Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies.

Unmet need is a core component of many Health Technology Assessment (HTA) processes at EU and national level. Most visibly, it is a core selection criterion for Joint Scientific Consultations (JSC) and Joint Clinical Assessment (JCA) for medical devices. This qualitative study explored how Unmet Medical Needs (UMNs) are understood and applied in drug development, with an emphasis on the European regulatory, HTA and access context, and examined their impact on regulatory and clinical development strategies. Twenty semi-structured interviews were conducted with representatives from regulatory authorities, HTA bodies, clinical development, industry, and patient insight roles. Data was analyzed using a thematic content approach combining deductive and inductive coding. Thematic analysis revealed general agreement on the importance of addressing UMNs, but also substantial variation in how they are defined and prioritized. Regulators often stressed disease severity and clinical evidence, while patients and clinicians emphasized quality of life. HTA representatives highlighted comparative benefit and long-term outcomes. These differing perspectives shaped how UMNs were integrated into development strategies, trial design, and regulatory planning. The findings indicate that clearer yet adaptable criteria could support earlier and more consistent alignment. Based on the analysis, a five-part roadmap to guide drug development is proposed, focusing on internal coordination, structured stakeholder engagement, collaboration between regulators and HTA bodies, adaptable definitions, and transparent decision-making. Together, these elements aim to support more systematic and predictable approaches to identifying and addressing unmet needs in drug development.