PharmacoEconomics Open最新文献

{"title":"Availability, Prices, and Affordability of Immune Checkpoint Inhibitors: A Cross-Sectional Study in Anhui Province, China.","authors":"Tongming Zhu, Kunkun Ge, Aizong Shen, Yingqi Wu","doi":"10.1007/s41669-026-00658-7","DOIUrl":"10.1007/s41669-026-00658-7","url":null,"abstract":"<p><strong>Background/aim: </strong>Immune checkpoint inhibitors (ICIs) have revolutionized cancer therapy, yet their uptake in low- and middle-income areas is hindered by high prices and uneven distribution. Anhui Province, China-characterized by marked urban-rural economic contrasts-offers a critical setting to examine real-world access to these agents. The objective of this study was to assess the availability, price levels, and affordability of ICIs across Anhui Province and to identify policy levers that could narrow observed access gaps.</p><p><strong>Materials and methods: </strong>We applied the World Health Organization/Health Action International (WHO/HAI) standardized survey methodology to evaluate the availability, price differentials, and economic burden of immune checkpoint inhibitors (ICIs) marketed in Anhui Province, China. The survey was conducted in 2025 across 199 public hospitals. Affordability was further assessed using a four-tier framework comprising the WHO/HAI standard indicator (defined as the number of days' wages required to afford 30 days of treatment), adjusted using local urban and rural per capita disposable income to account for China's socioeconomic heterogeneity, and incorporating insurance reimbursement scenarios on the basis of Anhui provincial policy (85% for urban employee insurance and 70% for urban-rural resident insurance) to estimate out-of-pocket expenditure. In addition, catastrophic health expenditure (CHE), defined as household out-of-pocket health spending exceeding 40% of non-food expenditure, was used to capture household-level financial risk beyond individual income-based measures. Availability was defined as the proportion of facilities stocking a medicine on the survey day. Prices were collected at unit level and summarized as medians across facilities, with affordability assessed using the median price ratio (MPR), calculated as median local unit price relative to the international reference price (IRP) from the MSH International Drug Price Indicator Guide, in line with WHO/HAI methodology.</p><p><strong>Results: </strong>Surveying 199 hospitals, we found that domestic PD-1 inhibitors-sintilimab, camrelizumab, and tislelizumab-were stocked in roughly 4 out of 5 facilities, whereas 11 of the 15 mainly imported ICIs appeared in fewer than 16% of hospitals; although some imported agents had acceptable median-price ratios (0-2), their absolute prices remained several-fold higher than domestic alternatives; thus even after insurance (85% urban, 70% rural), a year's treatment with cadonilimab or durvalumab still exceeded the catastrophic-expenditure threshold by up to 40 times for rural households, while domestic sintilimab or camrelizumab stayed well below that line.</p><p><strong>Conclusions: </strong>Domestic ICIs are broadly available and relatively affordable, whereas imported brands remain scarce and financially out of reach; boosting reimbursement limits, expanding centralized procurement, an","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"695-706"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379530/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148345938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost Effectiveness of the Adjuvanted Recombinant Zoster Vaccine in Older Adults in Portugal.","authors":"Eleftherios Zarkadoulas, Fábio Silva, Joana Alves, Francisco Madeira, Maarten J Postma","doi":"10.1007/s41669-026-00640-3","DOIUrl":"10.1007/s41669-026-00640-3","url":null,"abstract":"<p><strong>Background and objectives: </strong>Herpes zoster (HZ) and associated complications comprise a considerable public health burden in older adults. This study aimed to evaluate the cost-effectiveness of recombinant zoster vaccine (RZV) versus no vaccine for the prevention of HZ in older adults in Portugal.</p><p><strong>Methods: </strong>A static multicohort Markov model (ZONA) was adapted to the Portuguese context. The base-case compared RZV with no vaccination in a hypothetical cohort of 1 million adults aged ≥65 years. Real-world data were used for first-dose coverage (56.1%) and second-dose completion (67.2%), with vaccine efficacy and waning based on 11-year follow-up data. Outcomes were calculated over a lifetime horizon from the societal perspective, with a 4% discount applied to costs and quality-adjusted life years (QALYs). In the base-case, HZ incidence was based on previously reported European data. Sensitivity analyses were performed, and a scenario analysis explored the impact of alternative HZ incidence rates based on recent Portuguese data.