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Co-developing a comprehensive disease policy model with stakeholders: the case of malaria during pregnancy 与利益攸关方共同制定综合疾病政策模式:妊娠期疟疾案例
Pub Date : 2024-09-12 DOI: 10.1101/2024.09.10.24313438
Silke Fernandes, Andrew Briggs, Kara Hanson
Introduction Understanding the holistic impact of malaria during pregnancy is essential for improving maternal and child outcomes in malaria endemic settings. To be able to design appropriate research and conduct robust policy analyses, a comprehensive model of the underlying disease, representing the current understanding of mechanisms and consequences is essential. This study aimed to illustrate a methodology to co-develop a disease model with expert stakeholders using malaria during pregnancy as a case study. Methods An initial steering group was convened to develop a first model of malaria during pregnancy and its consequences for mother and child based on their understanding of the literature. Subsequently, this model was refined using a Delphi process to gain consensus amongst twelve experts, representing the disciplines of health economics, mathematical modelling, epidemiology and clinical medicine, working in the field of malaria during pregnancy. Experts reviewed drafts of the conceptual model and provided feedback in two rounds of semi-structured questionnaires with the aim of identifying the most important health outcomes and relationships in both mother and child as well as the most relevant stratifiers for the model. Consensus on any final disagreement was reached after two consensus meetings. Results The final model is a comprehensive disease model of malaria during pregnancy, including ten maternal and ten child outcomes with four stratifiers. The model developed in this study should be of value to malaria researchers, funders, evaluators and decision makers, though some adaptation will be required for each specific context and purpose. In addition, the methodology and process followed in this study is replicable and can guide researchers aiming to develop a conceptual model for other conditions. Discussion & Conclusion The model resulting from this study highlights the complexity required to depict appropriately the consequences of malaria during pregnancy for both the mother and the child. It also demonstrates how to conduct a rigorous process to develop a disease model. In addition the study has helped to identify a number of areas with scarce data and need for further research.
导言 了解孕期疟疾的整体影响对于改善疟疾流行地区的母婴预后至关重要。为了能够设计适当的研究并进行稳健的政策分析,必须建立一个全面的疾病模型,以反映目前对机制和后果的理解。本研究旨在以妊娠期疟疾为案例,说明与专家利益相关者共同开发疾病模型的方法。方法 召集了一个初步指导小组,根据他们对文献的理解,建立孕期疟疾及其对母婴影响的第一个模型。随后,采用德尔菲法对该模型进行了改进,以获得 12 位代表卫生经济学、数学建模、流行病学和临床医学等学科的孕期疟疾领域专家的共识。专家们审查了概念模型的草案,并通过两轮半结构式问卷调查提供了反馈意见,目的是确定母婴最重要的健康结果和关系,以及与模型最相关的分层因素。在两次共识会议后,就最终的分歧达成了共识。结果 最终模型是孕期疟疾的综合疾病模型,包括十项母婴结果和四项分层因素。本研究开发的模型对疟疾研究人员、资助者、评估者和决策者都有价值,但需要根据具体情况和目的进行一些调整。此外,本研究采用的方法和过程具有可复制性,可为旨在为其他情况开发概念模型的研究人员提供指导。讨论与印证;结论 本研究产生的模型凸显了适当描述孕期疟疾对母婴造成的后果所需的复杂性。它还展示了如何通过严格的程序来开发疾病模型。此外,这项研究还有助于确定一些数据稀缺和需要进一步研究的领域。
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引用次数: 0
How does participation in coal-to-gas policy and availability of natural gas pipelines affect residents' well-being? 参与煤制天然气政策和天然气管道的可用性如何影响居民的福祉?
