Cost-utility and cost consequence of a telehealth intervention targeting improvement in addictive eating for Australian adults (the TRACE program).

BACKGROUND: The TRACE (Targeted Research for Addictive and Compulsive Eating) intervention was evaluated in a 3-month randomized controlled trial which demonstrated significant improvement in Yale Food Addiction Scale scores favoring dietitian-led telehealth (active intervention) compared with passive and control groups. This study aimed to determine intervention costs and cost-utility. METHODS: Costs of each intervention (2021$AUD) and incremental net monetary benefit (iNMB; incremental benefit, defined as Quality-Adjusted Life Years (QALY) gained, multiplied by willingness to pay threshold minus incremental cost) were calculated to estimate differences between groups. RESULTS: The active intervention (n = 38) cost $294 (95% UI: $266, $316) per person compared to $47 (95% UI: $40, $54) in the passive intervention (n = 24), and $26 in the control group (n = 37). At a cost-effectiveness threshold of $50 000 per QALY score gained, the active intervention iNMB was -$186 (95% UI: -$1137, $834) and the passive group $127 (95% UI: -$1137, $834). Compared to the control group, estimates indicate a 30% chance of the active intervention, and a 60% chance of the passive intervention being cost effective. CONCLUSION: Although the overall cost of the active intervention was low, this was not considered cost-effective in comparison to the passive intervention, given small QALY score gains. TRIAL REGISTRATION: Australia New Zealand Clinical Trial Registry ACTRN12621001079831.

Exploring the potential cost-effectiveness of a new computerised decision support tool for identifying fetal compromise during monitored term labours: an early health economic model.

BACKGROUND: Around 60% of term labours in the UK are continuously monitored using cardiotocography (CTG) to guide clinical labour management. Interpreting the CTG trace is challenging, leading to some babies suffering adverse outcomes and others unnecessary expedited deliveries. A new data driven computerised tool combining multiple clinical risk factors with CTG data (attentive CTG) was developed to help identify term babies at risk of severe compromise during labour. This paper presents an early health economic model exploring its potential cost-effectiveness. METHODS: The model compared attentive CTG and usual care with usual care alone and simulated clinical events, healthcare costs, and infant quality-adjusted life years over 18 years. It was populated using data from a cohort of term pregnancies, the literature, and administrative datasets. Attentive CTG effectiveness was projected through improved monitoring sensitivity/specificity and potential reductions in numbers of severely compromised infants. Scenario analyses explored the impact of including litigation costs. RESULTS: Nationally, attentive CTG could potentially avoid 10,000 unnecessary alerts in labour and 2400 emergency C-section deliveries through improved specificity. A reduction of 21 intrapartum stillbirths amongst severely compromised infants was also predicted with improved sensitivity. Attentive CTG could potentially lead to cost savings and health gains with a probability of being cost-effective at £25,000 per QALY ranging from 70 to 95%. Potential exists for further cost savings if litigation costs are included. CONCLUSIONS: Attentive CTG could offer a cost-effective use of healthcare resources. Prospective patient-level studies are needed to formally evaluate its effectiveness and economic impact in routine clinical practice.

The effect of two speech and language approaches on speech problems in people with Parkinson's disease: the PD COMM RCT.

