An innovative business model using established Medicare items for delivery of cardiac rehabilitation: A value proposition for primary care.

BACKGROUND: Approximately 70% of Australians do not attend cardiac rehabilitation (CR). A potential solution is integrating CR into primary care OBJECTIVE: To propose a business model for primary care providers to implement CR using current Medicare items. DISCUSSION: Using the chronic disease management plan, general practitioners (GPs) complete four clinical assessments at 1-2 weeks, 8-12 weeks, and 6 and 12 months after discharge. The net benefit of applying this model, compared with claiming the most used standard consultation Item 23, in Phase II CR is up to $505 per patient and $543 in Phase III CR. The number of rural GPs providing CR in partnership with the Country Access To Cardiac Health (CATCH) through the GP hybrid model has increased from 28 in 2021 to 32 in 2022. This increase might be attributed to this value proposition. The biggest limitation is access to allied health services in the rural areas.

Measurement properties of utility-based health-related quality of life measures in cardiac rehabilitation and secondary prevention programs: a systematic review.

PURPOSE: To identify utility-based patient-reported outcome measures (PROMs) for assessing health-related quality of life (HRQoL) in cardiac rehabilitation and secondary prevention programs (CR) and appraise existing evidence on their measurement properties. Secondly, to link their items to the International Classification of Functioning Disability and Health (ICF) and the International Consortium of Health Outcome Measures (ICHOM) domains for cardiovascular disease (CVD). METHODS: Eight databases were searched. The review followed the COSMIN and JBI guidelines for measurement properties systematic reviews and PRISMA 2020 reporting guidelines. Non-experimental and observational empirical studies of patients ≥ 18 years of age with CVD undergoing CR and assessed quality of life (QoL) or HRQoL using utility-based PROMs or one accompanied by health state utilities were included. RESULTS: Nine PROMs were identified with evidence on measurement properties for three measures: the German translations of SF-12, EQ-5D-5L, and MacNew heart disease HRQoL questionnaire. There was moderate quality evidence for responsiveness and hypothesis testing of the SF-12 and EQ-5D-5L, and high-quality evidence for responsiveness and hypothesis testing for the MacNew. All items of SF-12 and EQ-5D were linked to ICF categories, but four items of the MacNew were not classified or defined. All the PROM domains were mapped onto similar constructs from the ICHOM global sets. CONCLUSION: Three utility-based PROMs validated in CR were identified: the German versions of the EQ-5D and SF-12 and the MacNew questionnaire. These PROMs are linked to a breadth of ICF categories and all ICHOM global sets. Additional validation studies of PROMs in CR are required.

Accuracy, Acceptability, and Application: Fecal Immunochemical Tests for Early Detection of Advanced Neoplasia in Colonoscopy-Based Surveillance.

BACKGROUND: The fecal immunochemical test (FIT) is widely used in colorectal cancer (CRC) screening, but limited data exist for its application in individuals at above-average risk for CRC who complete surveillance colonoscopies. AIM: To assess the accuracy, acceptability, and effectiveness of FIT in the interval between surveillance colonoscopies, for predicting advanced neoplasia (advanced adenoma or CRC) at the next colonoscopy. METHODS: Individuals enrolled in an Australian surveillance program were included. Diagnostic accuracy was determined for 614 individuals completing a two-sample FIT (OC-Sensor) ≤ 3 months preceding surveillance colonoscopy. 386 Individuals were surveyed to assess acceptability of interval FIT. Additionally, a retrospective analysis was performed on 7331 individuals offered interval FIT between colonoscopies, where a positive FIT (≥ 20 µg hemoglobin/g feces) triggered an early colonoscopy. Associations between interval FIT results and advanced neoplasia were determined using regression analysis. RESULTS: FIT detected CRC and advanced adenoma with sensitivities of 60.0% (3/5) and 27.1% (35/129), respectively. Most (89.1%, 344/386) survey respondents preferred completing interval FIT every 1-2 years. The detection rate of interval FIT for advanced neoplasia decreased with increasing FIT completion. Individuals returning a positive FIT had a higher risk of advanced neoplasia than those who did not complete FIT. Positive interval FIT reduced time-to-diagnosis for CRC and advanced adenoma by a median of 30 and 20 months, respectively. CONCLUSION: Interval FIT was well accepted and enabled earlier detection of advanced neoplasia in individuals at above-average risk of CRC. Given that interval FIT predicts advanced neoplasia, it may be used to personalize surveillance colonoscopy intervals.

