Clinical trials in idiopathic pulmonary fibrosis: where we have been and where we are going

Idiopathic pulmonary fibrosis (IPF) is the most common and most lethal diffuse fibrosing lung disease, with an increasing incidence and a mortality rate that exceeds that of many types of cancer. At present, there is no effective standard treatment recommended by guideline documents. As such, the unmet medical need is high. Recently, several high-quality clinical trials, evaluating safety and efficacy of different novel molecules, have been concluded. The results have mostly been disappointing, although some compounds have shown promising results. In particular, pirfenidone seems to be the most advanced molecule for IPF treatment, having been approved in Europe, Japan and India. Nintedanib, a triple kinase inhibitor, has almost completed enrolment of Phase III trials, based on promising Phase II results. Randomized controlled trials still represent a valid choice for IPF patients, and their completion is critically important to achieving the ultimate goal of curing IPF. Future approaches will probably include the evaluation of currently available agents alone and in combination, the identification of novel drugs with pleiotropic actions, and trials with validated, weighted composite endpoints.