Pub Date : 2024-09-09 DOI: 10.1101/2024.09.08.24313274
Shi Jiankui, Hu Lun, Xia yingge, Hu Xiangdong
With the rapid advancement of industrialization and urbanization, China is facing an increasingly serious challenge of air pollution. The dramatic deterioration of air quality not only compromises the quality of daily life, but also poses a serious threat to public health. In order to deal with the urgent environmental problems, the Chinese government actively seeks treatment methods, among which the coal-to-gas policy, with its advantages of clean and low-carbon, has become a key part of the environmental governance strategy. However, in the initial implementation of the coal-to-gas policy, problems such as shortage of gas sources and pipeline access hindered the promotion of the policy. To provide an in-depth analysis of the actual impact of the coal-to-gas policy on residents' well-being, a comprehensive analysis was conducted based on data from the China General Social Survey. The study found that the implementation of the coal-to-gas policy not only significantly improved residents' subjective well-being and made the environment more livable, but also significantly improved residents' physical health and reduced health problems caused by air pollution. This positive effect is particularly pronounced among young people, women and residents of northern regions, who have benefited more from the clean-air benefits of the coal-to-gas policy. In addition, the study found that the availability of natural gas pipelines has a non-linear effect on residents' well-being. Moderate pipeline coverage can significantly improve the quality of life of residents and provide more convenient and reliable clean energy. Therefore, under the premise of ensuring stable supply, it is necessary to seek the optimal pipeline layout scheme. Finally, the coal-to-gas policy has boosted regional economic vitality and residents' well-being by promoting industrial restructuring and reducing pollution emissions.
随着工业化和城市化的快速推进,中国正面临着日益严峻的空气污染挑战。空气质量的急剧恶化不仅影响了人们的日常生活质量,也对公众健康构成了严重威胁。为了应对迫在眉睫的环境问题,中国政府积极寻求治理方法,其中煤改气政策以其清洁低碳的优势,成为环境治理战略的重要组成部分。然而,在煤改气政策实施初期,气源短缺、管道不畅等问题阻碍了政策的推广。为了深入分析煤改气政策对居民福祉的实际影响,我们基于中国社会综合调查数据进行了全面分析。研究发现,煤改气政策的实施不仅显著改善了居民的主观幸福感,使环境更加宜居,还显著提高了居民的身体健康水平,减少了空气污染引起的健康问题。这种积极效应在年轻人、妇女和北方地区居民中尤为明显,他们从煤改气政策的清洁空气效益中获益更多。此外,研究还发现,天然气管道的可用性对居民的福祉有非线性影响。适度的管道覆盖可以显著提高居民的生活质量,提供更加便捷可靠的清洁能源。因此,在保障稳定供应的前提下,必须寻求最优的管道布局方案。最后,煤改气政策通过促进产业结构调整和减少污染排放,提升了区域经济活力和居民福祉。
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引用次数: 0
Causally-informative analyses of the effect of job displacement on all-cause and specific-cause mortality from the 1990s Finnish recession until 2020: A population registry study 对 20 世纪 90 年代至 2020 年芬兰经济衰退期间失业对全因和特因死亡率的影响进行因果信息分析:人口登记研究
Pub Date : 2024-08-31 DOI: 10.1101/2024.08.30.24312834
Stephanie Zellers, Elissar Azzi, Antti Latvala, Jaakko Kaprio, Terhi Maczulskij
Background Job loss is related to morbidity and mortality, but generation of causal evidence is challenging due to confounding factors. Finland suffered a severe economic recession in the early 1990s with unemployment reaching almost 25%, with many job losses due to mass layoff or company closure. Such job displacements are assumed to be exogenous to the individual and create a natural experiment for causal inference.
背景失业与发病率和死亡率有关,但由于存在混杂因素,要生成因果关系的证据具有挑战性。芬兰在 20 世纪 90 年代初经历了严重的经济衰退,失业率高达近 25%,许多工作岗位因大规模裁员或公司倒闭而丧失。这种工作岗位的转移被假定为个人的外生因素,为因果推论提供了一个自然实验。
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引用次数: 0
Privacy Protection of Sexually Transmitted Infections Information from Chinese Electronic Medical Records 中国电子病历中性传播感染信息的隐私保护
Pub Date : 2024-08-27 DOI: 10.1101/2024.08.13.24311908
Mengchun Gong, Yue Yu, Zihao OuYang, Wenzhao Shi, Chao Liu, Qilin Wang, Jiale Nan, Endi Cai, Fen Ding, Sheng Nie
Objectives: To formulate an efficacious approach for safeguarding the privacy information of electronic medical records.Design: Chinese patient electronic medical record text information.Setting: The Chinese Renal Disease Data System database.Participants: 3,233,174 patients between 1 Jan. 2010 and 31 Dec. 2023.Main outcome measures: Annotated patient privacy fields and the effectiveness of privacy protectionResults: We have developed an automated tool named EPSTII, designed to protect the privacy of patients' sexually transmitted infection information within medical records. Through the refinement of keywords and the integration of expert knowledge, EPSTII currently achieves a 100% accuracy and recall rate. Our privacy protection measures have reached a 99.5% success rate, ensuring the utmost protection of STI patients' privacy. As the first large-scale investigation into privacy leakage and STI identification in Chinese electronic medical records, our research paves the way for the future development of patient privacy protection laws in China and the advancement of more sophisticated tools.Conclusions:The EPSTII method demonstrates a feasible and effective approach to protect privacy in electronic medical records from 19 hospitals, offering comprehensive insights for infectious disease research using Chinese electronic medical records, with protocols tailored for accurate STI data extraction and enhanced protection compared to traditional methods.