Clinical effectiveness of cardiac rehabilitation and barriers to completion in patients of low socioeconomic status in rural areas: A mixed-methods study.

OBJECTIVE: To investigate cardiac rehabilitation utilisation and effectiveness, factors, needs and barriers associated with non-completion. DESIGN: We used the mixed-methods design with concurrent triangulation of a retrospective cohort and a qualitative study. SETTING: Economically disadvantaged areas in rural Australia. PARTICIPANTS: Patients (≥18 years) referred to cardiac rehabilitation through a central referral system and living in rural areas of low socioeconomic status. MAIN MEASURES: A Cox survival model balanced by inverse probability weighting was used to assess the association between cardiac rehabilitation utilization and 12-month mortality/cardiovascular readmissions. Associations with non-completion were tested by logistic regression. Barriers and needs to cardiac rehabilitation completion were investigated through a thematic analysis of semi-structured interviews and focus groups (n = 28). RESULTS: Among 16,159 eligible separations, 44.3% were referred, and 11.2% completed cardiac rehabilitation. Completing programme (HR 0.65; 95%CI 0.57-0.74; p < 0.001) led to a lower risk of cardiovascular readmission/death. Living alone (OR 1.38; 95%CI 1.00-1.89; p = 0.048), having diabetes (OR 1.48; 95%CI 1.02-2.13; p = 0.037), or having depression (OR 1.54; 95%CI 1.14-2.08; p = 0.005), were associated with a higher risk of non-completion whereas enrolment in a telehealth programme was associated with a lower risk of non-completion (OR 0.26; 95%CI 0.18-0.38; p < 0.001). Themes related to logistic issues, social support, transition of care challenges, lack of care integration, and of person-centeredness emerged as barriers to completion. CONCLUSIONS: Cardiac rehabilitation completion was low but effective in reducing mortality/cardiovascular readmissions. Understanding and addressing barriers and needs through mixed methods can help tailor cardiac rehabilitation programmes to vulnerable populations and improve completion and outcomes.

Clinical Effectiveness and Utilisation of Cardiac Rehabilitation After Hospital Discharge: Data Linkage Analysis of 84,064 Eligible Discharged Patients (2016-2021).

BACKGROUND: Despite the highest levels of evidence on cardiac rehabilitation (CR) effectiveness, its translation into practice is compromised by low participation. AIM: This study aimed to investigate CR utilisation and effectiveness in South Australia. METHODS: This retrospective cohort study used data linkage of clinical and administrative databases from 2016 to 2021 to assess the association between CR utilisation (no CR received, commenced without completing, or completed) and the composite primary outcome (mortality/cardiovascular re-admissions within 12 months after discharge). Cox survival models were adjusted for sociodemographic and clinical data and applied to a population balanced by inverse probability weighting. Associations with non-completion were assessed by logistic regression. RESULTS: Among 84,064 eligible participants, 74,189 did not receive CR, with 26,833 of the 84,064 (31.9%) participants referred. Of these, 9,875 (36.8%) commenced CR, and 7,681 of the 9,875 (77.8%) completed CR. Median waiting time from discharge to commencement was 40 days (interquartile range, 23-79 days). Female sex (odds ratio [OR] 1.12; 95% CI 1.01-1.24; p=0.024), depression (OR 1.17; 95% CI 1.05-1.30; p=0.002), and waiting time >28 days (OR 1.15; 95% CI 1.05-1.26; p=0.005) were associated with higher odds of non-completion, whereas enrolment in a telehealth program (OR 0.35; 95% CI 0.31-0.40; p<0.001) was associated with lower odds of non-completion. Completing CR (hazard ratio [HR] 0.62; 95% CI 0.58-0.66; p<0.001) was associated with a lower risk of 12-month mortality/cardiovascular re-admissions. Commencing without completing was also associated with decreased risk (HR 0.81; 95% CI 0.73-0.90; p<0.001), but the effect was lower than for those completing CR (p<0.001). CONCLUSIONS: Cardiac rehabilitation (CR) attendance is associated with lower all-cause mortality/cardiovascular re-admissions, with CR completion leading to additional benefits. Quality improvement initiatives should include promoting referral, women's participation, access to telehealth, and reduction of waiting times to increase completion.

