Objective: This work outlines innovative payment schemes currently being utilized in Germany with regards to cell and gene therapies (CGTs). Transferability to the German market of innovative contracting currently utilized in other countries was assessed.

Methods: CTGs available in Germany were identified via EMA and Paul-Ehrlich-Institute listings. Published HTA assessments, reimbursement status and current prices (LAUER-TAXE^®) were collected. Reimbursement models in place were determined through publicly available sources (e.g. news articles). Research was supplemented with CRA-internal expertise.

Results: Several CGTs have become available on the German market in recent years, most notably the CAR-T cell therapies Kymriah^® (Tisagenlecleucel) and Yescarta^® (Axicabtagene ciloleucel). Often aimed at rare diseases, CGTs struggle to maintain a balance between data requirements for market authorisation and local HTA assessments, leading to timely limited reimbursement decisions in Germany. Additionally, current reimbursement systems are based on assessing yearly treatment costs and thus struggle with extremely high one-time costs of CGTs. Consequently, many pharmaceutical manufacturers have negotiated pay-for-performance reimbursement with sick funds, e.g. Gilead’s Yescarta^® with the VDEK. However, such pay-for-performance models have challenges of their own. Manufacturers and payers need to agree on appropriate outcomes to be monitored, which is usually challenging given the limited availability of data, and the timeframe of long-term efficacy assessment. The administrative framework for proper monitoring, and funding thereof, should also not be neglected. For drug developers, the payment structure of annuity pricing models will mean that resources dedicated to clinical research might not deliver a full return on investment for many years, impacting cash flow and potential future R&D investments.

Outlook: Here, the different innovative payment models pharmaceutical manufacturers have chosen in Germany will further explored, followed by assessment of how contracting models currently used internationally could be adapted in Germany. The barriers will be analysed, uncovering potential pathways to affect change.