Rare diseases affect over 350 million people globally, half of whom are children. Despite this, more than 90% of these conditions still lack approved treatments, highlighting a significant unmet need and a substantial opportunity.
Orphan drugs, developed specifically for rare diseases, have demonstrated remarkable growth over the past decade, surpassing non-orphan drug sales and recently leading FDA approvals. While these drugs offer substantial rewards, their success depends on complex, high-stakes launches, making an optimal launch strategy essential.
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