Drug Regulators, EMEA, Publish Guidance on the Development of therapies for Cystic Fibrosis
full text here
CF is a life threatening and chronically debilitating disease, impairing the Quality of life, characterised by progressive bronchiectasis and obstructive pulmonary disease (> 90% patients). The lower respiratory tract involvement is the primary cause of morbidity and mortality in patients with CF (> 90% of fatalities). Ninety percent of CF patients are colonised with Pseudomonas aeruginosa (PA), and PA infections are the cause of mortality in 80% of those patients.
CF is mainly a paediatric disease. Life expectancy is considerably shortened due to respiratory damage-induced morbidity and mortality. However, due to the advances of past 20 years in the prophylaxis of chronic respiratory infection, along with the systematic supplement of pancreatic enzyme and decrease in malnutrition CF patients reach a mean 30 to 40 years of age, and the proportion of adult CF patients increases.
The guidance will focus on:
• clinical trials design in lung involvement and clinically relevant endpoints (mucociliary clearance and broncho-pulmonary infections (mortality), in particular: treatment of PA early colonisation, chronic infection and exacerbations; prophylaxis of chronic PA infection; slowing/stopping lung damages (fibrosis, bronchiectases)
• clinical trials design in pancreatic involvement, in particular management of exocrine pancreatic insufficiency and malnutrition.
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