Background: Speech impairments are common with Parkinson's disease (reported prevalence 68%), increasing conversational demands, reliance on family and social withdrawal. Objective(s): The PD COMM trial compared the clinical and cost-effectiveness of two speech and language therapy approaches: Lee Silverman Voice Treatment LOUD and National Health Service speech and language therapy for the treatment of speech or voice problems in people with Parkinson's disease to no speech and language therapy (control) and against each other. Design: PD COMM is a phase III, multicentre, three-arm, unblinded, randomised controlled trial. Participants were randomised in a 1 : 1 : 1 ratio to control, National Health Service speech and language therapy or Lee Silverman Voice Treatment LOUD via a central computer-generated programme, using a minimisation procedure with a random element, to ensure allocation concealment. Mixed-methods process and health economic evaluations were conducted. Setting: United Kingdom outpatient and home settings. Participants: People with idiopathic Parkinson's disease, with self-reported or carer-reported speech or voice problems. We excluded people with dementia, laryngeal pathology and those within 24 months of previous speech and language therapy. Interventions: The Lee Silverman Voice Treatment LOUD intervention included maximum effort drills and high-effort speech production tasks delivered over four 50-minute therapist-led personalised sessions per week, for 4 weeks with prescribed daily home practice. National Health Service speech and language therapy content and dosage reflected local non-Lee Silverman Voice Treatment speech and language therapy practices, usually 1 hour, once weekly, for 6 weeks. Trained, experienced speech and language therapists or assistants provided interventions. The control was no speech and language therapy until the trial was completed. Main outcome measures: Primary outcome: Voice Handicap Index total score at 3 months. Secondary outcomes: Voice Handicap Index subscales, Parkinson's Disease Questionnaire-39; Questionnaire on Acquired Speech Disorders; EuroQol-5D-5L; ICEpop Capabilities Measure for Older Adults; Parkinson's Disease Questionnaire - Carers; resource utilisation; and adverse events. Assessments were completed pre-randomisation and at 3, 6 and 12 months post randomisation. Results: Three hundred and eighty-eight participants were randomised to Lee Silverman Voice Treatment LOUD (n = 130), National Health Service speech and language therapy (n = 129) and control (n = 129). The impact of voice problems at 3 months after randomisation was lower for Lee Silverman Voice Treatment LOUD participants than control [-8.0 (99% confidence interval: -13.3, -2.6); p = 0.001]. There was no evidence of improvement for those with access to National Health Service speech and language therapy when compared to control [1.7 (99% confidence interval: -3.8, 7.1); p = 0.4]. Participants randomised to Lee Silverman Voice Treatment LOUD reported a lower impact of their voice problems than participants randomised to National Health Service speech and language therapy [99% confidence interval: -9.6 (-14.9, -4.4); p < 0.0001]. There were no reports of serious adverse events. Staff were confident with the trial interventions; a range of patient and therapist enablers of implementing Lee Silverman Voice Treatment LOUD were identified. The economic evaluation results suggested Lee Silverman Voice Treatment LOUD was more expensive and more effective than control or National Health Service speech and language therapy but was not cost-effective with incremental cost-effectiveness ratios of £197,772 per quality-adjusted life-year gained and £77,017 per quality-adjusted life-year gained, respectively. Limitations: The number of participants recruited to the trial did not meet the pre-specified power. Conclusions: People that had access to Lee Silverman Voice Treatment LOUD described a significantly greater reduction in the impact of their Parkinson's disease-related speech problems 3 months after randomisation compared to people that had no speech and language therapy. There was no evidence of a difference between National Health Service speech and language therapy and those that received no speech and language therapy. Lee Silverman Voice Treatment LOUD resulted in a significantly lower impact of voice problems compared to National Health Service speech and language therapy 3 months after randomisation which was still present after 12 months; however, Lee Silverman Voice Treatment LOUD was not found to be cost-effective. Future work: Implementing Lee Silverman Voice Treatment LOUD in the National Health Service and identifying alternatives to Lee Silverman Voice Treatment LOUD for those who cannot tolerate it. Investigation of less costly alternative options for Lee Silverman Voice Treatment delivery require investigation, with economic evaluation using a preference-based outcome measure that captures improvement in communication. Study registration: This study is registered as ISRCTN12421382. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 10/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 58. See the NIHR Funding and Awards website for further award information.

Most people with Parkinson's disease develop difficulties with their speech and voice. Communicating becomes difficult. This affects their relationships, work, social life and how they feel about themselves. Our PD COMM trial compared two types of speech and language therapy to find out if they helped the speech and voice problems people with Parkinson's have. We measured changes in the way their voice and speech problems affected their lives and how much therapy cost the National Health Service and families. Everyone taking part had speech or voice problems because of their Parkinson's disease. People could not take part if they had dementia, evidence of laryngeal pathology or previous laryngeal surgery or received speech and therapy for Parkinson's disease in the last 2 years. People who agreed to take part joined one of three groups, which were alike except for the therapy they received. A computer decided which group they joined by chance. National Health Service speech and language therapy Lee Silverman Voice Treatment LOUD No speech and language therapy for 12 months The 388 people who took part came from 41 outpatient clinics in Scotland, England and Wales. Most were older men. The people that received Lee Silverman Voice Treatment LOUD felt better about their speech and voice after 3 months compared to people in the other groups. A year later, they still felt better about it. People that received National Health Service therapy had no benefit compared to people with no access to therapy. Analysis of cost-effectiveness indicated that Lee Silverman Voice Treatment LOUD did not offer value for money and the intervention cost more because more speech and language therapy time was needed to deliver it. Our next question is to ask how we can provide Lee Silverman Voice Treatment LOUD in a way that costs less, for example, using therapy assistants and computer packages or at home. Clear speech and language therapy approaches for people with Parkinson's disease and speech or voice problems should be tested in trials that measure changes in people's lives.

Cost-effectiveness intervention thresholds for romosozumab and teriparatide in the treatment of osteoporosis in the UK.

