopausal and postmenopausal women.• This information is critical to informing pharmacoeconomic decisions about healthcare provision and publicly funded treatments in China.

Meta-analysis of health state utility values measured by EuroQol 5-dimensions (EQ5D) questionnaire in Chinese women with breast cancer.

BACKGROUND: To synthesise EQ5D health state utility values in Chinese women with breast cancer for parameterising a cost utility model. METHODS: Eligible studies had to report health state utility values measured by EQ-5D in Chinese women diagnosed with breast cancer. Risk of bias was assessed using the Newcastle Ottawa Scale (NOS). Data from single arm studies was pooled using meta-analysis of single proportions to provide overall point estimates and 95% confidence intervals for fixed and random effects models using the inverse variance and Der Simonian-Laird methods respectively. Heterogeneity was evaluated using the I2 statistic and sensitivity analysis and meta-regression were conducted. RESULTS: Five papers were included, when all studies were combined (n = 4,100) the mean utility (95% confidence interval) for random effects model was 0.83 (0.78, 0.89); for TNM 0-1 0.85 (0.75, 0.95); for TNM II 0.85 (0.78, 0.93); for TNM III 0.83 (0.77, 0.90) and for TNM IV 0.73 (0.63, 0.82).The utility of patients in State P (first year after primary breast cancer) 0.84 (0.80, 0.88); in State R (first year after recurrence) 0.73 (0.69, 0.76), in State S (second and following years after primary breast cancer or recurrence) 0.88 (0.83, 0.92); and in State M (metastatic disease) 0.78 (0.74, 0.82). Mean utility for duration since diagnosis 13 to 36 months was 0.88 (0.80, 0.96, I2 =95%); for 37 to 60 months 0.89 (0.82, 0.96, I2 =90%); for more than 60 months 0.86 (0.76, 0.96, I2 =90%). Mean utility for chemotherapy was 0.86 (0.79, 0.92, I2 =97%); for radiotherapy 0.83 (0.69, 0.96, I2 =97%); surgery 0.80 (0.69, 0.91, I2 =98%); concurrent chemo-radiation 0.70 (0.60, 0.81, I2 =86%) and endocrine therapy 0.90 (0.83, 0.97, I2 =91%). CONCLUSION: This study synthesises the evidence for health state utility values for Chinese women with breast cancer which is useful to inform cost utility models.

A comprehensive review of economic evaluations of therapeutic drug monitoring interventions for cancer treatments.

Therapeutic drug monitoring (TDM) of cancer drugs has been shown to improve treatment effectiveness and safety. The aim of this paper was to comprehensively review economic evaluations of TDM interventions for cancer drugs. Searches were conducted in 4 electronic databases, Medline, EMBASE, and Centre for Reviews and Dissemination databases (Database of Abstracts of Reviews of Effects and the NHS Economic Evaluation Database), from their inception to June 2019. Studies were included if they were economic evaluations of TDM interventions for an active cancer treatment. The quality of reporting of economic evaluations was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS checklist). Of the 11 publications identified, imatinib with TDM and 5-fluorouracil with TDM were the most commonly assessed interventions (4 publications each). Overall study quality was good, with publications meeting 61 to 91% (median 80%) of CHEERS checklist criteria. A variety of studies were used to estimate the clinical effectiveness inputs for the cost effectiveness models. All publications considered TDM to be cost-effective based on an incremental cost-effectiveness ratio below the willingness to pay threshold (64%) or being cost-saving (36%). TDM interventions were considered cost-effective across the economic evaluations. Further clinical research assessing the impact of TDM on overall survival or other long-term health outcomes may enhance the evidence base for TDM in oncology. Future economic evaluations of TDM should explicitly consider uncertainty in the underlying clinical evidence and incorporate changes in the use of newer targeted drugs that form the current standard of care.

Preschool children's preferences for sedentary activity relates to parent's restrictive rules around active outdoor play.

