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Biosimilar medicines in Malaysia: Unveiling new guidance for practice 马来西亚的生物仿制药:公布新的实践指南
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-21 DOI: 10.1016/j.hlpt.2025.101135
N Saad, Coleen SB Choo, H Chandriah, N Ahmad

Background

Biologics have transformed the treatment of chronic diseases, but their high-cost limits access and burdens healthcare budgets. Biosimilars, highly similar versions of approved biologics, offer a more affordable alternative. However, their complex nature necessitates distinct regulatory and clinical guidance to ensure their safe and effective use in the healthcare facilities.

Objective

This article highlights the policy initiatives undertaken by the Ministry of Health (MOH) Malaysia to develop a Position Statement, aimed at guiding the integration of biosimilars into Malaysia’s public healthcare system.

Method

A multi-stage process began in 2019 and involved structured stakeholder engagement, literature reviews, and consensus-building across medical and regulatory disciplines. Key areas addressed included interchangeability, switching, automatic substitution, prescribing, procurement, and pharmacovigilance. Two rounds of review were conducted, and stakeholder consensus was obtained through a binary survey, with a 70 % agreement threshold.

Results

Stakeholders from multiple specialties emphasized cautious but positive attitudes toward biosimilar use, especially for treatment-naïve or short-term therapy patients. The final guidance includes ten statements across five domains, emphasizing physician-led switching, prohibition of automatic substitution, indication-specific prescribing, and the need for robust pharmacovigilance. A total of 83 % of stakeholders supported the content, and 88 % endorsed its dissemination. The document was officially endorsed by the MOH Medicines Formulary Panel for implementation across all MOH facilities.

Conclusion

The position statements represent a critical step toward structured biosimilar adoption in Malaysia’s public healthcare. Ongoing implementation, education, and post-market evaluation will be essential to strengthen prescriber confidence, improve pharmacovigilance, and enhance patient trust in biosimilars.
生物制剂已经改变了慢性病的治疗方式,但其高昂的成本限制了获取,并增加了医疗预算负担。生物仿制药是与已获批准的生物制剂高度相似的版本,提供了一种更实惠的替代方案。然而,它们的复杂性需要独特的监管和临床指导,以确保它们在医疗机构中安全有效地使用。本文重点介绍了马来西亚卫生部(MOH)为制定立场声明而采取的政策举措,旨在指导将生物仿制药纳入马来西亚的公共医疗保健系统。方法一个多阶段的过程始于2019年,涉及结构化的利益相关者参与、文献综述以及跨医学和监管学科的共识建立。解决的关键领域包括互换性、切换、自动替代、处方、采购和药物警戒。进行了两轮审查,并通过二元调查获得利益相关者共识,同意阈值为70%。结果来自多个专业的利益相关者强调了对生物类似药使用的谨慎但积极的态度,特别是对于treatment-naïve或短期治疗患者。最终指南包括五个领域的十项声明,强调医生主导的转换、禁止自动替代、针对特定适应症的处方以及加强药物警戒的必要性。共有83%的利益相关者支持该内容,88%的人赞同其传播。该文件已得到卫生部药物处方小组的正式批准,以便在卫生部所有设施中实施。结论:该立场声明代表了马来西亚公共医疗保健采用结构化生物仿制药的关键一步。持续的实施、教育和上市后评价对于加强处方者信心、提高药物警戒性和增强患者对生物仿制药的信任至关重要。
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引用次数: 0
Impact of assistive technologies on caregiver burden and perseverance for people with dementia at home 辅助技术对在家照顾痴呆症患者的负担和毅力的影响
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-19 DOI: 10.1016/j.hlpt.2025.101134
G. Bagnasco , J. van Exel

Objectives

This study examines the relationship between the use of assistive technologies and the burden and perseverance time of informal caregivers of community-dwelling people with dementia.

Methods

An online survey of 342 informal caregivers in the Netherlands assessed objective burden (tasks and time involved in caregiving), subjective burden (personal experience of stress and strain), current perseverance time (time expected to be able to continue providing care), and well-being (overall happiness). Data included demographics, housing, neighbourhood characteristics, assistive technology use, and preferences for additional technologies. Multiple logistic regressions examined associations between assistive technology categories and caregiver outcomes (i.e., below-median weekly caregiving hours, Self-Rated Burden score; above-median CarerQol-7D score, CarerQol-VAS score, and Current Perseverance Time in months). Furthermore, multiple logistic regressions estimated the impact of the most desired technology category on the likelihood of extending the perseverance time by more than six months. Analyses were stratified by caregiver living arrangement (co-residing vs living nearby).

Results

Among co-residing caregivers, daily living support technologies were associated with reduced caregiving hours, whereas risk prevention technologies were linked to longer current perseverance time but also higher self-rated burden. Risk response technologies were also associated with higher self-rated burden. Among caregivers living nearby, risk prevention technologies were associated with higher quality of life. Moreover, daily living support and risk response technologies most strongly extended perseverance time beyond six months in both groups.

