Children and Adults With Rare Diseases Need Innovative Medical Devices
Children and Adults With Rare Diseases Need Innovative Medical Devices
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DOI：
10.1115/1.4040489
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发表时间：
2018-09-01
影响因子：
0.9
通讯作者：
Lewis, Debra Y.
中科院分区：
文献类型：
--
作者：
Peiris, Vasum;Xu, Kui;Lewis, Debra Y.
罕见病（RD）影响了大约3000万美国人，其中一半是儿童。这项研究是第一次通过对医生的调查来全面评估他们的医疗器械需求。该研究旨在确定和记录RD管理中假定未满足的诊断和治疗设备需求；阐明潜在未满足需求的程度；并生成有意义的数据，告知医疗设备利益相关者。进行了横断面非概率调查。研究人群从四个小组的成员文件中抽取：FDA医疗器械咨询委员会、儿科咨询委员会、儿科器械协会和国家卫生研究院（NIH） Rare。疾病临床研究网络。只有具有RD相关经验或知识的受访医师才有资格。在符合条件的受访者中，90%的人确认需要创新的设备来照顾RD患者。436个RD确定了850多个设备需求，其中74%的需求与儿童有关。儿科医生（OR = 2.11, 95% CI 1.01-4.39, P = 0.046）和有更多RD经验的医生对现有器械的不满程度更高（OR = 4.49, 95% CI 2.25-8.96, P < 0.0001）。创建全新的设备是缓解需求的首选建议。这项研究表明，创新的医疗设备对儿童和成人RD患者的护理具有重大的公共卫生需求。FDA和NIH支持并寻求机会加速这些弱势患者的设备开发。
Rare diseases (RD) affect approximately 30 million Americans, half of whom are children. This study is the first to comprehensively evaluate their medical device needs via a survey of physicians. The study sought to identify and document the presumed unmet diagnostic and therapeutic device needs for RD management; clarify the magnitude of the potential unmet need; and generate meaningful data to inform medical device stakeholders. A cross-sectional nonprobability survey was conducted. The study population was drawn from the membership files of four groups: FDA Medical Devices Advisory Committee, Pediatric Advisory Committee, Pediatric Device Consortia, and National Institutes of Health (NIH) Rare. Diseases Clinical Research Network. Only physician respondents with experience or knowledge regarding RD were eligible. Among eligible respondents, 90% confirmed the need for innovative devices to care for people with RD. Over 850 device needs were identified for 436 RD, with 74% of needs related to children. Pediatric physicians (OR = 2.11, 95% CI 1.01-4.39, P = 0.046) and physicians with more RD experience reflected greater dissatisfaction with existing devices (OR = 4.49, 95% CI 2.25-8.96, P < 0.0001). Creation of entirely new devices is the top recommendation for mitigating needs. This study demonstrates a major public health need for innovative medical devices to care for children and adults with RD. FDA and NIH support and seek opportunities to accelerate device development for these vulnerable patients.