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Cost-effectiveness analysis of enfortumab vedotin and pembrolizumab versus chemotherapy for patients with untreated advanced urothelial cancer in the United Kingdom

Por: Wu · Q. · Li · Q. · Qin · Y.
Objectives

Enfortumab vedotin and pembrolizumab (EV–Pem) have recently been shown to improve the prognosis of patients with untreated locally advanced or metastatic urothelial carcinoma (la/mUC) in a randomised phase three clinical trial. This study aims to evaluate the cost-effectiveness of EV–Pem as a first-line treatment for patients with la/mUC.

Design

We developed a three-state Markov model to simulate the treatment pathways for patients with la/mUC. Costs associated with drugs, treatment monitoring, adverse event management and utility inputs were obtained from national cost databases, trial data and existing literature. In addition to the base-case analysis, sensitivity, threshold and subgroup analyses were performed to assess uncertainty.

Setting

The National Health Service and Personal Social Services perspective.

Participants

A hypothetical English cohort of patients with la/mUC.

Interventions

First-line treatment with either EV–Pem or chemotherapy.

Main outcome measures

Costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) were calculated over a lifetime horizon as primary outcomes.

Results

In patients with la/mUC, EV–Pem increased health outcomes by 0.82 QALYs (95% uncertainty interval (UI) 0.66 to 0.92) at an incremental cost of £76 793.75 (95% UI 75 759.96 to 77 750.72) versus chemotherapy, yielding an ICER of £93 650.92 per QALY (95% UI £83 471.84 to 116 739.74). One-way sensitivity analysis revealed that factors such as utility values, EV prices and discount rates influence model outcomes, but these variations did not alter the conclusion. Probabilistic sensitivity analysis indicated that, at a willingness-to-pay threshold of £30 000 per QALY, the probability of cost-effectiveness is 0. Results remained robust across subgroup analyses.

Conclusion

In this economic model analysis, EV–Pem is not cost-effective compared with first-line chemotherapy in the UK, and a price reduction may be necessary.

The cost-effectiveness of penicillin allergy assessment pathway (PAAP): a decision analysis

Por: Yang · M. · Bestwick · R. · Howdon · D. · Ahmed · S. · Powell · N. · Armitage · K. F. · Fielding · J. · Porter · C. E. · Savic · S. · West · R. M. · Howard · P. · Galal · U. · Pavitt · S. · Sandoe · J. A. · Mujica-Mota · R. E.
Objective

To evaluate the cost-effectiveness of implementing a penicillin allergy assessment pathway (PAAP) versus usual care within the NHS.

Design

A decision tree analysis over a 5-year time-period, informed by a randomised controlled trial (RCT) of PAAP and systematic review. Value of information analysis was also conducted to estimate the value of conducting a new trial.

Data sources

Model inputs were informed by the ALABAMA RCT participants included in the primary analysis, 811 adults with penicillin allergy labels and recent antibiotic prescriptions, and data from published literature.

Interventions

Participants in the ALABAMA trial included in the primary analysis: PAAP (n=401) and usual care (n=410).

Primary and secondary outcome measures

Costs are presented in GBP (£) at 2022–2023 prices, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio, incremental net monetary benefit (INMB), the probability of cost-effectiveness at the £20,000 and £30,000 per QALY threshold, and the cost effectiveness of a new follow-on trial.

Results

PAAP had incremental costs of £–83 (probability of cost saving 47.5%) and incremental QALYs of 0.036 (probability of positive benefits 47.5%). The INMBs (probability of cost-effectiveness) were £806 (48%) and £1167 (48%) under the decision thresholds of £20,000 and £30,000 per QALY, respectively. PAAP was more cost-effective among females, people aged >65 years, and more frequent antibiotic users. A new follow-on trial involving 1267 participants was estimated to cost £2.4 million and, by reducing uncertainty in the evidence, would avoid £19.6 million in costs of incorrect management decisions for eligible patients over the next 10 years.

Conclusion

The PAAP was considered cost-effective, but significant uncertainty remained. Future trials with adequate power and longer follow-up are needed to determine the most cost-effective models for penicillin allergy testing.

Trial registration number

ISRCTN20579216.

How do musculoskeletal disorders impact on quality of life in Tanzania? Results from a community-based survey

Por: Grieve · E. · Deidda · M. · Krauth · S. J. · Biswaro · S. M. · Halliday · J. E. B. · Hsieh · P.-H. · Kelly · C. · Kilonzo · K. · Kiula · K. P. · Kolimba · R. · Msoka · E. F. · Siebert · S. · Walker · R. · Yongolo · N. M. · Mmbaga · B. · McIntosh · E. · NIHR Global Health Group · Biswaro
Objectives

There are little available data on the prevalence, economic and quality of life impacts of musculoskeletal disorders in sub-Saharan Africa. This lack of evidence is wholly disproportionate to the significant disability burden of musculoskeletal disorders as reported in high-income countries. Our research aimed to undertake an adequately powered study to identify, measure and value the health impact of musculoskeletal conditions in the Kilimanjaro region, Tanzania.

