Presentation (CTI)

OBJECTIVES: Precision therapies, targeting small patient subgroups based on genetic or molecular characteristics, challenge traditional Health Technology Assessment (HTA) frameworks. We aimed to summarize how HTA methodologies across the UK and EU4 (France, Germany, Italy, Spain) have evolved to accommodate precision therapies, highlighting key innovations and implications.
METHODS: A review of HTA guidance documents and published literature was conducted, focusing on methodological changes related to evidence requirements, appraisal processes, and frameworks for precision therapies.
RESULTS: A total of 23 documents including HTA guidance and published literature were identified. HTA agencies in Europe have adopted several key methodological innovations: increased acceptance of real-world evidence and surrogate endpoints; implementation of adaptive and flexible appraisal pathways, such as managed access agreements; incorporation of sophisticated subgroup analyses; joint assessment of therapies and companion diagnostics; and expansion of value frameworks to include broader outcome measures. Country-level differences were notable; the UK and Italy demonstrated the greatest adaptability in HTA for precision medicine, using managed access, real-world data, and flexible pathways. Conversely, France and Germany generally maintained standard methods, emphasizing clinical endpoints with limited use of adaptive tools. Spain showed mixed, regionally variable approaches. International collaboration through initiatives like Joint Clinical Assessment may help foster methodological harmonization and facilitate cross-HTA evidence sharing.
CONCLUSIONS: These methodological advances seek to address the challenges of small patient populations, clinical uncertainty, and evolving evidence in precision medicine. By enabling flexible, data-driven assessments, they facilitate earlier patient access while maintaining scientific rigor. Differences in implementation between countries reflect local policy priorities, but ongoing collaboration is promoting greater alignment. HTA methodologies in the UK and EU4 are evolving to meet the unique demands of precision therapies. Continued innovation, international collaboration, and adaptation of value frameworks are essential to ensure equitable, efficient access to personalized treatments across Europe.