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This study aimed to assess the cost-utility of romiplostim (ROMI) compared to eltrombopag (EPAG) as a second-line treatment for chronic primary immune thrombocytopenia (cITP) in Chinese adults. A decision tree-embedded Markov model with a lifetime horizon was used to estimate the quality-adjusted life years (QALYs) and costs for ROMI versus EPAG from the perspective of the Chinese health care system. The model was driven by platelet response with a 4-week cycle. Both QALYs and costs were discounted 5% per year. Clinical data comparing ROMI and EPAG were obtained by matching-adjusted indirect comparison (MAIC), utilizing individual patient data on ROMI and published Chinese Phase III trial data on EPAG. Costs were reported in 2022 US dollars and included drug acquisition costs, monitoring costs, bleeding-related costs, and costs associated with adverse events. Deterministic and probabilistic sensitivity analyses were performed. The CEA model indicated that treatment with ROMI resulted in an average of $4,344.4 higher costs for 0.004 QALYs. One-way sensitivity analysis (OSA) indicated that the model was most sensitive to the high bleeding rate in response (Markov stage) for EPAG and ROMI. Probabilistic sensitivity analysis (PSA) indicated that ROMI was likely to be cost effective in 0.16% cases at a willingness-to-pay threshold of $12039.1 (China per capita GDP in 2022) per QALY. If the price of ROMI is either lower than or equal to that of EPAG, ROMI could likely be considered cost-effective as a second-line treatment for Chinese adults with cITP.

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Background: The 13-MD is a new generic instrument developed to measure general health-related quality of life (GHRQoL). This instrument considers all aspects of health (i.e., physical, mental, and social) in a balanced way. A previous study led to minor changes in the original version of the 13-MD. The objective of this study was to confirm the validity of the modified 13-MD. Methods: Validity was assessed with recent data from the general population of Quebec, Canada. The meta-dimensions and items composing the 13-MD were also subjected to a ranking procedure, which allowed to determine the most important aspects for respondents. Results: A total of 1337 French-speaking participants were recruited with 1099 completing the 13-MD for validation purposes and 1084 completing the ranking procedure. The 13-MD showed very satisfactory results and confirmed to be a valid instrument. The ranking of the meta-dimensions revealed that "Well-being" received the most points, followed by "Sleep and energy" and "Body functioning." Conclusion: These results will be very useful in the continuous improvement of the 13-MD, ultimately leading to the valuation stage (i.e., development of a value set).

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BACKGROUND: In Jordan, no national value set is available for any preference-accompanied health utility measure. OBJECTIVE: This study aims to develop a value set for EQ-5D-3L based on the preferences of the Jordanian general population. METHODS: A representative sample of the Jordanian general population was obtained through quota sampling involving age, gender, and region. Participants aged above 18 years were interviewed via videoconferencing using the EuroQol Valuation Technology 2.1 protocol. Participants completed ten composite time trade-offs (cTTO) and ten discrete choice experiments (DCE) tasks. cTTO and DCE data were analyzed using linear and logistic regression models, respectively, and hybrid models were applied to the combined DCE and cTTO data. RESULTS: A total of 301 participants with complete data were included in the analysis. The sample was representative of the general population regarding region, age, and gender. All model types applied, that is, random intercept model, random intercept Tobit, linear model with correction for heteroskedasticity, Tobit with correction for heteroskedasticity, and all hybrid models, were statistically significant. They showed logical consistency in terms of higher utility decrements with more severe levels. The hybrid model corrected for heteroskedasticity was selected to construct the Jordanian EQ-5D-3L value set as it showed the best fit and lowest mean absolute error. The predicted value for the most severe health state (33333) was - 0.563. Utility decrements due to mobility had the largest weight, followed by anxiety/depression, while usual activities had the smallest weight. CONCLUSION: This study provides the first EQ-5D-3L value set in the Middle East. The Jordanian EQ-5D-3L value set can now be used in health technology assessments for health policy planning by the Jordanian health sector's decision-makers.