</p><p><strong>Results: </strong>Over a lifetime horizon, RZV was estimated to prevent 45,551 HZ cases, 9056 post-herpetic neuralgia (PHN) cases, 5580 cases with other non-PHN complications, and 6 HZ deaths. The incremental cost-effectiveness ratio (ICER) from the societal perspective was €48,707 per QALY gained, below a hypothetical willingness-to-pay threshold of two times the gross domestic product per capita per QALY gained (equivalent to €62,200). This base-case ICER was most sensitive to the annual incidence of initial HZ. In the scenario analysis, exploring higher HZ incidence based on Portuguese data, the estimated ICER was €41,877 per QALY gained. In both the base-case and the scenario analyses, ICERs were lowest in individuals aged 65-69 years, with ICERs ranging from €36,207 to €42,700 per QALY gained, in the scenario analysis and base case, respectively.</p><p><strong>Conclusions: </strong>RZV presents a cost-effective option for the prevention of HZ and its complications in Portuguese adults aged ≥ 65 years, with greater cost effectiveness in those aged 65-69 years.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"661-677"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379566/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147344854","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validation of the Obesity Lifecycle Model Built to Assess the Cost Effectiveness of Novel Therapies in the Management of Obesity.","authors":"Ben Modley, Ahmed Seddik, Marc Evans, Stephanie Stephens, Chloe Salter, Marilena Appierto","doi":"10.1007/s41669-026-00661-y","DOIUrl":"10.1007/s41669-026-00661-y","url":null,"abstract":"<p><strong>Background: </strong>Economic models support healthcare decision makers to efficiently assess value and allocate resources; formal validation is critical to ensure confidence in model outputs. The Obesity Lifecycle Model is a patient-level simulation model capturing natural history, clinical complications, quality-of-life and economic outcomes associated with obesity; however, it has yet to undergo formal external and cross-validation.</p><p><strong>Objective: </strong>The aim of this study was to validate the Obesity Lifecyle Model as per best practice guidelines from the Professional Society for Health Economics and Outcomes Research (ISPOR) and the Society for Medical Decision Making (SMDM) Task Force.</p><p><strong>Methods: </strong>Relevant data sources and outcomes were identified for all validations. Selected validation studies informed the model to simulate study outcomes. The model was populated with study characteristics to reproduce studies used in model development (dependent validation), studies not used in model development (independent validation), or other published models (cross-validation). Accuracy between predicted and observed outcomes was assessed using standard statistical methods and mean error calculations.</p><p><strong>Results: </strong>The model demonstrated overall concordance with observed outcomes, supported by coefficient of determination (R²) and ordinary least squares linear regression line (OLS LRL) estimates generally close to 1.0. Independent validation showed an underprediction for cardiovascular disease (CVD) and mortality with an OLS LRL of 0.9309 and an R² of 0.8984 across all populations (normoglycaemic/prediabetic populations: OLS LRL = 0.9485, R² = 0.8854; T2DM populations: OLS LRL = 0.9208, R² = 0.9068).</p><p><strong>Conclusions: </strong>The Obesity Lifecycle Model demonstrates favourable concordance with observed clinical and quality-of-life outcomes, supporting its use for evaluating obesity-related complications and informing healthcare decision making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"647-659"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379529/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148006390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Burden of Spinal Muscular Atrophy in Singapore: A Societal Perspective Cost-of-Illness Study.","authors":"Zhi Zhen Lim, Yan Ling Lim, Furene Sijia Wang, Simon Robert Ling, Stacey Kiat Hong Tay, Yi Wang","doi":"10.1007/s41669-026-00655-w","DOIUrl":"10.1007/s41669-026-00655-w","url":null,"abstract":"<p><strong>Objectives: </strong>The objective was to evaluate the overall economic burden of spinal muscular atrophy (SMA) in Singapore.