Conclusions

Assistive technologies play a nuanced role in supporting informal caregivers of community-dwelling people with dementia, with impacts differing by living arrangement and technology type.
目的探讨辅助技术的使用与社区痴呆患者非正式照护者负担和坚持时间的关系。方法对荷兰342名非正式护理人员进行在线调查,评估客观负担(护理任务和时间)、主观负担(个人压力和紧张经历)、当前坚持时间(预计能够继续提供护理的时间)和幸福感(总体幸福感)。数据包括人口统计、住房、社区特征、辅助技术使用和对其他技术的偏好。多重逻辑回归检验了辅助技术类别与护理人员结果(即每周护理时间低于中位数、自评负担评分、高于中位数的CarerQol-7D评分、CarerQol-VAS评分和当前坚持时间(以月为单位)之间的关系。此外,多重逻辑回归估计了最期望的技术类别对延长坚持时间超过六个月的可能性的影响。根据照顾者生活安排(共同居住vs住在附近)对分析进行分层。结果在共同居住的照顾者中,日常生活支持技术与减少照顾时间有关,而风险预防技术与更长的当前坚持时间有关,但也与更高的自评负担有关。风险应对技术也与较高的自评负担相关。在住在附近的护理人员中,风险预防技术与更高的生活质量相关。此外,日常生活支持和风险应对技术最有力地延长了两组的坚持时间超过6个月。结论辅助技术在支持社区痴呆患者非正式照护者方面发挥着微妙的作用,其影响因居住安排和技术类型而异。
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引用次数: 0
Value-centred commissioning in primary health care: reform lessons from the Portuguese experience 初级卫生保健中以价值为中心的委托:来自葡萄牙经验的改革教训
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-14 DOI: 10.1016/j.hlpt.2025.101133
Paulo Santos , Isabel Nazaré , Luísa Sá

Background

Portuguese Primary Health Care commissioning has transitioned from centrally managed agreements to performance-based models. Despite this evolution, persistent structural and procedural inefficiencies limit its capacity to generate value. Comparative European experiences highlight alternative frameworks that emphasise outcome relevance, decentralisation, and system learning.

Objective

To propose a conceptual model for primary health care commissioning aligned with structure–process–outcome logic.

Methods

We conducted a narrative review to trace the evolution of commissioning in Portuguese Primary Health Care, focusing on its regulatory, financial, and performance roles. We used several sources, including legislation, policy documents, national and European reports, and academic literature. Analysis followed a structure–process–outcome framework. Comparative insights highlighted governance models, incentive structures, and reform opportunities aligned with value-based care and European benchmarks.

Results

Portuguese commissioning systems lack clinically meaningful outcome indicators and remain rigid in design. The new model offers a framework for aligning commissioning processes with population health needs, clinical relevance, and system adaptability. Comparative analysis shows that decentralised, context-sensitive models from other European countries are applicable and promising.

Conclusions

Commissioning reforms should centre around auditable, evidence-based outcome indicators tailored to local contexts. These indicators can enhance motivation, accountability, and continuous learning. Reforms are achievable within current organisational structures and planning cycles. Policymakers should consider decentralisation and a value-oriented approach to improve Primary Health Care delivery and responsiveness, particularly in systems facing similar structural constraints.
葡萄牙初级卫生保健委托已从中央管理的协议过渡到基于绩效的模式。尽管有这种发展,但持续的结构和程序上的低效限制了其创造价值的能力。比较欧洲的经验突出了强调结果相关性、权力下放和系统学习的替代框架。目的提出符合结构-过程-结果逻辑的初级卫生保健委托概念模型。方法我们进行了一项叙述性回顾,以追踪葡萄牙初级卫生保健委托的演变,重点关注其监管、财务和绩效角色。我们使用了几个来源,包括立法、政策文件、国家和欧洲报告以及学术文献。分析遵循结构-过程-结果框架。比较见解强调了与基于价值的护理和欧洲基准相一致的治理模式、激励结构和改革机会。结果葡萄牙的调试系统缺乏临床意义的结果指标,设计仍然僵化。新模式提供了一个框架,使调试过程与人口健康需求、临床相关性和系统适应性保持一致。对比分析表明,来自其他欧洲国家的分散的、上下文敏感的模式是适用的和有前途的。委托改革应围绕可审计的、基于证据的、适合当地情况的结果指标展开。这些指标可以增强动机、责任和持续学习。在目前的组织结构和规划周期内,改革是可以实现的。决策者应考虑权力下放和以价值为导向的方法,以改善初级卫生保健的提供和响应能力,特别是在面临类似结构限制的系统中。
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引用次数: 0
Four years upscaling telemonitoring to future-proof health care delivery in Dutch university hospitals: Before and after - where do we stand and what are the results? 四年来,荷兰大学医院将远程监控升级为面向未来的医疗服务:前后——我们的立场是什么,结果是什么?
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-13 DOI: 10.1016/j.hlpt.2025.101132
HJ Harm Gijsbers , S Azam Nurmohamed , Linda W Dusseljee–Peute , Tom H van de Belt , Marlies P. Schijven

Objectives

Upscaling digital solutions is crucial for future-proofing healthcare, yet effective and sustainable methods are underrepresented in literature. In the Netherlands, a University Medical Center (UMC) network collaborated within the Citrien eHealth program to future-proof care using telemonitoring (TM). From 2020–2022, three TM activities were initiated: TM for cardiac rhythm abnormalities/heart failure (TMCardio), TM of blood pressure in high-risk pregnant women (TMAnteNatal), and continuous wireless monitoring of vital functions (TMVitals). This study evaluated the scale-up of these TM programs in seven Dutch UMCs.

