Comparison of four value sets derived using different TTO and DCE approaches: application to the new region-specific PBM, AP-7D.

BACKGROUND: AP-7D is a newly developed preference-based measure (PBM) in East and Southeast Asia. However, no value set has been established yet. Comparison of the characteristics of value sets obtained by different methods is necessary to consider the most appropriate methodology for valuation survey of AP-7D. METHOD: We surveyed the general population's preference of AP-7D health states by four valuation methods (a) composite time trade-off (cTTO); (b) simple discrete choice experiment (DCE); (c) DCE with duration; and (d) ternary DCE. In Japan, we collected approximately 1,000 samples for cTTO tasks through a face-to-face survey and 2,500 samples for each of the three DCE tasks. Respondents were selected through quota sampling based on the sex and age. The cTTO data were analyzed using a linear mixed and tobit model; the DCE data were analyzed using a simple and panel conditional logit model. Where the results of the analysis showed inconsistencies, a constrained model was used. RESULTS: Since all the unconstrained models, except simple DCE, showed one or more inconsistencies, the constrained model was used for the analyses. The minimum values for the models were as follows: TTO model, -0.101; simple DCE model, -0.106; DCE with duration model, -0.706; ternary DCE model, -0.306. The score for the DCE with the duration model was much lower than that for the other models. Although the value sets for AP-7D differed among the four valuation methods, the ternary DCE model showed intermediate characteristics between those of the cTTO and DCE with duration models. As compared with to EQ-5D-5L, the distributions of all the scores on the Japanese AP-7D moved to the left. Although "Energy" was one of the domains with the least influence on the AP-7D score in all four models, "Burden to others" had the largest impact on the preferences. CONCLUSION: We constructed four value sets using different TTO and DCE methods. Our findings are expected not only to contribute to the development of AP-7D, but also other preference-based measures.

Are Web-Based Valuation Surveys for Preference-Based Measures as Reliable as Face-to-Face Surveys? TTO, DCE and DCE with Duration.

BACKGROUND: Valuation surveys of preference-based measures are typically conducted face-to-face or on web panels. In this survey, we considered whether face-to-face and online surveys were reliable using three tasks: composite time trade-off (cTTO), discrete choice experiment (DCE), and DCE with duration. METHODS: Respondents (aged 20-69 years) for both face-to-face (N = 1000, target sample size) and web surveys were selected through quota sampling by sex and age from each panel of the general population in Japan. They were then allocated to one of the three tasks and divided into six groups (two survey modes × three tasks, N = 334 per group). For the cTTO, respondents were asked to rate ten health states described by the EQ-5D-5L. For the DCE and DCE with duration surveys, respondents were asked about 15 health-state pairs. For all participants, as in the second-stage survey, a similar process was repeated two weeks after the first survey. Reliability was evaluated by calculating the percentage of agreement and intraclass correlation coefficients. RESULTS: The cTTO scores of the face-to-face and web surveys were systematically different. Between the face-to-face and web surveys, the agreement of the TTO survey was not good. The intraclass correlation coefficient (ICC) was 0.37 for the face-to-face test-test and 0.59 for the web test-retest. Discrete choice experiment (DCE) and DCE with duration had similarly good agreement (more than 70%), regardless of face-to-face or web surveys. However, between the first and second surveys (test-retest) of DCE and DCE with duration, the agreement depends on whether the positions of the two cards (health states) are identical. CONCLUSION: If the face-to-face cTTO score is the gold standard, a web-based survey of cTTO is not recommended regardless of the ICC. If a DCE survey is performed, positioning effects should be considered.

Cost-effectiveness analysis of lifestyle interventions for preventing kidney disease in patients with type 2 diabetes.