BACKGROUND: With prevalence estimates indicating that young Australian children are increasingly sedentary, it is important to identify the relevant attributes that are shaping this lack of activity. Literature has identified safety concerns of parents as a consistent barrier to physical activity participation of young children. Despite safety being a plausible determinant of young children's activity preferences, the impact of restrictive parenting practices has rarely been examined through quantitative research. The current study investigates the link between controlling and supportive physical activity parenting practices and preschool children's physical activity knowledge, preferences and parent-reported behaviour. METHODS: The current cross-sectional study included 138 parent-child dyads and involved two components of data collection including a child and a parent questionnaire. Results of the parent and child questionnaires were matched to determine correlations between physical activity parenting practices and preschool children's physical activity knowledge, preference and parent-reported behaviour. RESULTS: Children's preferences for physical activity correlated with a number of demographic characteristics and physical activity parenting practices, with the most influential variables being parental age, parental rules around active play outdoors and parental use of screen-time to reward/control child behaviour. Based on parental-reporting, children who preferred to be physically active were more likely to engage in physical activity and were less likely to engage in screen-time on the weekend. CONCLUSIONS: This study identified that parenting practices are not only associated with children's active and sedentary behaviours (parent-reported), but also with how children prefer to play (parent-reported). Future research should seek to clarify the relationship between children's activity preferences and parent's use of screen-time to reward and control their child's behaviour, given the developmental and behavioural health risks associated with excessive media/screen exposure in early childhood. Further research should investigate whether competing societal values of the importance of encouraging children's risky play and the need to prevent children from being injured, coupled with parent's busy schedules are contributing to parental ambivalence regarding how to promote active play for their children. Finally, research should be conducted to establish the relationship between physical activity parenting practices and children's objectively-measured activity level.

Validation of an iPad activity to measure preschool children's food and physical activity knowledge and preferences.

BACKGROUND: Preschool children's knowledge of, and preference for food and physical activity play an important role in the development of lifestyle behaviors throughout childhood. Valid and reliable instruments that are interactive and appealing to preschool children are needed, to obtain quality information in a way that actively engages children and encourages willing participation. The purpose of the current research is to assess the reliability and validity of an adapted computerized (iPad) version of the photo-pair food and exercise questionnaire (PPFEQ). METHODS: The adaptation of the PPFEQ involved generating the questionnaire as an iPad-based tool, updating the photo-pairs within the questionnaire and testing for validity and reliability. This involved four phases of investigation to assess test-retest reliability, internal consistency, sensitivity to change and percent agreement of the questionnaire. RESULTS: The adaption of the PPFEQ resulted in an 18-item questionnaire, titled the preschool food and play questionnaire (Pre-FPQ). The Pre-FPQ demonstrated acceptable reliability and sensitivity to change. Test-retest reliability and internal consistency improved with age, however, it was evident that the tool was not suitable for children younger than 4 years of age. CONCLUSIONS: Children encounter a dynamic world that shapes their knowledge, preferences, choices and behaviors. The Pre-FPQ is an innovative tool to measure preschool children's knowledge of and preference for food and physical activity. The questionnaire offers the advantage of being presented in a well-received modality for preschool children as well as being easy and inexpensive to administer. This new tool is likely to be useful for the assessment of the effectiveness of healthy lifestyle programs implemented in the childcare setting. Future work is needed to refine and improve measures of physical activity preference in preschool children.

Cost-effectiveness of dual-energy X-ray absorptiometry plus antiresorptive treatment in Australian women with breast cancer who receive aromatase inhibitors.

