By Michal Rutkowski, Regional Patient Advocate for Central and Eastern Europe, Gulf Region & Middle East:
The penultimate weekend of October 2019 saw the largest gathering of the year of the HAE International Central Eastern Europe community. As has become the tradition, the capital city of Poland hosted for the fourth consecutive time the annual regional advocacy event, dedicated to patients, caregivers and physicians, who constantly have been working in the areas of awareness, diagnosis, access and reimbursement to make HAE patients’ lives better and easier. The workshop was also a great opportunity to meet and greet the newcomers to the HAE International world as well as current members of the global HAE family.
The meeting gathered patient’ advocacy leaders from Poland, Armenia, Belarus, the Czech Republic, Estonia, Georgia, Hungary, Kazakhstan, Lithuania, Russia, Slovakia and Ukraine, accompanied by patients and caregivers from Belgium and the Netherlands. The theme of the 2019 Workshop was “Importance of patient advocacy: knowledge as the key to success”.
The 2019 keynote speakers for the regional workshop were:
- Professor Henriette Farkas, MD, PhD, DSc, Director of the Hungarian HAE Center at Semmelweis University in Budapest,
- Professor Marc Riedl, MD, MS, Clinical Director of the US HAEA Hereditary Angioedema Center at UCSD in San Diego,
- Marcin Stobiecki, MD, PhD, Head of the Polish National HAE Center at the University Hospital in Krakow,
- Tricia Mullins, Forward thinking Patient Advocacy, Access, Engagement & MedEd Leader,
- Fiona Wardman, HAE International Board of Directors / Treasurer / Chief Regional Patient Advocate / Regional Patient Advocate Asia Pacific,
- Ole Frölich, HAE International Enterprise Technology Manager.
The Workshop topics were divided into four break-out sessions focusing on Advocacy, Scientific, Awareness and Clinical research.
Break-out Session 1 (Advocacy) was an introduction to the subject of patient advocacy:
- Fiona Wardman explained who HAE International is and what value the organization brings to the HAE community: patients, caregivers and physicians. Also, she informed attendees about the currently available projects and programs distributed by HAE International to its member organizations, and she made the participants more familiar with HAE International’s structure,
- Ole Frölich focused on advanced technology that enables patient organizations to grow quickly and easily with the help of HAE International hosted websites and HAEi Connect. These two online platforms were detailed explained by Ole, who was also available during the time of the meeting to help setting up websites for the member organizations,
- Tricia Mullins had an inspirational and motivational presentation on what a patient advocate is, how to become a patient advocate, and how the patient advocacy organization can help other HAE stakeholders. A patient herself, Tricia shared her multiyear experience in the field of increasing awareness and helping patients with rare diseases.
Break-out Session 2 (Scientific) was a display of knowledge of two HAE experts, who in words understandable to patients explained important issues related to HAE:
- Henriette Farkas presented the specificity of the disease in children and women based on data from clinical studies and her own clinical practice. Henriette highlighted similarities and differences in symptoms, attacks frequency, and trigger factors. Also, she focused on pregnancy and the best available treatments for different types of patients,
- Marc Riedl gave a real-time travel to the past, present and future of HAE therapy, going through the pathophysiology of HAE and medicine availability over the years. Marc explained current management guidelines and acute treatment recommendations. He also discussed topics related to preventive treatment.
Break-out Session 3 (Awareness) belonged to the leaders of patient organizations who presented the current state of management of HAE in their countries. The CEE region is very diverse in terms of diagnostic options as well as access to and reimbursement for modern HAE therapies.
It should be emphasized that the presentations delivered by the speakers were preceded by detailed surveys completed by HAE patients respectively in their home countries. Nearly 360 patients from 13 countries took part in the survey to help collect the data used in the presentations. This allowed gathering extremely reliable information on the needs, expectations and fears that patients are struggling with in everyday life.
Break-out Session 4 (Clinical researches) was the last workshop session during which participants could listen to the importance and impact of clinical studies on HAE patients based on Polish experiences. The meeting ended with a 30-minute Questions & Answers panel in which attendees had the opportunity to get along with detailed expertise of physicians present and broaden their knowledge on HAE:
- Marcin Stobiecki presented the essence of clinical trials, using the story of one HAE patient who has been participating in clinical studies for nearly 40 years and who was the first Polish patient ever to receive C1-Inhibitor. Marcin’s presentation was also a kind of story showing what patients and physicians from countries formerly behind the Iron Curtain have had to face,
- Professor Farkas, Professor Riedl and Dr Stobiecki were heavily exploited but patiently and in detail answering the attendees’ questions. The range of questions was varied. From the basic ones related to the diagnosis to more complex regarding children treatment and potential consequences of overdosing the HAE medications.
All participants emphasized that the fourth edition of the HAE International Central Eastern Europe Workshop was the best meeting so far. Very good quality venue, world-class HAE experts, motivated patients, caregivers and physicians, ubiquitous kindness, friendship and passion influenced the quality and success of the event.
As always during the meetings run by HAE International, there were also representatives of the pharmaceutical industry from (in alphabetical order) BioCryst Pharmaceuticals, CSL Behring, KalVista Pharmaceuticals, Pharming Group NV, and Takeda, who search for a new treatment options for patients and who support patient advocacy activities organized under the leadership of HAE International.
I would like to thank all the attendees of this year’s Workshop for their active participation in the meeting. Through a common goal, mutual respect and understanding, but above all unconditional friendship, we have been able to create over the years something truly unique that we should be absolutely proud of.
Central and Eastern Europe is a very special region, which, despite many differences, also has plenty of common features. This is a region with a lot of room for improvement, just to say that about 75 to 80 percent of HAE patients are still undiagnosed. And that is where the patient advocates’ role begins.