Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions

Abstract

Background: Despite increasing informal and formal use of unmet medical need (UMN) in drug development, regulation, and assessment, there is no insight into its definitions in use. This study aims to provide insight into the current definitions in use and to provide a starting point for a multi-stakeholder discussion on alignment. Methods: A scoping and a gray literature review were performed to locate definitions of UMN in literature and on stakeholder websites. These definitions were categorized and then discussed among the multi-stakeholder author group via semistructured group discussions and open session workshops with a broader stakeholder audience. Issues with the formation of a common definition and mechanisms for use were discussed. Results: The reviews yielded 16 definitions. Differences were evident, but all included 1 or more of the following elements: (adequacy of) available treatments (16 of 16: 100%), disease severity or burden (6 of 16: 38%), and patient population size (1 of 16: 6%). The stakeholder discussions led to a suggestion for a definition including the first 2 items and, depending on context, population size. The discussions also showed that quantification of UMN is highly dependent on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities. Conclusion: We encourage stakeholders that want to promote alignment on the concept of UMN to prospectively discuss the scope in which they want to apply the concept, what elements they find important for consideration in each case, and how they would measure UMN within the broader regulatory or value framework applicable.

The fourth edition of the European Network for Health Technology Assessment Forum: highlights and outcomes

“…The Forum has set up the basis for a continuing discussion on the concept of unmet medical need. The relevance of this topic has been confirmed by a recent review which identified sixteen different definitions used across different stakeholders (14). Overall, discussions on the different topics covered during this edition – joint production of assessment reports, engagement with payers, patients, and HCPs – emphasized the importance of synergies, pragmatism, and inclusiveness across Member States and stakeholders to put in place a collaboration that serves the interest of patients and public health in a truly European spirit.…”

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

“…The identified benefits and limitations of the MEAs may seem contradictory. However, this observation relates to the various perspectives or interests of stakeholders involved [82][83][84][85][86]. Restrictive reimbursement for a subgroup, for example, always has multiple perspectives, i.e., loss of revenue for the company or lack of access for a specific patient population, but an opportunity for an HTA organization or payer to gain evidence that is lacking.…”

The Qualitative Value of Facilitated Regulatory Pathways in Europe, USA, and Japan: Benefits, Barriers to Utilization, and Suggested Solutions

“…Overall, these recommendations support the need for continued agency-agency and agency-company discussions on aligning FRPs, which can benefit from better alignment on the criteria for unmet need and innovation. These have already started to take place, for example as directed by the EMA [22], as well as through International Coalition of Medicines Regulatory Authorities [23] and multistakeholder meetings [20,[24][25][26].…”