Review of the French Commission for Economic Evaluation and Public Healths (CEESP) Activity: Focus on Orphan Drug
Author(s)
Guylian Serisier, PharmD candidate1, Lou Chambry, MsC2, Laetitia Rey, PharmD3, Marguerite Kandel, PhD1, Sandy Leproust, PhD4, Chloé Gervès-Pinquié, PhD5.
1IQVIA, Courbevoie, France, 2IQVIA Opérations France, Paris, France, 3Iqvia, Courbevoie, France, 4IQVIA Opérations France, La Défense, France, 5IQVIA, Paris, France.
1IQVIA, Courbevoie, France, 2IQVIA Opérations France, Paris, France, 3Iqvia, Courbevoie, France, 4IQVIA Opérations France, La Défense, France, 5IQVIA, Paris, France.
OBJECTIVES: In France, access to reimbursement requires manufacturers to submit a dossier to the French Commission for Economic Evaluation and Public Health (CEESP), responsible for health technology assessment. Since 2013, when a high added value and significant revenue are claimed, submitting an economic evaluation dossier has been mandatory. Given the specific characteristics of the economic and clinical data associated with orphan drugs (ODs), this study aims to review CEESP’s economic appraisals over the past decade.
METHODS: A dedicated database of economic appraisals for ODs, published through May 2025, was developed using IQVIA's CEESP economic opinion database. It compiles administrative data, results, classification of CEESP reservations and justifications. A comparative analysis was conducted between this database and the broader dataset of all drugs.
RESULTS: Since 2014, CEESP has published 66 economic opinions for ODs. Compared to all drugs, ODs are more often assessed at initial market entry (+28%) and based on non-comparative data (+17%). These drugs are mainly indicated in oncology (−18%), neurology (+8%), and hematology (+7%). A higher proportion of OD dossiers were invalidated (+14%), primarily due to more frequent major methodological reservations (RMM) (+13%) while the rate of major global uncertainty (IGM) remained unchanged. RMMs were primarily driven by structuring choices, modeling, and the measurement of utilities in both databases, with variations in proportions (−3%, −7%, and +7%, respectively). The median ICER (Incremental Cost Effectiveness Ratio) for ODs (€175,918/QALY) was 30% higher than that of all drugs (€135,422/QALY).
CONCLUSIONS: This analysis suggests that ODs undergo specific evaluations by the CEESP, with more frequent invalidations and higher ICERs (+30% among validated dossiers). These outcomes may reflect limited clinical data, high product costs, and substantial incremental costs versus comparators, often limited to supportive care. These findings offer valuable insights for stakeholders aiming to improve the robustness of OD economic dossiers in line with CEESP expectations.
METHODS: A dedicated database of economic appraisals for ODs, published through May 2025, was developed using IQVIA's CEESP economic opinion database. It compiles administrative data, results, classification of CEESP reservations and justifications. A comparative analysis was conducted between this database and the broader dataset of all drugs.
RESULTS: Since 2014, CEESP has published 66 economic opinions for ODs. Compared to all drugs, ODs are more often assessed at initial market entry (+28%) and based on non-comparative data (+17%). These drugs are mainly indicated in oncology (−18%), neurology (+8%), and hematology (+7%). A higher proportion of OD dossiers were invalidated (+14%), primarily due to more frequent major methodological reservations (RMM) (+13%) while the rate of major global uncertainty (IGM) remained unchanged. RMMs were primarily driven by structuring choices, modeling, and the measurement of utilities in both databases, with variations in proportions (−3%, −7%, and +7%, respectively). The median ICER (Incremental Cost Effectiveness Ratio) for ODs (€175,918/QALY) was 30% higher than that of all drugs (€135,422/QALY).
CONCLUSIONS: This analysis suggests that ODs undergo specific evaluations by the CEESP, with more frequent invalidations and higher ICERs (+30% among validated dossiers). These outcomes may reflect limited clinical data, high product costs, and substantial incremental costs versus comparators, often limited to supportive care. These findings offer valuable insights for stakeholders aiming to improve the robustness of OD economic dossiers in line with CEESP expectations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE653
Topic
Economic Evaluation, Health Technology Assessment, Study Approaches
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases