In the lead up to the imminent FDA opinion on the biologics license application (BLA) for Novartis’ gene therapy Zolgensma for the treatment of spinal muscular atrophy (SMA), the financial media company and magazine Forbes has published an interesting editorial on the controversial issues surround the pricing and reimbursement for ATMPs.
The article includes an opinion on the potential issues surrounding the price for Zolgensma given the possible extended benefits from treating young paediatric patients, the severity of the condition and the relative price of the standard of care over the life time of patients, making it a new pricing paradigm compared to already approved ATMPs.