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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. [ABSTRACT FROM AUTHOR]

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. 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%). 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. 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. • Although modern machine learning methods have led to improvements in many fields of medicine, they are rarely applied to mapping from a nonpreference-based measure onto health utility. • This study developed 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 using the gradient boosted tree, a promising modern machine learning method. • The gradient boosted tree approach did not improve the predictive performance measured by the root mean squared error and mean absolute error compared with regression approaches but had the potential to reduce overprediction and underprediction in poor and good health, respectively. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Purpose: In developed countries, progressive rapid aging is increasing the need for social care. This study aimed to determine Japanese utility weights for the Adult Social Care Outcomes Toolkit (ASCOT) four-level self-completion questionnaire (SCT4).Methods: We recruited 1050 Japanese respondents from the general population, stratified by sex and age, from five major cities. In the best-worst scaling (BWS) phase, respondents ranked various social care-related quality of life (SCRQoL) states as "best," "worst," "second-best," or "second-worst," as per the ASCOT. Then, respondents were asked to evaluate eight different SCRQOL states by composite time-trade off (cTTO). A mixed logit model was used to analyze BWS data. The association between cTTO and latent BWS scores was used to estimate a scoring formula that would convert BWS scores to SC-QALY (social care quality-adjusted life year) scores.Results: Japanese BWS weightings for ASCOT-SCT4 were successfully estimated and found generally consistent with the UK utility weights. However, coefficients on level 3 of "Control over daily life" and "Occupation" domains differed markedly between Japan and the UK. The worst Japanese SCRQoL state was lower than that for the UK, as Japanese cTTO results showed more negative valuations. In general, Japanese SC-QALY score (for more than 90% of health states) was lower than that for the UK.Conclusions: We successfully obtained Japanese utility weights for ASCOT SCT4. This will contribute to the measurement and understanding of social care outcomes. [ABSTRACT FROM AUTHOR]

Background: The population is aging rapidly in many developed countries. Such countries need to respond to the growing demand and expanding costs of healthcare (HC) for the elderly. Therefore, it is important to investigate the factors correlating such HC costs. In Japan, HC is composed of two sections, namely medical care (MC) and long-term care (LTC). While many studies have examined MC and LTC costs on their own, few studies have conducted comprehensive investigations of HC costs. The aim of this study is to examine the risk factors that influence HC costs for the elderly who enroll in the LTC insurance system in Japan. Methods: The inclusion criteria in the present study are as follows: being 65 years of age, or older; certified eligibility for, and use of services offered by the LTC insurance system at home or in an institutional setting in December 2009; and being covered by the National Health Insurance (NHI) system. MC and LTC insurance data were obtained from claim records for the elderly in July and December of 2007, 2008, and 2009 (i.e., a total of six survey points). Panel data, per subject, were constructed using MC and LTC claim records. The sample included 810 subjects and 4029 observations. Results: We estimated a regression equation with a censored dependent variable using a Tobit model. Significant associations between MC or LTC costs and interaction terms (household composition × seasonal effects) were investigated. MC costs significantly decreased and LTC costs significantly increased among subjects living alone during winter. Income level was also a positive determinant of MC costs, while eligibility level was a positive determinant of LTC costs. Conclusions: We recommend that the health policy for the elderly focus more on seasonal effects, household composition, and income level, as well as on eligibility level. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to investigate the impact of adverse events (AEs) on health utility and health-related quality of life (HRQOL) in patients with metastatic breast cancer undergoing first-line chemotherapy.Methods: We analyzed the data from the SELECT BC study, a multicenter, open-label, randomized, phase III study conducted in Japan, which compared first-line S-1 with taxane therapies. Heath utility and HRQOL were assessed using the EQ-5D-3L and European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) at baseline and 3, 6, and 12 months after treatment initiation. Health utility was calculated based on societal preferences, and AEs were reported at each cycle of the study treatment. Linear marginal mean models were used to quantify the impact of the last AEs (with 10 or more incidences) observed before HRQOL assessment on health utility and HRQOL.Results: Analysis included 380 patients and 12 (of 15) AEs. Grade 1 nausea and oral mucositis, grade 1 and 2 edema, and grade 2 fatigue, motor and sensory neuropathy, and myalgia were significantly associated with disutility, measured using the EQ-5D-3L. Grade 1 oral mucositis, grade 1 and 2 fatigue, and grade 2 sensory neuropathy were significantly associated with impaired global health status in the EORTC QLQ-C30. AEs associated with decrements in the five functioning scales included fatigue, oral mucositis, nausea, edema, motor and sensory neuropathy, and myalgia.Conclusions: We reported disutilities caused by AEs in patients with metastatic breast cancer under chemotherapy. These findings can be applied to future model-based cost-effectiveness analyses.Trial Registration Number: C000000416. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Purpose: The purpose of this study was to measure the population norms for the Japanese versions of preference-based measures (EQ-5D-3L, EQ-5D-5L, and SF-6D). We also considered the relations between QOL score in the general population and socio-demographic factors.Methods: A total of 1143 adult respondents (aged ≥ 20 years) were randomly sampled from across Japan using data from the Basic Resident Register. The health status of each respondent was measured using the EQ-5D-3L, EQ-5D-5L, and SF-6D, and responses regarding socio-demographic data as well as subjective diseases and symptoms were obtained. The responses were converted to a QOL score using Japanese value sets.Results: The percentages of respondents with full health scores were 68 % (EQ-5D-3L), 55 % (EQ-5D-5L), and 4 % (SF-6D). The QOL score measured using the SF-6D was significantly lower than those measured using either EQ-5D score. The QOL score was significantly lower among respondents over the age of 60 years, those who had a lower income, and those who had a shorter period of education. Intraclass correlation coefficient showed a poor agreement between the EQ-5D and SF-6D scores. The differences in QOL scores between respondents with and those without any disease were 0.064 for the EQ-5D-3L, 0.061 for the EQ-5D-5L, and 0.073 for the SF-6D; these differences are regarded as between-group minimal important differences in the general population.Conclusion: The Japanese population norms of three preference-based QOL measures were examined for the first time. Such information is useful for economic evaluations and research examining QOL score. [ABSTRACT FROM AUTHOR]

