Health Utility Values Among Patients With Diabetic Retinopathy, Wet Age-Related Macular Degeneration, and Cataract in Thailand: A Multicenter Survey Using Time Trade-Off, EQ-5D-5L, and Health Utility Index 3.

OBJECTIVES: This study aimed to establish normative health utility data in Thai patients with diabetic retinopathy, wet age-related macular degeneration, and cataract; evaluate the sensitivity of different utility instruments to visual impairment; explore the relationship among these health utility values with the vision-specific quality of life (QoL); and assess the association of baseline characteristics and visual acuity level with health utility values and vision-specific QoL. METHODS: This multicenter cross-sectional survey included 309 patients from tertiary eye centers. We used health utility instruments (time trade-off [TTO], EuroQol five-dimension [EQ-5D-5L], and Health Utility Index 3 [HUI3]) and vision-specific QoL instrument (National Eye Institute Visual Function Questionnaire) for face-to-face interviews. Demographic data and Early Treatment Diabetic Retinopathy Study visual acuity were recorded during the participants ophthalmic visits. Univariable and multivariable mixed-effect models were used to evaluate factors associated with the utility scores. Health utility scores among each type of eye disease were compared. RESULTS: The overall mean utility values from the TTO, EQ-5D-5L, and HUI3 were 0.84 ± 0.25, 0.70 ± 0.19, and 0.68 ± 0.26, respectively. The health utility scores obtained from TTO and HUI3 showed a significant response to severe visual impairment or worse. Health utility scores from HUI3 (r = 0.54; P < .01) and EQ-5D-5L (r = 0.43; P < .01) displayed a moderate correlation with the National Eye Institute Visual Function Questionnaire score. There were no significant differences in health utility value among the 3 diseases upon adjusting for the visual acuity level and demographics. CONCLUSIONS: Visual acuity level has a greater impact on a patient's QoL than the type of eye disease. HUI3 and EQ-5D-5L and TTO are suitable for measuring health utility in leading causes of blindness.

Impact of Structural Differences on the Modeled Cost-Effectiveness of Noninvasive Prenatal Testing.

BACKGROUND: Noninvasive prenatal testing (NIPT) was developed to improve the accuracy of prenatal screening to detect chromosomal abnormalities. Published economic analyses have yielded different incremental cost-effective ratios (ICERs), leading to conclusions of NIPT being dominant, cost-effective, and cost-ineffective. These analyses have used different model structures, and the extent to which these structural variations have contributed to differences in ICERs is unclear. AIM: To assess the impact of different model structures on the cost-effectiveness of NIPT for the detection of trisomy 21 (T21; Down syndrome). METHODS: A systematic review identified economic models comparing NIPT to conventional screening. The key variations in identified model structures were the number of health states and modeling approach. New models with different structures were developed in TreeAge and populated with consistent parameters to enable a comparison of the impact of selected structural variations on results. RESULTS: The review identified 34 economic models. Based on these findings, demonstration models were developed: 1) a decision tree with 3 health states, 2) a decision tree with 5 health states, 3) a microsimulation with 3 health states, and 4) a microsimulation with 5 health states. The base-case ICER from each model was 1) USD$34,474 (2023)/quality-adjusted life-year (QALY), 2) USD$14,990 (2023)/QALY, (3) USD$54,983 (2023)/QALY, and (4) NIPT was dominated. CONCLUSION: Model-structuring choices can have a large impact on the ICER and conclusions regarding cost-effectiveness, which may inadvertently affect policy decisions to support or not support funding for NIPT. The use of reference models could improve international consistency in health policy decision making for prenatal screening. HIGHLIGHTS: NIPT is a clinical area in which a variety of modeling approaches have been published, with wide variation in reported cost-effectiveness.This study shows that when broader contextual factors are held constant, varying the model structure yields results that range from NIPT being less effective and more expensive than conventional screening (i.e., NIPT was dominated) through to NIPT being more effective and more expensive than conventional screening with an ICER of USD$54,983 (2023)/QALY.Model-structuring choices may inadvertently affect policy decisions to support or not support funding of NIPT. Reference models could improve international consistency in health policy decision making for prenatal screening.

Valuing quality of life for economic evaluations in cancer: navigating multiple methods.

INTRODUCTION: Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts. AREAS COVERED: This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research. EXPERT OPINION: We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials.

Measuring perceived utility of genomic sequencing: Development and validation of the GENEtic Utility (GENE-U) scale for adult screening.

INTRODUCTION: As population-based screening programs to identify genetic conditions in adults using genomic sequencing (GS) are increasingly available, validated patient-centered outcome measures are needed to understand participants' experience. We aimed to develop and validate an instrument to assess the perceived utility of GS in the context of adult screening. METHODS: Informed by a five-domain conceptual model, we used a five-step approach to instrument development and validation: (1) item writing, (2) cognitive testing, (3) pilot testing and item reduction, (4) psychometric testing, and (5) evaluation of construct validity. Adults undergoing risk-based or population-based GS who had received GS results as part of ongoing research studies participated in structured cognitive interviews and two rounds of surveys. After item pool refinement, we conducted an exploratory factor analysis and calculated Pearson correlations with related instruments. RESULTS: We derived the 18-item Adult Diagnostic version of the GENEtic Utility (GENE-U) scale (total sum score α = .87). Mirroring the Pediatric Diagnostic version, the instrument has a two-factor structure, including an Informational Utility subscale (14 items, α =.89) and an Emotional Utility subscale (4 items, α =.75). The Informational Utility subscale was strongly associated with empowerment and personal utility of GS. Correlations of the Emotional Utility subscale with psychosocial impact and anxiety and depression were weak to moderate. CONCLUSION: Initial psychometric testing of the Adult Screening GENE-U scale demonstrates its promise, and additional validation in translational genomics research is warranted.

Treatment Preferences of Adult Patients with Attention-Deficit/Hyperactivity Disorder - A Discrete Choice Experiment.

Background: Understanding patient preferences for treatments may facilitate shared decision-making. This study assessed adult patient preferences for attention-deficit/hyperactivity disorder (ADHD) treatments in a sample of 600 patients in the United States (US). Methods: A web-based discrete choice experiment (DCE) survey was conducted among treated adults with ADHD. Participants were recruited from Dynata's US panel (06/22/2023-07/06/2023). Attributes and levels, identified based on clinical inputs and published data, included efficacy and safety. Participants' preferences were estimated using conditional logistic regression. Willingness to trade-off and attributes' relative importance were calculated. Overall preferences for treatment profiles approximating centanafadine, lisdexamfetamine, atomoxetine, and viloxazine were estimated using adjusted total utilities. Results were stratified by current treatment status. Sensitivity analyses including participants who passed validity tests were conducted. Results: Among the 600 participants (mean age 37.9 years; 66.2% female; 50.8% treated), all attributes had a statistically significant impact on preferences for ADHD treatments (p < 0.001); the most important attribute was improvement in ADHD symptoms (36%), followed by risks of nausea (25%), insomnia (20%), anxiety (8%), dry mouth (6%), and feeling jittery (5%). Together, safety attributes accounted for >60% of relative importance in decision-making. Participants were willing to forgo 0.59, 0.57, 0.49, 0.32, and 0.17 percentage points of symptom improvement to achieve one-percentage-point reduced risk of insomnia, nausea, anxiety, feeling jittery, and dry mouth, respectively. Centanafadine profile had consistently higher adjusted total utilities than its comparators. Similar results were obtained in the subgroup and sensitivity analyses. Conclusion: Efficacy was the most important attribute for patients when making treatment decision, but taken together, AEs had greater relative importance than efficacy alone. Accordingly, a profile resembling that of centanafadine would be preferred by an average patient compared to key competitors due to its favorable safety profile. These findings may help improve treatment decision-making, enhance treatment satisfaction, and foster adherence.

Emerging biomarkers for non-invasive diagnosis and treatment of cancer: a systematic review.

Cancer remains a global health challenge, necessitating continuous advancements in diagnostic and treatment strategies. This review focuses on the utility of non-invasive biomarkers in cancer diagnosis and treatment, their role in early detection, disease monitoring, and personalized therapeutic interventions. Through a systematic review of the literature, we identified 45 relevant studies that highlight the potential of these biomarkers across various cancer types, such as breast, prostate, lung, and colorectal cancers. The non-invasive biomarkers discussed include liquid biopsies, epigenetic markers, non-coding RNAs, exosomal cargo, and metabolites. Notably, liquid biopsies, particularly those based on circulating tumour DNA (ctDNA), have emerged as the most promising method for early, non-invasive cancer detection due to their ability to provide comprehensive genetic and epigenetic information from easily accessible blood samples. This review demonstrates how non-invasive biomarkers can facilitate early cancer detection, accurate subtyping, and tailored treatment strategies, thereby improving patient outcomes. It underscores the transformative potential of non-invasive biomarkers in oncology, highlighting their application for enhancing early detection, survival rates, and treatment precision in cancer care. Systematic review registration: https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42023474749 PROSPERO, identifier CRD42023474749.

