Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands; National Health Care Institute, Diemen, The Netherlands.
DIA Europe, Middle East and Africa, Basel, Switzerland.
Value Health. 2019 Nov;22(11):1275-1282. doi: 10.1016/j.jval.2019.07.007. Epub 2019 Sep 6.
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.
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.
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.
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.
尽管在药物研发、监管和评估中越来越多地非正式和正式使用未满足的医疗需求（UMN），但对于其使用中的定义却没有深入了解。本研究旨在深入了解当前使用中的定义，并为利益相关者就一致性进行讨论提供一个起点。
进行了范围界定和灰色文献综述，以在文献和利益相关者网站上查找 UMN 的定义。这些定义进行了分类，然后通过半结构化小组讨论和更广泛的利益相关者参与的开放会议研讨会，由多利益相关者作者小组进行讨论。讨论了形成共同定义和使用机制的问题。
综述结果得到了 16 个定义。存在差异，但都包含以下 1 个或多个要素：（治疗的）充足性（16 个中有 16 个：100%）、疾病严重程度或负担（16 个中有 6 个：38%）和患者人群规模（16 个中有 1 个：6%）。利益相关者的讨论导致了一个包含前两个要素的定义建议，并根据具体情况包含人口规模。讨论还表明，UMN 的量化高度依赖于使用范围和价值框架，具体取决于不同利益相关者的偏好和责任。
我们鼓励希望在 UMN 概念上促进一致性的利益相关者，前瞻性地讨论他们希望在哪个范围内应用该概念，他们认为在每种情况下考虑哪些要素重要，以及他们将如何在适用的更广泛监管或价值框架内衡量 UMN。