Abstract: 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 &y& Elsevier]

Abstract: 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 &y& Elsevier]

Abstract: 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 &y& Elsevier]

Background: Cetuximab, a monoclonal antibody directed against the epidermal growth factor receptor, improves progression-free survival and overall survival in patients with metastatic colorectal cancer (mCRC). However, patients with a KRAS gene mutation do not benefit from cetuximab therapy. Methods: We performed a cost-effectiveness analysis of KRAS testing and cetuximab treatment as last-line therapy for patients with mCRC in Japan. In our analysis, we considered three treatment strategies. In the 'KRAS-testing strategy' (strategy A), KRAS testing was performed to guide treatment: patients with wild-type KRAS received cetuximab, and those with mutant KRAS received best supportive care (BSC). In the 'no-KRAS-testing strategy' (strategy B), genetic testing was not conducted and all patients received cetuximab. In the 'no-cetuximab strategy' (strategy C), genetic testing was not conducted and all patients received BSC. To evaluate the cost effectiveness of KRAS testing, the KRAS-testing strategy was compared with the no-KRAS-testing strategy; to evaluate the cost effectiveness of KRAS testing and cetuximab, the KRAS-testing strategy was compared with the no-cetuximab strategy; and to evaluate the cost effectiveness of cetuximab treatment without KRAS testing, the no-KRAS-testing strategy was compared with the nocetuximab strategy. A three-state Markov model was used to predict expected costs and outcomes for each group. Outcomes in the model were based on those reported in a retrospective analysis of data from the National Cancer Institute of Canada Clinical Trials Group CO.17 study. We included only direct medical costs from the perspective of the Japanese healthcare payer. A 3% discount rate was used for both costs and outcome. Two outcomes, life-years (LYs) gained and quality-adjusted life-years (QALYs) gained, were used to calculate the incremental cost-effectiveness ratio (ICER). Results: Our cost-effectiveness analysis revealed that the KRAS-testing strategy was dominant compared with the no-KRAS-testing strategy, with an expected cost reduction of ¥0.5 million per patient and an estimated budget impact of ¥3-5 billion ($US42-59 million; July 2010 values) per year. The ICER of the KRAS-testing strategy compared with the no-cetuximab strategy was ¥11 million ($US120 000) per LY gained and ¥16 million ($US160 000) per QALY gained, whereas the ICER of the KRAS-testing strategy compared with the no-KRAS-testing strategy was ¥14 million ($US180 000) per LY gained and ¥21 million ($US230 000) per QALY gained. These results were supported by the sensitivity analysis. Conclusions: KRAS testing is recommended before administering cetuximab as last-line therapy for patients with mCRC. However, our analysis suggests that the ICER of cetuximab treatment (with or without KRAS testing) is too high, even if treatment is limited to patients with wild-type KRAS. [ABSTRACT FROM AUTHOR]