Content Comparison of Quality-of-Life Instruments Used in Economic Evaluations of Sleep Disorder Interventions: A Systematic Review.

BACKGROUND: Assessment of quality of life (QoL) in people living with sleep disorders using questionnaires is necessary to compare intervention benefits. Knowledge of the content and concepts covered by specific QoL instruments is essential to determine which instruments are best suited for conducting economic evaluations of sleep-related interventions. OBJECTIVES: This review aims to identify the QoL instruments that have been applied in economic evaluations of sleep disorder interventions and compare their conceptual overlap and content coverage using the framework of the International Classification of Functioning, Disability and Health (ICF). METHODS: A systematic review of full economic evaluations in sleep published in peer-reviewed journals from conception to 30 May, 2023 was conducted. MEDLINE, PsychInfo, ProQuest, Cochrane, Scopus, CINAHL, Web of Science and Emcare were searched for eligible studies. Studies incorporating either generic or sleep-specific QoL instruments as the primary or secondary measures of effectiveness within a full economic evaluation were included. Quality appraisal against the JBI Critical Appraisal Checklist for Economic Evaluations and EURONHEED checklists and mapping of QoL items to ICF categories were performed by two reviewers, with a third helping settle any potential differences. RESULTS: Sixteen instruments were identified as having been used in sleep health economic evaluations. The EQ-5D-3L, Epworth Sleepiness Scale, and Insomnia Severity Index were the most widely used, but the latter two are predominantly diagnostic tools and not specifically designed to guide economic evaluations. Other instruments with broader ICF content coverage have been least used, and these include the Sleep Apnea Quality of Life Index, Functional Outcomes of Sleep Questionnaire, 15 Dimensions, Short-Form 6 Dimensions, 12-item Short Form Survey, 36-item Short Form Survey and the GRID Hamilton Rating Scale for Depression. CONCLUSIONS: This study provides an overview of current QoL instruments used in economic evaluations of sleep with respect to their content coverage. A combination of generic and sleep-specific instruments with broader ICF content coverage is recommended for such evaluations.

Exploring the Use of Pictorial Approaches in the Development of Paediatric Patient-Reported Outcome Instruments: A Systematic Review.

