Applied Health Economics and Health Policy最新文献

{"title":"Societal Preferences for Assessment Pathways of Rare Disease Drugs: A Discrete Choice Experiment","authors":"Constanza Vargas,&nbsp;Richard De Abreu Lourenco,&nbsp;Deborah J. Street,&nbsp;Manuel Espinoza,&nbsp;Stephen Goodall","doi":"10.1007/s40258-026-01047-1","DOIUrl":"10.1007/s40258-026-01047-1","url":null,"abstract":"<div><h3>Background</h3><p>Countries have implemented different assessment pathways for public funding of drugs for rare diseases, resulting in inequities in access. Understanding public preferences for these processes could enhance decision-making legitimacy and acceptance.</p><h3>Objective</h3><p>We aimed to elicit societal preferences for different assessment pathways that lead to public reimbursement to drugs for rare diseases.</p><h3>Methods</h3><p>A discrete choice experiment was conducted among adults from the general population in Australia who were asked to assume the role of a government advisor and chose between two different assessment pathways. Attributes describing the assessment pathways were identified through a literature review and expert focus groups. Each respondent completed eight choice tasks. Data were analysed using conditional logit, mixed logit and latent class models. Willingness to wait was calculated as the ratio of attribute coefficients to access time.</p><h3>Results</h3><p>There were 1099 respondents who completed the survey. Overall, respondents preferred assessment pathways that incorporated comprehensive evidence and stakeholder input. The mixed logit model revealed preference heterogeneity across four of the five attributes, with the latent class analysis identifying three groups: Class 1 (28.1%) preferred use of all available evidence, Class 3 (20.2%) opposed long wait times, and Class 2 (52%) showed no strong preferences. On average, respondents were willing to wait 32 additional months to incorporate all types of evidence into the decision-making process, and an additional 14 months for broad stakeholder involvement. Acceptable delays indicate the value placed on greater certainty and broader consultation rather than recommended policy waiting times.</p><h3>Conclusions</h3><p>This study examined societal preferences for assessment pathways for rare disease medicines. Robust evidence, covering safety, clinical effectiveness and cost effectiveness, and enhanced stakeholder involvement were the most influential factors. Respondents were also willing to delay patient access to ensure decisions were supported by strong evidence.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 5","pages":"857 - 867"},"PeriodicalIF":3.1,"publicationDate":"2026-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01047-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148025989","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases","authors":"Fatemeh Hosseini,&nbsp;Mohammadreza Mobinizadeh,&nbsp;William V. Padula","doi":"10.1007/s40258-026-01048-0","DOIUrl":"10.1007/s40258-026-01048-0","url":null,"abstract":"<div><p>Health technology assessment (HTA) informs evidence-based decision-making for resource allocation in healthcare; however, the topic selection phase that determines which technologies proceed to full assessment is underexplored and often lacks fairness and transparency, particularly disadvantaging orphan medicinal products (OMPs) for rare diseases. We drew on selectively identified literature and illustrative HTA experiences to advance an opinion-driven argument about equity in topic selection for OMPs, grouping insights into three domains: structural challenges, conceptual frameworks and policy strategies. Structural barriers in topic selection include a lack of institutionalised, participatory processes, overreliance on prioritisation criteria implicitly aligned with population-wide impact and minimal engagement with stakeholders representing the rare disease community, resulting in the systematic under-prioritisation of OMPs. Procedural and value frameworks, including multi-criteria decision analysis (MCDA), evidence-informed deliberative processes (EDPs), accountability for reasonableness (A4R), equity weighting and Health Equity Impact Assessment (HEIA), can help agencies incorporate social value and fairness before formal appraisal, but remain unevenly operationalised. Policy strategies such as equity-oriented horizon scanning, open nomination systems, and special pathways for OMPs are highlighted as feasible approaches to promote fairness. Achieving equity in HTA topic selection for OMPs requires both policy and cultural change, and embedding pluralistic value frameworks, engaging stakeholders and adopting specific policy tools are essential steps towards ensuring that rarity is met with heightened ethical attention rather than exclusion.