Increasing rare disease awareness brings promise to orphan drug innovation

rphan drug innovation has gone from strength to strength, propelled by advances in genetic medicine, such as mRNA therapies. However, challenges remain in patient identification and carrying out small-scale complex clinical trials. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON Biotech, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape.

Identifying patient populations
Role of patient advocacy organizationsScalable containment
Regulatory complexity
Reimbursement concerns
CRO partnerships

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Increasing rare disease awareness brings promise to orphan drug innovation

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