Idiopathic Pulmonary Fibrosis: Clinically Meaningful Primary Endpoints in Phase 3 Clinical Trials
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- Ganesh Raghu
- Department of Medicine, and
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- Harold R. Collard
- Department of Medicine, University of California San Francisco, San Francisco, California
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- Kevin J. Anstrom
- Duke Clinical Research Institute, Duke University, Durham, North Carolina
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- Kevin R. Flaherty
- Department of Medicine, University of Michigan, Ann Arbor, Michigan; and
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- Thomas R. Fleming
- Department of Biostatistics, University of Washington, Seattle, Washington
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- Talmadge E. King
- Department of Medicine, University of California San Francisco, San Francisco, California
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- Fernando J. Martinez
- Department of Medicine, University of Michigan, Ann Arbor, Michigan; and
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- Kevin K. Brown
- Department of Medicine, National Jewish Health, Denver, Colorado
書誌事項
- 公開日
- 2012-05
- 権利情報
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- https://academic.oup.com/pages/standard-publication-reuse-rights
- DOI
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- 10.1164/rccm.201201-0006pp
- 公開者
- Oxford University Press (OUP)
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説明
<jats:title>Abstract</jats:title> <jats:p>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.</jats:p>
収録刊行物
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- American Journal of Respiratory and Critical Care Medicine
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American Journal of Respiratory and Critical Care Medicine 185 (10), 1044-1048, 2012-05
Oxford University Press (OUP)