Validation of Patient-Reported Outcomes in the On-Demand Treatment of Hereditary Angioedema Attacks Published in Clinical Reviews in Allergy & Immunology

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• By validating patient-reported outcome measures in a real-world setting, we are ensuring that the experience and voices of people living with HAE directly inform how we assess new therapies in development

  60% confidence

• This study gives us greater confidence that clinical outcomes truly reflect meaningful benefits for patients. These results not only strengthen our clinical programs but also reinforce our commitment to advancing patient-centered care and providing oral alternatives that improve upon standard of care to people with HAE

  60% confidence

• By validating patient-reported outcome measures with rigorous evidence, we have supported the definition of clinical study endpoints and reinforced trust in the results of those studies, resulting in HAE treatment decisions ultimately being guided by patient experience

  60% confidence

• The findings support the use of PGI and AMRA instruments to evaluate endpoints in clinical trials for on-demand HAE treatments, including within our RAPIDe-3 study—the first-ever on-demand HAE study to be designed with prespecified endpoints being fully compliant with the Core Outcome Set recommended in the AURORA Consensus

  60% confidence

• 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

  60% confidence

• End of Progression was the first-in-time achieved endpoint, followed by symptom relief, then symptom resolution

  60% confidence

• The ability to truly understand the impact of meaningful symptom changes will not only support the development of novel treatments but provide additional evidence to clinicians and people living with HAE to inform decisions on the most appropriate treatment options for them

  60% confidence

• 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 ≥20% than with the timing of AMRA-3 ≥30%

  60% confidence

• 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

  60% confidence

• Most concepts (i.e., skin swelling, abdominal pain, difficulty swallowing, and voice change) were considered important by participants within the PRO assessments

  60% confidence