Health Care Resource Use and Costs of Hemophilia A in France

Author(s)

Cahoreau V¹, Giraud N², Delienne S³, Fagnani F⁴, Bouee S⁴, Cottin J⁴, Bureau I⁴, Rudant J⁵, Fahfouhi Y⁵, Martin L⁵, Lilliu H⁶, Frenzel L⁷, Lebreton A⁸
¹CHU Bordeaux, Bordeaux cedex, France, ²Association Française des Hémophiles, Paris, France, ³CHU Dijon, Dijon cedex, France, ⁴CEMKA, Bourg-la-Reine, France, ⁵Pfizer, Paris, France, ⁶Inbeeo, London, LON, UK, ⁷Hôpital Necker, Paris, France, ⁸CHU Clermont-Ferrand, Clermont-Ferrand, France

Presentation Documents

ISPOREurope23_BOUEE_RWD98_POSTER129979.pdf

OBJECTIVES: Hemophilia A is an inherited bleeding disorder characterized by a deficiency in blood clotting factor VIII (FVIII). Its severity varies based on the degree of FVIII deficiency, with the most severe cases experiencing frequent spontaneous hemorrhages (FVIII levels < 1 IU/dL). Treatment options include on-demand administration following a hemorrhage or prophylaxis to prevent bleeding. The most frequent complication is the production of inhibitory antibodies against the coagulation factors leading to more aggressive and expensive treatments. The aim of this study was to estimate the healthcare consumption and related costs of Hemophilia A according to severity in France in 2021.

METHODS: Data relating to all patients diagnosed with Hemophilia A were extracted from the nationwide French claims database (SNDS) for the year 2021.

Patients were categorized in 4 groups according to the pattern of treatment (on demand / in prophylaxis) as a proxy of the severity of the disease (mild or minor / severe), and to the presence of inhibitors.

Healthcare resource use and direct costs were estimated in a societal perspective.

RESULTS: 5,507 patients with Hemophilia A were identified. Mean age was 36 years and 97.3% were males. The mean annual direct medical costs (drugs, consultations, hospitalizations, ...) varied strongly according to treatment modalities and presence of inhibitors:

€11,471 for 4,056 patients treated on demand without inhibitors (73.7% of patients)
€76,332 for 36 patients treated on demand with inhibitors (0.7% of patients)
€247,937 for 1,243 patients treated in prophylaxis without inhibitors (22.6% of patients)
€349,616 for 172 patients treated in prophylaxis with inhibitors (3.1% of patients).

The majority of the costs was related to antihemophilic drugs: 64%, 60%, 94% and 93% respectively for the 4 treatment groups.

CONCLUSIONS: The cost of Hemophilia A is high, varies greatly with disease severity and presence of inhibitors, and is mostly due to the antihemophilic drugs.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

RWD98

Topic

Economic Evaluation, Health Policy & Regulatory, Study Approaches

Topic Subcategory

Public Spending & National Health Expenditures

Disease

Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)

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