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Optimizing Expert Identification and Engagement to Improve Rare Disease Outcomes

July 22, 2023
Author(s): Joseph B. Laudano, BS Pharm, PharmD, Nikky Oladunmoye, PharmD, MPH, Gail Dutton, BS and David Kelaher, BPharm, MSc

Orphan drug approvals by the U.S. Food & Drug Administration (FDA) have increased steadily since 2010. For four of the past five years, they have comprised more than half of all drugs approved per year,. Yet, despite this, only 10 percent of the 7,000 recognized rare diseases have FDA-approved therapies. Consequently, there is relatively little research regarding some 90 … [Read more...]

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