The cost of hemophilia treatment: the importance of minimizing it without detriment to its quality

ABSTRACT

Introduction: Hemophilia is a very expensive disease. Ameliorations in the management of hemophilia cause increased patient survival, more complex disease management, and augmented treatment costs. A literature search related to the cost of hemophilia was analyzed.

Areas covered: Clotting factor concentrates, which are the keystone of management, are costly. In fact, the yearly expenditure in the USA is 250,000 USD per adult patient. Indirect expenses also play a part in the economic load, and include lost productive capacity, uncompensated caretaker tariffs, and hemophilia-related physical handicap. Factor concentrates are responsible for more than 90% of the direct health-care expenditures of hemophilia. In an evaluation of the cost of management with high-dose prophylaxis in previously untreated patients from childbirth to puberty, including immune tolerance induction (ITI), the average annual treatment cost (Euros per kilogram) was 4391 EUR (approximately 4865 USD), and the average ITI cost was 383,448 EUR (approximately 424,860 USD). The expected ITI recovery period in the aforementioned evaluation was 1.8 years. To reduce costs, strategies such as disease management programs and drug pricing programs have also been implemented.

Expert opinion: Knowing the current cost of hemophilia is essential to ensure that patients are treated as efficiently as possible.

KEYWORDS:

• Hemophilia
• cost of care
• disease management programs
• drug pricing programs
• assistance programs
• inhibitors
• extended half-life products

Article highlights

• Factor concentrates account for more than 90% of the direct medical care costs of hemophilia.

• It is essential to carry out economic assessments in collaboration with clinical experts, budget managers and health economists.

• Disease management programs (DMPs) significantly reduce the average annual cost of hemophilia treatment.

• Drug pricing programs (DPPs) such as the 340B pharmacy program maintain a high quality of care and increase access to treatment.

• Assistance programs help many uninsured patients with hemophilia receive free factor products.

• The economic advantages of the new drugs (emicizumab) could be very important in patients with inhibitors and in those at risk of developing them.

Declaration of interest

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants, or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.