Innovative reimbursement mechanisms have long been considered potential solutions to the data uncertainty associated with one-off, high-value gene therapies that have long-term therapeutic potential, combined with limited supporting evidence at launch. The launches of increasing numbers of such gene therapies in Europe and the USA in the past 5 years provide valuable exemplars of how innovative reimbursement mechanisms are used by healthcare system decision makers in practice. This review details the use of such reimbursement schemes for recently launched gene therapies in key European countries and the USA, and shows that they are more widespread in Europe than in the USA. Although innovative payment schemes are increasingly used across countries, differences in healthcare system structures (e.g., single- vs multi-payer systems) and willingness to pay mean that decision makers in different countries have different incentives to manage uncertainties around long-term, real-world product value.
Keywords: chimeric antigen receptor T-cell therapy; gene therapy; health technology assessment; innovative payment mechanisms; market access; outcomes-based reimbursement.