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OBJECTIVE: To examine the test-retest reliability of the Functional Assessment of Cancer Therapy - 8 Dimension (FACT-8D) for the first time, and to conduct a head-to-head comparison of the distribution properties and validity between the FACT-8D and EQ-5D-5L in Colorectal Cancer (CRC) Patients. METHODS: We conducted a longitudinal study on Chinese CRC patients, employing Functional Assessment of Cancer Therapy-General (FACT-G) and EQ-5D-5L at baseline, and FACT-G during follow-up (2-7 days from baseline). Utility scores for FACT-8D were derived from all available value sets (Australia, Canada and USA), while EQ-5D-5L scores were obtained from corresponding value sets for various countries. We assessed convergent validity using pairwise polychoric correlations between the FACT-8D and EQ-5D-5L; known-groups validity by discriminating participants' clinical characteristics, and effect size (ES) was tested; test-retest reliability for FACT-8D using kappa and weighted Kappa for choice consistency, and intraclass correlation coefficient (ICC) and Bland-Altman method for utility consistency. RESULTS: Among the 287 patients with CRC at baseline, 131 were included in the retest analysis. The utility scores of FACT-8D were highly positively correlated with EQ-5D-5L across various country value sets (r = 0.65-0.77), and most of the dimensions of FACT-8D and EQ-5D-5L were positively correlated. EQ-5D-5L failed to discriminate known-groups in cancer stage across all value sets, whereas both were significant in FACT-8D (ES = 0.35-0.48, ES = 0.38-0.52). FACT-8D showed good test-retest reliability (Cohen's weighted Kappa = 0.494-0.722, ICC = 0.748-0.786). CONCLUSION: The FACT-8D can be used as a valid and reliable instrument for clinical evaluation of patients with CRC, outperforming EQ-5D-5L in differentiating clinical subgroups and showing promise for cancer practice and research.

Recently, the Multi-Attribute Utility in Cancer Consortium developed the Functional Assessment of Cancer Therapy − 8 Dimension (FACT-8D), a new cancer-specific multi-attribute utility instrument based on the Functional Assessment of Cancer Therapy - General (FACT-G). This addresses the FACT-G's limitation in directly generating utility values, which has broad application prospects in cost-utility analysis within the field of oncology. To our knowledge, this is the first study to examine the test-retest reliability of FACT-8D and to conduct a head-to-head comparison of its distribution properties and validity against the EQ-5D-5L in colorectal cancer (CRC) patients. The results indicate that FACT-8D is a valid and reliable instrument for clinical evaluation of CRC patients, demonstrating superior performance in differentiating between known clinical groups compared to the generic MAUI EQ-5D-5L, and is a promising instrument for use in cancer practice and research.

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BACKGROUND: Telementoring studies found technical challenges in achieving accurate and stable annotations during live surgery using commercially available telestration software intraoperatively. To address the gap, a wireless handheld telestration device was developed to facilitate dynamic user interaction with live video streams. OBJECTIVE: This study aims to find the perceived usability, ergonomics, and educational value of a first-generation handheld wireless telestration platform. METHODS: A prototype was developed with four core hand-held functions: (1) free-hand annotation, (2) cursor navigation, (3) overlay and manipulation (rotation) of ghost (avatar) instrumentation, and (4) hand-held video feed navigation on a remote monitor. This device uses a proprietary augmented reality platform. Surgeons and trainees were invited to test the core functions of the platform by performing standardized tasks. Usability and ergonomics were evaluated with a validated system usability scale and a 5-point Likert scale survey, which also evaluated the perceived educational value of the device. RESULTS: In total, 10 people (9 surgeons and 1 senior resident; 5 male and 5 female) participated. Participants strongly agreed or agreed (SA/A) that it was easy to perform annotations (SA/A 9, 90% and neutral 0, 0%), video feed navigation (SA/A 8, 80% and neutral 1, 10%), and manipulation of ghost (avatar) instruments on the monitor (SA/A 6, 60% and neutral 3, 30%). Regarding ergonomics, 40% (4) of participants agreed or strongly agreed (neutral 4, 40%) that the device was physically comfortable to use and hold. These results are consistent with open-ended comments on the device's size and weight. The average system usability scale was 70 (SD 12.5; median 75, IQR 63-84) indicating an above average usability score. Participants responded favorably to the device's perceived educational value, particularly for postoperative coaching (agree 6, 60%, strongly agree 4, 40%). CONCLUSIONS: This study presents the preliminary usability results of a novel first-generation telestration tool customized for use in surgical coaching. Favorable usability and perceived educational value were reported. Future iterations of the device should focus on incorporating user feedback and additional studies should be conducted to evaluate its effectiveness for improving surgical education. Ultimately, such tools can be incorporated into pedagogical models of surgical coaching to optimize feedback and training.