The purpose of our study was to estimate the out-of-pocket payment and cost-effectiveness of capecitabine plus oxaliplatin (XELOX) or XELOX plus bevacizumab from the perspective of patients with metastatic colorectal cancer (MCRC). Based on the NO16966 and NO16967 trials, the mean out-of-pocket payment was calculated from patient-level data. Out-of-pocket payments for 16 cycles (11 months) of first-line chemotherapy and 8 cycles (5 months) of second-line chemotherapy were included. In addition, incremental cost-effectiveness ratios (ICERs) for first-line bevacizumab were calculated by dividing the difference of the out-of-pocket payment by the difference of the mean number of progression-free survival (PFS) years or quality-adjusted PFS (QAPFS) years. The mean out-of-pocket payments for middle-income patients under 70 years of age were JPY 328,000 for 16 cycles of first-line XELOX and JPY 376,000 for XELOX plus bevacizumab. The mean out-of-pocket payment for 8 cycles of second-line XELOX was calculated to be JPY 175,000. Middle-income patients over 70 years of age were required to pay JPY 61,000 and JPY 72,000 for first-line XELOX and XELOX plus bevacizumab, respectively. The ICERs of middle-income patients <70 years of age were JPY 430,000/PFS-year and JPY 720,000/QAPFS-year, and those of middle-income patients >70 years of age were JPY 100,000/PFS-year and JPY 170,000/QAPFS-year. We clarified the out-of-pocket payment and cost-effectiveness of chemotherapy of MCRC patients in Japan. Our previous survey shows it is highly possible that many patients prefer to pay that incremental out-of-pocket payment to gain one additional QAPFS year. However, our cost-effectiveness analysis was not conducted from the perspective of society or healthcare payers. [ABSTRACT FROM AUTHOR]

We measured health utility scores of colorectal cancer (CRC) patients from a societal perspective in Japan. Twenty-five states of health were described: four metastatic states without severe adverse events (AEs), 16 metastatic states with Grade 3/4 AEs, four adjuvant states, and one terminal state. A total of 1,500 respondents stratified by age and gender were recruited randomly from the largest Web-panel in Japan. Respondents were allocated randomly to three of the 25 health states and answered questionnaires by standard gamble (SG) and time trade-off (TTO) methods. Although utility scores of metastatic CRC receiving XELOX (capecitabine plus oxaliplatin) chemotherapy were 0.48(SG and TTO) (with stoma) and 0.57(SG) or 0.59(TTO) (without stoma), utility scores of those receiving FOLFOX4 (5-fluorouracil/folinic acid and oxaliplatin) chemotherapy were 0.42(SG) or 0.43(TTO) (with stoma) and 0.52(SG) or 0.53(TTO) (without stoma). These differences between XELOX and FOLFOX4 were statistically significant ( P = 0.0198 in SG and P = 0.0059 in TTO). Stage 3/4 AEs decreased utility scores to 0.35–0.4 and 0.4–0.45 in the presence and absence of stoma, respectively. XELOX was generally considered a significantly preferable chemotherapy regimen compared to FOLFOX4 for CRC in Japan. Almost all Grade 3/4 AEs and stoma significantly decreased utility scores. These differences are dependent on the accuracy of the health state description and to confirm these results. In future research, it would be preferable that preference-based HRQoL measures are used directly in patients if utility scores are practically measurable by such method. [ABSTRACT FROM AUTHOR]

