Shifts in the French HTA Transparency Committee’s Use of Indirect Comparisons: Insights From a Systematic Review of the French Transparency Committee (TC) Opinions
Author(s)
Margot Séjourné, MSc, malika belamri, PharmD, PhD, Anne-Line Couillerot, MSc, Erwan Autin, MSc.
Alira Health, Paris, France.
Alira Health, Paris, France.
OBJECTIVES: This study aims to assess how the French Transparency Committee (TC) has evaluated indirect comparison (IC) methodologies over the past three years. By conducting a comparative analysis of TC opinions issued before and after the release of its updated evaluation doctrine (February 2023), the study highlights shifts in the appraisal and acceptance of ICs.
METHODS: This study presents a systematic review of TC opinions including IC over an 18-month period before and after the publication of the doctrine (May 2021 - October 2024). Data were manually extracted to identify the type of CI methodologies, its level of acceptance and its potential impact on ASMR (added value) ratings. Quantitative and qualitative analyses assessed changes over time. Specific focus was placed on rare diseases and pediatric indications, where randomized controlled trials are often unfeasible.
RESULTS: Among 713 CT opinions analyzed, 136 (17%) included an IC, with a slight increase following February 2023 (94 vs. 79). Single-arm trials supported 27% of ICs, with increased acceptance observed post-doctrine. NMAs remained the most frequently used method, although their use is declining. In contrast, MAICs and propensity score-based methods have gained traction since 2023. MAICs, absent prior to 2023, now represent 20% of accepted ICs despite ongoing methodological concerns. Mentions of ICs in ASMR wording increased, suggesting improved transparency, although fewer methodological details were reported. IC acceptance also rose in pediatric submissions, and transparency improved in rare disease cases. Notably, the reassessment of Libtayo represents the only case where an IC directly led to an improved ASMR rating.
CONCLUSIONS: Despite the growing recognition of CIs, the CT remains cautious, requiring rigorous justification and methodology. This rigor contrasts with other, more permissive European agencies, particularly within the framework of the JCA (Joint Clinical Assessment), where the harmonization of methodological requirements is becoming a central issue for manufacturers.
METHODS: This study presents a systematic review of TC opinions including IC over an 18-month period before and after the publication of the doctrine (May 2021 - October 2024). Data were manually extracted to identify the type of CI methodologies, its level of acceptance and its potential impact on ASMR (added value) ratings. Quantitative and qualitative analyses assessed changes over time. Specific focus was placed on rare diseases and pediatric indications, where randomized controlled trials are often unfeasible.
RESULTS: Among 713 CT opinions analyzed, 136 (17%) included an IC, with a slight increase following February 2023 (94 vs. 79). Single-arm trials supported 27% of ICs, with increased acceptance observed post-doctrine. NMAs remained the most frequently used method, although their use is declining. In contrast, MAICs and propensity score-based methods have gained traction since 2023. MAICs, absent prior to 2023, now represent 20% of accepted ICs despite ongoing methodological concerns. Mentions of ICs in ASMR wording increased, suggesting improved transparency, although fewer methodological details were reported. IC acceptance also rose in pediatric submissions, and transparency improved in rare disease cases. Notably, the reassessment of Libtayo represents the only case where an IC directly led to an improved ASMR rating.
CONCLUSIONS: Despite the growing recognition of CIs, the CT remains cautious, requiring rigorous justification and methodology. This rigor contrasts with other, more permissive European agencies, particularly within the framework of the JCA (Joint Clinical Assessment), where the harmonization of methodological requirements is becoming a central issue for manufacturers.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA297
Topic
Clinical Outcomes, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas