Katie McCarthy | SVP & Practice Lead, Clinical & Regulatory
Lumanity, Inc.

Day 1 - World Orphan Drug Congress USA 2026 @ 12:10

Rare Disease Regulatory Pathways in Flux: Navigating FDA and EMA Changes in 2026

Rare disease drug development is facing rapid regulatory change in 2026. Both FDA and EMA are finalizing guidance that will reshape trial design, accelerated approval standards, and manufacturing oversight for gene and cell therapies. This panel will deliver a real-time analysis of the most recent updates - from confirmatory trial requirements to global harmonization efforts - and explore how developers can adapt without losing momentum. Attendees will leave with actionable insight into the policies and processes that will define rare disease approvals in the years ahead. 

Objectives: 

• Analyze newly released FDA and EMA guidance impacting rare disease drug development in 2026. 
• Understand mid-year changes to accelerated approval standards, confirmatory trial expectations, and endpoint flexibility. 
• Prepare for updated manufacturing and CMC requirements for gene and cell therapies in rare indications. 
• Identify strategies to maintain development timelines while adapting to evolving regulatory frameworks. 

Moderator: Katie McCarthy, SVP & Practice Lead, Clinical & Regulatory, Lumanity, Inc.

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Katie McCarthy / Rare Disease Regulatory Pathways in Flux: Navigating FDA and EMA Changes in 2026

Katie McCarthy, SVP & Practice Lead, Clinical & Regulatory,
Lumanity, Inc.

Katie McCarthy is a seasoned professional with over 25 years of experience in the biopharmaceutical industry. As the Senior Vice President and Practice Lead of Clinical & Regulatory at Lumanity, Inc., she plays a pivotal role in driving the company's strategic initiatives and client...

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