INTRODUCTION: Children may find self-reporting health-related quality of life (HRQoL) using patient-reported outcome measures (PROMs) presented in text-based formats difficult, particularly younger children and children with developmental delays or chronic illness. In such cases, pictorial PROMs (where pictorial representations are used alongside or to replace text) may offer a valid alternative. AIM: This systematic literature review focused on identifying and describing paediatric PROMs that incorporate pictorial approaches, providing children with more effective means to express their HRQoL. METHODS: Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines were followed. Seven electronic databases were searched from inception to 1 March 2022. There were no country restrictions applied to the search; all English-language studies were considered for inclusion in the review. Characteristics and development methods of the identified pictorial PROMs were evaluated against context-specific good practice guidelines published by The Professional Society for Health Economics and Outcomes Research (ISPOR). RESULTS: A total of 22 paediatric pictorial PROMs, comprising 28 unique versions, were identified. These PROMs were predominantly developed in the USA and the UK, targeting children aged 3-18 years. Likert scales with pictorial anchors, particularly happy-sad faces, were commonly used for response options, appearing in 15 (54%) of the PROMs. Various graphic methods, such as happy-sad faces, cartoons, and thermometers, were adapted to specific content domains. These PROMs covered a wide range of domains, including physical and emotional health and social functioning. Emphasis was placed on content validity, including active child participation in developing pictorial elements. Notably, children's participation was sought during the development of the pictorial elements for 13 (46%) of the PROMs. Various development methods were employed, with 43% of paediatric PROMs using literature reviews, 43% using focus groups, and 32% involving expert consultation. Interviews emerged as the primary method, being employed in 61% of the studies. Additionally, three measures specifically addressed cross-cultural considerations. CONCLUSION: Paediatric pictorial PROMs offer child-friendly tools for assessing HRQoL for application with children who find reading and understanding text-based PROMs challenging. There is some evidence that pictorial PROMs facilitate self-report in this population and improve measurement properties compared to text-only PROMs. Further research is needed to develop, validate, and test paediatric pictorial PROMs, with an emphasis on including children from the inception in the co-design process.

Health economics in nursing research: what you need to know to include economic evaluation methodology in your research.

Due to limited resources and constant, ever-changing healthcare challenges, health economics is essential to support healthcare decisions while improving health outcomes. Economic evaluation methodology facilitates informed decision-making related to the efficient allocation of resources while positively impacting clinical practice. In this paper, we provide an overview of economic evaluation methods and a real-world example applying one method of economic evaluation (cost-utility analysis) in nursing research.

Effectiveness of cardiac rehabilitation programs on medication adherence in patients with cardiovascular disease: A systematic review and meta-analysis.

Background: Education to improve medication adherence is one of the core components of cardiac rehabilitation (CR) programs. However, the evidence on the effectiveness of CR programs on medication adherence is conflicting. Therefore, we aimed to summarize the effectiveness of CR programs versus standard care on medication adherence in patients with cardiovascular disease. Methods: A systematic review and meta-analysis was conducted. Seven databases and clinical trial registries were searched for published and unpublished articles from database inception to 09 Feb 2022. Only randomised controlled trials and quasi-experimental studies were included. Two independent reviewers conducted the screening, extraction, and appraisal. The JBI methodology for effectiveness reviews and PRISMA 2020 guidelines were followed. A statistical meta-analysis of included studies was pooled using RevMan version 5.4.1. Results: In total 33 studies were included with 16,677 participants. CR programs increased medication adherence by 14 % (RR = 1.14; 95 % CI: 1.07 to 1.22; p = 0.0002) with low degree of evidence certainty. CR also lowered the risk of dying by 17 % (RR = 0.83; 95 % CI: 0.69 to 1.00; p = 0.05); primary care and emergency department visit by mean difference of 0.19 (SMD = -0.19; 95 % CI: -0.30 to -0.08; p = 0.0008); and improved quality of life by 0.93 (SMD = 0.93; 95 % CI: 0.38 to 1.49; p = 0.0010). But no significant difference was observed in lipid profiles, except with total cholesterol (SMD = -0.26; 95 % CI: -0.44 to -0.07; p = 0.006) and blood pressure levels. Conclusions: CR improves medication adherence with a low degree of evidence certainty and non-significant changes in lipid and blood pressure levels. This result requires further investigation.

Development and evaluation of a co-designed website for delivering interactive self-directed cardiac rehabilitation.