目的:制定有效的方法来保护电子病历的隐私信息:制定保护电子病历隐私信息的有效方法:设计:中国患者电子病历文本信息:环境:中国肾脏病数据系统数据库:主要结果测量:注释患者隐私字段和隐私保护的有效性:我们开发了一种名为 EPSTII 的自动工具,旨在保护医疗记录中患者性传播感染信息的隐私。通过改进关键词和整合专家知识,EPSTII 目前的准确率和召回率均达到了 100%。我们的隐私保护措施成功率高达 99.5%,最大限度地保护了性传播感染患者的隐私。结论:EPSTII方法展示了一种保护19家医院电子病历隐私的可行而有效的方法,为利用中国电子病历进行传染病研究提供了全面的见解,与传统方法相比,该方法为准确提取性传播疾病数据和加强保护量身定制了方案。
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引用次数: 0
Cost-utility of a new psychosocial goal-setting and manualised support intervention for Independence in Dementia (NIDUS-Family) versus goal-setting and routine care: economic evaluation embedded within a randomised controlled trial. 痴呆症患者自立能力的新型心理社会目标设定和手动支持干预(NIDUS-Family)与目标设定和常规护理的成本效益对比:随机对照试验中的经济评估。
Pub Date : 2024-08-26 DOI: 10.1101/2024.08.24.24312530
Abdinasir Isaaq, Claudia Cooper, Victoria Vickerstaff, Julie Barber, Kate Walters, Iain Lang, Penny Rapaport, Vasiliki Orgeta, Kenneth Rockwood, Laurie Butler, Kathryn Lord, Gill Livingston, Sube Banerjee, Jill Manthorpe, Helen Kales, Jessica Budgett, Rachael M Hunter
Background: NIDUS-Family is a 6-8 session, psychosocial and behavioural intervention, delivered by non-clinical facilitators, tailored to goals set by dementia-unpaid/family carer dyads. It is effective in terms of attainment of personalised client goals. We aimed to determine if it is cost-effective. Methods: This cost utility and cost-effectiveness analysis is within a two-armed, single masked, multi-site, superiority Randomised Controlled Trial (RCT). We recruited 302 dyads from community settings. Randomisation was blocked and site-stratified, using a 2:1 ratio (intervention: control (goal-setting and routine care)), with allocation by remote web-based system. We calculated the probability that NIDUS-Family is cost-effective for a client with dementia based on Quality Adjusted Life Year (QALY) from health and personal social services and societal perspectives, at GBP 20,000-30,000 decision thresholds for QALY gained, compared to usual care over 12 months. Analyses were intention-to-treat. Trial registration: ISRCTN11425138. Findings: From 30.4.2020-9.5.2022, 204 participants (109 (53.4%) female) were randomised to intervention and 98 (60 (61.2%) female) to control. 218 (72.2%) participants at 6 months and 178 (58.9%) at 12 months provided cost data. There was 89% and 87% probability that NIDUS-Family was cost-effective compared to usual care from personal social services and societal perspectives respectively. Intervention participants accrued on average GBP 8934 (37%) less costs than control participants (95% CI -59,460 to 41,592). Interpretation: NIDUS-Family is the first personalised care and support intervention to demonstrate cost-effectiveness from the perspective of the quality of life of people with dementia, as well as clinical effectiveness and should be part of routine dementia care.