BACKGROUND: Lifestyle interventions in patients with type-2 diabetes contribute to reducing the incidence of chronic kidney disease. The cost-effectiveness of lifestyle interventions to prevent kidney disease in patients with type-2 diabetes remains undetermined. We aimed to develop a Markov model from a Japanese healthcare payer's perspective focusing on the development of kidney disease in patients with type-2 diabetes and examine the cost-effectiveness of lifestyle interventions. METHODS: To develop the model, the parameters, including lifestyle intervention effect, were derived from results of the Look AHEAD trial and previously published literature. Incremental cost-effectiveness ratios (ICER) were calculated from the difference in cost and quality-adjusted life years (QALY) between lifestyle intervention and diabetes support education groups. We estimated lifetime costs and effectiveness assuming patient's life span to be 100 years. Costs and effectiveness were discounted by 2% annually. RESULTS: ICER for lifestyle intervention compared to diabetes support education was JPY 1,510,838 (USD 13,031) per QALY. Cost-effectiveness acceptability curve showed that the probability that lifestyle intervention is cost-effective at the threshold of JPY 5,000,000 (USD 43,084) per QALY gained, compared to diabetes support education, is 93.6%. CONCLUSIONS: Using a newly-developed Markov model, we illustrated that lifestyle interventions for preventing kidney disease in patients with diabetes would be more cost-effective from a Japanese healthcare payer's perspective compared to diabetes support education. The model parameters in the Markov model must be updated to adapt to the Japanese setting.

Comparison of physicians' and dentists' incident reports in open data from the Japan Council for Quality Health Care: a mixed-method study.

BACKGROUND: Patient safety is associated with patient outcomes. However, there is insufficient evidence of patient safety in the dental field. This study aimed to compare incidents reported by dentists and physicians, compare the type of errors made by them, and identify how dentists prevent dental errors. METHODS: A mixed-methods study was conducted using open data from the Japan Council for Quality Health Care database. A total of 6071 incident reports submitted for the period 2016-2020 were analyzed; the number of dentists' incident reports was 144, and the number of physicians' incident reports was 5927. RESULTS: The percentage of dental intern reporters was higher than that of medical intern reporters (dentists: n = 12, 8.3%; physicians: n = 180, 3.0%; p = 0.002). The percentage of reports by dentists was greater than that by physicians: wrong part of body treated (dentists: n = 26, 18.1%; physicians: n = 120, 2.0%; p < 0.001), leaving foreign matter in the body (dentists: n = 15, 10.4%; physicians: n = 182, 3.1%; p < 0.001), and accidental ingestion (dentists: n = 8, 5.6%; physicians: n = 8, 0.1%; p < 0.001), and aspiration of foreign body (dentists: n = 5, 3.4%; physicians: n = 33, 0.6%; p = 0.002). The percentage of each type of prevention method utilized was as follows: software 27.8% (n = 292), hardware (e.g., developing a new system) 2.1% (n = 22), environment (e.g., coordinating the activities of staff) 4.2% (n = 44), liveware (e.g., reviewing procedure, double checking, evaluating judgement calls made) 51.6% (n = 542), and liveware-liveware (e.g., developing adequate treatment plans, conducting appropriate postoperative evaluations, selecting appropriate equipment and adequately trained medical staff) 14.3% (n = 150). CONCLUSION: Hardware and software and environment components accounted for a small percentage of the errors made, while the components of liveware and liveware-liveware errors were larger. Human error cannot be prevented by individual efforts alone; thus, a systematic and holistic approach needs to be developed by the medical community.

Impacts of the preceding cancer-specific health-related quality of life instruments on the responses to the subsequent EQ-5D-5L.

BACKGROUND: In clinical studies, the EQ-5D-5L is often employed with disease-specific health-related quality of life instruments. The questions in the former are more general than the latter; however, it is known that responses to general questions can be influenced by preceding specific questions. Thus, the responses to the EQ-5D-5L have the possibility of being influenced by the preceding disease-specific health-related quality of life instruments. This may lead to bias in the cost-effectiveness analysis results. Therefore, this study aimed to evaluate the impact of the preceding cancer-specific health-related quality of life instruments on the EQ-5D-5L responses. METHODS: We prepared questionnaire booklets containing the EQ-5D-5L, the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30, and the Functional Assessment of Cancer Therapy General with different orders. Using a quasi-randomized design, they were distributed to the patients undergoing drug therapy for advanced cancer, who were classified into three groups: Groups 1, 2, and 3 (the EQ-5D-5L placed first, second, and last, respectively). We compared the EQ-5D-5L index and the missingness of EQ-5D-5L among the groups. RESULTS: The mean EQ-5D-5L index was 0.796, 0.760, and 0.789 for groups 1 (n = 300), 2 (n = 306), and 3 (n = 331), respectively. The difference between Groups 2 and 1 was - 0.036 (95% CI - 0.065 to - 0.007; p = 0.015). The proportion of patients with an incomplete EQ-5D-5L was 0.11, 0.11, and 0.05 for Groups 1, 2, and 3, respectively. The difference of the proportions between group 3 and 1 and between 3 and 2 was - 0.06 (95% CI - 0.10 to - 0.02; p = 0.003) and - 0.06 (95% CI - 0.10 to - 0.02; p = 0.003), respectively. CONCLUSIONS: Although the EQ-5D-5L index differed according to the instrument orders, the difference size would not be considerably larger than the minimally important difference. The patients tended to complete the EQ-5D-5L when they were placed at the end of the questionnaire.

