Zynteglo Could Be Gene Therapy Test Case For Viability Of Outcomes-Based Contracts – UHC Exec
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.
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Gene Therapy: Bluebird’s Beti-Cel Sails Through US Panel Review On Strength of ‘Impressive’ Efficacy
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.
Draft evidence report from the Institute for Clinical and Economic Review on the potentially curative rare disease treatment also supports the narrative that the unrestricted pricing environment in the US invites greater access to innovative treatments compared to markets abroad.
Implementation of ‘multiple best price’ reporting approach to facilitate value-based payment arrangements postponed until 2022 to allow time for further guidance to be developed, but new definition of VBP arrangements is effective in March.