Journal of market access & health policy最新文献

When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation. 基于年金的支付何时有助于解决资助先进疗法的可负担性挑战？来自预算影响模拟的见解。

Journal of market access & health policy Pub Date : 2026-04-20 DOI: 10.3390/jmahp14020023

Walter Van Dyck, Sissel Michelsen, David Veredas, Isabelle Huys, Jeroen Luyten, Steven Simoens

{"title":"When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation.","authors":"Walter Van Dyck, Sissel Michelsen, David Veredas, Isabelle Huys, Jeroen Luyten, Steven Simoens","doi":"10.3390/jmahp14020023","DOIUrl":"https://doi.org/10.3390/jmahp14020023","url":null,"abstract":"Spreading payments over time by means of annuities has been proposed as a means to address the affordability challenge of funding very expensive advanced therapies, especially within managed entry agreements. This study aims to examine when annuities (in contrast with a single upfront payment) offer a viable solution for both healthcare payers and manufacturers to fund one-time advanced therapies. We put forward four conditions under which annuity-based payments can be considered an acceptable payment strategy: (1) excessive budget impact, (2) cost equivalence with upfront payment, (3) compensation for financial risk and (4) a limited annuity period. We develop an exploratory model that simulates how the budget impact of annuity-based payments for advanced therapies meets these conditions across several economic and epidemiological scenarios. Given our model parameter values, results suggest that annuity-based payments are most suitable when the initial patient volume (prevalence) significantly exceeds annual new cases (incidence), and when the financial risk premium for the annuity-based payment scheme does not exceed the social discount rate. While further refinement of the model is needed, this study demonstrates that annuity-based payments can only help control the annual budget need when the focus is on a high-prevalence disease, and the therapy is financed through health impact bonds issued by a governmental payer. This arrangement ensures a low-risk premium, which is typically only available to public payers.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108133/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791449","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework. 低收入和中等收入国家儿童肺炎的家庭自付负担费用：证据审查和计量经济学框架。

Journal of market access & health policy Pub Date : 2026-04-13 DOI: 10.3390/jmahp14020022

Ioannis Smaraidos, Maria Kyrmanidou, Asterios Kampouras

{"title":"Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework.","authors":"Ioannis Smaraidos, Maria Kyrmanidou, Asterios Kampouras","doi":"10.3390/jmahp14020022","DOIUrl":"https://doi.org/10.3390/jmahp14020022","url":null,"abstract":"Pediatric pneumonia remains a major cause of morbidity and mortality in low- and middle-income countries (LMICs), imposing both health and financial burdens. While the clinical aspects of pediatric pneumonia are well-studied, less attention has been paid to its economic implications for households, particularly regarding out-of-pocket (OOP) expenditure. This paper synthesizes current evidence from Kenya, India, Bangladesh, and Vietnam and introduces a proposed econometric framework designed to identify cost determinants and model policy interventions. The framework integrates microeconomic data, identifies cost determinants, and models the effects of clinical and policy factors (e.g., intensive care, vaccination, insurance coverage) on household expenditures. Simulated results illustrate potential findings from such an approach. Existing studies show substantial variability in hospitalization costs, with OOP payments ranging from US$30 to US$250 per episode, often exceeding 20% of monthly household income. Econometric modeling using generalized linear models (GLMs) and difference-in-differences (DiD) can disentangle the impact of hospital practices, disease severity, and policy interventions. Simulated regression results demonstrate that length of stay, intensive care admission, and absence of insurance significantly increase household costs, while pneumococcal conjugate vaccine (PCV) introduction reduces both admissions and financial burden. Hospitalization for pediatric pneumonia imposes significant OOP costs on households in LMICs. An econometric framework provides rigorous tools to estimate cost drivers, evaluate policy impacts, and guide equitable health financing reforms.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108171/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791277","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Accessibility of Biologic Drugs in Morocco: Comparison with FDA and EMA Approvals (2015-2025). 摩洛哥生物药品的可及性：与FDA和EMA批准的比较（2015-2025）

Journal of market access & health policy Pub Date : 2026-04-09 DOI: 10.3390/jmahp14020021

