Children and Adults With Rare Diseases Need Innovative Medical Devices

Vasum Peiris; Kui Xu; Heather L Agler; Eric A Chen; Rashmi Gopal-Srivastava; Brian M Lappin; Debra Y Lewis; Gayatri R Rao

doi:10.1115/1.4040489

Children and Adults With Rare Diseases Need Innovative Medical Devices

J Med Device. 2018 Sep;12(3):0347011-347018. doi: 10.1115/1.4040489. Epub 2018 Jul 13.

Authors

Vasum Peiris¹, Kui Xu², Heather L Agler³, Eric A Chen⁴, Rashmi Gopal-Srivastava⁵, Brian M Lappin⁶, Debra Y Lewis², Gayatri R Rao²

Affiliations

¹ Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 66, Suite 5422, Silver Spring, MD 20993.
² Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 32, Suite 5295, Silver Spring, MD 20993.
³ Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 66, Suite 5570, Silver Spring, MD 20993.
⁴ Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 32, Suite 5295, Silver Spring, MD 20993 e-mail: eric.chen@fda.hhs.gov.
⁵ National Center for Advancing Translational Sciences, Office of Rare Diseases Research at National Institutes of Health, 6701 Democracy Blvd, Bethesda, MD 20817.
⁶ Office of Planning, Food and Drug Administration, 10903 New Hampshire Avenue, WO Building 32, Suite 3352, Silver Spring, MD 20993.

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 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.