CAPLACIZUMAB: HOSPITAL BUDGET IMPACT AND CLINICALLY APPROPRIATE USE AT THE UNIVERSITY PUBLIC HOSPITALS OF PARIS - FRANCE (AP-HP)
Author(s)
Juliette Gayet, PharmD student1, Albane Degrassat-Théas, PharmD2, Marion TANO, PharmD1, Philippe Mougenot, PharmD1, VIRGINIE SIORAT, PharmD1, ISABELLE FUSIER, PharmD1.
1AGEPS - APHP, Paris, France, 2Faculté de pharmacie de Paris, Paris, France.
1AGEPS - APHP, Paris, France, 2Faculté de pharmacie de Paris, Paris, France.
OBJECTIVES: In France, hospital drugs are reimbursed through the Diagnosis-Related Group (DRG)-based payment system. Innovative and expensive drugs are financed in addition to the DRGs if they meet specific criteria. Since 08/31/2018 Caplacizumab is indicated for the treatment in patients ≥ 12 years and ≥40 kg experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange. It is expensive and not funded in addition to the DRGs. It is reimbursed via hospital outpatient dispensing (retrocession) after discharge. This study aimed to evaluate caplacizumab hospital budget impact at Public Hospital of Paris (AP-HP) and to assess how treatment duration recommendations influence episode cost.
METHODS: Data on caplacizumab spending were extracted from AP-HP discharge database between 01/01/2020 and 12/31/2025 and were cross-referenced with recommendations. Two hypothetical cost scenarios were developed. The main indicator analysed was the mean cost per treated aTTP episode and total annual expenditure.
RESULTS: Spending on caplacizumab within AP-HP increased by 220% in 2025 compared to 2024, reaching a total of €5.4 million (€2.2 million for DRGs and €3.2 million for retrocession). This was driven by an increase in the number of episodes treated (an average of 20 in 2024 and 45 in 2025). In the marketing authorisation scenario, the total cost was €132k per episode (€39k for DRGs and €93k for retrocession). The scenario based on the 2022 Haute Autorité de Santé (HAS) recommendations and real-world clinical practice amounts to €122k per episode (€50k for DRGs and €72k for retrocession).
CONCLUSIONS: Aligning real-world use with recommended treatment duration may help mitigate the impact of high-cost drugs funded within DRGs on hospital budgets. This study highlights the importance of monitoring real-world drug spending and using economic assessment tools to identify medicines that require targeted oversight and interventions to promote their appropriate use.
METHODS: Data on caplacizumab spending were extracted from AP-HP discharge database between 01/01/2020 and 12/31/2025 and were cross-referenced with recommendations. Two hypothetical cost scenarios were developed. The main indicator analysed was the mean cost per treated aTTP episode and total annual expenditure.
RESULTS: Spending on caplacizumab within AP-HP increased by 220% in 2025 compared to 2024, reaching a total of €5.4 million (€2.2 million for DRGs and €3.2 million for retrocession). This was driven by an increase in the number of episodes treated (an average of 20 in 2024 and 45 in 2025). In the marketing authorisation scenario, the total cost was €132k per episode (€39k for DRGs and €93k for retrocession). The scenario based on the 2022 Haute Autorité de Santé (HAS) recommendations and real-world clinical practice amounts to €122k per episode (€50k for DRGs and €72k for retrocession).
CONCLUSIONS: Aligning real-world use with recommended treatment duration may help mitigate the impact of high-cost drugs funded within DRGs on hospital budgets. This study highlights the importance of monitoring real-world drug spending and using economic assessment tools to identify medicines that require targeted oversight and interventions to promote their appropriate use.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE718
Topic
Economic Evaluation, Health Policy & Regulatory
Topic Subcategory
Budget Impact Analysis
Disease
Rare & Orphan Diseases