</p><p><strong>Methods: </strong>A retrospective cohort study of electronic medical records and billing data was used to obtain medical costs from public healthcare consumption. Private medical costs, nonmedical costs from transportation and hired domestic helpers, and indirect costs due to caregiver productivity loss were estimated with a cross-sectional caregiver survey. The survey also examined intangible negative and positive effects on these caregivers. All costs were adjusted to 2024 Singapore dollar (SGD).</p><p><strong>Results: </strong>A total of 61 patients were identified in the electronic medical records, and 15 caregivers were interviewed. One caregiver who took care of two patients with SMA was excluded from the analysis. The gross cost of inpatient admission is SGD 1663.81 (standard deviation [SD] = 986.03) per day with mean length of stay of 6.22 days (SD = 10.89) per year, while annual mean outpatient cost is SGD 940.31 (SD = 1071.28). In addition to public healthcare services, private services incur an additional SGD 5992.35 (SD = 7557.33) per year, with fixed costs for respiratory devices and mobility aids at SGD 5677.06 (SD = 4519.57) and SGD 12,412.70 (SD = 8307.35), respectively. Nonmedical costs from hiring a domestic helper, medical-related transport, and nonmedical transport amount to SGD 7002.22 (SD = 6516.36), SGD 387.86 (SD = 196.36), and SGD 840.95 (SD = 1067.81) per annum, respectively. Paid work productivity loss is estimated at SGD 42,932.88 per family per year. Average overall economic burden is estimated at SGD 62,004.03, SGD 10,840.13, and SGD 59,629.64 per patient per year from the societal perspective, healthcare system perspective, and patient perspective, respectively.</p><p><strong>Conclusions: </strong>SMA poses a significant economic burden on patients, families, and the healthcare system. This study informs policy discussions for SMA management, advocating for a comprehensive, compassionate approach and societal perspective in economic evaluations to capture all costs and benefits.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"721-731"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379547/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147841292","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating Eligibility for Lung Cancer Screening in Ireland: A Dynamic Modelling Study of Smoking Trends.","authors":"Tatiana Bezdenezhnykh, James F O'Mahony, Benjamin M Jacob, Patrick Redmond","doi":"10.1007/s41669-026-00653-y","DOIUrl":"10.1007/s41669-026-00653-y","url":null,"abstract":"<p><strong>Background: </strong>Lung cancer screening (LCS) with low-dose computed tomography (LDCT) reduces mortality. Ireland will likely adopt a national screening programme in the coming years, and evidence on the size of the eligible population is required to inform planning.</p><p><strong>Objective: </strong>The objective was to estimate the number and characteristics of individuals in Ireland eligible for LCS under different international criteria and to project future uptake.</p><p><strong>Methods: </strong>We combined population data from the 2022 Census and subsequent projections with smoking history from the 2017 Eurobarometer survey. Eligibility was assessed under four criteria: Irish pilot (55-74 years, ≥ 20 pack-years, quit < 15 years), US Preventive Services Task Force (USPSTF) 2013, USPSTF 2021, and NELSON. Projections to 2045 accounted for demographic change and smoking trends. Expected uptake was estimated using participation rates from SUMMIT, a large UK LCS implementation trial.</p><p><strong>Results: </strong>In 2022, 245,105 individuals met the Irish pilot criteria, and 345,328 met USPSTF 2021. Eligibility remains broadly stable until the mid-2030s, then declines across all criteria. Relative differences between criteria persist over time, with USPSTF 2021 consistently identifying the largest group. Applying SUMMIT uptake rates, around 36,000 LDCT scans annually would be required at steady state.</p><p><strong>Conclusions: </strong>Approximately a quarter of a million people in Ireland would currently be eligible for LCS, rising to more than 340,000 under broader criteria. Numbers are expected to remain high for the next decade before gradually declining. These findings provide key evidence for service capacity planning and highlight the importance of flexible programme design to meet future demand.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"707-720"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379544/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147819292","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trial-Based Economic Evaluations Conducted alongside Studies Embedded in Australian Healthcare Services and Programs: A Scoping Review.","authors":"Belinda Wang, Catherine Sherrington, Luiza R Pivotto, Stanley Saputra, Josielli Comachio, Joanne Scarfe, Zoe Szewczyk, Nathalia Costa, Alison Pearce, Leanne Hassett, Andrew Milat, Christopher Williams, Wing S Kwok, Justin Yu, Kirsten Howard, Marina B Pinheiro","doi":"10.1007/s41669-026-00652-z","DOIUrl":"10.1007/s41669-026-00652-z","url":null,"abstract":"<p><strong>Objectives: </strong>The objective of this study was to map the literature and provide an overview of economic evaluations (EEs) conducted alongside studies embedded in Australian healthcare services or programs by: (1) providing a broad overview of the volume, characteristics and types of EEs and (2) examining the subset of EEs impacting older Australians in greater depth including characteristics (type, costs, outcomes, perspectives adopted, time horizon) and quality.</p><p><strong>Methods: </strong>We conducted a scoping review of full trial-based EEs of studies embedded in Australian healthcare services and programs, published in peer-reviewed journal publications. Medline, Informit, ProQuest Central, Web of Science, Econlit, PsychINFO, Cumulative Index to Nursing and Allied Health Literature (CINAHL), Cost-Effectiveness Analysis (CEA) Registry, and the International Health Technology Assessment (HTA) database were searched from inception to May 2025. Quality assessment was conducted using the Consensus Health Economic Criteria (CHEC-list).</p><p><strong>Results: </strong>We identified 88 articles (86 studies) reporting 107 EEs. Cost-effectiveness analyses (CEA) were most common (n = 53, 50%). Most interventions were delivered in ambulatory care settings (n = 41, 47%) and included participants from metropolitan areas (n = 81, 92%). A total of 28 studies (29 comparisons) were with older adults, reporting 38 EEs. Among these, CEAs (n = 16, 42%) and adoption of a health system perspective (n = 11, 29%) were most frequent. Most EEs included intervention costs (n = 25, 86%) and healthcare costs (n = 28, 97%). Most interventions evaluated in CEA and cost-utility analysis were found to be more costly and more effective (n = 16, 43%) or less costly and more effective (n = 15, 41%). The median CHEC-list was 16/19 (IQR 13-17).</p><p><strong>Conclusions: </strong>Our review identified a diverse range of intervention types and evaluation methods, but a relative lack of cost-benefit analyses, despite their value for cross-sector comparisons. Most evaluations were conducted in metropolitan settings and few incorporated broader costs such as aged care, particularly regarding interventions targeting older adults. Future research should prioritize evaluations that include rural and remote populations and adopt broader perspectives, ensuring the full value of interventions is captured to better inform policy and decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"623-645"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379549/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147662927","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Updated Health Economics Simulation Model of the Lifetime Progression of Multiple Sclerosis in an Australian Setting.","authors":"Andrew J Palmer, Ting Zhao, Glen Henson, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Julie A Campbell","doi":"10.1007/s41669-026-00650-1","DOIUrl":"10.1007/s41669-026-00650-1","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) is a progressive disease leading to increasing disability. Our previous simulation model estimated lifetime impacts of MS on quality of life and associated economic costs but was limited by small cohort input data and simplistic structure.</p><p><strong>Objective: </strong>The aim was to improve our previous model by incorporating more granular disease states, improving the accuracy of input data using a large MS cohort, and including contemporary cost and health state utility (HSU) inputs, matching the more detailed health states.</p><p><strong>Methods: </strong>A Markov model was developed simulating MS progression from no disability to mild, moderate, and severe disability and death under contemporary treatment patterns. State-specific probabilities of progression, death, relapse, costs, HSUs, and relapse-related disutilities were considered. Life expectancy (LE), quality-adjusted life years (QALYs), and discounted lifetime total costs (2026 Australian dollars) were calculated. Probabilistic sensitivity analysis was performed.