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Adoption of contact tracing app during pandemic: Users’ resistance behavior 在大流行病期间采用联系人追踪应用程序:用户的抵制行为
IF 6 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-15 DOI: 10.1016/j.hlpt.2024.100901
Yogesh Bhatt, Karminder Ghuman, Safiya Mukhtar Alshibani, Usama Awan
The study investigates the key issues influencing different barriers resulting in user resistance toward adopting contact tracing smartphone apps launched to track COVID-19 infections. Indian users’ experiences regarding the Aarogya Setu app for preventing the spread of COVID-19 were examined in two phases. In Phase I, online users’ comments available at the Google Play Store were qualitatively analyzed using open and axial coding. These codes were then used to create an implication matrix and hierarchical value maps to illustrate and interpret the relationships between issues, barriers, and user behavior. In Phase II, a supplementary empirical study, data was collected from users and non-users of the app through semi-structured telephone interviews and then qualitatively analyzed. By drawing on innovation resistance theory, the current study mapped a set of adoption barriers with three types of user resistance, i.e., postponement, opposition, and rejection. Rejection emerged as the most prominent consumer resistance behavior; usage barriers, functional risk, and value barriers related to the app's usage were the key drivers of this behavior. Postponement was the second most observed consumer resistance behavior. If usage barriers, functional risk, and value barriers of the app resulted in functional barriers toward adoption of the app, then image barrier was the key reason behind the psychological barrier. Administrators and developers of future interventions need to be conscious of usage barriers, functional risks, and value barriers related to the app's usage through stakeholder engagement to secure broader and faster adoption of such apps to improve health information systems.
本研究调查了影响用户采用为追踪 COVID-19 感染而推出的接触追踪智能手机应用程序的不同障碍的关键问题。研究分两个阶段考察了印度用户对用于预防 COVID-19 传播的 Aarogya Setu 应用程序的体验。在第一阶段,我们使用开放式和轴向编码对 Google Play 商店中的在线用户评论进行了定性分析。然后,利用这些编码创建了影响矩阵和分层价值图,以说明和解释问题、障碍和用户行为之间的关系。第二阶段是补充性实证研究,通过半结构化电话访谈从应用程序的用户和非用户那里收集数据,然后进行定性分析。通过借鉴创新阻力理论,本研究将一系列采用障碍与三种类型的用户阻力(即推迟、反对和拒绝)进行了映射。拒绝是最突出的消费者抵制行为;与应用程序使用相关的使用障碍、功能风险和价值障碍是这一行为的主要驱动因素。推迟是第二大消费者抵制行为。如果说应用程序的使用障碍、功能风险和价值障碍导致了采用应用程序的功能障碍,那么形象障碍则是心理障碍背后的关键原因。未来干预措施的管理者和开发者需要通过利益相关者的参与,意识到与应用程序使用相关的使用障碍、功能风险和价值障碍,以确保更广泛、更快速地采用此类应用程序来改善医疗信息系统。
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引用次数: 0
A decade of HTA in Indonesia: Methodological challenges in conducting economic evaluation 印度尼西亚 HTA 十年:开展经济评估的方法挑战
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-15 DOI: 10.1016/j.hlpt.2024.100894
Septiara Putri , Lusiana Siti Masytoh
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引用次数: 0
The beauty premium: Physicians’ facial expressions and patients’ selection and evaluation behavior in online health communities 美丽溢价:在线健康社区中医生的面部表情与患者的选择和评价行为
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-13 DOI: 10.1016/j.hlpt.2024.100895
Yanbin Yang, Chengyu Ma, Haopeng Liu

Background

With the development of information technology, online health communities (OHCs) are becoming an increasingly popular source of health information.

Objectives

While the impact of beauty has been extensively studied in various research fields, its role in OHCs has received little attention. This study aims to evaluate the effect of physicians’ appearance, smile, and positive emotions on patients’ selection and evaluation behavior in OHCs. Additionally, it explores the difference in the beauty premium among different types of physicians.

