Defining unmet medical need: mapping stakeholder perspectives in a multi-level framework for Europe.

BACKGROUND: In the context of the 2023 reform of the EU pharmaceutical legislation, the concept of unmet medical need (UMN) has gained renewed attention. The proposal introduces a new definition of UMN and calls for greater alignment across regulatory and health technology assessment (HTA) procedures. However, stakeholders remain divided on the concept's scope, purpose, and definition. OBJECTIVE: To explore stakeholder perspectives on the UMN concept, and to identify perceived application purposes and definitional parameters. METHODS: Semi-structured interviews were conducted with (i) healthcare providers, (ii) policy-makers/advisors (e.g., HTA bodies, regulators, payers), (iii) pharmaceutical industry representatives, and (iv) patients (representatives) across Europe. Interviews were transcribed verbatim and analysed thematically. RESULTS: Interviews (N = 38) revealed that a universally accepted, one-size-fits-all definition of UMN is viewed as unattainable, given the differing application purposes and stakeholder priorities. Analysis identified key sources of variation, categorised into five framing dimensions, five assessment criteria, and eight overarching application purposes. Framing dimensions establish the conceptual boundaries of UMN, including the type of need considered (pharmaceuticals, diagnostics, preventive measures, healthcare services), perspective of need (patient, caregiver, healthcare provider, society), and assessment level (population or individual). Assessment criteria provide factors for UMN evaluation, including the availability of alternatives, severity of the condition, population vulnerability, availability of information or research, and the intervention's therapeutic advantage. CONCLUSIONS: This study presents a multi-level framework for defining UMN, grounded in diverse stakeholder perspectives and designed for practical use. By explicitly outlining the underlying variables, the framework supports the development of transparent and context-sensitive UMN definitions.