Mar. 11 at 11:24 AM
$PHVS Pharvaris reports results from study of patient experience during HAE attacks
Pharvaris announced that results from a Pharvaris-sponsored non-interventional, mixed methods, real-world study assessing the patient experience during acute HAE attacks across alternative endpoints used in clinical studies have been published in Clinical Reviews in Allergy & Immunology. The study evaluated the patient experience with HAE attack manifestations, and what constitutes meaningful changes in those manifestations, to support the inclusion of meaningful endpoints in clinical studies for on-demand treatment, or ODT, of HAE attacks, such as RAPIDe-3. Although approved medicines exist for the on-demand treatment of HAE attacks, there have been no head-to-head studies of on-demand therapies, and cross-trial comparisons have been challenging due to lack of uniformity in study design.
Additionally, generation of evidence related to the key symptoms experienced by people during HAE attacks, insights into the subsequent perception from patients as to what are clinically-meaningful changes in symptoms, validation of the content of various patient-reported outcome, or PRO, instruments to support the evaluation of those symptoms, and confirmation of patient understanding and interpretation of that content, all could help in the evaluation of on-demand therapies by regulators, payers, and the HAE community. This mixed methods study provides strong evidence that the PGI and AMRA instruments are reliable, valid, and sensitive tools for assessing PROs in HAE attacks. Within the PRO assessments, most concepts were considered important by participants.
Qualitative interviews confirmed that even relatively small improvements could be meaningful, such as the moment an attack was no longer worsening, as captured by the endpoint of End of Progression. Consistency across all PRO assessments of attack manifestation and response assessment were seen; End of Progression was the first-in-time achieved endpoint, followed by symptom relief, then symptom resolution. Importantly, the median time to onset of initial symptom relief as measured as PGI-C rating of at least "a little better" aligned more closely with the timing of AMRA-3 greater than or equal to20% than with the timing of AMRA-3 greater than or equal to30%. These results informed the hierarchy of endpoints in the Phase 3 RAPIDe-3 trial of deucrictibant and may also inform future trials of ODTs for HAE attacks.
