BMJ openArun S Karlamangla, Duncan Thomas, Sharon Stein Merkin, Kari A Moore, Karol E Watson, Teresa E Seeman
OBJECTIVE: Multiple prospective cohort studies, including the multi-ethnic study of atherosclerosis (MESA), have documented elevations in cardiovascular risk factors after the Great Recession (GR) of 2008, despite other studies that report mortality declines after the GR. It is possible that the deleterious effects of the GR on cardiovascular risk profiles are short-lived and reversed. We examine whether risk factor elevations seen in MESA in the 2010-2011 wave persist into the 2016-2018 wave of data collection. DESIGN: Individual fixed effects (IFE) analysis of prospective cohort data from MESA, with rate of change in the risk factor (from wave 2 to later waves) as the dependent variable, effectively controlling for individual-specific linear trends in ageing and all time-invariant characteristics. Models included controls for time-varying characteristics. SETTING: Communities centred around Baltimore, Maryland; Chicago, Illinois; Forsyth County, North Carolina; Los Angeles, California; New York City, New York; and St. Paul, Minnesota. PARTICIPANTS: All MESA participants who participated in at least two pre-GR waves and both post-GR waves; n=3101 (47% men, 25% African American, 21% Hispanic, 13% Asian). OUTCOMES: Primary: Systolic blood pressure (SBP), pulse pressure (PP), fasting blood glucose, serum C-reactive protein (CRP). Secondary: Serum low density lipoprotein cholesterol (LDL-C) and Body Mass Index (BMI) RESULTS: Mean SBP and PP in 2016-2018 were greater by 8.6 (95% CI 5.9 to 11.4) and 8.0 (95% CI 6.0 to 9.9) mm Hg, respectively, than predicted by individual-specific pre-GR linear ageing trends, and fasting blood glucose was 6.3% higher (95% CI 4.6 to 8.1). In comparison, corresponding increases in 2010-2011 were 2.5 and 3.0 mm Hg and 3.3%. CRP increased by 29.7% (95% CI 21.8 to 38.2) in 2010-2011 and 45.1% (95% CI 28.3 to 64.2) in 2016-2018. Mean BMI did not change in 2010-2011 but decreased by 0.5 kg/m2 (95% CI -0.7 to -0.2) in 2016-2018. LDL-C decreased by 4.4 (95% CI -6.5 to -2.2) and 5.2 (95% CI -9.5 to -0.9) mg/dL in the two post-GR waves. CONCLUSIONS AND RELEVANCE: Post-GR increases in blood pressure, blood glucose and CRP in the USA not only persisted for 8-10 years after the GR but also grew in magnitude. In contrast, mean BMI and mean LDL-C reduced post-GR. Further studies are needed to uncover mechanisms (behavioural or otherwise) underlying the BMI and LDL-C reductions seen here, and the mortality consequences of these risk factor changes.
Journal of oral rehabilitationMattias Ulmner, Elizabeth Stenwall, Selihattin Mermer, Bodil Lund
BACKGROUND: Temporomandibular joint (TMJ) involvement in rheumatic inflammatory arthropathies (RIA) has been sparsely described, and evidence regarding local treatment options for affected TMJs remains limited. OBJECTIVES: To perform a systematic review (SR) regarding local treatments for TMJ involvement in RIA and evaluate their effects on pain management, disease improvement, quality of life, health economics, and adverse events. METHODS: According to the PICO framework, patients with RIA were defined as the population, local TMJ treatment as the intervention, and alternative treatments, placebo, or no treatment as comparators. Outcome measures included maximum interincisal opening (MIO) and TMJ pain. A comprehensive search strategy was developed and applied to five databases. RESULTS: The search generated 14 193 studies of which 33 met the inclusion criteria. All included studies had moderate or high risk of bias. Meta-analyses were conducted with due consideration of these limitations. Patients with juvenile idiopathic arthritis (JIA) or RIA (excluding JIA) having total joint replacement were analysed and JIA patients demonstrated better outcome both regarding MIO (p = 0.006, I2 = 86.8%) and TMJ pain (p = 0.02, I2 = 82.2%). No significant findings were observed in the remaining meta-analyses. A critical appraisal suggests that intra-articular corticosteroid injections might be beneficial for JIA patients. CONCLUSION: The listed results have a low level of evidence and should be interpreted with caution. Nevertheless, this appears to be the only SR specifically evaluating local TMJ treatments in RIA. Ways of decreasing bias and increasing cohort sizes seem necessary for expanding knowledge.
VaccineHuyen Thi Thanh Dang, Andrew Clark, An Le Thanh Nguyen, Hong Thi Duong, Trung Thanh Nguyen, Thanh Trung Tran, Lam Ngoc Tung Cu, Dung Chi Tham, Nga Tuyet Nguyen…
BACKGROUND: In Vietnam, rotavirus remains a leading cause of severe acute gastroenteritis resulting in hospitalization. Although rotavirus vaccines are available in the private sector-primarily accessible to urban children-the Expanded Programme on Immunization has only piloted the vaccine in select provinces. This study assessed the impact and cost-effectiveness of national rotavirus vaccination in Vietnam to inform policy decision on nationwide implementation. METHODS: We used a transparent static cohort model with a granular weekly age structure (< 5 years) to project rotavirus gastroenteritis (RVGE) cases, visits, hospitalizations, and deaths in children under five years, comparing no vaccination to nationwide vaccination with the domestic ROTAVIN vaccine over 10 years starting in 2026. Model inputs drew from global, regional, and local data including pre-vaccine incidence in Vietnam, local vaccine effectiveness and waning data, and treatment costs (governmental and societal perspectives) sourced from a local cost of illness study and hospital records (converted to 2025 USD). Deterministic and probabilistic sensitivity analyses addressed parameter uncertainty. RESULTS: Over ten years, ROTAVIN vaccination was projected to reduce RVGE health burden by 42%, averting 97,776 (95% uncertainty interval 72,722-342,311) hospital admissions and 433 (330-1300) deaths. The program would cost US$39.5 million ($30.7-$117.7 million) for ten birth cohorts (2026-2036). From the governmental perspective, at least 62% of vaccination costs would be offset by healthcare savings. The cost per DALY averted was estimated at $982 (governmental perspective), while the program was cost-saving from the societal perspective in the base case. Sensitivity analyses confirmed robustness, with a 99% probability of cost-effectiveness from the governmental perspective at a willingness-to-pay threshold of 0.5 times Vietnam's GDP per capita (and 100% from the societal perspective). CONCLUSIONS: Nationwide ROTAVIN vaccination could avert a substantial health and economic burden in Vietnam and would be highly cost-effective given recently published thresholds.
Professional status in healthcare is determined not only by educational preparation or statutory scope of practice, but also by how a profession functions within reimbursement systems, labor markets, and healthcare delivery. This article applies a health economics framework to evaluate the current professional status of audiology in the United States using observable economic indicators rather than aspirational goals or regulatory intent. Drawing on evidence synthesized from companion analyses of workforce trends, professional earnings, educational investment, reimbursement policy, and labor market dynamics, the review examines how reimbursement authority, pricing power, workforce growth, earnings trajectories, and productivity collectively reflect professional standing. The analysis demonstrates that, despite doctoral-level education and expanding clinical competencies, audiology remains constrained by limited reimbursement authority, restricted pricing autonomy, narrow service valuation, and clinician-dependent delivery models that limit workforce scalability and economic returns. These structural conditions contribute to constrained earnings growth, workforce instability, and increasing debt-to-income pressures, placing the profession in an intermediate economic position relative to other doctoral health professions. The article concludes that professional advancement should be evaluated through measurable economic outcomes and that meaningful progress toward autonomous professional status will require coordinated alignment of reimbursement, revenue architecture, labor deployment, and professional classification within the U.S. healthcare system.
BMJ openJonathan A Staloff, Melinda J B Buntin, Bianca K Frogner
OBJECTIVES: To assess how 21st-century healthcare spending grew in four high-income countries, to examine the extent to which healthcare workforce growth is associated with spending growth and to compare how the composition of the healthcare workforce has changed over time and across countries. We hypothesised that (1) healthcare spending growth patterns in the USA, the UK, Australia and Canada would be similar after the Great Recession; (2) healthcare workforce growth would be positively correlated with healthcare spending growth across countries and (3) growth in the healthcare workforce would be driven by non-physician and non-nursing workers across all countries. DESIGN: Retrospective, descriptive cross-national longitudinal analysis. SETTING: National-level data from four high-income countries (Australia, Canada, the UK, the USA) using 2023 Economic Co-operation and Development Health Statistics and the WHO's National Health Workforce Accounts Data Portal. PARTICIPANTS: Australia, Canada, the UK, the USA. INTERVENTIONS: None. PRIMARY AND SECONDARY OUTCOME MEASURES: Primary outcomes were healthcare spending growth as a per cent of gross domestic product and per capita, and healthcare workers as a per cent of total civilian employment and per capita. Secondary outcomes included growth and composition of practising physicians, nursing personnel and 'other' healthcare workers. RESULTS: Healthcare employment and healthcare spending grew in all four countries over the study period. Healthcare employment accounted for over 90% of the variance in health spending over time in the USA, Canada and Australia and over 80% in the UK. Spearman correlation coefficients between employment and spending levels were 0.94 for Australia, 0.92 for Canada and 0.90 for both the UK and USA. While the USA led in healthcare spending growth prior to the Great Recession, its post-recession spending growth rate was similar to peer countries. Physicians and nurses collectively comprised less than one-third of total healthcare employment, with 'other' healthcare workers constituting the majority and a fast-growing component. CONCLUSIONS: Results were consistent with all three hypotheses. Healthcare workforce growth was strongly correlated with spending growth within each country, reinforcing the centrality of labour force dynamics to healthcare spending trends.