Sequential romosozumab-to-alendronate or sequential teriparatide-to-alendronate can be a cost-effective treatment option for postmenopausal women at very high risk of fracture. PURPOSE: To estimate the 10-year probability of a major osteoporotic fracture (MOF) at which sequential treatment with romosozumab or teriparatide followed by alendronate, compared with alendronate alone, becomes cost-effective in a UK setting. METHODS: A microsimulation model with a Markov structure was used to simulate fractures, costs, and quality-adjusted life years (QALYs), in women receiving sequential treatment with either romosozumab or teriparatide followed by alendronate, compared with alendronate alone. Patients aged 50 to 90 years with a recent MOF, hip or spine fracture were followed from the start of a 5-year treatment until the age of 100 years or death. The analysis had a healthcare perspective. Efficacy of romosozumab, teriparatide and alendronate was derived from phase III randomised controlled trials. Resource use and unit costs were derived from the literature. Cost-effectiveness intervention threshold (CEIT), defined as the 10-year probability of a major osteoporotic fracture at which treatment becomes cost-effective, was compared with clinically appropriate intervention thresholds for bone-forming treatment in women with very high fracture risk as recommended by the UK National Osteoporosis Guideline Group (NOGG). RESULTS: The base case analysis showed that sequential romosozumab-to-alendronate treatment was cost-effective from a 10-year MOF probability of 18-35% and above depending on age and site of sentinel fracture at a willingness to pay (WTP) of £30,000. For teriparatide-to-alendronate, treatment was cost-effective at a 10-year MOF probability of 27-57%. The results were sensitive to pricing of the drugs but relatively insensitive to treatment duration, romosozumab persistence assumptions, and site of sentinel fracture. The CEITs for romosozumab-to-alendronate treatment were lower than the clinical thresholds from the age of 70 years meaning that treatment could be considered both cost-effective and aligned with the NOGG treatment guidelines. By contrast, for teriparatide-to-alendronate the CEITs were higher than the clinical thresholds irrespective of age. However, cost-effective scenarios were found in the presence of strong clinical risk factors in addition to a recent sentinel fracture. CONCLUSION: The results of this study indicate that sequential romosozumab-to-alendronate or teriparatide-to-alendronate treatment can be a cost-effective treatment option for postmenopausal women at very high risk of fracture.

A systematic review and quality assessment of economic evaluations of kidney replacement therapies in end-stage kidney disease.

End-stage kidney disease (ESKD) is fatal without treatment by kidney replacement therapies (KRTs). However, access to these treatment modalities can be problematic given the high costs. This systematic review (SR) aims to provide an updated economic evaluation of pairwise comparisons of KRTs and the implications for the proportion of patients with access to the KRT modalities, i.e., kidney transplantation (KT), hemodialysis (HD), and peritoneal dialysis (PD). This SR was conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) 2020. We searched studies in PubMed, Embase, Scopus, and Cost Effectiveness Analysis (CEA) registry, from inception to March 2023. Thirteen studies were included with pairwise comparisons among three KRTs, with varying proportions of patients for each modality. Seven studies were from high-income countries, including five from Europe. Summary findings are presented on a cost-effectiveness plane and incremental net benefit (INB). KT was the most cost-effective intervention across the pairwise comparisons. KT and PD were both more cost-effective alternatives to HD. HD was more costly and less effective than PD in all studies except one. Concurrent efforts to increase both KT and PD represented the best scenario to improve treatment options for ESKD patients.

Health literacy and cumulative social disadvantage are associated with survival and transplant in patients with hepatocellular carcinoma: a prospective study.

OBJECTIVE: To investigate how individual social determinants of health (SDOH) and cumulative social disadvantage (CSD) affect survival and receipt of liver transplant (LT) in patients with hepatocellular carcinoma (HCC). METHODS: We enrolled 139 adult patients from two Indianapolis hospital systems between June 2019 and April 2022. Structured questionnaires collected SDOH and social risk factor data. We compared SDOH and CSD by race, gender and disease aetiology, assigning one point per adverse SDOH. Multivariable competing risk survival analysis assessed associations between SDOH, CSD, survival and LT receipt. RESULTS: Black patients experienced higher CSD than white patients in the cohort (5.4±2.5 vs 3.2±2.1, p<0.001). Black patients were significantly more likely to have household incomes

Evaluating cost-effectiveness of PFO management strategies: closure with cardioform vs. amplatzer, and treatment with medical therapy alone, for secondary stroke prevention.

AIM: The aim of this study was to evaluate the cost-effectiveness of patent foramen ovale (PFO) closure using CARDIOFORM Septal Occluders versus AMPLATZER Septal Occluders, as well as compared to Medical Therapy Alone, from a payor perspective in the United States. METHODS: An economic evaluation compared the value of CARDIOFORM, AMPLATZER, and Medical Therapy Alone. A Markov model simulated a cohort of 1,000 individuals with PFO and a history of cryptogenic stroke, with baseline demographic and clinical characteristics reflecting individuals enrolled in the REDUCE and RESPECT trials over a five-year time horizon. The costs and health consequences associated with complications and adverse events, including recurrent stroke, were compared over a time horizon of 5 years. RESULTS: PFO closure using CARDIOFORM was economically dominant, providing both cost-savings and improved effectiveness compared to closure with AMPLATZER. It resulted in an estimated savings of over $1.3 million, an additional 24.8 quality-adjusted life-years (QALYs) gained, and 26 strokes avoided in a cohort of 1,000 patients. When compared to Medical Therapy Alone, closure with CARDIOFORM was found to be cost-effective, with an incremental cost-effectiveness ratio (ICER) of $36,697 per QALY gained. Sensitivity and scenario analysis showed the model findings to be highly robust across reasonable changes to baseline input values and assumptions. CONCLUSIONS: The results of this analysis suggest that PFO closure using the CARDIOFORM Septal Occluder is the most cost-effective treatment strategy for patients with a PFO-associated stroke, particularly compared to AMPLATZER where it resulted in both cost-saving and improved patient outcomes.

