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Expert View

Food and Drug Administration's Humanitarian Device Exemption Marketing Approval Pathway: Insights for Developing Devices for Rare Diseases

[+] Author and Article Information
Gumei Liu

Office of Orphan Products Development,
Office of Special Medical Programs,
U.S. Food and Drug Administration,
10903 New Hampshire Avenue,
Silver Spring, MD 20993
e-mail: gumei.liu@fda.hhs.gov

Eric Chen

Office of Orphan Products Development,
Office of Special Medical Programs,
U.S. Food and Drug Administration,
10903 New Hampshire Avenue,
Silver Spring, MD 20993
e-mail: eric.chen@fda.hhs.gov

Debra Lewis

Office of Orphan Products Development,
Office of Special Medical Programs,
U.S. Food and Drug Administration,
10903 New Hampshire Avenue,
Silver Spring, MD 20993
e-mail: debra.lewis@fda.hhs.gov

Gayatri Rao

Office of Orphan Products Development,
Office of Special Medical Programs,
U.S. Food and Drug Administration,
10903 New Hampshire Avenue,
Silver Spring, MD 20993
e-mail: gayatri.rao@fda.hhs.gov

1Corresponding author.

2This number is based on the Orphan Drug Act prevalence definition of a rare disease—affecting fewer than 200,000 individuals in the U.S.

Manuscript received October 30, 2016; final manuscript received February 26, 2017; published online June 27, 2017. Assoc. Editor: Michael Eggen.This material is declared a work of the U.S. Government and is not subject to copyright protection in the United States. Approved for public release; distribution is unlimited.

J. Med. Devices 11(3), 034701 (Jun 27, 2017) (8 pages) Paper No: MED-16-1350; doi: 10.1115/1.4036333 History: Received October 30, 2016; Revised February 26, 2017

The Food and Drug Administration's (FDA) Humanitarian Device Exemption (HDE) is a unique marketing approval pathway for medical devices targeting diseases affecting small (rare) patient populations. In an effort to increase the utilization and success of this pathway, the FDA has analyzed data from HDE approvals from 2007 to 2015 to identify factors that have contributed to a successful HDE marketing application. There were 28 HDE approvals during the analysis period and were based on a broad range of data constituting valid scientific evidence. Most had at least one prospectively conducted clinical trial to support safety and probable benefit. An analysis of these HDE approvals demonstrates that the FDA exercises a high degree of flexibility when reviewing HDE applications.

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References

Griggs, R. C. , Batshaw, M. , Dunkle, M. , Gopal-Srivastava, R. , Kaye, E. , Krischer, J. , Nguyen, T. , Paulus, K. , and Merkel, P. A. , 2009, “ Clinical Research for Rare Disease: Opportunities, Challenges, and Solutions,” Mol. Genet. Metab., 96(1), pp. 20–26. [CrossRef] [PubMed]
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Figures

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Fig. 1

Distribution of population estimate of HDE approvals

Grahic Jump Location
Fig. 2

Distribution of the number of subjects enrolled in HDE clinical trials

Grahic Jump Location
Fig. 4

Total time taken from filing of HDE application to approval: a decreasing trend

Grahic Jump Location
Fig. 5

Total time taken from filing of HDE application to approval: treatment versus diagnostic devices

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