Managed care implications of hereditary angioedema

Hereditary angioedema (HAE) is a genetic syndrome caused by a functional deficit in complement C1 inhibitor that results in recurrent episodes of nonpruritic swelling of the hands, feet, arms, legs, trunk, face, genitalia, bowels, and larynx beginning in childhood or adolescence and continuing throughout the patient's lifetime. Delays in the diagnosis of HAE average several years owing to the rarity of the disorder and the mimicking of clinical symptoms of those of a variety of other diseases. The lives of people with HAE have been transformed in recent years by the approval of several effective new therapies for the treatment and prevention of angioedema attacks. Nonetheless, evidence from surveys shows that many patients still report suboptimal disease control accompanied by significant morbidity, psychological stress, and diminished productivity and quality of life. Further, new HAE agents are costly-the average wholesale price of a single dose of ecallantide (Kalbitor) is $9540-which will further strain an already stressed healthcare system. This article reviews the economic burdens, both direct and indirect, of HAE, and how changes in treatment practices, such as the wider adoption of home-based therapy, may improve patient outcomes and reduce costs.