Presentation (CTI)

OBJECTIVES: Assessment of orphan medicinal products (OMPs) is challenged by the need to capture value beyond conventional cost-effectiveness outcomes. While multiple value elements have been proposed, there is no consensus on how to systematically structure, classify, or prioritise them for use in health technology assessment (HTA). This limits comparability, transparency, and methodological development. The aim of this study is to develop a reproducible and theory-informed framework that systematically organises broader value elements of OMPs and explicitly integrates their operational feasibility for HTA application.
METHODS: A structured narrative review identified value elements and methodological considerations relevant to OMP assessment published between January 2015 and February 2026 (n=61 publications, 43 including one or more value elements, 18 including multi-criteria decision analysis approaches). Extracted elements were synthesized through an iterative, framework-building approach. The framework was constructed along two orthogonal dimensions: (1) Stakeholder perspective (patients/caregivers, healthcare system, society), and
(2) Whole-health domains (physical, behavioural, socio-economic, environmental, and spiritual). Value elements were mapped across these axes to ensure conceptual completeness and non-overlapping classification. A third, cross-cutting operational layer was incorporated to characterise each value element according to data availability, methodological maturity, and measurability. This layer was represented as a gradient reflecting increasing uncertainty and implementation complexity.
RESULTS: Twenty value elements were identified and positioned within the framework. The structured mapping demonstrates full coverage across stakeholder groups and whole-health domains, while revealing heterogeneity in operational applicability. Elements central to current HTA practice are well-defined and measurable, whereas outer elements are less standardized and methodologically underdeveloped.
CONCLUSIONS: This framework provides a systematic and extensible method for structuring OMP value by separating conceptual identification from operational feasibility. It enables prioritisation of methodological research, supports consistent interpretation across studies, and offers a foundation for integrating broader value into HTA decision-making.