Presentation (CTI)

OBJECTIVES: Decision-making on market access and reimbursement of innovative medicines is complex and involves multiple stakeholders. In this context, unmet medical need (UMN), defined as the gap between available healthcare interventions and patient needs, has gained increasing attention; however, its role in the Belgian reimbursement framework remains limited. This study aimed to identify the most relevant UMN and standard market access (MA) and reimbursement indicators for Belgian stakeholders, assess differences in prioritization across stakeholder groups, and identify areas of consensus to support the development of a decision-support tool integrating UMN into reimbursement decision-making.
METHODS: A cross-sectional survey was conducted among Belgian stakeholders, including policymakers, healthcare providers, academics, patient representatives, and pharmaceutical industry professionals. Respondents rated the relevance of predefined indicators, derived from literature and expert input, using a 5-point Likert-scale. Descriptive statistics were used to rank indicators overall and by stakeholder group. Differences in prioritization were assessed using the Kruskal-Wallis test. Consensus was defined as indicators ranked within the top 10 by at least three stakeholder groups.
RESULTS: Fifty respondents completed the survey. In the UMN domain, consensus was highest for general Health-Related Quality of Life (HRQoL), treatment effectiveness, life expectancy, and Quality of Life (QoL) score. In the standard MA and reimbursement domain, QoL score, life-saving effect, clinical effectiveness, and safety were ranked the highest, while economic indicators were generally less emphasized, except by policymakers. Overall, the UMN indicators received higher overall scores than standard MA and reimbursement indicators, with no statistically significant differences between stakeholder groups.
CONCLUSIONS: Stakeholders demonstrated similar preferences, particularly regarding clinical effectiveness and QoL. While no significant between-group differences were observed, this may reflect limited statistical power. Despite its exploratory nature and small sample size, this study provides preliminary insights into stakeholder priorities and highlights key indicators for incorporating UMN into reimbursement decision-making in Belgium.