‘We have achieved something unprecedented for an ultra-rare disease,’ Henrik told the assembled patient advocacy leaders at the beginning of the 2024 HAEi Global Leadership Workshop, with eight treatments approved and new options being studied. However, Henrik recognized that this access was not spread evenly, with patients in many countries only having access to anabolic steroids and tranexamic acid. This workshop, he said, is designed to win the fight for access by equipping every participant with the tools and skills needed.
A vital part of winning the fight for access is equipping every participant with the tools and skills needed. Henrik stated the first step was knowing what was out there and turned to Tony Castaldo for a review of currently available and forthcoming therapies.
Tony began by reminding all present they are an advocate and their goal is to improve the quality of life for people with HAE. The route to this is winning access to and reimbursement for modern HAE medicines. A crucial element of advocacy, Tony counseled, was to ‘be aggressive.’ He called on all patient advocates to interact with pharma company representatives to make sure their voices were heard and to not be afraid to ask for commitment and support.
Tony outlined the medicines available, noting that information can vary by country and that you should consult the prescribing information for your country for specifics.
>> Find a list of licensed medicines on haei.org
Looking to the future, Tony described the ‘astonishing level of pharmaceutical company interest in the next generation of HAE treatments,’ which resulted from ‘our motivated and united HAEi community’ and the consistent and proactive role in Navigating the Future.
Tony then presented information on the six companies and medicines being tested, which called for some very challenging pronunciations.
>> Find the list of HAE treatments in development on haei.org
Tony closed the session by praising the unselfish work of all who pave the way for a brighter future by participating in clinical research and the can-do attitude of our community.