Specialty Pharmacy Continuum: Specialty Pharmacies’ Role in Gene Therapy

By Gina Shaw
With contributions from:

– Allison Droba, Senior Operations and Program Manager, CGT, Orsini
– Eyad Farah, Chief Operating Officer, Orsini

Manufacturing of novel cell and gene therapy (CGT) products is extremely complex and patient-specific, so it can be a heavy lift to distribute these agents. However, several leading specialty pharmacies have developed dedicated programs focused on the unique financial, delivery, temperature control and patient support challenges associated with providing these therapies.

Orsini an Early Adopter

With three decades of experience in rare disease pharmacy, Illinois-based Orsini Specialty Pharmacy was well positioned to move into the CGT space in 2017, when it began supporting the cell therapy autologous cultured chondrocytes on a porcine collagen membrane (MACI, Vericel), used for the repair of full-thickness knee cartilage defects in adults. A year later, in 2018, Orsini entered the gene therapy channel with a program to support patients who are prescribed onasemnogene abeparvovec-xioi (Zolgensma, Novartis), an adeno-associated viral vector–based gene therapy for pediatric spinal muscular atrophy (SMA). . . .