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.
Skip Nav Destination
Article navigation
September 2017
Expert View
Food and Drug Administration's Humanitarian Device Exemption Marketing Approval Pathway: Insights for Developing Devices for Rare Diseases
Gumei Liu,
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
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
Search for other works by this author on:
Eric Chen,
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
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
Search for other works by this author on:
Debra Lewis,
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
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
Search for other works by this author on:
Gayatri Rao
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
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
Search for other works by this author on:
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
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
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
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
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. Sep 2017, 11(3): 034701 (8 pages)
Published Online: June 27, 2017
Article history
Received:
October 30, 2016
Revised:
February 26, 2017
Citation
Liu, G., Chen, E., Lewis, D., and Rao, G. (June 27, 2017). "Food and Drug Administration's Humanitarian Device Exemption Marketing Approval Pathway: Insights for Developing Devices for Rare Diseases." ASME. J. Med. Devices. September 2017; 11(3): 034701. https://doi.org/10.1115/1.4036333
Download citation file:
Get Email Alerts
Cited By
Related Articles
Children and Adults With Rare Diseases Need Innovative Medical Devices
J. Med. Devices (September,2018)
A High-Throughput In Vitro Model Illustrating Potential Microbiological Interactions During Treatment of Pseudomonas Aeruginosa Biofilm Associated Infections
J. Med. Devices (June,2008)
The Role of Computational Modeling and Simulation in the Total Product Life Cycle of Peripheral Vascular Devices
J. Med. Devices (June,2017)
Magnetic Chest Tube Positioning System
J. Med. Devices (June,2018)
Related Proceedings Papers
Related Chapters
Regulatory perspectives
Biocompatible Nanomaterials for Targeted and Controlled Delivery of Biomacromolecules
Process Components
Bioprocessing Piping and Equipment Design: A Companion Guide for the ASME BPE Standard
Applications of Macro-, Micro- and Nano-Biomaterials Prepared using Biopolymers
Biopolymers Based Micro- and Nano-Materials