Methods

An uncontrolled before-after study design was used.Primary outcome, ‘normalization of telemonitoring,’ was assessed using the 20-item NoMAD questionnaire. Results were structured according to the non-adoption, abandonment, scale-up, spread and sustainability (NASSS) framework. Secondary outcomes included the number of UMCs offering TM programs and the number of patients using TM.

Results

The NoMAD questionnaire had a 61 % response rate. Respondents were familiar with TM (N = 85, mean = 7.27, SD=2.13) and believed it would become more normal in their work (N = 98, mean = 8.42, SD=1.64). However, a significant difference between current practice and expected future use of TM was observed (p≤ 0.001). All UMCs ran TM programs, but not all implemented all three projects. TMCardio patients increased from 190 to 5185, TMAnteNatal from 41 to 1162, and TMVitals from 2666 to 13630.

Conclusion

Telemonitoring uptake increased across Dutch UMCs, but not uniformly. The complexity of scale-up is highest in NASSS domains 4, 5, and 6, with a focus on management, resources, health care reimbursement, and regulations. Understanding the impact of the Citrien network collaboration on scale requires further qualitative analysis.
升级数字解决方案对于面向未来的医疗保健至关重要,但文献中有效和可持续的方法代表性不足。在荷兰,一个大学医学中心(UMC)网络与Citrien eHealth项目合作,使用远程监控(TM)进行面向未来的护理。从2020-2022年,启动三个TM活动:心律异常/心力衰竭TM (TMCardio)、高危孕妇血压TM (tmtenatal)和生命功能连续无线监测(tmvital)。本研究评估了这些TM项目在荷兰七所大学的规模。方法采用前后对照研究设计。主要结果“远程监护的正常化”使用20项NoMAD问卷进行评估。结果根据不采用、放弃、扩大、传播和可持续性(NASSS)框架进行结构化。次要结果包括提供TM项目的UMCs数量和使用TM的患者数量。结果NoMAD问卷有效率为61%。受访者对TM较为熟悉(N = 85,均值= 7.27,SD=2.13),认为TM在工作中会变得更加正常(N = 98,均值= 8.42,SD=1.64)。然而,观察到当前实践和预期未来使用TM之间存在显著差异(p≤0.001)。所有umc都运行TM程序,但并不是所有umc都实现了这三个项目。TMCardio患者从190例增加到5185例,tmmantenatal从41例增加到1162例,TMVitals从2666例增加到13630例。结论远程监护的使用在荷兰各医院有所增加,但并不均匀。扩大规模的复杂性在NASSS领域4、5和6中最高,重点是管理、资源、医疗报销和法规。了解Citrien网络协作对规模的影响需要进一步的定性分析。
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引用次数: 0
Teleconsultation for chronic disease management among community-dwelling older adults: A call for innovation 社区老年人慢性病管理远程会诊:创新的呼唤
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-11 DOI: 10.1016/j.hlpt.2025.101131
Su Wei Wan , Lina Choe , Gretel Jianlin Wong , Wee Ling Koh , Janelle Shaina Ng , Wee Hian Tan , Joanna Li Xin Ooi , Jacquelyn Melody , Ker Kan Tan

Objectives

Telehealth services have been around for a while but utilization of teleconsultation among older adults remains low despite its extensive and known benefits. Behavioural intentions are multifactorial and nuanced, and qualitative evidence describing the acceptance of teleconsults for chronic disease management is scarce. Hence, this study explored the perspectives of older adults towards teleconsultation use in the primary care setting.

Methods

A descriptive multi-site qualitative study was conducted. Semi-structured interviews guided by the Unified Theory of Acceptance and Use of Technology (UTAUT) model were held with twenty community-dwelling older adults ≥ 65 years on routine follow-up for three common chronic diseases in Singapore: hypertension, hypercholesterol, and type II diabetes mellitus. Data was analysed using the inductive thematic approach.

Results

Five themes were yielded: (i) more than just a physical consult; (ii) the difference in modes – does it really matter?; (iii) psychological receptivity to new technology; (iv) appropriateness of teleconsultation for medical condition; and (v) moderating factors. Participants were generally ambivalent towards teleconsultation as they could foresee its advantages but also its limitations which were thought to affect treatment. Many lacked knowledge of this remote modality which led to preconceptions of it being non-intuitive and difficult to adopt in the absence of facilitators that promote their viability.

