重新评估II期心力衰竭临床试验：共识建议
Reassessing Phase II Heart Failure Clinical Trials: Consensus Recommendations.
作者信息
Butler Javed, Hamo Carine E, Udelson James E, O'Connor Christopher, Sabbah Hani N, Metra Marco, Shah Sanjiv J, Kitzman Dalane W, Teerlink John R, Bernstein Harold S, Brooks Gabriel, Depre Christophe, DeSouza Mary M, Dinh Wilfried, Donovan Mark, Frische-Danielson Regina, Frost Robert J, Garza Dahlia, Gohring Udo-Michael, Hellawell Jennifer, Hsia Judith, Ishihara Shiro, Kay-Mugford Patricia, Koglin Joerg, Kozinn Marc, Larson Christopher J, Mayo Martha, Gan Li-Ming, Mugnier Pierrre, Mushonga Sekayi, Roessig Lothar, Russo Cesare, Salsali Afshin, Satler Carol, Shi Victor, Ticho Barry, van der Laan Michael, Yancy Clyde, Stockbridge Norman, Gheorghiade Mihai
机构信息
From the Department of Medicine, Stony Brook University, NY (J.B., C.E.H.); Division of Cardiology and the CardioVascular Center, Tufts Medical Center, Boston, MA (J.E.U.); Division of Cardiology, Inova Heart & Vascular Institute, Falls Church, VA (C.O'C.); Department of Medicine, Division of Cardiovascular Medicine, Henry Ford Hospital, Detroit, MI (H.N.S.); Division of Cardiology, University of Brescia and Civil Hospital, Italy (M.M.); Division of Cardiology, Northwestern University Feinberg School of Medicine, Chicago, IL (S.J.S., C.Y.); Section on Cardiovascular Medicine, Wake Forest School of Medicine, Winston-Salem, NC (D.W.K.); Division of Cardiology, University of California San Francisco (J.R.T.); Merck & Co., Kenilworth, NJ (H.S.B., J.K.); Gilead Sciences, Foster City, CA (G.B., J.H., C.S.); Amgen Inc., Thousand Oaks, CA (C.D., M.K.); Bristol-Myers Squibb, Princeton, NJ (M.M.D., M.D., R.J.F., P.M., S.M., C.R.); Bayer, Wuppertal, Germany (W.D., M.v.d.L.); Department of Cardiology, HELIOS Clinic Wuppertal, University Hospital Witten/Herdecke, Germany (W.D.); AstraZeneca, Gaithersburg, MD (R.F.-D., J.H., L.-M.G.); AstraZeneca, Gothenburg, Sweden (R.F.-D., J.H., L.-M.G.); Relypsa Inc., Redwood City, CA (D.G., M.M.); Vifor Pharma, Opfikon, Switzerland (U.-M.G.); Department of Cardiology, Nippon Medical School Musashi-Kosugi Hospital, Kawasaki, Japan (S.I.); Novartis Pharmaceuticals Inc., East Hanover, NJ (P.K.-M., V.S.); Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA (C.J.L.); Bayer Pharma AG, Wuppertal, Germany (L.R.); Cardiology Division, Columbia University Medical Center, New York, NY (C.R.); Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, CT (A.S.); Moderna Therapeutics, Cambridge, MA (B.T.); Division of Cardiovascular and Renal Products, United States Food and Drug Administration, Silver Spring, MD (N.S.); Center for Cardiovascular Innovation, Feinberg School of Medicine, Northwestern University, Chicago, IL (M.G.).
出版信息
Circ Heart Fail. 2017 Apr;10(4). doi: 10.1161/CIRCHEARTFAILURE.116.003800.
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.
心力衰竭患者负担日益加重且治疗效果持续欠佳，这凸显了持续开展研究以开发针对该疾病新疗法的重要性。只有通过有效的临床试验设计与实施，将基础科学发现成功转化为直接应用于人类的疗法，才能实现这一目标，进而显著改善临床病程和治疗效果。在这方面，II期临床试验在确定众多潜在基础科学发现中哪些应进入大规模人体注册试验时起着关键作用。对心力衰竭II期试验的严格审查揭示了其在概念、设计、实施和解读方面存在的多重缺陷。为进一步探讨II期试验在心力衰竭患者中的挑战、改进潜力及作用，美国食品药品监督管理局于2016年10月17日组织了一次会议，参会人员包括临床医生、研究人员、行业成员和监管人员。本文档总结了此次会议的讨论内容，并为未来方向提供了关键建议。
Zotero 插件