New approaches to the design of clinical trials in idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis (IPF) is one of the major challenges for respiratory medicine since prognosis is particularly poor and few therapeutic options are available - in fact, at present to the only approved drug is pirfenidone. Winning this challenge will be based on successful design and completion of randomized clinical trials. The last decade witnessed an unprecedented increase in quality and quantity of trials in IPF: nonetheless, most have been negative and potential obstacles did emerge. In particular, the choice of the best endpoint, i.e. clinically meaningful and feasible at the same time, and the management of missing data still represent issues not fully resolved. The increasingly competitive environment and the heterogeneity in approach by different regulatory agencies also need to be considered. In the next few years more and more trials will be designed and completed, in the hope of taking quicker and safer treatments to IPF patients.