Patient perspectives on the treatment burden of injectable medication for hereditary angioedema

Abstract

Hereditary angioedema (HAE) is a rare, chronic disease characterized by debilitating swelling episodes in various parts of the body. Patients experience significant burdens related to the symptoms and management of HAE, which can affect their daily lives and reduce their overall quality of life. Prophylactic treatment options have expanded in the past decade to the benefit of patients; however, these therapies require scheduled injections, which can be painful, burdensome, and time consuming. We conducted an online survey of patients with HAE in the USA to better understand their experiences with available prophylactic medications and the associated treatment burdens. Our survey results suggest that most patients are satisfied with their current therapies but desire novel medications with a simpler route of administration and that, although most patients experience significant treatment-related burdens, they learn to cope with these challenges over time.

Berotralstat (BCX7353): Structure-Guided Design of a Potent, Selective, and Oral Plasma Kallikrein Inhibitor to Prevent Attacks of Hereditary Angioedema (HAE)

“…Prophylactic therapeutic options have grown in the past two decades to include lanadelumab (Takhzyro), SC C1-INH (Haegarda), and IV C1-INH (Cinryze). All these treatment options are either large biologics or peptides that must be administered via injection or infusion leading to a significant treatment burden; therefore, the development of an oral long-term prophylactic therapy could increase convenience and improve the ease of administration, reducing treatment burden and improving the quality of life (QoL) in patients with HAE . No oral drugs have been approved for the prevention or treatment of HAE, though a few small molecule PKal inhibitors have been reported. − We report the discovery of Berotralstat (BCX7353), an orally bioavailable small molecule PKal inhibitor that has been effective in preventing HAE attacks and has recently been approved by the U.S. Food and Drug Administration (FDA), Japan’s Ministry of Health, Labor, and Welfare (MHLW), and the European Commission for the prophylactic treatment of HAE in patients 12 years and older.…”

Current challenges and future opportunities in patient‐focused management of hereditary angioedema: A narrative review

“…Furthermore, the availability of published data on HAE treatment patterns also varies among countries (Figure 2). For example, six studies on the treatments received by patients with HAE and two studies on HAE treatments prescribed by the physicians in the United States included data from up to 1429 patients with HAE and up to 156 physicians treating HAE per study 36–43 . In contrast, we have found only one study from the continent of Africa reporting on HAE treatment patterns; this study included 43 patients with HAE from South Africa 44 .…”

“…Conversely, the studies from United States reported established use of first‐line HAE treatment options, with the use of C1‐INH reported in up to 60.7% of patients and the use of lanadelumab (since 2018) reported in up to 53.3% of patients in separate studies 37–42 . Widespread use of C1‐INH was also reported in Canada (93.5% of physicians reported prescribing C1‐INH for LTP), 64 Germany (ongoing LTP and/or STP with C1‐INH reported in up to 65.0% of patients), 65 and Puerto Rico (LTP with C1‐INH reported in up to 68.7% patients who were taking prophylaxis) 66 .…”

“…In several studies from the United States, patients reported anxiety around taking their HAE medication, 41 finding needles and/or injections/infusions unpleasant, 38 and being tired of injections/infusions 38,41 . However, the majority of patients also reported learning to cope with the difficult aspects of their HAE treatment and being satisfied with their current injectable medications 40,41 . Additionally, injectable medications for HAE have longer dosing intervals, which have been associated with higher treatment adherence in several different therapeutic areas, presumably due to more patient convenience with less frequent dosing 93–95 …”

Patient and caregiver perspectives on transitioning to oral prophylaxis in the emerging hereditary angioedema treatment landscape

“… 12 Many patients with HAE report having an informal caregiver, most commonly a family member with HAE, who assists in their HAE‐related medical care. 13 , 14 Patients with HAE can also experience burdens related to repetitive long‐term injectable treatments such as lanadelumab and C1‐INH‐SC, which are administered subcutaneously and can be burdensome, inconvenient, and associated with injection‐site reactions. 9 , 10 , 15 In a 2018 report by the Center for Biologics Evaluation and Research and the U.S. Food and Drug Administration, patients with HAE considered the route of treatment administration to be an important factor in making treatment decisions, with oral preferred over subcutaneous (SC) administration and SC preferred over intravenous.…”