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Background: The presence of dental caries impacts on children's daily lives, particularly among those living in deprived areas. There are successful interventions across the United Kingdom for young children based on toothbrushing with fluoride toothpaste. However, evidence is lacking for oral health improvement programmes in secondary-school pupils to reduce dental caries and its sequelae. Objectives: To determine the clinical and cost effectiveness of a behaviour change intervention promoting toothbrushing for preventing dental caries in secondary-school pupils. Design: A multicentre, school-based, assessor-blinded, two-arm cluster randomised controlled trial with an internal pilot and embedded health economic and process evaluations. Setting: Secondary schools in Scotland, England and Wales with above-average proportion of pupils eligible for free school meals. Randomisation occurred within schools (year-group level), using block randomisation stratified by school. Participants: Pupils aged 11-13 years at recruitment, who have their own mobile telephone. Interventions: Two-component intervention based on behaviour change theory: (1) 50-minute lesson delivered by teachers, and (2) twice-daily text messages to pupils' mobile phones about toothbrushing, compared with routine education. Main outcome measures: Primary outcome: presence of at least one treated or untreated carious lesion using DICDAS4-6MFT (Decayed, Missing and Filled Teeth) in any permanent tooth, measured at pupil level at 2.5 years. Secondary outcomes included: number of DICDAS4-6MFT; presence and number of DICDAS1-6MFT; plaque; bleeding; twice-daily toothbrushing; health-related quality of life (Child Health Utility 9D); and oral health-related quality of life (Caries Impacts and Experiences Questionnaire for Children). Results: Four thousand six hundred and eighty pupils (intervention, n = 2262; control, n = 2418) from 42 schools were randomised. The primary analysis on 2383 pupils (50.9%; intervention 1153, 51.0%; control 1230, 50.9%) with valid data at baseline and 2.5 years found 44.6% in the intervention group and 43.0% in control had obvious decay experience in at least one permanent tooth. There was no evidence of a difference (odds ratio 1.04, 95% confidence interval 0.85 to 1.26, p = 0.72) and no statistically significant differences in secondary outcomes except for twice-daily toothbrushing at 6 months (odds ratio 1.30, 95% confidence interval 1.03 to 1.63, p = 0.03) and gingival bleeding score (borderline) at 2.5 years (geometric mean difference 0.92, 95% confidence interval 0.85 to 1.00, p = 0.05). The intervention had higher incremental mean costs (£1.02, 95% confidence interval -1.29 to 3.23) and lower incremental mean quality-adjusted life-years (-0.003, 95% confidence interval -0.009 to 0.002). The probability of the intervention being cost-effective was 7% at 2.5 years. However, in two subgroups, pilot trial schools and schools with higher proportions of pupils eligible for free school meals, there was an 84% and 60% chance of cost effectiveness, respectively, although their incremental costs and quality-adjusted life-years remained small and not statistically significant. The process evaluation revealed that the intervention was generally acceptable, although the implementation of text messages proved challenging. The COVID-19 pandemic hampered data collection. High rates of missing economic data mean findings should be interpreted with caution. Conclusions: Engagement with the intervention and evidence of 6-month change in toothbrushing behaviour was positive but did not translate into a reduction of caries. Future work should include work with secondary-school pupils to develop an understanding of the determinants of oral health behaviours, including toothbrushing and sugar consumption, particularly according to free school meal eligibility. Trial registration: This trial is registered as ISRCTN12139369. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 15/166/08) and is published in full in Health Technology Assessment; Vol. 28, No. 52. See the NIHR Funding and Awards website for further award information.

