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.
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September 2018
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Children and Adults With Rare Diseases Need Innovative Medical Devices
Vasum Peiris,
Vasum Peiris
Center for Devices and Radiological Health,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5422,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5422,
Silver Spring, MD 20993
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Kui Xu,
Kui Xu
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
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Heather L. Agler,
Heather L. Agler
Center for Devices and Radiological Health,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5570,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5570,
Silver Spring, MD 20993
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Eric A. Chen,
Eric A. Chen
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
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
e-mail: eric.chen@fda.hhs.gov
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Rashmi Gopal-Srivastava,
Rashmi Gopal-Srivastava
National Center for Advancing
Translational Sciences,
Office of Rare Diseases
Research at National
Institutes of Health,
6701 Democracy Blvd,
Bethesda, MD 20817
Translational Sciences,
Office of Rare Diseases
Research at National
Institutes of Health,
6701 Democracy Blvd,
Bethesda, MD 20817
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Brian M. Lappin,
Brian M. Lappin
Office of Planning,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 3352,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 3352,
Silver Spring, MD 20993
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Debra Y. Lewis,
Debra Y. Lewis
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
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Gayatri R. Rao
Gayatri R. Rao
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Search for other works by this author on:
Vasum Peiris
Center for Devices and Radiological Health,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5422,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5422,
Silver Spring, MD 20993
Kui Xu
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Heather L. Agler
Center for Devices and Radiological Health,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5570,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 66, Suite 5570,
Silver Spring, MD 20993
Eric A. Chen
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
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
e-mail: eric.chen@fda.hhs.gov
Rashmi Gopal-Srivastava
National Center for Advancing
Translational Sciences,
Office of Rare Diseases
Research at National
Institutes of Health,
6701 Democracy Blvd,
Bethesda, MD 20817
Translational Sciences,
Office of Rare Diseases
Research at National
Institutes of Health,
6701 Democracy Blvd,
Bethesda, MD 20817
Brian M. Lappin
Office of Planning,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 3352,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 3352,
Silver Spring, MD 20993
Debra Y. Lewis
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Gayatri R. Rao
Office of Orphan Products Development,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 5295,
Silver Spring, MD 20993
1Corresponding author.
Manuscript received February 6, 2018; final manuscript received May 23, 2018; published online July 13, 2018. Assoc. Editor: Michael Eggen.
J. Med. Devices. Sep 2018, 12(3): 034701 (8 pages)
Published Online: July 13, 2018
Article history
Received:
February 6, 2018
Revised:
May 23, 2018
Citation
Peiris, V., Xu, K., Agler, H. L., Chen, E. A., Gopal-Srivastava, R., Lappin, B. M., Lewis, D. Y., and Rao, G. R. (July 13, 2018). "Children and Adults With Rare Diseases Need Innovative Medical Devices." ASME. J. Med. Devices. September 2018; 12(3): 034701. https://doi.org/10.1115/1.4040489
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