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

Author： Cerri Stefania Del Giovane Cinzia Balduzzi Sara Soncini Francesco Sdanganelli Antonia Richeldi Luca

ISSN： 2041-6792

Source： Clinical Investigation, Vol.3, Iss.6, 2013-06, pp. : 531-544

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Abstract

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 the only approved drug is pirfenidone. Overcoming this challenge will be dependent on successful design and completion of randomized clinical trials. The last decade witnessed an unprecedented increase in quality and quantity of trials in IPF; however, most have been negative and potential obstacles have emerged. In particular, the choice of the best end point; that is, one that is both clinically meaningful and at the same time feasible, as well as the management of missing data still represent issues are not fully resolved. The increasingly competitive environment and the heterogeneity in approach by different regulatory agencies also need to be considered. During the next few years, more and more trials will be designed and completed in the hope of developing quicker and safer treatments to IPF patients.