People with Cystic Fibrosis (CF) have their own views on scientific research into the disease that affects their airways and digestive system. When asked what kind of scientific research they consider important, projects emerge with great impact for those patients themselves, observes Jacquelien Noordhoek, director of the Dutch Cystic Fibrosis Foundation (NCFS). She received her PhD from Utrecht University today. 

Cystic Fibrosis is a rare hereditary disease. In the Netherlands, 25 to 30 babies are born with this disease every year. The complaints differ per person, but respiratory complaints, intestinal complaints and growth retardation are common. Worldwide research has led to major breakthroughs and recently there are effective medicines to combat the complaints. Unfortunately, not all variants of CF can be treated with these medicines. Moreover, there is still no treatment to cure people with CF. About 1650 people in the Netherlands have CF. The life expectancy of these people is about 50 years.”

Researcher Jacquelien Noordhoek became director of the Dutch Cystic Fibrosis Foundation (NCFS) in 1998. 'In the early days, I noticed that the NCFS did get involved in research, but at a late stage: we were asked to sign a letter of support for a grant application, or a patient was allowed to sit at the table of a research committee, who could talk about his experiences. The primary curiosity of the researcher, the department or the university was decisive. The question of patient needs came later and was developed and mapped in a structured way by the NCFS. 

Participation 

'It is not common for physician-researchers to structurally explore what questions patients would like to have answered; what they consider important within scientific research. That's where people with CF could really improve,' the researcher said. The NCFS takes a sustainable approach to patient participation. This started with a successful 5-year cycle and a second and a third cycle followed. Among other things, this thesis describes the conditions a research agenda prioritised by patients must meet.

'Part of my dissertation is also about how the quality of care can be improved when care is assessed from the patients' perspective. The NCFS has invested in this in several ways. By fulfilling our role in an active and professional way, medical centres in the Netherlands know how to find us and make use of our knowledge and experience.'

Coaching for parents

In collaboration with CF patients, quality criteria also emerged. Quality of care is often measured by goals that are important to doctors. The question from the NCFS is: What does the patient consider important and how can we use this information to improve care? 

More customised psychosocial support was provided for parents who have just learned of their child's CF diagnosis. We offer these parents a coaching programme during a hectic period in which they experience what changes when your child has a serious illness. Help that fits seamlessly with the needs of parents.'

Jacquelien Noordhoek sees that patient organisations can realise more impact by professionalising. 'The role of volunteers is of enormous importance and, in addition, I see the added value of professionals. To raise funds and expand our international network, run a disease-specific registration, and enable new research, the use of experts is something I think we will continue to focus on in the future.'