By Anthony J. Castaldo, HAEi President & CEO

Access to modern HAE medicines is the key to improving our community’s quality of life. HAEi’s experience working with member organizations shows that having robust and reliable real-world data is a key component winning approval for a therapy. HAEi conducts targeted, scientific based research designed to provide data that our member organizations can use in their advocacy efforts to convince Health Ministries to approve HAE therapies. 

HAEi has three core methodological approaches for patient-centric HAE studies that we conduct for our member organizations: Heat Map Survey, Pharmacoeconomic, Socioeconomic and Quality of Life Assessment, and Baseline Burden of Illness Study.

Heat Map Survey

HAEi designed our Heat Map Survey methodology to be used for countries where there are few, if any people diagnosed with HAE. The objective is to identify “pockets” of potential HAE so that we can pinpoint areas to concentrate HAE education and awareness campaigns. The Heat Map Survey is on-hold in Rwanda due to COVID-19, and we are looking at Thailand, Bangladesh, and the Philippines as study candidates. 

Pharmacoeconomic, Socioeconomic and Quality of Life Assessment

Our Pharmacoeconomic, Socioeconomic and Quality of Life Assessment studies are targeted for countries that already have access to modern HAE therapies. At present, we have completed these studies in the US, the United Kingdom, and the Nordic countries. We are also initiating studies in Australia and New Zealand. 

The results of the pharmacoeconomic study conducted in the US were published in March 2021 in the prestigious medical journal Proceedings of Asthma and Allergy.

Baseline Burden of Illness Study 

Baseline Burden of Illness study is conducted in countries where a patient organization exists, but no modern HAE therapies are yet available.  Surveys are used to quantify the number of attacks, emergency room visits, hospitalizations, and patient quality of life. This study provides data that opens the door to approaching health ministries with real world evidence of a catastrophic unmet medical need.  We have conducted the study India and are currently discussing plans for China and several other countries.