BACKGROUND: Differentiated service delivery (DSD) models have been introduced to optimise HIV care by tailoring services to patient needs, but evidence on their cost-effectiveness in Ethiopia is limited. OBJECTIVE: To evaluate the cost-effectiveness of DSD models for HIV care compared with standard of care (SOC) in North Wollo, Ethiopia. STUDY DESIGN: Retrospective economic evaluation using bottom-up microcosting from a healthcare provider perspective. SETTING: One hospital and three health centres in North Wollo Zone, Ethiopia, from January 2024 to December 2024. PARTICIPANTS: 786 adults (≥18 years) on antiretroviral therapy, including 393 receiving DSD (across nine models: 6-month multimonth dispensing, 3-month multimonth dispensing, appointment spacing, community antiretroviral therapy (ART) groups (CAG), FastTrack, maternal and child health, peer-led, adolescent groups and key population models) and 393 receiving SOC. MAIN OUTCOME MEASURES: Total annual cost per patient (USD), retention in care at 12 months (%) and incremental cost-effectiveness ratios (ICERs) expressed as cost per additional patient retained. STATISTICAL ANALYSIS: Costs were annualised at a 3% discount rate. ICERs were calculated comparing each DSD model to SOC. Deterministic one-way sensitivity analysis (±20% for medication cost and retention rate) assessed robustness. RESULTS: SOC had the highest annual cost per patient (US$295) and lowest retention (76.6%). All DSD models were dominant (lower cost, higher retention) with negative ICERs. CAG was the most cost-effective (US$199 per patient; 93.0% retention; ICER -5.85), followed by FastTrack (US$212; 92.9%; -5.09) and adolescent groups (US$238; 90.5%; -4.10). Sensitivity analyses confirmed robustness; no scenario reversed dominance. CONCLUSION: DSD models, particularly CAG, FastTrack and adolescent groups, provide more cost-effective HIV care than SOC in Ethiopia by achieving higher retention at lower costs. Scaling up these models could optimise resource allocation and reduce health system burden.
JMIR research protocolsStephen Morris, Jenny Shand, Theo Georghiou, Kevin Herbert, Rachel Lawrence, Raj Mehta, Pei Li Ng, Lucina Rolewicz, Holly Elphinstone, Holly Walton
BACKGROUND: Ambient voice technology (AVT) uses conversational AI to record and organize clinical consultations in real time. It is being adopted quickly across the National Health Service (NHS). However, evidence about its effects on productivity, costs, or staff experience across different health care settings is limited. Phase 1 of this research program developed a taxonomy, logic model, and outcome framework for evaluating AVT. Phase 2 will carry out a multisite, mixed methods evaluation of AVT in 4 NHS trusts. These include mental health outpatient services, acute hospital outpatient clinics, and accident and emergency departments. OBJECTIVE: This study aims to explore the real-world impact of AVT on productivity, costs, and staff experience across NHS adult services. METHODS: The study includes three parts: (1) a quantitative quasi-experimental analysis of routine NHS data to estimate the impact of AVT on documentation time, clinician activity, and service outcomes; (2) a comprehensive health economic evaluation comprising cost-consequence analysis, cost-benefit analysis, and budget impact modeling; and (3) interviews with up to 36 staff from 3 services (mental health outpatient, acute hospital outpatient, and accident and emergency) to explore their experience of using AVT, their views on its impact, and what helps and gets in the way of its use. Sites will be chosen to include different care settings, organizational environments, and AVT products. Quantitative and economic analyses will use NHS electronic health records and national datasets to understand changes over time and measure the impact of AVT. Interview data will be analyzed using thematic analysis and rapid assessment procedures to identify key themes. All study findings will be combined to give a clear view of AVT and its impact. RESULTS: Data collection is expected to begin in August 2026 and conclude by January 2027. Publication of results is anticipated in February 2027. CONCLUSIONS: This evaluation will provide real-world evidence on the productivity, economic, and experiential impacts of AVT in the NHS. Outputs will include peer-reviewed papers, a slide-deck summary for the funder, and a publicly available health economic decision-support tool to help NHS organizations decide whether they should adopt AVT.
JMIR research protocolsAnne F Klassen, Charlene Rae, Shane D Morrison, Natasha Johnson, Karine Khatchadourian, Jack Graesser, Manraj N Kaur
BACKGROUND: The need for gender-affirming care (GAC) for young people has increased rapidly worldwide. GAC includes social, psychological, and medical interventions aimed at alleviating gender-related distress. Given that many outcomes of GAC relate to how young people function and feel, a rigorously developed patient-reported outcome measure (PROM) is needed. To address this need, GENDER-Q Youth was developed with extensive input from youth with lived experience. Concept elicitation interviews were performed with 47 youth from Canada and the United States. A conceptual framework and draft PROM were developed and refined with feedback from 33 experts and 17 youth, and were pilot tested with 406 older youth. The field test version of GENDER-Q Youth includes 16 independently functioning scales (248 items) that measure health-related quality of life, gender practices, voice, and experience of care. OBJECTIVE: This paper outlines the protocol for an international cross-sectional study to field test GENDER-Q Youth in a sample of transgender and gender diverse (TGD) adolescents and young adults aged 12 to 25 years receiving GAC. METHODS: This study follows international guidelines for PROM development and validation (eg, US Food and Drug Administration and Consensus-Based Standards for the Selection of Health Measurement Instruments [COSMIN]). GENDER-Q Youth was translated into Danish, Dutch, and German following the Professional Society for Health Economics and Outcomes Research (ISPOR) guidelines for the translation and cultural adaptation of PROMs. For the field test, TGD youth are being recruited using multiple strategies (eg, face-to-face, emails, and so on). A REDCap survey is used to collect sociodemographic and clinical data. Branching logic is used to ensure relevant GENDER-Q Youth scales are completed as some scales are gender-specific. For psychometric analysis, Rasch measurement theory (RMT) analysis will be used to examine fit of the observed data to the Rasch model. A series of tests and criteria will examine item fit and scale reliability and validity. Test-retest reliability will be examined with intraclass correlation coefficients. For construct validity, 147 predefined hypotheses of expected group differences and correlations between GENDER-Q Youth scales will be examined. Acceptance of at least 75% of hypotheses is considered sufficient evidence of construct validity per COSMIN criteria. RESULTS: The research described in this protocol is funded by a Canadian Institutes of Health Research Sex and Gender Science Chair (April 2020) and supported by a Canada Research Chair in Patient-Reported Outcomes (April 2025). With the Danish, Dutch, and German translations now completed, the international field test is underway in Canada, Europe, Australia, and the United States. Data collection and analysis are expected to be completed by the end of 2026. CONCLUSIONS: This protocol describes the international GENDER-Q Youth field test study. Once developed, GENDER-Q Youth can be used to inform GAC, research, and quality improvement efforts.
BMJ openStefanie Lip, Pieter Theo Pepler, Alex McConnachie, Leslie J Sheffield, Sam Mostafa, Linsay McCallum, Neil Hawkins, Olivia Wu, Louise Bennett, Iain Frater, Kat…
INTRODUCTION: Pharmacogenomic (PGx) panel testing may reduce adverse drug reactions (ADRs), treatment failures and downstream healthcare use by aligning prescribing with germline genetic variation. The PREPARE trial reported an approximately 30% reduction in clinically relevant ADRs using a pre-emptive multigene panel, but its open-label design, patient-reported endpoint and subsequent methodological critiques limit its use for National Health Service (NHS) decision-making. Whether panel-based PGx delivers benefit in acute NHS secondary care, where prescribing is time-critical, patients are older and multimorbid and outcomes can be linkage-adjudicated, remains unknown. METHODS AND ANALYSIS: PHOENIX is a pragmatic, pilot, parallel-group, individually randomised controlled trial with blinded outcome adjudication, in NHS Greater Glasgow and Clyde (NHSGGC) hospitals and NHS Golden Jubilee National Hospital. Adults newly prescribed an eligible PGx-relevant index drug are identified through a data-driven screening system integrating electronic prescribing, community prescribing linkage and record review and randomised 1:1 to PGx-guided care or standard care with delayed PGx testing. In the intervention arm, buccal-swab DNA is genotyped (Clinical Laboratory Improvement Amendments/College of American Pathologist (CLIA/CAP)-accredited laboratory) and a Clinical Pharmacogenetics Implementation Consortium (CPIC)/Dutch Pharmacogenetics Working Group (DPWG)-based report returned within approximately 10 days; results are research-use-only. The trial aims to recruit 2000-4000 participants constrained by a fixed operational end-date (30 September 2026). The primary outcome is index-drug-related ADR or treatment failure within 3 months, adjudicated using Common Terminology Criteria for Adverse Events (CTCAE) V.6 and validated causality tools, with deterministic linkage via the West of Scotland Safe Haven. Secondary outcomes include ADR severity, hospitalisations, mortality, actionable prescribing uptake, biomarker levels, EuroQol 5-Dimension 5-Level questionnaire (EQ-5D-5L) and healthcare utilisation. The primary analysis is intention-to-treat logistic regression adjusted for stratification factors; a within-trial cost-utility analysis estimates cost per quality-adjusted life year (QALY). ETHICS AND DISSEMINATION: Approved by Scotland A Research Ethics Committee (24/SS/0064; IRAS 344412; NHS R&I INGN24MG113). Co-sponsors are NHSGGC and the University of Glasgow. Findings will be disseminated via peer-reviewed publication, conferences, patient and public involvement and engagement-co-produced lay summaries and the trial website. TRIAL REGISTRATION NUMBER: NCT06907784.
