Defining unmet medical need: mapping stakeholder perspectives in a multi-level framework for Europe

Area of Science:

• Pharmaceutical policy
• Health technology assessment
• Regulatory science

Background:

• The 2023 EU pharmaceutical legislation reform highlights the concept of unmet medical need (UMN).
• Stakeholders express differing views on the scope, purpose, and definition of UMN.
• There is a need for greater alignment in UMN definitions across regulatory and HTA procedures.

Purpose of the Study:

• To explore diverse stakeholder perspectives on the unmet medical need (UMN) concept.
• To identify perceived application purposes and definitional parameters of UMN.
• To inform the development of a practical framework for defining UMN.

Main Methods:

• Conducted semi-structured interviews with 38 stakeholders across Europe, including healthcare providers, policymakers, industry representatives, and patient representatives.
• Transcribed interviews verbatim and performed thematic analysis.
• Identified key sources of variation in UMN definitions and applications.

Main Results:

• A single, universally accepted definition of UMN is considered unattainable.
• Identified five framing dimensions (e.g., type of need, perspective, assessment level) influencing UMN definitions.
• Identified five assessment criteria (e.g., alternative availability, condition severity) and eight overarching application purposes for UMN.

Conclusions:

• A multi-level framework for defining UMN has been developed, integrating diverse stakeholder perspectives.
• This framework provides a practical tool for creating transparent and context-sensitive UMN definitions.
• Explicitly outlining underlying variables supports consistent and adaptable UMN definition development.