0
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,
10903 New Hampshire Avenue,
WO Building 66, Suite 5422,
Silver Spring, MD 20993

Kui Xu, Debra Y. Lewis, 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

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

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

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

Brian M. Lappin

Office of Planning,
Food and Drug Administration,
10903 New Hampshire Avenue,
WO Building 32, Suite 3352,
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 12(3), 034701 (Jul 13, 2018) (8 pages) Paper No: MED-18-1024; doi: 10.1115/1.4040489 History: Received February 06, 2018; Revised May 23, 2018

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.

FIGURES IN THIS ARTICLE
<>
Copyright © 2018 by ASME
Your Session has timed out. Please sign back in to continue.

References

Global Genes, 2015, “ RARE Diseases: Facts and Statistics,” Global Genes, Aliso Viejo, CA, accessed May 22, 2018, http://globalgenes.org/rare-diseases-facts-statistics/
Genetic and Rare Diseases Information Center, 2018, “ Frequently Asked Questions,” Genetic and Rare Diseases Information Center, Gaithersburg, MD, accessed May 22, 2018, https://rarediseases.info.nih.gov/about-gard/pages/31/frequently-asked-questions
Rare Disease Impact Report, 2018, “Insights From Patients and the Medical Community,” Global Genes, Aliso Viejo, CA, accessed May 22, 2018, https://globalgenes.org/wp-content/uploads/2013/04/ShireReport-1.pdf
Institute of Medicine (U.S.), 2010, “Committee on Accelerating Rare Diseases Research and Orphan Product Development,” M. J. Field and T. F. Boat, eds., National Academies Press, Washington, DC, accessed May 22, 2018, http://www.nationalacademies.org/hmd/~/media/Files/Report%20Files/2010/Rare-Diseases-and-Orphan-Products-Accelerating-Research-and-Development/Rare%20Disease%20Research%202010%20Report%20Brief.pdf
Department of Health and Human Services, Food and Drug Administration, 2018, “Report to Congress: Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases,” FDA, White Oak, MD, accessed May 22, 2018, https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm283745.htm
U.S. Food and Drug Administration, 2018, “Medical Device Advisory Committee,” Food and Drug Administration (FDA), Silver Spring, MD, accessed May 22, 2018, https://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/MedicalDevices/MedicalDevicesAdvisoryCommittee/default.htm
U.S. Food and Drug Administration, 2018, “Pediatric Advisory Committee,” Food and Drug Administration (FDA), Silver Spring, MD, accessed May 22, 2018, https://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/default.htm
U.S. Food and Drug Administration, 2018, “Pediatric Device Consortia Grants Program,” Food and Drug Administration (FDA), Silver Spring, MD, accessed May 22, 2018, https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/PediatricDeviceConsortiaGrantsProgram/
Rare Diseases Clinical Research Network, 2018, The National Center for Advancing Translational Sciences, Bethesda, MD, accessed May 22, 2018, https://www.rarediseasesnetwork.org/
Braithwaite, D. , Emery, J. , de Lusignan, S. , and Sutton, S. , 2003, “ Using the Internet to Conduct Surveys of Health Professionals: A Valid Alternative?,” Fam. Pract., 20(5), pp. 545–551. [CrossRef] [PubMed]
Cunningham, C. T. , Quan, H. , Hemmelgarn, B. , Noseworthy, T. , Beck, C. A. , Dixon, E. , Samuel, S. , Ghali, W. A. , Sykes, L. L. , and Jette, N. , 2015, “ Exploring Physician Specialist Response Rates to Web-Based Surveys,” BMC Med. Res. Methodol., 15(1), p. 32. [CrossRef] [PubMed]
Buntz, B. , 2016, “ 5 Reasons Why Medical Device Innovation Is so Tough, Medical Product Manufacturing News Medtech Pulse,” The Medical Device and Diagnostic Industry newsletter, Santa Monica, CA, accessed May 22, 2018, http://www.qmed.com/mpmn/medtechpulse/5-reasons-why-medical-device-innovation-so-tough
Bergsland, J. , Elle, O. J. , and Fosse, E. , 2014, “ Barriers to Medical Device Innovation,” Med. Devices, 7, pp. 205–209. [CrossRef]
Schieppati, A. , Henter, J. I. , Daina, E. , and Aperia, A. , 2008, “ Why Rare Diseases Are an Important Medical and Social Issue,” Lancet, 371(9629), pp. 2039–2041. [CrossRef] [PubMed]

Figures

Grahic Jump Location
Fig. 1

Survey organization and primary skip pattern

Grahic Jump Location
Fig. 2

Mean factor scores for impediments and exploratory factor analysis*. *Based on 385 respondents; a denominator less than 385 indicates item missing data; CI denotes confidence interval; P values were calculated using t-test comparing pediatric versus nonpediatric and medicine and nonmedicine; Score is calculated as 0 = not an impediment/no answer, 1 = small extent, 2 = moderate extent, and 3 = large extent. †Significant difference at P < 0.05.

Tables

Errata

Some tools below are only available to our subscribers or users with an online account.

Related Content

Customize your page view by dragging and repositioning the boxes below.

Related Journal Articles
Related eBook Content
Topic Collections

Sorry! You do not have access to this content. For assistance or to subscribe, please contact us:

  • TELEPHONE: 1-800-843-2763 (Toll-free in the USA)
  • EMAIL: asmedigitalcollection@asme.org
Sign In