Sinaï Francisco Dias MSc , Brian Asare MSc , Lumbwe Chola PhD
{"title":"Cost-Effectiveness of Hydroxyurea for Treatment of Children With Sickle Cell Anemia in Ghana","authors":"Sinaï Francisco Dias MSc , Brian Asare MSc , Lumbwe Chola PhD","doi":"10.1016/j.vhri.2025.101586","DOIUrl":"10.1016/j.vhri.2025.101586","url":null,"abstract":"<div><h3>Objectives</h3><div>Most African children born with sickle cell disease (SCD) will die before their fifth birthday. Hydroxyurea has proven therapeutic benefits for SCD yet remains inaccessible to many children in low- and middle-income countries. To inform policy on coverage and reimbursement, this study evaluates the cost-effectiveness of hydroxyurea for pediatric SCD in Ghana, compared with the standard of care.</div></div><div><h3>Methods</h3><div>We developed a Markov model of SCD progression, incorporating costs and effects from a Ghanaian payer perspective, over a 17-year time horizon. Outcomes were life-years gained and disability-adjusted life-years averted. Cost-effectiveness was determined using the World Health Organization willingness-to-pay threshold of 1 times the per-capita gross domestic product and an alternative country-specific threshold based on health opportunity cost ($580.52). One-way and probabilistic sensitivity analyses were undertaken. Costs and effects were discounted at 3% per annum.</div></div><div><h3>Results</h3><div>In the base case, the incremental cost-effectiveness ratio was $1440.34 per disability-adjusted life-year averted and $1823.89 per life-year gained. The intervention was potentially cost-effective using the World Health Organization threshold but not cost-effective using the alternative threshold. The 1-way sensitivity analysis highlighted the incremental cost-effectiveness ratio’s sensitivity to price changes, emphasizing the importance of price negotiations.</div></div><div><h3>Conclusions</h3><div>Hydroxyurea can provide substantial health benefits for Ghanaian children with SCD. The results provide a strong argument for the incorporation of hydroxyurea into the Ghana national health insurance benefits package. Future research should address the disparity in threshold outcomes and the need for country-specific data on the long-term effects and broader economic impact of hydroxyurea in Ghana.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"55 ","pages":"Article 101586"},"PeriodicalIF":1.5,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145982111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"How Can Mortality Trajectory Analysis Improve Health Policy and Research in Southeast Asia?","authors":"Jeff Clyde G. Corpuz PhD","doi":"10.1016/j.vhri.2025.101583","DOIUrl":"10.1016/j.vhri.2025.101583","url":null,"abstract":"","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101583"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145925614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-Utility and Budget Impact Analysis of Pharmacogenetic-Guided Antiplatelet Therapy for Acute Coronary Syndrome in Thailand","authors":"Nathapol Samprasit MPharm , Nattanichcha Kulthanachairojana PhD , Chamipa Phanudulkitti PhD , Jatapat Hemapanpairoa PharmD","doi":"10.1016/j.vhri.2025.101565","DOIUrl":"10.1016/j.vhri.2025.101565","url":null,"abstract":"<div><h3>Objectives</h3><div>Pharmacogenetic (PGx) testing for CYP2C19 genotypes offers a precision medicine approach to dual antiplatelet therapy in patients with acute coronary syndrome (ACS) undergoing percutaneous coronary intervention (PCI). PGx testing is limited in Thailand because of policy constraints. This study evaluated the cost-utility and budget impact of PGx-guided dual antiplatelet therapy compared with universal clopidogrel in Thailand.</div></div><div><h3>Methods</h3><div>A hybrid decision tree and Markov model were developed to estimate lifetime costs and health outcomes for patients with post-PCI ACS from a societal perspective. The model compared universal clopidogrel with 2 PGx-guided strategies for CYP2C19 loss-of-function allele carriers. Key outcomes included life-years and quality-adjusted life-years (QALYs) gained. One-way and probabilistic sensitivity analyses were conducted to assess model uncertainty. A 5-year budget impact analysis was performed from a payer perspective.