Presentation (CTI)

OBJECTIVES: Since the EU HTA Regulation introduced Joint Clinical Assessment (JCA) for oncology medicines, uncertainty remains whether common clinical assessment can reduce country-specific evidence generation need. Hence, we assessed how transferable EU JCA requirements are to national HTA settings in rare oncology and identified the remaining evidence burden after JCA.
METHODS: Public JCA and HTA requirements from Germany, France, Italy, and Spain were reviewed across six domains: comparator selection, endpoints, subgroups, indirect treatment comparisons (ITCs), real-world evidence (RWE), and patient-reported outcomes (PROs). Each country-domain pair was classified as fully aligned, partially aligned, or materially divergent relative to JCA. Scenario-testing framework was applied to three rare-oncology examples: two ongoing JCA-based therapy archetypes and one published JCA benchmark. PICO variation, comparator gaps, survival maturity, ITC or external controls needs, PRO availability, and country-specific evidence requirements were assessed for each scenario.
RESULTS: Across 24 country-domain comparisons, eight were fully aligned, 14 partially aligned, and two materially divergent. Differences were mainly observed in comparator selection, ITC acceptability, and immature survival evidence, while PRO expectations were relatively more consistent. Post-JCA burden differed by therapy archetype. Targeted-therapy scenarios were mainly affected by comparator heterogeneity, subgroup reinterpretation, and local therapeutic-positioning needs. The ATMP/cell-therapy scenario showed the highest burden due to possible reliance on single-arm evidence, external controls or supportive RWE, uncertainty in long-term durability and survival maturity. The published JCA benchmark demonstrated a lower evidence burden, although local adaptation of evidence framing was required for reimbursement.
CONCLUSIONS: JCA provides an evidence base but not a fully transferable HTA package in rare oncology. Early experience highlights some alignment, but it is too early to establish whether JCA will achieve cross-market harmonisation. These findings suggest that evidence planning should integrate JCA requirements with anticipated national HTA evidence needs, specifically for ATMPs and targeted therapies that face heterogeneous PICO expectations and launch uncertainty.