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"647 - 654"},"PeriodicalIF":3.3,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01048-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147961865","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Structured Expert Elicitation of Long-Term Survival in Non-small Cell Lung Cancer: A Case Study Applying Guidance from NICE Technical Support Document 26","authors":"Jessica E. Forsyth,&nbsp;Monika Achra,&nbsp;Javier Baena Espinar,&nbsp;Maximilian Hochmair,&nbsp;Astra M. Liepa,&nbsp;Alan Lenox-Smith,&nbsp;Tarun Puri,&nbsp;Rahul Rajesh,&nbsp;Michael D. Sonksen,&nbsp;Yvonne Summers,&nbsp;Anthonie J. van der Wekken,&nbsp;Min-Hua Jen,&nbsp;Shijie Ren","doi":"10.1007/s40258-026-01046-2","DOIUrl":"10.1007/s40258-026-01046-2","url":null,"abstract":"<div><h3>Background</h3><p>Extrapolation of overall survival in health technology assessment (HTA) is a key area of uncertainty, as equally well-fitting models can generate divergent lifetime projections. This can lead to significant implications for decision-making. Subject experts are often consulted on the plausibility of survival projections, but input is rarely obtained in a robust manner.</p><h3>Objective</h3><p>This case study applies guidance from the National Institute for Health and Care Excellence Decision Support Unit Technical Support Document (TSD) on using structured expert elicitation for long-term survival estimation (TSD 26). This study focuses on its feasibility of implementation from the perspective of a company preparing a HTA submission.</p><h3>Methods</h3><p>The REVEL trial, evaluating ramucirumab plus docetaxel for stage IV non-small cell lung cancer, was selected as a case study. The elicitation was based on the Sheffield Elicitation Framework (SHELF) and undertaken in accordance with TSD 26.</p><h3>Results</h3><p>Five experts from four countries attended across three virtual workshops. Experts provided quantitative judgements on overall survival at 4 years for the intervention and comparator from the REVEL trial. Qualitative discussion of the change in hazard and scenario testing demonstrated the internal consistency of experts’ judgments and provided valuable context for expert judgements. Group discussion in separate workshops posed challenges when the facilitation team had to relay the views of absent experts.</p><h3>Conclusions</h3><p>This case study demonstrates that it is feasible to follow the recommendations provided in TSD 26 when conducting elicitation for long-term survival outcomes and highlights the critical role of discussing the hazard function in group discussions.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"763 - 776"},"PeriodicalIF":3.3,"publicationDate":"2026-05-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01046-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147866470","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating System-Wide Healthcare Costs Using a Health System Model: Application to the Thanzi La Onse Model of Malawi","authors":"Sakshi Mohan,&nbsp;Newton Chagoma,&nbsp;Simon Walker,&nbsp;Christian Abraham Arega,&nbsp;Martin Chalkley,&nbsp;Joseph Collins,&nbsp;Emilia Connolly,&nbsp;Tim Colbourn,&nbsp;Eva Janoušková,&nbsp;Tara D. Mangal,&nbsp;Gerald Manthalu,&nbsp;Joseph Mfutso-Bengo,&nbsp;Margherita Molaro,&nbsp;Dominic Nkhoma,&nbsp;Andrew Phillips,&nbsp;Lalit Sharma,&nbsp;Bingling She,&nbsp;Wiktoria Tafesse,&nbsp;Pakwanja Desiree Twea,&nbsp;Paul Revill,&nbsp;Timothy B. Hallett","doi":"10.1007/s40258-026-01030-w","DOIUrl":"10.1007/s40258-026-01030-w","url":null,"abstract":"<div><h3>Objectives</h3><p>Modelling approaches that consider system-wide delivery platforms rather than single diseases can be instrumental in economic evaluation and forward-looking policy formulation. This study develops a costing approach tailored to the Thanzi La Onse (TLO) model of Malawi’s healthcare system, with general applicability to other health system models.</p><h3>Methods</h3><p>We developed a mixed-method costing approach to estimate the total cost of healthcare delivery (excluding high-level administrative costs) in Malawi using the TLO model, from a healthcare provider perspective. Through iterative adjustments of key parameters, we aligned model-based estimates as closely as possible with real-world expenditure and budget data. Costs were projected for 2023–2030 under alternative scenarios of health system capacity.</p><h3>Results</h3><p>A comparison with expenditure and budget data suggests our costing method is broadly reliable for the conditions captured by the model, though some mismatches remain owing to data limitations and definitional inconsistencies. Under current system capacity, total healthcare delivery costs for 2023–2030 were estimated at 2.83 billion US dollars [95% uncertainty interval (UI), $2.80–$2.87 billion], excluding non-medical infrastructure and administrative costs, averaging $390.98 million [$385.92–$396.71 million] annually or $16.89 [$16.75–$17.08] per capita. Scenario analysis highlighted strong interdependencies within the health system. Improving consumable availability alone increased consumables costs by 4.63%, while expanding human resources for health (HRH) alone increased them by 1.43%. When both HRH and consumable availability were expanded together, consumable costs rose by 5.93%, a combined effect larger than either change alone, illustrating how bottlenecks in one component constrain the impact of improvements in another.