Life Quality Gains Evident With Newer Preventive HAE Therapies, Survey Finds

Newer preventive therapies for hereditary angioedema (HAE) lead to a better quality of life and fewer attacks than on-demand treatments, according to a large patient survey study.

The study, “Assessing the cost and quality-of-life impact of on-demand‐only medications for adults with hereditary angioedema,” carried out by the U.S. HAE Association and HAE International (HAEi), was published in the journal Allergy & Asthma Proceedings.

Modern preventive medications, such Takhzyro (lanadelumab) and Haegarda, are designed to stop HAE attacks from occurring or to limit their frequency enough to allow patients to lead largely attack-free lives.

The HAE Association Medical Board expects the use of these therapies to shift the focus of HAE management toward long-term prevention. As these medications become increasingly available, investigators have wondered about their comparative cost-effectiveness. Less attention, however, has been devoted to how on-demand-only therapies impact patients’ quality of life.

This study surveyed HAE patients to assess their financial burden related to on-demand-only treatments, and compared how this affected their quality of life relative to individuals using preventive treatments. The survey, according to HAEi, came largely as a response to an insurance industry-funded study.

“A study of HAE medicines published by a group of economists who receive funding from the insurance industry concluded that new preventive HAE medicines are not cost-effective,” Anthony Castaldo, president and CEO of HAEi and the study’s lead author, said in a press release.

“The U.S. HAE Association and HAEi joined forces in questioning the methods and conclusions of this analysis and responded by initiating a comprehensive study to determine the financial and quality of life impact of the new preventive medicines,” he added.

Castaldo and his colleagues issued an anonymous online survey to HAE Association members, asking about their attack frequency, treatment use, and co-occurring illnesses, in addition to economic and socioeconomic factors.

The group received 737 complete responses from people with HAE types 1 and 2, representing almost 20% of Association members.

Their responses showed that the direct costs associated with on-demand-only treatment amounted to $363,795 in total per patient per year, with further indirect costs reaching $52,576 per patient each year.

Indirect costs, largely socioeconomic, included estimates for the financial impact of sick days, reduced working hours, choice of education, ability to win promotions, and disability benefits.

Preventive treatments led to a considerable reduction in attack frequency compared to those for on-demand treatment. Patients using newer prophylactics reported a mean of 5.92 attacks per year, while those using other preventive therapies experienced a mean of 16.6 annual attacks.

Patients with on-demand-only treatments had the highest attack frequency, with a mean of 26.27 attacks each year.

The type of treatment used also affected the proportion of respondents reporting no attacks in the three months prior to the survey. Response data indicated that 57% of people using newer preventive treatments experienced zero attacks during this period, as opposed to 26% of those using other types of preventive treatment, and 19% of those with on-demand-only medicines.

Overall, the use of any preventive therapy was associated with a “clinically meaningful” 33.3% improvement in median quality of life scores, compared to on-demand-only therapies. In those using newer preventive treatments, a 59.5% improvement in median quality of life scores was seen compared to patients using on-demand-only therapies.

These findings, the researchers pointed out, are consistent with those from recent clinical trials that also found that modern preventive therapies significantly lower attack frequency while improving quality of life measures.

The researchers concluded that the use of modern preventive therapies can effectively reduce attack frequency and lead to “statistically significant and clinically relevant” quality of life improvements.

“Our real-world patient data revealed that modeled on-demand–only therapy had high treatment and socioeconomic costs, and was associated with high levels of QoL [quality of life] impairment,” they wrote. “These data could be useful to clinicians and patients as they consider therapy options for patients with HAE.”