Critical Incident Reports Related to Ventilator Use: Analysis of the Japan Quality Council National Database.

OBJECTIVE: This study aimed to assess the factors associated with medical device incidents. METHODS: In this mixed-methods study, we used incident reporting data from the Japan Council for Quality Health Care. Of the 232 medical device-related reports that were downloaded, 34 (14.7%) were ventilator-associated incidents. Data related to patients, situations, and incidents were collected and coded. RESULTS: The frequencies of ventilator-associated accidents were 20 (58.8%) during the daytime and 14 (41.2%) during the night/early morning. Ventilator-associated accidents occurred more frequently in the hospital room (n = 22 [64.7%]) than in the intensive care unit (n = 4 [11.8%]). Problems with ventilators occurred in only 4 cases (11.8%); in most cases, medical professionals experienced difficulty with the use or management of ventilators (n = 30 [88.2%]), and 50% of them were due to misuse/misapplication of ventilators (n = 17 [50.0%]). Ventilator-associated accidents were caused by an entanglement of complex factors-hardware, software, environment, liveware, and liveware-liveware interaction. Communication and alarm-related errors were reported to be related, as were intuitiveness or complicated specifications of the device. CONCLUSIONS: Our study revealed that ventilator-associated accidents were caused by an entanglement of complex factors and were related to inadequate communication among caregivers and families. Moreover, alarms were overlooked owing to inattentiveness. Mistakes were generally caused by a lack of experience, insufficient training, or outright negligence. To reduce the occurrence of ventilator-associated accidents, hospital administrators should develop protocols for employment of new devices. Medical devices should be developed from the perspective of human engineering, which could be one of the systems approaches.

Gradient Boosted Tree Approaches for Mapping European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Onto 5-Level Version of EQ-5D Index for Patients With Cancer.

OBJECTIVES: This study aimed to develop direct and response mapping algorithms from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 onto the 5-level version of EQ-5D index based on the gradient boosted tree (GBT), a promising modern machine learning method. METHODS: We used the Quality of Life Mapping Algorithm for Cancer study data (903 observations from 903 patients) for training GBTs and testing their predictive performance. In the Quality of Life Mapping Algorithm for Cancer study, patients with advanced solid tumor were enrolled, and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 and 5-level version of EQ-5D were simultaneously evaluated. The Japanese value set was used for direct mapping, whereas the Japanese and US value sets were used for response mapping. We trained the GBTs in the training data set (80%) with cross-validation and tested the predictive performance measured by the root mean squared error (RMSE), mean absolute error (MAE), and mean error in the test data set (20%). RESULTS: The RMSE and MAE in the test data set were larger in the GBT approaches than in the previously developed regression-based approaches. The mean error in the test data set tended to be smaller in the GBT approaches than in the previously developed regression-based approaches. CONCLUSIONS: The predictive performances in the RMSE and MAE did not improve by the GBT approaches compared with regression approaches. The flexibility of the GBT approaches had the potential to reduce overprediction and underprediction in poor and good health, respectively. Further research is needed to establish the role of machine learning methods in mapping a nonpreference-based measure onto health utility.

Developing a New Region-Specific Preference-Based Measure in East and Southeast Asia.

