Multi-Stakeholder Perspectives on Interpretation, Challenges, and Ways Forward for the Unmet Medical Need Concept – Semi-Structured Interviews

OBJECTIVES: In the context of the EU pharmaceutical legislation reform, the importance of aligning research priorities with unmet medical needs (UMNs) has been emphasized. However, a lack of stakeholder alignment on what constitutes an UMN hinders targeted research. This qualitative study aims to (1) gain insight into the understanding of various stakeholder groups on the concept of UMN, assessing differences and commonalities and (2) identify challenges and ways forward for the identification and assessment of UMNs.

METHODS: Qualitative insights were gathered through semi-structured interviews with patients, healthcare providers, policymakers, and pharmaceutical industry experts across Europe.

RESULTS: Interviews (N=30) revealed a shared perspective on the necessity of a more concrete definition(s) of and framework(s) for identifying UMNs. While stakeholders agreed that more clarity is needed, their interpretations of UMN varied based on their own context, with regulatory stakeholders focusing on medicine characteristics, reimbursement stakeholders considering existing alternatives and societal impact, industry experts referring to the boundaries of feasibility and success rate of developing medicines in challenging scientific contexts and disease areas, and patients demanding an inclusive approach, including medical care in its broader meaning, extending beyond the mere availability of treatment. Stakeholders proposed various criteria for the identification and assessment of UMN, including disease severity, the innovative nature of the proposed treatment, and the burden of disease on patients and/or society. However, divergent opinions arose regarding the inclusion of the availability of alternative treatment options as criterium to assess UMN. Notably, all stakeholders recognized the significance of quality of life as a key measure for quantifying unmet needs.

CONCLUSIONS: While the application of the UMN concept holds promise for guiding R&D efforts, this study found that there is a need for more clarity and a more precise definition(s)/framework of UMNs to support and encourage aligning of stakeholder efforts with UMNs.