The Challenges of Outcomes-Based Reimbursement for Advanced Therapy Medicinal Products in Europe

ISPOR Europe 2022 -- Advanced therapy medicinal products (ATMPs), such as cell and gene therapies, are often associated with single administration and curative potential in many chronic, life-limiting and life-threatening diseases. These 'cures' are typically accompanied by extremely high prices, while limited evidence to date means that their true long-term benefit is unknown. Outcomes-based reimbursement (OBR) is an attractive risk-sharing approach to the managed entry of ATMPs as it addresses both the inherent uncertainty in value determination and apprehensions regarding treatment cost. Despite the apparent utility of OBR, uptake remains low. The aim of this study was to review the literature discussing OBR for ATMPs in European markets to identify the barriers to, and opportunities for its use.