OBJECTIVES: Almost all preference-based measures (PBMs) have been developed in Western countries, with none having been formulated in Asian countries. In this study, we construct a new generic PBM based on concept elicitation using interview surveys in East and Southeast Asian countries and qualitative analysis. METHODS: This cross-sectional study included 225 adults recruited from 9 East and Southeast Asian countries or regions (Indonesia, Japan, Korea, mainland China, Malaysia, the Philippines, Singapore, Taiwan, and Thailand). Trained interviewers conducted semistructured interviews with 25 participants from the general population of each country/region. Qualitative data were analyzed using a content analysis approach. The selection of items was determined based on interview surveys and team member discussions. The description of items was considered based on a detailed qualitative analysis of the interview survey. RESULTS: A new region-specific PBM-the Asia PBM 7 dimensions instrument-was designed. It reflects East and Southeast Asian values and comprises 7 items: pain, mental health, energy, mobility, work/school, interpersonal interactions, and burden to others. CONCLUSIONS: The new region-specific instrument is one of the first PBMs developed in the context of non-Western countries. The Asia PBM 7 dimensions contains 7 items that address the core concepts of health-related quality of life that are deemed important based on East and Southeast Asian health concepts.

Choking injuries: Associated factors and error-producing conditions among acute hospital patients in Japan.

Choking can lead to mortality and residual impairments. This study aimed to determine the factors associated with choking among acute hospital patients and examine error-producing conditions to suggest choking-prevention policies. Among 36,364 cases reported by hospital staff at an acute university hospital from 2012 to 2018 were examined using a retrospective study, 35,440 were analysis as the number of cases analysed for the study. We used descriptive statistics to present patient characteristics and conducted univariable and multivariable logistic regression analyses to identify factors associated with choking. Additionally, we conducted content analysis (root cause analysis) to examine error-producing conditions and prevention policies. Sixty-eight cases were related to choking injuries; of these, 43 patients (63.2%) were male, and 38 (55.9%) were aged 65 years and older. Choking cases had a high percent of adverse outcomes involving residual impairment or death (n = 23, 33.8%). Mental illness (adjusted odds ratio [95% confidence interval]: 3.14 [1.39-7.08]), and hospitalisation in the general wards (adjusted odds ratio [95% confidence interval]: 3.13 [1.70-5.76]) were associated with an increased probability of choking. Error production was caused by food (n = 25, 36.8%) and medical devices or supplies (n = 13, 19.1%). Almost all contributory factors were associated with inadequate checking (n = 66, 97.1%) and misperception of risk (n = 65, 95.6%). Choking poses a highly significant burden on patients, and hospital administrators should minimise the risk of choking to prevent related injuries. Hospital administrators should provide training and education to their staff and develop adequate protocols and procedures to prevent choking.

Cost-Effectiveness of Trastuzumab With or Without Chemotherapy as Adjuvant Therapy in HER2-Positive Elderly Breast Cancer Patients: A Randomized, Open-Label Clinical Trial, the RESPECT Trial.

BACKGROUND AND OBJECTIVE: Trastuzumab is a standard care as adjuvant chemotherapy (AdjCT) for patients with human epidermal growth factor receptor 2 (HER2)-positive primary breast cancer (BC) in Japan. However, no reports have evaluated its economics for patients with HER2-positive BC over 70 years of age. The objective of this study was to evaluate the cost-effectiveness of HER2-targeted trastuzumab + chemotherapy in Japan, comparing it with trastuzumab monotherapy. METHODS: A three-state-partitioned survival model was developed to evaluate the cost-effectiveness of trastuzumab + chemotherapy versus trastuzumab monotherapy for AdjCT in elderly patients with HER2-positive BC. We derived the efficacy data, utilities, and costs of both arms from individual patient data in the RESPECT trial (NCT01104935) and published studies. The costs and quality-adjusted life years (QALYs) were discounted at 2% per annum using a payer perspective. The respective cost estimates were reported in 2019 Japanese Yen (JPY) or US dollars (US$). The primary outcome was the incremental cost-effectiveness ratio (ICER). We assured robustness with deterministic and probabilistic sensitivity analyses. RESULTS: The cost per patient for trastuzumab + chemotherapy was JPY 14.6 million (US$137,000), and their QALYs were 9.308, compared with JPY 14.2 million (US$131,000) and 9.101, respectively, for trastuzumab monotherapy. The ICER of trastuzumab + chemotherapy versus trastuzumab monotherapy was JPY 2.7 milllion/QALY (US$17,200/QALY). The ICER for trastuzumab with chemotherapy varied from "Dominant" to "Dominated" in one-way sensitivity analysis. CONCLUSIONS: The base-case analysis suggests that AdjCT with trastuzumab + chemotherapy is likely to be a cost-effective choice for patients with HER2-positive BC aged 70 years or older. However, the sensitivity analysis suggested uncertainty regarding the cost-effectiveness of trastuzumab + chemotherapy.