Mounir Charrak, Yahia Cherrah, Samira Serragui

{"title":"Accessibility of Biologic Drugs in Morocco: Comparison with FDA and EMA Approvals (2015-2025).","authors":"Mounir Charrak, Yahia Cherrah, Samira Serragui","doi":"10.3390/jmahp14020021","DOIUrl":"https://doi.org/10.3390/jmahp14020021","url":null,"abstract":"This study aims to evaluate the rates and timeframes of the availability and reimbursement of biologic drugs in Morocco, after approval by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The results will help to identify disparities in access and promote rapid access to these innovative treatments. This descriptive study established an international reference list of biological medicines, based on data from the FDA and EMA for the period from 2015 to 2025. An analysis was conducted using national sources, focusing on the availability, reimbursement rates, and timeframes for each listed biological drug. Of the 233 listed biological drugs, only 13.7% (32/233) of those approved between 2015 and 2025 are available in Morocco. Of these, 87.5% (28/32) have been priced, and only 10.7% (3/28) have been approved for reimbursement. The average time between FDA/EMA approval and pricing in Morocco is 3.75 years and 3.41 years, respectively, while the average reimbursement approval time is 2.74 years. This study highlights the delay and limited access for Moroccan patients to internationally approved biologic drugs.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108164/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization. 绘制真实世界证据在欧盟卫生技术评估法规中的使用：方法考虑、挑战和协调的机遇。

Journal of market access & health policy Pub Date : 2026-04-08 DOI: 10.3390/jmahp14020020

Grammati Sarri, Bengt Liljas, Keith R Abrams, Stephen J Duffield, Murtuza Bharmal

{"title":"Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization.","authors":"Grammati Sarri, Bengt Liljas, Keith R Abrams, Stephen J Duffield, Murtuza Bharmal","doi":"10.3390/jmahp14020020","DOIUrl":"https://doi.org/10.3390/jmahp14020020","url":null,"abstract":"Methodological guidelines for real-world evidence (RWE) in European Union (EU) joint clinical assessments (JCA) are lacking. This manuscript explores RWE potential in EU health technology assessment (HTA) and offers recommendations for generating high-quality RWE. An environmental scan of peer-reviewed and gray literature was conducted to review RWE frameworks and documents in EU regulatory and HTA decision-making. Extraction elements were standardized across key RWE themes: data quality, methodological rigor, stakeholder engagement, and applications. In JCA, RWE has multiple uses, including informing PICO simulation exercises, understanding disease landscape, identifying prognostic factors and effect modifiers, and directly or indirectly informing comparative clinical assessments. Methodological guidance from the HTA Coordination Group is limited to cases in which evidence from non-randomized studies is used as direct inputs in comparative assessments. Individual HTA bodies provide more detailed guidance, missing an opportunity to leverage RWE within JCAs that can offer insight for local Member State submissions. Generating high-quality RWE that is credible, actionable, and acceptable for JCA submissions and local HTA bodies requires careful attention to methodological considerations and early planning. Broader RWE integration that reflects patient journeys is needed. Expanding the HTA Coordination Group guidance can unlock RWE's full potential in supporting EU JCA submissions.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108034/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791321","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans-A Health Policy Perspective. 当行动主义成为生存：巴尔干地区数字时代持续抵抗的心理健康成本——卫生政策视角。

Journal of market access & health policy Pub Date : 2026-04-02 DOI: 10.3390/jmahp14020019

Aleksandar Sič, Svetozar Mijuskovic, Nebojsa Brezic

{"title":"When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans-A Health Policy Perspective.","authors":"Aleksandar Sič, Svetozar Mijuskovic, Nebojsa Brezic","doi":"10.3390/jmahp14020019","DOIUrl":"https://doi.org/10.3390/jmahp14020019","url":null,"abstract":"Activism exposes individuals to sustained harassment, threat and psychological strain in contexts marked by discrimination and weak institutional protection. For LGBTQ communities, public engagement frequently increases vulnerability to both offline and digital harm, with cumulative consequences for mental health. Using the Balkans as a case example, this perspective sees activist mental health through a public health and health policy lens, framing distress not as an individual coping failure but as an outcome of structural barriers and minority stress processes, including inadequate legal protection, limited access to culturally competent mental health care and insufficient accountability for platform-mediated harm. This article highlights the population-level implications of unaddressed structural stressors, like burnout, disengagement and reduced sustainability of civil society participation, by situating activist mental health within broader questions of health system performance, access to care and governance. Upstream policy responses that strengthen institutional protection, ensure equitable access to mental health services and promote safer digital environments would address these challenges, positioning activist mental health as a critical public health policy issue.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108053/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791332","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis. 在腰椎融合术中，3d打印患者特异性椎间固定器与现成椎间固定器的成本-效益：马尔科夫模型成本-效用分析。

Journal of market access & health policy Pub Date : 2026-03-25 DOI: 10.3390/jmahp14020018

