There are more than 7,000 identified rare diseases today, 71.9% of which are genetic. And only around 5% of rare diseases have treatments. Development is complex and presents specific challenges. For example, many rare disease patients are children, since nearly 70% of rare diseases are exclusively pediatric-onset. The patient pool is often very limited for any one disease, and patients are often geographically dispersed, highlighting the importance of selecting a development partner with strong patient recruitment and global operational capabilities.
In this eBook, Parexel Biotech shares insights on maximizing the chances of success in rare disease drug development, providing our perspectives on four of the most significant challenges:
– Easing trial burdens on sites, and enhancing the clinical staff experience
– Ensuring that pediatric trials are ethical and feasible for patients and families
– Getting to market faster and stronger with natural history studies and
real-world evidence
– Learning from past successes and failures in orphan indications

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