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，但在临床神经科学中有许多问题阻碍了发现的步伐。其结果是，许多“学习”(I/II期)临床试验未能以一致的方式将有希望的治疗推进到“关键”(III期)试验。这导致了大量确证阶段的临床试验失败，并无法推动转化性神经科学的发展。临床试验复杂、昂贵、耗时，许多致力于开发和评估新疗法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通过临床试验专家、生物统计学家、数据经理、监测器、研究协调员和其他临床试验专业人员之间的密切跨大学合作，创造了规模经济。对于这项建议，我们的首要目标是利用这一资源来培训下一代研究人员，以交付来自精心设计和科学合理的“学习”阶段临床试验的重要结果，这些试验将为未来成功的验证性试验奠定基础。我们提议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活动，以一种综合的、纵向的、小组体验的方式进行。计划在未来几年在临床试验模拟和灵活的适应性设计方面进行先进的跟踪。我们的方法将使我们能够评估在特定结果衡量标准(如资助的资金数量)和内部绩效指标(临床试验规划和课程参与者实施方面的知识和能力)方面的进展情况。我们将利用我们的创新、跨机构合作来 完成以下具体目标：1)在临床神经科学领域找到有前途的初级研究人员，并在以患者为中心的临床研究的设计、资金、实施、监测/监督、道德表现和报告方面为他们提供严格的基础；2)通过在课程前、课程中和课程后支持参与者来促进持续的职业生涯发展，使他们能够贯彻以临床试验为基础的研究计划；3)加强科学合理、设计良好的早期临床研究的渠道，为关键的验证性试验提供基础，以减轻负担 4)为神经科学领域的临床试验人员建立协作和共享教育资源的合作基础设施，以促进科学合理、以患者为导向的临床试验。

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.