PNH treatment Fabhalta may save $4M in lifetime costs: Study
Newer therapy more convenient and more cost-effective than standard of care
The use of Fabhalta (iptacopan) — approved in the U.S. in December 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — may save about $4 million over a patient’s lifetime compared with intravenous, or into-the-vein, standard of care therapies, according to a study by U.S. researchers.
The oral therapy was found, in the clinical studies that served as the basis for its U.S. approval, to be more convenient for patients than the use of other complement inhibitors for treating PNH. Fabhalta also was shown to work better than standard of care treatment for keeping PNH symptoms controlled.
Now, a team of researchers at Yale School of Medicine in New Haven, Connecticut, and the University of Pennsylvania in Philadelphia have used trial data to calculate the cost-effectiveness of Fabhalta for PNH patients versus that standard treatment.
According to the team, their analysis showed that Fabhalta “remained cost-saving across … all scenario analyses.” And, they added that, “across all probabilistic … analyses, [Fabhalta] was favored over [standard of care] in 100% of 10,000 [testing] iterations.”
Their study, “Cost-effectiveness of iptacopan for paroxysmal nocturnal hemoglobinuria,” was published in the journal Thrombosis and Hemostasis.
Scientists calculated quality-adjusted life years for standard and new therapies
The complement system is a set of proteins that are part of the body’s immune system. It normally helps protect the body against infections. In PNH, however, the complement system fails to recognize red blood cells as a healthy part of the body, mistakenly launching an attack against them, and causing the disease’s symptoms, which include fatigue, shortness of breath, and abdominal pain.
Unlike other approved complement inhibitors that target a protein called C5 to block complement activation, Fabhalta works by targeting another complement protein called factor B. By inhibiting factor B, Fabhalta prevents damage to circulating red blood cells, thereby easing symptoms.
Clinical studies comparing Fabhalta with C5 inhibitors like Soliris (eculizumab) and Ultomiris (ravulizumab) showed not only that Fabhalta was more convenient for patients — because it’s available as capsules to be taken by mouth, rather than by intravenous administration — but that it also worked better to keep symptoms controlled.
Now, the team of researchers sought to evaluate Fabhalta’s efficacy in relation to its cost. To that end, the researchers drew on data from APPLY-PNH (NCT04558918), a clinical trial that assessed the safety and efficacy of Fabhalta in 97 patients who had residual anemia despite being on Soliris or Ultomiris for at least six months.
Fabhalta costs $488,220 per year, per the study. Calculations showed that, over a patient’s lifetime, it provides 12.6 quality-adjusted life years, or QALYs — a measure that accounts for both health-related quality of life and length of life, with 1 QALY corresponding to one year of perfect health — at $9.52 million. In contrast, C5 inhibitors were found to provide 10.8 QALYs at $13.5 million.
These calculations suggest, according to the researchers, that Fabhalta is more cost-effective than standard of care treatment with C5 inhibitors — in fact, saving approximately $4 million over a patient’s lifetime. The use of the PNH treatment is also expected to reduce patient and nurse time in care by 730 to 2,920 hours, which is the equivalent of about one to four months.
Fabhalta found to be ‘the cost-effective strategy’ for PNH treatment
To make sure these findings were reliable, the researchers ran thousands of different cost and outcome scenarios. In every case, Fabhalta came out as the better and more cost-effective option, the team noted. It was found to be cost-saving in different countries, specifically Brazil, Japan, and the U.S.
The use of Fabhalta “contributes to a higher quality of life for patients with PNH at a lower overall cost burden, as compared with C5 inhibitors,” the researchers wrote.
We hope that our study will help support the use of [Fabhalta] in anti-C5–treated patients living with PNH … while decreasing health resource utilization, saving nursing and patient time, increasing patient transfusion independence, and ultimately, allowing for improved quality-adjusted life expectancy.
The team noted that “our findings must be carefully interpreted within the existing pricing landscape of PNH,” adding that the study “does not support incrementally increased prices for newer … medications.”
Overall, however, the researchers noted that Fabhalta is “the cost-effective strategy” for treatment.
“We hope that our study will help support the use of [Fabhalta] in anti-C5–treated patients living with PNH … while decreasing health resource utilization, saving nursing and patient time, increasing patient transfusion independence, and ultimately, allowing for improved quality-adjusted life expectancy,” the researchers wrote.
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