Enhancing Scientific Inquiry in Clinical Neurosciences Through Methodology Traini

描述（由申请人提供）：临床试验是确定新疗法疗效的金标准方法，但有许多问题抑制了临床神经科学发现的步伐。结果是，许多“学习”（I/II期）临床试验未能以连贯的方式将有希望的治疗方法推进到“关键”、确认性（III期）试验。这导致了大量的验证期临床试验失败和无法推进转化神经科学。临床试验是复杂、昂贵和耗时的，许多从事新疗法开发和评估的初级研究人员在该领域的成功和进步面临着巨大的障碍。在NINDS的支持下，专注于神经科学的临床试验网络变得越来越重要。与制药公司内部的发现团队类似，NETT网络和NeuroNEXT通过临床医生、生物统计学家、数据管理人员、监测人员、研究协调员和其他临床试验专业人员之间的紧密跨大学合作，创造了规模经济。对于这一提议，我们的首要目标是利用这一资源来培训下一代研究人员，以提供设计良好且科学合理的“学习”阶段临床试验的重要结果，这将为未来成功的验证性试验奠定基础。我们提出的研究教育计划包括会前网络研讨会、面对面会议、会后网络研讨会和其他综合、纵向、小组体验的活动。在临床试验模拟和灵活的适应性设计的先进轨道计划在以后的几年。我们的方法将使我们能够评估具体结果指标（如资助的拨款数量）和内部绩效指标（临床试验计划和与课程参与者进行的知识和能力）的进展。我们将利用创新的跨机构合作来

DESCRIPTION (provided by applicant): Clinical trials are the gold standard method for determining the efficacy of new treatments, yet there are a number of issues inhibiting the pace of discovery in the clinical neurosciences. The result is that many "learn" (phase I/II) clinical trials are failing to move promising treatments forward into "pivotal," confirmatory (phase III) trials in a coherent way. This has led to a high number of confirmatory phase clinical trial failurs and an inability to advance translational neuroscience. Clinical trials are complicated, expensive, and time consuming and many junior investigators working on the development and evaluation of new therapies face substantial barriers to success and progression in the field. With NINDS support, clinical trial networks focused on neuroscience have grown in importance. Analogous to discovery teams within pharmaceutical companies, the NETT Network and NeuroNEXT have created economies of scale with dense cross-university collaborations between clinician trialists, biostatisticians, data managers, monitors, study coordinators and other clinical trial professionals. For this proposal, our overarching goal is to leverage this resource to train the next generation of researchers to deliver the important results from well-designed and scientifically-sound "learn" phase clinical trials that will form the foundation for successful confirmatory trials in the future. Our proposed research educational plan includes pre-conference webinars, an in-person conference, with post-conference webinars and other activities in an integrated, longitudinal, small group experience. An advanced track in clinical trial simulation and flexible adaptive design is planned for the later years. Our approach will allow us to evaluate progress on specific outcome measures (such as number of grants funded) and internal performance metrics (knowledge and competency in clinical trial planning and conduct with course participants). We will use our innovative, cross institutional collaboration to accomplish the following specific aims: 1) Identify promising junior investigators in the clinical neurosciences and provide them with a rigorous foundation in the design, funding, conduct, monitoring/oversight, ethical performance, and reporting of patient-oriented clinical research; 2)Promote ongoing professional career development by supporting participants before, during, and after the course to allow them to follow through on their plans for clinical trials-based research; 3)Enhance the pipeline of scientifically-sound, well-designed early phase clinical investigations that will provide a foundation for pivotal, confirmatory trials to reduce the burden of high-impact neurological diseases; and 4) Create a cooperative infrastructure for collaboration and sharing of educational resources for clinical trialists in the neurosciences to promote scientifically-sound, patient-oriented clinical trials.