</p><h3>Conclusions</h3><p>Mixed-method costing using health system models is a feasible and robust method to estimate and forecast healthcare delivery costs. Clarifying assumptions and limitations can improve their utility for economic analyses and evidence-based planning in the health sector.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"707 - 725"},"PeriodicalIF":3.3,"publicationDate":"2026-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147809960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost Effectiveness of Endovascular Thrombectomy for Large Ischemic Strokes: A US Healthcare Payer Markov Model Informed by Randomized Trials","authors":"Rami Z. Morsi,&nbsp;Yuan Zhang,&nbsp;Elena Badillo Goicoechea,&nbsp;Harsh Desai,&nbsp;Sachin A. Kothari,&nbsp;Sonam Thind,&nbsp;Archit B. Baskaran,&nbsp;Ahmad Chahine,&nbsp;James E. Siegler,&nbsp;Elisheva R. Coleman,&nbsp;James R. Brorson,&nbsp;Ali Mansour,&nbsp;Shyam Prabhakaran,&nbsp;Tareq Kass-Hout","doi":"10.1007/s40258-026-01045-3","DOIUrl":"10.1007/s40258-026-01045-3","url":null,"abstract":"<div><h3>Background and Objective</h3><p>Endovascular thrombectomy is standard for acute large-vessel occlusion stroke, but the value of endovascular thrombectomy in patients with large ischemic regions remains uncertain from a US healthcare payer perspective. This study evaluated the cost effectiveness of endovascular thrombectomy plus medical management (MM) versus MM alone in patients with large ischemic regions, synthesizing data from a recent meta-analysis of all available randomized trials.</p><h3>Methods</h3><p>We developed a decision tree linked to a Markov model to perform a cost-utility analysis of endovascular thrombectomy plus MM versus MM alone from a US healthcare payer perspective over 90-day, 1-year, 5-year, and 20-year horizons. The target population was adults with acute ischemic stroke from large-vessel occlusion and Alberta Stroke Program Early CT Score (ASPECTS) &lt; 6 or infarct core volume ≥ 50 mL. Clinical inputs came from a systematic review of randomized trials. The primary outcome was the incremental cost-effectiveness ratio, expressed as cost per quality-adjusted life-year (QALY) gained. Scenario analyses (90-day, 1-year, 5-year, and 20-year horizons) and sensitivity analyses evaluated uncertainty.</p><h3>Results</h3><p>Over 20 years, endovascular thrombectomy plus MM yielded 0.44 additional QALYs and US dollars 19,611 higher costs versus MM alone, with an incremental cost-effectiveness ratio of US dollars 45,117 per QALY. Endovascular thrombectomy plus MM was cost effective in 59.4% and 94.3% of simulations at willingness-to-pay thresholds of US dollars 50,000 and 100,000 per QALY, respectively, whereas MM alone was dominant over a 90‑day horizon.</p><h3>Conclusions</h3><p>From a US healthcare payer perspective, endovascular thrombectomy for large ischemic strokes is unlikely to be cost effective in the short term but becomes more likely to be cost effective over a 20-year time horizon at commonly used willingness-to-pay thresholds, highlighting the importance of adopting long‑term perspectives in coverage and reimbursement decisions.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"777 - 789"},"PeriodicalIF":3.3,"publicationDate":"2026-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01045-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147715707","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Costs and Budget Impact of a Health System Strengthening Intervention for Hypertension and Cardiovascular Disease Care in Ghana","authors":"Kavita Singh,&nbsp;Eugene Kallson,&nbsp;Elom Hillary Otchi,&nbsp;Rupal Shah-Rohlfs,&nbsp;Juliette Selom Edzeame,&nbsp;Fionn Harnischfeger,&nbsp;Alfred Edwin Yawson,&nbsp;Manuela De Allegri","doi":"10.1007/s40258-026-01043-5","DOIUrl":"10.1007/s40258-026-01043-5","url":null,"abstract":"<div><h3>Objectives</h3><p> Cardiovascular diseases (CVDs) are a leading contributor to morbidity, mortality, and healthcare spending in low- and middle-income countries (LMICs). Despite this burden, economic evidence on system-level interventions to strengthen hypertension and CVD care in sub-Saharan Africa remains limited. This study estimates the costs and budget impact of the Ghana Heart Initiative (GHI), a multi-component health systems strengthening intervention.</p><h3>Methods</h3><p> We conducted a retrospective cost analysis from the health system perspective using an activity-based costing approach. We estimated the financial and economic costs incurred during the design (2018–2019) and initial implementation phase (2020–2022) of the GHI across 42 public health facilities in the Greater Accra Region. Costs were disaggregated by activity cluster and input category. We then projected the budget impact of hypothetical national scale-up under three implementation scenarios that varied in scope and cost-sharing assumptions. All costs were inflation-adjusted and reported in 2024 US dollars (US $).