Postmenopausal women with breast cancer on aromatase inhibitor (AI) treatment are at increased risk of bone mineral density loss, which may lead to minimal trauma fractures. We examined the cost-effectiveness of dual energy X-ray absorptiometry (DXA) with antiresorptive (AR) therapy compared with fracture risk assessment, lifestyle advice, and vitamin supplementation. We used a hypothetical Markov cohort model of lifetime duration for 60-year-old women with early stage breast cancer receiving AIs. The data to inform the model came from medical literature, epidemiological reports, and costing data sets. Two eligibility scenarios for AR therapy were considered: (A) osteoporosis and (B) osteopenia or osteoporosis. The main outcomes were incremental cost per quality-adjusted life years gained and cumulative fractures per 1000 women, calculated relative to the comparator. Key aspects of the model were explored in sensitivity analyses. Due to relatively low effectiveness gains, the outcomes were primarily driven by the costs. The incremental cost per quality-adjusted life year gained was A$47,556 and A$253,000 for scenarios A and B, respectively. The numbers of fractures avoided were 56 and 77 per 1000 women, respectively. The results were most sensitive to the initial probability of osteoporosis, baseline risk of fracture, and cohort starting age. Compared with risk assessment and lifestyle advice only, a DXA scan followed by an AR treatment is potentially cost-effective for women aged 60 and over undergoing AI therapy for early breast cancer. However, the number of fractures averted through this intervention is small.

Understanding the context for pet cat and dog feeding and exercising behaviour among pet owners in Ireland: a qualitative study.

BACKGROUND: Pet cat and dog obesity contributes to increased risk of several diseases, including cancer and diabetes mellitus as well as a worsening of orthopaedic problems, and a reduction in survival rate. This study aims to develop a better understanding of cat and dog owners' self-reported beliefs and factors that influence owner behaviour around feeding and exercising their pet cat or dog, as there is a lack of in-depth understanding in this area. Seven focus group discussions, with 43 pet owners in total, were conducted. RESULTS: Pet owners often reported a perceived a low level of control over feeding; often undermined by other people feeding of their pet, their pets begging for food, and their pets attitude towards food. Treats were used in the absence of owner control over pet begging and emotional attachment, and to influence pet behaviour. The majority of participants had positive attitudes to pet exercise, which could be related to pet specific requirements, especially differences in cats and dogs. There were some negative experiences of stress associated with dog walking and fears over aggressive confrontations with other dogs. CONCLUSION: Feeding one's pet is influenced by beliefs about pet specific needs, pet food and pet health, pet owners' perceived control over feeding, and the implications for the pet owner. Pet exercise is influenced by beliefs about pet specific exercise needs, and the implications of exercising one's pet for the pet owner. Understanding owner behaviours on feeding and exercise allows for a more targeted approach to preventing and treating pet obesity.

The effect of a financial incentive on COVID-19 vaccination uptake, and predictors of uptake, in people experiencing homelessness: A randomized controlled trial.

People who are homeless are at increased risk of COVID-19 infection, and of poorer associated outcomes. Delivering vaccinations to, and ensuring uptake of multiple doses in, people who are homeless is complex. Financial incentives may improve vaccination uptake, particularly in people who have not received routine vaccinations previously, though evidence about the effect of incentives is limited and variable. This randomized controlled trial (ANZCTR 383156) assessed the effect of a financial incentive (an A$10 grocery voucher) on uptake of the second COVID-19 vaccination in Australian adults who were homeless, and who had received their first dose. Participants were recruited through a vaccination program for people experiencing homelessness between September 2021 and January 2022. They were followed-up for a minimum of 6.5 months. Uptake was measured 'on-time' at 6 weeks, and at any time during the trial period. Vaccination status was checked on the Australian Immunisation Register. Demographic and vaccination program characteristics associated with uptake were also investigated. Eighty-six people consented to participate, and 43 were randomly allocated to each of the 'incentive' and 'no incentive' groups. The incentive slightly increased the likelihood of a participant receiving a second vaccination on-time (risk difference (RD), 11.6 % [95 %CI, -9.0, 32.2 %]; p = 0.27), and at any time during the trial (RD, 14.0 % [95 %CI, -2.2, 30.1 %], p = 0.09). The incentive had a significant positive effect on uptake in people with no previous vaccination history, increasing their likelihood of receiving a second vaccination on-time (RD, 42.3 % [95 %CI, 15.7, 68.8 %]; p = 0.002) and at any time during the trial (RD, 38.7 % [95 %CI, 16.1, 61.3 %], p < 0.001). Financial incentives may increase COVID-19 vaccination uptake in people who are homeless, and particularly those who have no previous vaccination history. Future research should consider alternative incentive values, types, and cost-effectiveness.