Methods

Over 13,000 images of physicians and their relevant information were collected from the Good Doctor website, which is now China's leading OHC. We identified facial features in physicians’ photos based on deep learning and used Ordinary Least Squares (OLS) regression models to estimate the relationship between physicians’ facial expressions and patients’ behavior. We used PSM to address endogeneity issues and test the robustness of the results.

Results

This study found that physicians’ appearance and smile positively impact patients’ selection and evaluation behavior, the results indicated that the beauty premium does exist in OHCs. In addition, heterogeneity analysis showed that the beauty of high titles, longer service duration of physicians has a greater influence on patients’ selection and evaluation behavior.

Conclusions

The beauty premium exists in OHCs. Therefore, this study provides new evidence on the impact of physicians’ facial attractiveness in OHCs and provides useful insights for patients, physicians, and platforms about the relationship between physician's structured or unstructured information and patients’ decision-making behaviors.

背景随着信息技术的发展,在线健康社区(OHCs)正成为越来越受欢迎的健康信息来源。研究目的虽然美貌的影响已在多个研究领域得到广泛研究,但其在 OHCs 中的作用却鲜有人关注。本研究旨在评估医生的外表、微笑和积极情绪对患者在 OHC 中的选择和评价行为的影响。方法:我们从中国领先的在线健康门户网站--好大夫网站上收集了超过 13,000 张医生照片及其相关信息。我们基于深度学习识别了医生照片中的面部特征,并使用普通最小二乘法(OLS)回归模型估计了医生面部表情与患者行为之间的关系。结果本研究发现,医生的外表和微笑对患者的选择和评价行为有积极影响,结果表明在 OHC 中确实存在美丽溢价。此外,异质性分析表明,职称越高、服务时间越长的美女医师对患者的选择和评价行为影响越大。因此,本研究为OHC中医生面部吸引力的影响提供了新的证据,并为患者、医生和平台了解医生的结构化或非结构化信息与患者决策行为之间的关系提供了有益的启示。
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引用次数: 0
Guide for the efficient and safe delivery of Telepharmacy 高效安全的远程药学服务指南
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-10 DOI: 10.1016/j.hlpt.2024.100899
R. Collado-Borell , M. Gomis-Pastor , MA. Rodríguez-Cabezas , A. Parro-Martín , A. Linares-Alarcón , E. Gutiérrez , E. Colominas-González , A. Fernández-Polo , M. Domínguez-Cantero , A. Arrondo-Velasco , R. Morillo-Verdugo

Objectives

This study aims to develop a guide establishing the requirements for developing and implementing telepharmacy in Hospital Pharmacy Services (HPS). A secondary objective is providing a reference frame to promote and allocate the necessary resources.

Methods

The project was developed in 5 phases from May-October 2021: (1) constitution of a working team; (2) literature review; (3) semi-structured interviews using the nominal group technique (with direct communications between experts and face-to-face discussion); (4) development of online workshops to raise awareness and debate each of the aspects considered essential in the document; (5) preparation of the final document and validation by the working group.

Results

As a result of this methodology, the Guide for the Efficient and Safe Provision of Telepharmacy was obtained. This Guide proposes that pharmacists follow a Strategic Plan to develop a new telepharmacy program in an HPS under quality standards. This Strategic Plan has been structured in 4 organizational phases in which the requirements or resources necessary for its implementation are established: Strategy Phase, Planning Phase, Action Phase, and Phase of monitoring results and continuous improvement.

Conclusions

This study provides a practical guide for hospital pharmacists and managers to develop and implement telepharmacy in an HPS with quality guarantees. The Guide provides the pharmacists collective with a reference framework to promote and allocate the necessary resources in an increasingly complex healthcare environment where telepharmacy is an essential strategy for sustainability.

