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.
Two pharmas, Sanofi and Takeda Pharmaceutical Co. Ltd., discussed their strategies with BioCentury.
For decades, rare disease drug development was the domain of small specialty biotechs. Sanofi’s 2011 acquisition of Genzyme Corp. is often considered the tipping point, at which pharma started to get serious about the rare disease space. Genzyme’s success showed that the Orphan drug business could be sustainable and profitable.
Takeda followed suit with a major acquisition, its $62 billion takeout of Shire plc in 2018 (see “Takeda Hits the Gas”).
Pfizer has also joined the club, albeit with a more staged approach, progressively adding Orphan disease programs via licensing deals and small bolt-on acquisitions since 2010.
However, Orphan drug development is a different beast than drug development for larger indications.
Rare diseases require small trials with innovative approaches to design and endpoints, and greater interaction with the patient community at all stages along the R&D and commercial continuum. In preparation for commercial launches, Orphan drug companies must work with patient groups and clinicians