Presentation (CTI)

OBJECTIVES: In oncology, overall survival (OS) is considered the gold standard endpoint by the Transparency Committee (TC) when assessing clinical added value (ASMR). However, OS data are often immature at the time of initial assessment. This study aimed to identify drivers associated with higher ratings (ASMR III versus IV) when OS data are considered immature by the TC.
METHODS: A retrospective benchmark analysis was conducted using publicly available open data from the French HTA agency HAS. Opinions issued between 2018 and 2025 were screened for references to immature survival data or data maturity in the ASMR rationale. ASMR V ratings were excluded to focus on factors differentiating ASMR III from ASMR IV decisions in the presence of immature OS evidence.
RESULTS: Among the 11 oncology opinions eligible, 7 were granted an ASMR IV, while 4 achieved an ASMR III. ASMR IV ratings, were generally associated with persistent uncertainty resulting from immature OS combined with unfavorable safety profiles (e.g., Grade ≥3 toxicities), non-conclusive quality of life (QoL) data, or unresolved positioning. Conversely, ASMR III decisions were consistently supported by large treatment effect on clinically validated surrogate endpoint (e.g., Disease-Free Survival/ Event-Free Survival DFS/EFS), a high unmet medical need and acceptable safety profile. A temporal pattern was observed: while multiple ASMR III ratings were granted between 2018 and 2019 despite immature OS data, most decisions issued since 2022 resulted in ASMR IV.
CONCLUSIONS: Immature OS data alone does not appear sufficient to determine ASMR level. Rather, TC decision reflects the overall strength of evidence, including the clinical relevance of surrogate endpoints, magnitude of effect, unmet medical need, and residual uncertainty. Recent decisions suggest a more cautious interpretation of immature OS data, with ASMR IV increasingly assigned pending OS maturation.