Conclusions

Older adults’ acceptance of teleconsultation for chronic disease management seems conditional and non-optimistic at present. Physician advocacy, digital trainings, structured policies and age-appropriate teleconsult configurations with matured features that can mitigate the shortfalls of remote consultation will help enhance older adults’ acceptance of such services.

Public interest summary

While the potential and proven merits of telemedicine are plentiful, this remote modality remains foreign and intimidating to the majority of older adults. This study explored teleconsult acceptability for chronic disease management with the UTAUT framework. Findings revealed several pressing concerns which indicate the need for more digital initiatives and healthcare advocacy for its uptake in the community. Besides user-related programs, there is room for system improvements to include mature features that may offload the burden on users to learn the technology.
目的远程保健服务已经存在了一段时间,但老年人远程咨询的使用率仍然很低,尽管它有广泛和已知的好处。行为意向是多因素和微妙的,定性证据描述接受远程咨询的慢性病管理是稀缺的。因此,本研究探讨了老年人对远程会诊使用在初级保健设置的观点。方法采用描述性多地点定性研究。在接受和使用技术统一理论(UTAUT)模型的指导下,对20名≥65岁的新加坡社区老年人进行了半结构化访谈,并对高血压、高胆固醇和II型糖尿病这三种常见慢性病进行了常规随访。数据分析采用归纳专题方法。结果产生了五个主题:(i)不仅仅是物理咨询;(ii)模式的差异——这真的重要吗?(iii)对新技术的心理接受能力;(iv)就医疗状况进行远程会诊的适当性;(五)调节因素。参与者通常对远程咨询持矛盾态度,因为他们可以预见其优势,但也可以预见其局限性,这些局限性被认为会影响治疗。许多人对这种远程方式缺乏了解,导致先入为主地认为这种方式不直观,在缺乏促进其可行性的促进者的情况下难以采用。结论目前成人对慢性病远程会诊的接受程度有条件且不容乐观。医生倡导、数字化培训、结构化政策和适合年龄的远程会诊配置,这些成熟的功能可以缓解远程会诊的不足,有助于提高老年人对此类服务的接受度。虽然远程医疗的潜力和已被证明的优点很多,但这种远程模式对大多数老年人来说仍然是陌生的和令人生畏的。本研究探讨远程会诊在UTAUT框架下对慢性病管理的可接受性。调查结果揭示了几个紧迫的问题,表明需要更多的数字举措和医疗保健宣传,以便在社区中采用。除了与用户相关的程序之外,系统还有改进的空间,可以包括成熟的功能,从而减轻用户学习技术的负担。
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引用次数: 0
Orphan medicine policy and decision-making frameworks: A narrative review of HTA methodologies 孤儿药政策和决策框架:HTA方法的叙述性回顾
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-08 DOI: 10.1016/j.hlpt.2025.101130
Mohammadreza Mobinizadeh , William V. Padula , Min-Hua Jen , Efat Mohamadi , Sahar Salehi

Objectives

Orphan medicinal products (OMPs) often face unique policy and reimbursement challenges due to small patient populations, limited clinical data, and high costs. This review aims to provide the first comprehensive, cross‑methodological and cross‑stage synthesis of how CEA, GCEA, MCDA, and BIA have been applied in OMPs policymaking, addressing critical gaps in integrating findings across HTA stages and income‑level contexts.

Methods

This narrative review searched PubMed and Google Scholar for English‑language studies published between 2008 and 2024, using keywords related to rare diseases, policymaking, and the three key stages of the health technology policymaking process: topic selection, assessment, and appraisal. Studies were included if they addressed at least one of these stages in the context of OMPs.

Results

This review identified 17 studies related to policymaking for orphan medicinal products, of which 6 focused on topic selection—including 3 that explicitly examined horizon scanning—and 11 on the combined assessment and appraisal stages. Methodologically, 3 studies applied CEA or GCEA, 8 explored MCDA (2 for topic selection and 6 for assessment and appraisal stages), and 2 employed BIA, with some studies using more than one approach. CEA serves as the standard but struggles to incorporate societal, ethical, and dynamic market factors, often limiting its applicability for OMPs. GCEA addresses such gaps with additional considerations like disease severity and equity but remains methodologically complex. MCDA offers a more comprehensive framework by integrating clinical, economic, and qualitative benefits, although challenges like attribute weighting and stakeholder inclusion persist. BIA is instrumental in assessing affordability but lacks clinical outcome integration. Additionally, Managed Entry Agreements (MEAs) although not an HTA methodology in themselves - have emerged in several countries as practical policy tools for mitigating financial risk and bridging evidence gaps, thereby complementing the application of CEA, GCEA, MCDA, and BIA in real‑world OMPs decision‑making.

