Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical...

Area of Science:

• Health Technology Assessment (HTA)
• Drug Development
• Regulatory Science

Background:

• Unmet medical needs (UMNs) are central to Health Technology Assessment (HTA) and regulatory decision-making for medical devices in Europe.
• Existing processes for identifying and addressing UMNs lack consistent definition and prioritization across stakeholders.
• This variability impacts regulatory pathways, clinical trial design, and market access strategies for new therapies.

Purpose of the Study:

• To explore the understanding and application of Unmet Medical Needs (UMNs) in drug development within the European regulatory, HTA, and access landscape.
• To examine the influence of differing UMN perspectives on regulatory and clinical development strategies.
• To propose a roadmap for more systematic and predictable identification and addressing of UMNs.

Main Methods:

• Qualitative study employing twenty semi-structured interviews with diverse stakeholders (regulators, HTA bodies, industry, patients, clinicians).
• Thematic content analysis using a combination of deductive and inductive coding.
• Analysis focused on understanding UMN definitions, prioritization, and impact on development strategies.

Main Results:

• General consensus exists on the importance of addressing UMNs, but significant divergence in definitions and prioritization was observed.
• Regulators prioritized disease severity and clinical evidence; patients/clinicians focused on quality of life; HTA bodies emphasized comparative benefit and long-term outcomes.
• These differing perspectives influenced integration into development strategies, trial design, and regulatory planning.

Conclusions:

• Clearer, yet adaptable, criteria for defining UMNs are needed to foster earlier and more consistent alignment between stakeholders.
• A proposed five-part roadmap emphasizes internal coordination, stakeholder engagement, regulator-HTA collaboration, adaptable definitions, and transparent decision-making.
• Implementing these recommendations can lead to more systematic and predictable approaches for identifying and addressing unmet medical needs in drug development.