Van Onvervulde Nood naar Besluitvorming: Empirische en Conceptuele Inzichten in het Farmaceutisch Beleid in Europa

The concept of unmet medical needs is been used in, and beyond, regulatory and market access practices, determining the eligibility of innovative therapeutics in pathways for accelerated market access. These pathways are developed to speed up market access for products meeting the most pressing needs but what are the highest needs? No generally accepted understanding or definition of the unmet medical need concept is available. This complicates its application in guiding decision-making and impeding a clear signal for drug developers. In this Ph.D. project, we want to assess and align stakeholders' understanding of the concept of unmet medical needs via a combination of qualitative studies, including interactions with different stakeholders (including policymakers, regulatory authorities, developers, payers/HTA bodies, patients, and healthcare providers), and quantitative studies, such as surveys and analysis of market data and policy documents. Based on these multi-stakeholder insights we aim to formulate policy recommendations and develop tools and guidance that can support stakeholders in their decision-making process and contribute to a drug evaluation framework that drives and supports the industry to meet the most pressing unmet medical needs.