Reported Incidents Involving Non-medical Care Workers and Nursery Teachers in Hospitals in Japan: An Analysis of the Japan Council for Quality Health Care Nationwide Database.

Objective With the shortage of medical staff, the birth rate decline, and aging populations in some countries, task shifting from specific medical staff to non-medical care workers in hospitals has been implemented as a short-term solution. Incident reporting reduces preventable patient errors, improves the quality of healthcare services, and contributes to patient safety. However, research focused on the expanding roles of non-medical staff who provide direct care for patients is lacking. The present study aimed to bridge this gap by examining reported incidents involving non-medical care workers and nursery teachers in hospitals in Japan. Methodology A retrospective mixed-methods study was conducted using data published by the Japan Council for Quality Health Care. A total of 21,876 cases were reported between 2016 and 2020, and 97 out of 21,876 cases were analysed, after excluding incidents involving workers or staff other than care workers/nursery teachers. Descriptive statistics were used to examine the incidents, and textual data included in the incident reports were analysed by two registered nurses. Results The occupations of the people involved were care worker (n=80, 82.5%) and nursery teacher (n=17, 17.5%). There were two reports of worker injuries (n=2, 2.1%), which were excluded. A total of 95 cases were included in the final analysis to examine the effects on patients. Among the remaining 95 cases, there were five severe patient incidents (death, n=2, 2.1%; cerebral hemorrhage, n=3, 3.2%), and the most frequent incident was bone fracture (n=64, 67.4%). Some patients had cognitive impairment (n=29, 30.5%) and osteoporosis (n=25, 26.3%). We divided the factors related to incident occurrence into software (procedures and protocols), environment (wards and theaters), and liveware (people, including care workers, nursery teachers, and patients). Regarding the reasons for the incidents, the percentages for the three factors were as follows: education/training 34.7% (n=33), in software; patient state 4.1% (n=39), in environment; and neglect to observe 45.3% (n=43), in liveware. Conclusion Our study involved a secondary analysis of published data, and the sample size was small. However, incident reports from care workers and nursery teachers working in hospitals included serious errors. The role of non-medical care staff in hospitals is broad and diverse, and has been shifting from direct care for patients with mild illnesses to direct care for patients with severe illnesses. An efficient clinical environment that ensures quality of care and service is lacking. By focusing on patient safety outcomes, policymakers and hospital teams should consider adjusting the working environment.

Estimating Value-Based Price and Quantifying Uncertainty around It in Health Technology Assessment: Frequentist and Bayesian Approaches.

BACKGROUND: Although several statistical methods have been developed to inform decision making on reimbursement under uncertainty (e.g., expected net benefit, cost-effectiveness acceptability curves, and expected value of perfect information [EVPI]), those for value-based pricing are limited. This research develops methods for estimating the value-based price and quantifying the uncertainty around it in health technology assessment. METHODS: We defined the value-based price of a medical product under assessment as the price at which the incremental cost-effectiveness ratio is just equal to a cost-effectiveness threshold. According to this definition, we derived an explicit form of the value-based price. Using this explicit form, we developed frequentist and Bayesian approaches to value-based pricing under uncertainty. Our proposed methods were illustrated via 2 hypothetical case studies. RESULTS: The value-based price can be expressed explicitly using cost, effectiveness, and a cost-effectiveness threshold and is a linear function of a cost-effectiveness threshold. In the frequentist framework, point estimation, interval estimation, and hypothesis testing for the value-based price are available. In the Bayesian framework, the best estimate of the value-based price under uncertainty is the weighted median value-based price with the weight of the expected consumption volume of a medical product under assessment. This is based on the opportunity loss incurred by a decision error in value-based pricing. This opportunity loss also provides a basis for the calculation of EVPI associated with value-based pricing. These methods provided estimates of the value-based prices of medical products and the uncertainty around them in 2 hypothetical case studies. CONCLUSIONS: Our developed methods can improve decision making on value-based pricing in health technology assessment.

