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

G Raghu, HR Collard, KJ Anstrom… - American journal of …, 2012 - atsjournals.org
G Raghu, HR Collard, KJ Anstrom, KR Flaherty, TR Fleming, TE King Jr, FJ Martinez
American journal of respiratory and critical care medicine, 2012atsjournals.org
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 funtional status in IPF …
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 funtional 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.
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