OBJECTIVES: Hereditary angioedema (HAE) is a rare genetic disease caused by the deficiency or dysfunction of C1 esterase inhibitor (C1-INH), and characterized by recurring episodes of severe swelling, which can be life-threatening. Frequency and severity of attacks vary. Replacement C1-INH therapies provide effective relief; however, there is a lack of comparative data on economic outcomes in Central and Eastern European countries (CEE).

METHODS: Acquisition costs for human plasma-derived and recombinant C1-INH were collected from public databases for Bulgaria, Czech Republic, Hungary, Poland, and Romania. Attack rates and the mean number of doses required to control each attack were collected from published studies; data from randomized controlled studies were analysed separately from real-world and open-label studies. Cost per attack was estimated considering the distribution of patient weights and labelled dose regimens.

RESULTS: The proportion of patients requiring multiple doses to control an attack ranged from 9,1% to 65,7% in randomized studies and 0,2% to 30,9% in real-world studies. In general, recombinant C1-INH was less expensive than plasma-derived options, due to lower re-dosing rates. In the analysis using randomized studies, average savings per patient when using recombinant C1-INH were €312 per attack (€8,391 per year) in Bulgaria, €469 (€12,610) in Czech Republic, €122 (€3,284) in Hungary, €188 (€5,060) in Poland, and €88 (€2,368) in Romania. In the analysis using real-world studies, average savings per patient were €166 per attack (€4,454 per year) in Bulgaria, €288 (€7,735) in Czech Republic, €259 (€6,993) in Hungary, €339 (€9,124) in Poland, and €220 (€5,931) in Romania.

CONCLUSIONS: Based on published studies and drug prices, treatment with recombinant C1-INH may require fewer repeat doses to control an HAE attack and provide savings in CEE countries, compared to plasma-derived products. In addition to the economic impact of re-dosing, future analyses should consider the humanistic impact of rapid control of attacks.