Design

A community-based cross-sectional survey was undertaken between January 2021 and September 2021. A two-stage cluster sampling with replacement and probability proportional to size was used to select a representative sample of the population.

Setting

The survey was conducted in 15 villages in the Hai District, Kilimanjaro region, Tanzania.

Participants

Economic and health-related quality of life (HRQOL) questionnaires were administered to a sample of residents (aged over 5 years old) in selected households (N=1050). There were a total of 594 respondents, of whom 153 had a confirmed musculoskeletal disorder and 441 matched controls. Almost three-quarters of those identified as having a musculoskeletal disorder were female and had an average age of 66 years.

Primary and secondary outcome measures

Questions on healthcare resource use, expenditure and quality of life were administered to all participants, with additional more detailed economic and quality of life questions administered to those who screened positive, indicating probable arthritis.

Results

There is a statistically significant reduction in HRQOL, on average 25% from a utility score of 0.862 (0.837, 0.886) to 0.636 (0.580, 0.692) for those identified as having a musculoskeletal disorder compared with those without. The attributes ‘pain’ and ‘discomfort’ were the major contributors to this reduction in HRQOL.

Conclusions

This research has revealed a significant impact of musculoskeletal conditions on HRQOL in the Hai district in Tanzania. The evidence will be used to guide clinical health practices, interventions design, service provisions and health promotion and awareness activities at institutional, regional and national levels.

Measuring and valuing patient and caregiver productivity costs: a scoping review protocol

Por: Yeretzian · S. T. · Sillcox · C. · Loshak · H. · Ramsay · L. · Sahakyan · Y. · Sander · B.
Introduction

Economic evaluations are essential for informing healthcare resource allocation. When conducted from a societal perspective, they may include productivity costs such as paid and unpaid productivity losses for patients and their caregivers. Although several methods exist to measure and value productivity costs, there is limited methodological consensus on which methods should be used. This scoping review aims to synthesise existing methods for measuring and valuing patient and caregiver productivity costs.

Methods and analysis

This review will follow the Arksey and O’Malley framework, enhanced by subsequent methodological guidance from Levac and the Joanna Briggs Institute. The six stages include identifying the research question; identifying relevant studies; selecting studies; charting the data; collating, summarising and reporting the results; and consultation. We will search MEDLINE, Embase and EconLit from 1996 to July 2025. Eligible sources will include peer-reviewed literature that reports methods for the measurement or valuation of productivity costs related to paid or unpaid work among patients or caregivers. Screening and data extraction will be conducted independently by two reviewers. Extracted data will include types of productivity costs, instruments used, valuation approaches, as well as recommendations on preferred measurement and valuation methods. Results will be synthesised thematically and reported using the Preferred Reporting Items for Systematic Review and Meta-Analysis for Scoping Reviews checklist.

Ethics and dissemination

Ethics approval is not required as this review will rely exclusively on publicly available literature and does not involve human participants or the use of primary data. The findings will first be shared with Canada’s Drug Agency as a report and then disseminated through peer-reviewed publication and academic presentations to inform future research and practice.

Registration

This protocol has been registered with the Open Science Framework (https://doi.org/10.17605/OSF.IO/FK9D4).

Effect of the 'digital plus approach to upscaling early childhood development services in rural China: study protocol for a cluster randomised controlled trial

Por: Wang · B. · Xiao · Y. · Qian · Y. · Li · S. · Zhang · H. · Reiling · K. · Rozelle · S.
Introduction

The high prevalence of developmental delays among young children poses significant barriers to long-term social mobility in low-income and middle-income countries. Digital interventions aimed at early childhood development (ECD) have shown promise in improving developmental outcomes, yet scalable and effective strategies remain under-explored. This study aims to evaluate the effectiveness of a digitally delivered parenting intervention to improve early child development in rural China.

Method and analysis

We design a parallel cluster-randomised controlled trial to evaluate a 12-month digitally delivered ECD intervention in rural China. Key inclusion criteria are households with children aged 6–24 months and without relocation intentions. Key exclusion criteria are children with a severe disability. Participants are masked to treatment assignment. The project implements village-level interventions in three counties in central China, involving 70 villages (clusters). Implementation involves two phases: the preparatory phase and the online phase, in which ‘Parenting the Future’ curriculum will be delivered through a newly developed mobile application. During the preparatory phase, county-level parenting trainers will conduct weekly home visits to each sampled household. In the online phase, caregivers will primarily access weekly parenting training through prerecorded videos on a mobile application. Trainers will conduct short, fortnightly home visits to manage toys and picture books loaned through the same application in accordance with the training. The planned total treatment involves 48 sessions over 1 year. Assessments were conducted via home visits at baseline and endline. The primary outcomes include cognitive development measured with the Bayley Scales of Infant and Toddler Development, third edition and social-emotional development measured with the age and stage questionnaire: social emotional. Secondary outcomes include caregiver engagement and the home learning environment measured by family care indicators and responsive caregiving rating scale. The trial aims to test the effectiveness and scalability of a hybrid ECD intervention.