Cost-effectiveness Analysis of the Oncomine™ Dx Target Test MultiCDx System Using Next-generation Sequencing and Single-gene Test in Advanced and Recurrent Nonsquamous Non-small-cell Lung Cancer.

Introduction: To determine the appropriate treatment for patients with advanced/recurrent nonsquamous non‒small-cell lung cancer (NSCLC), a companion diagnostic was conducted to detect driver mutations through genetic testing. In Japan, Oncomine Dx Target Test (DxTT) using next-generation sequencing (NGS) that can comprehensively detect gene mutations or single-gene tests are conducted as companion diagnostics. Furthermore, cost-effectiveness analysis was conducted to compare the cost-effectiveness of Oncomine DxTT using NGS with that of single-gene test in Japan. Methods: The target population included patients with advanced/recurrent nonsquamous NSCLC. A model structure was constructed for the Oncomine DxTT strategy and three single-gene tests (i.e., epidermal growth factor receptor (EGFR) mutations and anaplastic lymphoma kinase (ALK)/c-ros oncogene 1 (ROS1) rearrangements) with reference to previous studies and the Clinical Practice Guidelines of Lung Cancer 2022 in Japan. The model structure assumed that genetic testing would be conducted and first-line treatment used the drug most recommended in the 2022 Japanese Lung Cancer Clinical Practice Guidelines, depending on the driver mutation,. Model inputs were obtained from the literature and price list in Japan, and cost-utility analysis was conducted. Results: For the Oncomine DxTT strategy, the expected incremental costs and effectiveness were estimated to be approximately JPY 172,361 (JPY 12,285,228 vs. JPY 12,112,867 for strategies A and B, respectively) and -0.51 quality-adjusted life-year (QALY) per patient (21.93 QALY vs. 22.44 QALY for strategies A and B). As a result, the costs increased but the effectiveness decreased. Therefore, the Oncomine DxTT strategy was dominated by the three single-gene tests. Sensitivity and scenario analyses revealed that the test success rate of Oncomine DxTT affected the results. Conclusions: The genetic test using Oncomine DxTT before the first-line treatment is not cost-effective compared with the three single-gene tests (EGFR/ALK/ROS1) for patients with advanced/recurrent nonsquamous NSCLC.

Long-term quality of life and influencing factors in elderly patients after vertebroplasty based on the EQ-5D-3L Chinese utility scoring system: a retrospective study.

BACKGROUND: The quality of life of elderly patients after vertebroplasty is influenced by various factors. Although the EuroQol 5-Dimension 3-Level (EQ-5D-3L) scale has been widely used to assess quality of life, the factors affecting the long-term postoperative quality of life of elderly vertebroplasty patients in China have not been thoroughly studied. METHODS: This retrospective study included 519 patients aged 65 years and older who underwent elective vertebroplasty. We collected baseline data from these patients and conducted telephone follow-ups 12 months postoperation to evaluate their EQ-5D-3L health utility scores and EuroQol Visual Analogue Scale (EQ-VAS) scores. Univariate and multivariate linear regression models were used to analyse the factors affecting quality of life. RESULTS: Of the 519 patients, the majority were female (78.0%), aged 65 to 95 years, with an average age of 75.2 years. Twelve months postoperation, pain/discomfort was the most commonly reported issue for 68.4% of patients. The median EQ-5D-3L health utility score was 0.783, with a range between 0.450 and 0.887; the median EQ-VAS score was 75, ranging from 60 to 85. Multivariate linear regression analysis indicated that older age, hormone use, higher American Society of Anesthesiologists (ASA) grades, nondrinking habits, and low albumin levels were found to be independent risk factors affecting long-term quality of life in elderly patients after vertebroplasty. Additionally, a history of tumours, the number of vertebral compression fractures, and bone mineral density were also crucial influencing factors. CONCLUSIONS: Based on the use of the EQ-5D-3L Chinese utility scoring system, we evaluated the quality of life of patients aged 65 and above 12 months after vertebroplasty. This study identified several factors related to postoperative quality of life in elderly vertebroplasty patients, providing crucial evidence for further clinical decisions and patient education.

