Pharmas’ challenge: learning from rare disease units while leaving them alone

How do you build a rare disease portfolio inside a pharma focused on large indications, and do right by both the new and the old? That’s a question faced by pharmas expanding from their traditional terrains to build Orphan disease beachheads.

On the one hand, the goal is to integrate best practices and mechanistic insights from Orphan drug development across programs for all types of disease. But that has to be offset against giving the units enough autonomy to meet the unique demands of rare diseases...