Ethics and dissemination

Ethical approval for this study has been obtained from the Institutional Review Board of Southwestern University of Finance and Economics. Informed consent will be obtained from all participants, with appropriate measures in place to ensure participant confidentiality. Results from the study will be shared through academic publications, policy briefs and presentations to government stakeholders and international organisations, contributing to policy discussions on the scalability of digital ECD interventions.

Trial registration number

AEA RCT Registry (AEARCTR-0013908); ISRCTN Registry (ISRCTN15854033).

Curative care expenditure across different comorbidity states of chronic hepatitis B: a prevalence study in Sichuan Province, China

Por: Qiaoying · W. · Lanming · F. · Yang · L. · Jia · L. · Qun · S. · Yuelin · Z.
Objective

The study aims to calculate the curative care expenditure (CCE) from different comorbidity states of chronic hepatitis B (CHB), to provide a reference for the relevant government departments to optimise health insurance protection measures for this disease.

Design

A prevalence-based retrospective study.

Setting

This study included 1600 medical institutions, covering general hospitals, traditional Chinese medicine hospitals, specialised hospitals and maternal and child health hospitals.

Participants

The cohort included 238,617 CHB outpatients and 4312 inpatients.

Primary and secondary outcome measures

CCE for CHB was estimated based on System of Health Accounts 2011.

Results

The total CCE of CHB in Sichuan Province in 2019 was USD 85.49 million. The majority of CCE was spent by outpatient services (USD 67.16 million), the main source of financing schemes was household out-of-pocket payment (75.71%). Conversely, the CCE for inpatient services (USD 18.33 million) was mainly financed by public financing schemes (46.38%). More than 70% of the CCE flowed to general hospitals, and less than 20% flowed to primary healthcare institutions. The CCE varied significantly across different comorbidity groups. The CCE for patients with no additional comorbidities and those in the low comorbidity group was mainly spent by patients aged 30–39 years and those aged 40–49 years, respectively. The CCE in the high comorbidity group was mainly spent by patients aged 50–54 years.

Conclusion

The present study shows that a high proportion of the CCE allotted to CHB is financed by household out-of-pocket payment. This situation poses a heavy medical burden not only on individuals, but also on society. Therefore, the financing structure needs to be optimised urgently. Potential policy directions may improve the outpatient mutual aid protection mechanism.

Evaluating health system expenditure across the rural-urban spectrum in New Zealand: a study protocol

Por: Liepins · T. · Atkinson · J. · Davie · G. · Leung · W. · Crengle · S. · Keenan · R. · Whitehead · J. · Stokes · T. · Nixon · G.
Introduction

Inequities in health status exist in New Zealand across the rural–urban spectrum. In parallel, rural–urban differences in health service utilisation vary by service type. Despite the New Zealand public health system being based on principles of universal access, equity and fairness, levels of health expenditure on rural and urban populations are not well understood. The purpose of the study is to undertake a rural–urban analysis of public health system expenditure, based on individual-level service utilisation and national pricing of health service events.

Methods and analysis

Individual-level service utilisation and pricing will be collated from national collection databases for all eligible users of publicly funded services. The analysis will include calendar years 2017–2024. Descriptive analysis and a two-part generalised linear regression model will be used to identify if rural–urban differences in expenditure exist, and what the association of rurality is with expenditure (if any). The model will also be used to identify geographic regions with expenditure levels that vary from those predicted using regression model weights. As the specific statistical approach will be determined by data attributes, this protocol outlines the intended approach to construct the analytical model.

Ethics and consultation

Ethics approval was obtained from the University of Otago Human Research Ethics Committee (HD23/052). Māori consultation has been undertaken with the Ngāi Tahu Research Consultation Committee and will continue throughout the research process.

Economic evaluation of a hybrid type 1 effectiveness-implementation trial comparing a community health worker palliative care intervention to enhanced standard of care in African American patients across four cancer centres in the USA: a study protocol

Por: Monton · O. · Drabo · E. F. · Masroor · T. · Fuller · S. · Woods · A. P. · Siddiqi · A. · Malone · T. B. · Johnston · F. M.
Introduction

Despite increasing palliative care capabilities in the USA, utilisation rates remain low for patients with advanced cancer, particularly among African American patients. To address this gap, a theory-driven, stakeholder-informed community health worker (CHW) palliative care intervention for African American patients with advanced cancer and their informal caregivers is currently being assessed through a hybrid type 1 effectiveness-implementation trial at four cancer centres across the USA. To improve the quality and efficiency of palliative care delivery, inform resource allocation and guide broad-scale implementation, it is essential to generate evidence on the economic value of palliative care programmes. The objectives of this study are to evaluate the cost-effectiveness and estimate the social value of a CHW palliative care intervention for African American patients with advanced cancer and their caregivers.

Methods and analysis

We will conduct cost-effectiveness analyses (CEAs) and a social return-on-investment (SROI) analysis to assess the value of the CHW palliative care intervention compared with enhanced standard of care. Standard, extended and distributional CEAs will be performed from the perspectives of an adopting organisation or payer (eg, Medicaid), the US healthcare sector and society. An SROI analysis will also be conducted to assess the social value of the intervention. These analyses will focus on estimating the costs, health and distributional impacts of the intervention.