Why was this study done? Patent foramen ovale (PFO) is a small opening in the heart that can allow blood clots to pass from one side to the other, increasing the risk of strokes, particularly those without a clear cause, known as cryptogenic strokes. Closing the PFO can help prevent these strokes. This study aimed to determine which treatment option is the best value for money and most effective for preventing secondary strokes in patients with PFO: using the CARDIOFORM Septal Occluder, the AMPLATZER Septal Occluder, or using only medications.What did the researchers do? We used a computer model to simulate the health outcomes and costs for 1,000 patients with PFO who had experienced a cryptogenic stroke. The model followed these patients over five years and compared three treatment strategies: closing the PFO with the CARDIOFORM device, closing it with the AMPLATZER device, and using medications alone (Medical Therapy Alone).What did the researchers find? Our findings showed that:The CARDIOFORM device was more cost-effective and provided better health outcomes than the AMPLATZER device.Using the CARDIOFORM device saved over $1.3 million, added nearly 25 more years of good-quality life (measured as quality-adjusted life years or QALYs), and prevented 26 strokes in a group of 1,000 patients.Compared to using medications alone, the CARDIOFORM device was cost-effective, with a cost of about $36,700 for each year of good-quality life gained. What do the results mean? These results suggest that using the CARDIOFORM device to close a PFO is the best strategy for preventing secondary strokes when compared to AMPLATZER and Medical Therapy Alone. This approach not only saves money but also improves patient outcomes compared to the AMPLATZER device and using medications alone.How could this study help patients? This study provides clear and well-grounded information that helps patients, healthcare providers, and policymakers make informed decisions about the best treatment strategies for preventing secondary strokes in patients with PFO. Using the CARDIOFORM device can lead to better health outcomes and cost savings, ultimately improving the quality of life for patients.

Validity of a visual analogue scale to measure and value the perceived level of sanitation - evidence from Ghana and Mozambique.

Two billion people globally lack access to a basic toilet, and sanitation is a critical determinant of health and wellbeing. Evaluations of sanitation programmes typically measure disease or behaviour, and visual analogue scales (VAS) have not been used to measure users' feelings about their level of sanitation. In this study, we assess the validity of a horizontal sanitation VAS numbered 0-10, with end-anchors "best imaginable" and "worst imaginable" sanitation. In Kumasi, Ghana, we surveyed 291 participants before and after uptake of a container-based sanitation service. In Maputo, Mozambique, we surveyed 424 participants from treatment groups of a prior trial. We assessed construct validity by testing hypothesised associations between VAS scores and toilet characteristics, and by respondents valuing three hypothetical sanitation states. We assessed responsiveness by comparing VAS with/without sanitation interventions. There was evidence (p<0.05) for 60% of hypothesised associations in Ghana, and 100% in Mozambique. For responsiveness, there was a 3.4 point increase (2.1 SD) in VAS 10 weeks post-intervention in Ghana, and a 2.9 point difference (1.3 SD) in Mozambique. In valuation exercises, the mean was higher (p<0.001) for the objectively better sanitation state. The sanitation VAS could be useful in economic evaluation to identify which improvements achieve quality of life gains most efficiently. For future studies we recommend a vertical sanitation VAS numbered 0-100 with emojis at end-anchors but retaining a 0-10 option for those who struggle with numeracy.

Medical Costs and Caregiver Burden of Delivering Disease-Modifying Alzheimer's Treatments with Different Duration and Route of Administration.

