POTENTIAL PATIENT OPPORTUNITY LOSS ASSOCIATED WITH DELAYED AND ABSENT ACCESS TO NEWLY APPROVED MEDICINES IN FRANCE: A CROSS-COUNTRY ANALYSIS
Author(s)
Rahma Sellami, PharmD, MSc1, Marine Sivignon, PharmD2, Clement Francois, MSc, PhD1, Yosra Boukhris, PharmD3, Nicolas Virely, PharmD, MSc1, Loris Blasco, PharmD, MSc4, Pol-Antoine Hamon, PharmD4.
1Inizio Ignite Putnam, Paris, France, 2Inizio Ignite Putnam, Lyon, France, 3Inizio Ignite Putnam, Tunis, Tunisia, 4LEEM, Paris, France.
1Inizio Ignite Putnam, Paris, France, 2Inizio Ignite Putnam, Lyon, France, 3Inizio Ignite Putnam, Tunis, Tunisia, 4LEEM, Paris, France.
OBJECTIVES: To identify newly approved medicines reimbursed in major European countries but not in France and characterize the potential patient opportunity loss associated with delayed or absent access.
METHODS: A cross-country analysis was conducted on medicines receiving a first European Medicines Agency (EMA) marketing authorization between 2022 and 2025. Access status was assessed in France, Germany, England, Spain, and Italy using harmonized definitions based on national reimbursement status. Medicines reimbursed in four comparator countries but not in France as of December 31, 2025 were identified. Access trajectories, health technology assessment (HTA) outcomes, and reimbursement status were reviewed to characterize access barriers. Potential patient impact was characterized using target population estimates reported in Haute Autorité de Santé (HAS) assessments and contextualized using disease burden indicators.
RESULTS: Out of 193 medicines identified, 19 (10%) were reimbursed in Germany, England, Spain, and Italy but remained inaccessible through routine reimbursement pathways in France. Most belonged to oncology, hematology, neurology, and rare disease therapeutic areas. Among them, 13 were in a prolonged intermediate access phase despite a positive HTA assessment, 2 in a recent intermediate access phase, 2 received a negative HTA recommendation, and 2 had not been submitted for reimbursement by manufacturers. Most medicines received a moderate-to-important SMR, indicating recognized clinical value, but an ASMR V rating, limiting their ability to achieve routine reimbursement despite availability in comparator countries. The cumulative target populations reported in HAS assessments for these 19 medicines exceeded 640,000 patients. While not all eligible patients would necessarily receive treatment, this figure illustrates the potential magnitude of the populations affected by differences in access between France and comparator countries.
CONCLUSIONS: These findings suggest that access disparities may affect substantial patient populations despite recognized clinical value and may contribute to potential patient opportunity loss associated with delayed or absent access to newly approved therapies.
METHODS: A cross-country analysis was conducted on medicines receiving a first European Medicines Agency (EMA) marketing authorization between 2022 and 2025. Access status was assessed in France, Germany, England, Spain, and Italy using harmonized definitions based on national reimbursement status. Medicines reimbursed in four comparator countries but not in France as of December 31, 2025 were identified. Access trajectories, health technology assessment (HTA) outcomes, and reimbursement status were reviewed to characterize access barriers. Potential patient impact was characterized using target population estimates reported in Haute Autorité de Santé (HAS) assessments and contextualized using disease burden indicators.
RESULTS: Out of 193 medicines identified, 19 (10%) were reimbursed in Germany, England, Spain, and Italy but remained inaccessible through routine reimbursement pathways in France. Most belonged to oncology, hematology, neurology, and rare disease therapeutic areas. Among them, 13 were in a prolonged intermediate access phase despite a positive HTA assessment, 2 in a recent intermediate access phase, 2 received a negative HTA recommendation, and 2 had not been submitted for reimbursement by manufacturers. Most medicines received a moderate-to-important SMR, indicating recognized clinical value, but an ASMR V rating, limiting their ability to achieve routine reimbursement despite availability in comparator countries. The cumulative target populations reported in HAS assessments for these 19 medicines exceeded 640,000 patients. While not all eligible patients would necessarily receive treatment, this figure illustrates the potential magnitude of the populations affected by differences in access between France and comparator countries.
CONCLUSIONS: These findings suggest that access disparities may affect substantial patient populations despite recognized clinical value and may contribute to potential patient opportunity loss associated with delayed or absent access to newly approved therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR96
Topic
Health Policy & Regulatory, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Health Disparities & Equity, Insurance Systems & National Health Care, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas