The Unmet Medical Needs Concept – A Structured Analysis of Its Application in Authorisation and Belgian Reimbursement Practices

OBJECTIVES: Unmet Medical Need (UMN) is a criterion for specific regulatory and market access procedures that are in place to accelerate access to highly needed medicines. As the interpretation of the concept differs among stakeholders, this study aims to (i) identify the criteria by which an UMN is characterised, (ii) identify the type of criteria reflected upon in (European) regulatory and (national) market access documents and the extent to which they are discussed, and (iii) identify differences and similarities between regulatory and market access documents in the reporting of UMN.

METHODS: A structured full-document analysis was performed of regulatory documents (European Public Assessment Reports (EPARs)) and Belgian market access documents (Managed Entry Agreements (MEA), and reimbursement documents) for products granted a European centralised marketing authorization via a procedure based on UMNs between 2015 and 2020. This analysis was informed by a structured literature review of policy documents to identify UMN definitions, applications and characterizing criteria.

RESULTS: A total of 118 products were identified of which 79 were reimbursed in Belgium, with 18 under a MEA. For these products, documents were screened for two main UMN criteria, as identified via the literature review, being the severity/burden of the disease and the presence of alternative treatment methods. The analysis showed that most EPARs lacked full substantiation of the identified criteria (90%) while more than half mentioned at least one without further explanation (64%). In the market access documents, reference to the UMN criteria were limited but therapeutic need and societal need were extensively discussed.

CONCLUSIONS: The extent to which the regulatory and market access documents reflect upon commonly used UMN criteria is limited. Increased and more systematic reflection in publicly available documents could help to generate improved transparency in decision-making and advance understanding of the application of the UMN concept among stakeholders.