AIFA Retrospective Analysis Reveals Added Therapeutic Value as Key Driver of Drug Innovativeness Recognition in Oncology
核心洞察
Between 2017 and 2024, AIFA (搜索) evaluated over 160 oncohaematological indications, granting full innovativeness in 36% of cases based on a multidimensional assessment model.
Added therapeutic value emerged as the primary determinant of evaluation outcomes, outweighing therapeutic need when considered in isolation.
A multidisciplinary working group has reached consensus on new parameters for defining added therapeutic advantage, particularly for endpoints not based on overall survival in early-stage malignancies.
The Italian Medicines Agency (AIFA (搜索)) has relied on a multidimensional assessment model since 2017 to determine drug innovativeness, and a new retrospective analysis covering the period 2017–2024 now reveals that added therapeutic value consistently served as the strongest predictor of a positive evaluation outcome—surpassing even the criterion of therapeutic need.
Under Determination No. 519 of 31 March 2017, AIFA (搜索) established three pivotal criteria for assessing innovativeness: therapeutic need, added therapeutic value, and quality of evidence. Over the subsequent seven years, the Agency evaluated more than 160 oncohaematological therapeutic indications, recognising full innovativeness in 36% of cases. The retrospective analysis of these decisions demonstrates that added therapeutic value was the main determinant of the evaluation outcome, even more than therapeutic need considered in isolation.
Institutional Evolution and Updated Criteria
The landscape for drug innovativeness assessment in Italy is undergoing significant change. AIFA (搜索) has recently restructured its institutional framework with the establishment of a single Scientific and Economic Commission (CSE) and has published updated criteria for the recognition of innovativeness. These domestic reforms coincide with the implementation of the Health Technology Assessment (HTA) Regulation at the European level, creating what the Agency describes as an opportunity for critical and prospective reflection on current evaluation parameters.
A key area of focus is the definition of added therapeutic advantage in relation to endpoints not based on overall survival (OS), particularly in the treatment of early-stage malignancies—a methodological challenge that has drawn increasing attention from regulators and clinicians alike.
Multidisciplinary Consensus on New Parameters
To address these evolving challenges, a multidisciplinary working group was convened, bringing together experts in oncology clinics, research methodology, regulatory procedures, and health economics. The group included, alongside the authors, Francesco De Lorenzo, Maria Carmela Piccirillo, and Entela Xoxi, operating within the framework of the Project "Oncology Early Asset Consensus Pathway with NGT (Nominal Group Technique) Methodology," with the non-contributing support of AstraZeneca.
Integrating these diverse disciplinary perspectives, the working group reached a shared consensus on a series of summarising elements aimed at refining how added therapeutic advantage is defined and measured—particularly when overall survival data are not yet available or feasible as primary endpoints.
Implications for Early Access and Resource Allocation
The recognition of drug innovativeness remains one of the central tools through which AIFA (搜索) guides early access to therapies, promotes clinical value, and governs the allocation of public resources. The finding that added therapeutic value drives evaluation outcomes underscores the Agency's emphasis on demonstrable clinical benefit over unmet need alone, a principle likely to be reinforced as the new CSE operationalises the updated criteria.
As the European HTA Regulation moves toward full implementation, Italy's experience with its multidimensional model—and the ongoing refinement of how therapeutic value is measured—may offer valuable lessons for other member states navigating similar assessments of oncology therapies in early-stage disease settings.