Objective: A cost-effectiveness analysis of oral capecitabine versus intravenous bolus 5-fluorouracil/l-leucovorin (FU/LV) as adjuvant therapy in patients with stage 3 colon cancer was performed from a Japanese healthcare payer perspective. Methods: Adjuvant therapy comprised 24 weeks of treatment with either oral capecitabine 1250 mg/m² twice daily on days 1-14 of a 21-day cycle or intravenous bolus FU 500mg/m² and LV 250mg/m² weekly for 6 weeks of an 8-week cycle (Roswell Park regimen). The analysis comprised short-term (1 year after initiation of adjuvant therapy) and long-term (up to 15 years) components. The long-term analysis involved a three-state (disease-free, recurrence and death) Markov model. Estimates for transition probabilities, costs and utilities were derived from the X-ACT trial, a Japanese phase II trial, and other published sources. Cost estimates were considered from the perspective of a healthcare payer. Costs were expressed in Japanese Yen (f), year 2007 values. A discount rate of 3% was applied to costs and outcomes. Cost effectiveness was expressed as a cost per QALY. The effects of uncertainty were explored through one-way and probabilistic sensitivity analyses. Results: In the 1-year analysis, direct costs were f440 000 ($US4000) less per patient with capecitabine than with FU/LV. In the long-term analysis, differences between treatments in direct medical costs ranged from f470 000 ($US4300) to f580 000 ($US5300) depending on the time horizon used. Capecitabine was also projected to increase the number of QALYs compared with FU/LV. The sensitivity analysis suggested that the model outcome was robust. The probabilistic sensitivity analysis estimate of capecitabine being the dominant regimen was 96.6% at a zero willingness to pay. Direct costs remained lower with capecitabine if the price of generic LV was ⩾50% of the branded product. Conclusion: This analysis suggests that capecitabine improves health outcomes and lowers direct costs compared with bolus FU/LV (i.e. dominant treatment strategy) when used as adjuvant therapy in patients with stage 3 colon cancer in Japan. [ABSTRACT FROM AUTHOR]

Abstract: Background: Rapid progress has been made in the treatment of metastatic colorectal cancer (mCRC). New treatment regimens for mCRC include not only cytotoxic chemotherapy but also targeted monoclonal antibodies, including bevacizumab. However, bevacizumab is an expensive medication, which costs from ¥300,000 to ¥400,000 (US $2500–$3300) per month. Objective: The purpose of this cost-effectiveness analysis was to examine the economic efficiency of treating mCRC with bevacizumab plus chemotherapy versus chemotherapy alone in Japan. Methods: We searched an electronic database (MEDLINE, UpToDate, and American Society of Clinical Oncology [ASCO] Virtual Meeting; key terms: bevacizumab limited to randomized controlled trial; years: 2000 to present [June 29, 2007]) to detect randomized controlled trials (RCTs) that compared chemotherapy alone with chemotherapy plus bevacizumab. To analyze the cost-effectiveness of bevacizumab, we used the Weibull regression model and determined an expected treatment duration at each state using reported survival curves of RCTs. We included only the direct medical costs (2006) of these medications to estimate the expected values of incremental costs; thus, the analysis was conducted from the perspective of the health care payer. The incremental cost-effectiveness ratios (ICERs) were calculated from these expected values of incremental life-years and incremental costs. Results: We identified 5 articles using MEDLINE and 1 trial found on UpToDate and ASCO Virtual Meeting; these data composed the final analysis group. First-line chemotherapy regimens included in this analysis were bevacizumab + 5-fluorouracil/leucovorin (FU/LV), irinotecan/FU/LV (IFL), infusional FU/LV/ oxaliplatin (FOLFOX6), bolus FU/LV/oxaliplatin (bFOL), and capecitabine/oxaliplatin (CAPOX). The only second-line chemotherapy regimen included was FOLFOX4. The ICERs of additional bevacizumab when combined with FU/LV,IFL,FOLFOX6, bFOL, and CAPOX were ¥17.4 million (US $145,000), ¥11.9 million ($99,000), ¥13.5 million ($113,000), ¥16.9 million ($141,000), and ¥8.5 million ($71,000), respectively, per life-year gained; the ICER was ¥14.1 million ($118,000) with second-line FOLFOX4. Conclusions: In this cost-effectiveness analysis in Japan, the ICERs of bevacizumab + FU/LV combination treatment, IFL, and second-line FOLFOX4 were high compared with other chemotherapies for mCRC. It remains difficult to assess first-line therapies comprising bevacizumab with oxaliplatin-based regimens, especially CAPOX. Further information is needed to assess cost-effectiveness. [Copyright &y& Elsevier]

