Rare disease development is entering a new era—one defined by clearer regulatory expectations, deeper patient involvement, and a growing emphasis on feasibility, flexibility, and transparency.
In this eBook, we examine how evolving FDA programs, modern trial designs, and patient-centered practices are reshaping rare disease development. Drawing on recent regulatory guidance and real-world execution insights, the eBook explores how sponsors can design rigorous, feasible programs that meet both regulatory and patient expectations—while maintaining momentum in small, complex populations.
What You’ll Learn
- How FDA initiatives like RDEA, RDEP, and the Rare Disease Innovation Hub are shaping regulatory expectations
- Where regulatory flexibility applies—and where traditional rigor remains essential
- Practical strategies for integrating patient insight into trial design and feasibility planning
- How operational flexibility can reduce burden while protecting data quality
- Emerging expectations around returning individual patient data responsibly and transparently
Download the eBook
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