Presentation (CTI)

OBJECTIVES: The EU Health Technology Assessment Regulation (HTAR), implemented in January 2025, offers the potential to harmonise clinical evidence generation and streamline market access through Joint Clinical Assessments (JCAs). This study examines outstanding clinical trial design challenges and leverages expert insights to recommend policy interventions for successful JCAs moving forward.
METHODS: This research employed a scoping review complemented by a stakeholder policy roundtable. The literature search was conducted across major databases (PubMed, Scopus, ScienceDirect, Web of Science), resulting in 34 publications included from 2022 to 2025. Additionally, the policy roundtable validates literature findings and identifies practical strategies moving forward with clinical trial design experts, industry participants, HTA representatives and patient advocates.
RESULTS: Literature review findings highlight significant complexities associated with PICO (Population, Intervention, Comparator, Outcomes) harmonisation across Member States. A lack of PICO alignment could result in 10-16 distinct evidence requirements for one oncology indication, meaning manufacturers may face 280-720 analyses per product, creating substantial logistical and methodological burdens within short timeframes. Preliminary stakeholder feedback indicates optimism about reducing fragmentation, alongside concerns regarding implementation complexity. The policy roundtable explores outstanding challenges concerning clinical trial design under the HTAR, including varied acceptance of Real-World Evidence (RWE) within HTA assessments. The roundtable participants develop practical solutions for clinical trial optimisation under the new regulation and highlight outstanding policy work that needs to be done for the JCA to produce high quality and effective assessments. As the landscape is continuously evolving, ongoing developments will be incorporated between abstract submission and the conference.
CONCLUSIONS: Stakeholders’ perceptions of the HTAR reflect a blend of optimism and caution. While HTAR is seen as an improvement over fragmented national assessments, concerns remain about PICOs and integration of RWE persist, thus strategic refinements will be essential to realise the Regulation’s aims of streamlined market access across the EU.