An Economic Evaluation of Family-Based Versus Traditional Helicobacter pylori Screen-and-Treat Strategy: Based on Real-World Data and Microsimulation Model.

OBJECTIVE: There is an economic evaluation on the family-based Helicobacter pylori screen-and-treat strategy (FBHS) in China. This study aimed to compare the cost-effectiveness of the FBHS with the traditional H. pylori screen-and-treat strategy (TBHS). MATERIALS AND METHODS: A seven-state microsimulation model, including H. pylori infection and gastric cancer states, was constructed on the basis of the target family samples from 29 provinces in China. Taking a lifetime horizon from a healthcare system perspective, the long-term costs and health outcomes of the FBHS and TBHS screening strategies were simulated separately, and economic evaluations were performed. The model parameters were primarily derived from real-world data, published literature, and expert opinions. The primary outcome was the incremental cost-effectiveness ratio (ICER) expressed as cost/quality-adjusted life-year (QALY) gained. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analysis were performed to assess the uncertainty of the results. RESULTS: The base-case analysis revealed that the average costs for FBHS and TBHS were 563.67 CNY and 574.08 CNY, respectively, with corresponding average QALYs of 14.83 and 14.79. The ICER for the comparison between the two strategies was -214.07, indicating that FBHS was an absolutely dominant strategy with better cost-effectiveness. The results of both one-way sensitivity analysis and probabilistic sensitivity analysis were robust. When taking into account the added benefit of the higher H. pylori eradication rate in FBHS, the average costs were further reduced, and the average QALYs were increased, solidifying its position as an unequivocally dominant strategy. CONCLUSION: The FBHS is an absolutely dominant and cost-effective strategy that enables an optimized allocation of screening resources. Decision-makers should prioritize FBHS when developing H. pylori prevention and control strategies.

[Evaluation of the Viral Hemorrhagic Fever Surveillance System in the Assaba region, Mauritania (2020-2022)]. / Évaluation du système de surveillance des fièvres hémorragiques virales dans la wilaya d'Assaba, Mauritanie (2020-2022).

Introduction: Several arboviral diseases have been known to be endemic (e.g., Crimean-Congo hemorrhagic fever, Rift Valley fever) or are emerging (dengue fever, chikungunya, O'nyong-nyong) in human populations in Mauritania, while others have become rare in recent years (e.g. yellow fever). Moreover, domestic animals, especially cattle, camels, goats, and sheep, are also known to be infected with some of these arboviruses (e.g. Crimean-Congo hemorrhagic fever, Rift Valley fever). For these reasons, viral hemorrhagic fever surveillance in Mauritania is part of the Integrated Disease Surveillance and Response (IDSR). However, limited information is available on the efficacy of the viral hemorrhagic fever surveillance system in the Assaba region of Mauritania. The aim of the present study was to assess the performance of the surveillance system, in particular its general utility, simplicity, flexibility, acceptability, and reactivity. Methods: A descriptive cross-sectional study was conducted from July to August 2022 in the Assaba region with the objective of evaluating the characteristics of the system by interviewing key actors involved in the surveillance of viral hemorrhagic fevers, with a focus on Rift Valley fever and Crimean-Congo hemorrhagic fever, using questionnaires developed following the guidelines of the Centers for Disease Control and Prevention (Atlanta, Georgia, USA). Data from 2020-2022 on viral hemorrhagic fevers from the National Institute of Public Health laboratory were analyzed. Medians, interquartile ranges, and proportions were calculated using Epi Info® 7.2.5.0 and Excel® 2021. Results: The questionnaire was answered by all twenty-six persons involved in the viral hemorrhagic fever surveillance system in Assaba region. The majority of survey respondents found the system to be useful (51%), simple (63%), acceptable (46%), responsive (64%), and flexible (46%). An analysis of the data revealed a positive predictive value of 28% for Rift Valley Fever. The weekly distribution of cases within the wilaya indicates that the moughataa of Kiffa recorded the highest number of cases in September, with a notable weekly peak during that month in 2020. According to the analysis of the National Institute of Public Health database, cases of viral hemorrhagic fevers were promptly handled. Survey responses and database analysis revealed issues related to data quality and data management mechanisms. These limitations in the surveillance system are likely to be due to insufficient resources and training of the personnel, in particular with regards to data collection and management, which in turn led to incomplete or missing data and invalid data entry. These weak points can be ascribed, at least in part, to financial constraints and inadequate attribution of priority to arboviral diseases. Despite these limitations, disease data generated by the surveillance system were generally reliable. Conclusion: The viral hemorrhagic fever surveillance system in the Assaba region adheres to the organization and functioning of the national viral hemorrhagic fever surveillance system, which is part of the IDSR. The characteristics of utility, simplicity, responsiveness, and flexibility of the viral hemorrhagic fever surveillance system are good, but acceptability and flexibility need further improvement. The earlier the first arboviral human or animal cases are detected, the more likely an active intervention can be organized in response to the emerging epidemic or epizootic and prevent the spread of the disease. An efficient viral surveillance system is the key to reducing the negative impact of arboviral diseases in Assaba region.

