Orphans Should Live Alone

Two recent events have brought into relief important considerations around the business models for developing and selling drugs for rare (ultra-orphan) diseases. Shire PLC’s latest acquisition, of Baxalta Inc., will create a specialist rare disease firm of unprecedented size, raising the question of whether the effectiveness of individual programs that by definition are targeted to very small patient populations can maintain their identity and integrity within the organizational structure. Sanofi’s decision to more fully integrate its Genzyme Corp. into the larger organization as the Sanofi Genzyme specialty care business unit similarly calls into question whether the move will remove some of the independence that is widely acknowledged as being critical to the successful development of rare disease drugs. Sanofi completed its acquisition of Genzyme in February 2011. [See Deal]

Large, established pharmaceutical firms have shown interest in drugs for ultra-orphan diseases, either through R&D or licensing/acquisition. Shire and Sanofi can argue a degree of success. Much of the Shire...