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. [ABSTRACT FROM AUTHOR]

Background: To develop direct and indirect (response) mapping algorithms from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and the Functional Assessment of Cancer Therapy General (FACT-G) onto the EQ-5D-5L index.Methods: We conducted the QOL-MAC study where EQ-5D-5L, EORTC QLQ-C30, and FACT-G were cross-sectionally evaluated in patients receiving drug treatment for solid tumors in Japan. We developed direct and indirect mapping algorithms using 7 regression methods. Direct mapping was based on the Japanese value set. We evaluated the predictive performances based on root mean squared error (RMSE), mean absolute error, and correlation between the observed and predicted EQ-5D-5L indexes.Results: Based on data from 903 and 908 patients for EORTC QLQ-C30 and FACT-G, respectively, we recommend two-part beta regression for direct mapping and ordinal logistic regression for indirect mapping for both EORTC QLQ-C30 and FACT-G. Cross-validated RMSE were 0.101 in the two methods for EORTC QLQ-C30, whereas they were 0.121 in two-part beta regression and 0.120 in ordinal logistic regression for FACT-G. The mean EQ-5D-5L index and cumulative distribution function simulated from the recommended mapping algorithms generally matched with the observed ones except for very good health (both source measures) and poor health (only FACT-G).Conclusions: The developed mapping algorithms can be used to generate the EQ-5D-5L index from EORTC QLQ-C30 or FACT-G in cost-effectiveness analyses, whose predictive performance would be similar to or better than those of previous algorithms. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the effects of S-1, an orally administered 5-FU agent, versus taxane on patient-reported outcomes (PROs) in the SELECT BC trial. Methods: Patients with HER2-negative and endocrine treatment-resistant breast cancer with metastasis or recurrence after surgery were randomly assigned to receive first-line taxane or S-1. PROs (secondary endpoint) were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and Patient Neurotoxicity Questionnaire (PNQ) at baseline and at 3, 6, and 12 months. We conducted a responder analysis for the QLQ-C30 and PNQ and created cumulative distribution function (CDF) plots as a sensitivity analysis. Results: The questionnaire response rates were over 80% from 386 patients, who completed at least one baseline questionnaire. S-1 was significantly superior to taxane with respect to 6 scales (physical functioning [p = 0.03], role functioning [p = 0.04], social functioning [p < 0.01], financial difficulties [p = 0.01], global health status [p = 0.02], and constipation [p < 0.01]) and sensory neuropathy (p = 0.01). The CDF plots partially supported the conclusions and their robustness. Conclusion: First-line S-1 therapy has clinical benefits with respect to many aspects of health-related quality of life for metastatic breast cancer patients. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Background: Inaccurate estimates of diabetes-related healthcare costs can undermine the efficiency of resource allocation for diabetes care. The quantification of these costs using claims data may be affected by the method for defining diagnoses.Objectives: The aims were to use panel data analysis to estimate diabetes-related healthcare costs and to comparatively evaluate the effects of diagnostic definitions on cost estimates.Research Design: Monthly panel data analysis of Japanese claims data.Subjects: The study included a maximum of 141,673 patients with type 2 diabetes who received treatment between 2005 and 2013.Measures: Additional healthcare costs associated with diabetes and diabetes-related complications were estimated for various diagnostic definition methods using fixed-effects panel data regression models.Results: The average follow-up period per patient ranged from 49.4 to 52.3 months. The number of patients identified as having type 2 diabetes varied widely among the diagnostic definition methods, ranging from 14,743 patients to 141,673 patients. The fixed-effects models showed that the additional costs per patient per month associated with diabetes ranged from US$180 [95 % confidence interval (CI) 178-181] to US$223 (95 % CI 221-224). When the diagnostic definition excluded rule-out diagnoses, the diabetes-related complications associated with higher additional healthcare costs were ischemic heart disease with surgery (US$13,595; 95 % CI 13,568-13,622), neuropathy/extremity disease with surgery (US$4594; 95 % CI 3979-5208), and diabetic nephropathy with dialysis (US$3689; 95 % CI 3667-3711).Conclusions: Diabetes-related healthcare costs are sensitive to diagnostic definition methods. Determining appropriate diagnostic definitions can further advance healthcare cost research for diabetes and its applications in healthcare policies. [ABSTRACT FROM AUTHOR]