Health-related quality of life and its association with disease activity/functional status in rheumatoid arthritis: A cross-sectional study from South India.

BACKGROUND: Rheumatoid arthritis (RA) is an autoimmune disease with chronic pain that gradually becomes incapacitating and negatively influences the health-related quality of life (HRQoL). This study estimates HRQoL in RA using the EuroQol five dimensions (EQ-5D) tool and its association with functional status and disease activity. METHODS: RA patients (n = 320) aged above 18 years, visiting outpatient clinic at a tertiary care multispecialty hospital in south India were the study participants. Sociodemographic, clinical, and laboratory data were collected from them. EQ-5D-5L questionnaire and the EQ Global Health Visual Analogue Scale (EQ-VAS) were used to measure HRQoL. Disease activity was measured using Disease Activity Score-28 (DAS-28), and the Health Assessment Questionnaire (HAQ) was used to assess functional status. Pearson's correlation and multiple linear regression were used to measure association, and statistical significance was considered at p < 0.05. RESULTS: The EQ-5D utility score was 0.54 ± 0.36, pain and anxiety were the most affected domains, and the mean EQ-VAS was 63.05 ± 18.54%. A moderate to high disease activity was present in 85% (DAS-28 > 3.2), and a severe functional disability in 32.8% (HAQ > 1.5) of study participants. The mean EQ-5D scores for RA patients were 0.78 (0.65-0.90) for no disease activity, 0.73 (0.65-0.80) for mild, 0.53 (0.32-0.74) for moderate and 0.47 (0.32-0.62) for high disease activity. In multiple linear regression analysis, HAQ and age independently predicted EQ-5D. CONCLUSION: RA significantly impacts HRQoL, and interventions focussing on pain and anxiety management are essential. The study's EQ-5D values could help estimate Quality Adjusted Life Years (QALY) while conducting economic evaluation studies in RA within an Indian context.

Cost-Utility Analysis of Antipsychotic Reduction and Discontinuation in Patients with Long-term Schizophrenia and Psychosis in English Mental Health Trusts: The RADAR Study.

OBJECTIVES: The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared to maintenance treatment over 24 months from a mental health services, health and social care, and societal perspectives. METHODS: Nineteen mental health trusts recruited patients to the RADAR randomised controlled trial. Quality adjusted life years (QALYs) were calculated from patient-reported EQ-5D-5L, with years of full capability (YFCs) calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources. RESULTS: 253 participants were randomised: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in QALYs (-0.035; 95% CI: -0.123 to 0.052), whereas YFCs were significantly lower in the reduction arm compared to the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective. CONCLUSIONS: It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over two-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.

Direct and Indirect Mapping of AQoL-6D onto EQ-5D-5L Utilities Using Data from a Multi-centre, Cross-Sectional Study of Malaysians with Chronic Heart Failure.

OBJECTIVE: The AQoL-6D, a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population. METHODS: Cross-sectional data from a multi-centre cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using five sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalised linear model, two-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models' predictive performance was assessed through ten-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified. RESULTS: Among the models evaluated, TPM, which included age, sex, and five AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy. CONCLUSION: This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data is unavailable.

Developing a risk score to inform the use of rhBMP-2 in adult spinal deformity surgery.