Japanese preference weights of the Adult Social Care Outcomes Toolkit for Carers (ASCOT-Carer).

PURPOSE: We developed preference weights of the Adult Social Care Outcomes Toolkit for Carers (ASCOT-Carer) in Japan. METHODS: We used best-worst scaling (BWS) and composite time trade-off (cTTO) to determine the preference weights for ASCOT-Carer states in the general population. TTO values were applied to convert the BWS scores to utilities. The sample number was approximately 1000 for the BWS survey and 200 for the TTO survey. Whereas face-to-face surveys by computer-assisted interviewing were adopted for the TTO tasks, a web-based survey was used for the BWS tasks. In the BWS tasks, the ASCOT-Carer states were presented, and the "best," "worst," "second best," and "second worst" domains in a profile were selected. A mixed logit model was applied to the BWS data. RESULTS: The respondents' background was similar to that of the general population, although the number of people in the age and sex categories was equal. The preference weights for calculating the utilities of the ASCOT-Carer states were estimated. The estimated utilities of the ASCOT-Carer states were distributed between 1 and 0.02. All preference weights were consistent. The item with the highest preference weight was level 1 in the "space and time to be yourself." The least preferred item was level 4 in the "space and time to be yourself" and "control over daily life" domains. CONCLUSION: We established Japanese preference weights for ASCOT-Carer states, the first weights of an Asian country. The estimated utilities can contribute to the measurement of caregivers' social care-related QoL and perform of cost-effectiveness analyses.

Minimal important differences of EORTC QLQ-C30 for metastatic breast cancer patients: Results from a randomized clinical trial.

PURPOSE: To establish minimal important differences (MIDs) for the European Organisation for Research and Treatment for Cancer Quality of life Questionnaire core 30 (EORTC QLQ-C30) in patients with metastatic breast cancer. METHODS: The dataset was obtained from the SELECT BC-CONFIRM randomized clinical trial. Anchors obtained from patients (transition items) and clinicians (performance status) were used for anchor-based methods. Anchors obtained through 6 months after starting treatment were used for this analysis. Correlation coefficients of anchor and change in QLQ-C30 and effect size were used to qualify for estimating MIDs. Mean change method and generalized estimating equation were applied to estimate MIDs. Distribution-based methods were used for comparison. RESULTS: We analyzed a dataset of 154 metastatic breast cancer patients. MIDs were estimated in 8 of 15 scales of QLQ-C30. Estimated MIDs for within-group improvement varied from 7 to 15 and those for deterioration varied from - 7 to - 17. Estimated MIDs for between-group improvement varied from 5 to 11 and those for deterioration varied from - 5 to - 8 across QLQ-C30 scales. Patient-reported anchors were more susceptible to early changes in health status than clinician-reported anchors. CONCLUSION: We provided the MIDs of the QLQ-C30 using both patient- and clinicians-reported anchors measured in a randomized trial of Japanese patients with metastatic breast cancer. We recommend patient-reported anchors for anchor-based estimation of MID. Our results can aid patients and clinicians, as well as researchers, in the interpretation of QLQ-C30.

Cost-effectiveness of 12 months of capecitabine as adjuvant chemotherapy for stage III colon cancer: preplanned cost-effectiveness analysis of the JFMC37-0801 study.

OBJECTIVES: We evaluated the cost-effectiveness of a 12-month regimen of oral capecitabine versus a standard 6-month regimen as postoperative adjuvant chemotherapy for stage III colon cancer. METHODS: We utilized patient-level data from a multi-institutional randomized controlled trial (JFMC37-0801) that investigated prolonged oral fluoropyrimidine monotherapy. The analysis considered three health states: stable disease, post-metastasis, and death. A parametric statistical model with a cure model was used to estimate the survival curve. The analysis was conducted from the Japanese public healthcare payer's perspective, considering only direct medical costs. A lifetime horizon was used, with a discount rate of 2% for both cost and health outcomes. Health outcomes were evaluated in terms of quality-adjusted life-years (QALYs). RESULTS: The estimated cure rates for colon cancer were 0.726 [95% confidence interval (CI) 0.676-0.776] and 0.694 (95% CI 0.655-0.733) with the 12- and 6-month regimens, respectively; and the estimated 5-year relapse-free survival rates were 74.4% and 69.8%, respectively. The estimated lifetime cost for 12 months of capecitabine was JPY 3.365 million (USD 31,159), compared with JPY 3.376 million (USD 31,262) for 6 months. The estimated QALY were 12.48 and 11.77 for the 12- and 6-month regimens, respectively. Thus, the 12-month capecitabine regimen was dominant. Using a willingness-to-pay threshold of JPY 5 million per QALY, we determined a 97.4% probability that the 12-month capecitabine regimen is more cost-effective than the 6-month regimen. CONCLUSIONS: Twelve months of capecitabine is the favorable option for postoperative adjuvant chemotherapy for stage III colon cancer from the perspective of cost-effectiveness.