</div></div><div><h3>Results</h3><div>Compared with universal clopidogrel, the PGx-guided ticagrelor strategy was dominant with lower costs and higher QALYs. The PGx-guided prasugrel strategy exceeded the willingness-to-pay threshold, with an incremental cost-effectiveness ratio of 247 604 Thai Baht (THB)/QALY. Probabilistic sensitivity analyses indicated a 99.9% probability that the ticagrelor strategy would be a cost-effective strategy at the Thai willingness-to-pay threshold. The 5-year budget impact analysis, assuming 100% access to PGx testing, estimated a budget saving of 240.54 million THB for ticagrelor. In contrast, prasugrel was associated with an additional requirement of 1520.89 million THB.</div></div><div><h3>Conclusions</h3><div>In Thailand’s healthcare setting, PGx-guided ticagrelor is a dominant strategy for patients with post-PCI ACS. These findings support revising the National List of Essential Medicines to incorporate genotype-guided prescribing and enable broader access to precision medicine.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101565"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145834810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hanna Gyllensten PhD , Gunnar Bergström PhD , Sara Freyland MSc , Elisabeth Björk Brämberg PhD
{"title":"Economic Evaluation of a Problem-Solving Intervention With Workplace Involvement Among Employees With Common Mental Disorder in Swedish Primary Care: Results From a Cluster-Randomized Controlled Trial","authors":"Hanna Gyllensten PhD , Gunnar Bergström PhD , Sara Freyland MSc , Elisabeth Björk Brämberg PhD","doi":"10.1016/j.vhri.2025.101558","DOIUrl":"10.1016/j.vhri.2025.101558","url":null,"abstract":"<div><h3>Objectives</h3><div>The aim of this study was to evaluate the cost-effectiveness of a problem-solving intervention with workplace involvement (PSI-WPI) compared with care as usual (CAU) among employees on sick leave due to common mental disorders in Swedish primary healthcare centers.</div></div><div><h3>Methods</h3><div>Employees aged 18 to 59 years on sick leave for common mental disorders (<em>n</em> = 197) were included in a controlled cluster-randomized trial by coordinators (<em>n</em> = 19) recruited at primary care centers in Region Västra Götaland, Sweden. The study was conducted between February 2018 and August 2021, with 18 months of follow-up per participant. The economic evaluation included a cost-effectiveness analysis comparing changes in sick leave with direct costs from a healthcare perspective, calculated from register data, and intervention costs; a cost-utility analysis based on EQ-5D questionnaires and a societal perspective included indirect costs calculated from registered sick leave. Sensitivity analyses explored robustness to alternative missingness assumptions.</div></div><div><h3>Results</h3><div>The PSI-WPI resulted in more sickness absence (average 40 extra days; 95% confidence interval, 3-77 days) compared with CAU and higher healthcare costs (SEK 21 650, SEK 4962-48 262) over 18 months. At 12 months, the cost of care was SEK 23 734 (SEK 537-46 931) higher in the PSI-WPI group. The difference in quality-adjusted life-years between the groups was negligible. The sensitivity analysis indicated robust results, and diagnoses not targeted by the study were important cost drivers.</div></div><div><h3>Conclusions</h3><div>The PSI-WPI was not cost-effective for employees on sick leave due to common mental disorders compared with CAU only.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101558"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145775945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Florian Naye PhD , Maxime Sasseville PhD , Karine Toupin-April PhD , France Légaré PhD , Chloé Cachinho MSc , Thomas Gérard MSc , Valentin Vaillant MSc , Alison M. Hoens MSc , Yannick Tousignant-Laflamme PhD , Simon Décary PhD
{"title":"Measurement Properties and Cross-Cultural Invariance of the French Decisional Conflict Scale in Chronic Pain: Secondary Analysis of the DECIDE-PAIN Survey","authors":"Florian Naye PhD , Maxime Sasseville PhD , Karine Toupin-April PhD , France Légaré PhD , Chloé Cachinho MSc , Thomas Gérard MSc , Valentin Vaillant MSc , Alison M. Hoens MSc , Yannick Tousignant-Laflamme PhD , Simon Décary PhD","doi":"10.1016/j.vhri.2025.101551","DOIUrl":"10.1016/j.vhri.2025.101551","url":null,"abstract":"<div><h3>Objectives</h3><div>Evidence on the validity of the French version of the Decisional Conflict Scale (DCS) remains limited, especially among people living with chronic noncancer pain in Canada. This study examined the measurement properties of the French 16-item DCS and assessed its cross-cultural measurement invariance in this population.