Abstract
Heart failure (HF) patients experience a high burden of symptoms and functional limitations, and morbidity and mortality remain high despite successful therapies. The majority of HF drugs in the United States are approved for reducing hospitalization and mortality, while only a few have indications for improving quality of life, physical function, or symptoms. Patient-reported outcomes that directly measure patient's perception of health status (symptoms, physical function, or quality of life) are potentially approvable endpoints in drug development. This paper summarizes the history of endpoints used for HF drug approvals in the United States and reviews endpoints that measure symptoms, physical function, or quality of life in HF patients.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 429-440 |
| Number of pages | 12 |
| Journal | JACC: Heart Failure |
| Volume | 8 |
| Issue number | 6 |
| DOIs | |
| State | Published - Jun 2020 |
Keywords
- FDA
- clinical trials
- endpoints
- heart failure
- patient-reported outcomes
ASJC Scopus subject areas
- Cardiology and Cardiovascular Medicine
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Endpoints in Heart Failure Drug Development : History and Future. / Fiuzat, Mona; Lowy, Naomi; Stockbridge, Norman et al.
In: JACC: Heart Failure, Vol. 8, No. 6, 06.2020, p. 429-440.Research output: Contribution to journal › Review article › peer-review
TY - JOUR
T1 - Endpoints in Heart Failure Drug Development
T2 - History and Future
AU - Fiuzat, Mona
AU - Lowy, Naomi
AU - Stockbridge, Norman
AU - Sbolli, Marco
AU - Latta, Federica
AU - Lindenfeld, Jo Ann
AU - Lewis, Eldrin F.
AU - Abraham, William T.
AU - Teerlink, John
AU - Walsh, Mary
AU - Heidenreich, Paul
AU - Bozkurt, Biykem
AU - Starling, Randall C.
AU - Solomon, Scott
AU - Felker, G. Michael
AU - Butler, Javed
AU - Yancy, Clyde
AU - Stevenson, Lynne W.
AU - O'Connor, Christopher
AU - Unger, Ellis
AU - Temple, Robert
AU - McMurray, John
N1 - Funding Information: Drs. Lowy and Stockbridge are employees of the U.S. Food and Drug Administration. Dr. Latta is an employee of Inova. Dr. Lindenfeld has received consulting and/or research support from Abbott, AstraZeneca, Edwards Lifesciences, Boehringer Ingelheim, V-wave, Vifor, CVRx, and Impulse DYnamics. Dr. Abraham is a consultant for Abbott, ARCA biopharma, Boehringer Ingelheim, CVRx, Edwards Lifesciences, Sensible Medical; a compensated principal investigator for studies conducted by various companies; and is a member of the board of Cardionomic. Dr. Teerlink has received research support from and is a consultant for Abbott, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Cytokinetics, EBR Systems, Medtronic, Merck, Novartis, scPharma, and Windtree Therapeutics. Dr. Bozkurt serves as a member of the Clinical Event Committee, Abbott Vascular, Respicardia Registry Steering Committee; and has received consulting and/or research support from Anthem; and is a consultant for scPharmaceuticals. Dr. Solomon has received research support from Alnylam, Amgen, AstraZeneca, Bellerophon, Bayer, Bristol-Myers Squibb, Celladon, Cytokinetics, Eidos, Gilead, GlaxoSmithKline, Ionis, Lone Star Heart, Mesoblast, MyoKardia, U.S. National Institutes of Health (NIH)/National Heart Lung and Blood Institute (NHLBI), Novartis, Sanofi, Pasteur, Theracos; and is a consultant for Akros, Alnylam, Amgen, Arena, AstraZeneca, Bayer, Bristol-Myers Squibb, Cardior, Corvia, Cytokinetics, Daiichi-Sankyo, Gilead, GlaxoSmithKline, Ironwood, Merck, Myokardia, Novartis, Roche, Takeda, Theracos, Quantum Genetics, Cardurion, AoBiome, Janssen, Cardiac Dimensions, and Tenaya. Dr. Felker has received research support from NHLBI (NIH grant U-10HL110312), American Heart Association, Amgen, Merck, Cytokinetics, and Roche Diagnostics; and is a consultant for Novartis, Amgen, Bristol-Myers Squibb, Cytokinetics, Medtronic, Cardionomic, Relypsa, V-wave, Myokardia, Innolife, EBR Systems, Arena, Abbott, Sphingotec, Roche Diagnostics, Alnylam, LivaNova, Windtree Therapeutics, Rocket Pharma, and SC Pharma. Dr. Butler has received consulting and/or research support from Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CVRx, Janssen, Luitpold, Medtronic, Merck, Novartis, NovoNordisk, Relypsa, Sanofi, and Vifor. Dr. Lewis is a consultant for and has received research support from Novartis, Amgen, Merck, and Dal-Cor. The views expressed in this manuscript are those of the authors and do not necessarily represent the views of the FDA or the US Department of Health and Human Services. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose. Barry Greenberg, MD, served as Guest Editor for this paper. Funding Information: Drs. Lowy and Stockbridge are employees of the U.S. Food and Drug Administration. Dr. Latta is an employee of Inova. Dr. Lindenfeld has received consulting and/or research support from Abbott, AstraZeneca, Edwards Lifesciences, Boehringer Ingelheim, V-wave, Vifor, CVRx, and Impulse DYnamics. Dr. Abraham is a consultant for Abbott, ARCA biopharma, Boehringer Ingelheim, CVRx, Edwards Lifesciences, Sensible Medical; a compensated principal investigator for studies conducted by various companies; and is a member of the board of Cardionomic. Dr. Teerlink has received research support from and is a consultant for Abbott, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Cytokinetics, EBR Systems, Medtronic, Merck, Novartis, scPharma, and Windtree Therapeutics. Dr. Bozkurt serves as a member of the Clinical Event Committee, Abbott Vascular, Respicardia Registry Steering Committee; and has received consulting and/or research support from Anthem; and is a consultant for scPharmaceuticals. Dr. Solomon has received research support from Alnylam, Amgen, AstraZeneca, Bellerophon, Bayer, Bristol-Myers Squibb, Celladon, Cytokinetics, Eidos, Gilead, GlaxoSmithKline, Ionis, Lone Star Heart, Mesoblast, MyoKardia, U.S. National Institutes of Health (NIH)/National Heart Lung and Blood Institute (NHLBI), Novartis, Sanofi, Pasteur, Theracos; and is a consultant for Akros, Alnylam, Amgen, Arena, AstraZeneca, Bayer, Bristol-Myers Squibb, Cardior, Corvia, Cytokinetics, Daiichi-Sankyo, Gilead, GlaxoSmithKline, Ironwood, Merck, Myokardia, Novartis, Roche, Takeda, Theracos, Quantum Genetics, Cardurion, AoBiome, Janssen, Cardiac Dimensions, and Tenaya. Dr. Felker has received research support from NHLBI (NIH grant U-10HL110312), American Heart Association, Amgen, Merck, Cytokinetics, and Roche Diagnostics; and is a consultant for Novartis, Amgen, Bristol-Myers Squibb, Cytokinetics, Medtronic, Cardionomic, Relypsa, V-wave, Myokardia, Innolife, EBR Systems, Arena, Abbott, Sphingotec, Roche Diagnostics, Alnylam, LivaNova, Windtree Therapeutics, Rocket Pharma, and SC Pharma. Dr. Butler has received consulting and/or research support from Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CVRx, Janssen, Luitpold, Medtronic, Merck, Novartis, NovoNordisk, Relypsa, Sanofi, and Vifor. Dr. Lewis is a consultant for and has received research support from Novartis, Amgen, Merck, and Dal-Cor. The views expressed in this manuscript are those of the authors and do not necessarily represent the views of the FDA or the US Department of Health and Human Services. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose. Barry Greenberg, MD, served as Guest Editor for this paper. Publisher Copyright: © 2020
PY - 2020/6
Y1 - 2020/6
N2 - Heart failure (HF) patients experience a high burden of symptoms and functional limitations, and morbidity and mortality remain high despite successful therapies. The majority of HF drugs in the United States are approved for reducing hospitalization and mortality, while only a few have indications for improving quality of life, physical function, or symptoms. Patient-reported outcomes that directly measure patient's perception of health status (symptoms, physical function, or quality of life) are potentially approvable endpoints in drug development. This paper summarizes the history of endpoints used for HF drug approvals in the United States and reviews endpoints that measure symptoms, physical function, or quality of life in HF patients.
AB - Heart failure (HF) patients experience a high burden of symptoms and functional limitations, and morbidity and mortality remain high despite successful therapies. The majority of HF drugs in the United States are approved for reducing hospitalization and mortality, while only a few have indications for improving quality of life, physical function, or symptoms. Patient-reported outcomes that directly measure patient's perception of health status (symptoms, physical function, or quality of life) are potentially approvable endpoints in drug development. This paper summarizes the history of endpoints used for HF drug approvals in the United States and reviews endpoints that measure symptoms, physical function, or quality of life in HF patients.
KW - FDA
KW - clinical trials
KW - endpoints
KW - heart failure
KW - patient-reported outcomes
UR - http://www.scopus.com/inward/record.url?scp=85084702906&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85084702906&partnerID=8YFLogxK
U2 - 10.1016/j.jchf.2019.12.011
DO - 10.1016/j.jchf.2019.12.011
M3 - Review article
C2 - 32278679
AN - SCOPUS:85084702906
SN - 2213-1779
VL - 8
SP - 429
EP - 440
JO - JACC: Heart Failure
JF - JACC: Heart Failure
IS - 6
ER -