InjuryHenry Olivera Perez, Anna Huang Perez, Andrea M Gochi, Nathan Alcasid, Lucas Thornblade, Naveen Balan, Gregory P Victorino, April Mendoza, Adam Gutierrez, Genn…
INTRODUCTION: Recent studies have shown thoracic irrigation after tube thoracostomy drainage of traumatic hemothorax (HTX) reduces the rate of retained HTX and need for secondary interventions such as video-assisted thoracoscopic surgery (VATS) but the cost-effectiveness of thoracic irrigation is not well-studied. We hypothesized that thoracic irrigation after thoracostomy tube drainage of traumatic HTX would be more cost-effective compared to standard tube thoracostomy drainage. METHODS: A decision-analytic model was created to evaluate the cost-effectiveness of thoracic irrigation after thoracostomy tube placement for traumatic HTX. Our base case was a 38-year-old hemodynamically stable patient presenting with a traumatic HTX requiring thoracostomy tube placement on presentation. Cost, probabilities, and utilities were obtained from the published literature, with cost represented in 2025 US dollars. Utilities were represented in Quality-Adjusted Life Years (QALY's). One-way sensitivity analysis was performed. RESULTS: The decision-analytic model identified that thoracic irrigation after thoracostomy tube placement for traumatic HTX was the dominant strategy with lower cost at $17,818 with QALY 0.69 compared to no irrigation at $34,527 with QALY 0.67. The strategy of thoracic irrigation demonstrated an increased net monetary benefit compared to no irrigation ($51,086 vs $32,581) with increased QALY. Using one-way sensitivity analysis, thoracic irrigation was cost-effective when its net monetary benefit surpasses $33,581. CONCLUSION: In hemodynamically stable patients, adoption of routine thoracic irrigation for traumatic HTX is cost-effective providing additional benefit at less cost. Adoption of this management strategy should be strongly considered as it may reduce cost and improve quality of life, in addition to previously demonstrated improvement in clinical outcomes.
Journal of the American College of Clinical Pharmacy : JACCPBrian Murray, Jackie Rowe, Duncan X Dobbins, Phuong Duong, Megan Kunka Fritz, Madison Brooke Grizzle, Steven T Johnson, Abbie D Leino, Rajsumeet Macwan, Farah …
Artificial intelligence (AI) is increasingly shaping the pharmaceutical industry. This ACCP commentary examines the implications of AI for industry-based clinical pharmacists across the pharmaceutical life cycle, including drug development, regulatory affairs, medical affairs, health economics and outcomes research, and pharmacovigilance. Artificial intelligence-enabled tools may support target identification, clinical trial design, regulatory intelligence, evidence synthesis, medical content generation, real-world evidence analysis, economic modeling, adverse event processing, and safety signal detection. As these tools mature, the role of the clinical pharmacist is likely to shift from primarily task execution toward clinical interpretation, quality assurance, strategic decision-making, and governance of AI-supported outputs. However, AI implementation also introduces important risks and practical implementation challenges. These limitations reinforce the need for clinical pharmacists to remain actively engaged as human-in-the-loop experts who can assess whether AI-generated insights are scientifically valid, clinically relevant, ethically sound, and appropriate for decision-making. Ultimately, AI may expand the reach and efficiency of pharmaceutical industry functions, but successful integration will depend on pharmacist leadership in evaluation, oversight, and responsible implementation.
Current opinion in ophthalmologyMark Yu Zheng Wong, Marcus Ang
PURPOSE OF REVIEW: Artificial intelligence (AI) has emerged as a promising tool across multiple stages of myopia care, including screening, risk prediction and treatment planning. However, most published studies remain focused on algorithm development and validation, with considerably less attention directed towards implementation, regulation and real-world deployment. This review examines the current state of AI implementation in myopia and discusses the challenges that must be addressed before successful clinical adoption can occur. RECENT FINDINGS: Recent studies have applied AI across multiple stages of the myopia care pathway, from population screening and risk stratification to personalized intervention planning. Nevertheless, evidence regarding real-world deployment remains limited. A small number of implementation studies have begun evaluating integration within existing healthcare systems, though few studies focus on actual patient outcomes and change in management, beyond algorithmic performance alone. Early economic evaluations suggest that AI-enabled programmes may be cost-effective through improved resource allocation and prevention of long-term visual morbidity. No myopia-specific AI system has yet achieved routine clinical implementation as a regulated medical device. SUMMARY: The principal challenges facing AI in myopia care are implementation, regulation, economics and clinician trust. Future progress will depend not only on algorithmic performance but also on prospective implementation studies, demonstrable economic value and robust governance frameworks. Successful translation will ultimately require demonstration of clinical value within real-world healthcare systems.
Cancer cytopathologyJiayuan Guo, Bo Franzén, Rolf Lewensohn, Martin Hysek, Linus Jönsson
BACKGROUND: Precision oncology requires diagnostic sampling that delivers molecularly actionable material quickly and safely. In non-small cell lung cancer (NSCLC), core-needle biopsy (CNB) remains common. Yet fine-needle aspiration (FNA) has re-emerged with rapid on-site evaluation (ROSE), optimized cell blocks, and next-generation sequencing. The objective of this study was to compare the economic costs of FNA versus CNB with respect to an FNA-first with predefined CNB escalation strategy for NSCLC clinical pathways. METHOD: The authors synthesized evidence on diagnostic adequacy, complication rates, and molecular performance of cytology versus core tissue. A microcosting framework decomposed total diagnostic episode costs into sampling, processing, complications, and remedial procedures. This framework was applied to Sweden's Standardized Cancer Care Pathway for NSCLC using decision-tree modeling and sensitivity analyses. RESULTS: Modern cytology workflows with ROSE achieve a nondiagnostic sampling rate ≤6% and support programmed death-ligand 1 and broad next-generation sequencing with high mutation concordance to surgical specimens. Compared with CNB, FNA incurs fewer major complications and facilitates repeat sampling. In the Swedish illustration, an FNA-first pathway with ROSE reduced expected per-patient diagnostic cost by approximately 33% (7748 vs. 11,637 Swedish krona). The results were robust to ±50% variation in complication rates and ±5%-10% variation in adequacy. CONCLUSIONS: Reframing adequacy around molecular fitness and time to actionability supports FNA where ROSE exists, with codified, same-episode CNB escalation. This approach advances safety, capacity, and timeliness without compromising molecular yield. The authors provide a transferable cost-consequence model and implementation checklist for health systems. Local recalibration is required for absolute cost values, and multisite calibration is encouraged to establish generalizability across the different settings.
Journal of evaluation in clinical practiceNadja Alexandrov, Durk-Jouke van der Zee, Maud B R C Eurlings, Maritta N van Stigt, Monique Theunissen, Erik Buskens, Koen M van der Waaij, Erik D Gommer, Jona…
RATIONALE: Effective prehospital stroke triage enables faster treatment and better patient outcomes. Although prehospital triage innovations are rapidly emerging, evidence regarding the balance between costs and benefits as well as implementation issues remains limited. AIMS AND OBJECTIVES: This review aims to synthesise published evidence on health economics and implementation determinants of prehospital triage tools for acute suspected ischaemic stroke patients. METHOD: A systematic search was conducted across five databases (CINAHL, Cochrane, EMBASE, PubMed, and Web of Science) between 1995 and 2025. Results were reported narratively, and reporting quality was assessed using CHEERS, STROBE-simulation, COREQ, CONSORT, SQUIRE, and GRAMMS. Included were empirical studies of EMS-operated prehospital triage innovations for acute suspected stroke patients. The main outcome measures were health economic outcomes (e.g., ICER) and system-level implementation determinants (CFIR constructs). RESULTS: From 6491 screened studies, thirty-one were included: thirteen studies included health economic outcomes and eighteen reported implementation determinants. Prehospital triage tools assessed were: MSU (n = 14), telemedicine (n = 13), clinimetrics (n = 1), prehospital MRI (n = 1), biomarkers (n = 1), and an AI-based microwave tool (n = 1). Most studies (68%) were of high reporting quality. Positive results regarding cost-effectiveness were reported (n = 11), including cost-savings (n = 1), as well as implementation determinants (n = 11). However, evidence on sustained implementation was limited. Barriers included innovation design, complexity, workflow compatibility, work and IT infrastructure, and costs. CONCLUSION: Included studies suggest most prehospital stroke triage tools are cost-effective, but implementation remains largely context-dependent. Barriers, including innovation complexity, role delineation, workflow fit, design, and costs, highlight the need for future studies to integrate health economics into an implementation science framework alongside clinical outcomes.