Jackson C Hill, Ralph J Mobbs, Marc Coughlan, Kevin A Seex, Chloe A Amaro, William R Walsh, William C H Parr

{"title":"Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis.","authors":"Jackson C Hill, Ralph J Mobbs, Marc Coughlan, Kevin A Seex, Chloe A Amaro, William R Walsh, William C H Parr","doi":"10.3390/jmahp14020018","DOIUrl":"https://doi.org/10.3390/jmahp14020018","url":null,"abstract":"The aim of the present study was to compare the cost-effectiveness of 3DMorphic's spinal 3DFusion Lumbar (3DFL) cages versus Off-The-Shelf (OTS) cages for patients undergoing lumbar interbody fusion in an Australian healthcare setting. 3DFL cages differ from generic OTS cages in that they are Patient-Specific Interbody Cages (PSICs). While several studies have discussed the clinical benefits of PSIC versus OTS cages, no studies have evaluated the cost-effectiveness of this technology. Without a direct randomised controlled trial between the two implant categories, an indirect treatment comparison was performed. The indirect comparison was informed by a clinical trial of 3DFL cages, the Australian Spine Registry and an analysis of reoperation rates for patients undergoing spinal fusion in an Australian cohort. In conclusion, the PSICs were demonstrated to be clinically superior to OTS cages as measured by Health Related Quality of Life (HRQoL) and reoperation rates. The cost-utility analysis demonstrated that 3DFL cages were cost-effective compared to OTS cages in an Australian healthcare setting.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108168/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791331","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand. 使用泰国高成本抗癌药物真实世界数据的管理准入协议技术的比较成本评估。

Journal of market access & health policy Pub Date : 2026-03-20 DOI: 10.3390/jmahp14010017

Piyapat Owat, Chaoncin Sooksriwong, Hataiwan Ratanabunjerdkul, Tuangrat Phodha

{"title":"Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand.","authors":"Piyapat Owat, Chaoncin Sooksriwong, Hataiwan Ratanabunjerdkul, Tuangrat Phodha","doi":"10.3390/jmahp14010017","DOIUrl":"10.3390/jmahp14010017","url":null,"abstract":"High-cost innovative anticancer drugs pose challenges for health systems in balancing timely patient access with long-term financial sustainability. In Thailand, reliance on Health Technology Assessment for reimbursement decisions may delay access, highlighting the potential role of Managed Entry Agreements (MEAs) as complementary policy instruments to manage uncertainty related to price, effectiveness, and use; however, MEA application remains limited and lacks an analytical framework for technique selection. This study used real-world data from Thammasat University Hospital to examine and compare the cost-saving performance of five MEA techniques-discount, free initiation treatment, utilization cap, conditional treatment continuation, and pay-by-result-across six high-cost anticancer drugs representing dominant uncertainty characteristics. Drug procurement costs were modeled over a 24-month horizon from the payer's perspective, and one-way sensitivity analyses were conducted using ±10% variation in median progression-free survival. Free initiation treatment generated the highest cost savings across uncertainty types, followed by conditional treatment continuation, while utilization cap and discount produced more moderate savings. Pay-by-result demonstrated the lowest cost-saving potential. Sensitivity analyses confirmed the robustness of comparative rankings. Overall, the findings indicate that MEA performance varies according to dominant sources of drug-related uncertainty and support a more structured, context-appropriate approach to MEA selection to strengthen market access and value-based pricing in Thailand.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13027474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147535001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40. 芬尼酮治疗左心室射血分数≥40心力衰竭的全新健康经济模型验证

Journal of market access & health policy Pub Date : 2026-03-11 DOI: 10.3390/jmahp14010016

Tobiasz Lemański, Kerstin Folkerts, Phil McEwan, Paul Mernagh, Mateusz Robert Żemojdzin, Michał Pochopień

