Towards a New Understanding of Unmet Medical Need

Abstract

‘Unmet medical need’ (UMN) is a central concept in the incentivisation and development of new health technologies, and the identification of a particular medical need as ‘unmet’ is intended to encourage innovation in that area [1]. A meaningful distinction is essential for informed decision making and priority setting by a range of stakeholders including public research funders (e.g. Horizon Europe), health regulators (e.g. the European Medicines Agency), national and multi-national health technology assessment (HTA) agencies (e.g. EUnetHTA), patients and patient advocates, and the pharmaceutical industry. Incentives available to these stakeholders include, amongst others, preferential access to public research funds; protocol assistance for small-sized and medium-sized enterprises; access to alternative or ‘adaptive’ regulatory pathways; consideration of UMN as a value element in HTAs; and financial incentives or innovative payment models at the reimbursement stage. Targeted incentives for innovation around UMN are part of larger efforts to ensure the efficiency, effectiveness, quality, sustainability, safety and affordability of health systems [2], and there was broad agreement at a recent workshop on a pharmaceutical strategy for Europe that incentives are an important tool in ensuring the development of innovative medicines to address pressing health needs [3]. However, despite the centrality of UMN in the development pathway, this concept is not understood in the same way by different stakeholders [4]. Inconsistencies in its understanding diminish the usefulness of UMN as a concept that, at its essence, is meant to distinguish more urgent societal health needs from less urgent needs [2, 4]. We suggest that, to agree on shared and consistent criteria for distinguishing pressing from less urgent health needs, it will first be necessary for stakeholders to agree on what they are trying to achieve though this distinction.