Tooth decay has an impact on children and young people's daily lives, particularly those living in deprived areas. For young children, programmes to improve toothbrushing with fluoride toothpaste help prevent tooth decay. The Brushing RemInder 4 Good oral HealTh trial (BRIGHT) investigated whether a secondary-school-based toothbrushing programme would work. We developed a new programme which included a lesson and twice-daily text messages sent to pupils' phones. In total, 4680 pupils, aged 11­13 years, from 42 secondary schools in the United Kingdom took part in the trial. At each school, one year group was randomly selected to receive the programme, while the other year group did not receive it. All pupils were followed up for 2.5 years to see whether there were any differences in levels of tooth decay, frequency of toothbrushing, plaque or quality of life. We also considered the programme's value for money and the views of pupils and school staff. We followed up 2383 pupils and found no difference in tooth decay, plaque or quality of life. We found those who had the programme were more likely to brush their teeth twice daily after 6 months than those who did not. The programme was not good value for money overall. However, the programme appeared to be of more benefit at preventing tooth decay in pupils eligible for free school meals compared to those not eligible. In the schools with more pupils eligible for free school meals, the chance of the programme representing good value for money increased. The programme was generally liked by the pupils and school staff. Some pupils found the text messages useful, although others said they were annoying. The programme helped pupils brush their teeth more frequently in the short term, but this did not lead to less tooth decay. Further research is needed to understand how to prevent tooth decay in secondary-school pupils.

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BACKGROUND: Vision impairment and blindness are significant global public health challenges, particularly in low- and middle-income countries, where access to eye care services remains limited. India has significantly reduced the prevalence of Blindness and Vision Impairment (VI) over the last two decades. This was achieved with the help of greater investments towards blindness control programs. The use of utility values helps in conducting economic evaluations of various eye health programs and empirically justify investing in these programs. This study aimed to estimate utility values for various childhood eye conditions in central India using the EuroQol-Five-Dimension-Youth (EQ-5D-Y) instrument. METHODS: This is a before and after study with data collected at two time points for few participants and at only one time point for others. This study was undertaken at Shri Sadguru Netra Chikitsalaya (SNC) and included children representing central and north India. Participants were randomly sampled in the hospital. After comprehensive eye examination, participants completed the EuroQol-Five-Dimension-Youth (EQ-5D-Y) questionnaire along with EuroQol Visual Analogue Scale (EQ VAS) measurement to elicit their health state for their condition which was repeated after six months post-intervention to measure the change in utility value. We have used Indonesian value set to analyze the preference scores of each dimension of EQ-5D-Y. RESULTS: Utility values of 16 eye conditions were estimated at baseline and seven conditions were followed up for post-intervention utility value estimation. There is a statistically significant improvement in the utility values post-intervention amongst six conditions. Blindness and Pediatric cataract had the greatest change (0.23 and 0.2 respectively) in utility value whereas mild Vision Impairment (VI) showed the least change (0.02) in the utility value post-intervention. Blindness had the lowest baseline (0.62) and post-intervention (0.85) utility value. CONCLUSION: The utility values estimated in this study showed that generic measures such as EQ-5D-Y may be used to elicit health states for various eye conditions amongst children. These estimates are helpful in undertaking cost-utility analyses of eye health programs and interventions aimed at these eye conditions.

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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.

