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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., (Copyright © 2022 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc. All rights reserved.)

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., (Copyright © 2022 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

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., (Copyright © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Objectives: Although numerous mapping algorithms from a non-preference-based measure to a target health utility measure have been developed and applied in cost-utility analyses (CUAs), conditions for a mapping algorithm to work well in a CUA are still unclear. In this research, we formulate the mapping problem as a missing data problem and clarify these conditions., Methods: We defined a valid mapping algorithm based on the purpose of mapping (ie, not for prediction but for CUA), and derived a sufficient set of conditions for a valid mapping algorithm. We also conducted a simulation study to investigate properties of a mapping algorithm under situations where the conditions are satisfied and violated., Results: The derived sufficient conditions indicate that the complete overlap of the source measure with the target health utility measure is important and that a covariate that is omitted from a mapping algorithm but has an effect on the target health utility measure not captured by the source measure may invalidate a mapping algorithm. The conditions cannot be verified from data in a CUA but can be supported using external data. A simulation study showed that when at least 1 of the 3 conditions was violated, a mapping algorithm provided biased health utility estimates in a CUA, and that prediction accuracy did not necessarily reflect performance of a mapping algorithm in a CUA., Conclusion: The derived conditions provide a fundamental basis for better practices in developing and selecting a mapping algorithm., (Copyright © 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Objective: We investigated the quantification of the response shift-adjusted treatment effect on quality-of-life (QOL) data in a randomized controlled trial of taxane versus S-1 for patients with metastatic breast cancer (SELECT-BC)., Methods: This study was a secondary data analysis of a previously published trial. The response shift-adjusted treatment effect on health-related QOL (HRQOL) data measured by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) was estimated using structural equation modeling techniques in addition to quantifying the "true" treatment effect. Measurement invariances in the values of the common factor loadings, intercepts, and residual variances between before treatment and at the 3-, 6-, and 12-month visits were considered the response shift effects., Results: In the taxane group, we observed positive recalibration effects for role functioning and positive reprioritization and negative recalibration effects for emotional functioning. The observed change of -4.56 for role functioning comprised +2.26 response shifts and -6.82 "true" change. The observed change of +9.41 for emotional functioning comprised +12.43 response shifts and -1.17 "true" change. In the S-1 group, we observed positive reprioritization and negative recalibration effects for emotional functioning and positive reprioritization effects for social functioning. The observed change of +10.54 for emotional functioning comprised +10.07 response shifts and +0.47 "true" change. The observed change of +2.43 for social functioning comprised +3.50 response shifts and -1.07 "true" change., Conclusion: Detailed analysis of the response shift effects will improve the evaluation reliability of observed HRQOL data during clinical trials., (Copyright © 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

In April 2019, Japan formally introduced health technology assessment (HTA) and, more specifically, a cost-effectiveness analysis, to inform healthcare decision making, mainly when it comes to the pricing of new technologies. This article provides an overview of this new policy, which was implemented formally after a pilot program. In the fiscal year (FY) 2012, discussions on cost-effectiveness assessments were initiated in Japan. After 7 years of deliberations, a cost-effectiveness assessment was implemented formally in April 2019. In Japan, the cost-effectiveness analysis has been used to inform price adjustments of healthcare technologies, although it has not yet been used for decision making on insurance coverage. Selection criteria were established because not all drugs and medical devices could be evaluated owing to a shortage of experts. Exclusion criteria have also been applied to prevent access restriction. The scope of the evaluation's price adjustment target is limited to part of the product price. If the cost per quality-adjusted life-year (QALY) threshold falls below ¥5 million per QALY, the price adjustment rate changes stepwise according to the cost per QALY. In addition to price reduction, a price-raising scheme has also been implemented for scenarios where products are evaluated to be highly cost-effective and innovative. This article describes the first formally implemented HTA system in Japan. Although it is too early to make any conclusions about its effect, the Japan-specific context makes this system unique. To fully understand the opportunities and challenges of the new system, it is vital that Japan accumulates experience with this system and develops human resources in health economic evaluation., (Copyright © 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Objectives: A new rechargeable dual-channel deep brain stimulation (DBS) system has been introduced for the treatment of Parkinson's disease and other movement disorders. However, the clinical value of the device, which has a high cost, remains unclear., Materials and Methods: We conducted a cost-minimization analysis using a national database of health insurance claims in Japan. DBS-related costs were compared between rechargeable and non-rechargeable devices and estimated across a 20-year period., Results: Although the price of rechargeable DBS was higher than that of non-rechargeable DBS, we observed total cost-savings of 8.4 million yen across 20 years by considering costs related to implantation surgery, frequency of replacement, and risk of complications., Conclusions: In this study, real-world evidence indicated that rechargeable dual-channel DBS is a reasonable choice for saving total medical costs. Price revisions should consider cost-effectiveness findings for medical devices., (© 2018 International Neuromodulation Society.)

Objectives: In Japan, cost-effectiveness evaluation was implemented on a trial basis from fiscal year 2016. The results will be applied to the future repricing of drugs and medical devices. On the basis of a request from the Central Social Insurance Medical Council (Chuikyo), our research team drafted the official methodological guideline for trial implementation. Here, we report the process of developing and the contents of the official guideline for cost-effectiveness evaluation., Methods: The guideline reflects discussions at the Chuikyo subcommittee (e.g., the role of quality-adjusted life-year) and incorporates our academic perspective. Team members generated research questions for each section of the guideline and discussions on these questions were carried out. A draft guideline was prepared and submitted to the Ministry of Health, Labour and Welfare (MHLW), and then to the subcommittee. The draft guideline was revised on the basis of the discussions at the subcommitte, if appropriate., Results: Although the "public health care payer's perspective" is standard in this guideline, other perspectives can be applied as necessary depending on the objective of analysis. On the basis of the discussions at the subcommittee, quality-adjusted life-year will be used as the basic outcome. A discount rate of 2% per annum for costs and outcomes is recommended. The final guideline was officially approved by the Chuikyo general assembly in February 2016., Conclusions: This is the first officially approved guideline for the economic evaluation of drugs and medical devices in Japan. The guideline is expected to improve the quality and comparability of submitted cost-effectiveness data for decision making., (Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Background: The valuation study of the five-level version of the EuroQol five-dimensional questionnaire (EQ-5D-5L) involved composite time trade-off (cTTO) and a discrete choice experiment (DCE). The DCE scores must be anchored to the quality-of-life scale from 0 (death) to 1 (full health). Nevertheless, the characteristics of the statistical methods used for converting the EQ-5D-5L DCE results by using TTO information are not yet clearly known., Objectives: To present the Japanese DCE value set of the EQ-5D-5L and compare three methods for converting latent DCE values., Methods: The survey sampled the general population at five locations in Japan. 1098 respondents were stratified by age and sex. To obtain and compare the value sets of the EQ-5D-5L, the cTTO and DCE data were analyzed by a linear mixed model and conditional logit, respectively. The DCE scores were converted to the quality-of-life scale by anchoring to the worst state using cTTO, mapping DCE onto cTTO, and a hybrid model., Results: The data from 1026 respondents were analyzed. All the coefficients in the cTTO and DCE value sets were consistent throughout all the analyses. Compared with the cTTO algorithm, the mapping and hybrid methods yielded very similar scoring coefficients. The hybrid model results, however, produced a lower root mean square error and fewer health states with errors exceeding 0.05 than did the other models. The DCE anchored to the worst state overestimated the cTTO scores of almost all the health states., Conclusions: Japanese value sets based on DCE were demonstrated. On comparing the observed cTTO scores, we found that the hybrid model was slightly superior to the simpler methods, including the TTO model., (Copyright © 2016 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Objectives: When quality-adjusted life-years are used for economic evaluation, the controversial issue of "double counting" of productivity loss emerges, particularly given the lack of empirical data., Methods: In this study, we performed a Web-based, large-sample survey to address the issue of double counting. To determine the influence of income reduction on utility scores, we obtained utility scores of eight health states with three instruction types: a) no instruction, b) instructed to consider income reduction, and c) instructed not to consider income reduction (compensated). Respondents were randomly sampled from the online panel and asked to evaluate 1 of 24 patterns by both standard gamble and time trade-off methods., Results: A total of 6551 respondents completed the questionnaire. First, despite the lack of instruction on income reduction, many respondents spontaneously assumed lost income. The proportion tended to