目标本研究旨在制定一份指南,确定在医院药房服务(HPS)中发展和实施远程药学的要求。方法从 2021 年 5 月至 10 月,该项目分 5 个阶段进行:(1) 成立工作小组;(2) 文献综述;(3) 使用名义小组技术进行半结构化访谈(专家之间直接交流和面对面讨论);(4) 开展在线研讨会,以提高认识并就文件中认为至关重要的各个方面进行讨论;(5) 编写最终文件并由工作小组进行验证。结果 通过这一方法,获得了《高效安全提供远程药学服务指南》。该指南建议药剂师遵循战略计划,在符合质量标准的情况下,在医保局内制定新的远程药学计划。该战略计划分为 4 个组织阶段,其中确定了实施计划所需的要求或资源:结论 本研究为医院药剂师和管理人员提供了一份实用指南,帮助他们在有质量保证的情况下在医保局开发和实施远程药学。该指南为药剂师集体提供了一个参考框架,以便在日益复杂的医疗环境中促进和分配必要的资源,而远程药学是实现可持续发展的一项重要战略。
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引用次数: 0
'Where have my patients gone?': A simulation study on real-world data processing in Clinical Data Warehouses 我的病人去哪儿了?临床数据仓库中真实世界数据处理的模拟研究
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-02 DOI: 10.1016/j.hlpt.2024.100893
Sonia Priou , Emmanuelle Kempf , Rémi Flicoteaux , Marija Jankovic , Gilles Chatellier , Christophe Tournigand , Christel Daniel , Guillaume Lamé

Objective

To access Electronic Health Record (EHR) data, hospitals have implemented Clinical Data Warehouses (CDWs) using Extract Transform and Load (ETL) processes. While ETL performances are typically evaluated individually, our study examines the cumulative impact of ETLs on data availability.

Methods

Using a real multi-hospital CDW as a case study, we modeled EHR data processing from the software sources to the CDW's data store. We simulated a scenario where researchers aimed to reconstruct breast cancer care trajectories using EHR data. We calculated the size and characteristics of the data store population, and compared them to the original population.

Results

EHR data are recorded in various software depending on data category, hospital, and year, each requiring specific series of ETLs for integration in the CDW. Despite acceptable transfer rates for each ETL (range 73 %-100 %), cumulative losses led to study populations in the data store being up to 90 % smaller than anticipated when researchers required data exhaustivity for patients. Population size decreased steeply with the more data categories required. No difference was found in population characteristics between the data store and the original cohorts.

Discussion & Conclusion

Researchers should scrutinize data availability in CDWs as missing data could result from outsourced care, incomplete input, or underperforming ETLs. Integrating more data sources in CDWs increases the number of data routes, necessitating time for ETL implementation and maintenance, and increases data loss risks. Though commonly perceived as a “black box”, data transformation can significantly influence the reliability of populations studied in CDWs.

Public interest Summary

To access data generated during care, researchers build Clinical Data Warehouses (CDWs). CDWs are infrastructures composed of a series of processing steps to extract the data from the data source, transform it according to the needs and load it into a data store. Usually, the performances of these processing steps are evaluated one a time. However, each data point goes through a series of processing steps before being made available for research. In this study, we aim to evaluate the impact of the entire data processing pipeline on the availability of data points in a CDW by simulating a study on breast cancer and evaluating the impact on the size and the characteristics of the final cohort. The cumulative losses of the processing steps resulted in a population 90 % smaller than anticipated. The characteristics of the final population showed no difference to those of the original cohort.