</p><h3>Results</h3><p> The total economic cost of designing and implementing the GHI over 4 years was US$1.96 million, of which 91.5% was attributable to implementation activities. Human resources were the primary cost driver. Average annual economic costs were US$11,960 per health facility, US$997 per targeted health provider trained, and US$39.9 per outpatient attendee. Projected annual costs for nationwide scale-up ranged from US$12.1 million to US$30.1 million, depending on implementation scenario, with a streamlined service-delivery–only model representing a lower-bound estimate.</p><h3>Conclusions</h3><p> This study provides granular, activity-level cost estimates for a complex health system strengthening intervention targeting hypertension and CVD care. The findings offer policy-relevant inputs for budget planning and highlight how implementation choices influence the fiscal implications of scaling up system-level CVD interventions in LMICs.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"751 - 762"},"PeriodicalIF":3.3,"publicationDate":"2026-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01043-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147687631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality-Adjusted Life Expectancy and Cost-Effectiveness Thresholds in Three High-Income Regions in Asia","authors":"Xinyu Zhao,&nbsp;Yawen Jiang","doi":"10.1007/s40258-026-01044-4","DOIUrl":"10.1007/s40258-026-01044-4","url":null,"abstract":"<div><h3>Background and Objective</h3><p>Cost-effectiveness thresholds (CETs) are essential for health technology assessments and resource allocation in healthcare. However, empirical CET estimates alongside quality-adjusted life expectancy are absent for the Hong Kong Special Administrative Region of China (Hong Kong SAR), Taiwan Province of China (Taiwan Province), and Singapore. This study aims to estimate quality-adjusted life expectancy and derive CETs in these regions.</p><h3>Methods</h3><p>Quality-adjusted life expectancy was calculated by using the Sullivan method. The CET was estimated using the value of a statistical life-based method, adjusted for age-specific life expectancy and health utilities to derive value of a statistical quality-adjusted life-year. Population-weighted value of a statistical quality-adjusted life-year values yielded regional CETs. Final estimates were corrected for overestimation, and sensitivity analyses were conducted.</p><h3>Results</h3><p>Quality-adjusted life expectancy estimates at birth were 79.98, 76.73, and 79.48 quality-adjusted life-years for Hong Kong SAR, Taiwan Province, and Singapore, respectively. Base-case CETs, expressed as times of gross domestic product per capita, were 3.12 (95% confidence interval 2.38–3.88), 2.15 (95% confidence interval 0.85–3.37), and 3.30 (95% confidence interval 1.77–4.74) for the three regions, respectively. Sensitivity analyses showed CET ranges of 2.86–3.46 (Hong Kong SAR), 2.00–2.39 (Taiwan Province), and 3.07–3.66 (Singapore) times of gross domestic product per capita for utility variations, and 2.02–3.98 (Hong Kong SAR), 1.43–2.71 (Taiwan Province), and 2.11–4.23 (Singapore) times of gross domestic product per capita for discount rate changes.</p><h3>Conclusions</h3><p>This study provides the first value of a statistical life-based CET estimates integrated with quality-adjusted life expectancy for three high-income regions in Asia. Focusing on context-specific thresholds beyond the World Health Organization benchmarks, the current findings offer a scientific basis for health technology assessments and policy decisions.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"739 - 750"},"PeriodicalIF":3.3,"publicationDate":"2026-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147662059","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lost in Translation? A Scoping Review to Explore the Translation and Cultural Adaptation of Preference-Based Measures of Quality of Life","authors":"Jenny Cleland,&nbsp;Kiri Lay,&nbsp;Julie Ratcliffe,&nbsp;Jyoti Khadka","doi":"10.1007/s40258-026-01042-6","DOIUrl":"10.1007/s40258-026-01042-6","url":null,"abstract":"<div><h3>Background</h3><p>Preference-based measures (PBMs) of quality of life are an important tool to inform the evaluation of health and social care interventions and services. Traditionally, PBMs have tended to be developed in a single country, therefore, reflecting the linguistic and cultural norms of that country. Given increases in the cultural and linguistic diversity of populations in many countries, it is important that PBMs are culturally and linguistically adapted. Linguistic adaptation ensures the translated words are correct and accurate, whilst cultural adaptation ensures the meaning and relevance of the concepts are understood as intended by the target audience. Several different guidelines for translation and cultural adaptation exist, including the guidelines developed by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).