</div></div><div><h3>Methods</h3><div>We conducted a secondary analysis of a pan-Canadian cross-sectional online survey following the Consensus-Based Standards for the Selection of Health Measurement Instruments guidance. We evaluated score distribution, including ceiling and floor effects. We assessed readability, internal consistency, structural validity of a hypothesized 5-factor model, and convergent and discriminant validities. We tested measurement invariance between the French and English versions.</div></div><div><h3>Results</h3><div>We analyzed data from 270 French-speaking Canadians (mean age = 51; 60% male; 54.8% with less than a university diploma; and 70.4% with limited health literacy). The French DCS showed good readability (Flesch-Kincaid Grade Level: 5.6), no ceiling or floor effects for the total score, and high internal consistency (ω = 0.95). The 5-factor structure was supported (CFI = 0.98). Convergent validity was confirmed (AVE: 0.61-0.72), but discriminant validity was poor (AVE < squared interfactor correlations). Measurement invariance was supported at configural, metric, scalar, and strict levels when compared with 985 English-speaking respondents (mean age = 51; 47.5% male; 50% with less than a university diploma; and 71.3% with limited health literacy).</div></div><div><h3>Conclusions</h3><div>The French 16-item DCS has acceptable measurement properties and cross-cultural measurement invariance in people living with chronic noncancer pain. Further research is necessary to enhance its validity, possibly by reexamining its factor structure.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101551"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145617555","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-Utility Analysis of Adjuvant Osimertinib in Resected Epidermal-Growth-Factor-Receptor-Mutated Early-Stage Non-Small Cell Lung Cancer","authors":"Supitchaya Changsatja PharmD, MA , Chayanis Kositamongkol PharmD, MSc , Lucksamon Thamlikitkul MD, PhD , Pochamana Phisalprapa MD, PhD , Touchanun Komonpaisarn PhD","doi":"10.1016/j.vhri.2025.101567","DOIUrl":"10.1016/j.vhri.2025.101567","url":null,"abstract":"<div><h3>Objectives</h3><div>Lung cancer is the most common cancer and the leading cause of cancer death worldwide. One-third of non-small cell lung cancer (NSCLC) patients are diagnosed at a resectable stage, for which surgery is the standard curative treatment. However, most patients recur within 2 years. Adjuvant osimertinib, a third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor, significantly reduces recurrence and prolongs survival in completely resected EGFR-mutated NSCLC. This study assessed the cost-utility and budget impact of adjuvant osimertinib versus placebo for resected stage IB-IIIA EGFR-mutated NSCLC from Thailand’s societal perspective.</div></div><div><h3>Methods</h3><div>A Markov model with a lifetime horizon and 4-week cycle was developed, including 3 health states: disease-free, recurrent disease, and death. Patients received osimertinib or placebo in the disease-free state, with recurrence categorized as locoregional or distant. Subsequent treatments were modeled based on recurrence timing. Health outcomes were obtained from a systematic review, and costs followed Thai health technology assessment guidelines. Results were reported as incremental cost-effectiveness ratios in 2023 USD per quality-adjusted life-years (QALY) gained. Sensitivity analyses addressed uncertainty.</div></div><div><h3>Results</h3><div>Osimertinib yielded 2.36 additional QALYs at an incremental cost of USD 62 604.90, with an incremental cost-effectiveness ratio of USD 26 474.02/QALY, exceeding Thailand’s willingness-to-pay threshold (USD 4619). An 85.07% price reduction is required for cost-effectiveness. Probabilistic sensitivity analysis showed 0% probability of cost-effectiveness at the current threshold, increasing to 52% at USD 25 981.97/QALY. The 5-year budget impact analysis was estimated at USD 678.73 million.</div></div><div><h3>Conclusions</h3><div>Adjuvant osimertinib is not cost-effective in Thailand under current pricing but could be with major price reductions or risk-sharing strategies.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101567"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145840089","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Estimation of Health Utility Values for Health States in Head and Neck Cancers Using Real-World Data","authors":"Krishnakumar Thankappan MCh , Shivali Kamath PharmD , Lakshmi Ravunniarth Menon MCh , Tejal Patel MCh , Deepak Balasubramanian MCh , Manu Raj DNB , Moni Abraham Kuriakose MD , Sujha Subramanian PhD , Subramania Iyer MCh","doi":"10.1016/j.vhri.2025.101576","DOIUrl":"10.1016/j.vhri.2025.101576","url":null,"abstract":"<div><h3>Objectives</h3><div>To generate India-specific health state utility values for head and neck cancers (HNCs) using EuroQol 5-Dimension 5-Level (EQ-5D-5L) and EuroQol Visual Analog Scale (EQ-VAS) instruments, enabling context-relevant cost-utility analyses and health technology assessments.