Journal of cancer research and clinical oncologyAnn-Cathrine Froitzheim, Melina Sophie Kurte, Katja Gehrke, Sebastian Lempfert, Katja Hesse, Florian Kron
PURPOSE: Chimeric Antigen Receptor (CAR) T-cell therapy has transformed the treatment of diffuse large B-cell lymphoma (DLBCL) but carries higher drug acquisition costs than autologous stem cell transplantation (autoSCT), while administration and follow-up costs remain underrepresented in economic evaluations and reimbursement decision-making. This study compares administration-related and subsequent treatment costs of CAR-T and autoSCT for DLBCL from the German statutory health insurance (SHI) perspective. METHODS: A two-step analysis based on two German databases was performed. In step 1, SHI billing data (representing 5 million insured individuals, ≙ 8% of SHI) from 2020-2022 were analyzed longitudinally. Costs were divided into initial regime (IR) (all services except CAR-T drug acquisition) and a following regime (FR). In step 2, a cross-sectional analysis of all German inpatient cases from 2020-2023 (17 million cases/year) assessed inpatient costs. RESULTS: Step 1 included 12 CAR-T and 59 autoSCT patients. Median IR costs per patient were €61,703 (CAR-T) versus €55,802 (autoSCT), while FR costs were lower for CAR-T (€44,832 versus €69,453). Most frequently reported adverse events (any grade) for CAR-T versus autoSCT were: B-cell aplasia/neutropenia (77% versus 64%), thrombocytopenia (38% versus 64%). Step 2 included 1,229 CAR-T and 2,239 autoSCT inpatient cases (2020-2023). Average LOS was comparable (27.3 versus 27.8 days), and average inpatient costs were lower for CAR-T (€13,991 versus €29,432). CONCLUSION: Although associated with higher IR costs, CAR-T therapy in DLBCL demonstrated lower inpatient and FR costs compared to autoSCT. This analysis focused on administration and follow-up costs, excluding drug acquisition costs, characterizing the economic burden beyond drug pricing.
OBJECTIVES: To assess temporal trends and regional disparities in the costs and affordability of non-insulin antidiabetic drugs in China between 2015 and 2022. DESIGN: A retrospective observational study using monthly provincial procurement data. SETTING: 27 provincial-level administrative regions in mainland China from 2015 to 2022. PARTICIPANTS: No individual participants were included. The units of analysis were aggregated monthly procurement records for 29 non-insulin antidiabetic drugs classified into nine therapeutic categories. PRIMARY AND SECONDARY OUTCOME MEASURES: The primary outcomes were the 30-day treatment cost and affordability ratio of non-insulin antidiabetic drugs at the national and regional levels. The 30-day treatment cost was calculated using defined daily doses and affordability was assessed as the ratio of the 30-day treatment cost to the corresponding minimum daily wage. Secondary outcomes included temporal changes in costs and affordability by therapeutic category, active ingredient and region. RESULTS: National average monthly costs fluctuated before 2019, reaching a peak of US$18.53 in 2019 and subsequently declined to US$11.34 in 2022. Costs declined across all therapeutic categories from 2019 to 2022, although the magnitude of reduction varied considerably. Affordability also improved over the study period, but substantial differences remained across therapeutic categories. By 2022, only biguanides and sulfonylureas had achieved good affordability at the category level, while several other categories remained above the affordability threshold. Regional disparities persisted throughout the study period; in 2022, affordability was relatively more favourable in the central region and poorer in the northwest, and no region achieved overall good affordability. CONCLUSIONS: Between 2015 and 2022, the cost and affordability of non-insulin antidiabetic drugs in China improved overall, but disparities persisted across therapeutic categories and regions. These findings highlight the continuing challenge of ensuring equitable access to higher-cost therapies and provide a historical benchmark for evaluating subsequent changes in drug costs and affordability.
Clinical spine surgeryRichard N Storey, Yoshinori Maki, Stacey Darwish, Joseph S Bulter
INTRODUCTION: Odontoid process fractures in elderly patients are common and significant for their poor clinical and radiologic prognosis. This controversy debates the role of MRI in odontoid process fractures in elderly patients without a neurological deficit. CONTROVERSY: Elderly patients generally sustain odontoid process fractures following low-energy mechanisms. Ligamentous structures provide a significant secondary constraint to progressive fracture displacement. The additional contribution of MRI after CT and/or radiographs is controversial. SUMMARY: Potentially the most justifiable reason for getting an MRI in the setting of an odontoid process fracture in a neurologically intact elderly patient is to identify occult adjacent segment injury, particularly in the setting of advanced degenerate disease or ankylosis. This debate is useful in guiding imaging criteria and hypothesis generation, particularly the role of discrete MRI findings as indicators for surgery. The authors suspect that ligamentous injury findings are nuanced in their predictive features; ie, anterior longitudinal ligament disruption in extension fractures and posterior longitudinal ligament disruption in flexion injuries. Translational work to demonstrate CT thresholds which are consistent with MRI-confirmed ligamentous injury may give the ability to anticipate ligamentous injury on CT alone. This is of high importance in healthcare systems where access to MRI is limited.
BMJ openMalcolm A West, Kashuf Khan, Charles T West, Zoe Li, Elias Brown, Samantha Sodergren, Helen Moyses, Sam A Rose, Errol Bignall, Layla Stephen, Sharon A Applin, …
INTRODUCTION: Advanced and recurrent pelvic cancers pose significant public health challenges due to their invasive nature, affecting pelvic structures like the pelvic floor, other organs and the perineum. Treatment involves chemoradiotherapy followed by extended margin cancer surgery (EMCS) to achieve cancer cure. EMCS results in an empty pelvis prone to major complications like collections, abscesses, bowel obstruction, fistulae and perineal wound breakdown. Known as the empty pelvis syndrome (EPS), this affects >50% of patients. EPS leads to poor quality of life (QoL), reduced return to function and significant financial burden to patients and healthcare services. Perineal reconstruction using myocutaneous flaps or biological meshes may mitigate these complications, but their longer-term clinical outcomes and effectiveness are not well-documented. This study addresses key unmet needs and aligns with strategic healthcare priorities.The Reconstruction after Extended Margin Cancer Surgery (REMACS) study aims to assess the effects of EMCS and perineal reconstruction on QoL, complications and health economic utilisation up to 12 months post-surgery.Objectives include:a. Providing real-world data on 12-month QoL and complications to aid shared decision-making consultations regarding reconstructive techniques post-ECMS.b. Informing clinical practice and patient expectations, improving communication and reducing regret throughout the initial stages of cancer survivorship.c. Evaluating economic impacts on patients and healthcare settings, optimising resource utilisation.d. Uncovering hidden treatment toxicities post-ECMS. METHODS AND ANALYSIS: Design: Multi-centre observational cohort study. PARTICIPANTS: 236 patients undergoing EMCS with perineal reconstruction in tertiary cancer centres, ensuring diverse representation. DATA COLLECTION: Patients will complete QoL (European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC-QLQ-C30), EuroQol-5 Dimension 5-Level Questionnaire (EQ-5D-5L), Locally Recurrent Rectal Cancer-QoL (LRRC-QoL)) and Decision-Regret questionnaires pre-surgery and 3, 6, 12 months post-surgery. Patient economic impact (COST, patient employment, health resource utilisation), quality-adjusted life years, incremental cost-effectiveness ratios (EQ-5D-5L) and healthcare economic impact will be recorded over 12 months after surgery. Complications (Clavien-Dindo) and pathology outcomes. 5-year survival and cancer recurrence will also be recorded as part of standard of care. Purposefully selected patients (n=30) representing different cancer types and from different geographical regions in the UK, ensuring the inclusion of different ethnic and under-represented minority backgrounds (ie, sexual and gender minorities and those from low socio-economic groups) will be interviewed at 3 and 12 months post-surgery. Patient experiences including preparedness, complex decision-making, complications, regret and hidden cancer treatment toxicities will be explored. ANALYSES: EORTC-QLQ-C30 global health scores at 12 months will be compared between groups (primary outcome: flap versus mesh reconstruction). Descriptive statistics, causal inference techniques and regression modelling will be used to account for allocation bias and heterogeneity. Propensity scoring will weight cases and causal inference techniques will be used to visualise and understand confounding effects. Cost-utility economic analyses will compare reconstructive techniques. Longitudinal interview analyses will use constant comparative methods using reflexive thematic analysis. ETHICS AND DISSEMINATION: REMACS is approved by HRA REC (22/NE/0032). The study is funded by Penguins Against Cancer (Registered Charity Number 1159905), Bowel Research UK (BRUK SG 23019), PLANETS Cancer Charity (Registered Charity Number 1175572) and an NIHR Research for Patient Benefit Grant (NIHR207881).This study will benefit patients internationally by providing real-world outcomes to improve shared decision-making around complex consultations, informing patient expectations and clinical outcomes for QoL, complications and cost after EMCS and perineal reconstruction. Results will be disseminated to researchers, healthcare professionals, patients, carers, charities and policymakers through online platforms, media, scientific conferences and journals. This will raise awareness of late effects of cancer treatment, influence international guidelines and cancer commissioning. TRIAL REGISTRATION NUMBER: Clinical Trials.gov registration number-NCT05219058 and ISRCTN registry number-ISRCTN81241096.
Advances in therapyBatyrkhan Kuatov, Alanna MacDonald, Stacey Priest, Diana Tran, Laurence Undreiner, Melike Inkaya Taskin, Sara Villa, Andrea Marcellusi
INTRODUCTION: Primary immunodeficiency (PID) and secondary immunodeficiency (SID) are chronic conditions associated with impaired immune function and increased susceptibility to infections. Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare autoimmune neurological disorder characterized by progressive motor and sensory deficits due to immune-mediated demyelination of peripheral nerves. Patients with these diseases often require long-term immunoglobulin (IG) therapy, which can be costly and burdensome, particularly when administered intravenously in hospital settings. IgPro20 pre‑filled syringes (PFS) offer a simplified, subcutaneous IG alternative that may reduce treatment burden and improve cost‑efficiency. The aim of this study was to estimate the budget impact of introducing IgPro20 PFS into the Italian healthcare system for patients with PID, SID, and CIDP. METHODS: A population-based budget impact model was developed in Microsoft Excel® from the perspective of the Italian National Health Service (Servizio Sanitario Nazionale). The model used a 3-year time horizon and incorporated drug acquisition, administration, and adverse event costs. Model comparators included intravenous and subcutaneous IG and IgPro20 vials. Population estimates and market shares were informed by Italian affiliates. RESULTS: The introduction of IgPro20 PFS resulted in total cost savings of €31,524,488 for 4794 patients over 3 years. Savings were distributed across PID (€8,840,041), SID (€5,413,035), and CIDP (€17,271,412). Sensitivity analyses identified IgPro20 PFS and vial pricing as key drivers. CONCLUSION: IgPro20 PFS are a cost-saving, patient-centric treatment option for PID, SID, and CIDP. These findings support their adoption in Italy's healthcare system and may inform policy and payer decisions at regional and national levels.
