Cystic Fibrosis in Belgium: Its Challenges and Its Future
Cystic Fibrosis (or Mucoviscidose) is an incurable life-threatening disease that affects over a thousand persons in Belgium. Ken De Marie helps us understand the disease, the challenges it poses to patients, and the latest advancements in his cure. Ken works for the Belgian Cystic Fibrosis association, where he tries to find solutions for Cystic Fibrosis related financial problems that patients can encounter. He also helps with lobbying efforts towards policymakers
What is Cystic Fibrosis?
Cystic Fibrosis (also known as Mucoviscidose or muco) is the most common life-threatening hereditary disease in Belgium. More than 1360 patients are known in our country. The most common symptoms are recurring respiratory infections, mucus that is hard to evacuate through coughing, stomach pain, and other digestive problems. Cystic Fibrosis doesn’t only have an impact on the lungs, but it can also affect several other organs. The disease is very complex and can be rather heterogeneous in the way it can manifest itself in patients. As of yet, Cystic Fibrosis remains incurable.
What’s the current situation of Cystic Fibrosis and its patients in Belgium like? Where is it heading? What would you like to see happening for them in the future?
Cystic Fibrosis is an incurable disease which requires intensive therapy. Patients manage to control their symptoms through daily aerosols, physiotherapy sessions, the use of medicines and various other treatments. Nevertheless, Cystic Fibrosis is a progressive and life-shortening disease causing patients to pass away prematurely or needing a lung transplantation. Up until a few years ago the therapy was purely symptomatic (it eased the symptoms without dealing with the cause of the disease).
Recently new medicines came on the market that are disease-modifying. They address the underlying cause of the disease and improve lung function, lower the number of hospitalisations that require antibiotics, etc. These so-called Cystic FibrosisTR-modulators are a true medical revolution, but they are expensive and therefore face a number of challenges with regards to their reimbursement. We are working to make these Cystic FibrosisTR-modulators available for people with Cystic Fibrosis.
In the future I would like to see the burden of treatment and the impact of the disease on patients’ lives become much lower, but of course my biggest wish is that, thanks to the continuing efforts of researchers worldwide, an actual cure for Cystic Fibrosis can be found.
Which changes would you like to see happening?
Patients should be in the center of their data collection, owning their data and also co-deciding which data is collected. Up till now, only clinical data was collected, and patient-reported outcomes were only collected through outdated quality of life (QoL) measures. In the context of disease-modifying therapies, and to put patients at the heart of their own care, variables that really reflect their health and feelings need to be collected. Also, data collection in between clinical visits (real-world data collection) will lead to better care and potentially broader access to innovative treatments.
What brings you to Hack Healthcare?
The Flemish umbrella association for patient organizations (VPP) contacted me with the proposal to participate in this event. Since I was interested in the We-Are-Health project in which they are involved, I gladly accepted. We-Are-Health focuses on empowering patients with regards to the use of their personal data.
Moreover, the current covid-19 crisis was a wake-up call for a lot of patient organisations about the importance of keeping up-to-date with technological advancements. In a few months time, remote consultations and digital meetings became a part of our lives. This is also a theme that is being addressed by Hack Healthcare.
Which Hack Healthcare themes and challenges interest you the most and why?
Telemedicine is an important development that proved its usefulness in recent times. Furthermore, for people with Cystic Fibrosis sports is not just a past-time, it’s an integral part of their treatment. Anything encouraging regular sports or fitness activities is therefore potentially useful. And last, but certainly not least, rare disease data. The collection of real-world data can help us understand more about the actual impact of new therapies in our daily lives. Due to small sample sizes, research about Cystic Fibrosis is often confronted with a number of limitations. Real world data can potentially provide us with interesting findings and more answers.
What would you like to see coming out of Hack Healthcare
I think it’s important for developers of technological solutions in healthcare to realise that it’s necessary to involve patients in the early stages of development. Patient organisations can make an important contribution in this regard. I hope Hack Healthcare will further underscore the importance of early patient-involvement.
About Muco, the Belgian Cystic Fibrosis Association
Muco is the national Belgian Cystic Fibrosis Association. It strives for a better and longer life for all people with Cystic Fibrosis in Belgium. We try to achieve this by supporting patients and their families by means of providing information, lobbying, financial support and financing scientific research.