How Is the Unmet Medical Need Concept Applied in Marketing Authorization and P&R Assessments? A Structured Document Analysis

OBJECTIVES: Unmet Medical Need (UMN) serves as a criterion for specific regulatory and market access procedures aimed at expediting access to highly necessary medicines. Due to varying interpretations of the UMN concept among stakeholders, this study aims to (A) gain insights into the frequency and consistency in the application of the UMN concept in assessment documents, and (B) identify the elements used to substantiate the UMN in specific cases.

METHODS: A structured full-document analysis was conducted on regulatory documents (European Public Assessment Reports (EPARs)) and Belgian pricing and reimbursement documents (CRM documents) for products granted European centralized marketing authorization through UMN procedures from 2015 to 2020.

RESULTS: A total of 142 innovative medicines authorized between 2015 and 2020 were included in the study, having been applied a European and/or Belgian UMN action. Of these, 103 were also reimbursed in Belgium, allowing for a comparative analysis in both EPARs and CRM documents. (A) Application of the concept: The UMN concept was mentioned in 80% of EPARs and 57% of CRM documents; however, it was not mentioned in 20% of EPARs and 43% of CRM documents. In cases where the concept was mentioned, 44% of EPARs and 22% of CRM documents explaining the rationale behind the UMN. Inconsistencies were observed between European and national levels, within the levels themselves, and the respective documents. (B) Characterizing or substantiating elements: The elements used to describe UMN included (i) the availability of alternative treatments and their added value, (ii) disease severity and burden on both the individual patient and society, and (iii) the vulnerability of the population, including rare diseases and children.

CONCLUSIONS: The analysis revealed that UMN rationale is often unclear in decision-making processes and inconsistently applied within and between organizations. Greater and more systematic reflection in publicly available documents could enhance transparency in decision-making.