Conclusion

Integrating GCEA, MCDA, BIA, and complementary tools such as MEAs alongside systematic horizon scanning within the topic selection stage creates a robust framework for addressing the complexities of orphan drug policymaking across all HTA stages. Future work should focus on developing a technical input–output design for the proposed integrated chain HTA model, ensuring that outputs from horizon scanning and topic selection feed directly into structured assessment modules, and that appraisal outputs are systematically linked to policy tools such as MEAs and BIA.
由于患者人数少、临床数据有限和成本高,孤儿药(omp)往往面临独特的政策和报销挑战。本综述旨在首次全面、跨方法、跨阶段地综合分析CEA、GCEA、MCDA和BIA是如何应用于omp政策制定的,解决在整合HTA各阶段和收入水平背景下的研究结果方面的关键差距。方法本叙述性综述检索了PubMed和谷歌Scholar在2008年至2024年间发表的英语研究,使用与罕见病、政策制定以及卫生技术决策过程的三个关键阶段(主题选择、评估和评估)相关的关键词。如果研究在omp的背景下涉及这些阶段中的至少一个,则纳入研究。结果本综述共发现17项与孤儿药政策制定相关的研究,其中6项集中在选题上,其中3项明确考察了水平扫描,11项集中在综合评估和评价阶段。方法学上,3项研究采用CEA或GCEA, 8项研究采用MCDA(2项用于选题,6项用于评估和评价阶段),2项研究采用BIA,其中一些研究使用了多种方法。CEA作为标准发挥作用,但努力将社会、伦理和动态市场因素纳入其中,这通常限制了其对omp的适用性。GCEA通过诸如疾病严重程度和公平性等额外考虑来解决这些差距,但在方法上仍然很复杂。MCDA通过整合临床、经济和定性效益提供了更全面的框架,尽管属性加权和利益相关者包容等挑战仍然存在。BIA有助于评估可负担性,但缺乏临床结果整合。此外,管理准入协议(MEAs)虽然本身不是一种HTA方法,但已在一些国家作为减轻财务风险和弥合证据差距的实用政策工具出现,从而补充了CEA、GCEA、MCDA和BIA在现实世界OMPs决策中的应用。将GCEA、MCDA、BIA和mea等辅助工具与主题选择阶段的系统水平扫描相结合,可以为解决所有HTA阶段孤儿药政策制定的复杂性创建一个强大的框架。未来的工作应侧重于为拟议的综合链HTA模型开发技术投入产出设计,确保水平扫描和主题选择的产出直接提供给结构化评估模块,并确保评估产出系统地与多边环境协定和BIA等政策工具联系起来。
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引用次数: 0
A real-world comparison of outcomes and healthcare resource utilization of Transcatheter Aortic Valve Implantation (TAVI) and Surgical Aortic Valve Replacement (SAVR) 经导管主动脉瓣植入术(TAVI)与外科主动脉瓣置换术(SAVR)的临床疗效及医疗资源利用比较
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-11-06 DOI: 10.1016/j.hlpt.2025.101129
Giaele Moretti , Chiara Seghieri , Claudio Passino , Milena Vainieri

Objectives

This study aims to assess and compare the real-world outcomes and healthcare resource utilization of Transcatheter Aortic Valve Implantation (TAVI) and Surgical Aortic Valve Replacement (SAVR) procedures, acknowledging the challenges associated with medical device evaluations.

Methods

The two cohorts of TAVI and SAVR patients were identified using individual-level administrative data in the Tuscany region of Italy from 2016 to 2021. Patients in treatment cohorts were followed for up to one-year post-procedure, with outcomes and costs assessed. Clinical indicators were selected from the Valve Academic Research Consortium-3 (VARC-3) consensus document. Follow-up costs, in euros (€), were calculated for each patient up to three years post-procedure, from the perspective of the Italian National Healthcare System.

Results

No significant differences in 30-day and 1-year mortality were found between TAVI and SAVR, though TAVI showed a slight increase in 3-year mortality (OR 1.05, p = 0.004). TAVI patients had higher rates of conduction disturbances and pacemaker implantation at all time points. They also experienced more ER admissions and hospital readmissions at 3 years, but shorter hospital stays. At 1 year, TAVI incurred in €234 higher total costs, driven mainly by higher ER costs, while pharmaceutical costs were similar. At 3 years, total costs were €2132 for TAVI and €1915 for SAVR, with higher ambulatory and ER costs in the TAVI group but lower pharmaceutical costs.

Conclusion

The study explores the potential of Real-World Evidence to inform the clinical and economic evaluation of new technologies and procedures. The study differs from some prior randomized controlled trial-based studies, highlighting the impact of diverse analytical approaches and patient populations.