BACKGROUND: Multiple disease modifying treatment for Alzheimer's disease are currently in clinical development or have been recently approved for use. They have vastly different treatment properties but so far, little work has been done to quantify the impact of treatment properties on the treatment's value in terms of medical and social care costs and caregiver burden. OBJECTIVES: This study aims to analyze how the mode of treatment administration, treatment frequency and duration, and monitoring requirements affect the value of disease modifying treatments. In order to isolate these effects, we compare five hypothetical disease modifying treatments with equal efficacy and safety: (1) chronic bi-weekly intravenous infusion, (2) chronic four-weekly intravenous infusion, (3) 52 weeks fixed duration four-weekly intravenous infusion, (4) chronic subcutaneous injections, and (5) chronic oral prescription on their direct medical costs, caregiver burden, and preservation of treatment value. DESIGN: Survey of Alzheimer's disease treatment clinics and retrospective data analysis. SETTING: United States. MEASUREMENTS: Direct medical cost and caregiver burden of treatment administration and monitoring compared to gross treatment benefit. RESULTS: Chronic bi-weekly infusion treatment had the highest direct medical cost ($45,208) and caregiver burden ($6,095), reducing the treatment value by 44%, while oral treatment with the lowest direct medical cost ($1,983) and caregiver burden ($457) reduced the treatment value by only 2%. Substantial caregiver burden was reported from the survey, with a reported average of 2.3 hours for an office visit and infusion, 44 minutes of round-trip travel time, and 78% of patients being accompanied by a caregiver for treatment. CONCLUSION: Burden of chronic intravenous treatments exceed the gross medical and social care cost savings and value of caregiver benefit. The results suggest the need for less complex treatments that require fewer clinic visits to preserve the economic value of disease modifying treatments.

Insights and networks: methodological assessment and scientometric analysis of economic evaluations in dentistry.

INTRODUCTION: Assessing the methodological quality of economic evaluations (EEs) is crucial for evidence-based decision-making. The study aimed to evaluate EEs in restorative dentistry and endodontics, while also analyzing the scientific landscape of researchers and publications through co-authorship and citation network analysis providing an insight into the distribution of scientific expertise. METHODOLOGY: A systematic search for relevant articles from 2012 to 2022 was conducted using PubMed, Scopus, and EBSCO. The ten-point Drummond checklist was used to appraise the methodological quality of included studies. Bibliometric data for network analysis were extracted from the Dimensions database and visualized using VOSviewer software. RESULTS: Of the 37 articles, 81.08% scored good, 16.21% average, and 2.7% poor on the methodological rating scale. Most of the included studies were in Q1 journals, with limited representation in Q2 and Q3 journals. Compliance was highest in Q2 journals (95%), followed by Q1 (88.36%), while it dropped to 40% for Q3 journals. Co-authorship analysis revealed a dense network of researchers, with Prof. Falk Schwendicke V. having a significant influence. Moreover, the Journal of Dentistry had the highest impact, followed by Journal of Endodontics and BMC Oral Health. CONCLUSIONS: Despite a diverse scientific landscape, participation from developing countries was limited emphasizing the need for inclusivity and diversity in the scientific network. While the quantity of good-quality studies was encouraging, the overall quality of evidence remains paramount for decision-making in healthcare policy and practice. Therefore, continuous efforts to improve methodological rigor and reporting practices are essential to contribute robust evidence.

Cost-effectiveness of proton beam therapy vs. conventional radiotherapy for patients with brain tumors in Sweden: results from a non-randomized prospective multicenter study.

BACKGROUND: This study assessed the cost-effectiveness of proton beam therapy (PBT) compared to conventional radiotherapy (CRT) for treating patients with brain tumors in Sweden. METHODS: Data from a longitudinal non-randomized study performed between 2015 and 2020 was used, and included adult patients with brain tumors, followed during treatment and through a one-year follow-up. Clinical and demographic data were sourced from the longitudinal study and linked to Swedish national registers to get information on healthcare resource use. A cost-utility framework was used to evaluate the cost-effectiveness of PBT vs. CRT. Patients in PBT group (n = 310) were matched with patients in CRT group (n = 40) on relevant observables using propensity score matching with replacement. Costs were estimated from a healthcare perspective and included costs related to inpatient and specialized outpatient care, and prescribed medications. The health outcome was quality-adjusted life-years (QALYs), derived from the EORTC-QLQ-C30. Generalized linear models (GLM) and two-part models were used to estimate differences in costs and QALYs. RESULTS: PBT yielded higher total costs, 14,639 US$, than CRT, 13,308 US$, with a difference of 1,372 US$ (95% CI, -4,914-7,659) over a 58 weeks' time horizon. Further, PBT resulted in non-significantly lower QALYs, 0.746 compared to CRT, 0.774, with a difference of -0.049 (95% CI, -0.195-0.097). The probability of PBT being cost-effective was < 30% at any willingness to pay. CONCLUSIONS: These results suggest that PBT cannot be considered a cost-effective treatment for brain tumours, compared to CRT. TRIAL REGISTRATION: Not applicable.

The Return on Investment for the Prevention and Treatment of Childhood and Adolescent Overweight and Obesity in Beijing: A Modeling Study.

