Pharma 5.0

Rare diseases: overcoming clinical development challenges with data

Published: 28-Feb-2025

From design to patient recruitment, investigator site selection and data interpretation, rare diseases present unique challenges for clinical trials. But, with only 5% of rare diseases having approved treatments, sponsors must find ways to make these studies more feasible and effective

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Thankfully, we live in an increasingly digital data-driven world, notes Dr Gen Li (pictured), founder and CEO of Phesi.

The clinical development industry has more access to real-world data on rare diseases, patients and clinical trial designs than ever before — as well as advanced AI tools that can rapidly extract precise insights from this information.

Here we look at five challenges in rare disease clinical development and why sponsors should be taking a data-driven approach to address them. 

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