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.

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.

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.

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.

The Australian Reproductive Genetic Carrier Screening Project (Mackenzie's Mission): Design and Implementation.

Reproductive genetic carrier screening (RGCS) provides people with information about their chance of having children with autosomal recessive or X-linked genetic conditions, enabling informed reproductive decision-making. RGCS is recommended to be offered to all couples during preconception or in early pregnancy. However, cost and a lack of awareness may prevent access. To address this, the Australian Government funded Mackenzie's Mission­the Australian Reproductive Genetic Carrier Screening Project. Mackenzie's Mission aims to assess the acceptability and feasibility of an easily accessible RGCS program, provided free of charge to the participant. In study Phase 1, implementation needs were mapped, and key study elements were developed. In Phase 2, RGCS is being offered by healthcare providers educated by the study team. Reproductive couples who provide consent are screened for over 1200 genes associated with >750 serious, childhood-onset genetic conditions. Those with an increased chance result are provided comprehensive genetic counseling support. Reproductive couples, recruiting healthcare providers, and study team members are also invited to complete surveys and/or interviews. In Phase 3, a mixed-methods analysis will be undertaken to assess the program outcomes, psychosocial implications and implementation considerations alongside an ongoing bioethical analysis and a health economic evaluation. Findings will inform the implementation of an ethically robust RGCS program.

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.

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.

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.

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.

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.

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.

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.

The Horse as a Therapist: Effects of an Equine Program Without "Therapy" on the Attention and Behavior of Youth Disengaged from Traditional School.

Background: Equine-assisted therapy may promote positive behavior change in young people "at risk." However, it is not always clear what therapeutic content is involved and if a trained therapist is included. The therapeutic effects of the key part of the "therapy," the horse, are not understood. Objectives: To investigate the impact of an equine program without a therapist on attention and behavioral outcomes of young people "at risk." Design: A within subjects pre-post design. A small sample also completed a control period. Setting/location: A small riding center in a rural area of outer Brisbane, Australia. Subjects: Twelve- to 17-year olds (N = 50; 20 girls; mean age 13.88), attending nontraditional flexischool. Intervention: A 5-week program of 2-h long sessions of equine activities that did not include a trained therapist or specific therapeutic content. Outcome measures: Teacher-report measures of externalizing and internalizing behavior were reported before and after the program through the Behavioral Rating Inventory of Executive Function (BRIEF) and the Strength and Difficulties Questionnaire (SDQ). Results: A paired samples t test resulted in the statistically significant reduction of the BRIEF Global Executive Score between pre and post participation in the equine program (mean difference = -5.89), t(36) = -3.377, p = 0.002 and the SDQ Hyperactivity score (mean difference = -0.727), t(43) = -2.244, p = 0.030. Equine activities may reverse a trajectory of worsening problems. This may especially affect symptoms related to attention deficit hyperactivity disorder. Conclusion: Equine programs may offer an alternative method to reduce poor behavior and improve attention in young people. Benefits in attention may occur even without specific therapeutic content or therapist involvement. It is proposed that some of these benefits come directly from the horse and the interactions with the horse; others are contextual.

Interventions for children and adolescents who stutter: A systematic review, meta-analysis, and evidence map.

PURPOSE: This systematic review critically appraises and maps the evidence for stuttering interventions in childhood and adolescence. We examine the effectiveness of speech-focused treatments, the efficacy of alternative treatment delivery methods and identify gaps in the research evidence. METHODS: Nine electronic databases and three clinical trial registries were searched for systematic reviews, randomised controlled trials (RCTs) and studies that applied an intervention with children (2-18 years) who stutter. Pharmacological interventions were excluded. Primary outcomes were a measure of stuttering severity and quality assessments were conducted on all included studies. RESULTS: Eight RCTs met inclusion criteria and were analysed. Intervention approaches included direct (i.e. Lidcombe Program; LP) and indirect treatments (e.g. Demands and Capacities Model; DCM). All studies had moderate risk of bias. Treatment delivery methods included individual face-to-face, telehealth and group-based therapy. Both LP and DCM approaches were effective in reducing stuttering in preschool aged children. LP had the highest level of evidence (pooled effect size=-3.8, CI -7.3 to -0.3 for LP). There was no high-level evidence for interventions with school-aged children or adolescents. Alternative methods of delivery were as effective as individual face-to-face intervention. CONCLUSION: The findings of this systematic review and evidence mapping are useful for clinicians, researchers and service providers seeking to understand the existing research to support the advancement of interventions for children and adolescence who stutter. Findings could be used to inform further research and support clinical decision-making.

Utility Values for the CP-6D, a Cerebral Palsy-Specific Multi-Attribute Utility Instrument, Using a Discrete Choice Experiment.

BACKGROUND AND OBJECTIVE: The CP-6D is a new preference-based measure derived from the CPQOL, a cerebral palsy-specific quality-of-life questionnaire. The CP-6D contains six dimensions, each with five levels. A preference-based value set is required to score the CP-6D on a utility scale and render it suitable for cost-utility analysis. This study aims to estimate the utility value set for the CP-6D for interventions for people with cerebral palsy (CP). METHODS: A discrete choice experiment was designed and administrated to an adult Australian online panel. Each respondent answered 12 choice sets. Each choice was presented as a combination of the health state from the CP-6D and duration spent in that health state before death. Conditional logit and mixed logit regression were used to analyse the data. The utility values were estimated as a ratio of the coefficient of each dimension to the coefficient of the duration. RESULTS: A total of 2002 participants completed the survey and responded to each choice. Generally, the dimension levels were monotonic, meaning the coefficients reflected the ordered nature of the levels in each dimension. The dimensions relating to manual ability, social well-being and acceptance had the greatest effect on choice. The value of the worst 'pits' health state is - 0.582. CONCLUSION: This study provides the first CP-specific utility value set that can potentially be used in cost-utility analyses of interventions for people with CP where the CPQOL has been applied, both prospectively and retrospectively.

