Idiopathic pulmonary fibrosis: from clinical trials to real-life experiences

Abstract

Randomised controlled clinical trials are fundamental in medicine to develop new effective drugs and new therapeutic regimens and are the strength of evidence-based medicine. These studies allow us to avoid the repetition of misleading experiences that have been reported in the past, where drugs or associations were utilised without compelling evidence and ultimately proven to be ineffective. In recent years, randomised clinical trials have been conducted and concluded for many rare diseases, including idiopathic pulmonary fibrosis. However, clinical trials do not always reflect the real-life scenario. Patients selected for clinical trials present fewer comorbidities, they fall between certain age limits, and the severity of their disease is defined; therefore, they do not always reflect the whole of the population affected by a specific disease. These are the reasons why we also need data that mirror real-life experience. The limitations that these kind of studies present are always several and the studies should be interpreted with caution, although they can fill the important gap between efficacy and effectiveness. In this article, we will review the existing clinical data on real-life treatment of idiopathic pulmonary fibrosis. @ERSpublications Real-life studies are useful in confirming the experimental data obtained from randomised controlled clinical trials http://ow.ly/PYUCC

A Real-Life Multicenter National Study on Nintedanib in Severe Idiopathic Pulmonary Fibrosis

“…Such a population has generally been excluded from randomized controlled trials. Our study has the typical pros and cons of real-life studies already discussed in previous papers [14, 16]. It is at the same time a national multicenter retrospective analysis truly representative of the real situation of patients affected by a more severe form of IPF.…”

Treatment of idiopathic pulmonary fibrosis: a position paper from a Nordic expert group

“…The ASCEND study was more rigorous with regard to patient inclusion criteria as it did not allow marked emphysema (extent of IPF should be greater than that of emphysema in HRCT) or airway obstruction. In smaller observational studies , the treatment effect of pirfenidone was stronger in patients with progressive IPF compared to those who were considered stable.…”

Role of pirfenidone in the management of pulmonary fibrosis

“…Because pirfenidone had already been approved for prescription in many countries outside of the US prior to FDA approval, “real world” experiences with pirfenidone in other countries have appeared in the literature and report that the drug is relatively well-tolerated and appears to benefit a substantial number of patients 44,45. Recent publications also support safety and efficacy of pirfenidone for the treatment of IPF.…”