Reimbursing the value of gene therapy care in an era of uncertainty.

Early-stage gene therapy (GT) clinical trials are demonstrating exciting results for persons with haemophilia (PWH), with the first products possibly licenced over the next few years for haemophilia A and B. These new treatments offer the possibility of a one-off approach to the treatment of haemophilia, with demonstrated increases in factor level expression and substantial reductions in both bleeds and factor utilization. However, clinical trial participants have demonstrated variable expression in factor levels, including decreases, over time, suggesting in some cases the effect may not last. The consequence of this uncertainty has led to challenging discussions on value and reimbursement. In most national healthcare systems, the relatively high cost of paying for GT on a one-off basis may be prohibitive, resulting in a lack of access and less post-marketing data generated, ultimately keeping these performance uncertainties high for payers. Economic models have demonstrated the cost-effectiveness of GT in haemophilia based on current clinical trial inputs, but it is in the certainty of these inputs and concomitant budget impacts where the lack of available data will be a concern for payers. To overcome the 'chicken and egg' discussion in relation to reimbursement and data, GT will necessitate new pricing and reimbursement models that share the risk between the manufacturer and the payer. New models have been described for other conditions. The aim of this paper is to propose illustrative concepts of haemophilia reimbursement models that may be further considered in the assessment of a less predictable therapeutic such as GT.