背景:NIDUS-Family 是一项为期 6-8 个疗程的社会心理和行为干预措施,由非临床促进者根据痴呆-无偿/家庭照护者二元组合设定的目标提供。从实现客户个性化目标的角度来看,该干预措施非常有效。我们的目标是确定它是否具有成本效益。方法:这项成本效用和成本效益分析是在一项双臂、单人、多地点、优越性随机对照试验(RCT)中进行的。我们从社区环境中招募了 302 对夫妇。随机分配采用阻断和地点分层方式,比例为 2:1(干预:对照(目标设定和常规护理)),并通过远程网络系统进行分配。我们从健康、个人社会服务和社会角度出发,按照获得 QALY 的 20,000-30,000 英镑决策阈值,计算了 NIDUS-Family 在 12 个月内与常规护理相比,对痴呆症患者而言具有成本效益的概率。分析方法为意向治疗。试验注册:ISRCTN11425138。研究结果:从 2020 年 4 月 30 日至 2022 年 5 月 9 日,204 名参与者(109 人(53.4%)为女性)被随机分配到干预组,98 人(60 人(61.2%)为女性)被随机分配到对照组。218 名参与者(72.2%)提供了 6 个月的成本数据,178 名参与者(58.9%)提供了 12 个月的成本数据。从个人社会服务和社会角度来看,"NIDUS-家庭 "与常规护理相比,分别有89%和87%的可能性具有成本效益。干预参与者的费用比对照参与者平均少 8934 英镑(37%)(95% CI -59460-41592)。解释:NIDUS-Family是首个从痴呆症患者的生活质量和临床效果角度证明具有成本效益的个性化护理和支持干预措施,应该成为常规痴呆症护理的一部分。
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引用次数: 0
Quantifying the health impact of crop breeding: Revisiting the Disability-Adjusted Life Years Approach 量化作物育种对健康的影响:重新审视残疾调整寿命年数法
Pub Date : 2024-08-26 DOI: 10.1101/2024.08.26.24312574
Bert Lenaerts
Crop breeding, through methods like biofortification, plays a crucial role in increasing agricultural yields, contributing to a stable and healthy food supply by boosting the micronutrient content of staples, which is pivotal for combating chronic and hidden hunger, especially in rural areas. This shift towards improved nutrition can be sustainably integrated into regular diets, marking a significant stride in public health. The burden of hunger and its consequences on health are increasingly quantified using the Disability-Adjusted Life Years (DALYs) approach, which merges years of life lost and years lived with disability, offering a comprehensive view of health impacts and aiding in resource allocation despite its complexity and subjective nature. A practical formula for quantifying the health impact of biofortification was introduced by Stein et al. (2005) and Zimmermann and Qaim (2004). This entails calculating the efficacy or relative reduction in hunger burden based on the current and post-intervention nutrient intake against the recommended dietary allowances. As data on consumption and recommended intake levels are variable and not readily available, this paper proposes relying on relative estimates to bridge the data gaps and uncertainties, thus streamlining the quantification of crop breeding's impact on diets and overall health.
通过生物强化等方法进行的作物育种在提高农业产量方面发挥着至关重要的作用,通过提高主食中的微量营养素含量,促进稳定和健康的粮食供应,这对于消除长期和隐性饥饿,尤其是农村地区的长期和隐性饥饿至关重要。这种向改善营养的转变可持续地融入常规饮食中,标志着公共卫生领域的一大进步。饥饿的负担及其对健康的影响越来越多地采用残疾调整生命年(DALYs)方法进行量化,该方法将损失的生命年数和残疾生活年数合并在一起,尽管具有复杂性和主观性,但它提供了对健康影响的全面看法,并有助于资源分配。Stein 等人(2005 年)以及 Zimmermann 和 Qaim(2004 年)提出了量化生物强化对健康影响的实用公式。这需要根据当前和干预后的营养摄入量与建议的膳食摄入量来计算饥饿负担的有效性或相对减少量。由于消耗量和推荐摄入量的数据不固定且不易获得,本文建议依靠相对估计值来弥补数据差距和不确定性,从而简化作物育种对膳食和整体健康影响的量化工作。
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引用次数: 0
The impact of financial support and budgeting on medication availability and purchasing behavior in the Nigerian primary healthcare system 财政支持和预算编制对尼日利亚初级卫生保健系统中药品供应和购买行为的影响
Pub Date : 2024-08-23 DOI: 10.1101/2024.08.23.24312494
Brittany Hagedorn, Rui Han
Previous work has shown that primary healthcare facilities can benefit from both in-kind support (e.g., medication shipments) as well as increased cash-on-hand to spend to address service readiness gaps. However, there is limited evidence on how facility managers choose to spend available cash or how their decisions to manage their facility budgets are affected by in-kind support. Economic theory suggests that the optimal allocation of cash resources would depend on the context and constraints to how it can be spent, and expenditures would in turn affect the availability of supplies and medications. We test this theory using regression analysis on data from the Nigeria Service Delivery Indicators for Health (SDI), a health facility survey from twelve states in 2013 that included both