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Expert View

Children and Adults with Rare Diseases Need Innovative Medical Devices

[+] Author and Article Information
Vasum Peiris

Center for Devices and Radiological Health, Food and Drug Administration
vasum.peiris@fda.hhs.gov

Kui Xu

Office of Orphan Products Development, Office of Medical Products and Tobacco, Food and Drug Administration
kui.xu@fda.hhs.gov

Heather Agler

Center for Devices and Radiological Health, Food and Drug Administration
heather.agler@fda.hhs.gov

Eric Chen

10903 New Hampshire Avenue, WO Building 32, Suite 5202, Silver Spring, MD 20993
eric.chen@fda.hhs.gov

Rashmi Gopal-Srivastava

National Center for Advancing Translational Sciences, Office of Rare Diseases Research at National Institutes of Health
rashmi.gopal-srivastava@nih.gov

Brian Lappin

Office of Planning, Office of Commissioner, Food and Drug Administration
brian.lappin@fda.hhs.gov

Debra Lewis

Office of Orphan Products Development, Office of Medical Products and Tobacco, Food and Drug Administration
debra.lewis@fda.hhs.gov

Gayatri Rao

Office of Orphan Products Development, Office of Medical Products and Tobacco, Food and Drug Administration
grao77@gmail.com

1Corresponding author.

ASME doi:10.1115/1.4040489 History: Received February 06, 2018; Revised May 23, 2018

Abstract

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 non-probability 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 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=.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.

Copyright (c) 2018 by ASME
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