Maximizing the chances of success in rare disease development is complex. In this eBook, our biotech experts share their perspectives on what it takes to streamline every step of the rare disease drug development journey.
Rare disease drug development is complicated and rife with unique and specific challenges. Limited patient pools, geographic logistics and the unfortunate fact that many rare disease patients are children are some of the more daunting to overcome. But by selecting a development partner with specialized rare disease expertise and strong global operational capabilities, it can be done. In this eBook, Parexel Biotech shares perspectives on four ways to address challenges, including:
- Easing trial burdens on sites
- Ensuring pediatric trials are ethical and feasible for families
- Speeding development with natural history studies and real-world evidence
- Learning from previous orphan indications