</div></div><div><h3>Methods</h3><div>A cross-sectional study was conducted at a tertiary HNC center in India from July 2023 to June 2024. Adults with confirmed HNCs across 53 predefined health states were enrolled. A health state is a specific condition of overall well-being, represented by a utility value indicating how preferable it is compared with perfect health or death. Utility scores were derived from the EQ-5D-5L index using the Indian tariff set and the EQ-VAS. Utility variations across treatment phases, tumor subsites, stage, and comorbidity status were also examined.</div></div><div><h3>Results</h3><div>Of 767 recruited patients, 753 were included for analysis across 48 health states, excluding 5 health states with less sample size. The mean EQ-5D-5L utility was 0.84 (SD 0.20), significantly higher than EQ-VAS (mean 0.73, SD 0.18; <em>P</em> < .001). Utilities varied significantly across treatment phases (<em>P</em> < .001). Clinically, patients in active treatment and palliative recurrence had the poorest perceived health, whereas those in remission showed the highest utility, mirroring expected functional recovery patterns. EQ-5D-5L produced consistently higher utility values than EQ-VAS, reflecting differences between population-weighted and self-perceived health assessments.</div></div><div><h3>Conclusions</h3><div>To our knowledge, this is the first Indian data set of real-world health state utility values in HNC. These values provide essential inputs for economic evaluations and health technology assessments in oncology, supporting more accurate, locally relevant healthcare decision making in resource-limited settings.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101576"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145913188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rana Aljaber MSc , Rawan F. Al Froukh MSc , Amna Al Hashar PhD
{"title":"Budget Impact Analysis of Fam-Trastuzumab-Deruxtecan as a Second Line in Patients With HER2-Positive Metastatic Breast Cancer in Oman","authors":"Rana Aljaber MSc , Rawan F. Al Froukh MSc , Amna Al Hashar PhD","doi":"10.1016/j.vhri.2025.101575","DOIUrl":"10.1016/j.vhri.2025.101575","url":null,"abstract":"<div><h3>Objectives</h3><div>Despite advancements, breast cancer remains a major global health and financial challenge. Trastuzumab-based therapies, particularly Fam-Trastuzumab-Deruxtecan (T-DXd), have shown superior outcomes, leading to its recommendation as a preferred second-line treatment. However, its high cost necessitates a budget impact analysis (BIA) to evaluate financial feasibility. This study assesses the 1-year budget impact of replacing Trastuzumab Emtansine (TDM1) with T-DXd for HER2+ metastatic breast cancer in an Omani cancer center.</div></div><div><h3>Methods</h3><div>A static BIA model was built based on National Institute for Health and Care Excellence template with adaptation as required. The model time horizon was 1 year then projected over another 2 years, using 2022 as the baseline year, adopting Omani healthcare-payer perspective. The analysis considered 2 scenarios: (1) TDM1 as the current formulary drug and (2) T-DXd is introduced instead of TDM1, assuming that treatment share would be 100%. One-way sensitivity analysis is performed to assess the model’s robustness.</div></div><div><h3>Results</h3><div>The BIA demonstrated that replacing TDM1 with T-DXd for the eligible patients would lead to increase in the budget expenditure. Over a 3-year period, considering the provided assumptions and input data, the cumulative excess amount to €5 909 481.67. The primary contributing factors are the drug acquisition cost and the median treatment duration. The sensitivity analysis results suggest the model is robust to changes in adverse drug reaction costs, unlike changes in treatment duration and drug cost.</div></div><div><h3>Conclusions</h3><div>Although T-DXd improves clinical outcomes, its adoption as a second-line treatment in Oman poses a substantial financial burden, necessitating careful budget planning to ensure long-term affordability.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101575"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145925613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Carla Castillo-Laborde MSc, PhD , Oscar Arteaga MD, MSc, DrPH
