The curse of idiopathic

Excerpt

The anti-fibrotic drug pirfenidone has proven efficacy in slowing lung function decline in idiopathic pulmonary fibrosis (IPF). The registration studies were designed to include patients with IPF and to exclude patients with pulmonary fibrosis associated with other disorders. Although the patients investigated in these studies comprised a pathogenically heterogeneous cohort, Food and Drug Administration (FDA) approval of pirfenidone applies only to patients deemed to have idiopathic causes for their pulmonary fibrosis. Consequently, use of pirfenidone for non-idiopathic causes of pulmonary fibrosis is considered off-label, subject to prescribing liabilities and insurance denial. …