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Background: We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis. Methods: We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence. Results: The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal cancer but may be cost-effective for the remaining comparisons. Limitations: We were unable to obtain individual participant data as planned. The limited number of randomised controlled trials for each comparison and the paucity of data on health-related quality of life mean that the recommendations may change as new evidence (from trials with a low risk of bias) emerges. Conclusions: In people with peritoneal metastases from colorectal cancer with limited peritoneal metastases and who are likely to withstand major surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should not be used in routine clinical practice (strong recommendation). There is considerable uncertainty as to whether hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy or cytoreductive surgery + systemic chemotherapy should be offered to patients with gastric cancer and peritoneal metastases (no recommendation). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered routinely to women with stage III or greater epithelial ovarian cancer and metastases confined to the abdomen requiring and likely to withstand interval cytoreductive surgery after chemotherapy (strong recommendation). Future work: More randomised controlled trials are necessary. Study registration: This study is registered as PROSPERO CRD42019130504. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.

Cancers of the bowel, ovary or stomach can spread to the lining of the abdomen ('peritoneal metastases'). Chemotherapy (the use of drugs that aim to kill cancer cells) given by injection or tablets ('systemic chemotherapy') is one of the main treatment options. There is uncertainty about whether adding cytoreductive surgery (cytoreductive surgery; an operation to remove the cancer) and 'hyperthermic intraoperative peritoneal chemotherapy' (warm chemotherapy delivered into the lining of the abdomen during cytoreductive surgery) are beneficial. We reviewed all the information from medical literature published until 14 April 2022, to answer the above uncertainty. We found the following from eight trials, including about 1000 participants. In people with peritoneal metastases from bowel cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably does not provide any benefits and increases harm compared to cytoreductive surgery + systemic chemotherapy, while cytoreductive surgery + systemic chemotherapy appears to increase survival compared to systemic chemotherapy alone. There is uncertainty about the best treatment for people with peritoneal metastases from stomach cancer. In women with peritoneal metastases from ovarian cancer who require systemic chemotherapy before cytoreductive surgery to shrink the cancer to allow surgery ('advanced ovarian cancer'), hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably increases survival compared to cytoreductive surgery + systemic chemotherapy. In people who can withstand a major operation and in whom cancer can be removed, cytoreductive surgery + systemic chemotherapy should be offered to people with peritoneal metastases from bowel cancer, while hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered to women with peritoneal metastases from 'advanced ovarian cancer'. Uncertainty in treatment continues for gastric cancer. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.

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OBJECTIVE: In the Brazilian public national healthcare system, botulinum toxin type A has traditionally been the sole treatment option for patients with dystonia. However, as of October 2022, Deep Brain Stimulation (DBS) garnered positive recommendations for the condition. This study aims to assess the cost-effectiveness of DBS in treating adults with generalized and cervical dystonia within the Brazilian healthcare context, considering its recent inclusion. METHODS: A systematic review identified randomized controlled trials (RCTs) assessing DBS efficacy in treating adults with generalized and cervical dystonia. Two cost-utility analyses compared the cost-effectiveness of DBS plus the Best Clinical Practice (BCP) to BCP alone. Markov models, which included three health states (no clinical improvement, clinical improvement, and death), employed a one-year cycle and a lifetime horizon. The study utilized both one-way and probabilistic sensitivity analyses. RESULTS: Two RCTs, one for each condition, revealed superior clinical improvement with DBS when compared to sham simulation. The Incremental Cost-Utility Ratio (ICUR) was $ 1,121.66 for generalized dystonia and $4,556.50 for cervical dystonia. Effectiveness discount rates and age at surgery were identified as influential parameters. In 1,000 Monte Carlo simulations, 99.9% of the ICUR values for generalized dystonia and 74.2 % for cervical dystonia fell below the cost-effectiveness threshold in Brazil ($8,146.64 per QALY). CONCLUSIONS: From the perspective of the Brazilian public health system, the combination of DBS and BCP appears to be cost-effective for the treatment of both generalized and cervical dystonia when compared to BCP alone.