Background: The increasing prevalence of child and adolescent overweight and obesity (CAOAO) in Beijing poses significant health and economic challenges. This study assesses the potential health and economic outcomes of implementing specific interventions to address CAOAO in Beijing. Methods: A deterministic Markov cohort model was used to estimate the impact of five interventions from 2025 to 2115: restrictions on unhealthy food marketing to children, mandatory front of package labeling (FOPL), family-based nutrition and exercise education, school-based nutritional health education, and nutritional counseling in primary healthcare. The model evaluated societal costs, healthcare savings, wages, and economic productivity in adulthood, calculating the return on investment (ROI) for each intervention and their combined effect. Result: Without intervention, Beijing is projected to experience a loss of 13.4 million disability-adjusted life years (DALYs) due to CAOAO. The health and economic impact of childhood obesity in Beijing is projected to be CNY 14.6 trillion (USD 2.1 trillion), or a lifetime loss of CNY 6.8 million (USD 0.96 million) per affected child, exceeding the sum of Beijing's GDP from 2021 to 2023. Restrictions on unhealthy food marketing to children and nutrition counseling in primary healthcare are projected to yield the highest returns, with benefits accruing within one year of implementation. Owing to the substantial upfront costs, including personnel, materials, and training, school-based and family-based interventions require a longer time horizon to realize significant health and economic benefits. Conclusions: Effective management of CAOAO in Beijing demands a multifaceted approach. The combination of restrictions on unhealthy food marketing to children, mandatory front of package labeling, nutrition counseling in primary healthcare, school-based intervention, and family-based intervention presents the most substantial health and economic returns. This comprehensive strategy aligns with global best practices and addresses the unique challenges faced by Beijing in combating childhood obesity.

The development of a capability wellbeing measure in economic evaluation for children and young people aged 11-15.

The capability approach provides a broad evaluative space for making funding decisions for health and care interventions, with capability wellbeing as the outcome of value. A range of capability measures have been developed for the economic evaluation of health and care interventions for adults. However, such measures have not been previously developed for children and young people for this purpose and may be valuable. This study aimed to identify important capabilities for children and young people aged 11-15, and to develop these into attributes for an economic measure that can inform funding decisions. Thirty-three qualitative in-depth interviews were undertaken with children and young people aged 11-15 (n = 19) and parents (n = 14) in urban and rural areas of England between September 2019 and November 2021. Purposeful maximum variation sampling ensured representation from different backgrounds. Children and young people were asked to think of things important to them and place these on sticky notes around a drawing/photograph of themselves; the interview asked them about these important things. Parents were asked to identify factors that enhanced and negatively impacted their child's quality of life. Analysis using constant comparison facilitated exploration of similarities and differences in important capabilities. A second phase of semi-structured interviews with children and young people (n = 15) explored how these attributes should be expressed in a meaningful way. Eight overarching capability wellbeing themes were identified, with some variation across children and young people, and parent groups: Fun and enjoyment; Learning and experiencing; Attachment; Emotional security andsupport; Achievement; Identity and choice; Physical safety; Aspiration. Potentially, this information will help to provide an alternative approach to the measurement of benefits to children and young people for economic evaluation of health and care interventions, one that will be better able to capture benefits associated with interventions to improve the social determinants of health.

Improving the Care of Severe, Open Fractures and Postoperative Infections of the Lower Extremities: Protocol for an Interdisciplinary Treatment Approach.

BACKGROUND: Patients with open fractures often experience complications during their injury. The treatments incur high costs. Interdisciplinary cooperation between different medical disciplines may improve treatment outcomes. Such cooperation has not yet been envisaged in the German health care system. OBJECTIVE: The aim of the study is to improve the treatment of fractures with open soft tissue damage or postoperative complications in terms of duration and sustainability in a region in northwest Germany. Largely standardized diagnostics and therapy are intended to optimize processes in hospitals. In addition, a reduction in the duration of treatment and treatment costs is to be achieved. METHODS: Using a digital platform, physicians from 31 hospitals present patient cases to an interdisciplinary group of experts from the fields of plastic surgery, infectiology, hygiene, and others. The group of experts from the environment of the University Hospital Münster promptly makes a joint treatment recommendation for the individual case. The plan is to examine 3300 patients with open fractures or surgical complications. As consortium partners, there are also 3 statutory health insurance companies. The extent to which the therapy recommendations are effective and contribute to cost reduction in the health care system will be empirically investigated in a stepped-wedge cluster-randomized design. In addition, medical and nonmedical professional groups involved in the project will be asked about their work in the project (in total, 248 clinic employees). The primary outcome is the complication rate of open fractures or the occurrence of postoperative complications. As secondary outcomes, the number of antibiotics administered, limb function, and quality of life will be assessed. The health economic evaluation refers to the costs of health services and absenteeism. For the work-related evaluation, workload, work engagement, work-related resources, readiness for technology, and ergonomic aspects of the new telemedical technology will be collected. In addition, clinic employees will give their assessments of the success of the project in a structured telephone interview based on scaled and open-ended questions. RESULTS: The project started in June 2022; data collection started in April 2023. As of mid-June 2024, data from 425 patients had been included. In total, 146 members of staff had taken part in the questionnaire survey and 15 had taken part in the interviews. CONCLUSIONS: Standardized treatment pathways in the standard care of patients with open fractures and postoperative infections will be established to reduce complications, improve chances of recovery, and reduce costs. Unnecessary and redundant treatment steps will be avoided through standardized diagnostics and therapy. The interdisciplinary treatment perspective allows for a more individualized therapy. In the medium term, outpatient or inpatient treatment centers specialized in the patient group could be set up where the new diagnostic and therapeutic pathways could be competently applied. TRIAL REGISTRATION: German Clinical Trials Register DRKS00031308; https://drks.de/search/de/trial/DRKS00031308. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/57820.