Public interest abstract

Aortic stenosis (AS) is a common heart valve disease in the elderly, whose standard treatment consists of Surgical Aortic Valve Replacement (SAVR). For patients that are ineligible for surgery due to high risk or comorbidities, Transcatheter Aortic Valve Implantation (TAVI), has emerged as a less invasive option. This study used real-world data from Tuscany, Italy, to compare clinical outcomes and costs. We found no significant difference in mortality rates between TAVI and SAVR at 30 days and one year and higher mortality at 3 years. TAVI patients were more likely to experience conduction disturbances, often requiring pacemaker implantation. Cost differences were modest, with TAVI patients incurring €234 more in total healthcare expenses over one year and €217 at three years. These findings highlight the value of real-world evidence in assessing the safety, effectiveness, and financial sustainability of new treatments like TAVI.
本研究旨在评估和比较经导管主动脉瓣植入术(TAVI)和外科主动脉瓣置换术(SAVR)的实际结果和医疗资源利用,并承认与医疗器械评估相关的挑战。方法使用意大利托斯卡纳地区2016年至2021年的个人行政数据确定TAVI和SAVR患者的两个队列。治疗组的患者在手术后随访长达一年,评估结果和成本。临床指标选择自瓣膜学术研究联盟-3 (VARC-3)共识文件。从意大利国家医疗保健系统的角度出发,计算每位患者术后三年的随访费用,以欧元(€)为单位。结果TAVI组与SAVR组30天死亡率和1年死亡率无显著差异,但TAVI组3年死亡率略有升高(OR 1.05, p = 0.004)。TAVI患者在所有时间点都有较高的传导障碍和起搏器植入率。他们在3年内也经历了更多的急诊和再入院,但住院时间更短。1年后,TAVI的总成本增加了234欧元,主要是由于ER成本增加,而药品成本也相似。3年,TAVI的总成本为2132欧元,SAVR的总成本为1915欧元,TAVI组的门诊和急诊成本较高,但药品成本较低。结论:本研究探索了真实世界证据的潜力,为新技术和新程序的临床和经济评估提供信息。该研究不同于先前的一些基于随机对照试验的研究,突出了不同分析方法和患者群体的影响。主动脉瓣狭窄(AS)是老年人常见的心脏瓣膜疾病,其标准治疗方法为外科主动脉瓣置换术(SAVR)。对于因高风险或合并症而不适合手术的患者,经导管主动脉瓣植入术(TAVI)已成为一种侵入性较小的选择。这项研究使用了来自意大利托斯卡纳的真实数据来比较临床结果和成本。我们发现TAVI和SAVR在30天和1年的死亡率没有显著差异,3年的死亡率更高。TAVI患者更容易出现传导障碍,通常需要植入起搏器。成本差异不大,TAVI患者一年的总医疗费用多出234欧元,三年多出217欧元。这些发现突出了现实世界证据在评估TAVI等新疗法的安全性、有效性和财务可持续性方面的价值。
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引用次数: 0
Co-design and formative usability evaluation of a multifunctional mhealth app for chronic vestibular syndrome management 慢性前庭综合征管理多功能移动健康应用程序的共同设计和形成性可用性评估
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-10-30 DOI: 10.1016/j.hlpt.2025.101128
Xuejiao Cao , Yanping Yu , Ruiqi Zhang , Huawei Li , Wenyan Li , Peixia Wu

Objectives

Chronic vestibular syndrome (CVS) refers to chronic dizziness and imbalance symptoms persisting for ≥3 months. The treatment and management of patients with CVS remain a global challenge. The effectiveness of mobile application in assisting people with other chronic conditions has been proved. This study aims to co-design and develop an app prototype for patients with CVS innovatively, then explore users’ formative evaluation for this app prototype.

Methods

This research adopted the qualitative research method. Two rounds of focus groups were conducted to determine the modules and functions of the app. Then the app prototype was developed accordingly. Subsequently, patients or their main caregiver were interviewed to explore their formative evaluation and optimization requirements, which in accordance with the ISO 9241–11 usability framework.

Results

During the focus groups, eight modules were identified for this app, including 20 specific functions in total. On the basis of focus group results, an app prototype was developed over a period of six months. Interview results indicated that participants expressed positive evaluations regarding the usefulness of this app. However, there were several impediments in terms of usability. In the end, seven user requirements for app optimization were obtained.

Conclusions

This study designed and developed an app based on expert focus groups. Target users provided relatively positive evaluations on this prototype. This study provided preliminary evidence on the acceptability and usability of mobile apps for managing patients with CVS.

