Expanding the definition of unmet medical need: Findings from a multistakeholder workshop.

Abstract

Background: Unmet medical need (UMN) is frequently referenced in health policy and health care decision-making, including the Centers for Medicare & Medicaid Services' (CMS) Drug Price Negotiation Program (DPNP). However, the concept of UMN is often defined inconsistently and has a narrow focus, primarily centered on clinical effectiveness and the availability of treatment. This approach may overlook the wider needs of patients, caregivers, and society as a whole. A broader conceptualization of UMN has been proposed, yet there remains a lack of an actionable implementation framework.

Objective: To examine how stakeholders interpret and distinguish among UMN elements, prioritize these elements based on perceived importance, and elicit perspectives on the applicability of a prioritized UMN framework in a decision-making context.

Methods: A multistakeholder workshop was conducted in June 2025 involving 17 participants representing researchers, industry, payers, policymakers, and a patient organization. Participants evaluated 38 UMN elements grouped into 7 domains derived from a prior structured literature review. Using an interactive polling platform, participants assessed the conceptual distinctness of elements within domains, ranked domains by importance, and completed a pairwise weighting exercise grounded in the Simple Multi-Attribute Rating Technique. Quantitative voting results were aggregated, and qualitative feedback from plenary discussions was recorded and thematically synthesized.

Results: Participants confirmed the conceptual distinctness of the proposed domains and elements. In the weighting exercise, traditional clinical effectiveness received the highest importance (24%), followed by availability of other treatments (19%) and impacts on patient, caregiver, or family quality of life (16%), although differences across domains were modest and should be interpreted with caution, given the exploratory sample. Economic burden on patients and families (13%), societal perspective elements (11%), and economic burden on society (10%) received moderate weights, whereas treatment administration attributes received the lowest weight (7%). Participants agreed that the broader element set captures dimensions of UMN not addressed by the current framework under CMS's DPNP. Key qualitative themes included perceived overlap within elements of the quality-of-life domain, the need for clearer conceptual boundaries between UMN and comparative effectiveness, and feasibility concerns around implementation.

Conclusions: UMN is inherently multidimensional and extends beyond traditional clinical measures. These exploratory findings suggest that structured prioritization of UMN domains may support more transparent and policy-relevant frameworks for evaluating unmet need in the United States, including within CMS's DPNP.