ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

256    www.ecfs.eu OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS PATIENTS’ PRIORITIES AND THE CF PHARMACEUTICAL PIPELINE CHAPTER 14 In a further ongoing study, the same group is examining how to choose a set of outcome measures to assess the validity of the results from clinical trials from the perspective of patients and stakeholders. Preliminary observations point to rather low concordance between the scoring given by health professionals and by stakeholders. Despite these limitations, this should repre- sent an important step toward the defini- tion of a core outcome set to address the patient-centeredness of clinical trial results. 2.4. Dutch Cystic Fibrosis Foundation The Dutch NCFS has been working for the past 12 years to try to align its research programme with the priorities of its patients [5]. In 2007, 15 people with CF and 15 parents were asked to specify research ideas that were personally important to them. The 30 people then prioritized their top 10 research ideas and then, similarly to the JLA project, these were ranked online by almost 200 Dutch people with CF who answered the Dutch registry’s invitation to participate. The top 5 were then agreed with clinicians and scientists. These topics were: insight into the basic defect, infec- tion, inflammation, segregation policy, and psychosocial issues. This set the NCFS research agenda for 2007-2011 with several projects funded to try to address these priorities. In 2012 the NCFS reviewed the priori- ties using a similar method. The priorities remained unchanged except for segrega- tion policy being replaced by fatigue in CF. However the impact of this research agenda was subsequently judged to be disap- pointing as the funded projects were highly diverse, with little promise of follow-up work, so a new approach was taken. A specific consortium of basic scientists, clinicians and patient representatives was set up who designed a research program called “HIT CF” with the aim to address the top priorities in 2012 of targeting the basic defect in people with CF, infection, and inflammation. Patients and parents of patients were involved in review of projects. This programme has brought in € 5 million of funding and resulted in many publications. The organoid technology, which is a key component of the programme, has been particularly successful. A further review of by patients and parents in 2016 resulted in the decision to now focus entirely on new therapies targeting the basic defect. 2.5. Comparing the patient priorities between these initiatives Table 4 , taken from an editorial in the Journal of Cystic Fibrosis [8], shows shared priorities across the different priority setting exercises. It is reassuring to see similar themes evolving throughout the various research priority exercises, adding validation to their processes. However, not all of these priori- ties will be of interest to the pharmaceutical industry. For example, research into strate- gies to reduce disease burden may be more suitable for funding by patient organizations or national research funding schemes. It is important to note the differences in objec- tives set out for each priority setting exer- cise; the JLA PSP aimed to prioritize ques- tions that could be answered by clinical research whereas the Insight CF exercise aimed to prioritize questions that could be answered through the CFF registry. The