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Idiopathic pulmonary fibrosis (IPF) is a chronic fibrotic lung disease with increasing incidence; the median survival is only 35 months and as yet no therapy has been proven to prolong survival. Recent unexpected randomised controlled trial (RCT) results and the conflicting evaluations of drug efficacy by regulatory agencies when considering the approval of pirfenidone have emphasised that we remain in the first stages of both our understanding of disease-relevant therapeutic targets and in our ability to investigate these putative targets with well-designed RCT. Three phase III trials are, however, anticipated to report results in 2014 and there is cautious optimism that we may be entering an era of mechanism-based anti-fibrotic therapies proven by large RCT to modify disease progression. We must now address how to practically translate these therapies safely and with maximal efficacy from a homogenous RCT population to the ‘real-life' heterogeneous population of patients with IPF. The role of a formal multi-disciplinary team meeting in a specialist centre with expertise in IPF is key to this. New methodological and ethical research challenges will arise as we enter an era of potential combination therapy; standardized, robust RCT design will be central to meeting these challenges if we are to enable ongoing progress in our aim of increasing both the length and quality of life of patients with IPF.
Jones et al. (Tue,) studied this question.