</p><p><strong>Results: </strong>A typical MS cohort of 35-year-old females had a projected mean LE of 44.0 years, 27.3 and 11.3 undiscounted/discounted QALYs, respectively, and lifetime costs of $1,449,597. Compared with the Australian general population, those with MS had 6.8 years lower LE, 15.0 lower undiscounted QALYs, and $1.24 million higher costs. Our updated model showed higher LE and costs but lower QALYs compared with our previous model, due to differences in probability utilities and cost inputs.</p><p><strong>Conclusions: </strong>Our improved model, based on a larger cohort and updated inputs, provides a more nuanced assessment of the impact of MS on LE, QALYs, and costs, with improved face validity. It will be used for the cost-effectiveness analyses of current and future MS interventions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"759-767"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379532/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147691467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Biosimilar Denosumab Versus Bisphosphonates in Postmenopausal Osteoporosis.","authors":"Jeanine Flanigan, Sarah Kane, Fatemeh Mirzayeh Fashami, Anna Zhou, Lamees Almuallem, Mike Tindal, Marion Schauf, Edward Li, Gary H Lyman","doi":"10.1007/s41669-026-00651-0","DOIUrl":"10.1007/s41669-026-00651-0","url":null,"abstract":"<p><strong>Background/objectives: </strong>Postmenopausal osteoporosis (PMO) is a major public health and economic burden in the USA. The objective of this study was to evaluate the cost-effectiveness of biosimilar denosumab compared with alendronate, risedronate, ibandronate, and zoledronic acid in treating women with PMO at high risk of fracture from a US payer perspective.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was conducted using a Markov cohort model and a lifetime horizon was applied to capture long-term costs and effects. Treatment effects were based on published network meta-analyses. The cost of biosimilar denosumab was estimated on the basis of assumptions; other costs were obtained from publicly available sources. Main outcomes included quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs).</p><p><strong>Results: </strong>Biosimilar denosumab was associated with greater efficacy and higher costs versus all comparators. Over a lifetime horizon, the ICER was $101,017, $93,544, $100,515, and $144,995 per QALY gained for biosimilar denosumab compared with alendronate, risedronate, ibandronate, and zoledronic acid, respectively. Cost-effectiveness was most sensitive to starting age, discount rate, treatment duration, and biosimilar price. At a willingness-to-pay (WTP) threshold of $150,000 per QALY gained, biosimilar denosumab was likely to be cost-effective compared with alendronate, risedronate, ibandronate, and zoledronic acid in the treatment of PMO. At a WTP threshold of $100,000 per QALY gained, biosimilar denosumab was likely to be cost-effective relative to risedronate, while ICERs relative to alendronate and ibandronate only marginally exceeded this WTP threshold.</p><p><strong>Conclusions: </strong>Biosimilar denosumab was estimated to be cost-effective compared with alendronate, risedronate, ibandronate, and zoledronic acid at a WTP threshold of $150,000/QALY gained and compared with risedronate at a threshold of $100,000/QALY gained. As a lower-cost alternative to reference denosumab, biosimilar denosumab may enhance patient access to effective PMO treatment in the USA.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"733-748"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147675755","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adverse Drug Reaction Costs and Study Quality in Africa: A Systematic Review of Cost-of-Illness Studies.","authors":"Bezie Kebede, Bereket Bahiru, Chernet Tafere","doi":"10.1007/s41669-026-00657-8","DOIUrl":"10.1007/s41669-026-00657-8","url":null,"abstract":"<p><strong>Objective: </strong>An adverse drug reaction (ADR) is a harmful, unwanted patient response to a drug that happens at doses normally used for prevention, diagnosis, or treatment. Despite limited and inconsistent primary evidence on the cost of ADRs in Africa, this systematic review aims to synthesize the magnitude of ADR-related costs and assess the reporting quality of existing studies.