Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials.

Published

Journal Article

Definitive evidence of clinical efficacy in a Phase 3 trial is best shown by a beneficial impact on a clinically meaningful endpoint-that is, an endpoint that directly measures how a patient feels (symptoms), functions (the ability to perform activities in daily life), or survives. In idiopathic pulmonary fibrosis (IPF), we believe the endpoints that best meet these criteria are all-cause mortality and all-cause nonelective hospitalization. There are no validated measures of symptoms or broader constructs such as health status or functional status in IPF. A surrogate endpoint is defined as an indirect measure that is intended to substitute for a clinically meaningful endpoint. Surrogate endpoints can be appropriate outcome measures if validated. However, validation requires substantial evidence that the effect of an intervention on a clinically meaningful endpoint is reliably predicted by the effect of an intervention on the surrogate endpoint. For patients with IPF, there are currently no validated surrogate endpoints.

Full Text

Duke Authors

Cited Authors

  • Raghu, G; Collard, HR; Anstrom, KJ; Flaherty, KR; Fleming, TR; King, TE; Martinez, FJ; Brown, KK

Published Date

  • May 15, 2012

Published In

Volume / Issue

  • 185 / 10

Start / End Page

  • 1044 - 1048

PubMed ID

  • 22505745

Pubmed Central ID

  • 22505745

Electronic International Standard Serial Number (EISSN)

  • 1535-4970

Digital Object Identifier (DOI)

  • 10.1164/rccm.201201-0006PP

Language

  • eng

Conference Location

  • United States