Data Points to Potential for Patient Centricity in Rare Disease Trial Success

Clinical Trials Arena August 19, 2025

Global orphan drug sales are on the rise. To dodge potential pitfalls associated with reaching ultra-rare populations, mapping patient pathways, reducing trial burden and partnering with advocacy groups is crucial

For health policymakers at a national level, rare diseases are those affecting a tiny fraction of the population. Worldwide, however, this adds up to over 300 million people. And it has emerged as a major growth area in the biopharmaceutical industry.

Thanks to incentives like the Orphan Drug Act and advances in genetics, drug development for uncommon ailments is booming. According to GlobalData projections, global orphan drug sales are projected to reach nearly $275 billion by 2028, reflecting roughly 12% year-on-year growth. Investment is pouring in from many biopharma firms;...

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