BACKGROUND: Recombinant human bone morphogenetic protein-2 (rhBMP-2) has not shown superior benefit overall in cost-effectiveness during adult spinal deformity (ASD) surgery. STUDY DESIGN/SETTING: Retrospective PURPOSE: Generate a risk score for pseudarthrosis to inform the utilization of rhBMP-2, balancing costs against quality of life and complications. METHODS: ASD patients with 3-year data were included. Quality of life gained was calculated from ODI to SF-6D and translated to quality-adjusted life years (QALYs). Cost was calculated using the PearlDiver database and CMS definitions for complications and comorbidities. Established weights were generated for predictive variables via logistic regression to yield a predictive risk score for pseudarthrosis that accounted for frailty, diabetes, depression, ASA grade, thoracolumbar kyphosis and three-column osteotomy use. Risk score categories, established via conditional inference tree (CIT)-derived thresholds were tested for cost-utility of rhBMP-2 usage, controlling for age, prior fusion, and baseline deformity and disability. RESULTS: 64% of ASD patients received rhBMP-2 (308/481). There were 17 (3.5%) patients that developed pseudarthrosis. rhBMP-2 use overall did not lower pseudarthrosis rates (OR: 0.5, [0.2-1.3]). Pseudarthrosis rates for each risk category were: No Risk (NoR) 0%; Low-Risk (LowR) 1.6%; Moderate Risk (ModR) 9.3%; High-Risk (HighR) 24.3%. Patients receiving rhBMP-2 had similar QALYs overall to those that did not (0.163 vs. 0.171, p = .65). rhBMP-2 usage had worse cost-utility in the LowR cohort (p < .001). In ModR patients, rhBMP-2 usage had equivocal cost-utility ($53,398 vs. $61,581, p = .232). In the HighR cohort, the cost-utility was reduced via rhBMP-2 usage ($98,328 vs. $211,091, p < .001). CONCLUSION: Our study shows rhBMP-2 demonstrates effective cost-utility for individuals at high risk for developing pseudarthrosis. The generated score can aid spine surgeons in the assessment of risk and enhance justification for the strategic use of rhBMP-2 in the appropriate clinical contexts. LEVEL OF EVIDENCE: III.

LPDi GAN: A License Plate De-Identification Method to Preserve Strong Data Utility.

License plate (LP) information is an important part of personal privacy, which is protected by law. However, in some publicly available transportation datasets, the LP areas in the images have not been processed. Other datasets have applied simple de-identification operations such as blurring and masking. Such crude operations will lead to a reduction in data utility. In this paper, we propose a method of LP de-identification based on a generative adversarial network (LPDi GAN) to transform an original image to a synthetic one with a generated LP. To maintain the original LP attributes, the background features are extracted from the background to generate LPs that are similar to the originals. The LP template and LP style are also fed into the network to obtain synthetic LPs with controllable characters and higher quality. The results show that LPDi GAN can perceive changes in environmental conditions and LP tilt angles, and control the LP characters through the LP templates. The perceptual similarity metric, Learned Perceptual Image Patch Similarity (LPIPS), reaches 0.25 while ensuring the effect of character recognition on de-identified images, demonstrating that LPDi GAN can achieve outstanding de-identification while preserving strong data utility.

The cost-utility analysis of adding ocrelizumab to the treatment plan for primary progressive multiple sclerosis in Iran.

BACKGROUND: This study evaluates the cost-effectiveness of adding ocrelizumab to supportive care for primary progressive multiple sclerosis (PPMS) in Iran. RESEARCH DESIGN AND METHODS: Using a lifetime horizon from the payer's perspective, we developed a decision analytic model with Expanded Disability Status Scales (EDSS) as Markov health states while taking transition probabilities and treatment effects into account. Data were sourced from clinical trials and other literature. The target population was PPMS patients receiving either supportive care or ocrelizumab. We assessed cost- effectiveness through total costs, quality-adjusted life-years (QALYs), and the incremental cost- effectiveness ratio (ICER). Sensitivity analyses addressed uncertainties. RESULTS: The addition of ocrelizumab to supportive care provided an incremental gain of 0.89 QALYs and an additional cost of US$76,771.34, resulting in an ICER of US$86,220.35 compared to supportive care, which is 5.2 times Iran's GDP per capita (US$16,557). Thus, ocrelizumab is not cost-effective at the threshold of one time GDP per capita. However, the probability of cost-effectiveness increases at higher thresholds. Sensitivity analyses confirmed the robustness of the results. CONCLUSION: While ocrelizumab is not cost-effective at the threshold of one-time GDP per capita, its clinical benefits are significant. Formulating healthcare policies for high-cost medications with low alternatives like ocrelizumab is essential.

