Presentation (CTI)

OBJECTIVES: The Joint Clinical Assessment (JCA) mandated by the EU HTA Regulation (2021/2282) is closely tied to EMA milestones—specifically, the EMA submission (start of the EU HTA procedure) and the CHMP opinion (JCA dossier submission 45 days prior). While clinical study reports of pivotal trials satisfy key regulatory questions on efficacy and safety, they often lack elements required for HTA, such as appropriate comparators, subgroup analyses, HTA-relevant endpoints, or additional data cuts. However, the tight window between PICO scoping and JCA submission leaves little room for late-stage adjustments in statistical analyses. We aim to pinpoint where regulatory and HTA evidence-generation intersect, quantify timing gaps, and identify practical levers for delivering HTA-ready analyses.
METHODS: We overlaid EMA and JCA milestones to create a unified timeline and retrospectively analysed 8 recent marketing authorisation procedures (infectious diseases, cardiovascular disease, oncology) from 6 health technology developers. For each product we recorded the dates for database lock, reported top-line results, CSR analyses, full CSR completion and corresponding complementary analyses for German HTA (AMNOG) submissions, assessing when HTA-relevant data became available.
RESULTS: EMA and EU HTA clocks tick in parallel but demand different evidence. The median time from database lock to top-line disclosure was 10 days. CSR analyses were finalised after ≈ 30 days; full CSR completion required an additional 6 weeks. HTA-specific analyses were seldom initiated before CSR finalisation, limiting time to prepare HTA-specific analyses and risking delays in JCA submissions.
CONCLUSIONS: To meet the demanding EU HTA timelines, HTA analyses must be planned, prespecified, and initiated early, ideally in parallel with primary regulatory analyses, even in seemingly straightforward cases.