HAE Patients in US Favored Oral Prophylactic in 2018 Survey

People with hereditary angioedema (HAE) would prefer an oral medication that could prevent their attacks, even when satisfied with their current treatment, a survey of patients in the U.S. reports.

The study, “Hereditary angioedema patients would prefer newer-generation oral prophylaxis,” was published in the Journal of Drug Assessment.

BioCryst Pharmaceuticals, which developed and markets Orladeyo (berotralstat), an oral HAE preventive treatment recently approved in the U.S. and Japan, and available in the U.K., funded the study.

In recent years, new medications have become available to treat HAE and are considered effective. But until Orladeyo’s approval late last year, no oral therapies were available in the U.S. for the prophylactic (preventive) treatment of HAE.

Researchers with BioCryst and other institutions reported the results of a survey of HAE patients in the U.S. that was conducted in late 2018 (two years before Orladeyo’s approval).

A total of 75 people with an average age of 39 responded to the survey. The majority (97.3%) had HAE type 1 — only two patients had type 2 disease — and the average length of time since their HAE diagnosis was 16.7 years.

Nearly all (97%) respondents reported using some form of prescription medication to manage their HAE, including 64% who were actively taking prophylactic treatments. Demographics were generally similar between those who were and were not on prophylaxis, except that those on prophylactic treatment were significantly more likely to have health insurance that covers prescription drugs.

Survey respondents widely agreed on the importance of taking prophylactic medications as prescribed. But more than half (57%) also thought such treatment is burdensome. This viewpoint was more common among people who were not actively on prophylaxis.

Almost all (98-100%) of these patients said that they would be willing to try an oral prophylactic treatment. Willingness to try an oral prophylactic generally declined when the hypothetical medication was described as less effective than other options; nonetheless, about two-thirds of respondents not on prophylaxis said they would try an oral treatment even if it wasn’t very effective.

Nearly all respondents (98%) taking prophylactic medications agreed that they liked their current medication, but would prefer an oral medication. Many (85%) said they would switch to an oral medication if one became available.

Respondents generally agreed that convenience and lifestyle fit were important factors in choosing a treatment. Of note, two-thirds of respondents actively using injectable prophylaxis agreed that avoiding needle sticks would be their main reason for trying an oral alternative.

“Our research demonstrates strong patient preference for an oral prophylactic HAE medication option,” the researchers concluded.

They noted that a more convenient treatment could increase adherence — that is, help patients take their medications as prescribed — in addition to easing treatment burden.