Enhancing Patient Care: Evaluating Clinical Utility in Measurement Instruments.

The evaluation of clinical utility is essential for the successful adoption of new technology in clinical practice. An approach to evaluating clinical utility is presented here using the example of digitized measurement instruments.

Establishing a Consensus-Based Framework for the Use of Wearable Activity Trackers in Health Care: Delphi Study.

BACKGROUND: Physical activity (PA) plays a crucial role in health care, providing benefits in the prevention and management of many noncommunicable diseases. Wearable activity trackers (WATs) provide an opportunity to monitor and promote PA in various health care settings. OBJECTIVE: This study aimed to develop a consensus-based framework for the optimal use of WATs in health care. METHODS: A 4-round Delphi survey was conducted, involving a panel (n=58) of health care professionals, health service managers, and researchers. Round 1 used open-response questions to identify overarching themes. Rounds 2 and 3 used 9-point Likert scales to refine participants' opinions and establish consensus on key factors related to WAT use in health care, including metrics, device characteristics, clinical populations and settings, and software considerations. Round 3 also explored barriers and mitigating strategies to WAT use in clinical settings. Insights from Rounds 1-3 informed a draft checklist designed to guide a systematic approach to WAT adoption in health care. In Round 4, participants evaluated the draft checklist's clarity, utility, and appropriateness. RESULTS: Participation rates for rounds 1 to 4 were 76% (n=44), 74% (n=43), 74% (n=43), and 66% (n=38), respectively. The study found a strong interest in using WATs across diverse clinical populations and settings. Key metrics (step count, minutes of PA, and sedentary time), device characteristics (eg, easy to charge, comfortable, waterproof, simple data access, and easy to navigate and interpret data), and software characteristics (eg, remote and wireless data access, access to multiple patients' data) were identified. Various barriers to WAT adoption were highlighted, including device-related, patient-related, clinician-related, and system-level issues. The findings culminated in a 12-item draft checklist for using WATs in health care, with all 12 items endorsed for their utility, clarity, and appropriateness in Round 4. CONCLUSIONS: This study underscores the potential of WATs in enhancing patient care across a broad spectrum of health care settings. While the benefits of WATs are evident, successful integration requires addressing several challenges, from technological developments to patient education and clinician training. Collaboration between WAT manufacturers, researchers, and health care professionals will be pivotal for implementing WATs in the health care sector.

Discriminatory Properties of Quality-Adjusted Life Year Based Cost-Effectiveness Analyses for Patients With Disabilities: A Duchenne Muscular Dystrophy Case Study.

OBJECTIVES: Quality-adjusted life years (QALYs) have been challenged as a measure of benefit for people with disabilities, particularly for those in low-utility health states or with irreversible disability. This study examined the impact of a QALY-based assessment on the price for a hypothetical treatment for Duchenne muscular dystrophy (DMD), a progressive, genetic neuromuscular disease. METHODS: A previously published, 5-state model, which analyzed treatments for early ambulatory (EA) DMD patients, was replicated, validated, and adapted to include early nonambulatory (ENA) DMD patients. The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively). All inputs were replicated including willingness-to-pay thresholds of $50 000 to $200 000/QALY. RESULTS: In contrast to EA patients, ENA patients had a 98% modeled decline in QALY-based threshold price at a willingness-to-pay of $150 000/QALY or higher, despite equal treatment benefit (delayed progression/death). At $100 000/QALY or lower, net nontreatment costs exceeded health benefits, implying any treatment for ENA patients would not be considered cost-effective, even at $0 price, including an indefinite pause in disease progression. CONCLUSIONS: For certain severe, disabling conditions, traditional approaches are likely to conclude that treatments are not cost-effective at any price once a patient progresses to a disabled health state with low utility value. These findings elucidate theoretical/ethical concerns regarding potential discriminatory properties of traditional QALY assessments for people with disabilities, particularly those who have lost ambulation or have other physical limitations.