RATIONALE: Hereditary angioedema (HAE) is a genetic disorder characterized by swelling episodes (attacks). New prophylactic treatments are being investigated and indicated for HAE. Little is known about patients’ preferences for attributes of investigational or existing prophylactic treatments.
METHODS: Patients invited through an HAE patient advocacy group completed an online survey with a discrete-choice experiment. Hypothetical treatments were described by onset of drug action; reduction in attack frequency; mode and frequency of administration; and risks of mild-to-moderate injection site reactions (ISRs) and gastrointestinal (GI) side effects. Preference weight estimates from random-parameters logit models were used to calculate the relative importance of attributes and risk tolerance.
RESULTS: 250 patients completed the survey (81.6% female; age [mean, min - max]: 39 years, 18 - 79). Respondents placed most importance on reducing HAE attack frequency (from a 45% to 90% reduction in attacks), followed by avoiding a 25% risk of a GI side effect, moving from injection to oral administration, less frequent dosing, and avoiding a 55% risk of ISRs. Reducing the time to onset of action from 10 weeks to 1 week was least important. On average, respondents would accept >50% ISR and >25% GI risks for large (40%) reductions in attack frequency, and 19.3% ISR and 7.6% GI risks for smaller (5% reduction in attack frequency) improvements at high levels of efficacy.
CONCLUSIONS: These findings help elucidate which attributes of long-term prophylactic HAE treatments are most important to patients and may help inform shared decision-making between patients with HAE and their physicians.
