High clinical, economic burden with hemophilia A or B in France
Patients have higher mortality, work disability risk, higher healthcare visit rate
Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors.
That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of mortality, work disability, and a higher rate of healthcare visits, including outpatient care, emergency room visits, and inpatient care.
“Overall, the hemophilia clinical burden remained significantly higher as compared to [people who don’t have bleeding disorders], but it is likely that these outcomes have been considerably mitigated over time due to the organization of care, therapeutic innovations, and full coverage of patients’ expenses,” the study’s researchers wrote. The study, “Clinical and Economic Burden of Patients with Haemophilia A and B in France: Analysis of a Nationwide Claims Database,” was published in Haemophilia.
Hemophilia is mainly caused by genetic mutations that lead to a deficiency in proteins, called clotting factors, needed for the blood to clot, putting patients at an increased risk of excessive and prolonged bleeding episodes. Hemophilia A is caused by a deficiency in factor VIII (FVIII), while hemophilia B is driven by a deficiency in factor IX (FIX).
The standard treatment is factor replacement therapy, which supplies patients with a functional version of the missing clotting factor. It can be used as a prophylactic to prevent or reduce the frequency of bleeding episodes, or on demand to treat active bleeds. Some patients develop neutralizing antibodies, called inhibitors, against the supplied clotting factors, which may lower their effectiveness.
Clinical burden to patients
However, “there are few data on healthcare resource use and related costs of French hemophilia A and B patients,” wrote the researchers, who retrospectively analyzed data from the nationwide French claims database SNDS, which contains health-related information from more than 99% of the French population. A total of 6,909 hemophilia patients were included in the analysis, 5,577 with hemophilia A and 1,332 with hemophilia B. The patients were a mean age of 36 and mostly men (97.3% of those with hemophilia A; 83.1% of those with hemophilia B).
Most patients used on-demand therapies (72.8% of those with hemophilia A, 76.6% of those with hemophilia B), while prophylaxis was used by 27.2% of those with hemophilia A and 23.4% of those with hemophilia B. Inhibitors were present in 3.6% of those with hemophilia A and 1.1% with hemophilia B.
The researchers also compared hemophilia patients with 20,705 people without bleeding disorders matched by age, gender, and where they lived. Compared with them, the patients had a significantly higher clinical burden. The risk of mortality was 42% higher among hemophilia patients, with work disability being nearly three times higher.
This burden has been mitigated over the last 10 years due to the organization of care, but there are still gaps and push for even more coordination of care and prophylaxis to all patients who need it.
Hemophilia patients were 7.2 times more likely to have a history of HIV infection and 15.4 times more likely to have hepatitis, or liver inflammation, under active treatment. They were also more likely to use painkiller medications, visit healthcare providers, and have emergency room visits and be admitted to a hospital.
These results varied significantly when analyzing subgroups of patients according to their treatment regimen. Specifically, patients without inhibitors who were treated on demand had better indications than other patient groups.
Economic burden of hemophilia
People with hemophilia A had a mean total cost per patient of €87,764 (about $97,747), mainly associated with disease-specific treatments, compared with €1,656 (about $1,844) for those without a bleeding disorder. The most significant contributor to medication costs was Hemlibra (emicizumab) (67.8%), followed by FVIII replacement therapy (30.9%).
The costs varied significantly between subgroups of patients, spanning a total mean annual cost of €10,967 (about $12,214) for those without inhibitors who took on-demand therapies to €360,363 ($401,355) for those with inhibitors on a prophylactic. Extrapolating to the total hemophilia A French population, the economic burden for 2022 was estimated to amount to €480.2 million, or about $534 million.
For hemophilia B patients, the mean annual total cost per patient was €50,205 (about $55,915) compared with €1,590 (about $1,770) for people without a bleeding disorder. These costs were also mainly associated with medications, particularly extended half-life FIX products (84.8%). The results were limited to patients without inhibitors, with mean annual costs varying from €6,734 (about $7,500) for those on on-demand treatment to €181,566 (about $202,219) for those on a prophylactic. The national extrapolated economic annual burden of hemophilia B was estimated to be of €64.7 million (about $72 million) for 2022.
“Our study found that the clinical burden of hemophilia patients was significantly higher compared to [people without bleeding disorders], even though most patients on prophylaxis without inhibitors were treated by extended half-life factors for hemophilia B and [Hemlibra] for hemophilia A,” the researchers wrote. “We also estimated from a societal perspective the direct cost of hemophilia. This burden has been mitigated over the last 10 years due to the organization of care, but there are still gaps and push for even more coordination of care and prophylaxis to all patients who need it.”
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