The launch of cell and gene therapies can be challenging due to limitations in supporting clinical data, leading to uncertainty around their value claims. Additionally, their high costs create affordability issues for payers. To improve patient access and reduce payer uncertainty, outcomes-based reimbursement can be implemented; however, this requires operational data collection infrastructure and practice.|
This study aimed to identify the indications most likely to see the launch of cell or gene therapies in the UK over the next five years and assessed the suitability of the existing data collection infrastructure and clinical practice for enabling outcomes-based reimbursement in these indications. Target indications for cell or gene therapies in Phase III clinical development with EU and/or US trial sites were identified through secondary research and assessed for the availability of relevant data collection infrastructures in the UK. The suitability of existing infrastructure in supporting outcomes-based reimbursement was evaluated based on factors such as the time horizon of data collection, mandatory data entry requirements, and whether the infrastructure is product or therapy area-specific. These secondary research findings were validated through primary research involving expert interviews.
58 cell or gene therapies across 47 indications were identified, of which 20 are in oncology. Oncology indications have a better infrastructure for collecting outcomes data through the mandatory Systemic Anti-Cancer Treatment database, but data entry compliance can be limited due to resource constraints, and infrastructure upgrading will be required for outcomes-based reimbursement. Among non-oncology indications, around two-thirds have data collection infrastructures, but only three are suitable for outcomes-based reimbursement requirements.
Overall the study found that the existing data collection infrastructure in indications with potential cell or gene therapy launches in the UK over the next five years is insufficient to support outcomes-based reimbursement.