hospitals and primary healthcare centers (PHCs). We find that facilities with financial resources available to them have higher availability of essential medicines, especially if the facility had earmarked some cash for medication expenditures. However, earmarking for other expenditure categories did not have the same effect on medication availability, which indicates that budgeting processes are an important factor in ensuring medication availability. We find that cash support had large effect (p < 0.001) on availability and that in-kind donations had a negative effect on the probability of expenditure of medications. Additionally, we find the difference between hospitals and PHCs is due to their financial situation (variables become insignificant once support variables were in regressions). Regression analyses also showed that facilities that received in-kind medications had higher availability, but this only had a significant effect in facilities that did not have cash available to spend on medications, implying that facilities are able to address their own supply needs when they have resources available to them. Thus, in-kind supplies should be targeted to facilities that cannot otherwise procure them. Overall, facilities appear to be making effective trade-offs in the context of limited resources and they should receive both cash and support for appropriate budgeting and procurement practices.
以往的工作表明,基层医疗机构既可以从实物支持(如药品运输)中获益,也可以从增加的可用现金中获益,以解决服务准备不足的问题。然而,关于医疗机构管理者如何选择使用可用现金或他们管理医疗机构预算的决策如何受到实物支持影响的证据却很有限。经济学理论认为,现金资源的最佳分配将取决于使用现金的环境和限制因素,而支出反过来又会影响物资和药品的可用性。我们对尼日利亚卫生服务提供指标(SDI)的数据进行了回归分析,从而验证了这一理论。SDI 是 2013 年对 12 个州的卫生机构进行的一项调查,其中包括医院和初级保健中心(PHC)。我们发现,拥有财政资源的医疗机构拥有更高的基本药物可用性,尤其是在医疗机构已为药物支出预留部分现金的情况下。然而,为其他支出类别划拨专项资金对药品可用性的影响并不相同,这表明预算编制过程是确保药品可用性的一个重要因素。我们发现,现金支持对药品供应有很大影响(p < 0.001),而实物捐赠对药品支出概率有负面影响。此外,我们还发现医院和初级保健中心之间的差异是由其财务状况造成的(一旦支持变量进入回归,这些变量就变得不显著了)。回归分析还显示,获得实物药品的医疗机构有更高的可用性,但这只对没有现金用于药品支出的医疗机构有显著影响,这意味着医疗机构在有可用资源的情况下能够满足自身的供应需求。因此,实物供应应针对无法以其他方式采购的机构。总的来说,医疗机构似乎在有限的资源范围内做出了有效的权衡,它们应该同时获得现金和适当的预算和采购支持。
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引用次数: 0
Determining Cost-Saving Risk Thresholds for Statin Use 确定使用他汀类药物的成本节约风险阈值
Pub Date : 2024-08-20 DOI: 10.1101/2024.08.14.24311999
Afschin Gandjour
Background: Statins are widely prescribed to reduce cholesterol levels and decrease the risk of cardiovascular diseases. This study aims to determine cost-saving risk thresholds for statin use in Germany to inform better prescription practices and healthcare policies.Methods: A decision-analytic approach was employed, using secondary data to compare statin use with no statin use from the perspective of German sickness fund insurees. The analysis focused on cost savings from avoided cardiovascular (CV) events, translating these avoided events into net savings after accounting for treatment costs and potential side effects. The study considered the German adult population insured by sickness funds and used a lifetime horizon for the analysis.Results: The maximum number needed to treat (NNT) to achieve cost savings over 10 years was found to be 40, leading to a minimum risk threshold for savings of 9.8%. It was estimated that approximately 22% of the adult population in Germany has a 10-year CV risk of 9.8%, potentially avoiding between 307,049 and 705,537 CV events over 10 years, with net population savings of approximately 18 billion euros.Conclusions: The current official threshold for statin prescription in Germany, set at a 20% 10-year risk, is too stringent. Lowering the threshold to 9.8% could significantly increase the number of patients benefiting from statin therapy, reducing CV events and generating substantial cost savings. These findings suggest that adjustments to prescription guidelines could improve cardiovascular outcomes and economic efficiency within the German healthcare system.