be higher when considering more severe health states. Second, the degree of assumed income reduction was related to utility scores. For a 10% income reduction, respondents assumed a 0.02- to 0.04-decrease in utility score (both standard gamble and time trade-off methods). Third, utility scores did not change significantly when instruction was given not to consider income reduction (compensated) compared with when no instruction was given., Conclusions: An assumed income reduction clearly influenced utility scores; however, compensation for lost income failed to sufficiently improve utility scores. In our view, the effect of income on utility scores does not only reflect wage loss. Our results suggest that the impact of double counting is negligible., (Copyright © 2013 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Objective: To evaluate the cost-effectiveness of adjuvant FOLFOX therapy versus 5-fluorouracil/leucovorin (FU/LV) for patients with stage III colorectal cancer., Methods: We performed the cost-effectiveness of FOLFOX compared with standard FU/LV treatment by the retrospective analysis of patient-level data from the randomized controlled Multicenter International Study of Oxaliplatin, 5-Fluorouracil, and Leucovorin in the Adjuvant Treatment of Colon Cancer (MOSAIC) trial. Predicted mean time spent in each disease state was calculated by our statistical model, which takes into account the cure rate and treats death from causes other than colon cancer as a competing risk. We performed this analysis from the perspective of the health-care payer. Using a time horizon of 30 years, both cost and effectiveness were discounted by 3% per year., Results: Estimated cure rates for colon cancer were 0.715 (FOLFOX) and 0.622 (FU/LV). Estimated medical costs of FOLFOX were JPY 3.1 million (USD 34,000) compared with JPY 1.9 million (USD 22,000) of FU/LV. The mean estimated quality-adjusted life-year was 9.83 with FOLFOX and 9.07 with that of FU/LV. The incremental cost-effectiveness ratio of FOLFOX was JPY 1.5 million (USD 17,000) per quality-adjusted life-year compared with FU/LV, which was supported by sensitivity analysis. Even if we assume that Japanese outcomes were better than those reported by the MOSAIC trial, which would reduce the difference between cure rates for each treatment to 5%, the incremental cost-effectiveness ratio remained below 5.0 million (USD 56,000) per quality-adjusted life-year., Conclusions: Adjuvant FOLFOX is a cost-effective treatment for stage III colon cancer in Japan compared with FU/LV therapy. Even when parameters were changed to reflect smaller improvements with FOLFOX, the conclusion is the same., (Copyright © 2012 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Objective: To examine health-related quality of life, we investigated the effect of adjuvant chemotherapy regimens on utility scores assessed by the EuroQoL-5D (EQ-5D) instrument in a randomized, controlled trial for breast cancer patients after surgery. We also investigated the relationship between Functional Assessment of Cancer Therapy (FACT) scale scores and EQ-5D utilities., Methods: Patients were randomly assigned to the following four chemotherapy regimens: four cycles of anthracycline followed by paclitaxel (ACP), four cycles of anthracycline-containing regimens followed by docetaxel (ACD), eight cycles of paclitaxel (PTX), and eight cycles of docetaxel (DTX). Of 1060 registered, the first 300 consecutive patients were included in the current utility study. Utility scores were assessed using the EQ-5D instrument at baseline; cycles 3, 5, and 7; 7 months; and 1 year. We also evaluated the correlation between these scores and FACT-G, -B, and -Taxane scores at each time point., Results: Utility scores were significantly lower in the DTX group than in the ACP and ACD groups. Mean utility scores in the DTX group were lowest at 7 months and tended to remain low for a long time. The combined anthracycline followed by taxane group had significantly higher utility scores that the taxane-alone group, with no significant difference depending on the type of taxane. Only the FACT-G social/family well-being subscale had no relationship with EQ-5D responses and utility scores., Conclusions: Although the regimens in this study were similar in that they included taxane, the mean utility scores and longitudinal patterns of utility scores were different among regimens., (Copyright © 2011 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)