Skip to main content

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

Publication ,  Journal Article
Raghu, G; Collard, HR; Anstrom, KJ; Flaherty, KR; Fleming, TR; King, TE; Martinez, FJ; Brown, KK
Published in: Am J Respir Crit Care Med
May 15, 2012

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.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Am J Respir Crit Care Med

DOI

EISSN

1535-4970

Publication Date

May 15, 2012

Volume

185

Issue

10

Start / End Page

1044 / 1048

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Survival Analysis
  • Severity of Illness Index
  • Respiratory System Agents
  • Respiratory System
  • Lung Transplantation
  • Idiopathic Pulmonary Fibrosis
  • Humans
  • Hospitalization
  • Endpoint Determination
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Raghu, G., Collard, H. R., Anstrom, K. J., Flaherty, K. R., Fleming, T. R., King, T. E., … Brown, K. K. (2012). Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials. Am J Respir Crit Care Med, 185(10), 1044–1048. https://doi.org/10.1164/rccm.201201-0006PP
Raghu, Ganesh, Harold R. Collard, Kevin J. Anstrom, Kevin R. Flaherty, Thomas R. Fleming, Talmadge E. King, Fernando J. Martinez, and Kevin K. Brown. “Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials.Am J Respir Crit Care Med 185, no. 10 (May 15, 2012): 1044–48. https://doi.org/10.1164/rccm.201201-0006PP.
Raghu G, Collard HR, Anstrom KJ, Flaherty KR, Fleming TR, King TE, et al. Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials. Am J Respir Crit Care Med. 2012 May 15;185(10):1044–8.
Raghu, Ganesh, et al. “Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials.Am J Respir Crit Care Med, vol. 185, no. 10, May 2012, pp. 1044–48. Pubmed, doi:10.1164/rccm.201201-0006PP.
Raghu G, Collard HR, Anstrom KJ, Flaherty KR, Fleming TR, King TE, Martinez FJ, Brown KK. Idiopathic pulmonary fibrosis: clinically meaningful primary endpoints in phase 3 clinical trials. Am J Respir Crit Care Med. 2012 May 15;185(10):1044–1048.

Published In

Am J Respir Crit Care Med

DOI

EISSN

1535-4970

Publication Date

May 15, 2012

Volume

185

Issue

10

Start / End Page

1044 / 1048

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Survival Analysis
  • Severity of Illness Index
  • Respiratory System Agents
  • Respiratory System
  • Lung Transplantation
  • Idiopathic Pulmonary Fibrosis
  • Humans
  • Hospitalization
  • Endpoint Determination