BMJ openKaren Hughes, Kat Ford, Mark A Bellis, Zara Quigg, Joanne C Hopkins
OBJECTIVES: To update estimates of the health and financial burden of adverse childhood experiences (ACEs) in England and Wales and generate estimates for English regions. DESIGN: Population attributable fractions (PAFs) for ACEs were calculated for risks and causes of ill-health using data from 10 randomly stratified cross-sectional ACE studies. PAFs were applied to disability-adjusted life years (DALYs) for England, Wales and English regions for 2023, with financial costs estimated using UK statistical life year values (societal willingness to pay). SETTING: Households in England and Wales. PARTICIPANTS: 28 449 residents aged ≥18 years. OUTCOME MEASURES: PAFs for ACE exposure categories (1 ACE, 2-3 ACEs and ≥4 ACEs) for four health risks (smoking, high alcohol use, drug use and high body mass index (BMI)) and seven causes of ill health (violence, mental illness, cancer, type 2 diabetes, heart disease, respiratory disease and stroke). Annual estimated DALYs and financial costs attributable to ACEs. RESULTS: Relative risks for all outcomes increased with ACEs, with risk ratios for ≥4 ACEs ranging from 1.063 (95% CI 1.000 to 1.129, p=0.048) for high BMI to 9.147 (95% CI 7.062 to 11.847, p<0.001) for drug use. For health risks, PAFs for ACEs were highest for drug use (Wales 60.3% and England 56.7%), while ACE-attributable costs were highest for smoking (Wales £1.8 billion and England £22.7 billion). For causes of ill health, PAFs for ACEs were highest for violence (Wales 49.2% and England 46.2%), and costs were highest for mental illness (Wales £1.8 billion and England £37.8 billion). Across all outcomes (accounting for duplication by excluding DALYs for causes linked to the four health risks), total ACE-attributable costs were £7.8 billion for Wales and £115.6 billion for England; costs for English regions ranged from £6.3 billion (North East England) to £18.0 billion (South East England). CONCLUSIONS: Findings highlight the substantial financial burden of ACEs across England and Wales and provide important information to inform regional activity. There is an urgent and economically justified need for action to prevent ACEs and support those affected by them to reduce the health and economic burden of poor-quality childhoods.
International journal of cardiologyKenneth K Cho, Derek P Chew, Hariharan Sugumar, Liang-Han Ling, Aleksandr Voskoboinik, Christopher Reid, Jonathan M Kalman, Peter M Kistler, Louise Segan, Sand…
BACKGROUND: The WITHDRAW-AF trial demonstrated HFrEF pharmacotherapy can be safely withdrawn in carefully selected patients with recovered atrial fibrillation-mediated cardiomyopathy post-ablation. In this study, we examined the economic implications of this strategy. METHODS: We compared costs and outcomes of each strategy during the trial and through a 5-year Markov model. Functional status was assessed by VO2max and 6-min walk test distance (6MWT), and quality of life by the Minnesota Living with Heart Failure Questionnaire (MLHFQ) mapped to the EQ-5D-5L. Probabilistic sensitivity analysis was through 2000 Monte Carlo simulations. RESULTS: In-trial medication cost savings were $1031/patient ($1572 vs $2604, p < 0.001). Over 5 years, projected modelled costs for withdrawal versus continuation were $3901 (95% CI: $3852-$3950) versus $8446 (95% CI: $8375-$8517). Outcomes favoured withdrawal; 6MWT in 61.8% of simulations (difference 29.39 m, 95% CI: 23.03-35.74), VO2max in 68.1% (difference 4.02 ml/kg/min, 95% CI: 3.49-4.44), and MLHFQ in 53.7% (difference 1.71, 95% CI: 0.57-2.84). QALYs were comparable (difference 0.003, 95% CI: -0.05-0.01). CONCLUSION: In recovered atrial fibrillation-mediated cardiomyopathy, HFrEF pharmacotherapy withdrawal represented cost-minimisation without detriment to functional status or quality of life.
BMJ global healthSutinee Soopairin, Parnnaphat Luksameesate, Nattiya Arthonchaikul, Suda Punrin, Terapong Tantawichien, Suthira Taychakhoonavudh
INTRODUCTION: Rabies remains a life-threatening disease affecting children in endemic regions including Thailand. The cost-effectiveness of pre-exposure prophylaxis (PrEP) rabies vaccination in the Thai context is unclear. This study analysed the cost-utility of adding PrEP rabies vaccination to post-exposure prophylaxis (PEP) rabies vaccination compared with PEP alone in 5-year-old children. METHODS: A decision tree model compared eight strategies (four PEP alone and four PrEP+PEP), classified by route of administration (intradermal (ID) or intramuscular (IM)) and vaccine product (purified chick embryo cell vaccine (PCEC) or chromatographically purified Vero cell vaccine (CPRV)/purified Vero cell rabies vaccine (PVRV)). Costs and outcomes were derived from published literature and discounted at 3% annually. The analysis was conducted from a societal perspective over a 20-year horizon. Incremental cost-effectiveness ratios (ICERs) were evaluated against Thailand's willingness-to-pay threshold of THB 160 000 per quality-adjusted life year (QALY). RESULTS: PrEP+PEP ID using PCEC was cost-effective, with an ICER of THB 40 977 (US$1160.8) per QALY versus PEP alone ID using PCEC; the remaining strategies were dominated. The one-way sensitivity analysis identified annual bite incidence as the most influential parameter, with the ICER ranging from cost-saving to not cost-effective (THB 454 265 (US$12 868.7) per QALY). The results were robust to variation in all other parameters. CONCLUSION: PrEP+PEP ID using PCEC is cost-effective for children in Thailand, reducing reliance on equine rabies immunoglobulin and disease-related QALY losses. These findings support integrating PrEP into Thailand's National Immunization Program for children in high-risk areas, prioritising early vaccination in high-risk populations to advance the goal of eliminating rabies by 2030.
Health economics reviewEva Juliane Beer, Michaela Kirschneck, Michaela Coenen, Rosa Maria Sija Visscher
BACKGROUND: Health promotion and disease prevention are receiving increasing attention in Germany because of their potential to improve population health and reduce healthcare costs. Health-economic evaluations (HEEs) are essential for informing evidence-based decision-making in this field. However, HEEs of preventive interventions often face methodological challenges. This scoping review examines the current state and characteristics of HEEs conducted in the context of health promotion and disease prevention in Germany. METHODS: A scoping review was conducted to map the landscape of HEE of health promotion and disease prevention interventions in Germany, following a priori-registered protocol. Three bibliographic databases (MEDLINE, EMBASE and Web of Science) were searched from their inception to March 2025. The screening of titles, abstracts, and full texts was performed in a blinded, double-reviewer, and stepwise manner using predefined inclusion criteria: (i) focus on the German population, (ii) application of an HEE as the primary research method, and (iii) evaluation of a health-promoting or disease-preventing intervention. Studies focusing on tertiary prevention were excluded. RESULTS: Of the 4,632 records identified, 162 eligible original studies met the inclusion criteria. The volume of published HEEs has increased over time. Most studies (53.7%) assessed primary preventive interventions. Cost-effectiveness analysis was the most frequently applied HEE analysis type. Incremental cost-effectiveness ratios (ICERs) are commonly reported as incremental costs per quality-adjusted life year (QALY) gained (42.4% of all ICERs). In 85.2% of the studies, the intervention was judged to be cost-effective. A model-based approach was used in 75.9% of the studies, and 81.3% of these studies relied partly on non-German data sources. More than half of all studies (56.1%) reported limitations due to a lack of German-specific data. CONCLUSIONS: HEEs related to health promotion and disease prevention interventions in Germany exhibit substantial methodological heterogeneity. Frequent reliance on non-German data and the large proportion of interventions deemed cost-effective may reflect contextual data gaps and potential publication bias. These issues complicate the interpretation of economic evidence and pose challenges for decision-makers seeking to prioritize preventive strategies. TRIAL REGISTRATION: https://osf.io/h6749/overview.
