Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries

摘要

ABSTRACT Background : The experience of Kymriah? and Yescarta? provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. Objective : To provide an overview of the reimbursement schemes used for Kymriah? and Yescarta? in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders. Findings : Kymriah? and Yescarta? have relatively uniform list prices across the EU5, and are reimbursed?according to their marketing authorisations. In France and the UK, reimbursement is on the condition of collecting additional data (at the cohort level) and subject to future reassessments; elsewhere, rebates (Germany) or staged payments (Italy and Spain) are linked to individual patient outcomes. Conclusions : The experience of Kymriah? and Yescarta? shows an increased appetite for outcomes-based reimbursement (OBR) in the EU5, with notably novel approaches applied in Italy and Spain (outcomes-based staged payments). Thus, real-world evidence (RWE) has become an increasingly powerful lever for demonstrating the value of health benefits in the clinical setting.