为了访问电子健康记录(EHR)数据,医院使用提取、转换和加载(ETL)流程实施了临床数据仓库(CDW)。虽然 ETL 性能通常是单独评估的,但我们的研究考察了 ETL 对数据可用性的累积影响。以一个真实的多医院 CDW 为案例,我们模拟了从软件源到 CDW 数据存储的 EHR 数据处理过程。我们模拟了研究人员利用电子病历数据重建乳腺癌护理轨迹的情景。我们计算了数据存储群体的规模和特征,并与原始群体进行了比较。根据数据类别、医院和年份的不同,电子病历数据记录在不同的软件中,每种数据都需要特定系列的 ETL 才能集成到 CDW 中。尽管每个 ETL 的传输率都在可接受的范围内(73%-100%),但当研究人员要求患者数据穷尽时,累积损失导致数据存储中的研究群体比预期的要少多达 90%。随着所需的数据类别越多,群体规模急剧下降。数据存储与原始队列之间的人群特征没有差异。研究人员应仔细检查 CDW 中的数据可用性,因为数据缺失可能是由于外包护理、输入不完整或 ETL 性能不佳造成的。在 CDW 中集成更多数据源会增加数据路径的数量,从而需要时间来实施和维护 ETL,并增加数据丢失的风险。虽然数据转换通常被视为 "黑盒子",但它能极大地影响 CDW 中研究人群的可靠性。为了访问护理过程中生成的数据,研究人员建立了临床数据仓库(CDW)。临床数据仓库是由一系列处理步骤组成的基础设施,这些步骤包括从数据源提取数据、根据需要转换数据以及将数据加载到数据存储区。通常,这些处理步骤的性能一次评估一次。然而,每个数据点都要经过一系列处理步骤,然后才能用于研究。在本研究中,我们旨在通过模拟一项乳腺癌研究,评估整个数据处理流程对 CDW 中数据点可用性的影响,以及对最终队列规模和特征的影响。处理步骤的累积损失导致群体规模比预期小 90%。最终人群的特征与原始人群没有区别。
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引用次数: 0
Senior employment technology: Leveraging opportunities in population ageing 老年就业技术:利用人口老龄化带来的机遇
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-08-01 DOI: 10.1016/j.hlpt.2024.100883

Rapid population ageing, compounded by declines in the younger workforce that could offer timely caregiving assistance, is undermining society's ability to effectively and sufficiently protect people's health and quality of life as they grow old. This paper explores the promise of senior employment technology, a group of technological innovations that have the potential to alleviate or even eliminate the barriers faced by older individuals in the workforce. By doing so, these technologies empower older individuals to proactively safeguard their current and future living standards. This discussion delves into the potential of senior employment technologies in facilitating the continued participation of older individuals in the workforce and highlights the existing barriers preventing the widespread adoption of these technologies.

人口的迅速老龄化,再加上能够及时提供护理帮助的年轻劳动力的减少,正在削弱社会在人们年老时有效和充分保护其健康和生活质量的能力。本文探讨了老年就业技术的前景,这是一组有可能减轻甚至消除老年人在工作中面临的障碍的技术创新。通过这样做,这些技术能使老年人积极主动地保障他们当前和未来的生活水平。本讨论深入探讨了老年就业技术在促进老年人继续参与劳动力队伍方面的潜力,并强调了阻碍广泛采用这些技术的现有障碍。
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引用次数: 0
Defining change: Exploring expert views about the regulatory challenges in adaptive artificial intelligence for healthcare 定义变革:探索专家对医疗保健领域适应性人工智能监管挑战的看法
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-07-15 DOI: 10.1016/j.hlpt.2024.100892
Yves Saint James Aquino , Wendy A. Rogers , Susannah Louise Sage Jacobson , Bernadette Richards , Nehmat Houssami , Maame Esi Woode , Helen Frazer , Stacy M. Carter

Objective

Continuously learning or adaptive artificial intelligence (AI) applications for screening, diagnostic and other clinical services are yet to be widely deployed. This is partly due to existing device regulation mechanisms that are not fit for purpose regarding the adaptive features of AI. This study aims to identify the challenges in and opportunities for the regulation of adaptive features of AI.

