Reassessing Phase II Heart Failure Clinical Trials: Consensus Recommendations.
Butler J, Hamo CE, Udelson JE, O'Connor C, Sabbah HN, Metra M, Shah SJ, Kitzman DW, Teerlink JR, Bernstein HS, Brooks G, Depre C, DeSouza MM, Dinh W, Donovan M, Frische-Danielson R, Frost RJ, Garza D, Gohring UM, Hellawell J, Hsia J, Ishihara S, Kay-Mugford P, Koglin J, Kozinn M, Larson CJ, Mayo M, Gan LM, Mugnier P, Mushonga S, Roessig L, Russo C, Salsali A, Satler C, Shi V, Ticho B, van der Laan M, Yancy C, Stockbridge N, Gheorghiade M. Reassessing Phase II Heart Failure Clinical Trials: Consensus Recommendations. Circ Heart Fail. Apr 2017;10(4).
Circ Heart Fail
The increasing burden and the continued suboptimal outcomes for patients with heart failure underlines the importance of continued research to develop novel therapeutics for this disorder. This can only be accomplished with successful translation of basic science discoveries into direct human application through effective clinical trial design and execution that results in a substantially improved clinical course and outcomes. In this respect, phase II clinical trials play a pivotal role in determining which of the multitude of potential basic science discoveries should move to the large and expansive registration trials in humans. A critical examination of the phase II trials in heart failure reveals multiple shortcomings in their concept, design, execution, and interpretation. To further a dialogue on the challenges and potential for improvement and the role of phase II trials in patients with heart failure, the Food and Drug Administration facilitated a meeting on October 17, 2016, represented by clinicians, researchers, industry members, and regulators. This document summarizes the discussion from this meeting and provides key recommendations for future directions.
Medical Subject Headings
Cardiovascular Agents; Clinical Trials, Phase II as Topic; Consensus; Heart Failure; Humans; Research Design; Treatment Outcome; United States; United States Food and Drug Administration