ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

European Cystic Fibrosis Society   257  OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS PATIENTS’ PRIORITIES AND THE CF PHARMACEUTICAL PIPELINE CHAPTER 14 Table 4 : Shared research priorities across the different priority setting exercises [8] IPaCOR prioritized research ideas from gaps that were identified in Cochrane reviews, and may be finalized through translational medicine approaches that bridge the clas- sical “bench-to-bedside” gap. The NCFS group used a combination of focus groups and surveys to suggest topics felt to be important, and have changed these topics as the program has evolved. 3 The role of clinical trials networks in accelerating the pharmaceutical pipeline. Clinical trial networks exist to facilitate and accelerate clinical research trials with the aim to speed up the process of bringing new medicines to patients. Examples of these include the US CFF CF thera- peutics development network (CFF-TDN) launched in 1998 and the ECFS Clinical Trials Network (ECFS-CTN) which has been active since 2008. The ECFS-CTN currently provides access to 58 large and experi- enced CF centres, located in 17 different countries throughout Europe. Clinical trial networks will be discussed in further detail in Chapter 15. Trial networks should work closely with the CF patient community to ensure that their priorities are aligned. They should also balance the motivations of the pharmaceutical industry and the curiosi- ty-driven science of academic investigators against the priorities of the patient commu- nity, derived from their experience of living with CF [9]. Priority area JLA PSP rank CCF rank IPaCOR rank NCFS 2016 Treatment burden 1 3 5 Gastrointestinal 2 4 Exercise 7 8 Respiratory microorganism detection/ treatment 3,10 1 3,6,13 CF related diabetes 5 5 14 Mental health 2 Therapies targeting basic defect 1 Only priority

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