Bluebird Pushes For Zynteglo Pricing Of Five €315K Annual Installments
CEO Nick Leschly talked to Scrip about the European payment strategy for the one-time gene therapy, which will cost €1.575m over five years if treatment is successful.
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The cost-effectiveness watchdog now assumes an upfront $2.1m payment instead of five annual payments. The company switched gears to the US after the TDT gene therapy stumbled in Europe.
The company will prioritize US market for gene therapies; FDA also placed eli-cel for CALD on clinical hold following a case of MDS.
Value-based contracting in the US is moving toward high-cost drugs for rare disease and away from lower cost chronic treatments as payers plan for the coming pipeline of cell and gene therapies. Marketed gene therapies all have some kind of risk sharing deal in place but further progress on the most innovative payment models is hampered by regulatory and operational challenges.