Medicaid Spending on Hemophilia Therapies in US Tripled in 2005-19

Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported.

Its researchers expect this finding will help state agencies with decisions regarding treatment coverage based on their Medicaid budget, given that the program “provides insurance coverage for approximately half of all US patients with hemophilia,” they wrote.

Results from this analysis were described in the research letter “Trends in the Use of Conventional and New Pharmaceuticals for Hemophilia Treatments Among Medicaid Enrollees, 2005-2020,” published in the journal JAMA Network Open.

Hemophilia is caused by mutations that lead to the lack of functional blood clotting proteins — factor VIII (FVIII) in type A, and factor IX (FIX) in type B. One of its standard treatments is replacement therapy, which involves administering a version of the missing clotting protein to patients. Bypassing agents, which can circumvent the need for clotting factor treatment, are another common therapy.

In recent decades, there have been substantial advances in how hemophilia is treated. Starting in 2014, extended half-life products, which are replacement therapies modified to last longer and so require less frequent dosing, became available. Hemlibra (emicizumab), an antibody-based treatment for hemophilia A, was also approved in 2018.

While these therapies have improved hemophilia care, they come at a high cost. According to the U.S.-based research team that authored this study, hemophilia is now “one of the most expensive medical conditions to manage.”

These researchers performed an analysis of Medicaid spending on hemophilia products in the U.S. from 2005 to 2020. Medicaid provides free or low-cost health coverage to qualifying low-income individuals, families and children, pregnant women, the elderly, and people with certain disabilities.

Findings showed a steady increase in the use of standard half-life replacement therapies prior to 2015. Then, after extended half-life products became available, the use of these standard therapies dropped substantially — by 66% for FVIII and by 57% for FIX. The use of bypassing agents also decreased by 69% from 2017 to 2019.

In 2005, Medicaid spending on hemophilia treatments totaled $521 million. By 2019, spending had more than tripled, to $1.57 billion.

Between these years, spending on FVIII products more than doubled (from $330 million in 2005 to $779 million in 2019), and spending on FIX products more than quadrupled (from $52 million in 2005 to $238 million in 2019).

“The transition of factors VIII and IX from plasma-derived to recombinant pharmaceuticals, the transition from [standard to extended half-life] pharmaceuticals, and the use of bypassing agents and emicizumab have contributed to the transformation of hemophilia from a disease of significant morbidity to a condition that allows affected individuals to lead active lives,” the researchers wrote. “However, these advances have increased costs substantially.”

As of 2019, FVIII products accounted for 50% of all Medicaid spending on hemophilia treatments. FIX products accounted for 15%, bypassing agents for 17%, and Hemlibra for 19% of total spending.

Researchers noted these findings may be useful for states determining their Medicaid budgets. According to the investigators, these findings are particularly relevant since gene therapies for hemophilia are expected to become available in the near future, and state agencies will have to make coverage decisions.

Gene therapies work to deliver a non-mutated version of the defective gene to patients’ cells, thereby restoring the production of the functional clotting factor they are missing.

“These gene therapies, which will cost between $2 to $3 million per patient, have the potential to decrease requirements for factor replacement dramatically,” the researchers wrote.