Design of a “Lean” Case Report Form for Heart Failure Therapeutic Development
Author + information
- Mitchell A. Psotka, MD, PhDa,∗ (Mitchell.Psotka{at}inova.org), @mpsotka@hfcollaboratory,
- Mona Fiuzat, PharmDb,
- Peter E. Carson, MDc,
- David P. Kao, MDd,
- Jeffrey Cerkvenik, MSe,
- Daniel E. Schaber, PharmDe,
- Patrick Verta, MDf,
- Robert T. Kazmierski, PhDg,
- Meir Shinnar, MD, PhDg,
- Norman Stockbridge, MD, PhDg,
- Ellis F. Unger, MDg,
- Bram Zuckerman, MDg,
- Javed Butler, MD, MPH, MBAh,
- G. Michael Felker, MD, MHSi,
- Marvin A. Konstam, MDj,
- JoAnn Lindenfeld, MDk,
- Scott D. Solomon, MDl,
- John R. Teerlink, MDm,
- Christopher M. O'Connor, MDa and
- William T. Abraham, MDn
- aInova Heart and Vascular Institute, Falls Church, Virginia
- bDepartment of Medicine, Duke University School of Medicine, Durham, North Carolina
- cDepartment of Cardiology, Washington Veterans Affairs Medical Center, Washington, DC
- dDivision of Cardiology, University of Colorado School of Medicine, Aurora, Colorado
- eMedtronic Inc., Minneapolis, Minnesota
- fEdwards Lifesciences, Irvine, California
- gU.S. Food and Drug Administration, Silver Spring, Maryland
- hDepartment of Medicine, University of Mississippi, Jackson, Mississippi
- iDivision of Cardiology, Duke University Medical Center, Durham, North Carolina
- jCardioVascular Center of Tufts Medical Center, Boston, Massachusetts
- kHeart Failure and Transplantation Section, Vanderbilt Heart and Vascular Institute, Nashville, Tennessee
- lCardiovascular Division, Brigham and Women’s Hospital, Boston, Massachusetts
- mSection of Cardiology, San Francisco Veterans Affairs Medical Center and School of Medicine, University of California San Francisco, San Francisco, California
- nDepartments of Medicine, Physiology, and Cell Biology, Division of Cardiovascular Medicine, Davis Heart and Lung Research Institute, Ohio State University, Columbus, Ohio
- ↵∗Address for correspondence:
Dr. Mitchell A. Psotka, MD PhD, c/o Heart Failure Collaboratory, 3300 Gallows Road, Falls Church, Virginia, 22042.
Central Illustration
Highlights
• The development of treatments for heart failure is challenged by inefficient and burdensome clinical trials.
• The Heart Failure Collaboratory created a lean case report form for use in heart failure clinical trials.
• The lean case report form can be used and iterated to become the standard for clinical data capture.
Abstract
The development of treatments for heart failure (HF) is challenged by burdensome clinical trials. Reducing the need for extensive data collection and increasing opportunities for data compatibility between trials may improve efficiency and reduce resource burden. The Heart Failure Collaboratory (HFC) multi-stakeholder consortium sought to create a lean case report form (CRF) for use in HF clinical trials evaluating cardiac devices. The HFC convened patients, clinicians, clinical researchers, the U.S. Food and Drug Administration (FDA), payers, industry partners, and statisticians to create a consensus core CRF. Eight recent clinical trial CRFs for the treatment of HF from 6 industry partners were analyzed. All CRF elements were systematically reviewed. Those elements deemed critical for data collection in HF clinical trials were used to construct the final, harmonized CRF. The original CRFs included 176 distinct data items covering demographics, vital signs, physical examination, medical history, laboratory and imaging testing, device therapy, medications, functional and quality of life assessment, and outcome events. The resulting, minimally inclusive CRF device contains 75 baseline data items and 6 events, with separate modular additions that can be used depending on the additional detail required for a particular intervention. The consensus electronic form is now freely available for use in clinical trials. Creation of a core CRF is important to improve clinical trial efficiency in HF device development in the United States. This living document intends to reduce clinical trial administrative burden, increase evidence integrity, and improve comparability of clinical data between trials.
Footnotes
Dr. Psotka is a consultant for Amgen, Cytokinetics, Roivant, and Windtree. Dr. Fiuzat has received research support from and is a consultant for ResMed. Mr. Cerkvenik and Dr. Schaber are employees of Medtronic. Dr. Verta is an employee of Edwards Lifesciences. Dr. Butler has received research support from U.S. National Institutes of Health, European Union, and Patient-Centered Outcomes Research Institute; and is a consultant for Amgen, Array, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squib, CVRx, G3 Pharma, Innolife, Janssen, Luitpold, Medtronic, Merck, Novartis, NovoNordisk, Relypsa, and Vifor. Dr. Felker has received research funding from U.S. NIH/National Heart, Lung, and Blood Institute, American Heart Association, Novartis, Amgen, Cytokinetics, Merck, and Roche Diagnostics; and is a consultant for Novartis, Amgen, Roche Diagnostics, Medtronic, Bristol-Myers Squibb, GlaxoSmithKline, Cardionomic, Cytokinetics, Myokardia, Stealth, Innolife, and EBR Systems. Dr. Konstam is a consultant for Amgen, Bristol-Myers Squibb, Boehringer Ingelheim, and Novartis; and has received research support from Ironwood and Livanova. Dr. Lindenfeld is a consultant for Novartis, Abbott, ResMed, VWave, CVRx, and Cytokinetics. Dr. Solomon has received research support from Alnylam, Amgen, AstraZeneca, Bellerophon, Bristol-Myers Squibb, Celladon, Cytokinetics, Eidos, Gilead, GlaxoSmithKline, Ionis, Lone Star Heart, Mesoblast, MyoKardia, U.S. NIH /National Heart, Lung, and Blood Institute, Novartis, Sanofi Pasteur, and Theracos; and is a consultant for Akros, Alnylam, Amgen, AstraZeneca, Bayer, Bristol-Myers Squibb, Cardior, Corvia, Cytokinetics, Gilead, GlaxoSmithKline, Ironwood, Merck Sharpe and Dohme, Novartis, Roche, Takeda, Theracos, Quantum Genetics, Cardurion, AoBiome, Janssen, and Cardiac Dimensions. Dr. Teerlink is a consultant for Amgen, Bayer, Cytokinetics, Novartis, and Stealth Health; and has received research support from Abbott, Amgen, Bayer, Bristol-Myers Squibb, Novartis, and scPharma. Dr. O’Connor has received research support from and is a consultant for ResMed, Merck, and Bristol-Myers Squibb; is a consultant for Stealth Peptides; and is a co-owner of Biscardia. Dr. Abraham has served as a consultant for Amgen, AstraZeneca, CHF Solutions, GSK, Guidant, Medtronic, Merck, Novartis, Pfizer, and Zoll; and has received research support from Amgen, Biotronik, CHF Solutions, GSK, HFSA, Medtronic, Myogen, and Otsuka. 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.
- Received June 3, 2019.
- Revision received July 1, 2019.
- Accepted July 8, 2019.
- 2019 The Authors
