Evaluating Payment Models for ATMPs: A Comparative Study of Different Payment Models for Hemgenix®

Abstract

Introduction Advanced Therapy Medicinal Products (ATMPs), like gene therapy Hemgenix® for Hemophilia B, offer curative potential for rare diseases with unmet needs. Despite their clinical promise, high upfront costs and uncertain long-term efficacy pose significant challenges to their uptake through traditional healthcare reimbursement models. Aim This study aims to compare the costs incurred by different payment models for the one-time high-cost ATMP, Hemgenix®, and the budget impact of these payment models on healthcare payers in Sweden. Methods A decision support framework was applied to determine the suitable alternative payment methods for Hemgenix®. Followed by a cost assessment using a Markov model simulated over 5,10, and 15 years for a hypothetical cohort of Hemophilia B patients across three scenarios of clinical response (according to the Hope B trial, full, and partial response). Three payment models: full upfront payment, 30% reduced upfront payment, and outcome-based annuity payment, were compared alongside standard prophylaxis. Results In the short term, Hemgenix® was more expensive than prophylaxis, but over time, the reduced upfront payment and outcome-based models resulted to be less expensive as compared to prophylaxis. At 15 years, outcome-based annuity models proved most affordable under partial response scenarios, while the reduced upfront payment model was cheaper under full response. Sensitivity analyses showed that with increasing clinical uncertainty, the cost incurred by outcome-based models became lower compared to other Hemgenix® payment models. Conclusions: While the reduced upfront payment models with a considerable percentage of concession are a financially viable option, outcome-based annuity models effectively mitigate financial risk and budget impact under clinical uncertainty. Tailored reimbursement strategies can ensure early and sustainable access to ATMPs like Hemgenix® and reduce long-term burden of rare diseases like Hemophilia B.