BMJ openOurega-Zoé Ejebu, Jane Ball, Mesfin G Genie, Julia Philippou, Joanne Turnbull
BACKGROUND: In England, disproportionate numbers of registered nurses leave the National Health Service (NHS) early in their careers. Retaining early-career nurses could ease supply challenges. Most evidence-largely from outside the UK-indicates that retention factors among early-career nurses are within their employers' control. Research is needed to understand the job preferences of early-career NHS nurses to inform and develop tailored retention policies. OBJECTIVES: To identify the job preferences of early-career NHS nurses and estimate the potential trade-offs between these job characteristics. DESIGN AND METHODS: A cross-sectional discrete choice experiment (DCE) administered through an online survey distributed by the Nursing and Midwifery Council. A DCE is a quantitative stated-preference method that presents respondents with hypothetical job scenarios with varying attributes and levels. Nurses chose their preferred job package from each set of alternatives, enabling the estimation of their job preferences and trade-offs. Following established practice, we employed qualitative work, including a literature review, focus groups and consultation with academic experts and stakeholders, which was undertaken before the DCE to inform the selection of attributes and levels. Quantitative findings are reported separately. We specified a mixed logit model to estimate the job preferences of early-career NHS nurses and explore preference heterogeneity. SETTING: England, NHS. PARTICIPANTS: 11 early-career nurses participated in the qualitative focus groups that informed DCE development, and 5696 early-career nurses were included in the final DCE analysis. PRIMARY AND SECONDARY OUTCOME MEASURES: The DCE included eight job attributes: career development, organisational support, teamwork, management, personalised working, work pressure, recognition and salary. Salary was included as a continuous attribute to estimate the amount of annual salary respondents would be willing to forgo for improvements in the non-monetary job attributes. RESULTS: Early-career NHS nurses preferred good teams with good collaboration,[Formula: see text]supportive and accessible managers [Formula: see text] and low work pressure [Formula: see text]. They were willing to forgo £9419 in annual salary for good teamwork and collaboration, £6534 for supportive and accessible managers and £6055 for low-pressure work environments. CONCLUSIONS: Good teamwork and collaboration, supportive and accessible managers and low work pressure were highly valued by early-career NHS nurses. Healthcare managers should prioritise workplace environments that reflect these preferences as part of broader retention strategies.
Human reproduction updateErik Iwarsson, Christina Bergh, Kersti Lundin, Britt Friberg, Jenny Berg, Martina Lundqvist, Maria Ahlberg, Jan Adolfsson, Maja Kärrman Fredriksson, Erik Malmq…
BACKGROUND: Preimplantation genetic testing for aneuploidy (PGT-A) aims to increase live birth rate (LBR) after IVF/ICSI. While observational studies and most systematic reviews have indicated benefits with increased LBRs and reduced miscarriage rates, results from most randomised controlled trials (RCTs) have failed to show any increase in LBR when calculated per randomised patient. According to international, professional societies, PGT-A is currently not recommended for routine clinical use, still the use of PGT-A is increasing. OBJECTIVE AND RATIONALE: The purpose of this systematic review was to evaluate if PGT-A results in increased LBR in general or in specific patient groups. Efficacy is summarised in meta-analyses, and certainty of evidence was assessed. It also includes systematic reviews on possible complications for children and mothers and a health economic analysis. SEARCH METHODS: A systematic search was conducted from inception to November 2024 in MEDLINE, Embase, CINAHL, Cochrane Library, PsycINFO, PubMed, and Scopus. Only RCTs where the PGT-A technique analysed all chromosomes were selected for efficacy. Concerning safety, observational studies were also included. Risk of bias (RoB) was assessed using Cochrane tools. Results from meta-analyses were presented as odds ratio (OR) and risk difference (RD) with 95% CI. Certainty of evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) system. OUTCOMES: The search for efficacy and complications resulted in 9673 references. We included seven RCTs on efficacy, 55 studies on complications, and two studies on health economics. The primary outcome, live birth per randomised woman, assessed both for the first planned embryo transfer (ET) and cumulatively per oocyte retrieval, was comparable between IVF with PGT-A and IVF without PGT-A (OR 1.16; 95% CI 0.94-1.45 and OR 0.87; 95% CI 0.64-1.19) (low certainty of evidence), also for women over 35 years. The secondary outcome, live birth per ET, was higher after PGT-A (low certainty of evidence). It was not possible to evaluate if miscarriage rate is affected by PGT-A since the certainty of evidence was very low. Only two studies evaluated time to pregnancy. None of them showed a shorter time to live birth by use of PGT-A. There was no indication of an increase in complications for the child or the pregnant woman after PGT when comparing IVF with or without embryo biopsy using PGT-A, PGT-M, or PGT-SR. In a Swedish setting, adding PGT-A to IVF almost doubles the cost of an IVF treatment without any increase in live births. WIDER IMPLICATIONS: The strength of this report is that the main outcome is live birth per randomized woman. Many observational studies, earlier systematic reviews/meta-analyses, as well as two RCTs using all-chromosome PGT-A, were excluded from this systematic review; they only report live birth for those women who received an ET, which gives an incorrect and too positive view. No increase in complications for the child or the pregnant woman after PGT was observed; thus, embryo biopsy seems to be safe. Since our systematic review does not show any clear patient benefit, and PGT-A almost doubles the cost of an IVF treatment, PGT-A is currently not considered ethically justifiable. REGISTRATION NUMBER: PROSPERO-CRD42024529876.
OBJECTIVES: To analyse temporal trends, clinical outcomes and healthcare costs of Alzheimer's disease (AD)-related hospitalisations in Southwest China over 14 years and identify modifiable risk factors for adverse outcomes. DESIGN: Retrospective time-series analysis of electronic health records. SETTING: A tertiary referral centre in Southwest China (covering Sichuan, Guizhou, Yunnan and Xinjiang provinces); single-centre study. PARTICIPANTS: 4970 hospitalisations of patients with a confirmed diagnosis of AD (ICD-10: G30) discharged between January 2011 and December 2024, with a mean age of 77.8±0.14 years, and 51.7% were female. INTERVENTIONS: Not applicable (observational study). PRIMARY AND SECONDARY OUTCOME MEASURES: Primary outcomes were poor hospital outcomes, defined as prolonged length of stay (>14 days), frequent readmissions (≥4 admissions over the study period) and in-hospital mortality. Secondary outcomes included annual trends in hospitalisation rates, total costs and prevalence of comorbidities. RESULTS: Comorbidities (64.75% of admissions, mainly infections and fractures) and admission to non-neurology departments independently increased risks of prolonged stay (OR=5.248, 95% CI 4.287 to 6.425; p<0.001), frequent readmissions (OR=1.735, 95% CI 1.236 to 2.435; p=0.001) and in-hospital mortality (OR=2.176, 95% CI 1.506 to 3.146; p<0.001). Hospitalisations rose annually by 7.65% (95% CI 3.00% to 12.31%; p=0.001) and total costs by 8.32% (p<0.001), yet the composite poor-outcome rate declined from 70% to 50% after 2018 (annual percent change, -6.32%, p=0.004), coinciding with fewer comorbidity-driven admissions and increased neurology department care. CONCLUSIONS: Comorbidities and non-neurology admissions were associated with increased risks of adverse outcomes in this cohort. The post-2018 temporal improvements coincided with a decline in comorbidity-driven admissions and increased neurology department use. Further prospective studies are needed to confirm these associations. TRIAL REGISTRATION: Not applicable (observational study; not registered).
BMC medical ethicsInês Martins Esteves, Márcia Pestana-Santos, Filipa Sampaio, Margarida Reis Santos
BACKGROUND: Discrete choice experiments (DCEs) are quantitative preference-elicitation methods, developed largely within health economics, in which participants choose repeatedly between hypothetical scenarios composed of systematically varied attributes. Their growing use with children supports children's right to be heard in decisions affecting their care, yet it also raises ethical challenges that general paediatric research ethics guidance does not address in operational detail: sustained cognitive demand, potential emotional distress, assent that can blur under clinical stress, parental influence, clinician-researcher role conflict, and the processing of children's personal data. METHODS: We developed a prospective ethical governance framework during the preparatory phase of the PREF-ANX Kids study (Preferences for Preoperative Anxiety Management in Children), a planned multicentre DCE examining preferences for nursing interventions to manage preoperative anxiety among children aged 6-12 years undergoing elective surgery in Portuguese public hospitals, their parents, and healthcare professionals. Development combined a pragmatic, non-systematic synthesis of ethical, regulatory, and methodological guidance with iterative structured discussion within an interdisciplinary team. No formal consensus method was applied, and no children or families were involved in development; we identify this as a limitation. FRAMEWORK: The framework comprises four domains operating across the research lifecycle: relational participation, cognitive and emotional safeguarding, reflexive professional responsibility, and privacy stewardship. For each domain it specifies planned procedures (continuous assent and dissent monitoring, adapted task burden, structured reflexive documentation, and a prospective data protection impact assessment) together with their normative justification, and it distinguishes elements specific to PREF-ANX Kids from elements potentially adaptable to other paediatric DCEs. Trade-offs between competing ethical and methodological priorities are made explicit rather than resolved silently. CONCLUSIONS: The framework is prospective: it has not yet been implemented or empirically evaluated, and no claims of demonstrated effectiveness are made. It is intended to support transparent ethical planning of DCEs involving children aged 6-12 years and will be assessed during the PREF-ANX Kids study. Future refinement should incorporate co-design with children and families and empirical evaluation of feasibility, acceptability, participant experience, and data quality.