{"title":"Retail Sales of Drugs for Diabetes, Dyslipidemia, and Hypertension in Chile Under 2 Health Policies","authors":"Carla Castillo-Laborde MSc, PhD , Oscar Arteaga MD, MSc, DrPH","doi":"10.1016/j.vhri.2025.101577","DOIUrl":"10.1016/j.vhri.2025.101577","url":null,"abstract":"<div><h3>Objectives</h3><div>The retail market represents 60% of the pharmaceutical market in Chile. Medicines for diabetes, dyslipidemia, and hypertension are among the most commonly used by the Chilean population, and these conditions have been central to health policies aimed at improving access and coverage. This study aimed to analyze the effects of the implementation of the Explicit Health Guarantees program and the Pharmaceutical Fund on retail sales of drugs for hypertension, diabetes, and dyslipidemia.</div></div><div><h3>Methods</h3><div>Drugs related to the 3 conditions were classified using Anatomical Therapeutic Chemical codes. Interrupted time-series analyses were conducted to identify changes in retail sales trends (physical and monetary units) between 2000 and 2020. Key drugs for each condition were also analyzed in greater detail.</div></div><div><h3>Results</h3><div>From 2000 to 2020, hypertension drugs dominated sales in physical units, whereas since 2016, diabetes drugs have led in monetary terms. After the Explicit Health Guarantees program was introduced in 2005, sales in physical units rose for diabetes (trend) and dyslipidemia drugs (trend and level). Monetary sales declined for hypertension (level and trend) and dyslipidemia (level) and increased for diabetes (trend). In 2014, with the Pharmaceutical Fund, sales in physical units decreased for dyslipidemia (trend) and increased for hypertension (level). In monetary terms, dyslipidemia sales declined (trend), and hypertension sales rose (level and trend). Brand-name drug preference in retail remains strong, even when multiple unbranded generics are available, such as metformin, losartan, and atorvastatin.</div></div><div><h3>Conclusions</h3><div>Despite public-sector policies, retail sales of these drugs increased over time, although growth slowed during the second decade.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101577"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146022382","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-Effectiveness of Pembrolizumab as First-Line Therapy for Advanced Colorectal Cancer With High Microsatellite Instability or Mismatched Repair Deficiency in Japan","authors":"Seiya Taniguchi BS , Kensuke Moriwaki MS, PhD , Kosuke Morimoto MS , Kojiro Shimozuma MD, PhD","doi":"10.1016/j.vhri.2025.101555","DOIUrl":"10.1016/j.vhri.2025.101555","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to evaluate the cost-effectiveness of pembrolizumab (PEM) for treating patients with advanced colorectal cancer with high microsatellite instability or mismatched repair deficiency in Japan.</div></div><div><h3>Methods</h3><div>A partitioned survival analysis model was developed to predict costs and quality-adjusted life-years (QALYs) in PEM and standard-of-care (SoC) groups. Survival data for model extrapolation were obtained from the KEYNOTE-177 trial. Drug costs were based on official Japanese pricing, and other medical costs were estimated using a claims database provided by a health insurance association. A 2% discount rate was applied. Utility values were derived from previously published studies. The incremental cost-effectiveness ratio (ICER) of PEM compared with SoC was calculated and evaluated against Japan’s willingness-to-pay threshold. Sensitivity analyses were conducted to assess parameter uncertainty.</div></div><div><h3>Results</h3><div>The incremental cost of PEM compared with SoC was estimated at JPY15 554 008 with an incremental gain of 2.47 QALYs. Based on these results, the ICER for PEM compared with SoC was JPY6 299 749 per QALY. In a scenario without a restriction on the duration of PEM administration, the ICER was estimated at JPY21 408 155 per QALY.</div></div><div><h3>Conclusions</h3><div>Although outcomes varied depending on the PEM treatment duration and the medical cost setting after disease progression in the SoC group, PEM was suggested to be more cost-effective than SoC at the reference willingness-to-pay threshold of JPY15 million per QALY in Japan.</div></div>","PeriodicalId":23497,"journal":{"name":"Value in health regional issues","volume":"54 ","pages":"Article 101555"},"PeriodicalIF":1.5,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145737652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}