Presentation (CTI)

OBJECTIVES: Identifying and evaluating innovative medicines is essential for informed healthcare decision-making, particularly in health technology assessment (HTA) and resource allocation. However, the concept of pharmaceutical innovation remains ambiguous. The aim of this study was to identify existing evaluation frameworks used to assess the innovativeness of medicines and to analyse the key domains emphasised in these assessments.
METHODS: A systematic literature review was conducted using MEDLINE, Embase, Healthcare Administration Database and PsycInfo. Articles that classified or evaluated the innovativeness of medicines were included. From these, relevant frameworks and their components were extracted and analysed to identify common domains and criteria used in innovation assessments.
RESULTS: A total of 36 articles met the inclusion criteria, and 24 distinct evaluation frameworks were identified. No universally accepted definition or framework assessing the innovativeness of medicines was found. At its simplest, an innovative medicine is defined as a new active substance, but the reviewed frameworks revealed a more nuanced understanding. Three main domains emerged: 1) therapeutic (including therapeutic benefit, additional therapeutic benefit, quality of clinical evidence), 2) therapeutic need (including availability of existing treatments, unmet medical need, accessibility), and 3) technological (including new mechanism of action, safety, administration, cost). Among these, therapeutic benefit and the availability of alternative treatment options were the most frequently emphasised criteria. The heterogeneity in definitions and evaluation criteria underscores the complexity of operationalising pharmaceutical innovation in practice.
CONCLUSIONS: While innovation is often defined simply as the introduction of a new active substance, most frameworks incorporate broader elements such as clinical benefit and therapeutic need - especially relevant in HTA and decision-making. A transparent and consistent framework is needed to support evidence-based decision-making and ensure timely adoption of truly innovative medicines.