The high cost of research, development, manufacturing and delivery of cell and gene therapies results in them having high prices that may strain healthcare budgets. However, methods involving price reduction, limiting patient access or linking payments to product performance can help to address this affordability challenge. This paper examines how patient access to one-off high value cell and gene therapies may be impacted by the net budget impact test recently introduced in England, and how access can be improved by managed entry agreements.|
To compare results for scenarios where a full upfront payment was used versus an annuity-based payment, and to determine the number of patients that can be treated without exceeding the £20 million net budget impact threshold, a hypothetical scenario was produced where a new high-value one-off therapy displaced a comparatively low-cost chronic treatment. It was determined that suboptimal patient access occurs when charging a full upfront payment at the time of treatment, and that increased patient access can be achieved by a performance-based renumeration system coupled with annuity-based payments.