Four ways to increase rare disease drug development success

There are thousands of identified rare diseases today, yet only a small percentage of them have treatments readily available. In this new eBook, we present the Parexel Biotech perspective on maximizing the chances of success in rare disease drug development, including 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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