Objectives The combination use of gefitinib and epidermal growth factor receptor (EGFR) testing is a standard first-line therapy for patients with non-small cell lung cancer (NSCLC). Here, we examined the cost-effectiveness of this approach in Japan. Materials and methods Our analysis compared the ‘EGFR testing strategy’, in which EGFR mutation testing was performed before treatment and patients with EGFR mutations received gefitinib while those without mutations received standard chemotherapy, to the ‘no-testing strategy,’ in which genetic testing was not conducted and all patients were treated with standard chemotherapy. A three-state Markov model was constructed to predict expected costs and outcomes for each strategy. We included only direct medical costs from the healthcare payer's perspective. Outcomes in the model were based on those reported in the Iressa Pan-Asia Study (IPASS). The incremental cost-effectiveness ratio (ICER) was calculated using quality-adjusted life-years (QALYs) gained. Sensitivity and scenario analyses were conducted. Results The incremental cost and effectiveness per patient of the ‘EGFR testing strategy’ compared to the ‘no-testing strategy’ was estimated to be approximately JP¥122,000 (US$1180; US$1 = JP¥104 as of February 2014) and 0.036 QALYs. The ICER was then calculated to be around JP¥3.38 million (US$32,500) per QALY gained. These results suggest that the ‘EGFR testing strategy’ is cost-effective compared with the ‘no-testing strategy’ when JP¥5.0 million to 6.0 million per QALY gained is considered an acceptable threshold. These results were supported by the sensitivity and scenario analyses. Conclusion The combination use of gefitinib and EGFR testing can be considered a cost-effective first-line therapy compared to chemotherapy such as carboplatin–paclitaxel for the treatment for NSCLC in Japan. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Background: Task shifting and task sharing in health care are rapidly becoming more common as the shortage of physicians increases. However, research has not yet examined the changing roles of hospital administrative staff. This study clarified: (1) the adverse incidents caused by hospital administrative staff, and the direct and indirect impact of these incidents on patient care; and (2) the incidents that directly involved hospital administrative staff.Methods: This study used case report data from the Japan Council for Quality Health care collected from April 1, 2010 to March 31, 2019, including a total of 30,823 reports. In April 2020, only the 88 self-reported incidents by hospital administrative staff were downloaded, excluding incidents reported by those in medical and co-medical occupations. Data from three reports implicating pharmacists were rejected and the quantitative and textual data from the remaining 85 case reports were analyzed in terms of whether they impacted patient care directly or indirectly.Results: Thirty-nine reports (45.9%) involved direct impact on patient care, while 46 (54.1%) involved indirect impact on patient care. Most incidents that directly impacted patient care involved administrative staff writing prescriptions on behalf of a doctor (n = 24, 61.5%); followed by errors related to system administration, information, and documentation (n = 7, 17.9%). Most reported errors that indirectly affected patient care were related to system administration, information, and documentation used by administrative staff (n = 22, 47.8%), or to reception (n = 9, 19.6%). Almost all errors occurred during weekdays. Most frequent incidents involved outpatients (n = 23, 27.1%), or occurred next to examination/operation rooms (n = 12, 14.1%). Further, a total of 14 cases (16.5%) involved patient misidentification.Conclusions: Incidents involving hospital administrative staff, the most common of which are medication errors from incorrect prescriptions, can lead to severe consequences for patients. Given that administrative staff now form a part of medical treatment teams, improvements in patient care may require further submission and review of incident reports involving administrative staff. [ABSTRACT FROM AUTHOR]