AIMS: The aim of this study is to report on the development and evaluation of the co-designed website for delivering interactive self-directed cardiac rehabilitation (CR). METHODS AND RESULTS: Multi-method user experience design framework was used to co-design the web application and complete usability testing. Participants were recruited based on their eligibility for CR. Thematic analysis collected the participants' design specifications and lived experiences. The System Usability Scale (SUS) was administered at the completion of the website development and the usability testing workshops. This collected the participants' perceptions of the website's effectiveness, efficiency, and their satisfaction. Website development and usability testing workshops included 39 and 35 participants with a mean age of 66.5 (SD 11.7) and 68.6 (SD 11.2), respectively. Both genders were equally represented across both workshops with 19 (48.7%) and 16 (45.7%) women. Workshop themes guided the design process. The mean SUS scores increased from 66.7 (SD 16.8) to 73.6 (21), P = 0.26. Easiness of use (P = 0.03), integration of the website functions (P ≤ 0.001), and consistency (P = 0.038) significantly improved from website development to usability testing. The proportion of participants rating it as excellent increased from 20.5% to 42.9%, P = 0.11. CONCLUSION: The evolution of our CR website development was completed with an improvement in usability. Upcoming evaluation of this intervention will report on its effectiveness.

Effectiveness of alternative vs traditional exercises on cardiac rehabilitation program utilization in women with or at high risk of cardiovascular disease: a systematic review protocol.

OBJECTIVE: This review will evaluate the effectiveness of alternative vs traditional forms of exercise on cardiac rehabilitation program utilization and other outcomes in women with or at high risk of cardiovascular disease. INTRODUCTION: Exercise-based cardiac rehabilitation programs improve health outcomes in women with or at high risk of cardiovascular disease. However, such programs are underutilized worldwide, particularly among women. Some women perceive traditional gym-based exercise in cardiac rehabilitation programs (eg, typically treadmills, cycle ergometers, traditional resistance training) to be excessively rigorous and unpleasant, resulting in diminished participation and completion. Alternative forms of exercise such as yoga, tai chi, qi gong, or Pilates may be more enjoyable and motivating exercise options for women, enhancing engagement in rehabilitation programs. However, the effectiveness of these alternative exercises in improving program utilization is still inconsistent and needs to be systematically evaluated and synthesized. INCLUSION CRITERIA: This review will focus on randomized controlled trials of studies measuring the effectiveness of alternative vs traditional forms of exercise on cardiac rehabilitation program utilization as well as clinical, physiological, or patient-reported outcomes in women with or at high risk of cardiovascular disease. METHODS: The review will follow the JBI methodology for systematic reviews of effectiveness. Databases including MEDLINE (Ovid), CINAHL (EBSCOhost), Cochrane CENTRAL, Embase (Ovid), Emcare (Ovid), Scopus, Web of Science, LILACS, and PsycINFO (Ovid) will be searched. Two independent reviewers will screen articles and then extract and synthesize data. Methodological quality will be assessed using JBI's standardized instruments. GRADE will be used to determine the certainty of evidence. REVIEW REGISTRATION: PROSPERO CRD42022354996.

Health utility assessments in individuals undergoing diagnostic and surveillance colonoscopy: improved discrimination with a cancer-specific scale.

PURPOSE: To compare the sensitivity and discriminant validity of generic and cancer-specific measures for assessing health-related quality of life (HRQoL) for individuals undergoing diagnostic or surveillance colonoscopy for colorectal cancer. METHODS: HRQoL was assessed using EQ-5D-5L (generic), and EORTC QLQ-C30 (cancer-specific) scales, 14 days after (baseline) and one-year following colonoscopy (follow-up). Utility scores were calculated by mapping EORTC-QLQ-C30 onto QLU-C10D. Differences between participants with different indications for colonoscopy (positive faecal occult blood test (FOBT), surveillance, or symptoms) and colonoscopy findings (no polyps, polyps, or cancer) were tested using Wilcoxon-Mann-Whitney and Kruskal-Wallis H tests. Sensitivity was assessed by calculating the ceiling effects (proportion reporting the best possible level). RESULTS: 246 adults completed the survey, including those undergoing colonoscopy for symptoms (n = 87), positive FOBT (n = 92) or surveillance (n = 67). Those with symptoms had the lowest HRQoL at both baseline and follow-up, with differences observed within the HRQoL domains/areas of role function, appetite loss and bowel function on the QLU-C10D. No differences were found in HRQoL when stratified by findings at colonoscopy with both measures or when comparing baseline and follow-up responses. Participants reporting full health with EQ-5D-5L (21% at baseline and 16% at follow-up) still had problems on the QLU-C10D, with fatigue and sleep at baseline and with role function and fatigue at follow-up. CONCLUSION: Patients undergoing colonoscopy for symptoms had lower HRQoL compared to surveillance or positive FOBT. The cancer-specific QLU-C10D was more sensitive and had greater discriminant ability between patients undergoing colonoscopy for different indications.

