Unique challenges and opportunities in orphan diseases

The Orphan Drug Act was a pivotal moment for healthcare. Almost 40 years on, real-world evidence represents the next frontier. Jeremy Edwards, our CEO, outlines the challenges and opportunities in the orphan space

Before the Orphan Drug Act (ODA), only 38 drugs in total had been approved by the FDA in rare diseases, compare that with last year when 44% of FDA drug approvals were in rare, a total of 21 in one year alone. Evaluate Pharma predicts that orphan drugs will capture one-fifth of worldwide prescription sales by 2024. This ever-increasing focus on orphan diseases is driven mainly by the various financial and tax incentives attached to the ODA. It’s also driven by patient demand and advocacy. Digital channels have given a voice to the previously unheard.

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