Randomized Controlled Trial of Paper-Based at a Hospital versus Continual Electronic Patient-Reported Outcomes at Home for Metastatic Cancer Patients: Does Electronic Measurement at Home Detect Patients' Health Status in Greater Detail?

PURPOSE: This study aimed to determine whether continual electronic patient-reported outcome (ePRO) measurements at home can capture the fluctuations in health-related quality of life (HRQOL) scores between visits. METHODS: We performed a randomized controlled trial to compare the scores obtained by standard practice (paper-based measurements in the hospital) to scores by continuous measurement of ePRO at home. Metastatic cancer patients were randomly assigned to either the paper-based (n = 50) or the ePRO group (n = 52). EQ-5D-5L and EORTC QLQ C-30 scores were obtained on 3 different chemotherapy days in the paper-based group. Meanwhile, scores were obtained on the chemotherapy day and on days 3, 7, 10, and 14 in the ePRO group during 2 cycles. The first hypothesis of our study was that both scores at the same time points would be equivalent despite different measurement frequency, place, or mode of measurement. The second hypothesis was that PRO score-adjusted time would be different between the groups. For equivalence, the endpoint was the mean EQ-5D-5L index value on the chemotherapy day before the outpatient treatment. Only if equivalence was shown, quality-adjusted life-days (QALDs) were considered using all the data. RESULTS: The adjusted mean difference in the EQ-5D-5L index was determined to be -0.013 (95% confidence interval [CI]: -0.049 to 0.022); the 95% CI did not exceed the equivalence margin. Similarly, the mean difference in global health status (2.28 [95% CI: -2.55 to 7.11]) also showed equivalence. However, the QALD by EQ-5D-5L was significantly lower in the ePRO group by 1.36 per 30 d (95% CI: -2.22 to -0.51; P = 0.0021). CONCLUSIONS: Continual measurements of the HRQOL at home by ePRO may yield more detailed profiles of the HRQOL.

Estimation of Lifetime QALYs Based on Lifestyle Behaviors.

Various lifestyle behaviors have been known to affect health-related quality of life (HRQL) and life expectancy. However, the impact on quality-adjusted life years (QALYs), which can be used for health economics, has not been clarified. The purpose of this study was to estimate the impact of lifestyle behaviors on lifetime QALYs. We first examined the relationship between lifestyle behaviors and HRQL as measured by the EQ-5D-5L among 4000 participants via a web-based survey. The results of multiple regression analysis showed that physical activity and sleep were significantly related to HRQL. Therefore, we used microsimulation to estimate QALYs from physical activity and sleep, which were determined to be significant in the regression analysis. The results showed that there was a difference of 3.6 QALYs between the recommended lifestyle scenario (23.4 QALYs; 95%CI 3.6 to 35.1) and the non-recommended lifestyle scenario (19.8 QALYs; 95%CI 3.1 to 31.6). This difference was greater in the younger age group than in the older age group. The results also indicated a large difference in QALYs between physical activity and sleep. These findings may provide a significant suggestion for future health promotion measures.

Japanese Population Norms of EQ-5D-5L and Health Utilities Index Mark 3: Disutility Catalog by Disease and Symptom in Community Settings.