Journal of pain researchShiming Huang, Shuai Qing, Qishan Yi
OBJECTIVE: Short-term spinal cord stimulation (st-SCS) is increasingly used for acute and subacute herpes zoster neuralgia (HZNP), but postoperative management typically requires prolonged hospitalization. We compared two management strategies-continuous inpatient programming versus early discharge with remote programming-on pain outcomes, patient-reported outcomes, safety, and costs. METHODS: This single-center retrospective cohort study included 28 patients with HZNP (disease duration ≤3 months) who underwent percutaneous st-SCS between May 2021 and May 2024. Fourteen patients received inpatient programming throughout the stimulation period; 14 were discharged 1-2 days post-implantation with remote parameter adjustment. Pain (VAS), sleep (PSQI), anxiety/depression (HADS), quality of life (SF-36), opioid consumption (MME), complications, and costs were assessed preoperatively, at electrode removal, and at 3, 6, and 12 months. RESULTS: Both groups achieved substantial pain relief after st-SCS (time effect P<0.001). Between-group differences in VAS scores were less than 0.5 points at all postoperative time points-well below the minimal clinically important difference of approximately 2 points-indicating comparable analgesic efficacy regardless of programming setting (group effect P=0.325; group×time interaction P=0.902). At 3 months, the home group had numerically lower PSQI (5.8±1.3 vs 7.1±1.5) and HADS scores (6.2±1.8 vs 7.8±2.1) and higher SF-36 scores (78.4±6.2 vs 72.5±6.8), but these differences did not survive Bonferroni correction. Opioid consumption was lower in the home group at 3 months (13.2±4.0 vs 18.0±4.2 mg/day, P=0.005). Complication rates were similar (14.3% each). Total healthcare costs were 28.5% lower in the home group (9464±938 vs 13,236±1499 CNY, P<0.001), driven primarily by reduced bed and nursing fees from shorter hospitalization. CONCLUSION: For patients with HZNP treated with st-SCS, early discharge with remote programming achieved analgesic results comparable to inpatient management-both in statistical terms and, more importantly, in clinical magnitude-while reducing opioid use and healthcare costs. The cost advantage reflects shorter hospital stay rather than superior analgesia. These findings support remote programming as a practical alternative for appropriately selected patients, though confirmation in larger prospective studies is needed.
Journal of the American College of SurgeonsPatrick L Johnson, Cody L Mullens, Scott C Levy, Raymond Jean, Mark R Hemmila, John W Scott
BACKGROUND: Emergency general surgery (EGS) conditions account for 4 million unplanned hospital admissions annually, but whether these acute, usually curable conditions are associated with lasting medical debt is unknown. We hypothesized that post-EGS increases in medical debt would be more strongly associated with patient demographics than treatment factors. STUDY DESIGN: We evaluated 4,216 adults admitted for appendicitis, gallbladder disease, small bowel obstruction, or emergency laparotomy in a statewide EGS collaborative (April 2020-June 2021), linking clinical registry data to quarterly consumer credit reports from 1 year before through 1 year after admission. The primary outcome was any increase in medical debt in collections from admission to 12 months; the secondary outcome was debt magnitude among affected patients. Modified Poisson and negative binomial regression evaluated incidence and magnitude, respectively. RESULTS: Overall, 382 patients (9.1%) had increased medical debt in collections (mean increase $1,764; median $535). Estimated income demonstrated a dose-response relationship (15.2% <$40,000 vs 2.7% >$100,000; aRR 5.67, 95% CI 3.69-8.69). Uninsured patients had 4.06-fold higher risk than privately insured patients (95% CI 2.82-5.84; adjusted probability 29.5% vs 7.3%). Operative intervention, intensive care, and hospital length of stay were not associated with increased debt. CONCLUSIONS: Nearly 1 in 10 patients had increased medical debt in collections after EGS. Associations with income and insurance, but not treatment intensity, identify financial risk protection rather than changes in clinical care as a target for reducing post-EGS financial hardship.
BMJ openYanxiao Zhang, Xiaoge Wang, Zimeng Huang, Li Huang, Guojun Sun
OBJECTIVES: To systematically evaluate the clinical efficacy, cost-effectiveness and impact on medical insurance budgets of transarterial chemoembolisation (TACE) combined with apatinib versus TACE alone for the treatment of unresectable hepatocellular carcinoma (uHCC) in China, from an integrated health technology assessment (HTA) perspective. DESIGN: An HTA incorporating evidence from a randomised controlled trial, a Markov model with parametric extrapolation of survival data over a lifetime horizon and a static 5-year budget impact model from the Chinese healthcare payer perspective. SETTING: Analysis was conducted from the perspective of the Chinese public healthcare payer. Clinical evidence was derived from a multicentre, randomised, open-label, prospective, phase III trial conducted at 15 hospitals in China. PARTICIPANTS: The published trial included 196 patients with uHCC. Participants were predominantly male (overall 85%), with a mean age of 52.7 years in the combination group and 52.9 years in the TACE-alone group. All had an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 and were of Chinese ethnicity. The present analysis used the de-identified, aggregated data from this published trial. INTERVENTIONS: The experimental regimen comprised TACE (intra-arterial injection of lipiodol and epirubicin) combined with oral apatinib (500 mg/day, initiated 4 days after the first TACE). The control regimen was TACE alone. MAIN OUTCOME MEASURES: Clinical outcomes included median progression-free survival (PFS) and overall survival (OS). The primary economic outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained. The primary budgetary outcome was the 5-year cumulative incremental expenditure on the national basic medical insurance fund. RESULTS: Clinical evidence showed that the combination therapy significantly prolonged median PFS (6.1 vs 3.4 months; HR 0.45, 95% CI 0.33 to 0.63; p<0.0001) and OS (28.9 vs 24.0 months; HR 0.55, 95% CI 0.40 to 0.77; p=0.0005). The economic evaluation indicated that at the 2021 centralised procurement price, the ICER for the combination regimen was US$42 864/QALY, exceeding the willingness-to-pay threshold (US$37 653/QALY); however, when applying the 2025 national centralised procurement price, the ICER decreased to US$30 650/QALY, rendering it cost-effective. Budget impact analysis results showed that under the base-case penetration rate (reaching 50% by year 5), the cumulative incremental expenditure over 5 years would be approximately US$365 million, a finding highly sensitive to both the penetration rate and drug price. CONCLUSIONS: The TACE-apatinib regimen provides clear clinical benefit for uHCC in China, but its cost-effectiveness and budget feasibility are contingent on drug price. Successful national centralised procurement is the key policy lever to enable value-based access, as demonstrated by this integrated evidence framework. TRIAL REGISTRATION NUMBER: ChiCTR1800018621.
BMJ openTsegaye G Haile, Richard Norman, Winnie Chen, Gavin Pereira, Gizachew A Tessema
OBJECTIVE: Early childhood health and economic burdens are disproportionately distributed across population groups. We examined the vulnerability-related (First Nations status and remoteness) disparities of early childhood healthcare costs from birth to age 5 years and quantified the contribution of adverse perinatal outcomes, such as preterm birth (PTB), small-for-gestational-age (SGA) and low birthweight, to these disparities in the Northern Territory (NT), Australia. DESIGN AND PARTICIPANTS: We conducted a population-based retrospective cohort study of all singleton live births in the NT, Australia, between 1 July 2000 and 30 June 2016, using the Perinatal Trends dataset. Birth records were linked to hospitalisation, emergency department (ED) and national hospital costing data and followed to age 5. OUTCOMES AND ANALYSIS: Healthcare costs, adjusted to 2024 Australian Dollars (AUD), from a health system perspective, included hospitalisation and ED costs. A generalised linear model with log link and gamma distribution was fitted, and Oaxaca-Blinder decomposition was used to partition cost differences into explained (differences in population characteristics) and unexplained (differential effects/responses) components. RESULTS: The overall median hospitalisation costs per child were AUD 9608 (IQR: 4873 to 20 681) and ED costs AUD 2156 (IQR: 1075 to 4274) in the first 5 years. Among children with PTB and SGA, median hospitalisation costs were AUD 30 527 (IQR: 16 765 to 54 365) for First Nations and AUD 16 098 (IQR: 7577 to 28 048) for non-First Nations children; within this subgroup, costs were AUD 29 614 (IQR: 15 085 to 51 400) in remote areas compared with AUD 19 776 (IQR: 9620 to 33 263) in urban areas. After adjustment, mean hospitalisation costs were 98% higher for First Nations children (AUD 23 223 vs 11 716; difference AUD 11 507, 95% CI 10 798 to 12 215) and 19% higher for children in remote areas (AUD 19 454 vs 16 322; difference AUD 3132, 2479 to 3786); corresponding differences in mean ED costs were 67% (AUD 1866, 95% CI 1750 to 1982) and 17% (AUD 557, 95% CI 460 to 655). Remoteness-related disparities in hospitalisation costs were largely explained by child characteristics (85.7%; βE=-0.549, 95% CI -0.581 to -0.517), while disparities by First Nations status were primarily driven by differential cost responses (71%; βc=-0.606, 95% CI -0.651 to -0.561). For ED costs, characteristics explained 42% of the remoteness-related gap and 3.4% of the gap by First Nations status. CONCLUSIONS: Our findings highlight that vulnerability-related disparities in healthcare costs were substantial, with adjusted costs 67% to 98% higher for First Nations children and 17% to 19% higher for children in remote areas, and driven by distinct mechanisms. Thus, strategies addressing structural disadvantage and inequitable experiences are needed to reduce avoidable healthcare costs.
Public health genomicsJessica Ezzell Hunter, Caitlin G Allen, Jonathan S Berg, W David Dotson, Kimberly S Foss, Aaron J Goldenberg, Yue Guan, Christina Gutierrez-Ford, Kandamurugu M…
BACKGROUND: As population-based genomic screening (PGS) programs are increasingly implemented, standardized, evidence-based approaches are needed to support decision-making about which genetic findings should be returned based on clinical actionability. The Clinical Genome Resource (ClinGen) Actionability Framework, which guides decision-making on the return of monogenic secondary findings, was adapted to create a framework for the context of PGS in adults. METHODS: ClinGen convened a group of experts in public health, medical genetics and genomics, genetic counseling, population screening, health economics, and bioethics. Adaptation of the ClinGen Actionability Framework included special considerations for reporting genetic findings in the PGS context, such as screening in unselected populations, penetrance certainty, and cost-effectiveness. Several frameworks relevant to PGS, including public health decision-making and bioethics frameworks, were reviewed and items were abstracted. For items deemed relevant to the PGS context, a thematic clustering method was used to group similar items into conceptually related clusters. Through an iterative process of discussion and consensus building, the clusters were reviewed, refined, and used to define domains of actionability specific to the PGS context. These final domains were selected based on conceptual clarity, distinctiveness, and their relevance to decision-making about the return of genomic findings in unselected populations. RESULTS: The ClinGen PGS Actionability Framework includes a set of expanded domains that integrate clinical and public health perspectives on actionability while preserving alignment with the original structure and purpose of the ClinGen AWG framework. The framework guides the review and curation of evidence of actionability for genes and associated conditions, consensus scores for domains of actionability for specific outcome-intervention pairs, and assertions on the level of actionability. CONCLUSIONS: The ClinGen PGS Actionability Framework provides an evidence-based resource to support clinical communities and decision-makers with policy development on the return of genetic findings in PGS programs.