Evaluating the cost utility of estradiol plus dydrogesterone for the treatment of menopausal women in China.

AIM: Evaluate the cost utility of menopausal hormone therapy for women in China. MATERIALS AND METHODS: A bespoke Markov cost utility model was developed to evaluate a cohort of symptomatic perimenopausal women (>45 years) with intact uterus in China in accordance with China's Pharmacoeconomic guideline. Short (5-year) and long (10-year) treatment durations were evaluated over a lifetime model time horizon with 12-month cycle duration. Societal and healthcare payer perspectives were evaluated in the context of a primary care provider/prescriber, outpatient setting with inpatient care for patients with chronic conditions. Disease risk and mortality parameters were derived from focused literature searches, and China Diagnosis-related Group cost data was included. Comprehensive scenario, univariate and probabilistic sensitivity analysis were undertaken along with independent validation. This is the first model to include MHT-related disease risks. RESULTS: According to base case results, the total cost for MHT was 22,516$ (150,106¥) and total quality adjusted life years 12.32 versus total cost of no MHT 30,824$ (205,495¥) and total quality adjusted life years 11.16 resulting in a dominant incremental cost effectiveness ratio of -7,184$ (-47,898¥) per QALY. Results hold true over a range of univariate deterministic sensitivity and scenario analyses. Probabilistic analysis showed a 91% probability of being cost effective at a willingness to pay threshold of three times Gross Domestic Product per capita in China. CONCLUSION: Contingent on the structure and assumptions of the model, combination of estradiol plus dydrogesterone MHT is potentially cost saving in symptomatic women over the age of 45 years in China.

Menopausal hormone therapy is publicly funded in many countries to alleviate symptoms of menopause; however, uptake has been comparatively slow in China. This has implications for the estimated 168 million menopausal-aged women. This analysis is the first to evaluate the cost effectiveness of menopausal hormone therapy in China using best practice principles and incorporating longer term disease risks. Menopausal hormone therapy is potentially cost saving in the context of China.

Methods used to estimate the size of the owned cat and dog population: a systematic review.

BACKGROUND: There are a number of different methods that can be used when estimating the size of the owned cat and dog population in a region, leading to varying population estimates. The aim of this study was to conduct a systematic review to evaluate the methods that have been used for estimating the sizes of owned cat and dog populations and to assess the biases associated with those methods.A comprehensive, systematic search of seven electronic bibliographic databases and the Google search engine was carried out using a range of different search terms for cats, dogs and population. The inclusion criteria were that the studies had involved owned or pet domestic dogs and/or cats, provided an estimate of the size of the owned dog or cat population, collected raw data on dog and cat ownership, and analysed primary data. Data relating to study methodology were extracted and assessed for biases. RESULTS: Seven papers were included in the final analysis. Collection methods used to select participants in the included studies were: mailed surveys using a commercial list of contacts, door to door surveys, random digit dialled telephone surveys, and randomised telephone surveys using a commercial list of numbers. Analytical and statistical methods used to estimate the pet population size were: mean number of dogs/cats per household multiplied by the number of households in an area, human density multiplied by number of dogs per human, and calculations using predictors of pet ownership. CONCLUSION: The main biases of the studies included selection bias, non-response bias, measurement bias and biases associated with length of sampling time. Careful design and planning of studies is a necessity before executing a study to estimate pet populations.

Clinical utility of reproductive carrier screening for preconception and pregnant couples for multiple genetic conditions: a systematic review and meta-analysis.

