Presentation (CTI)

OBJECTIVES: Reimbursement of advanced therapy medicinal products (ATMPs) is perceived as particularly challenging. While the causes underlying their limited reimbursement are widely recognized, they are often described from specific perspectives or in isolation. This study aims to move beyond identifying challenges by providing a structured basis for root-cause-oriented strategies to improve their reimbursement.
METHODS: A literature-based root cause analysis was conducted to systematically identify the root causes of reported reimbursement challenges for ATMPs. First, a scoping review was conducted to identify scientific literature through both scientific and grey literature. Second, we applied causal mapping to the identified barriers and solutions to extract their structural root causes. Finally, key root causes were identified based on their interconnectedness across identified themes.
RESULTS: Identified causes and solutions were grouped into four overarching themes: budget impact, evidentiary uncertainty, and policies and other barriers. Key root causes identified across these themes are the small patient population, uncertain long-term value claim, and the limited knowledge and experience in the assessment and application of ATMPs.
CONCLUSIONS: The identified key root causes are not unique to ATMPs and occur for other therapies as well, indicating that it is not the uniqueness of characteristics, but rather the combination of characteristics that complicate reimbursement. Proposed solutions require a collaborative effort between stakeholders to foster knowledge and experience, update methodologies and guidelines, and multi criteria decision-analyses that explicitly consider innovativeness. As characteristics were not unique to ATMPs, proposed solutions are likely to be transferable and beneficial for the reimbursement of other therapies as well.