Health state utility values of type 2 diabetes mellitus and related complications: a systematic review and meta-regression.

BACKGROUND: This study aimed to synthesize and quantitatively examine Health State Utility Values (HSUVs) for Type 2 Diabetes Mellitus (T2DM) and its complications, providing a robust meta-regression framework for selecting appropriate HSUV estimates. METHOD: We conducted a systematic review to extract HSUVs for T2DM and its complications, encompassing various influencing factors. Relevant literature was sourced from a review spanning 2000-2020, supplemented by literature from PubMed, Embase, and the Web of Science (up to March 2024). Multivariate meta-regression was performed to evaluate the impact of measurement tools, tariffs, health status, and clinical and demographic variables on HSUVs. RESULTS: Our search yielded 118 studies, contributing 1044 HSUVs. The HSUVs for T2DM with complications varied, from 0.65 for cerebrovascular disease to 0.77 for neuropathy. The EQ-5D-3L emerged as the most frequently employed valuation method. HSUV differences across instruments were observed; 15-D had the highest (0.89), while HUI-3 had the lowest (0.70) values. Regression analysis elucidated the significant effects of instrument and tariff choice on HSUVs. Complication-related utility decrement, especially in diabetic foot, was quantified. Age <70 was linked to increased HSUVs, while longer illness duration, hypertension, overweight and obesity correlated with reduced HSUVs. CONCLUSION: Accurate HSUVs are vital for the optimization of T2DM management strategies. This study provided a comprehensive data pool for HSUVs selection, and quantified the influence of various factors on HSUVs, informing analysts and policymakers in understanding the utility variations associated with T2DM and its complications.

Should Scotland provide genome-wide sequencing for the diagnosis of rare developmental disorders? A cost-effectiveness analysis.

AIMS: This study aims to evaluate the cost effectiveness of genetic and genomic testing strategies for the diagnosis of rare developmental disorders in NHS Scotland. METHODS: Six genetic and genomic testing strategies were evaluated using a decision tree model. First-line, second-line and last-resort trio genome sequencing (GS), and second-line and last-resort trio exome sequencing (ES) were compared with standard genetic testing. The cost effectiveness of each strategy was expressed in terms of incremental cost per additional diagnosis. The impact of uncertainty on cost-effectiveness results was explored using deterministic and probabilistic sensitivity analysis. RESULTS: 2nd-line ES was a cost-saving option, increasing diagnostic yield by 13.9% and decreasing cost by £1027 per trio compared to standard genetic testing. Compared to ES, strategies involving GS increased costs significantly, with only a moderate or zero improvement in diagnostic yield. Sensitivity analysis indicated that significant reductions in cost or improvements in diagnostic yield are required before 1st-line GS becomes cost effective. CONCLUSION: 2nd-line ES (after chromosomal microarray; replacing gene panel testing) for the diagnosis of developmental disorders is a cost-saving option for the Scottish NHS. Ongoing economic evaluation is required to monitor the evolving cost and diagnostic yield of GS and ES over time.

Impact of Integrative Health and Medicine on Costs Associated with Adult Health System Beneficiaries with Musculoskeletal Conditions: A Retrospective Cohort Study.

Objective: Owing to perceived additional costs, patients may avoid integrative health and medicine (IHM) treatments, while insurers may not cover IHM. We hypothesized that adult beneficiaries of a health system's employee insurance plan with musculoskeletal (MSK) conditions receiving covered outpatient IHM would have reduced total allowed costs over the 1-year follow-up compared with matched controls, secondarily exploring medical and pharmaceutical cost subsets. Methods: We queried medical records and claims spanning 2018-2023 for beneficiaries aged 18-89 years with a new MSK episode. Patients were divided into cohorts: (1) IHM within 3 months after MSK diagnosis and (2) no IHM after initial primary care. After inflation adjustment and trimming, propensity score matching was used to balance cohorts on demographics, comorbidity, health care utilization, and prior 12-month spend. Least-squares mean total, medical, and pharmaceutical allowed costs (United States Dollar) over the 1-year follow-up were analyzed using a linear mixed model. Findings were compared with a generalized linear model without trimming. Results: There were 251 patients per matched cohort, with adequate covariate balance. There was no meaningful between-cohort difference (IHM minus No IHM) in least-squares mean total cost (+703 [95% CI: -314, 1720]). Secondary outcomes included medical cost (+878 [95% CI: 61, 1695]) and pharmaceutical cost (+6 [95% CI: -71, 83]). A generalized linear model revealed no meaningful difference in estimated mean total medical costs (-2561 [95% CI: -7346, +2224]). Conclusions: IHM use among adult health system beneficiaries with MSK conditions was not associated with meaningful differences in 1-year follow-up total health care costs compared with matched controls. Our study was underpowered for secondary outcomes, which should be interpreted with caution. Future research should include a larger sample of patients and examine longitudinal changes in patient-reported outcomes.