</p><h3>Objective</h3><p>The aim of this scoping review was to outline which PBMs have been translated and/or culturally adapted and specify any guidelines that have been followed.</p><h3>Methods</h3><p>Four databases (PubMed, SCOPUS, Web of Science, CINAHL) were searched (February 2025) along with grey literature to identify studies that had translated and/or culturally adapted PBMs.</p><h3>Results</h3><p>Forty-seven articles were included in the review. Translations of PBMs were common, but there was typically limited information about whether PBMs had been culturally adapted. The studies varied significantly as to whether guidelines had been followed and what steps were taken in the process.</p><h3>Conclusion</h3><p>This review highlights the variable transparency and inconsistency in the methods adopted for the translation and cultural adaptation of PBMs. The application of standardised translation and cultural adaptation guidelines, such as the ones developed by ISPOR, would enhance the methodological quality and cultural validity of PBMs.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"691 - 706"},"PeriodicalIF":3.3,"publicationDate":"2026-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01042-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147632250","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Towards Environmentally Extended Health Economic Analysis Plans: A Case Study for the MEL-SELF Trial","authors":"Jake T. W. Williams,&nbsp;Katy J. L. Bell,&nbsp;Scott McAlister,&nbsp;Rachael L. Morton","doi":"10.1007/s40258-026-01041-7","DOIUrl":"10.1007/s40258-026-01041-7","url":null,"abstract":"","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"791 - 796"},"PeriodicalIF":3.3,"publicationDate":"2026-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147626915","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Agreement in Qualitative and Quantitative Assessments of Disease Severity: Evidence from Pharmaceutical Reimbursement in Sweden","authors":"Martin Henriksson,&nbsp;Lars Sandman,&nbsp;Jonathan Siverskog","doi":"10.1007/s40258-026-01040-8","DOIUrl":"10.1007/s40258-026-01040-8","url":null,"abstract":"<div><h3>Background and Objectives</h3><p>Publicly funded healthcare systems that consider a trade-off between efficiency and equity by allowing a higher cost per patient benefit in patients with more severe conditions must somehow assess disease severity. Some countries employ quantitative measures of shortfall, whereas others rely on qualitative assessments. Despite its importance in pharmaceutical reimbursement and pricing, the operationalisation of disease severity in real-world decision making has rarely been scrutinized. The aim of this study was to investigate the relationship and agreement between qualitative disease severity assessments and quantitative measures of disease severity in Swedish pharmaceutical reimbursement.</p><h3>Methods</h3><p>Information from 36 pharmaceutical reimbursement decisions made by the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden from 2018 to 2023 was extracted, including the qualitative assessment of disease severity (moderate, high, or very high). Based on publicly available decision documents from the agency, we calculated absolute QALY shortfall (AS) and proportional QALY shortfall (PS). Linear regression was used to describe the mean shortfall across severity classifications. Ordinal logistic regression was used to analyse the role of AS and PS as predictors of TLV’s qualitative disease-severity assessments and the predictive ability of both measures was compared using the coefficient of discrimination (D’).</p><h3>Results</h3><p>The mean AS and PS was 12.2 and 0.796, respectively, in the very high disease severity category, which was approximately twice the mean shortfall observed in the moderate and high severity categories (Moderate: AS = 6.0, PS = 0.340; High: AS = 6.2, PS = 0.405). When the quantitative measures of severity were used as predictors of the qualitative assessments, PS was better able to discriminate between TLV’s severity classifications than was AS (<i>D</i>’ = 34.6% vs 22.3%). However, both measures frequently predicted low probabilities of the qualitative assessments that were observed and there was both substantial variation in shortfall for diseases with the same qualitative assessment (AS, <i>R</i>² = 35.8%; PS, <i>R</i>² = 61.0%) and overlaps in observed shortfall across different severity classifications.</p><h3>Conclusion</h3><p>Proportional QALY shortfall agrees more closely than AS with qualitatively assessed disease severity applied in the Swedish reimbursement system but there are large variations in the qualitative assessments that cannot be explained by either measure. Further investigation is warranted to understand if this is an intended and desired outcome.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 4","pages":"727 - 737"},"PeriodicalIF":3.3,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01040-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147580141","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}