</p><p><strong>Method: </strong>The authors followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist. Three databases (PubMed, Scopus, and ScienceDirect) and a hand search were employed. All empirical African cost-of-illness studies on ADRs were included regardless of publication year, disease type, or population, while letters to the editor, case reports, conference proceedings, and abstracts were excluded. Two independent authors conducted screening, data extraction, and quality assessment. A consensus-based cost-of-illness checklist was used to assess reporting quality. The primary outcome was the cost of adverse drug reactions, reported in 2024 international dollars (I$); the secondary outcome was the reporting quality of studies.</p><p><strong>Results: </strong>A total of 324 studies were identified, of which eight were included in the final analysis. Nearly all studies reported only direct medical costs, excluding direct non-medical and indirect costs. ADR-related hospitalization costs per patient ranged from I$54.75 in Nigeria to I$7438.47 in South Africa. Costs were mainly driven by hospitalization, medications, and investigations, and influenced by patient, disease, and health system-related factors. While all studies described their population, objectives, time horizon, and data collection methods, many failed to report important methodological details.</p><p><strong>Conclusion: </strong>Adverse drug reactions impose a substantial economic burden through direct medical costs influenced by multiple factors; however, many studies lacked key reported details. Future research should adopt recommended methodologies and comprehensive cost reporting to improve the reliability of cost estimates.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"609-621"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379527/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147974448","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prediction Error in Quality-Adjusted Life Years in Economic Evaluations of Immune Checkpoint Inhibitors: A Comparison Based on Projected and Observed Updated Survival.","authors":"Jinyu Chen, Yichen Zhang, Kexin Han, Sheng Han, Luwen Shi, Xiaodong Guan","doi":"10.1007/s41669-026-00660-z","DOIUrl":"10.1007/s41669-026-00660-z","url":null,"abstract":"<p><strong>Background: </strong>Survival data from pivotal clinical trials are critical for estimating quality-adjusted life years (QALYs). However, immature survival data require extrapolation beyond observed follow-up to project outcomes, introducing potential prediction error into QALY estimates used in economic evaluations. Immune checkpoint inhibitors (ICIs) present unique extrapolation challenges due to delayed responses and extended survival benefits. We therefore quantify the QALY prediction error of early extrapolations by benchmarking them against updated follow-up at a common time horizon.</p><p><strong>Methods: </strong>Using reconstructed individual patient data derived from published Kaplan-Meier curves of pivotal trials, this study assessed the accuracy of early survival extrapolations for ICIs approved in China by comparing early projections with QALYs obtained from updated data at the same horizon. A partitioned-survival framework using overall survival (OS) and progression-free survival (PFS) informed state occupancy, and QALYs were obtained via restricted mean survival time (RMST) integration of health-state utilities at the target horizon. Statistical analyses evaluated bias, precision, and agreement between extrapolated and updated QALY estimates. Linear regression and sensitivity analyses assessed the impact of target extrapolation horizon (T) on prediction error.</p><p><strong>Results: </strong>In total, 14 randomized controlled trials (4839 patients) were included for analysis. The mean deviation between extrapolated and observed QALYs was - 0.01 (95% CI - 0.03 to 0.01), with a mean absolute error (MAE) of 0.03 (95% CI 0.01 to 0.04). Strong agreement existed between extrapolated and updated QALYs (Spearman's ρ = 0.98, 95% CI 0.94 to 0.99, P < 0.001). Consistently, OS extrapolations showed minimal deviation (MAE: 0.56 months), while PFS tended to be underestimated (MAE: 1.91 months). Moreover, predictive error increased significantly with longer extrapolation periods for QALY (MAE increase: 0.011 QALY/year, P = 0.004) and OS (MAE increase: 0.342 months/year, P = 0.010).</p><p><strong>Conclusions: </strong>Near-horizon QALY prediction error was modest on average but increased with longer target extrapolation horizons. These findings support transparent reporting of extrapolation uncertainty and suggest that structured evidence reassessment may be particularly valuable as longer follow-up becomes available.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"769-779"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379523/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148016756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}