{"title":"Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40.","authors":"Tobiasz Lemański, Kerstin Folkerts, Phil McEwan, Paul Mernagh, Mateusz Robert Żemojdzin, Michał Pochopień","doi":"10.3390/jmahp14010016","DOIUrl":"10.3390/jmahp14010016","url":null,"abstract":"This study aimed to validate the health economic model for finerenone in the treatment of patients with heart failure (HF) and left ventricular ejection fraction (LVEF) ≥40% in the United Kingdom. A Markov model informed by the pivotal FINEARTS-HF trial compared finerenone + standard of care (SoC) to SoC alone. Cross-validation was performed on the results (life years [LYs] and quality adjusted life years [QALYs]) for the SoC arm against three models in HF with LVEF >40%. External validation compared cardiovascular (CV) mortality and the number of total HF events (hospitalisation for heart failure [HFF] and urgent heart failure visit [UHFV]) against FINEARTS-HF. The model estimated similar discounted outcomes to other models in HF (6.47 vs. 6.63-7.91 LYs and 4.78 vs. 4.63-5.27 QALYs). CV deaths (22 vs. 27) and UHFV events (60 vs. 61) avoided with finerenone were similar between the model and FINEARTS-HF. The broad estimated range of avoided HHF events (205-303 vs. 219 in FINEARTS-HF) was largely driven by baseline patient age. This comprehensive validation exercise demonstrated that the finerenone model accurately estimated observed clinical data and was well aligned in its projections with previous models assessing similar populations.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13028132/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147534994","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies. 解决药物开发中未满足的医疗需求：对监管和临床开发策略的评估和影响。

Journal of market access & health policy Pub Date : 2026-03-09 DOI: 10.3390/jmahp14010015

Carla Domingo-Esteban, Inka Heikkinen, Nanco Hefting

{"title":"Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies.","authors":"Carla Domingo-Esteban, Inka Heikkinen, Nanco Hefting","doi":"10.3390/jmahp14010015","DOIUrl":"10.3390/jmahp14010015","url":null,"abstract":"Unmet need is a core component of many Health Technology Assessment (HTA) processes at EU and national level. Most visibly, it is a core selection criterion for Joint Scientific Consultations (JSC) and Joint Clinical Assessment (JCA) for medical devices. This qualitative study explored how Unmet Medical Needs (UMNs) are understood and applied in drug development, with an emphasis on the European regulatory, HTA and access context, and examined their impact on regulatory and clinical development strategies. Twenty semi-structured interviews were conducted with representatives from regulatory authorities, HTA bodies, clinical development, industry, and patient insight roles. Data was analyzed using a thematic content approach combining deductive and inductive coding. Thematic analysis revealed general agreement on the importance of addressing UMNs, but also substantial variation in how they are defined and prioritized. Regulators often stressed disease severity and clinical evidence, while patients and clinicians emphasized quality of life. HTA representatives highlighted comparative benefit and long-term outcomes. These differing perspectives shaped how UMNs were integrated into development strategies, trial design, and regulatory planning. The findings indicate that clearer yet adaptable criteria could support earlier and more consistent alignment. Based on the analysis, a five-part roadmap to guide drug development is proposed, focusing on internal coordination, structured stakeholder engagement, collaboration between regulators and HTA bodies, adaptable definitions, and transparent decision-making. Together, these elements aim to support more systematic and predictable approaches to identifying and addressing unmet needs in drug development.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13027914/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147534983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations. 罕见病我们能相信PAICs吗？方法论的挑战和局限性。

Journal of market access & health policy Pub Date : 2026-03-06 DOI: 10.3390/jmahp14010014

Mikolaj Parkitny, Samuel Aballéa, Piotr Wojciechowski, Mondher Toumi

{"title":"Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations.","authors":"Mikolaj Parkitny, Samuel Aballéa, Piotr Wojciechowski, Mondher Toumi","doi":"10.3390/jmahp14010014","DOIUrl":"10.3390/jmahp14010014","url":null,"abstract":"Population-adjusted indirect comparisons (PAICs), including Matching-Adjusted Indirect Comparison and Simulated Treatment Comparison, are increasingly used to inform health technology assessments. These methods offer a pragmatic approach to generating comparative evidence between treatments when head-to-head trial data are unavailable and standard indirect treatment comparison methods are unfeasible. In rare diseases, however, PAICs often face substantial methodological challenges arising from small sample sizes, limited covariate overlap, and the frequent use of unanchored comparisons that rely on unverifiable assumptions. These limitations can lead to unstable estimates, reduced precision, and bias that may undermine the reliability of findings. Methodological refinements-such as optimized weighting, Bayesian approaches, and doubly robust estimators-provide some improvements but do not resolve these fundamental issues. Current European Joint Clinical Assessment guidance recommends that anchored PAICs be applied with great caution, while unanchored PAICs are considered highly problematic, and other methods should be used instead. We argue that PAICs can play a supportive role within a multidimensional and deliberative HTA process, contributing to comparative assessment alongside other evidence sources when available data are limited. However, their results require careful interpretation and transparent communication of uncertainty. Future research should prioritize the further development of formal frameworks to quantify bias and systematically assess robustness, thereby preventing overstatement of the credibility of PAIC-derived evidence in rare disease contexts.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13028332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147534979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0