WIB-Chicago: Rare to Ready – Navigating the Path to Rare Disease Drug Approval

On Thursday, June 26, we hosted a compelling panel discussion at Evanston Lab that brought together professionals and advocates in the life sciences community to explore the complexities of rare disease drug development. The panel featured Faith Eriks (Business Development Associate at Xentria), Tom Matthews (VP Clinical Development at Xentria), Amit Pithadia (CMC & Clinical Supply Director at Xentria), Noopur Singh (VP Marketing & Patient Affairs at Xentria), and Izabella Tyszler was the moderator. The panel shared their insights on the clinical, regulatory, and strategic challenges of bringing rare disease treatments to market.

The discussion began with an in-depth look at the unique challenges of clinical development in rare diseases, particularly in startup settings. Panelists stressed the importance of strong preclinical data, early engagement with regulators, and close collaboration between CMC and clinical teams. They also emphasized the need to define patient-focused endpoints, educate stakeholders, manage limited resources, and stay informed about competitor activity. A recurring theme throughout the discussion was the central role of patients in advancing rare disease research. Panelists spoke to the value of engaging both large advocacy groups and smaller, more targeted patient communities. They discussed the difficulty of identifying meaningful clinical endpoints, the need to remain adaptable in the face of supply chain issues and other disruptions, and the importance of using data to guide decisions and build contingency plans.

As the conversation shifted to career advice, panelists encouraged those new to the field to prioritize patient-centricity, seek out direct patient interactions, and remain humble in their approach. They also recommended networking early, attending advocacy events, and volunteering as valuable ways to gain insight into specific diseases and build credibility in the space. A special thank you to CBRE and Xentria for their generous support in making this event a success. 

Submitted by Melissa Tanner