Background: The EuroQol 5-dimension scale (EQ-5D) is one of the most frequently used preference-based quality of life (QOL) measures for health technology assessment. The 3-level version of the EQ-5D comprises a descriptive system (the EQ-5D-3L) and a visual analog scale (EQ-VAS). It remains unclear whether this five-item scale correlates with the QOL of patients with oral cancer during the perioperative period. We sought to clarify this point in the present study. Methods: Participants were 84 patients with oral malignancies who underwent radical treatment and completed the EQ-5D-3L and Functional Assessment of Cancer Therapy-Head and Neck (FACT-H&N) at regular intervals over 3 months after treatment. We analyzed the correlations between the EQ-5D-3L, EQ-VAS, and FACT-H&N, and conducted multiple regression analyses to examine how the FACT-H&N subscales relate to the EQ-5D-3L and EQ-VAS. We also investigated whether the EQ-5D-3L shows ceiling effects. Results: The EQ-5D-3L and EQ-VAS were strongly correlated with the FACT-H&N (rs = 0.621 and 0.638, respectively; P < 0.01). Furthermore, the EQ-5D-3L was significantly related with all FACT-H&N subscales except for social/family well-being. Particularly, the physical well-being subscale had the strongest relationship with the EQ-5D-3L. The FACT H&N and EQ-5D-3L showed similar changes over time. The EQ-5D-3L did not have a ceiling effect statistically. Conclusions: Our results indicate that actual physical performance might be most important for cost–utility analysis, whereas the assessment of familial feelings or friendship seems less important. However, the EQ-5D-3L appears to generally correlate with the FACT-H&N of patients with oral cancer during the perioperative period. Therefore, it is reasonable to assess the cost performance of oral cancer treatment using the EQ-5D-3L in Japan. [ABSTRACT FROM AUTHOR]

Background: This study evaluated the cost-effectiveness of replacing standard intravenous therapy (taxane) with oral S-1 therapy for first-line metastatic breast cancer treatment.Methods: This cost-effectiveness analysis was based on data from a randomized phase III trial (SELECT BC). As cost-effectiveness was a secondary endpoint of the SELECT BC trial, some of the randomized patients participated in an EQ-5D survey (N = 391) and health economic survey (N = 146). The EQ-5D responses, claims, and prescription data were collected for as long as possible until death. The expected quality-adjusted life years (QALY) obtained from each treatment were calculated using patient-level EQ-5D data, and the expected cost was calculated using patient-level claim data. The analysis was performed from the perspective of public healthcare payers.Results: The estimated EQ-5D least-square means and 95% CI up to 48 months were 0.764 (95% CI, 0.741-0.782) and 0.742 (95% CI, 0.720-0.764) in the S-1 and taxane arms, respectively. The expected QALY was 2.11 for the S-1 arm and 2.04 for the taxane arm, with expected costs of JPY 5.13 million (USD 46,600) and JPY 5.56 million (USD 50,500), respectively. These results show that S-1 is cost-saving. According to probabilistic sensitivity analysis, S-1 was dominant with a probability of 63%. When the willingness to pay (WTP) value was JPY 5 million (USD 45,500) per QALY, the probability of being cost-effective was 92%.Conclusions: Our results show that the introduction of oral S-1 therapy for metastatic breast cancer is highly likely to be cost-effective.Trial Registration: UMIN CTR C000000416 . Registered on May 10, 2006. [ABSTRACT FROM AUTHOR]

Background: The aim of this study was to develop and perform cross-cultural validation of a Japanese version of the Adult Social Care Outcomes Toolkit (ASCOT) four-level Self-Completion questionnaire (SCT4) instrument to measure Social-Care Related Quality of Life. It was important to develop a Japanese version of the ASCOT-SCT4 and validate it in the Japanese context, given the interest in measuring outcomes of social care services in Japan.Methods: The original version of ASCOT-SCT4 was translated into Japanese following good practice guidelines. Additionally, comments and feedback were obtained from an independent committee engaged in managing and providing social care services to refine the flow of sentences of the newly developed translated version. The resulting version was tested for cross-cultural validation among community-dwelling adults who use social care services to confirm the factorial structure and the scale system of the Japanese version, using Structural Equation Modeling and Item Response Theory.Results: Vigorous discussion was needed to translate the original version into Japanese especially for the items control over daily life and dignity. These two items were linguistically difficult to express in everyday language so potential participants could easily understand the intended concepts. In the cross-cultural validation, we obtained values for model fit within the acceptable range: between 0.706 and 0.550 for factor loadings, 0.923 for the Comparative Fit Index, 0.910 for the Tucker-Lewis Index, and 0.083 for the Root Mean Square Error of Approximation. This confirmed the factorial structure of the Japanese version. The IRT analysis, however, revealed that the scale system needed refinement to facilitate appropriate differentiation between each response option.Conclusions: This study provided preliminary evidence that the Japanese version of ASCOT-SCT4 is valid. As a result, the Japanese version was finalized and approved by the instrument developer. [ABSTRACT FROM AUTHOR]