Global & regional health technology assessmentStefania Brancone, Claudia Passante, Mariangela Prada
INTRODUCTION: Artificial Intelligence (AI) is rapidly expanding across the pharmaceutical value chain; however, empirical evidence on its application in Market Access (MA) remains limited. Given the strategic relevance and regulatory complexity of MA, understanding current AI adoption and perception represents a significant evidence gap. This study provides one of the first exploratory, European-focused assessments specifically investigating the role of AI in pharmaceutical MA. METHODS: A structured, closed survey was conducted among European pharmaceutical MA executives and senior leaders. The questionnaire investigated AI adoption levels, governance and implementation models, perceived benefits and barriers, and current and future applications, with a focus on MA activities. The survey was distributed between June and November 2025 through targeted professional outreach and conference-based data collection. RESULTS: Fifty responses were collected. Overall, 44% of respondents reported structural AI implementation within their organizations, with MA being the most impacted function (59%), contrasting with previous analyses where MA was among the least affected areas. AI adoption was strongly linked to company size, favoring large companies. Key barriers to AI adoption in MA included limited availability of high-quality data, data integration challenges, lack of internal expertise, and regulatory uncertainty. Reported benefits were mainly operational, while respondents identified strong future potential in market analysis, forecasting, health economics and outcomes research, and pricing and reimbursement strategy. CONCLUSIONS: AI is increasingly perceived as a valuable enabler in MA, although adoption remains heterogeneous and at an intermediate maturity stage. Effective integration requires robust governance, regulatory clarity, and AI-generated insights that support-rather than replace-human expertise.
ESMO real world data and digital oncologyJ Marin-Corral, N Iglesias, S Menke, D Casadevall, I H Medrano, J Tello, A J Muñoz, B E Miller, H Mehanna, M Rodríguez, M Taberna
BACKGROUND: The secondary use of electronic health records (EHRs) offers a major opportunity to generate real-world evidence (RWE) to complement clinical trials and support regulatory and clinical decision making. However, a large proportion of clinically relevant information remains embedded in unstructured text, limiting its systematic reuse, particularly in complex areas such as oncology. This article presents the Savana Next-Generation Registry (SNGR), a methodology for building oncology-focused registries through the extraction and structuring of unstructured EHR data. MATERIALS AND METHODS: SNGR uses EHRead®, a clinical natural language processing engine trained on multilingual EHRs, to transform free-text data into standardized clinical variables. By extracting clinically relevant information from unstructured clinical narratives, the approach enriches existing structured EHR data, enabling more complete and clinically meaningful datasets. The solution integrates medical expert input, terminology curation, and a multilayer quality assurance framework designed to support data validity, reproducibility, and traceability. RESULTS: SNGR is aligned with European regulatory frameworks, including the European Health Data Space, General Data Protection Regulation, and Data Act, enabling compliant and interoperable secondary data use. To date, it has supported over 65 real-world use cases across multiple therapeutic areas, including oncology, generating >30 peer-reviewed publications and analysis-ready datasets in multicenter and international collaborations. CONCLUSIONS: SNGR demonstrates how unstructured EHR data can be transformed into analysis-ready datasets for RWE generation. It provides a scalable and methodologically robust infrastructure for next-generation registries, particularly suited for oncology and other data-rich clinical domains, supporting research, drug development, and health care decision making. As the framework evolves, future studies will continue to expand its evidence base.
AIM: The primary aim of this study was to assess the feasibility, acceptability, and procedures of conducting a randomised controlled trial and to determine whether the prespecified progression criteria for advancing to a full trial were met. Secondary outcomes explored the clinical outcomes, process evaluation and health economics. METHODS: This was a two-arm, randomised feasibility study with 1:1 allocation conducted in the UK. The intervention lasted 6-months. Clinical outcome variables (quality of life, anxiety, loneliness) were collated at baseline, 6- and 12-months post intervention. The RE-AIM framework (Acceptability, Reach, Effectiveness, Adoption, Implementation and Maintenance) was used to undertake the process evaluation. A health economic evaluation was also explored. RESULTS: We recruited 57 participants of our target population of 64 (89%), 28 participants were randomly allocated to MATILDA and 29 to an active control group. Two participants died and one withdrew from the intervention arm prior to commencement of the intervention. We were able to match all 25 participants to a community group, most with a mentor(s). With regards reach and implementation, most older adults with an intellectual disability were still attending their community groups at 6-months (N = 18, 72%) and at 12-months (N = 17, 68%). There was a slight decrease in the anxiety scores for the participants in Matilda over the 6- and 12-months follow-up periods compared to the control group. MATILDA was found to be clearly acceptable and adoptable by the community groups and mentors. There were some challenges identifying and recruiting community groups and getting mentors to complete their questionnaires. DISCUSSION: MATILDA appears to be a promising community-based intervention to support older adults with an intellectual disability that promotes social inclusion, quality of life, and wellbeing. This study advances our empirical knowledge in understanding the contextual factors, mechanisms and outcomes in developing and delivering a community intervention for this population.
BMJ openFabio Veneruso, Anna Barnes, Christian Graeff, Heidi Probst, Michael Schlander, Tracy Underwood, Lennart Volz, Vincent Grégoire, Lionel Perrier
INTRODUCTION: Modern radiotherapy modalities have increased treatment precision and clinical effectiveness. However, the economic evidence that underpins their adoption is highly heterogeneous, with substantial variation in costing methods, data sources, analytic designs (trial-based, real-world/observational and decision-analytic modelling), perspectives and the treatment of uncertainty. We propose a systematic mapping review to characterise the peer-reviewed evidence base and identify methodological gaps relevant to health technology assessment and policy-making. This systematic mapping review aims to: (1) systematically catalogue the published, peer-reviewed evidence and the methodological approaches used in economic evaluations of modern radiotherapy; (2) identify methodological trends, including the uptake and implementation of value of information analysis and (3) highlight evidence gaps by tumour type, geographical region and methodological approach. METHODS AND ANALYSIS: Following the Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR) guidelines, we searched PubMed (MEDLINE), Embase, Scopus, Web of Science Core Collection and EconLit for economic evaluations of radiotherapy published from 2005 onwards, with an initial search on 24 February 2026 and a planned search update before synthesis. Study selection is ongoing; extraction and quality appraisal in duplicate have not been undertaken and no results are reported. Reporting completeness, methodological limitations and uncertainty will be appraised as three separate dimensions using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist, the Bias in Economic Evaluation (ECOBIAS) checklist and the TRansparent Uncertainty ASsessmenT (TRUST) framework, applied descriptively and not combined into a single quality score. Methodological information will be extracted, including model structure (eg, Markov model), analytical perspective (eg, healthcare payer), time horizon, costing approach (eg, diagnosis-related group (DRG)-based) and utility measurement (eg, EQ-5D). The methods used to assess uncertainty will be mapped for all studies, with the ECOBIAS checklist and the TRUST framework only applied to decision-analytic model-based evaluations. Findings will be synthesised descriptively as an evidence gap map and visualised as heat maps and bubble plots. A conceptual co-occurrence network, analysed in R with visualisation in VOSviewer, will map how modalities, tumour sites and economic characteristics cluster across studies. No meta-analysis is planned, given the expected heterogeneity of our findings. ETHICS AND DISSEMINATION: This review does not require ethical approval, as it will use data from previously published studies. The results will be disseminated through peer-reviewed publications, conference presentations and open-access releases of data extraction files and visual maps in the Open Science Framework (OSF). REGISTRATION: This review protocol was prospectively registered (9 February 2026) in the OSF Registries (DOI: 10.17605/OSF.IO/6W2TJ).
Bundesgesundheitsblatt, Gesundheitsforschung, GesundheitsschutzSebastian Böhme, Jessica Paas, Zoe Konjer, Jonas Meyrer, Jonas Schreyögg
Rising health expenditures and demographic change increasingly challenge the financing and steering of healthcare in Germany. Cost-sharing instruments are recurrently discussed as a means of reducing inappropriate utilisation, most recently in the context of a primary care gatekeeping system. This article analyses the institutional design and empirical evidence on financial steering instruments in four European health systems (Germany, the Netherlands, France, Denmark) and synthesises the findings from a health economics perspective. The effectiveness of copayments emerges as a function of three interrelated conditions: the type of service, the behavioural-economic design and the institutional architecture. Uniform copayments are regressive and disproportionately burden insured persons with low income. Based on the international evidence, a reintroduction of the previous German practice fee (Praxisgebühr) is not advisable. A more promising approach would design copayments as a targeted steering instrument at points where inappropriate utilisation is empirically identified, such as when patients inappropriately bypass a coordinated care pathway. This requires accounting for behavioural-economic effects and income-related differences. Configured in this way, copayments can contribute to improving patient steering without jeopardising access to standard care.