Bringing treatments from concept to clinic presents unique challenges, and rare indications amplify the complexity
- Small patient populations make trial recruitment arduous
- Limited natural history data complicates endpoint selection and trial design
- Manufacturing specialized therapies on a small scale adds cost and strains logistics
To address these challenges and more, leaders in clinical development, translational science, and advanced therapy manufacturing collected their experiences into a free ungated eBook.
Expert insights from 200 rare disease development programs
Set your study up for success with practical (and actionable) advice:
- Early HEOR planning and support
- Patient identification and engagement strategies
- Central lab tactics for small sample sizes
- Enhanced sample management approaches
- Considerations for specialized manufacturing