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BACKGROUND: Duchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is challenging, highlighting the need for proxy measures. This study aims to catalog and compare existing published health state utility estimates for DMD and related conditions. METHODS: Using two search strategies, relevant utilities were extracted from the Tufts Cost-Effectiveness Analysis Registry, including health states, utility estimates, and study and patient characteristics. Analysis One identified health states with comparable utility estimates to a set of published US patient population utility estimates for DMD. A minimal clinically important difference of ± 0.03 was applied to each DMD utility estimate to establish a range, and the registry was searched to identify other health states with associated utilities that fell within each range. Analysis Two used pre-defined search terms to identify health states clinically similar to DMD. Mapping was based on the degree of clinical similarity. RESULTS: Analysis One identified 4,308 unique utilities across 2,322 cost-effectiveness publications. The health states captured a wide range of acute and chronic conditions; 34% of utility records were extrapolated for US populations (n = 1,451); 1% were related to pediatric populations (n = 61). Analysis Two identified 153 utilities with health states clinically similar to DMD. The median utility estimates varied among identified health states. Health states similar to the early non-ambulatory DMD phase exhibited the greatest difference between the median estimate of the sample (0.39) and the existing estimate from published literature (0.21). CONCLUSIONS: When available estimates are limited, using novel search strategies to identify utilities of clinically similar conditions could be an approach for overcoming the information gap. However, it requires careful evaluation of the utility instruments, tariffs, and raters (proxy or self).

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With the rapid development of new energy and smart technology, the demand for inter-device communication in medium-low-voltage smart distribution grids has sharply increased, leading to a surge in the variety and quantity of communication services. To meet the needs of diverse and massive communication services, deploying service function chains to flexibly combine virtual resources has become crucial. This paper proposes an optimization method based on fit entropy and network utility to address the limited communication network resources in medium-low-voltage smart distribution grids. This was conducted by modeling the distribution grid as a three-domain model consisting of a service domain, a logical domain, and a physical domain and transforming it into a hierarchical bipartite hypergraph-matching problem, which is a complex combinatorial optimization problem. This paper introduces two matching optimization algorithms: "business domain-logic domain-physical domain integration" and "service domain-logic domain, logic domain-physical domain two-stage", which effectively address this problem based on fit entropy and utility. The simulation results demonstrate that these algorithms significantly improve service success rates and resource utilization, enhancing overall network utility.

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Mobile Edge Computing (MEC) is crucial for reducing latency by bringing computational resources closer to the network edge, thereby enhancing the quality of services (QoS). However, the broad deployment of cloudlets poses challenges in efficient network slicing, particularly when traffic distribution is uneven. Therefore, these challenges include managing diverse resource requirements across widely distributed cloudlets, minimizing resource conflicts and delays, and maintaining service quality amid fluctuating request rates. Addressing this requires intelligent strategies to predict request types (common or urgent), assess resource needs, and allocate resources efficiently. Emerging technologies like edge computing and 5G with network slicing can handle delay-sensitive IoT requests rapidly, but a robust mechanism for real-time resource and utility optimization remains necessary. To address these challenges, we designed an end-to-end network slicing approach that predicts common and urgent user requests through T distribution. We formulated our problem as a multi-agent Markov decision process (MDP) and introduced a multi-agent soft actor-critic (MAgSAC) algorithm. This algorithm prevents the wastage of scarce resources by intelligently activating and deactivating virtual network function (VNF) instances, thereby balancing the allocation process. Our approach aims to optimize overall utility, balancing trade-offs between revenue, energy consumption costs, and latency. We evaluated our method, MAgSAC, through simulations, comparing it with the following six benchmark schemes: MAA3C, SACT, DDPG, S2Vec, Random, and Greedy. The results demonstrate that our approach, MAgSAC, optimizes utility by 30%, minimizes energy consumption costs by 12.4%, and reduces execution time by 21.7% compared to the closest related multi-agent approach named MAA3C.

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Social decision-making is guided by a complex set of social norms. Computational modeling can play a significant role in enriching our understanding of these norms and how precisely they direct social choices. Here, we highlight three major advantages to using computational modeling, particularly models derived from Utility Theory, in the study of social norms. We illustrate how such models can help generate detailed processes of decision-making, enforce theoretical precision by delineating abstract concepts, and unpack when, and why, people adhere to specific social norms. For each benefit, we discuss a recent study which has employed modeling in the service of assessing the role of norms in decision-making, collectively revealing how computational modeling enables better prediction, description, and explanation of important social choices.

