Presentation (CTI)

OBJECTIVES: The interest in synthetic data in evidence generation is progressively increasing when access to real-world data is limited or not feasible within decision timelines. This study aimed at a pragmatic exploration of their potential role, highlighting conditions for reliable use in clinical, HTA and regulatory contexts from the perspective of Italian experts.
METHODS: Insights were derived from a structured multidisciplinary board including clinical, methodological, and HTA experts. The guided discussion explored familiarity with synthetic data, use cases, requirements, opportunities, and actions to facilitate its utilization.
RESULTS: Participants considered synthetic data as promising and complementary to real-world data evidence. Their value was considered greatest when evidence generation with established methods is constrained by small populations, clinical heterogeneity, privacy regulations, governance barriers, or decision timelines. Key usability requirements included high-quality, representative, traceable source data; transparent and trustworthy generation methods; preservation of relevant clinical and statistical patterns; privacy risk assessment; and a clear rationale for the intended use. Potential applications included integrative analyses when requested by decision-makers, expansion of subpopulations, and preliminary comparative scenarios or synthetic control arms based on retrospective data. Broader acceptability in HTA settings will require validation against real-world datasets, pilot cases with regulators, clinicians, payers, and data owners, and controlled environments, such as regulatory sandboxes, to test validation and governance standards.
CONCLUSIONS: Synthetic data are a valuable instrument to address selected evidence gaps, especially when real-world data cannot be generated, accessed, or shared in time for decision-making. Upon consideration of source data quality, clinical plausibility, statistical utility, privacy safeguards, and decision relevance, their use is a valuable complement to support clinical and HTA assessments, timely responding to needs of decision makers when they feel the need for integrative information. While their application spread, shared validation criteria, and regulatory dialogue are becoming essential to set clearer standards and support acceptable use.