Measurement properties of utility-based health-related quality-of-life measures in cardiac rehabilitation: a systematic review protocol.

OBJECTIVE: This review will identify and appraise existing evidence on the measurement properties of utility-based health-related quality-of-life (HRQoL) measures used in cardiac rehabilitation programs. The review will map the measure domains against the International Classification of Functioning, Disability and Health and the International Consortium of Health Outcome Measures domains for cardiovascular disease. INTRODUCTION: Improving HRQoL is an international key indicator for delivering high-quality and person-centered secondary prevention programs. Many instruments and measures assess HRQoL in individuals undergoing cardiac rehabilitation. Utility-based measures are suitable for calculating quality-adjusted life years, a required outcome metric in cost-utility analysis. Cost-utility analysis requires the use of utility-based HRQoL measures. However, there is no consensus on which utility-based measure is best for populations undergoing cardiac rehabilitation. INCLUSION CRITERIA: Eligible studies will include patients aged ≥18 years with cardiovascular disease who are undergoing cardiac rehabilitation. Empirical studies that assess quality of life or HRQoL using a utility-based, health-related, patient-reported outcome measure or a measure accompanied by health state utilities will be eligible. Studies must report at least 1 of the following measurement properties: reliability, validity, responsiveness. METHODS: This review will follow the JBI methodology for systematic reviews of measurement properties. The following databases will be searched from inception to the present: MEDLINE, Emcare, Embase, Scopus, CINAHL, Web of Science Core Collection, Informit, PsyclNFO, REHABDATA, and the Cochrane Library. Studies will be critically appraised using the The COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) risk of bias checklist. The review will be reported in line with the Preferred Reporting Items for Systematic reviews and Meta-Analysis (PRISMA) guidelines. REVIEW REGISTRATION: PROSPERO CRD42022349395.

Responsiveness and construct validity of EPIC-26, AQoL-6D and SF-6D following treatment in prostate cancer.

PURPOSE: To assess construct validity and responsiveness of the Expanded Prostate Cancer Index Composite Instrument (EPIC-26) relative to the Short-Form Six-Dimension (SF-6D) and Assessment of Quality of Life 6-Dimension (AQoL-6D) in patients following treatment for prostate cancer. METHODS: Retrospective prostate cancer registry data were used. The SF-6D, AQoL-6D, and EPIC-26 were collected at baseline and one year post treatment. Analyses were based on Spearman's correlation coefficient, Bland-Altman plots and intra-class correlation coefficient, Kruskal Wallis, and Effect Size and the Standardised Response Mean for responsiveness. RESULTS: The study sample was comprised of 1915 patients. Complete case analysis of 3,697 observations showed moderate evidence of convergent validity between EPIC-26 vitality/hormonal domain and AQoL-6D (r = 0.45 and 0.54) and SF-6D (r = 0.52 and 0.56) at both timepoints. Vitality/hormonal domain also showed moderate convergent validity with coping domain of AQoL-6D (r = 0.45 and 0.54) and with role (r = 0.41 and 0.49) and social function (r = 0.47 and 0.50) domains of SF-6D at both timepoints, and with independent living (r = 0.40) and mental health (r = 0.43) of AQoL-6D at one year. EPIC-26 sexual domain had moderate convergent validity with relationship domain (r = 0.42 and 0.41) of AQoL-6D at both timepoints. Both AQoL-6D and SF-6D did not discriminate between age groups and tumour stage at both timepoints but AQoL-6D discriminated between outcomes for different treatments at one year. All EPIC-26 domains discriminated between age groups and treatment at both timepoints. The EPIC-26 was more responsive than AQoL-6D and SF-6D between baseline and one year following treatment. CONCLUSIONS: AQoL-6D can be used in combination with EPIC-26 in place of SF-12. Although EPIC-26 is not utility based, its popularity amongst clinicians and ability to discriminate between disease-specific characteristics and post-treatment outcomes in clinical trials makes it a candidate for use within cost-effectiveness analyses. The generic measure provides a holistic assessment of quality of life and is suitable for generating quality adjusted life years (QALYs).