Ethics and dissemination

This study protocol was approved by the Johns Hopkins Medicine Institutional Review Board (IRB00372476). All methods will be carried out in accordance with relevant guidelines and regulations. Written informed consent will be obtained from all subjects prior to study participation. This manuscript does not contain participant-level data. The full protocol will be available from the corresponding author on reasonable request. The dissemination of findings from the clinical trial and accompanying economic evaluation outlined in this manuscript will be multifaceted to maximise reach and impact. Research findings will be presented at relevant scientific conferences, submitted for publication in peer-reviewed journals and shared with community stakeholders, including hospital leaders and administrators, providers, CHWs and patient advocacy groups.

Trial registration number

NCT05407844.

Morphine for chronic breathlessness (MABEL) in the UK: a health economic evaluation of a multisite, parallel-group, dose titration, double-blind, randomised, placebo-controlled trial

Por: Atter · M. J. · Hall · P. · Evans · R. A. · Norrie · J. · Cohen · J. · Williams · B. · Chaudhuri · N. · Bajwah · S. · Higginson · I. · Pearson · M. · Currow · D. · Stewart · G. · Fallon · M. · Johnson · M.
Objectives

To compare costs and health consequences and to assess the cost-effectiveness of using low-dose oral long-acting morphine in people with chronic breathlessness.

Design

Within-trial planned cost-consequences and cost-effectiveness analysis of data from a multisite, parallel-group, double-blind, randomised, placebo-controlled trial of low-dose, long-acting morphine.

Setting

11 hospital outpatients across the UK.

Participants

Consenting adults with chronic breathlessness due to long-term cardiorespiratory conditions.

Intervention

5–10 mg two times a day oral long-acting morphine with a blinded laxative for 56 days.

Primary outcome measures

Mean and SD of healthcare resource use (HRU) by trial arm; mean differences and 95% CI of costs between trial arms.

Secondary outcome measures

Mean differences in 28- and 56-day quality-adjusted life years (QALYs based on EuroQol five-dimension five-level score), Short Form-six dimensional scores and ICEpop CAPability-Supportive Care Measure scores; cost-utility of long-acting morphine for chronic breathlessness.

Results

143 participants (75 morphine and 67 placebo) were randomised; 140 (90% power, males 66%, mean age 70.5 (SD 9.4)) formed the modified intention-to-treat population (participants receiving at least one dose of study medication). There were more inpatient and fewer outpatient services used by the morphine group versus the placebo. In the base-case analysis at 56 days, long-acting morphine was associated with similar mean per-patient costs and QALYs. There was an increase of £24 (95% CI –£395 to £552) and 0.002 (95% CI –0.004 to 0.008) QALYs. Hospitalisations were the main driver of cost differences. The corresponding incremental cost-effectiveness ratio was £12 000/QALY, with a probability of cost-effectiveness of 54% at a £20 000 willingness-to-pay threshold. In the scenario analysis that excluded costs of adverse events considered unrelated to long-acting morphine by site investigators and researchers, the probability of cost-effectiveness increased to 73%.

Conclusion

Oral morphine for chronic breathlessness is likely to be a cost-effective intervention provided adverse events are minimised, but the effect on outcome is small and cautious interpretation is warranted.

Trial registration number

ISRCTN87329095.

Evaluating economic outcomes in the management of temporomandibular disorders: a protocol for a systematic review of randomised controlled trials

Por: Komarraju · P. · AlSabbagh · B. · Ohinmaa · A. · Armijo-Olivo · S. · Morris · M. · Li · J. · Major · P. W. · Goldberg · M. · Naik · A. · Abdelkarim · A. · Velly · A. M.
Introduction

Systematic reviews (SRs) on the management of temporomandibular disorders (TMDs) have predominantly focused on evaluating the effectiveness of various treatments, identifying those that provide the greatest benefits. However, the economic evaluation of these treatments has not been systematically explored. This SR aims to address this gap by evaluating the economic outcomes of the most common treatment modalities for TMDs, including cost-effectiveness, cost-utility, cost-benefit, cost-minimisation and the burden of illness.

Methods and analysis

This SR will be conducted using the following electronic databases Business Source Complete, CINAHL, EconLit (ProQuest), Embase (Ovid), MEDLINE (PubMed), MEDLINE (Ovid) and Scopus to identify studies evaluating the economic outcomes of treatments for TMDs. The eligibility criteria are as follows: (1) studies examining the costs and/or impact of treatments for TMDs and (2) articles published between 2000 and 2025. The primary outcomes of interest are the economic findings outlined earlier. Data extraction will include the following: author(s), year of publication, country, study objectives, study design, eligibility criteria, TMD diagnosis and screening, study groups, randomisation, blinding, sample size, number of participants invited, enrolled and completed, duration of treatment, follow-up, study duration, settings, assessment instruments, study outcomes, statistical analyses, results, limitations, strengths and funding sources. The quality of studies will be evaluated using the Consolidated Health Economic Evaluation Reporting Standards 2022 checklist, with risk of bias assessed using the Cochrane Effective Practice and Organization of Care’s risk-of-bias tool; where applicable, the Outcome Reporting Bias in Trials will be used to detect reporting biases. A narrative synthesis and summary tables will outline study characteristics, economic outcomes and the overall quality of evidence. We will conduct qualitative secondary and sensitivity analyses.

