Humanitarian Use Device and Humanitarian Device Exemption regulatory programs: pros and cons

Abstract

The US FDA established the Humanitarian Use Device (HUD) and Humanitarian Device Exemption (HDE) program to encourage medical device firms to address rare diseases. Despite being in existence for over a decade, there has only been one peer-reviewed publication examining this field. The objective of this report is to investigate how the HUD/HDE program differs from the standard regulatory system, discuss its potential advantages and disadvantages, and to speculate which humanitarian devices will be brought to market within the next 5 years. A total of 40 semistructured interviews with stakeholders, representing approximately half (n = 20, 49%) of the firms that have successfully obtained HDE-approved products, were performed in order to acquire the primary data for this paper. There appear to be short-term gains and long-term drains associated with launching humanitarian devices to market. This report aims to provide sponsors with information that may allow them to make better decisions during their product development of humanitarian devices and may, hopefully, also play a role in encouraging other sponsors to take the necessary steps forward in helping to find treatments for patients with rare diseases.

Keywords::

• Humanitarian Device Exemption
• Humanitarian Use Device
• medical device
• Pediatric Medical Device Safety and Improvement Act of 2007
• product development
• regulatory system

Acknowledgements

This manuscript benefited from the insightful comments and constructive criticisms provided by the reviewers. The author is grateful for David Hampton’s patience and generosity. The author would also like to thank Geraldine Rodgers and Anna Kempinska for their helpful comments. Lastly, the author is indebted to Professor Chris Lowe for sharing his pearls of wisdom and to Linda Allan and Sabine Deering for their tireless support.

Financial & competing interests disclosure

Daniel Maxwell Bernad is thankful for the generous funding received from the Cambridge Overseas and Commonwealth Trusts, without which this opportunity would have never transpired. Some of the data used in this manuscript was collected while completing thesis work during the author’s Master’s in Bioscience Enterprise at the University of Cambridge. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.