背景:他汀类药物被广泛用于降低胆固醇水平和减少心血管疾病风险。本研究旨在确定德国他汀类药物使用的成本节约风险阈值,为更好的处方实践和医疗保健政策提供依据:方法:采用决策分析方法,利用二手数据,从德国疾病基金投保人的角度对使用他汀类药物和不使用他汀类药物进行比较。分析的重点是避免心血管(CV)事件带来的成本节约,在考虑治疗成本和潜在副作用后,将这些避免的事件转化为净节约。研究考虑了德国疾病基金投保的成年人口,并采用终生视角进行分析:结果:研究发现,10 年内实现成本节约所需的最大治疗人数(NNT)为 40 人,节约成本的最低风险阈值为 9.8%。据估计,德国约有 22% 的成年人 10 年的心血管疾病风险为 9.8%,10 年内可能避免 307,049 到 705,537 起心血管疾病事件,净节省人口成本约 180 亿欧元:德国目前官方规定的他汀类药物处方阈值为 10 年风险 20%,这一阈值过于严格。将阈值降至 9.8%,可显著增加他汀类药物治疗的受益患者人数,减少冠心病事件,并节省大量成本。这些研究结果表明,调整处方指南可以改善心血管疾病的治疗效果,提高德国医疗系统的经济效益。
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引用次数: 0
Quantifying the intangible: Evidence from Nigeria on the impact of supervision, autonomy, and management practices on PHC performance in the context of Direct Facility Financing 量化无形资产:来自尼日利亚的证据:在直接融资机制下,监督、自治和管理实践对初级保健绩效的影响
Pub Date : 2024-08-16 DOI: 10.1101/2024.08.15.24312076
Brittany Hagedorn, Benjamin Loevinsohn, Oluwole Odutolu
Previous studies have shown that facility autonomy, especially control over budget allocation, can have a modest positive effect on performance, but the findings depend on the context. Similarly, management practices are often cited as important contributors to facility performance, but the evidence is limited and usually qualitative. Data from the large-scale randomized evaluation of the Nigeria States Health Investment Project (NSHIP) offers an opportunity to quantitatively examine these relationships in the context of a lower middle-income country. We utilize non-parametric statistics to test for difference in means and apply regression analysis to test the hypothesis that autonomy and management affected facility performance. Our results show that facilities with greater autonomy, more budget control, and better management practices generally outperform their peers on a range of facility readiness and service delivery measures. For example, regression results found that facilities with high autonomy held on average 2.1 more outreach sessions per month than those without, and facilities with an annual business plan offered 1.8 additional outreach services. Supervision practices, such as more frequent visits and use of a quantitative checklist, are associated with 26% higher productivity and up to a 28.6% increase in equipment availability (percentage points), respectively. We conduct sensitivity analyses on our variable selection and use a random forest approach to validate that results are robust to changes in the model structure. We conclude that facility-level autonomy and especially budget control can improve primary healthcare facility readiness and service availability, even in resource-constrained contexts, Further, this can be achieved through good management practices that are reinforced through supportive supervision and routine performance monitoring to maximize the gains that result from incremental financing. This shows that these policies and practices can be critical contributors to efficiently achieving the goals of universal healthcare policies in the context of limited resources.