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Background: Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome. Methods: We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing. Results: We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for path_PMS2 Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery. Limitations: Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would immediately take up risk-reducing surgery if offered, and it is possible that risk-reducing surgery would be more effective and cost-effective if it was taken up when offered. Conclusions: There is insufficient evidence to recommend for or against gynaecological cancer surveillance in Lynch syndrome on clinical grounds, but modelling suggests that surveillance could be cost-effective. Further research is needed but it must be rigorously designed and well reported to be of benefit. Study registration: This study is registered as PROSPERO CRD42020171098. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR129713) and is published in full in Health Technology Assessment; Vol. 28, No. 41. See the NIHR Funding and Awards website for further award information.

Lynch syndrome is an inherited condition which puts people at a higher risk of getting bowel cancer, womb cancer and ovarian cancer. Although people with Lynch syndrome are more likely to get these cancers, they are more likely to survive cancer if they get it. People diagnosed with Lynch syndrome get regular testing (surveillance) using a camera to check for bowel cancer or polyps. For womb and ovarian cancer, surveillance may also be an option, but it is less well studied in these cancers. This means that many women are not offered surveillance. Women with Lynch syndrome are recommended to have risk-reducing surgery when their risk starts rising, if they do not want any more children. We wanted to find out whether surveillance for womb and ovarian cancer would work and would be good value for money. Doctors and patients have said that these are important research questions. We searched for published research on this subject and found a lot of studies, but these studies were often small or not well designed, so they could only tell us a limited amount. Studies did not always measure the things that patients want to know. There was some evidence that people having surveillance might live longer than people not having surveillance, but there was also some evidence that risk-reducing surgery is better than surveillance. Surveillance has detected some cancers which had no symptoms, but there are also cancers diagnosed soon after a surveillance visit where nothing was found. People often find surveillance painful, but experiences vary. Our work shows that surveillance and surgery could be good value for money for many women with Lynch syndrome. We need better research to help patients and doctors decide whether surveillance is right for them.

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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.

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AIMS: This study investigated the cost-utility of real-time continuous glucose monitoring (rt-CGM) versus self-monitoring of blood glucose (SMBG) in people with type 2 diabetes (T2D) receiving intensive insulin therapy in South Korea. METHODS: The IQVIA Core Diabetes Model (CDM v9.5) was used, with clinical effectiveness data obtained from a large-scale real world study. Costs were obtained from South Korean sources and inflated to 2022 South Korean Won (KRW). A South Korean payer perspective was adopted over a lifetime horizon, with future costs and effects discounted at 4.5% per annum. Baseline characteristics included a mean baseline HbA1c level of 8.6% (71 mmol/mol), and a mean age of 64.4 years. A willingness-to-pay (WTP) threshold of KRW 46.0 million was used. RESULTS: Rt-CGM led to an increase of 0.683 quality-adjusted life years (QALYs) versus SMBG (7.526 QALYs for rt-CGM versus 6.843 QALYs for SMBG). An increase in costs of KRW 16.4 million (from KRW 90.4 million to KRW 106.8 million) was associated with rt-CGM. The incremental cost-utility ratio was KRW 24.0 million per QALY gained, significantly lower than the KRW 46 million threshold. CONCLUSIONS: For individuals with T2D managed by intensive insulin therapy in South Korea, rt-CGM is cost-effective relative to SMBG.

