Accelerate RARE Working Groups

Monday, September 14, 2026

Objectives:

Develop stakeholder consensus on rare disease–specific clinical development topics.

Create briefing documents, guidelines and white papers to meaningfully accelerate rare disease clinical development.

Topic Proposals:

Please feel free to propose a meaningful 2026 workshop topic by sending an email to miro@raretrialssummit.com.

2025 Report:

37 experts from biopharma and patient advocacy convened at the 2025 Summit to draft the priorities of the rare disease clinical development community ahead of the upcoming PDUFA VIII reauthorization.

Download the Report