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.

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.

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.

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.

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.

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.

Prospective observational study estimating willingness-to-pay for breast cancer treatments through contingent valuation method in Japanese breast cancer patients (JCOG1709A).

In April 2016, the Japanese government introduced health technology assessment as a response to rising medical expenses due to 'medical innovation'. This study investigates how Japanese breast cancer patients who received treatment in Japan consider the financial value (willingness-to-pay; WTP) for their life and health by using the contingent valuation method (CVM) prospectively. First, 168 patients (84 primary breast cancer patients and 84 metastatic breast cancer patients) were pre-examined their WTP with dichotomous-choice method survey form. Next, 1,596 patients (798 primary breast cancer patients and 798 metastatic breast cancer patients) will be surveyed to their WTP for hypothetical scenarios in CVM. Based on our results, we will construct an evaluation axis from the patients' viewpoint for the cost-effectiveness of clinical trials to establish standard treatments for breast cancer. We believe this research can contribute to create a meaningful healthcare system for patients, clinicians, industries, and healthcare policymakers.

What Is a Valid Mapping Algorithm in Cost-Utility Analyses? A Response From a Missing Data Perspective.

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.

Formal Implementation of Cost-Effectiveness Evaluations in Japan: A Unique Health Technology Assessment System.

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.

Response Shift-Adjusted Treatment Effect on Health-Related Quality of Life in a Randomized Controlled Trial of Taxane Versus S-1 for Metastatic Breast Cancer: Structural Equation Modeling.

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.

Mapping EORTC QLQ-C30 and FACT-G onto EQ-5D-5L index for patients with cancer.

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.

Development of Japanese utility weights for the Adult Social Care Outcomes Toolkit (ASCOT) SCT4.

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.

Incident reports involving hospital administrative staff: analysis of data from the Japan Council for Quality Health care nationwide database.

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.

Cost-effectiveness evaluation for pricing medicines and devices: A new value-based price adjustment system in Japan.

OBJECTIVES: In Japan, a new cost-effectiveness evaluation system for medicine and medical device pricing was employed in April 2019 after a trial implementation. This study describes the discussions held from April 2016 to March 2019 concerning the newly introduced system. METHODS: Using published government documents, discussions with stakeholders, and the minutes of the Chuikyo committee meetings, the following issues are addressed: (i) the results of the trial implementation and (ii) an overview of the newly introduced system. RESULTS: During the trial implementation, thirteen products were evaluated and their prices adjusted. The process of the new system-which was to be implemented in FY 2019-takes about 15-18 months to complete after listing of the target products by the National Health Insurance. The target products are selected principally based on sales volume, degree of innovation (premium), and disclosure of rationale for price setting. First, a manufacturer submits the cost-effectiveness data, which is then reviewed by the Center for Outcomes Research and Economic Evaluation for Health (C2H) in collaboration with academics. The results of the cost-effectiveness evaluation are not considered during the decision-making process concerning the product's listing. The price adjustment system is similar to value-based pricing (VBP); hence, the new system can be considered as VBP adjustment. CONCLUSION: Cost-effectiveness evaluation can help promote both technological innovation and sustainability of the healthcare system. We need to create a greater capacity for enhancing this academic review system.

Japanese translation and cross-cultural validation of the Adult Social Care Outcomes Toolkit (ASCOT) in Japanese social service users.

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.

To what extent does the EQ-5D-3L correlate with the FACT-H&N of patients with oral cancer during the perioperative period?

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.

Exploring the Application of Cost-Effectiveness Evaluation in the Japanese National Health Insurance System.

OBJECTIVES: Advances in health care due to the development and introduction of new drugs and medical devices have brought considerable benefits to people and patients in terms of upgraded quality of life and extended years of survival. However, some are concerned that the very advancement of health care would increase further the inflation of national healthcare costs. In response to these concerns, Japan's Central Social Insurance Medical Council ("Chuikyo") began in 2012 to examine how cost-effectiveness evaluation might be applied to the national health insurance system, and has been working toward establishing a system for its usage. METHODS: Cost-effectiveness evaluation was adopted on a trial basis in fiscal year (FY) 2016, targeting seven drugs and six medical devices. Analyses and re-analyses were performed by manufacturers and a public expert organization, respectively. Based on these analyses, a cost-effectiveness evaluation expert organization conducted an overall assessment ("appraisal"). Results of the evaluation were used to adjust the prices of the target items. RESULTS: Following the trial adoption of cost-effectiveness evaluation, price adjustments were performed for three items in April 2018. Meanwhile, a decision was also made to examine seven items for which technical requirements were identified due to differences in the understanding of analysis methods between involved parties. CONCLUSIONS: The Chuikyo will examine how to meet the newly identified technical requirements and discuss specific details with regard to establishing a system that incorporates cost-effectiveness evaluation. The Chuikyo plans to reach a conclusion by the end of FY 2018.

Patient-Reported Outcome Results from the Open-Label Randomized Phase III SELECT BC Trial Evaluating First-Line S-1 Therapy for Metastatic Breast Cancer.

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.