Materials and Methods

We performed in-depth qualitative, semi-structured interviews with a diverse group of 72 experts in high-income countries (Australia, Canada, New Zealand, US, and UK) who are involved in the development, acquisition, deployment and regulation of healthcare AI systems.

Results

Our findings revealed perceived challenges in the regulation of adaptive features of machine learning (ML) systems. These challenges include the complexity of AI applications as products subject to regulation; lack of accepted definitions of adaptive changes; diverse approaches to defining significant adaptive change; and lack of clarity about regulation of adaptive change. Our findings reflect potentially competing interests among different stakeholders and diversity of approaches from regulatory bodies and legislators in different jurisdictions across the globe. In addition, our findings highlight the complex regulatory implications of adaptive AI that differ from traditional medical products, drugs or devices.

Conclusion

The perceived regulatory challenges raised by adaptive features of AI applications require high-level coordination within a complex regulatory ecosystem that consists of medical device regulators, professional accreditation agencies, professional medical organisations, and healthcare service providers. Regulatory approaches should complement existing safety protocols with new governance mechanisms that specifically take into account the variety of roles and responsibilities that will be required to monitor, evaluate and oversee adaptive changes.

目标用于筛查、诊断和其他临床服务的持续学习或自适应人工智能(AI)应用尚未得到广泛应用。部分原因是现有的设备监管机制不适合人工智能的自适应功能。本研究旨在确定人工智能自适应功能监管所面临的挑战和机遇。材料与方法我们对高收入国家(澳大利亚、加拿大、新西兰、美国和英国)参与医疗人工智能系统开发、获取、部署和监管的 72 位专家进行了深入的半结构式定性访谈。结果我们的研究结果显示了机器学习(ML)系统自适应功能监管所面临的挑战。这些挑战包括:人工智能应用作为受监管产品的复杂性;缺乏公认的适应性变化定义;定义重大适应性变化的方法多种多样;对适应性变化的监管缺乏明确性。我们的研究结果反映了不同利益相关者之间潜在的利益冲突,以及全球不同司法管辖区监管机构和立法者的方法多样性。此外,我们的研究结果还强调了适应性人工智能与传统医疗产品、药品或设备不同的复杂监管影响。结论人工智能应用的适应性特征所带来的监管挑战需要在复杂的监管生态系统中进行高层协调,该生态系统由医疗设备监管机构、专业认证机构、专业医疗组织和医疗服务提供商组成。监管方法应通过新的治理机制来补充现有的安全协议,这些机制应特别考虑到监测、评估和监督适应性变化所需的各种角色和责任。
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引用次数: 0
Recommended exercises for lower back pain on YouTube videos; Are they useful or harmful? "YouTube视频中推荐的治疗下背部疼痛的运动;它们有用还是有害?
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-07-14 DOI: 10.1016/j.hlpt.2024.100891
Rabia Aydogan Baykara , Nevsun Pıhtılı Taş

Objective

Low back pain (LBP), a common public health problem affecting nearly 80 % of the population, causes disability and work absence, while exercise is recommended as an adjuvant treatment. YouTube is one of the most frequently used source of information, especially by patients. The aim of the study was to analyze clinically the reliability, feasibility and the content quality of exercises videos recommended for LBP on YouTube. channel.

Methods

Based on the observational study of the number of views of YouTube videos, 154 videos worth evaluation were selected using the keywords "low back pain” and “exercises",. The content quality of each video was evaluated with a) the American Medical Association Journal (JAMA), b) DISCERN, c) Global Quality Score (GQS), and d) Medical Quality Video Assessment Tool (MQ-VET) scales, while Pearson correlation analysis was performed to determine the correlation between the JAMA, GQS, DISCERN, MQ-VET scale scores.