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.

Using Rasch and factor analysis to develop a Proxy-Reported health state classification (descriptive) system for Cerebral Palsy.

PURPOSE: The Cerebral Palsy quality of life instrument is a well-known health-related quality of life measure for children with Cerebral Palsy. Due to its length it is not suitable as the basis of a preference-based instrument. The aim of this study is to develop a short version of the Cerebral Palsy quality of life instrument that can subsequently be scored as a multi-attribute utility instrument through assigning preference-based values. METHODS: A sample of 473 participants who have a child with Cerebral Palsy completed the Cerebral Palsy quality of life instrument(proxy-version) instrument. After deleting questions related only to the proxy, the dimensional structure was obtained using exploratory factor analysis. Extended Rasch analysis was then undertaken to test the psychometric performance of items and select the best item to represent each dimension. Expert opinion was sought to confirm the dimensions and items. RESULTS: A six-dimension classification system was identified, in which four domains were extracted from the factor analysis. Following expert opinion, two other domains were also added, as these were considered to have significant impact on health-related quality of life in children with Cerebral Palsy. CONCLUSIONS: The combination of Factor and Rasch analysis along with consultation with patients, clinicians and experts in health-related quality-of-life instrument development, has resulted in a short version of the Cerebral Palsy quality of life instrument.IMPLICATION FOR REHABILITATIONThis study provides the first classification system for children with Cerebral Palsy.The Cerebral Palsy-six dimension (CP-6D) survey, which is a short version of Cerebral Palsy Quality Of Life instrument, can be timesaving when measuring quality of life in children with Cerebral Palsy.The short version (CP-6D) can be used in preference based measurement and generate quality adjusted life years for children with Cerebral Palsy.

Cost-Effectiveness of Lenvatinib Compared with Sorafenib for the First-Line Treatment of Advanced Hepatocellular Carcinoma in Australia.

BACKGROUND AND OBJECTIVE: In the REFLECT trial, lenvatinib showed superior clinical benefits to sorafenib in terms of progression-free survival and was non-inferior for overall survival in the treatment of advanced hepatocellular carcinoma (HCC). We assessed the cost-effectiveness of lenvatinib compared with sorafenib for patients with advanced HCC in Australia. METHOD: A partitioned-survival model was built to perform a cost-effectiveness analysis comparing lenvatinib and sorafenib from an Australian health-system perspective. Survival curves were obtained from the REFLECT trial and fitted with parametric survival functions for extrapolation purposes beyond the trial follow-up. Cost and quality-adjusted life-years (QALYs) were accrued over the 10-year time horizon of the model. Deterministic and probability sensitivity analysis (PSA) were carried out to verify the validity of the model. RESULTS: Lenvatinib incurred higher costs (A$96,325) and superior health outcomes (QALYs: 1.205), while sorafenib had lower costs (A$92,394) and inferior health outcomes (QALYs: 1.086). Thus, lenvatinib yielded an incremental cost-utility ratio of A$33,028/QALY gained. Further, the results of the PSA found that the probability of lenvatinib being cost-effective at a willingness-to-pay threshold of A$50,000/QALY was 64%. CONCLUSION: Our study found that, at current prices, lenvatinib is a cost-effective treatment option compared with sorafenib for the first-line treatment of patients with advanced HCC.

Does policy change to allow pharmacist provision of influenza vaccination increase population uptake? A systematic review.

Objective The aims of this study were to estimate the effect of pharmacists' vaccinating for influenza on overall vaccination rates and to assess whether any effect differs for at-risk subgroups compared with the general population. Methods A systematic review was undertaken, adhering to the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines. Databases were searched during July 2019 and included Medline (Ovid), Cumulative Index to Nursing and Allied Health Literature (CINAHL), Scopus and the Cochrane Library. Results The largest difference reported in overall population vaccination rates associated with pharmacists undertaking influenza vaccinations was an increase of 10%; the smallest showed no discernible effect. The effect was graduated: pharmacists with the most autonomy demonstrated the largest rate increases. There was evidence of substitution by pharmacists, but the effect size was small. Conclusions The effect of allowing pharmacists to administer influenza vaccinations appears positive, but small. Given that pharmacists are likely to provide vaccinations at a lower cost than doctors, there may be cost-savings to the health system and consumers. Future research may include evaluating pharmacist-provided vaccinations compared with (or in combination with) other strategies, such as advertising, to increase access and uptake across the range of providers, as well as ongoing research to address vaccine hesitancy. What is known about the topic? In Australia, and many other countries, community pharmacies provide an alternative and accessible option for influenza vaccination; however the effect on overall vaccination rates remains unclear. What does this paper add? This systematic review of the international literature suggests that pharmacist-provided vaccinations increase uptake; substitution of doctors by pharmacists may result in cost savings. What are the implications for practitioners? The findings of this study are important for health policy makers and health workforce researchers aiming to maximise population vaccination rates and workforce efficiency. In the absence of available Australian data, data from the international experience of legislating pharmacists to vaccinate against influenza are summarised and critiqued. Results can be used when determining the best health workforce and policy mix with regard to the vaccination workforce.