OBJECTIVES:

To illustrate potential consequences for the Dutch healthcare system of implementing different payment models for autologous gene therapy atidarsagene autotemcel (AA, Libmeldy®).

METHODS:

A calculation tool calculated three payment models: (1) a price discount of 60%, (2) an outcome-based spread payment with a 60% discount, and (3) an outcome-based spread payment linked to the willingness to pay (WTP) model with a 60% discount. Financial consequences were estimated for when patients are full responders (A), patients follow the transition probabilities to all health states provided by the marketing authorization holder (B), and when patients are assumed to be unstable responders (C). The associated costs for an average patient during the timeframe of the payment agreement (five years), the total budget impact (eight patients during the five-year interval), and associated benefits expressed in quality-adjusted-life-years for the total expected lifetime of the patient population were calculated.

RESULTS:

When patients respond according to the MAHs assumptions (Scenario B) implementing an outcome-based reimbursement model (payment models 2 and 3) has equal or lower associated budget impact while gaining similar benefits compared to a discount (€9,4 million to €5,6 million vs. €9.2 million). In the case of unstable responders (Scenario C) costs for payers are lower in the outcome-based scenarios (€3.4 million and €2.3 million, Scenario 2.C and 3.C, respectively) compared to implementing a discount (€9.2million, Scenario 1.C). Discounts should only be considered if patients are full responders.

CONCLUSIONS:

Outcome-based models were suitable to mitigate the financial risk and are preferred, from the payer perspective, for AA over simple discounts in situations when clinical performance was similar to or worse than predicted. The framework and calculation tool can aid reimbursement decision-makers to weigh the desirability of each of the different payment scenarios and support them with negotiating and implementing an outcome-based spread payment model.