Results

According to the findings, the primary source for uploading exercise videos were physicians (32.5 %), with a high content quality. There was a statistically significant positive correlation between the total scores of all quality scales and the independent variables defined as view rates and number of comments (p < .05). The analysis of the video content showed that only 35 % (n = 54) of the videos explained the risks of the recommended exercises in detail

Conclusion

It can be concluded that the relability and feasibility of exercise videos for LBP on YouTube depend highly on the upload source. Thus digital content in health should be created with a multi-disciplinary team including healthcare professionals and also standardized to inform patients with the risks and benefits to avoid the potential harm due to the complex causes of low back pain.

Public Interest Summary

Low Back Pain, which a major health problem, is the most common musculoskeletal disorder leading to increased medical expenses and work absenteeism, while the most appropriate intervention remains elusive.. The therapeutic exercise program might differ according to patient's age, physical condition and the severity of the disease, while sustainability of the exercise program depends on the preference of exercise type of each patient. From this perspective, it is an important advantage that different exercise videos are available on YouTube. However, the content quality and reliability of exercise videos are the discriminators for the expected outcomes, since good quality content will benefit patients and prevent possible injuries and worsening of pain.

目的腰背痛(LBP)是一种常见的公共健康问题,影响着近 80% 的人口,导致残疾和缺勤,而运动被推荐为一种辅助治疗手段。YouTube 是最常用的信息来源之一,尤其是患者。本研究旨在对 YouTube 频道上推荐的腰背痛锻炼视频的可靠性、可行性和内容质量进行临床分析。 方法基于对 YouTube 视频浏览量的观察研究,使用关键词 "腰背痛 "和 "锻炼",选择了 154 个值得评估的视频。用 a) 美国医学会杂志 (JAMA)、b) DISCERN、c) 全球质量得分 (GQS) 和 d) 医疗质量视频评估工具 (MQ-VET) 量表对每个视频的内容质量进行评估,同时进行皮尔逊相关分析,以确定 JAMA、GQS、DISCERN、MQ-VET 量表得分之间的相关性。结果根据研究结果,上传运动视频的主要来源是医生(32.5%),内容质量较高。所有质量量表的总分与自变量(观看率和评论数量)之间存在统计学意义上的正相关(p <.05)。对视频内容的分析表明,只有 35% 的视频(n = 54)详细解释了推荐运动的风险。因此,健康领域的数字内容应由包括医护人员在内的多学科团队共同创建,同时还应标准化,以告知患者相关风险和益处,避免因腰背痛的复杂病因而造成潜在伤害。公益摘要腰背痛是一个主要的健康问题,是最常见的肌肉骨骼疾病,导致医疗费用和缺勤率增加,而最合适的干预措施却仍然难以确定。治疗性锻炼计划可能因患者的年龄、身体状况和疾病的严重程度而异,而锻炼计划的可持续性则取决于每位患者对锻炼类型的偏好。从这个角度来看,YouTube 上提供不同的运动视频是一个重要的优势。然而,运动视频的内容质量和可靠性是预期结果的判别因素,因为高质量的内容将使患者受益,并防止可能出现的损伤和疼痛恶化。
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引用次数: 0
The status of the AI medical industry in China: A database and statistical analysis 中国人工智能医疗产业的现状:数据库和统计分析
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-07-14 DOI: 10.1016/j.hlpt.2024.100889
Siwen Zhang , Zhe Huang , Guihong Feng , Xiaowen Yuan , Qi Zhang , Zicheng Wang , Yuwen Chen

Background

Artificial intelligence (AI) technology has developed rapidly in recent years, leading to exponential growth in the AI medical industry. However, a comprehensive investigation of approved medical devices in China is needed.

Methods

We utilized a web crawler to collect data on all medical devices from the China National Medical Products Administration website since 2018. Through natural language processing techniques and manual analysis, we identified all medical devices developed by artificial intelligence medical devices (AIMD) companies and conducted a statistical analysis.

