Peiris Vasum, Xu Kui, Agler Heather L, Chen Eric A, Gopal-Srivastava Rashmi, Lappin Brian M, Lewis Debra Y, Rao Gayatri R
Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 66, Suite 5422, Silver Spring, MD 20993.
Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 32, Suite 5295, Silver Spring, MD 20993.
J Med Device. 2018 Sep;12(3):0347011-347018. doi: 10.1115/1.4040489. Epub 2018 Jul 13.
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.
罕见病影响着约3000万美国人，其中一半是儿童。本研究首次通过对医生的调查全面评估了他们对医疗设备的需求。该研究旨在识别并记录在罕见病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规模；并生成有意义的数据以为医疗设备利益相关者提供信息。开展了一项横断面非概率调查。研究人群来自四个组织的成员档案：美国食品药品监督管理局医疗设备咨询委员会、儿科咨询委员会、儿科设备联盟以及美国国立卫生研究院（NIH）罕见病临床研究网络。只有对罕见病有经验或了解的医生受访者才有资格参与。在符合条件的受访者中，90%确认需要创新设备来照顾罕见病患者。针对436种罕见病确定了850多项设备需求，其中74%的需求与儿童相关。儿科医生（比值比=2.11，95%置信区间1.01 - 4.39，P = 0.046）以及有更多罕见病经验的医生对现有设备的不满程度更高（比值比=4.49，95%置信区间2.25 - 8.96，P < 0.0001）。创建全新设备是缓解需求的首要建议。本研究表明，对于照顾患有罕见病的儿童和成人的创新医疗设备存在重大公共卫生需求。美国食品药品监督管理局和美国国立卫生研究院支持并寻求机会加速为这些弱势患者开发设备。