INTRODUCTION: Many scientific societies have emphasized the importance of evaluating the clinical utility of reproductive carrier screening (RCS). This systematic review aims to assess the clinical utility of RCS and synthesize the outcomes in a meta-analysis. AREAS COVERED: A total of eleven studies were included. The number of conditions screened in the studies varied from three to 176 and led to the identification of one to 24 high-risk couples (HRCs) per 1,000 screened individuals. Pooled estimations were as follows: the prenatal diagnosis (PND) rate among pregnant HRCs 0.644 (95% CI = 0.364, 0.923), the termination rate among affected pregnancies 0.714 (95% CI = 0.524, 0.904), and the rate of in-vitro fertilization (IVF) with preimplantation genetic testing (PGT) 0.631 (95% CI = 0.538, 0.725). There is a statistically significant decrease in the rates of undertaking PND and termination as the number of screened conditions increases. Carriers of conditions classified as having a more severe impact were found to be more likely to choose termination or IVF with PGT. EXPERT OPINION: Our review suggests that the number and the severity of screened conditions can significantly impact HRCs' reproductive decisions. Future work needs to investigate the definition of clinical utility and the design of screening panels.

Demographics and economic burden of un-owned cats and dogs in the UK: results of a 2010 census.

BACKGROUND: The population of dogs and cats passing through rescue shelters may be subject to compromised welfare and increased susceptibility to disease. Little information exists to describe this population, its dynamics and associated management practices. The aim of this study was to carry out a census of un-owned cats and dogs in the UK in 2010, and to document the origins, destinations, husbandry and costs associated with the care of these animals. RESULTS: A sampling frame was constructed by searching the databases of publicly registered charities for England, Scotland and Wales, registers of breed rescues, and by internet searches of animal welfare websites. Overall, 2,352 contacts for 1,380 organisations were identified. All were sent a postal questionnaire asking for data on the number of dogs and cats housed, their origins and eventual outcomes, and details of husbandry between January 1st and December 31st 2010. For those which were registered charities (595), financial records were also obtained.A response rate of 38.8% was obtained. Overall, in 2010, 89,571 dogs and 156,826 cats entered the care of the participating organisations. Approximately half of these animals were relinquished by their owners. Other origins included being found as strays or confiscated for welfare purposes. Seventy-five per cent of dogs and 77.1% of cats were rehomed. The next most common outcome was euthanasia, accounting for 10.4% of dogs and 13.2% cats. For dogs and cats, 44.3% and 62% of participants respectively reported having a waiting list, which frequently exceeded the actual capacity of the facility. Over 19,000 people were involved in the care of these animals, on a paid or voluntary basis. Financial records were available for 519/595 (87.2%) of the registered charities, and their total expenditure in 2010 was £340 million. CONCLUSIONS: This study showed that a large number of animals become un-owned each year, which could have considerable implications for their welfare. Despite the resources expended, demand still exceeds capacity for many organisations, and a substantial number of both cats and dogs are euthanased, suggesting that further understanding of how and why these animals become un-owned is essential in order to target interventions.

Comparing multi-attribute utility instruments: CP-6D, a Cerebral palsy specific instrument, vs AQoL-4D.

BACKGROUND: Economic-evaluations of Cerebral palsy (CP) were based on utility estimates of health-related quality of life (HRQoL) from generic multi-attribute utility instruments (MAUIs). However, generic instruments had limited use as they could not capture some of the important aspects of living with CP. The Cerebral palsy 6 Dimension (CP-6D) is a disease specific MAUI. In this study, we compared the results of CP-6D with the Assessment of Quality of Life (AQoL-4D), a generic MAUI, and tested the criterion validity of the CP-6D in the general population. METHODS: An online survey of the Australian general population (n = 2002), who completed both the AQoL-4D and CP-6D MAUIs, was conducted. Validity was assessed from the correlations between the domains, items and instruments. ANOVA and t-tests were used to assess the instrument's discrimination in different social demographic categories. RESULT: There was a moderate correlation between the instruments (0.64). Differences in socio-demographic characteristics showed a medium effect size (p < 0.001) in both instruments and had a similar effect on utility weights in both instruments. Although the CP-6D was more sensitive to changes in income and education. CONCLUSIONS: Our results suggest that CP-6D and AQoL-4D were measuring a similar underlying construct. Both instruments responded similarly to socio-demographic differences.

Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type I in Australia.

INTRODUCTION: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder and regarded as one of the most frequent genetic causes of infant mortality. The aim of this study is to develop a cost-effectiveness analysis of AVXS-101 (Onasemnogene Abeparvovec/Zolgensma®) and nusinersen (Spinraza®) for SMA to inform decision-making on reimbursement policies in Australia. METHODS: A Markov model was developed with five health states to evaluate the costs and effects for patients with SMA Type I from a healthcare system perspective over a time-horizon of 100 years. The model parameters were based on clinical trials, parametric distributions, published literature, and Australian registries. One-way and probabilistic sensitivity analysis were performed to appraise the uncertainties of the parameters in the model. A threshold analysis was conducted to estimate the cost of AVXS-101 of being cost-effective. RESULTS: The incremental cost-effectiveness ratio (ICER) of AVXS-101 was $1,808,471 per quality-adjusted life year (QALY) and that of nusinersen was $2,772,798 per QALY, compared to standard of care, respectively. The ICER of AVXS-101 was $1,238,288 per QALY compared to nusinersen. The key drivers influencing on ICERs were costs of using treatments and utility values of sitting and walking independently. CONCLUSION: Both nusinersen and AVXS-101 resulted in health benefits, but they were not cost-effective with a commonly used willingness-to-pay (WTP) threshold of $50,000 per QALY. Developing high-quality clinical data and exploring appropriate WTP thresholds are critical for decision-making on reimbursement policies in the treatment of rare diseases.

Impacts of the COVID-19 pandemic, and of government responses to the pandemic, on people who are homeless in Australia: Mapping perceptions of a national sample of homelessness service workers.

People who are homeless are disproportionately impacted by the COVID-19 pandemic, and by government responses to the pandemic. This study maps the perceptions of homelessness service workers in Australia, about the impacts of the COVID-19 pandemic and government responses to it on people who are homeless. An electronic survey was distributed to homelessness service across Australia in June/July 2020, following Australia's 'first wave' of COVID-19. Fifty-nine homelessness services from all eight states/territories responded. Perceptions of impacts on people who are homeless were mapped in six themes: (1) changes in the types of people presenting, (2) overall impacts on peoples' lives, (3) impacts on mental health, (4) impacts of changes in service delivery, (5) impacts of government support and (6) ongoing impacts. The COVID-19 pandemic, and government responses to the pandemic, have affected every aspect of the lives of people who are homeless in Australia. There is a continuing need to support people who are homeless as the world transitions to the 'new normal' of COVID-19, particularly as rates of homelessness increase. Understanding impacts is vital to informing relevant and effective health, social and other supports for this group.

Strategies to improve vaccination rates in people who are homeless: A systematic review.

People who are homeless experience higher rates of vaccine-preventable disease, including COVID-19, than the general population, and poorer associated health outcomes. However, delivering vaccinations to people who are homeless is complex, and there is a lack of evidence to inform practice in this area. The aim of this systematic review is to: (a) identify, (b) analyse the characteristics of, and (c) evaluate the outcomes of, strategies to improve vaccination rates in people who are homeless. Literature was retrieved from eight electronic databases. Studies undertaken in high-income countries, published in English, in a peer-reviewed journal, and in full-text were considered. No limits were placed on study design or date. A total of 1,508 articles were retrieved and, after the removal of duplicates, 637 were screened. Twenty-three articles, reporting on nineteen separate vaccination strategies for hepatitis A/B, influenza, herpes zoster, invasive pneumococcal disease, and diphtheria in people who are homeless, were selected for inclusion. All the strategies were effective at improving vaccination rates in, people who are homeless. Most strategies involved vaccination clinics and most were delivered, at least in part, by nurses. Other characteristics of successful strategies included: delivering vaccinations at convenient locations; using accelerated vaccination schedules (if available); vaccinating at the first appointment, regardless of whether a person's vaccination history or serological status were known (if clinically safe); operating for a longer duration; offering training to staff about working with people who are homeless; widely promoting clinics; considering education, reminders, incentives, and co-interventions; ensuring no out-of-pocket costs; and working collaboratively with stakeholders, including people who are homeless themselves. These findings will inform evidence-based vaccination strategies, including for COVID-19, in people who are homeless, and improve associated health outcomes in this at-risk, hard-to-reach group.

