Make a difference – partnering for progress – patient-driven and targeted research

Debs Corcoran, HAEi Director, Research, and Tony Castaldo, HAEi CEO and Chairman of the Board, discussed the advocacy research work HAEi supports to further the ambitions of Member Organizations (MOs) to secure real-world data, which is so crucial to winning access to and reimbursement for HAE medications.

Tony outlined that, in the US and internationally, it is clear that while emotive personal stories have the power to engage people and encourage action, they need to be accompanied by hard evidence from data to demand action from health officials and bureaucrats.

Debs added that while patient-driven research may sound daunting and complicated, in reality, it is very simple. HAEi’s team handles all data collection, analysis, and development, providing MOs with a compelling evidence package to convince health officials and insurers. The three research programs currently offered are:

1. Heat map survey

The heat map survey, Debs and Tony explained, is used in countries where few people are currently diagnosed with HAE. The study aims to identify pockets of people who have symptoms that could be HAE, so that education and awareness campaigns can be targeted in those areas or regions. Ultimately, this means more patients diagnosed with HAE and increases the priority that health services give to HAE.

The survey is underway in Egypt, and the fieldwork has just ended in Bangladesh. Additionally, a pilot project in Brazzaville, Republic of the Congo, has adapted the original premise to suit local needs, with teams of volunteer researchers visiting local doctors to share a video about HAE and ask some Heat Map-style questions to identify potential patients.

2. Baseline burden of illness study

In countries where there’s an MO but limited or no access to modern HAE therapy, the baseline burden of illness study translates patient experiences into hard data. It quantifies the unmet need in terms of attacks, emergency visits, hospital stays, and patient quality of life, and can then be used to argue for better care.

So far, HAEi has conducted this program with MOs in India, Mexico, and South Africa, where they have successfully used the data in advocacy work. Future programs are in the works with the Philippines, Vietnam, Thailand, China, and more.

3. Pharmacoeconomic, socioeconomic, and quality of life assessment

The third research resource supports MOs where modern HAE medicines are available and where they are seeking to improve access or retain the access they have. Tony and Debs outlined that data from this study show societal and personal benefits to having HAE-specific medication available.

The US HAEA first published data from this type of survey in March 2021. Since then, HAEi has worked with the United Kingdom, Nordic countries, Australia, New Zealand, Germany, Austria, and Switzerland on this survey.

HAEi State of Management Report

Another HAEi resource that started HAEi’s commitment to real-world evidence is the State of Management series. Drawing on the knowledge and experience of the HAEi MO leaders, it brings together the HAE management reality in a series of countries. There is a State of Management report for Latin America and two for Europe, published on the HAEi website. These are soon to be joined, Debs said, by one covering HAEi’s 25 MOs in the European Union, and another for the 13 countries of the South Eastern Europe region.

These studies are just one example of the HAE community’s commitment to supporting research.

‘HAEi absolutely cannot conduct research without you. Thank you very much to everyone.’

– Debs Corcoran, HAEi Director, Research

Targeted research

These studies represent the bulk of the research team’s work, but, as Tony said, HAEi is always looking for new ways to give people with HAE an edge.

In a new development, Tony announced the launch of an initiative called Targeted Research as part of HAEi’s Advocacy Research work. To help explain further, Beverly Yamamoto, HAEi’s new Coordinator, Targeted Research Projects, joined Tony and Debs onstage. Working alongside Debs, a pilot project is underway in Pakistan to create HAE management guidelines tailored to the country’s specific needs and situation. This project has led to another, in Malaysia, which uses the same methodology: Desk research identifies HAE knowledgeable clinicians who can work together, with support from international experts, to develop an expert consensus on HAE management in their country.

Although these are country-specific, targeted guidelines, designed to be practical and to ensure patients receive the best available care, the aim is to expand horizons and always keep in mind the aspiration of international guidelines to normalize life for people with HAE.

In addition to the 11 doctors involved in Pakistan and 16 in Malaysia, the plan is to involve medical societies and universities to gain their endorsement, and to pursue an ambitious timeline for journal publication by the end of 2026.

So, whether it’s an MO just starting or an established community looking to protect hard-won access to medication, the HAEi Research team has the time, tools, and expertise to help. “Please, please be in touch if you want more information about how our work might be able to support you,” Debs told the participants.