HAE specialists cite costs, supply gaps as barriers to recommended care
Nearly two-thirds in Latin American survey reported preventive drug disruptions
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Specialists treating hereditary angioedema (HAE) across Latin America largely agree with international treatment guidelines, but high costs, limited access to medications, and frequent supply interruptions may keep patients from getting the care they need, a survey has found.
Nearly two-thirds of the 30 specialists surveyed across 10 countries reported frequent or constant interruptions in supplies of preventive medications. This included specialists who said they worked in settings with public healthcare or insurance coverage. Gaps also emerged in disease monitoring and access to recommended diagnostic testing.
Survey reveals gap between HAE guidelines and care
The findings show a gap “between what international guidance recommends and what participating experts reported their settings could deliver,” the researchers wrote, pointing to the need to address barriers to diagnosis, treatment access, and routine disease monitoring.
The study, “Hereditary angioedema in Latin America: international recommendations, expert consensus, and reported implementation barriers,” was published in Frontiers in Pharmacology.
HAE is a rare genetic disorder that causes sudden swelling attacks affecting almost any part of the body. Treatment of HAE includes on-demand medications to treat attacks when they occur and long-term preventive medications to reduce the risk of future attacks.
International guidelines recommend specific blood tests to help confirm an HAE diagnosis, as well as access to on-demand medications to treat attacks as soon as they begin. They also recommend considering long-term preventive treatment based on each patient’s needs, with targeted therapies preferred. Guidelines also call for regular assessments of how well the disease is controlled and its impact on quality of life to determine whether treatment is working or needs adjustment.
However, little is known about how consistently these recommendations are followed across Latin America or what barriers may prevent their implementation.
To investigate, researchers developed a 74-item questionnaire and conducted a two-round Delphi survey, which uses repeated surveys to identify areas of agreement among experts. The survey involved 30 HAE specialists from Argentina, Brazil, Chile, Colombia, Ecuador, Mexico, Panama, Peru, Uruguay, and Venezuela.
Experts agree on care standards but report access barriers
Participants reviewed recommendations covering diagnosis, treatment, disease monitoring, and care for specific patient groups. They also described how they treated patients and the challenges they faced in following those recommendations.
The experts agreed that blood tests for the most common forms of HAE should measure C4, a protein that’s usually low in people with these forms of the disease. They also agreed that tests should measure the levels and activity of C1 esterase inhibitor (C1-INH), a protein that may be present at low levels or not work properly. Genetic testing was considered particularly useful when the diagnosis remained uncertain.
However, the specialists reported differences in diagnostic testing across countries. According to the researchers, these differences may reflect unequal access to specialized tests and specialist care, potentially contributing to delays in diagnosis.
For long-term prevention, the panel supported three targeted treatments as first-line options: human plasma-derived C1-INH, which replaces the missing or poorly functioning protein; Takhzyro (lanadelumab); and Orladeyo (berotralstat). Takhzyro and Orladeyo both block a protein involved in triggering swelling attacks. The experts also agreed that on-demand medications should remain available to all patients, even those receiving preventive treatment, because attacks can still happen.
Yet the specialists reported major barriers to treatment access. Nineteen (63%) reported frequent or constant interruptions in supplies of preventive medications. That included 13 of 23 (57%) who said they practiced in settings with public healthcare or insurance coverage. High treatment costs were reported by 19 specialists (63%), while eight (27%) cited limited medication availability.
Although the panel supported newer targeted therapies, some specialists reported prescribing older medications, including attenuated androgens and antifibrinolytics. The researchers suggested that differences in insurance coverage, medication distribution, patient preferences, or clinicians’ experience may help explain why these older treatments were used.
Recommendations cover pregnancy, children, and disease monitoring
The panel also agreed that plasma-derived C1 inhibitor should be a first-line option for long-term prevention during pregnancy and breastfeeding. It was also the most frequently reported treatment for short-term prevention before procedures that may trigger attacks. For children, experts favored plasma-derived C1 inhibitor and Takhzyro, depending on the child’s age and individual circumstances.
The experts also agreed with international recommendations to regularly assess how well HAE is controlled and how it affects quality of life, using validated questionnaires. However, although 28 specialists (93%) said they assessed quality of life, only 18 (60%) named an assessment tool, and not all were validated specifically for HAE.
Overall, only 15 specialists (50%) said international guidelines were highly or fully applicable in their daily practice. Seven (23%) considered them moderately applicable, six (20%) rated them as having low applicability, and one (3%) said they were not applicable at all. One did not respond.
“Within this selected panel, consensus favored guideline-concordant HAE management, while reported implementation constraints were prominent,” the researchers wrote.
“Candidate [implementation] priorities arising from these reports include stronger laboratory capacity, more stable procurement and distribution, simpler coverage pathways, referral networks, telemedicine, and routine outcome measurement,” they added.
However, “because these reports were not independently verified and several countries were represented by only one to three respondents, the findings should not be generalized as national or regional estimates,” the researchers cautioned, emphasizing the need to validate the findings with “patient-level and objective health-system data.”
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