A Comparison of the Acceptability of Evidence in the New Joint Clinical Assessment and Nine European Union Countries

OBJECTIVES: In 2025, Joint Clinical Assessment (JCA) will become a mandatory process for manufacturers seeking reimbursement in the European Union (EU). The Methodological and Procedural Guidance (MPG) subgroup dictates evidence requirements for JCA. This analysis compared the acceptability of evidence from nine EU member states relative to JCA requirements.

METHODS: A review of published HTA guidance from Germany, France, Sweden, Netherlands, Poland, Portugal, Slovakia, Spain and Italy was compared to MPG guidance. Acceptability was assessed for single-arm trials (SATs), anchored & unanchored indirect treatment comparisons (ITCs), real-world evidence (RWE), progression-free survival (PFS) and patient-reported outcomes (PROs). The review was validated with ex-payers and access experts.

RESULTS: MPG guidance indicates low acceptability of SATs, unanchored ITCs, and RWE; acceptability of anchored ITCs is case-dependent. Acceptability of common trial endpoints is low for PFS, but high for PROs. MPG guidance is in line with most requirements from Germany and, with a varying alignment across the remaining countries. MPG guidance is more restrictive than many local guidelines, particularly for PFS, SAT, RWE.

CONCLUSIONS: This analysis supports that JCA evidentiary expectations reflect some of the most restrictive requirements across EU HTA bodies, may further challenge interpretability and utility of comparative evidence for decision-making, and deepen access disparities despite the goal to reduce variability in the EU. The JCA scoping process may lead to multiple non-trial comparisons, for which a lack of available evidence can be expected due to strict JCA requirements for ITCs, particularly unanchored networks. The lack of acceptance of PFS as a surrogate endpoint represents an additional challenge, given it is commonly used in oncology trials and is recognized by medical societies. Flexibility in evidence requirements and acceptance of innovative approaches to evidence generation from JCA assessors will be necessary to ensure continuity of patient access to new medicines.