1. Pink Sheet

Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway

 
• By Derrick Gingery

Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.

orphan drug credit
Advocates want more incentives to encourage drug development for ultra-rare diseases. • Source: Shutterstock

Rare disease advocates want Congress to initiate a process that could result in a formal definition for an ultra-rare disease and additional incentives for drug development in the space.

Among the legislative asks that the EveryLife Foundation for Rare Diseases encouraged participants to push during a 2 March Capitol Hill lobbying day is a $1

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