Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

The Humanitarian Device Exemption of the United States as a Pioneering Concept in Orphan Device Regulation and its Implications for the Policy of the Regulation of Medical Devices for Small Populations in the European Union ***

Ute Worringen (Abschlußjahr: 2015)

Summary
Language: English
This thesis aims to analyze aspects of the regulatory premarket pathways in the European Union (EU) and the United States regarding medical devices intended for use in rare diseases and conditions. The specificities of rare diseases are primarily the limited number of patients and scarcity of expertise . These medical products are suggested to be referred to as "orphan devices" in analogy to "orphan drugs". Due tot he recognition that such devices are less likely to be developed by industry because their market is small and research and development costs usually cannot be compensated through market revenues, their development seems to be little attractive. Therefore the outlines of the United States’ FDA pioneering concept of Humanitarian Use Device (HUD)/ Humanitarian Device Exemption(HDE) procedures was analysed.
These regulatory concepts were specifically designed to accelerate the market access of orphan devices under the condition of continuous additional approval of a local investigational review board. As there is no comparable regulatory pathway implemented in the EU, the question was raised, whether any elements of the FDA’s concept may be suitable as a model for a tailored legislation in the EU. The necessity to accelerate orphan device development in the EU has been postulated by the EU umbrella patient advocacy organization for rare diseases, EURORDIS.
As relevant structural differences between the two regulatory systems in the EU and U.S. have to be taken into account in judging about the transferability of regulatory processes, critical elements of both legislations were briefly described with emphasis on the premarket assessment of novel high-risk therapeutic medical devices.
Both medical device legislations share common elements like risk classification, premarket assessment and postmarket vigilance and the regulatory elements risk management, application of a quality systems, design dossier and labeling. Nevertheless, the administrative structures are contrasting regarding the centralization of authorities at the U.S.’s FDA versus the decentralized and supranational system in the EU. With regard to HUDs and orphan devices, the U.S.’s context may have accelerated the political process behind the regulatory concept intended for fostering therapeutic innovation for small populations. The U.S. concept seems to be well suited as a model for the HDE designation procedure for the EU. The implementation of an "orphan device designation" procedure into revised EU legislation is therefore proposed. Any effort concerning diseases and conditions that are rare and pediatric are probably more effective in international collaboration, which is therefore proposed regarding the further development of guidelines and standards for device development in special populations.
Pages: 71