Navigating Global Regulatory Pathways for Orphan Medical Devices

This session provides in-depth regulatory and strategic insights into the ecosystem, development and approval of orphan medical devices, with an emphasis on the United States, European Union, and China. Designed for regulatory professionals, the session will compare the legislative frameworks and regulatory requirements across these jurisdictions, highlighting critical divergences in policy, evidentiary expectations, and market access pathways.

Expert speakers - including regulatory consultants, representatives from a Notified Body and the medical device industry - will examine the unique challenges of generating clinical evidence for devices intended for small patient populations. Topics will include optimizing limited clinical data, alternative trial designs, and integrating real-world evidence to meet regulatory expectations.

The session will feature a detailed review of specific programs such as the FDA’s Humanitarian Device Exemption (HDE), with discussion of associated incentives and support mechanisms. These will be contrasted with the more constrained approaches in the EU and China. Attendees will benefit from practical case studies and interactive dialogue addressing regulatory engagement strategies, the use of expanded access or compassionate use programs, and the development of globally aligned clinical and regulatory plans.

By the conclusion of the workshop, participants will be equipped with actionable knowledge to navigate orphan device approval pathways, manage cross-jurisdictional regulatory complexities, and optimize clinical and market access strategies for rare disease populations.