Department of Internal Medicine-Cardiology, Charité Universitäts Medizin, Berlin, Germany.
Department of Cardiology, University of L'Aquila, L'Aquila.
J Cardiovasc Med (Hagerstown). 2018 Aug;19(8):411-421. doi: 10.2459/JCM.0000000000000669.
: Despite recent advances in chronic heart failure treatment, prognosis of acute heart failure patients remains poor with a heart failure rehospitalization rate or death reaching approximately 25% during the first 6 months after discharge. In addition, about half of these patients have preserved ejection fraction for which there are no evidence-based therapies. Disappointing results from heart failure clinical trials over the past 20 years emphasize the need for developing novel approaches and pathways for testing new heart failure drugs and devices. Indeed, many trials are being conducted without matching the mechanism and action of the drug with the clinical event. The implementation of these novel approaches should be coupled with the training of a new generation of heart failure physicians and scientists in the art and science of clinical trials. Currently, drug development is led by opinion leaders and experts who, despite their huge personal experience, were never trained systematically on drug development. The aim of this article is to propose a training program of 'drug development in Heart Failure'. A physician attending this course would have to be trained with a major emphasis on heart failure pathophysiology to better match mechanisms of death and rehospitalization with mechanism of action of the drug. Applicants will have to prove their qualifications and special interest in heart failure drug development before enrollment. This article should serve as a roadmap on how to apply emerging general principles in an innovative drug-development-in-heart-failure-process as well as the introduction of a new educational and mentorship program focusing on younger generations of researchers.
尽管在慢性心力衰竭治疗方面取得了最近的进展,但急性心力衰竭患者的预后仍然很差,出院后 6 个月内心力衰竭再住院率或死亡率达到约 25%。此外,这些患者中有大约一半的射血分数保留,针对这部分患者还没有基于证据的治疗方法。过去 20 年心力衰竭临床试验令人失望的结果强调了需要开发新的方法和途径来测试新的心力衰竭药物和设备。事实上,许多试验在进行时并没有使药物的机制和作用与临床事件相匹配。这些新方法的实施应该与新一代心力衰竭医生和科学家在临床试验的艺术和科学方面的培训相结合。目前,药物开发由意见领袖和专家主导,尽管他们个人经验丰富,但他们从未接受过系统的药物开发培训。本文的目的是提出一个“心力衰竭药物开发”培训计划。参加该课程的医生必须接受心力衰竭病理生理学的重点培训,以更好地将死亡和再住院的机制与药物的作用机制相匹配。申请人在注册前必须证明其在心力衰竭药物开发方面的资格和特殊兴趣。本文旨在提供一份路线图,说明如何将新兴的一般原则应用于心力衰竭药物开发过程中的创新,以及引入一个专注于研究人员的年轻一代的新的教育和指导计划。