Results

Since 2018, the number of AI-related medical devices approved in China has significantly increased. Most devices (79 %) were classified as Class II with moderate risk, whereas 21 % were classified as Class III with high risk. Most devices (74.2 %) were categorized as medical device software, and the most common application was medical image processing (63.2 %). In terms of target body areas, devices related to the heart accounted for the highest proportion (12.8 %), followed by those related to the lungs (11.3 %) and brain (6.7 %).

Conclusion

This study establishes a comprehensive database of medical devices developed by AIMD companies in China, enabling the public to gain a coherent understanding of their current development status.

背景人工智能(AI)技术近年来发展迅速,导致人工智能医疗行业呈指数级增长。然而,需要对中国已获批准的医疗器械进行全面调查。方法我们利用网络爬虫从中国国家医疗器械监督管理局网站收集了2018年以来所有医疗器械的数据。通过自然语言处理技术和人工分析,我们确定了人工智能医疗器械(AIMD)公司开发的所有医疗器械,并进行了统计分析。结果自 2018 年以来,中国批准的人工智能相关医疗器械数量显著增加。大多数器械(79%)被归类为中度风险的二级器械,21%被归类为高度风险的三级器械。大多数器械(74.2%)被归类为医疗器械软件,最常见的应用是医学图像处理(63.2%)。就目标身体部位而言,与心脏有关的器械所占比例最高(12.8%),其次是与肺有关的器械(11.3%)和与脑有关的器械(6.7%)。
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引用次数: 0
Health technology assessments of new specialized therapies: A typology of key challenges and potential policy solutions 新专科疗法的卫生技术评估:关键挑战和潜在政策解决方案类型学
IF 3.4 3区 医学 Q1 HEALTH POLICY & SERVICES Pub Date : 2024-07-14 DOI: 10.1016/j.hlpt.2024.100890
Sarah Wadmann , Malene S. Sørensen , Betina Højgaard

Objectives

Current transformations in the pharmaceutical sector raise pressing questions about what is considered acceptable evidence for the effects of new therapies. This article aims to identifying key challenges in clinical effectiveness evaluation of new therapies and discuss possible policy responses to these challenges.

Methods

The study builds on a systematic review of the 41 appraisals issued in 2019 by the Danish Medicines Council (DMC), which is responsible for the appraisal of new specialized therapies in Denmark.

Results

While much political attention currently centers on the use of ‘real-world evidence’, we find that clinical effectiveness evaluation based solely on non-RCT evidence still constitutes an exception in the Danish setting (9% of the evaluations). Yet, challenges of indirectness were prevalent even when evaluations were based on RCT data (54% of the evaluations). Challenges of effect extrapolation arose in about a third of the evaluations.

Discussion

As the identified challenges are likely to increase with the current trend from ‘blockbuster’ to ‘niche’ products in the pharmaceutical sector, we point to a need for regulators and health technology assessment agencies to collaborate about the development of guidance on the use of other study designs than traditional RCTs, methods that can reduce the risk of bias when conducting indirect comparisons, and principles for managed entry agreements.

目的当前制药行业的转型提出了一个紧迫的问题,即什么是新疗法效果的可接受证据。本文旨在确定新疗法临床疗效评估中的关键挑战,并讨论应对这些挑战的可能政策。方法本研究基于对丹麦药品委员会(DMC)2019年发布的41项评估报告的系统性回顾,该委员会负责丹麦新的专业疗法的评估。结果尽管目前许多政治关注的焦点是 "真实世界证据 "的使用,但我们发现,在丹麦环境中,仅基于非RCT证据的临床疗效评估仍然是一个例外(占评估的9%)。然而,即使是基于 RCT 数据的评估(占评估的 54%),也普遍存在间接性问题。讨论 由于目前制药行业从 "大片 "到 "小众 "产品的趋势,已确定的挑战可能会增加,我们指出监管机构和卫生技术评估机构有必要合作,就使用传统 RCT 以外的其他研究设计、进行间接比较时可降低偏倚风险的方法以及管理下进入协议的原则制定指南。
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Health Policy and Technology
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