The study, “Modeling Cost-Effectiveness of On-Demand Treatment for Hereditary Angioedema Attacks,” was published in the Journal of Managed Care & Specialty Pharmacy.
Hereditary angioedema (HAE) is most commonly caused by a lack of the protein C1 inhibitor (C1-INH) in the blood. Although there are preventive (prophylactic) therapies available that can reduce the frequency of HAE attacks, they still happen, often requiring “on-demand” treatments.
Some such treatments include the administration of C1-INH. This protein can either be recombinant (made in a lab), as is Ruconest, or it can be derived from plasma (the liquid component of blood), such as in Berinert. Other on-demand therapies, such as Kalbitor (ecallantide) and Firazyr (icatibant), work by targeting the pathways downstream of C1-INH that cause swelling.
Researchers now compared the cost-effectiveness of these four on-demand HAE therapies in a study funded by Pharming Healthcare, the manufacturer of Ruconest.
They constructed cost-effectiveness models for each of the therapies. These models included costs for the treatment itself, as well as related costs such as the potential need for hospitalization or re-dosing, which were estimated based on previously published studies for each of the therapies. Whether therapies could be self-administered or required administration from a healthcare professional also was considered.
Overall, the models suggested Ruconest as the most cost-effective of the four treatments analyzed, at an estimated $12,905 per attack. This was followed by Berinert ($14,668 per attack), Firazyr ($14,806 per attack), and Kalbitor ($21,068 per attack).
The effectiveness of each therapy also was estimated using a model that represented the amount of time per year during which an individual was not dealing with an HAE attack — with more effective therapies conferring less time spent with attacks. Due to the particular mathematics of the model, a maximum score of 0.83 was possible (indicating no attacks).
Ruconest had the highest estimated efficacy with a score of 0.806, followed by Kalbitor at 0.792, then Berinert at 0.769, and Firazyr at 0.765.
“This model finds that rhC1-INH [Ruconest] is both less costly and more effective than the remaining comparators and therefore represents the dominant therapy,” the researchers wrote.
These numbers were used to estimate the cost to an insurance plan covering 1 million people, assuming 20 people with HAE who experienced 26.9 attacks per person-year; these numbers are based on previously published HAE statistics.
From this, the overall cost incurred by an insurance plan for Ruconest would be $6.94 million per year, equating to $0.58 per member per month (PMPM). The overall cost of Firazyr was $7.97 million ($0.66 PMPM), of Berinert was $7.90 million ($0.66 PMPM), and of Kalbitor was $11.33 million ($0.94 PMPM).
“The budget impact model demonstrates that on-demand treatment using rhC1-INH [Ruconest] could represent cost savings of more than $1 million per year compared with icatibant [Firazyr] and more than $4 million compared with ecallantide [Kalbitor],” researchers wrote.
“Those figures are outright costs, and when combined with better effectiveness, rhC1-INH may also contribute to a higher quality of life for patients due to a low redose rate,” they added.
Further analysis of the model suggested that the low re-dose rate was a primary driver of Ruconest’s cost-efficacy, particularly in comparison to Firazyr and Berinert, which have comparable costs for the drug itself. The ability to self-administer — as opposed to requiring a healthcare professional to administer, as is the case for Kalbitor — also was a noteworthy factor.
It should be emphasized that these values are cost estimations.
“The most significant limitation to cost-effectiveness modeling of HAE is that as a rare disease there is not a large volume of research in general, and even less that examines costs and utilities associated with disease management,” the researchers wrote. “Therefore, a model such as this will necessarily have wide ranges for variables and at times require estimates in the place of empirical evidence.”
As such, according to the team, these estimations are most applicable to large-scale decisions of the sort faced by insurers. “We do not propose cost-effectiveness calculations for an individual HAE patient,” the researchers wrote.
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