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Open Access Assessing the cost and quality-of-life impact of on-demand‐only medications for adults with hereditary angioedema

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Background:

Novel subcutaneous (SC) prophylactic therapies are transforming the treatment landscape of hereditary angioedema (HAE). Although questions are being raised about their cost, little attention has been paid to the cost and quality of life (QoL) impact of using on-demand‐only medications.

Objective:

We assessed the overall economic burden of on-demand‐only treatment for HAE and compared patient QoL with patients who received novel SC prophylactic therapies.

Methods:

US Hereditary Angioedema Association members were invited to complete an anonymous online survey to profile attack frequency, treatment use, and the presence of comorbidities as well as economic and socioeconomic variables. We modeled on-demand treatment costs by using net pricing of medications in 2018, indirect patient and caregiver costs, and attack-related direct billed costs for emergency department admissions, physician office visits, and/or hospitalizations. QoL was assessed by using the Angioedema Quality of Life questionnaire.

Results:

A total of 1225 patients (31.4%) responded. Of these, 737 adults with HAE (type I or II) met the inclusion criteria and completed the survey. Per patient/year direct costs associated with modeled on-demand‐only treatment totaled $363,795, with additional indirect socioeconomic costs of $52,576 per patient/year. The greatest improvement in QoL was seen in patients who used novel SC prophylactic therapies, with a 59.5% (p < 0.01) improvement in median impairment scores versus on-demand‐only treatment. In addition, patients who used novel SC prophylactic therapies reported a 77% reduction in the number of attacks each year when compared with those who used on-demand‐only treatment.

Conclusion:

Our real-world patient data showed the cost and QoL burden of HAE treatment with on-demand‐only therapy. Use of novel SC prophylaxis can lead to sizeable reductions in attack frequency and statistically significant and clinically relevant improvements in QoL. These data could be useful to clinicians and patients as they consider therapy options for patients with HAE.

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Keywords: : hereditary angioedema (HAE); C1 inhibitor; C1inhibitor deficiency; health economics; lanadelumab; on-demand treatment; pharmacoeconomic; prophylaxis; quality of life (QoL); quality of life impairment

Document Type: Research Article

Affiliations: 1: From the US Hereditary Angioedema Association, Fairfax, Virginia; 2: Copenhagen Economics, Copenhagen, Denmark; 3: Hereditary Angioedema International, Fairfax, Virginia; and 4: Department of Medicine, University of California San Diego, La Jolla, California

Publication date: March 1, 2021

This article was made available online on February 13, 2021 as a Fast Track article with title: "Assessing the cost and quality-of-life impact of on-demand– only medications for adults with hereditary angioedema".

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  • Allergy and Asthma Proceedings is a peer reviewed publication dedicated to distributing timely scientific research regarding advancements in the knowledge and practice of allergy, asthma and immunology. Its primary readership consists of allergists and pulmonologists.

    The goal of the Proceedings is to publish articles with a predominantly clinical focus which directly impact quality of care for patients with allergic disease and asthma and by having the potential to directly impact the quality of patient care. AAP welcomes the submission of original works including peer-reviewed original research and clinical trial results. Additionally, as the official journal of the Eastern Allergy Conference (EAC), AAP will publish content from EAC poster sessions as well as review articles derived from EAC lectures.

    Featured topics include asthma, rhinitis, sinusitis, food allergies, allergic skin diseases, diagnostic techniques, allergens, and treatment modalities. Published material includes peer-reviewed original research, clinical trials and review articles.

    Articles marked "F" offer free full text for personal noncommercial use only.

    The journal is indexed in Thomson Reuters Web of Science and Science Citation Index Expanded, plus the National Library of Medicine's PubMed service.
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