The joint audience of healthcare professionals, patient advocates, and pharmaceutical industry representatives welcomed HAEi’s Chief Scientific Officer, Debs Corcoran, onto the stage. She and Tony Castaldo wanted to tell people about the power of patient-driven research.
Tony set the scene by reminding everyone that while emotive patient stories have power, for decisions makers who decide on access to HAE medicines are motivated and convinced only by data and real-world evidence of disease burden and benefit of treatment.
As a result, HAEi is at the forefront of producing data to support this decision-making, and Debs is in charge of all these projects.
Debs told the audience: ‘Research could sound complicated, but in reality, it means that we bring together the data with your support. We do the analysis, and it’s all to give you our MOs the evidence to convince health ministries or insurers.’
HAEi offers three core studies to MOs, these are:
- The heat map survey
- Baseline burden of illness study
- Pharmacoeconomic, socioeconomic, and quality of life assessment
Tony asked Debs to tell the audience all about the heat map survey. She told the audience that the heat map survey:
- Is for countries where there are few people diagnosed with HAE
- Helps to identify pockets of people with symptoms of chronic angioedema so that you can effectively target HAE education and awareness
- Leads to more diagnosed patients and helps increase the priority health services places on HAE
- Has already been done in the Philippines, with further studies planned in Thailand, Vietnam, Bangladesh and the Republic of the Congo
Moving on to the baseline burden of illness study, this is for countries where there are patients with HAE but little or no access to modern HAE medicines. Debs took up the job of explaining in more detail. ‘This study turns patient experience into evidence of unmet need,’ she told the audience.
The survey seeks to quantify:
- Number of attacks
- Emergency room visits
- Hospitalizations
- Patient quality of life
Debs told them that the end product is evidence to convince your health service that people with HAE need better care. This study has been conducted in India, Mexico, and South Africa. Work is ongoing to bring this study to the Phillippines, Vietnam, Thailand, and China.
As evidence of success, the Mexican data was used in a presentation to politicians, leading to significant benefits for HAE patients. Similarly, in South Africa, the data was accepted as an oral presentation at a rare disease conference (when HAE South Africa submitted it for only a poster!).
Finally, the pharmacoeconomic, socioeconomic, and quality of life assessment analysis.
Here, Debs highlighted two main aims. First, to quantify the overall economic value of modern HAE medicines, such as the ability to work, learn, and care for families. The second was to show the often dramatic quality of life improvements for people with access to modern HAE medication.
This study was completed in the US, Nordic countries, Australia, New Zealand, and the United Kingdom. Projects are ongoing in Germany, Austria, and Switzerland.
The goal is always to have the data published in a medical journal to maximize its visibility and credibility; it was an outstanding achievement that we published the US PE data in March 2021.
‘We need to have a seat at the table; having publications is a surefire way to get that.’
– Tony Castaldo, Chief Executive Officer, HAEi
Debs and Tony said that the State of Management reports add a fourth dimension to our patient-driven research. This research asks leaders of MOs to provide a snapshot of:
- Medication availability and reimbursement
- Government health priorities
- Challenges faced by people with HAE in their country
HAEi published the reports on the HAEi website, and additional State of Management surveys are now in-process.
Debs asked every advocacy leader to think about how they could get involved in research. Interested readers can contact Debs via d.corcoran@haei.org
‘We cannot conduct research without you, and we cannot conduct research without the people in your organization.’
– Debs Corcoran, Chief Scientific Officer, HAEi
