© 2022 BioMarin. All rights reserved.

Improvements and gaps in reaching treatment goals for patients with hereditary angioedema

Science | November 23, 2022

David Hinds is a senior director of epidemiology & real world evidence in BioMarin's global medical affairs group

The contents of this post were first presented at a poster talk during the annual scientific meeting of the American College of Allergy, Asthma & Immunology’s November 2022 symposium.

Hereditary angioedema (HAE) is a genetic disorder that results in unpredictable episodes of severe swelling and can be life threatening. The swelling can affect different parts of the body and can be painful and debilitating. Attacks can even last for a few days. Fortunately, treatments are available that help prevent attacks when taken regularly. These treatments are called long-term prophylaxis (LTP). In this blog post I look at how much has improved for HAE patients and where there is still room for improvement towards reaching the established treatment goal of normalizing patients’ lives.

Steps in the right direction

Three long-term prophylactic therapies used to treat HAE due to C1-inhibitor deficiency have become available in the past five years. We wanted to see if use of healthcare resources (such as visits to emergency rooms or filling prescriptions of medication for relief of acute HAE symptoms) by patients with HAE had decreased in the real world after those treatments became available. Use of real-world data, in addition to data generated during clinical trials, is critical to building an understanding of a treatment’s efficacy.

We looked at data from an anonymized US insurance claims database in adults treated with selected LTP for a minimum of 180 days. We found ~40-50 new users of each of the treatments of interest, with similar characteristics between the two groups.

We found that the annualized use of healthcare decreased by greater than 50% after treatment initiation:

  • Medication fills for relief of acute HAE symptoms dropped from ~10 to 3-4 per year
  • Emergency visits/hospitalizations due to HAE dropped from 1-2 to ~0.5 per year

Our conclusion is that, while LTP treatments reduced resource use and costs considerably, they did not eliminate the need for continued prescriptions and hospitalization. Use of these LTP treatments also cost between ~$730,000 to $870,000, with pharmacy costs accounting for >95% of total costs.