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Disseminated intravascular coagulation (DIC) is a critical, life-threatening disorder characterized by widespread activation of the coagulation cascade, leading to microthrombi formation, consumption of clotting factors and platelets, and a paradoxically increased risk of bleeding. Accurate and timely diagnosis is crucial for effective management and improved patient outcomes. This narrative review aims to evaluate the diagnostic accuracy and clinical utility of various scoring systems used to assess DIC. We examine prominent systems, including the International Society on Thrombosis and Haemostasis (ISTH) scoring system, the Japanese Association for Acute Medicine (JAAM) DIC criteria, and other regional or institutional criteria such as the Chinese DIC scoring system (CDSS). The review compares these systems based on their criteria, sensitivity, specificity, and accuracy across different patient populations and discusses their strengths and limitations. Additionally, we explore the impact of these scoring systems on patient management and therapeutic decisions, identify challenges and limitations, and highlight emerging trends and future directions in DIC diagnosis. By providing a comprehensive analysis, this review aims to enhance understanding of DIC scoring methods and inform clinical practice to improve patient care.

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INTRODUCTION: Seasonal influenza outbreaks in France cause a surge in patients, exacerbating the overburdened healthcare system each winter. Older adults are particularly vulnerable to serious events related to influenza. Quadrivalent influenza high dose (QIV HD) vaccines have been developed to offer better clinical protection in older adults, who often exhibit suboptimal immune response to quadrivalent influenza standard dose vaccines (QIV SD). This study aims to evaluate the public health impact and cost-effectiveness of administering HD versus SD vaccines to individuals aged 65+ in France. METHODOLOGY: Using a static model and decision-tree approach, the study analyzed health outcomes such as influenza cases, GP (general practitioner) visits, hospitalizations, and mortality; relative vaccine efficacy (rVE) estimates were derived from a pivotal randomized-controlled trial and a meta-analysis comparing HD to SD vaccines. Two approaches were implemented to model hospitalizations (conditional on influenza or not), and analyses on bed occupancy were performed. RESULTS: Results showed that using QIV HD instead of QIV SD during an average influenza season in France led to the prevention of 57,209 additional cases of influenza, 13,704 GP visits, and 764 influenza-related deaths. Moreover, switching to QIV HD resulted in additional 1,728 to 15,970 hospitalizations avoided and 15,124 to 138,367 reduced days of hospitalization depending on the hospitalization approach used. The cost-utility analysis showed a cost per quality-adjusted life year (QALY) gained ranging from 24,020 €/QALY to 5,036 €/QALY. CONCLUSIONS: Switching to QIV HD in older adults showed to be cost-effective, with even greater public health benefits at higher coverage rate, regardless the season severity.

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INTRODUCTION: Intravenous (IV) iron is the recommended treatment for patients with iron deficiency anemia (IDA) unresponsive to oral iron treatment, in whom oral iron is contraindicated, or where rapid iron replenishment is required. Ferric derisomaltose (FDI) and ferric carboxymaltose (FCM) are high-dose, rapid-infusion, IV iron formulations that have recently been compared in three head-to-head randomized controlled trials (RCTs), which showed significantly higher incidence of hypophosphatemia after administration of FCM than FDI. The present study objective was to evaluate the cost-utility of FDI versus FCM in a population of patients with IDA in China. METHODS: A previously-published patient-level simulation model was used to model the cost-utility of FDI versus FCM in China. The number of infusions of FDI and FCM was modeled based on the approved posology of the respective formulations using simplified tables of iron need in a population of patients with body weight and hemoglobin levels informed by a Chinese RCT of FCM. Data on the incidence of hypophosphatemia was obtained from the PHOSPHARE-IDA RCT, while data on disease-related quality of life were obtained from SF-36v2 data from the PHOSPHARE-IBD RCT. RESULTS: Over the 5-year time horizon, patients received 3.98 courses of iron treatment on average, requiring 0.90 fewer infusions of FDI than FCM (7.69 vs. 6.79). This resulted in iron procurement and administration cost savings of renminbi (RMB) 206 with FDI (RMB 3,519 vs. RMB 3,312). Reduced incidence of hypophosphatemia-related fatigue resulted in an increase of 0.07 quality-adjusted life years and further cost savings of RMB 782 over 5 years, driven by reduced need for phosphate testing and replenishment. FDI was therefore the dominant intervention. CONCLUSIONS: The results showed that FDI would improve patient quality of life and reduce direct healthcare expenditure versus FCM in patients with IDA in China.