OBJECTIVES: In 2020, the European Commission (EC) developed its Pharmaceutical Strategy for Europe with the aim of reinforcing the European healthcare system. A central topic is the definition of unmet medical need (UMN) and its positioning as a director for pharmaceutical innovation. This study aims to explore stakeholders' perspectives on UMN-related proposals within the EC Pharmaceutical Strategy for Europe.

METHODS: To follow up on the proposed strategy, the EC launched a survey to capture stakeholders' views on proposed optimization avenues. The EC's survey comprised 15 questions including multiple-choice and open questions. Descriptive and quantitative statistics were performed on the multiple-choice questions and thematic framework analysis on the open questions. Subgroup analysis was performed at the stakeholder group level (i.e. end-users, researchers, healthcare providers, public bodies, industry, and other) to assess differences and similarities.

RESULTS: A total of 478 participants completed the survey, consisting of 19% end-users, 7% researchers, 17% healthcare providers, 7.5% public bodies, 36% industry, and 13.5% other. Findings indicate consensus among stakeholder groups on the key criteria for defining UMNs, including the absence of satisfactory authorized treatment and the seriousness of the disease. However, disagreement arose regarding the inclusion of "lack of access for patients" as a defining element, with public bodies and industry less in favor compared to other stakeholder groups. Stakeholders from public bodies, researchers, end-users, and healthcare providers expressed concerns about the EU pharmaceutical policy's ability to encourage innovation in disease areas with UMNs. Stakeholders also raised questions about disease severity, therapy availability, and defining major therapeutic advantages.

CONCLUSIONS: This study provides concrete insights into stakeholders’ perceptions of the relative importance of elements to define UMNs and measures that could support innovation to answer these UMNs. Addressing the disagreement among stakeholders regarding the definition of UMNs requires careful consideration and dialogue.