Public Interest Summary

Chronic dizziness and imbalance can significantly affect daily life, making it hard for people with chronic vestibular syndrome (CVS) to manage their condition. In our study, we created a user-friendly mobile health app specifically designed for CVS patients. The app offers features to support symptom tracking, rehabilitation exercises, and educational resources. By involving both medical experts and patients in the co-design process, we ensured the app meets the real needs of users. Preliminary feedback from patients suggests that the app is practical and could help improve their quality of life. This innovation has the potential to make managing CVS more accessible and effective, reducing the challenges these patients face every day.
目的慢性前庭综合征(chronic vestibular syndrome, CVS)是指持续≥3个月的慢性头晕和失衡症状。CVS患者的治疗和管理仍然是一个全球性的挑战。移动应用程序在帮助其他慢性病患者方面的有效性已得到证实。本研究旨在创新性地共同设计和开发CVS患者应用原型,并探索用户对该应用原型的形成性评价。方法本研究采用定性研究方法。通过两轮焦点小组来确定app的模块和功能,并据此开发app原型。随后,根据ISO 9241-11可用性框架,对患者或其主要照顾者进行访谈,探讨其形成性评估和优化要求。结果在焦点小组中,确定了该应用程序的8个模块,包括20个具体功能。根据焦点小组的结果,我们花了6个月的时间开发了一款应用原型。采访结果表明,参与者对这款应用的实用性表达了积极的评价。然而,在可用性方面存在一些障碍。最后得到了7个用户对应用优化的需求。本研究设计并开发了一款基于专家焦点小组的app。目标用户对该原型的评价比较积极。本研究为移动应用程序管理CVS患者的可接受性和可用性提供了初步证据。慢性头晕和失衡会严重影响日常生活,使患有慢性前庭综合征(CVS)的人难以控制自己的病情。在我们的研究中,我们创建了一个专门为CVS患者设计的用户友好的移动健康应用程序。该应用程序提供了支持症状跟踪、康复练习和教育资源的功能。通过让医学专家和患者共同参与设计过程,我们确保了应用程序满足用户的真正需求。患者的初步反馈表明,该应用程序是实用的,可以帮助提高他们的生活质量。这项创新有可能使CVS管理更容易获得和有效,减少这些患者每天面临的挑战。
{"title":"Co-design and formative usability evaluation of a multifunctional mhealth app for chronic vestibular syndrome management","authors":"Xuejiao Cao ,&nbsp;Yanping Yu ,&nbsp;Ruiqi Zhang ,&nbsp;Huawei Li ,&nbsp;Wenyan Li ,&nbsp;Peixia Wu","doi":"10.1016/j.hlpt.2025.101128","DOIUrl":"10.1016/j.hlpt.2025.101128","url":null,"abstract":"<div><h3>Objectives</h3><div>Chronic vestibular syndrome (CVS) refers to chronic dizziness and imbalance symptoms persisting for ≥3 months. The treatment and management of patients with CVS remain a global challenge. The effectiveness of mobile application in assisting people with other chronic conditions has been proved. This study aims to co-design and develop an app prototype for patients with CVS innovatively, then explore users’ formative evaluation for this app prototype.</div></div><div><h3>Methods</h3><div>This research adopted the qualitative research method. Two rounds of focus groups were conducted to determine the modules and functions of the app. Then the app prototype was developed accordingly. Subsequently, patients or their main caregiver were interviewed to explore their formative evaluation and optimization requirements, which in accordance with the ISO 9241–11 usability framework.</div></div><div><h3>Results</h3><div>During the focus groups, eight modules were identified for this app, including 20 specific functions in total. On the basis of focus group results, an app prototype was developed over a period of six months. Interview results indicated that participants expressed positive evaluations regarding the usefulness of this app. However, there were several impediments in terms of usability. In the end, seven user requirements for app optimization were obtained.</div></div><div><h3>Conclusions</h3><div>This study designed and developed an app based on expert focus groups. Target users provided relatively positive evaluations on this prototype. This study provided preliminary evidence on the acceptability and usability of mobile apps for managing patients with CVS.</div></div><div><h3>Public Interest Summary</h3><div>Chronic dizziness and imbalance can significantly affect daily life, making it hard for people with chronic vestibular syndrome (CVS) to manage their condition. In our study, we created a user-friendly mobile health app specifically designed for CVS patients. The app offers features to support symptom tracking, rehabilitation exercises, and educational resources. By involving both medical experts and patients in the co-design process, we ensured the app meets the real needs of users. Preliminary feedback from patients suggests that the app is practical and could help improve their quality of life. This innovation has the potential to make managing CVS more accessible and effective, reducing the challenges these patients face every day.</div></div>","PeriodicalId":48672,"journal":{"name":"Health Policy and Technology","volume":"15 1","pages":"Article 101128"},"PeriodicalIF":3.7,"publicationDate":"2025-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145467652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Healthcare providers’ perspectives of first-tier NIPS' implementation in Canada 医疗保健提供者对加拿大一级NIPS实施的看法
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-10-24 DOI: 10.1016/j.hlpt.2025.101127
Tierry M. Laforce , Vardit Ravitsky , Marie-Christine Roy , Anne-Marie Laberge

Objective

This article presents an analysis of barriers and facilitators concerning the implementation of first-tier non-invasive prenatal screening (NIPS) in Canada, as identified by healthcare providers, using the Consolidated Framework for Implementation Research (CFIR).

Study Design

Semi-structured interviews were conducted with 16 healthcare professionals. Obstetricians-Gynecologists (OBGYN), family physicians, geneticist, midwives, and genetic counselors from 5 provinces were recruited. CFIR was used both for the interview guide and the coding dictionary. Discourse analysis was done for all interviews after data saturation was attained.

Results

The findings reveal a contrast in participant perspectives. While NIPS is perceived as a superior screening tool with the potential to replace traditional screening, concerns linger regarding its impact on pregnant individuals and the healthcare system. Participants acknowledge the positive perception of NIPS and its potential to replace traditional screening. They emphasize that NIPS does not address a gap in the screening pathway since other methods already detect the screened conditions. Despite facilitators like positive perceptions and increased accessibility to information, it necessitates careful consideration concerning NIPS' place in the prenatal screening pathway. Access to NIPS is considered a facilitator, potentially reducing inequalities, but widespread availability may compromise care for some pregnant individuals. Barriers include the lack of novelty in NIPS information and its inability to replace diagnostic tests.

