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Overcoming Hurdles in Rare Disease Research and Development

Rare Disease Research and Development

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

Download: Rarefied Thinking In Rare Disease Research >