Economic evaluation of reproductive carrier screening for recessive genetic conditions: a systematic review.

INTRODUCTION: Autosomal recessive (AR) and x-linked (XL) conditions are rare but collectively common which impact millions of people globally on morbidity, mortality and costs. Advanced medical technologies allow prospective parents to make informed reproductive decisions to avoid having affected children. Economic evaluations targeting on reproductive carrier screening (RCS) for AR and/or XL conditions have been conducted, but there has not been a systematic review in this area. AREAS COVERED: A systematic search of economic evaluations for RCS was undertaken using the following databases - EMBASE, MEDLINE and SCOPUS. The search strategy was designed to capture full economic evaluations related to RCS since 1990. This review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) strategy. The included 23 studies adopted various types of methodologies to conduct economic evaluations. The majority of studies examined a single condition. The various clinical strategies and screened conditions caused the different cost-effectiveness conclusions in the published studies. EXPERT OPINION: Establishing a validated and practical clinical strategy of RCS and investigating the cost-effectiveness of multiple conditions in one economic evaluation are critical for implementing RCS in the future. Further economic evaluations are essential to provide evidence-based practice for decision-makers.

Evaluating the cost utility of racecadotril in addition to oral rehydration solution versus oral rehydration solution alone for children with acute watery diarrhea in four low middle-income countries: Egypt, Morocco, Philippines and Vietnam.

AIM: To evaluate the cost utility of adjunct racecadotril and oral rehydration solution (R + ORS) versus oral rehydration solution (ORS) alone for the treatment of diarrhoea in children under five years with acute watery diarrhoea in four low-middle income countries. METHOD: A cost utility model, previously developed and independently validated, has been adapted to Egypt, Morocco, Philippines and Vietnam. The model is a decision tree, cohort model programmed in Microsoft Excel. The model structure represents the country-specific clinical pathways. The target population is children under the age of five years presenting with symptoms of acute watery diarrhea to an outpatient clinic or general physician practice. A healthcare payer perspective has been analysed with the model parameterised with local data, where available. Most recent cost data has been used to inform the drug, outpatient and inpatient costs. Uncertainty has been explored with univariate deterministic sensitivity. RESULTS: According to the base case models, R + ORS is dominant (cost-saving, more effective) versus ORS alone in Egypt, Morocco, Philippines and Vietnam. The incremental cost-effectiveness ratios in each country fall in the southeast (cost-saving, more effective) quadrant and represent a cost savings of -304,152 EGP per QALY gain in Egypt; -6,561 MAD per QALY gain in Morocco; -428,612 PHP per QALY gain in Philippines and -113,985,734 VND per QALY gain in Vietnam. Univariate deterministic sensitivity analysis shows that the three most influential parameters across all country adaptations are the utility of children without diarrhea; the utility of inpatient children with diarrhea and the cost of one night of inpatient care. CONCLUSION: In keeping with similar findings in upper-middle and high-income countries, the cost utility of R + ORS versus ORS is favourable in low-middle income countries for the treatment of children under five with acute watery diarrhoea.

PLAIN LANGUAGE SUMMARYDecision-makers rely on cost utility models to inform decisions about whether to publicly fund treatments as part of Universal Health Care. In low-middle income countries, the capacity to prepare cost utility models may be limited and using existing validated models is a practical solution to assist decision making. This study uses a cost utility model developed and independently validated for the United Kingdom, and adapts it to Philippines, Egypt, Morocco and Vietnam. The model evaluates the clinical benefit and economic impact of using racecadotril in addition to rehydration solution to treat diarrhoea in children. The results show that racecadotril is cost-saving and improves the quality of life for children in Philippines, Egypt, Morocco and Vietnam.