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.
We propose a training course to enable young investigators to learn how to design the best early clinical trials to enable discovery of effectiv treatments. Our team is comprised of experts who collaborate within large, NIH funded research networks focused on neurological diseases. Our approach will enhance and maintain the pipeline of scientifically-sound clinical trials and promising young investigators in a way that promotes health by discovering important, effective new treatments.
