Pitfalls in developing new compounds for idiopathic pulmonary fibrosis.
The development and availability of nintedanib and pirfenidone has heralded a new era in the management of idiopathic pulmonary fibrosis (IPF). Both agents demonstrate that the disease can be successfully modulated with therapeutic interventions, but neither are a cure and IPF remains a deadly disease.
There have been many lessons about the natural history of IPF and clinical trial design, not only from the clinical development programs for nintedanib and pirfenidone, but also the numerous negative trials that predated these.
In this review, we attempt to synthesize how the field of IPF and clinical trials has evolved, the lessons learnt, and how these might inform and enable more efficient future clinical trial designs that are primed for success.