Clinical Trial Design, Endpoints, and Regulatory Requirements
Abstract
A new therapeutic agent for heart failure can be approved if it improves survival and/or reduces hospitalisations or if it safely improves functional capacity. Therefore its clinical development program must demonstrate clinically relevant improvement in a robust clinical end point and adequate safety to justify regulatory approval and clinical use. Mortality and hospitalisations are now combined with new composite end points in order to improve trial efficiency and adequate assessment of efficacy of newer molecules, biologicals and cell therapies developed for the treatment of heart failure. Newer regulatory practices have been developed in the past few years and they will require design of innovative study designs able to demonstrate a sound clinical benefit alongside with adequate safety profile.
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Hospitalization for heart failure has been regarded as a reliable surrogate of disease progression in heart failure.
“Although hospitalization for heart failure has been regarded as a reliable surrogate of disease progression in heart failure,9,10”
From Effect of Empagliflozin on the Clinical Stability of Patients With Heart Failure and a Reduced Ejection Fraction: The EMPEROR-Reduced Trial (2021) · cites this paper as [9]
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References 19 · Cited by 7
- Authors
- Rosano, G. M. C.
- Affiliations
- St George's Hospital (GB)
- Corresponding author
- Rosano, Giuseppe M. C.
- Venue
- Handbook of Experimental Pharmacology
- Publisher
- Springer International Publishing
- Published
- 2016
- Volume
- 243
- Pages
- 67-78
- ISSN
- 0171-2004 (ISSN-L)
- 1865-0325
- OpenAlex
- W2543056759
- Cited by
- 7
- References
- 19
- Citations by year
- 2024: 2 · 2022: 1 · 2020: 2 · 2016: 1
- FWCI
- 1.91
- Type
- book chapter
- Language
- en
- Open access
- closed
- Locations
- Handbook of experimental pharmacology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Heart Failure Treatment and Management
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
- Keywords
- heart failure, clinical trial design, composite endpoints, regulatory approval, drug safety, survival, hospitalization, functional capacity, cell therapy, biologicals, trial efficiency
- MeSH terms
- Patient Reported Outcome Measures, Clinical Trials as Topic, Heart Failure, Hemodynamics, Hospitalization, Humans, Quality of Life, Research Design, Biomarkers, Survival Rate, Ventricular Function, Left, Disease Progression
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