Measuring the Wellbeing of Cancer Patients with Generic and Disease-Specific Instruments.

Different wellbeing measures have been used among cancer patients. This study aimed to first investigate the sensitivity of health state utility (HSU), capability, and subjective wellbeing (SWB) instruments in cancer. A cancer-specific instrument (QLQ-C30) was included and transferred onto the cancer-specific HSU scores. Furthermore, it examined the relative importance of key life domains explaining overall life satisfaction. Data were drawn from the Multi-instrument Comparison survey. Linear regression was used to explore the extent to which the QLQ-C30 sub-scales explain HSU and SWB. Kernel-based Regularized Least Squares (KRLS), a machine learning method, was used to explore the life domain importance of cancer patients. As expected, the QLQ-C30 sub-scales explained the vast majority of the variance in its derived cancer-specific HSU (R2 = 0.96), followed by generic HSU instruments (R2 of 0.65-0.73) and SWB and capability instruments (R2 of 0.33-0.48). The cancer-specific measure was more closely correlated with generic HSU than SWB measures, owing to the construction of these instruments. In addition to health, life achievements, relationships, the standard of living, and future security all play an important role in explaining the overall life satisfaction of cancer patients.

Cost-Utility Analysis of Rituximab vs Mycophenolate Mofetil for the Treatment of Pemphigus Vulgaris.

Importance: There is an increasing body of literature that supports the use of rituximab as a first-line steroid-sparing agent in pemphigus vulgaris. However, the cost of rituximab is substantial compared with conventional agents, and there are limited health economic data to justify its use. Objective: To evaluate the cost-effectiveness of rituximab biosimilars relative to mycophenolate mofetil as a first-line steroid-sparing agent for moderate to severe pemphigus vulgaris. Design, Setting, and Participants: A cost-utility analysis over a 24-month time horizon was conducted from the perspective of the Australian health care sector using a modeled cohort of treatment-naive adult patients with moderate to severe pemphigus vulgaris. A Markov cohort model was constructed to simulate disease progression following first-line treatment with rituximab biosimilars or mycophenolate mofetil. The simulated cohort transitioned between controlled disease, uncontrolled disease, and death. Efficacy and utility data were obtained from available published literature. Cost data were primarily obtained from published government data. One-way and probabilistic sensitivity analyses were performed to assess uncertainty. Primary outcomes were the changes in cost and quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) over the 24 months. Interventions: Rituximab biosimilars and mycophenolate mofetil. Results: The simulated cohort of treatment-naive patients had a mean age of 50.8 years, a female-to-male ratio of 1.24, and moderate to severe disease as classified by the Harman criteria. First-line rituximab biosimilars were associated with a cost reduction of AU$639 and an improvement of 0.07 QALYs compared with mycophenolate mofetil, resulting in an ICER of -AU$8818/QALY. Rituximab biosimilars were therefore more effective and less costly compared with mycophenolate mofetil. Sensitivity analyses demonstrated that rituximab biosimilars remained cost-effective across a range of values for cost, utility, and transition probability input parameters and willingness-to-pay thresholds. Conclusions and Relevance: In this cost-utility analysis, rituximab biosimilars were cost-effective compared with mycophenolate mofetil for moderate to severe pemphigus vulgaris. Further investigation into its cost-effectiveness over a longer time horizon is necessary, but the favorable results of this study suggest that the high acquisition costs of rituximab biosimilars may be offset by its effectiveness and provide economic evidence in support of its listing on the Pharmaceutical Benefits Scheme for pemphigus vulgaris.