OBJECTIVES: This study aimed to establish the Japanese population norms of the EQ-5D-5L and Health Utilities Index Mark 3 (HUI3) and estimate the disutility associated with diseases and symptoms. METHODS: We performed a door-to-door survey of the general population by random sampling. The planned sample size was 10 000 residents (age ≥16 years) of 334 districts in Japan. In addition to the EQ-5D-5L and HUI3 questionnaires, questions regarding demographic factors and self-reported main diseases and symptoms were asked. The EQ-5D-5L and HUI3 responses were converted to index values on the basis of Japanese value sets. Summary values by age and sex were calculated to obtain Japanese normative values. A multiple linear model was used to examine relationships between these values and diseases and symptoms. RESULTS: We collected 10 183 responses from 334 districts. The mean EQ-5D-5L index values were 0.821 (male) and 0.774 (female) in the age group of 80 to 89 years, which were lower compared with 0.978 (male) and 0.967 (female) in the age group of 16 to 19 years. Similar trends were observed for the HUI3 values. Age, sex, household income, and education level had a significant influence on the values of both instruments. When measured with the EQ-5D-5L, Parkinson disease, dementia, and stroke were associated with the largest disutility (>0.2), and the disutility for depression was approximately 0.18. In contrast, the HUI3 disutility values for Parkinson disease and dementia were approximately 0.4. CONCLUSIONS: This study established the Japanese population norms of the EQ-5D-5L and HUI3, which can be used in healthcare decision making and contribute to a more reliable analysis of economic evaluations.

EQ-5D-Y Population Norms for Japanese Children and Adolescents.

OBJECTIVE: This study was aimed at establishing population norms of EQ-5D-Y in Japan by a nationwide large sample survey. METHODS: We performed a door-to-door survey by visiting the homes of children/adolescents aged 8-15 years selected by random sampling. The planned sample size was 3600 from 100 districts in Japan. Children/adolescents were asked to respond to the EQ-5D-Y instrument, and their parents, to background questions on themselves and their children. Summary statistics by age/sex were calculated to obtain the Japanese population norms. A multiple linear regression model was used to examine the relationships between the EQ-5D-Y index and their parents' demographic factors, the children/adolescents' diseases/symptoms and the family environment. RESULTS: We collected 3636 responses from 100 districts. The overall EQ-5D-Y index values (all sexes, ages) ranged from 0.90 to 0.95. The percentage of respondents reporting full health ranged from 40 to 60%. In regard to the influence of the children/adolescents' diseases/symptoms on disutility, developmental disability showed the largest disutility values of 0.090. Sleeplessness and body pain were the symptoms that exerted the greatest influence on the EQ-5D-Y index; the effect sizes ranged from - 0.030 to - 0.098 for sleeplessness, and from - 0.023 to - 0.079 for body pain. The EQ-5D-Y index of children/adolescents with parents who reported severe stress was lower by 0.072 (p < 0.001), as compared with that of children/adolescents with parents reporting no stress. CONCLUSIONS: Population norms of EQ-5D-Y were established for the first time. We also clarified the relationship between the EQ-5D-Y index value and the children/adolescents' diseases/symptoms and the family environment.

Valuation Survey of EQ-5D-Y Based on the International Common Protocol: Development of a Value Set in Japan.

BACKGROUND: EQ-5D-Y is a preference-based measure for children and adolescents (aged 8-15 y). This is the first study to develop an EQ-5D-Y value set for converting EQ-5D-Y responses to index values. METHODS: We recruited 1047 respondents (aged 20-79 y) from the general population, stratified by gender and age group, in 5 Japanese cities. All data were collected through face-to-face surveys. Respondents were asked to value EQ-5D-Y states for a hypothetical 10-y-old child from a proxy perspective using composite time tradeoff (cTTO) and a discrete choice experiment (DCE). The discrete choice data were analyzed using a mixed logit model. Latent DCE values were then converted to a 0 (death)/1 (full health) scale by mapping them to the cTTO values. RESULTS: The mean observed cTTO value of the worst health state [33333] was 0.20. Analysis of the DCE data showed that the coefficients of the domains related to mental functions ("Having pain or discomfort" and "Feeling worried, sad, or unhappy") were larger than those for the domains related to physical and social functions. By converting latent DCE values to a utility scale, we constructed a value set for EQ-5D-Y. No inconsistencies were observed. The minimum predicted score was 0.288 [33333], and the second-best score was 0.957 [12111]. CONCLUSION: A value set for EQ-5D-Y was successfully constructed. This is the first survey of an EQ-5D-Y value set. Interpreting the differences between EQ-5D-Y and EQ-5D-5L value sets is a future task with implications for health care policy.