ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

European Cystic Fibrosis Society   33  Authors Siân Bentley, Daniel Peckham, Carlo Castellani, Nicola Shaw Introduction The treatment of cystic fibrosis (CF) has dramatically improved over the past few decades with life expectance increasing to over 40 years of age in many countries [1, 2]. This change in clinical outcome reflects a proactive multidisciplinary approach to treatment, with a focus on early interven- tions to avoid disease progression. The international culture of clinical, scientific and patient collaboration in the field of CF has also been pivotal in driving an evidence- based approach to treatment and provided the foundation for translational research and drug development. High standards of care are essential if clin- ical outcome is to continue improving. This includes facilities for neonatal screening and systems and processes in place to ensure prompt and accurate diagnosis, delivery of care in a multidisciplinary center with appropriate expertise, and easy access to medications which will minimize disease progression and reduce CF related compli- cations. Therapeutic intervention in CF can be challenging due to the complex multi- system nature of the disease, variability in concordance and limited effectiveness of some drug therapies [3]. Treatment should ultimately aim to maximize quantity and quality of life for people with CF, while minimizing treatment burden. The pharma- cological management of CF care is particu- larly challenging due to the polypharmacy nature of prescribing, variability in doses and regimens schedules, drug interactions, frequency of side effects, drug accessibility issues and adherence to treatment. 1 Antibiotics Despite the growing number of drugs avail- able for the treatment of CF, there is often limited evidence to support interventions. Surprisingly this can relate to everyday practice, including routine administration of antibiotics. Treatment decisions are often made based on the best possible evidence in combination with clinical experience. A recent systematic review identified 148 gaps in the evidence base available when making treatment decisions for themanage- ment of CF [4]. Most of these evidence CHAPTER 2 Cystic fibrosis medication: present and future