ISSUE: Over 2,600 gene therapy clinical trials have been reported worldwide, however, ensuring access to these therapies for patients has been challenging due to million-dollar price tags and uncertainty around long-term effectiveness. In 2021, the gene therapy manufacturer bluebird bio announced they will focus on the U.S. market after failing to receive reimbursement for Zyntelgo at the cost of $1.8 million dollars, claiming the European reimbursement system is “broken”. However, the Institute for Clinical and Economic Review (ICER) found Zyntelgo to be cost-effective in the U.S. at a price of $2.1 million dollars over five years, given FDA approval and evidence of durable treatment effectiveness over the same period. Further highlighting this heterogeneity, U.S. health plans have historically reimbursed more gene therapies than European payers, but often place significant restrictions on coverage, such as only providing insurance for patients meeting the strict eligibility criteria of pivotal trials. Despite these challenges, some gene therapies, such as Novartis’ Zolgensma^®, have secured reimbursement in both Europe and the U.S. The variability in reimbursement decisions across Europe and the U.S. has led to a disparity in patient access. It is important to understand what factors are driving this heterogeneity

OVERVIEW: Overview provided by Kate Hanman will be approximately 15 minutes. The issue panel will aim to explore whether differences in local reimbursement decision-making frameworks and payers’ willingness to adopt innovative payment agreements across geographies affect reimbursement outcomes. Furthermore, the session will discuss how the size and financial strength of the manufacturer contributes to variable reimbursement success in Europe and the U.S. With many gene therapies expected to come to market over the next few years, the panelists will consider what can be learned from gene therapy reimbursement outcomes to date and what different approaches need to be considered in each market to secure positive reimbursement outcomes.