HAEi Research in 2023

It has been a research and science-filled year for HAEi. We’ve been active with our research programs, have had more inquiries about our methodologies than ever before, delivered very successful scientific tracks at our Regional Conferences, and convened a unique healthcare professional Symposium.

Here, we focus on some highlights from 2023, including a first look into the HAEA/HAEi sponsored HAE with Normal C1-INH Symposium.

HAEi Research Methodologies: Baseline Burden of Illness

Our Burden of Illness survey has been conducted in Mexico and South Africa. This study provides data that opens the door to approaching health ministries with real-world evidence of a catastrophic unmet medical need.

Dr. Sandra Nieto from HAE Mexico has used the results of their Burden of Illness survey with great effect. Firstly, in a presentation to the Latin America Society for Immunodeficiencies (LASID) meeting in October in Mexico City. More recently, Dr. Nieto presented the data to the Congress of Deputies as part of an HAE Mexico event. The event was a great success, resulting in the Congress of Deputies declaring 16 May as National Hereditary Angioedema Day and agreeing on other important benefits for HAE patients.

Dr. Sandra Nieto said, “The study results mean we have a clear picture of the burden of people living with HAE in Mexico. We have been able to shine a light on this to doctors and, most importantly, to our Congress of Deputies, which makes decisions about healthcare in Mexico. The Burden of Illness study was taken into account by the Congress of Deputies when making their decisions.”

For HAE South Africa, the final results from the Burden of Illness study were used to prepare an abstract submitted for the Africa Rare-X Conference in February 2024. Rare-X is Africa’s leading conference on rare disease treatment and access, and brings together rare disease patients, dedicated healthcare professionals, and invested stakeholders. The abstract has been accepted, and Janice Strydom, CEO of HAE South Africa, will give an oral presentation at the conference.

Janice comments, “These results give us the baseline of the burden of HAE in South Africa right now. Before we had the results, we had a feel for what was happening, but now we have real-life numbers. The data helps show us where to target future awareness and education activities for the biggest impact. We’re delighted to be a part of the Rare-X Conference and share the experience of people with HAE in South Africa. To be accepted as oral, rather than poster, is an honor.”

HAEi Research Methodologies: Heat Map

HAEi designed our Heat Map Survey methodology 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. We have been working with several countries on the potential to implement Heat Map, and mention one example here.

Along with Michal Rutkowski, Regional Patient Advocate for Central Eastern Europe, Benelux, and the Middle East, we are working with Dr. Elham Hossny from Egypt to pilot the Heat Map project within her medical society in Egypt. There are currently only 44 people identified with HAE in Egypt, a country with a population of 110 million.

HAEi Research Methodologies: Pharmaco, socioeconomic, and quality of life assessment

These studies are targeted at countries that already have access to modern HAE therapies. The data
collected can be used with Health Ministries to advocate for continued access to modern HAE therapies.

In 2023, this study was run in Australia and New Zealand. In New Zealand, patients do not have access to prophylaxis; HAE Australasia can use the data to show the Government the benefits of funding modern preventative therapies. In addition, this study is currently being run in Germany, Austria, and Switzerland.

The HAEA/HAEi HAE with Normal C1-INH Symposium 2023

The US HAEA and HAEi realized that a decade had passed since the last supported symposium on the subject. With Co-Chairs Prof. Bruce Zuraw, Prof. Konrad Bork, and Prof. Marcus Maurer, the HAEA/HAEi team convened a 1-day symposium before the 2023 HAEi Regional Conference EMEA with the ambition of producing a new HAE with normal C1-INH scientific publication covering the latest science regarding this important subset of the HAE population.

At the start of the symposium, Anthony J. Castaldo, HAEi Chief Executive Officer and Chairman of the Board, thanked all the assembled experts, especially the three Co-Chairs, for agreeing to participate. He told the healthcare professionals present, “Well over a decade ago, the US HAEA began seeing a consistent number of patients joining after receiving a diagnosis of what was then called HAE Type-III. At that time, US HAEA had no way to answer the many questions we received from this subset of patients. Since then, thanks to HAEA and HAEi, multiple scientific meetings have addressed this lack of knowledge, and this new session will further advance the science to help this important subset of patients.”

The project involves 31 HAE healthcare professional experts from 16 countries worldwide and is split into three task forces. Three critical areas of science were covered: