OBJECTIVES : Gene therapies promise revolutionary benefits for patients and healthcare systems, with a clinical trial pipeline indicating a further paradigm shift in medicine, but the novel logic of a one-off, potential cure creates challenges for both healthcare payers and manufacturers. Risk-sharing reimbursement models such as pay for performance are often hailed as effective remedies to reconcile value for money and affordability for healthcare systems with high prices as innovation incentives for developers, but their practicability and effectiveness remain contested. This research intends to assess the potential of innovative risk-sharing reimbursement models for gene therapies and to identify barriers preventing their effective use in a multi-payer healthcare system, using the German system as a case study.

METHODS : The research is based on explorative, semi-structured expert interviews with both German healthcare payers (n=5) and industry representatives (n=5). Key experts were selected by purposive sampling, according to expertise and experience in gene therapy reimbursement and the German pharmaceutical reimbursement system.

RESULTS : We find that performance-based reimbursement models may be attractive and practical to reconcile payer and manufacturer interests by risk shifting and inclusion of the outcome, but that barriers intrinsic to a multi-payer healthcare system impede their effective use for regenerative medicines, such as gene therapies. Risk-sharing models coupled with annuity payment plans may secure the sustainability of healthcare systems, distributing the financial burden for payers over several years, while manufacturers generate regular revenue streams. Furthermore, healthcare reforms such as centralized negotiations of risk-sharing deals linked with a cost pooling across funds may be a prerequisite for a futureproof remuneration strategy, enhancing innovation to be commercially viable and easily accessible.

CONCLUSIONS : We claim that performance-based agreements can be an effective, and practical tool to solve the gene therapy reimbursement conundrum, but that disruptive healthcare reforms may be additionally necessary in a multi-payer system.