Novartis is once again looking to be a pioneer in cell and gene therapy, though this time is selecting a potentially less complex administration method.

Outcomes and disease progression could be clear cut, lending the rare disease treatment to innovative contracting. But the amount of money at stake is likely to complicate things.

AHIP arrangement is a big win for ICER and will facilitate access to the organization’s cloud-based cost effectiveness review platform for a broad range of payers, particularly small and mid-size companies that may not have the resources to subscribe to the service on their own.

Treatment is a potential game-changer for patients with transfusion-dependent beta-thalassemia, committee members said; panel was hesitant to extrapolate the hematological malignancies seen with bluebird’s other lentiviral vector programs to beti-cel.