This EVERSANA white paper examines the critical success factors for commercializing orphan drugs developed for rare diseases under the U.S. Orphan Drug Act. While orphan designation offers incentives such as tax credits, fee waivers, market exclusivity, and expedited regulatory pathways, manufacturers face challenges including limited clinical data, small patient populations, low disease awareness, and complex market access. The paper outlines seven key commercialization pillars: generating compelling clinical evidence, building disease awareness, delivering personalized patient support, improving diagnosis and referrals, partnering with advocacy organizations, establishing therapy leadership, and maintaining agile operations. It concludes that integrated commercialization strategies and p

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