Importance of time horizon and analogue selection for value-based pricing of cell and gene therapies

AUTHOR(S) Anstee K, Jao R



  • Since the first approval of novel CGTs in 2017, patients with severe, debilitating, or life-threatening rare diseases have benefited from this one-time potentially curative treatment option.¹
  • With the opportunity to revolutionize patient care, many CGTs are currently in development. However, successful market access for CGTs is associated with many challenges for both manufacturers and payers:
    • Due to the personalized and complex manufacturing process of CGTs, these are high-cost products for reimbursement within a health care system.
    • Payer decision-making is limited to immature short-term clinical data from which curative or long-term results are highly uncertain.
    • Few CGTs have been launched and successfully reimbursed, which limits evidence and price benchmarking for new CGTs.
    • There remains uncertainty on the appropriate time horizon to capture the cost and curative benefits of CGTs for payer decision-making.²


Applying a 5-year time horizon, accounting for benefits of one-time treatment, and including both CGT and non-CGT orphan analogues improves the accuracy of value-based price forecasts for a new CGT.

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