Conclusion

Our research offers a comprehensive understanding of barriers and facilitators, aiding anticipation of potential implementation as a first-tier test and maximizing the likelihood of successful implementation. The use of CFIR to analyze the prenatal screening pathway provides a structured approach to identify possible tensions in implementing first-tier NIPS, and actionable insights. This research provides valuable insights for policymakers and decision-makers, underscoring the importance of education and deliberate consideration when determining NIPS's appropriate role in prenatal screening.
目的:本文分析了在加拿大实施一线非侵入性产前筛查(NIPS)的障碍和促进因素,由医疗保健提供者确定,使用实施研究的综合框架(CFIR)。研究设计:对16名医疗保健专业人员进行半结构化访谈。从5个省招募了妇产科医生、家庭医生、遗传学家、助产士和遗传咨询师。CFIR被用于面试指南和编码字典。在达到数据饱和后,对所有访谈进行语篇分析。结果研究结果揭示了参与者视角的差异。虽然NIPS被认为是一种优越的筛查工具,有可能取代传统筛查,但人们仍然担心它对孕妇和医疗保健系统的影响。与会者承认NIPS的积极看法及其取代传统筛查的潜力。他们强调,NIPS并没有解决筛选途径中的空白,因为其他方法已经检测到筛选条件。尽管有积极的看法和更多的信息可及性等促进因素,但需要仔细考虑NIPS在产前筛查途径中的地位。获得NIPS被认为是一个促进因素,可能会减少不平等,但广泛的可获得性可能会损害对一些孕妇的护理。障碍包括NIPS信息缺乏新颖性,无法替代诊断测试。我们的研究提供了对障碍和促进因素的全面理解,帮助预测潜在的实施,作为第一级测试,最大限度地提高成功实施的可能性。使用CFIR分析产前筛查途径提供了一种结构化的方法,以确定在实施一线NIPS时可能存在的紧张关系,并提供可操作的见解。这项研究为政策制定者和决策者提供了有价值的见解,强调了在确定NIPS在产前筛查中的适当作用时,教育和深思熟虑的重要性。
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引用次数: 0
Determining fair medicines prices. What do citizens think? 确定公平的药品价格。市民是怎么想的?
IF 3.7 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2025-10-22 DOI: 10.1016/j.hlpt.2025.101126
Alexander Roediger , Lennart Pirktl , Georg Schönbächler , Helmut Brand

Objectives

There is a lively debate about the fair price of medicines, mainly led by experts. Little is known about the public’s opinion, although in most health systems with universal coverage medicines are procured by public money through payers or governments. This study assesses public opinion about fair medicine prices, the criteria that define fairness, and the policy implications.

Methods

A population survey amongst 1000 people across the Swiss population in all 3 language regions has been conducted between September and October 2024.

Results

Access for all (986), transparency of the cost structure (914) and a price reflecting the costs of a medicine (911) have ranked as the most relevant factors for a fair price of medicine. In contrast, the additional benefit of a new therapy is considered less important as a criterion. A large majority supports pharmaceutical companies making a profit. Asked how to align the different objectives, the majority supports the statement that medicines prices should be the result of a fair process (568), followed by the statement that governments should control profits (431).

Conclusions

This study suggests that citizens consider the balance between patient access and investment in research and development as the most relevant for a fair price of a medicine. When access and cost create a dilemma, citizens favour fair procedures. In contrast to expert opinions, the additional benefit of a medicine seems to be a less relevant criterion. The survey results indicate a nuanced and pragmatic approach to fairness, considering societal, scientific, and economic factors.
目的围绕药品公平价格展开了一场激烈的辩论,主要由专家主导。尽管在大多数全民覆盖的卫生系统中,药品是由公共资金通过付款人或政府购买的,但公众的意见知之甚少。本研究评估了公众对公平药品价格的看法、界定公平的标准以及政策影响。方法在2024年9月至10月期间,对瑞士所有3个语言区的1000人进行了人口调查。结果“人人可及性”(986)、“成本结构透明度”(914)和“反映药品成本的价格”(911)是影响药品公平价格的最相关因素。相比之下,一种新疗法的额外益处被认为是不那么重要的标准。绝大多数人支持制药公司盈利。当被问及如何协调不同的目标时,大多数人支持药品价格应该是公平过程的结果这一说法(568人),其次是政府应该控制利润的说法(431人)。本研究表明,公民认为患者可及性和研发投资之间的平衡与药品的公平价格最为相关。当获取和成本造成两难境地时,公民倾向于公平的程序。与专家意见相反,药物的额外益处似乎是一个不太相关的标准。调查结果表明,考虑到社会、科学和经济因素,公平是一种微妙而务实的方法。
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引用次数: 0
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Health Policy and Technology
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