A Systematic Review of International Guidance for Self-Report and Proxy Completion of Child-Specific Utility Instruments.

OBJECTIVES: This study aimed to identify and summarize published guidance and recommendations for child self- and proxy assessment of existing child-specific instruments of health-related quality of life (HRQoL) that are accompanied by utilities. METHODS: A total of 9 databases plus websites of (1) health technology assessment and health economics outcomes research organizations and (2) instrument developers were systematically searched. Studies were included if they reported guidance for child self- and proxy assessment for child populations (0-18 years old). Three reviewers independently screened titles, abstracts, and full-text reviews against the inclusion criteria. Key features of the guidance identified were summarized. RESULTS: A total of 19 studies met the inclusion criteria. In general, journal articles provided little guidance on child self- and proxy assessment, with the majority focused on instrument development and psychometric performance more broadly. Instrument developers' websites provided more guidance for child self- and proxy reports with specific guidance found for the EQ-5D-Y and the Pediatric Quality of Life Inventory. This guidance included the minimum age for self-report and mode of administration; recommended proxy types, age range of child for whom proxy report can be completed, and target population; and recall period. Websites of leading organizations provided general guidance on HRQoL evaluation in children but lacked specific guidance about self- and proxy completion. CONCLUSIONS: EQ-5D-Y and Pediatric Quality of Life Inventory developers' websites provided the most comprehensive guidance for self-report and proxy report of their respective instruments. More evidence is required for developing best practice guidance on why, when, and how to use self- and proxy reports in assessing HRQoL in child populations.

Evaluation of selective bone scan staging in prostate cancer - external validation of current strategies and decision-curve analysis.

BACKGROUND: Recommendations for staging newly diagnosed prostate cancer patients vary between guidelines and literature. METHODS: Our objective was to validate and compare prediction models selecting newly diagnosed prostate cancer patients for bone scan staging. To achieve this, we validated eleven models in a population-based cohort of 10,721 patients diagnosed with prostate cancer between 2005 and 2019. The primary outcome was net-benefit. This was assessed at different balances of conservatism and tolerance, represented by preference ratio and number-willing-to-test (NWT). Secondary outcomes included calibration slope, calibration-in-the-large (intercept), and discrimination measured by Area-under-the-receiver-operator-characteristics curve (AUC). RESULTS: For preference ratios less than 1:39 (NWT greater than 40), scanning everyone provided greater net-benefit than selective staging. For preference ratios 1:39 to 3:97 (NWT 33-40), the European Association of Urology (EAU) 2020 guideline recommendation was the best approach. For preference ratios 3:97-7:93 (NWT 14-33), scanning EAU high-risk patients only was preferable. For preference ratios 7:93-1:9 (NWT 10-13), scanning only Gnanapragasam Group 5 patients was best. All models had similar fair discrimination (AUCs 0.68-0.80), but most had poor calibration. CONCLUSIONS: We identified three selective staging strategies that outperformed all other approaches but did so over different ranges of conservatism and tolerance. Scanning only EAU high-risk patients provided the greatest net-benefit over the greatest range of preference ratios and scenarios, but other options may be preferable depending upon the local healthcare system's degree of conservatism and tolerance.