Ethics and dissemination

This SR does not require an ethics approval. The results will be disseminated through international and national conferences and peer-reviewed journals.

PROSPERO registration number

CRD42024613553.

Crowding-out and crowding-in effects of out-of-pocket expenditures for non-communicable diseases care on household consumption patterns in Bangladesh: a cross-sectional analysis of the 2022 household income and expenditure survey

Por: Islam · M. T. · Odunyemi · A. · Bruce · M. · Alam · K.

This study aims to analyse the crowding-out and crowding-in effects of non-communicable diseases (NCDs) related out-of-pocket (OOP) health expenditures on household consumption in Bangladesh.

Objective

Design

This study used data from the nationally representative Bangladesh household income and expenditure survey (HIES) 2022.

Setting

Eight divisions of Bangladesh.

Participants

14 395 households.

Primary and secondary outcome measures

We examined how household consumption patterns across 20 expenditure categories were impacted by OOP health spending for NCD management, with a focus on income-level disparities.

Results

In Bangladesh, OOP health expenditures for NCDs crowded out household expenditures on essential items like food and rent. Across all households, OOP health expenditures for NCDs by 10 US$ crowded out –3.8 US$ of expenditure on food (95% CI –5.1 to –2.5), and more specifically on protein-rich foods (–2.0 US$, 95% CI –2.8 to –1.2), spices (–0.2 US$, 95% CI –0.3 to –0.1), and restaurant and café meals (–0.9 US$, 95% CI –1.4 to –0.5). Crowding-out was also seen for tobacco, rent, durable goods and miscellaneous. In lower-income households, expenditures on food (–4.1 US$, 95% CI –7.2 to –1.1), restaurant and café meals (–2.0 US$, 95% CI –3.1 to –0.8), spices (–0.4 US$, 95% CI –0.7 to –0.09), and rent (–3.1 US$, 95% CI –4.5 to –1.6) were significantly crowded out.

Conclusion

This research demonstrates that NCD-related spending in Bangladesh reduces budgets for both food and non-food expenditures, with a stronger crowding-out effect on food items and rent, particularly in lower-income households. Effective financial and social protection mechanisms against NCDs are warranted to safeguard the consumption of the NCD-affected households in Bangladesh.

Economic evaluation of a person-centred care intervention with a digital platform and structured telephone support for people with chronic heart failure and/or chronic obstructive pulmonary disease: results from a randomised controlled trial in Sweden

Por: Harvey · B. P. · Barenfeld · E. · Fors · A. · Ekman · I. · Swedberg · K. · Gyllensten · H.
Objectives

The aim of the study was to evaluate the healthcare costs and effects of a remote person-centred care (PCC) add-on intervention compared with usual care for people with chronic heart failure (CHF) and/or chronic obstructive pulmonary Disease (COPD) from a societal perspective.

Design

A cost-effectiveness analysis (CEA) based on the results from a randomised controlled trial.

Setting

The study was conducted from August 2017 until June 2021 within nine primary care centres across Western Sweden.

Participants

Participants in the study had a diagnosis of COPD (J43.0, J44.0–J44.9) and/or CHF (I50.0–I50.9).

224 patients were randomly allocated to the study groups. After two withdrawals, the final intention-to-treat analysis included 110 participants in the intervention group and 112 in the control group.

Interventions

Both the intervention and control group received usual care through their primary care centres. In addition, the intervention group participated in a remote PCC add-on intervention consisting of a digital platform and structured telephone support.

Primary outcome

Incremental cost-effectiveness ratio using direct healthcare costs, productivity loss and prescription drug costs, compared with health effects measured using the EuroQoL questionnaire (EQ-5D-3L) over a 2-year time horizon.

Results

The intervention group had lower healthcare utilisation in inpatient care, specialised outpatient care and reduced productivity loss. The CEA showed incremental effects of 0.0469 quality-adjusted life years and incremental costs of SEK –68 533 (Swedish crowns). The PCC alternative was both more effective and resulted in lower healthcare costs compared with usual care, that is, PCC was dominant.

Conclusions

The results of this CEA demonstrated that a remote PCC add-on intervention for people with COPD and/or CHF had lower healthcare costs and higher health-related quality of life compared with usual care.

Trial registration number

NCT03183817 ClinicalTrials.gov.

Model-based pharmacoeconomic analysis of anti-VEGF strategies for neovascular age-related macular degeneration: a value-based comparison of real-world administration approaches

Objectives

To evaluate the cost-effectiveness of anti-vascular endothelial growth factor (VEGF) treatments for neovascular age-related macular degeneration (nAMD) using a value-based model that considers drug durability, dosing regimens and real-world administration strategies, including safe vial fractionation.