以往的研究表明,设施自主权,特别是对预算分配的控制权,可对绩效产生适度的积极影响,但研究结果取决于具体情况。同样,管理实践也经常被认为是提高医疗机构绩效的重要因素,但证据有限,而且通常是定性的。尼日利亚各州卫生投资项目(NSHIP)的大规模随机评估数据为在中低收入国家背景下定量研究这些关系提供了机会。我们利用非参数统计来检验平均值的差异,并运用回归分析来检验自主权和管理对医疗机构绩效的影响这一假设。我们的结果表明,自主权更大、预算控制更强、管理措施更完善的医疗机构在一系列医疗机构准备和服务提供措施方面的表现普遍优于其他医疗机构。例如,回归结果发现,自主性高的机构平均每月比没有自主性的机构多举办 2.1 次外联活动,而有年度业务计划的机构则多提供 1.8 次外联服务。监督实践,如更频繁的访问和使用量化检查表,分别与生产率提高 26% 和设备可用性提高 28.6% 有关(百分点)。我们对变量选择进行了敏感性分析,并使用随机森林方法验证了结果对模型结构变化的稳健性。我们的结论是,即使在资源有限的情况下,医疗机构层面的自主权,尤其是预算控制,也能改善基层医疗机构的准备情况和服务可用性。此外,这还可以通过良好的管理实践来实现,而良好的管理实践又可以通过支持性监督和日常绩效监测得到加强,从而最大限度地提高增量融资带来的收益。这表明,在资源有限的情况下,这些政策和做法可以成为有效实现全民医疗保健政策目标的关键因素。
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引用次数: 0
Is the United Kingdom (UK) medicines pricing policy failing patients? The impact of terminated National Institute for Health and Care Excellence (NICE) appraisals for multi-indication products on patients 英国的药品定价政策是否令患者失望?英国国家健康与护理优化研究所(NICE)终止多适应症产品评估对患者的影响
Pub Date : 2024-08-07 DOI: 10.1101/2024.08.06.24311489
Helen Mitchell, Qian Xin, Jack Hide, Clement Halin, Swarali Sunil Tadwalkar, Sabera Hashim, Richard Hudson
BackgroundNational Institute for Health and Care Excellence (NICE) data regarding manufacturer driven terminations indicate that some patients in the United Kingdom (UK) are unable to access treatments that are available in other European countries, which may result in reduced survival and quality of life (QoL). This study aims to quantify the health impact of NICE appraisals for multi-indication products terminated for reasons not related to clinical trial failure on the UK population. MethodsTerminated NICE appraisals (2014 to 2023) for multi-indication products were identified and a targeted literature search was conducted to identify data on the health impact of the interventions. The potential incremental quality-adjusted life year (QALY) loss and impact on overall survival (OS), progression-free survival (PFS), and QoL was calculated. ResultsOver 16,000 QALYs/year were potentially lost (with one QALY equal to one year of life in perfect health) across approximately 829,000 patients in the UK due to NICE appraisals for multi-indication products being terminated for reasons not related to clinical trial failure. Across oncology indications (approximately 18,900 patients), OS and PFS may have been reduced by over 9,400 years and 9,000 years, respectively. The potential impact of the treatments for non-oncology indications for which NICE appraisals were terminated on QoL was an incremental improvement of 13% (weighted average). ConclusionsDue to the increasing number of NICE terminations for multi-indication products, patients cannot access therapies that could lengthen their lives and increase their QoL. As the UK uniform pricing policy is likely to influence manufacturer-driven terminations, introducing alternative reimbursement arrangements such as indication-based pricing (IBP) agreements to ensure that prices remain commensurate with therapeutic value could improve access to therapies in the UK, thereby improving public health.
背景英国国家健康与护理卓越研究所(NICE)关于制造商驱动的终止的数据表明,英国的一些患者无法获得在其他欧洲国家可以获得的治疗,这可能会导致患者的生存率和生活质量(QoL)下降。本研究旨在量化因与临床试验失败无关的原因而终止的 NICE 多适应症产品评估对英国人口健康的影响。方法确定了已终止的 NICE 多适应症产品评估(2014 年至 2023 年),并进行了有针对性的文献检索,以确定有关干预措施对健康影响的数据。计算了潜在的增量质量调整生命年(QALY)损失以及对总生存期(OS)、无进展生存期(PFS)和 QoL 的影响。结果 在英国,由于 NICE 因与临床试验失败无关的原因而终止对多种适应症产品的评估,约有 829,000 名患者可能损失了超过 16,000 个质量调整生命年(一个质量调整生命年等于健康状态下一年的寿命)。在肿瘤适应症方面(约 18,900 名患者),OS 和 PFS 可能分别减少了 9,400 多岁和 9,000 多岁。NICE评估终止的非肿瘤适应症治疗对QoL的潜在影响为13%(加权平均值)的增量改善。结论由于越来越多的多适应症产品被NICE终止评估,患者无法获得可延长其生命并提高其QoL的治疗方法。由于英国的统一定价政策很可能会影响制造商驱动的终止,因此引入替代性报销安排,如基于适应症的定价协议(IBP),以确保价格与治疗价值相匹配,可以改善英国治疗的可及性,从而改善公众健康。
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medRxiv - Health Economics
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