General Population Mortality Adjustment in Survival Extrapolation of Cancer Trials: Exploring Plausibility and Implications for Cost-Effectiveness Analyses in HER2-Positive Breast Cancer in Sweden.

BACKGROUND: In economic evaluations of novel therapies, assessing lifetime effects based on trial data often necessitates survival extrapolation, with the choice of model affecting outcomes. The aim of this study was to assess accuracy and variability between alternative approaches to survival extrapolation. METHODS: Data on HER2-positive breast cancer patients from the Swedish National Breast Cancer Register were used to fit standard parametric distribution (SPD) models and excess hazard (EH) models adjusting the survival projections based on general population mortality (GPM). Models were fitted using 6-y data for stage I and II, 4-y data for stage III, and 2-y data for stage IV cancer reflecting an early data cutoff while maintaining sufficient events for comparison of model estimates with actual long-term outcomes. We compared model projections of 15-y survival and restricted mean survival time (RMST) to 15-y registry data and explored the variability between models in extrapolations of long-term survival. RESULTS: Among 11,224 patients compared with the observed registry 15-y RMST estimates across the disease stages, EH cure models provided the most accurate estimates in patients with stage I to III cancer, whereas EH models without cure most closely matched survival in patients with stage IV cancer, in which cure assumption was less plausible. The Akaike information criterion-averaged model projections varied as follows: -8.2% to +5.3% for SPD models, -4.9% to +5.2% for the EH model without a cure assumption, and -19.3% to -0.2% for the EH model with a cure assumption. EH models significantly reduced between-model variance in the predicted RMSTs over a 50-y time horizon compared with SPD models. CONCLUSIONS: EH models may be considered as alternatives to SPD models to produce more accurate and plausible survival extrapolation that accounts for general population mortality. HIGHLIGHTS: Excess hazard (EH) methods have been suggested as an approach to incorporate background mortality rates in economic evaluation using survival extrapolation.We highlight that EH models with or without a cure assumption can produce more accurate survival projections and significantly reduce between-model variability in comparison with standard parametric distribution models across cancer stages.EH models may be a preferred modeling method to reduce model uncertainty in health economic modeling since models that would otherwise have produced implausible extrapolations are constrained by the EH framework.Reduced uncertainty in economic evaluations will enhance the application of evidence-based health care decision making.

The "Other E" in ELSI: The Use of Economic Evaluation to Inform the Expansion of Newborn Screening.

The utilization and application of genomic information generated from precision medicine continues to increase with the goal of improving health outcomes. Increasingly researchers, health care professionals, and public health teams include an examination of the ethical, legal, and social issues (ELSI) in their consideration of the use of precision medicine for newborn and pediatric health. In addition to ELSI considerations, stakeholders could benefit from an understanding of economics, the other "E" in ELSI. The use of an economic evaluation could aid decision-making on whether to screen newborns who may be at risk for disease, to diagnose newborns and children who present with symptoms, to inform the treatment and management of diagnosed individuals. In this manuscript we review the core concepts of economic evaluation, the framework of decision-analysis, and key parameters for consideration in assessing the economics of NBS program(s). We describe the common language used in the economic evaluation and provide a practical overview of health economic evaluations including 1) their purpose, 2) different types and components, 3) evaluation of the different types and components of economic evaluations (i.e., cost-effectiveness vs. cost-benefit analysis), 4) impact of societal or healthcare perspectives on the analysis, 5) health outcomes, 6) time horizon for the analysis, 7) identification of appropriate comparators, and 8) resources for economic data. We conclude with a use case to demonstrate the application and understanding of economic considerations for in the advancement and expansion of NBS.

Calculating the Expected Net Benefit of Sampling for Survival Data: A Tutorial and Case Study.

HIGHLIGHTS: The net value of reducing decision uncertainty by collecting additional data is quantified by the expected net benefit of sampling (ENBS). This tutorial presents a general-purpose algorithm for computing the ENBS for collecting survival data along with a step-by-step implementation in R.The algorithm is based on recently published methods for simulating survival data and computing expected value of sample information that do not rely on the survival data to follow any particular parametric distribution and that can take into account any arbitrary censoring process.We demonstrate in a case study based on a previous cancer technology appraisal that ENBS calculations are useful not only for designing new studies but also for optimizing reimbursement decisions for new health technologies based on immature evidence from ongoing trials.