Design and setting

Model-based pharmacoeconomic analysis using data from randomised clinical trials and network meta-analyses. Analysis conducted from the payer perspective using cost data from the Spanish National Health System.

Methods

A model-based analysis compared five anti-VEGF agents—innovator and biosimilar ranibizumab, aflibercept 2 mg, brolucizumab and faricimab—across three dosing regimens: fixed, Pro Re Nata and Treat-and-Extend (TAE). Administration formats included single-use vials, prefilled syringes and vial fractionation (VF), with or without dead-space-free (DSF) syringes to minimise waste. The primary outcome was cost per optimal responder, defined as a patient gaining ≥15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters, with and without adverse events. Cost-effectiveness was evaluated using Number Needed to Treat (NNT), Net Efficacy Adjusted for Risk-NNT (adjusted for safety) and incremental cost-effectiveness ratios. Secondary outcomes included the number of treated patients and optimal responders achievable within a fixed 1 000 000 budget.

Results

The most cost-effective strategy was aflibercept 2 mg under a TAE regimen using DSF VF, with a total cost of 6214 per patient and a cost per optimal responder of 27 155. Under a fixed budget of 1 000 000, this approach allowed treatment of 160 patients, yielding 36 optimal responders. Faricimab with DSF VF ranked second, with a total cost of 5847 and a cost per optimal responder of 28 652, treating 171 patients and achieving 34 responders. In contrast, single-use vials without VF led to substantially higher total costs (eg, 11 305 for aflibercept TAE) and lower treatment capacity (eg, 88 patients treated).

Conclusions

This model demonstrates that combining durable agents, extended dosing intervals and optimised delivery strategies (eg, prefilled syringes and DSF VF) can substantially improve the cost-effectiveness and sustainability of anti-VEGF therapy in public health systems.

Cost analysis of post-stroke dysphagia during acute hospitalisation: a cross-sectional study in Vietnam

Por: Thong · T. H. · Hien · N. T. T. · Tung · L. T. · Thang · P. · Trung · T. H. · Tien · V. D. · Hanh · H. T. · Lam · T. H.
Objectives

To estimate the healthcare costs associated with post-stroke dysphagia during acute hospitalisation and to identify factors influencing these costs in a tertiary hospital setting in Vietnam.

Design

A cross-sectional study using clinical and billing data from hospital records.

Setting

The study was conducted at the Neurology Center of Bach Mai Hospital, a tertiary care facility in Hanoi, Vietnam, between June 2020 and January 2022.

Participants

A total of 951 patients aged ≥18 years with acute ischaemic stroke confirmed by CT or MRI were included. Dysphagia was assessed using the Gugging Swallowing Screen.

Outcome measures

Direct healthcare costs during hospitalisation were collected from the hospital billing system and categorised as medications, diagnostic imaging, medical supplies, accommodation, food, procedures and laboratory tests. All costs were converted to USD. Associations between patient characteristics and total healthcare costs were analysed using generalised linear models (Gamma distribution with log link), applying robust standard errors.

Results

The median treatment cost was 10.08 million VND (436.24 USD) in the dysphagia group vs 6.37 million VND (275.78 USD) in the non-dysphagia group. Costs increased with dysphagia severity, reaching 22.64 million VND (979.49 USD) among patients with severe dysphagia. In multivariate analysis, dysphagia was associated with a 21% increase in costs (exp(β) = 1.21; 95% CI 1.10 to 1.33; p14, pneumonia, prolonged hospitalisation and higher educational level.

Conclusions

Post-stroke dysphagia substantially increases acute hospitalisation costs in Vietnam. Early screening, standardised management and preventive care for complications may improve outcomes and reduce costs.

Trial registration number

The study was registered on the Research Registry website (https://www.researchregistry.com/) under the unique identification number: researchregistry8203.

Estimating the economic and societal burden of suicide and suicide attempts in India: a study protocol

Por: Jain · N. · Wijnen · B. · Lohumi · I. · Madan · J. · Evers · S.
Introduction

Suicide has a substantial economic burden, yet in low- or middle-income countries, the analysis is constrained by the absence of disaggregated economic data. Existing studies primarily rely on gross costing methods, overlooking sector-specific costs such as healthcare, law enforcement and productivity losses at the family and community levels. Furthermore, the societal perspective, essential for understanding the multifaceted economic impacts of suicide, remains insufficiently explored in the Indian context.

Methods and analysis

This study will quantify the economic burden of suicide and suicide attempts in India. The initial phase will involve developing a resource-use measure instrument to capture the societal costs associated with suicide. Subsequently, a retrospective, incidence-based study will be conducted in India using the developed instrument. This will also be complemented with the Health-Related Quality of Life Assessment using EuroQOL (EQ-5D-5L). Exploratory subgroup cost comparisons (eg, suicide methods, healthcare facility type) will use non-parametric tests, including the Mann–Whitney U and Kruskal–Wallis tests. Generalised linear mixed models (gamma distribution and log link) will analyse longitudinal cost and HRQoL data, besides bootstrapping techniques and sensitivity analyses.

Ethics and dissemination

Ethics approval was obtained for the study from the institutional review board of the Indian Law Society (ILS/141/2024). All data collected during the study will adhere to ethical guidelines, taking informed consent. The findings of the study will be disseminated through peer-reviewed journals to aid data-driven decision-making.

Trial registration

Clinical Trial Registry of India (CTRI/2025/02/080380), date of registration: 12/02/2025.

Determining the indirect costs of suicide in Sweden: a national population-based cross-sectional study, 2010-2019

Por: Wikström · D. · Nystrand · C. · Hadlaczky · G. · Gedin · F.
Objectives

Globally, more than 700 000 people commit suicide annually. In Sweden, the yearly incidence ranges between 1000 and 1500 people, which is higher than the global average. The aim of this study is to estimate the economic burden related to indirect costs that suicide has imposed on Swedish society between 2010 and 2019.

Design

National population-based cross-sectional study.

Participants and setting

All suicides in Sweden between 2010 and 2019, using data from the Swedish National Cause of Death Registry.

Outcomes

Indirect costs associated with suicides, estimated using the human capital approach, including productivity loss over 1-year and lifetime horizons.

Results

Between 2010 and 2019, 1406 to 1591 suicides occurred annually in Sweden, resulting in approximately 26 500 productive life years lost each year. In 2019, the productivity loss due to suicides was estimated at 44 million over a 1-year horizon and 935 million over a lifetime horizon. The corresponding per-person costs were 37 000 and 778 000, respectively.

Conclusions

This study provides valuable insights into the economic burden of suicide on Swedish society. It underlines the potential economic benefits of effective suicide prevention, aligning with previous research highlighting the substantial returns—both monetary and in terms of human well-being—that successful prevention strategies can yield.

Health economic assessment of the Landes public service 'Vivre a Domicile (VIVADOM) (Living at Home) based on personalised care for frail older adults with human support and digital solutions (telecare, tablet, home automation and connected objects): a Ma

Por: Sawadogo · A. R. · Gayot · C. · Nys · J. F. · Le Goff-Pronost · M. · Tchalla · A.
Introduction

Preventing loss of autonomy has become a public health issue due to the increase in healthcare costs associated with ageing. It has become even more pressing with the arrival of the baby-boomer generation. This has given rise to several initiatives. This is the background to the VIVADOM project. The project provides a complete kit for older adults aged 60 years and over living at home. First, the kit includes a technological package (telecare, light path and digital tablet). Then, these older adults benefit from personalised human support provided by postal workers trained in gerontology. The aim of this study will be to carry out a health economic assessment (HEA) of the VIVADOM project as part of the prevention of frailty and/or dependency (by comparing beneficiaries of the complete kit with non-beneficiaries). The comparator will be the fact of not benefiting from the complete kit. In addition, the efficiency of the project in preventing falls and cognitive problems will be studied. We will calculate three incremental cost-effectiveness ratios (ICER) for these three issues.

Methods and analysis

The economic model used will be the Markov model. Transition probabilities, average costs and average quality-adjusted life year (QALY) will be calculated for the two groups being compared. The ICER will be obtained by dividing the difference in average costs by the difference in average QALYs. Finally, ICERs will be compared with willingness-to-pay (WTP) to assess the efficiency of the system. Thus, the VIVADOM project will be efficient when these ICERs are lower than the WTP. Univariate and probabilistic sensitivity analysis will be carried out to ensure the robustness of the analysis results.

Ethics and dissemination

The HEA of the VIVADOM project has been approved by the research unit of the University of Limoges in France. The results will be published in a peer-reviewed journal and presented at relevant national and international conferences.

Budget impact analysis of glecaprevir/pibrentasvir treatment for hepatitis C in Iran: a modelling study

Por: Jafari · M. · Mehdizadeh · P. · Keshavarz · K. · Teymourzadeh · E. · Abyazi · M. A. · Gholamreza Shirazi · P.
Objectives

Glecaprevir/pibrentasvir (GLE/PIB), despite being a highly costly medication, is considered a cost-effective approach compared with sofosbuvir/velpatasvir (SOF/VEL) and sofosbuvir/daclatasvir (SOF/DCV) in the treatment of hepatitis C virus (HCV) infection. No study has evaluated the effect of GLE/PIB’s introduction into Iran’s drug list from a health policy perspective and estimated the budgetary impact change. Therefore, this study was conducted to analyse the fiscal effect of the introduction of GLE/PIB into Iran’s drug list.

Design

Budget impact analysis. The assumptions and costs of including GLE/PIB in Iran’s drug list for the treatment of patients with hepatitis C were derived from a conducted cost-effectiveness analysis.

Setting

National level. In this study, the budgetary changes in Iran’s pharmaceutical market and health system, from the Ministry of Health’s perspective, have been estimated for a 5-year time horizon following the introduction of GLE/PIB in the country.

Results

Based on the results obtained from the budget impact model, currently, 4112 patients are receiving SOF/DCV and SOF/VEL therapeutic regimens, which is expected to decrease to 1093 in 2029 owing to the affordability of medications and a 50% estimated market share for GLE/PIB. According to the results, with the introduction of GLE/PIB into the market and assuming a market share of 10% in the first year, growing to 50% by the fifth year, the healthcare system costs will increase by approximately $0.61, $1.77, $3.86, $7.45 and $13.51 million over the next 5 years, respectively. Additionally, based on the drug’s selling price, there will be a 468% increase in hepatitis C drug market costs after 5 years, resulting in an overall budget increase of approximately 0.13% for Iran’s pharmaceutical market. According to the sensitivity analysis, a 20% reduction in chronic hepatitis C (CHC) costs could decrease the projected increase in health sector costs from $13.51 million (an 18.84% increase) to $10.52 million (an 18.16% increase). Conversely, a 20% rise in CHC costs would raise those costs to $16.49 million (a 19.31% increase).

Conclusion

Considering the high price of the GLE/PIB compared with the available options in Iran, with the introduction of GLE/PIB into Iran’s drug list, insurance coverage and appropriate allocation of necessary resources, a reduction in the cost burden because of hepatitis C treatment is expected for individuals and households. Additionally, with a well-regulated market share of existing medications, the optimal treatment choice for patients will be feasible.

Study Protocol for a Delphi Process to Develop a Climate Impact Extension to the Consolidated Health Economic Evaluation Reporting Standard (CHEERS) 2022--the CHEERS ClimatE Checklist

Por: Oldenburg · J. · Keil · M. · Maass · L. · Lange · O. · Rogowski · W.
Introduction

The healthcare sector has significant environmental impacts, particularly through greenhouse gas emissions. Reducing its climate footprint is therefore essential for achieving political goals such as net-zero and climate-friendly healthcare. While health economic evaluation (HEE) methods compare the costs and consequences of two or more interventions, these analyses rarely consider climate impacts. Some studies have begun to determine climate impacts parallel to or integrated into HEEs. Life cycle assessment (LCA) could be used to integrate climate impacts by considering these results as effects or monetised climate footprints. However, a reporting standard is needed for using these climate-extended economic evaluations in evidence-based decision-making. This protocol describes using an online Delphi process to incorporate climate impacts into the Consolidated Health Economic Evaluation Reporting Standard (CHEERS), called CHEERS Climate Extension (CHEERS ClimatE).

Methods and analysis

The development of CHEERS ClimatE will proceed through five key stages. First, the preliminary steering group develops in consultation with an advisory board a proposal for the CHEERS ClimatE reporting standard based on a transparency checklist that combines three standards for carbon footprint calculations into the CHEERS framework. The mapping was complemented by reviewing studies that incorporate climate impacts in HEE. Second, for the Delphi process, international experts in HEE and LCA with at least one year of academic experience will be invited to participate in an online pre-survey. We aim to recruit at least 40 participants. Expecting various drop-outs, we aim to reach a consensus with at least 20 participants per Delphi round. Third, an expected three-round Delphi process will be conducted to validate and refine the proposed elements. Participants will rate each item using a 9-point Likert scale and will have the opportunity to comment on each item and propose new items. Consensus is defined with the target of a 70% agreement. Unless consensus is reached, a moderated video conference may be held as a fourth round. Fourth, following other CHEERS extensions, the consented checklist will be piloted using thematically relevant case studies. While substantial changes are not anticipated, minor revisions to individual items may be considered and ratified by the steering group and advisory board. The fifth stage is the publication of the final checklist.

Ethics and dissemination

This study has been approved by the ethics committee of the University of Bremen (2024–25). The findings of the Delphi study will be published in a peer-reviewed journal and presented at conferences.

Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis

Por: Majda · T. · Lee · J. S. · Curtis · R. · Kowal · S. L.
Objectives

Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how health effects and costs from funding an intervention are distributed among population subgroups. The objective was to evaluate how funding emicizumab for people with severe haemophilia A (PwSHA) impacts population health and health disparities in the USA.

Design

Population-level model of PwSHA from the perspective of the US healthcare system, using published sources and considering a lifetime time horizon.

Intervention

Emicizumab versus other haemophilia A prophylaxis treatments.

Primary outcome measures

Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.

Results

When an estimated 6512 PwSHA in the USA were treated with emicizumab, the US healthcare system would save US$160 billion over those individuals’ lifetimes. If these cost savings fund additional healthcare interventions in the overall population, funding emicizumab would improve overall US population health (1 068 903 QALYs gained, using a threshold of US$150 000/QALY) and reduce existing overall US inequities (–0.01% on the Atkinson index).

In all scenarios tested for sensitivity, increased emicizumab and prophylaxis utilisation led to further reductions in health disparities and greater increases in population health. Results were robust to deterministic variations in the allocation of cost savings due to emicizumab use.

Conclusion

Funding emicizumab treatments for PwSHA improves overall population health and reduces overall health inequities in the USA. Cost savings from the use of emicizumab free up important resources that can be leveraged to support other healthcare interventions, but decisions on how these funds are used have large consequences for equity.

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