Pilot-Feasibility

摘要-试点/可行性核心 试点/可行性核心作为一个“孵化器”，以培育创新的诊断方法， 管理尿素循环障碍（UCD）。核心在患者需求之间提供了重要的联系--正如 由临床医生和患者权益团体代表提出的新的研究建议， 有时是高风险的。一个关键的目的是促进临床试验，重点是发现新的 生物标志物，识别和验证最显著的结果指标，以及开发新的 诊断和护理方法。试点项目的结果将加强为纵向调查收集的数据， 研究和改进临床试验设计。 申请、审查和选择过程：征求尿素循环障碍的建议 联盟（UCDC）成员和其他人通过资助机会公告（FOA）。意向书 （1页）由遴选委员会主席审查，以确保提案明确与UCD相关 并推进FOA中定义的UCDC的总体目标和优先事项。符合这些条件的申请人 筛选标准是邀请提交一份完整的申请（5页的研究计划和预算信息）。的 遴选委员会根据以下标准审查全部申请：UCDC相关性，重要性，创新性和 使用NIH量表评分系统对每个标准和总体影响进行评估。其他主要考虑因素 审查中使用的方法包括使用UCDC中的现有资源和基础设施， 成果指标和里程碑，以及支持初级调查员职业生涯的潜力，包括 代表性不足的少数民族。评审会进行评分，并由评选委员会（包括一个 NIH科学官员），并根据NIH研究部分的格式进行优先排序。推选委员会 建议被送到作为“理事会”的UCDC执行委员会，以提供资金 这些决定将由NIH审查批准。申请第二年试点资金的申请人是 用同样的标准来判断。第二年供资的其他考虑因素包括： 第一年成功地达到了里程碑和取得了成就，第二年成功的理由 资金将促进UCDC的目标和优先事项，以及如何增加一年的明确理由 供资将增加取得有意义成果的可能性和外部供资的潜力。的请求 第二年的资助是在慈善基金可用时考虑的，它们不直接竞争 新的提议。 监督和评价：每个试点项目的结果作为平台介绍报告， 一年一度的UCDC会议。获奖者提交年度进度报告，直到项目完成。这 该报告被纳入向NIH提交的UCDC年度进展报告。获奖者每季度与核心 PI（Nagamani博士）审查进展并解决可能发生的任何挑战。

ABSTRACT – PILOT/FEASIBILITY CORE The Pilot/Feasibility Core serves as an “incubator” to nurture innovative approaches to diagnosis and management of urea cycle disorders (UCD). The Core affords an essential link between patient needs—as articulated by clinicians and patient advocacy group representatives—and novel research proposals that may be, on occasion, high risk. A key purpose is to promote clinical trials, with an emphasis on discovery of novel biomarkers, identification and validation of the most salient outcome measures, and development of new approaches to diagnosis and care. Results of the pilot projects will enhance data collected for the longitudinal study and improve clinical trial design. APPLICATION, REVIEW AND SELECTION PROCESS: Proposals are solicited from Urea Cycle Disorders Consortium (UCDC) members and others though a Funding Opportunity Announcement (FOA). Letters of Intent (1 page) are reviewed by the chair of the Selection Committee to ensure that the proposal clearly relates to UCD and advances the overall goals and priorities of the UCDC as defined in the FOA. Applicants meeting these screening criteria are invited to submit a full application (5-page research plan and budget information). The Selection Committee reviews full applications based on: UCDC relevance, Significance, Innovation and Approach using the NIH scale scoring system for each criterion and for overall impact. Other key considerations used in the review include use of existing resources and infrastructure in the UCDC, articulation of specific outcome measures and milestones, and the potential for supporting the career of junior investigators, including underrepresented minorities. The reviews are scored, discussed by the Selection Committee (which includes an NIH Scientific Officer), and prioritized according to the format of a NIH study section. Selection Committee recommendations are sent to the UCDC Executive Committee that serves as “Council” to make funding decisions that will be reviewed for approval by the NIH. Applicants seeking a second year of pilot funding are judged using the same criteria. Additional considerations for a second year of funding include having successfully met milestones and accomplishments in the first year, justification for how the second year of funding will further the goals and priorities of the UCDC, and a clear rationale for how the additional year of funding will increase the likelihood of meaningful outcomes and potential for outside funding. Requests for a second year of funding are considered when philanthropic funds are available, and they do not compete directly with new proposals. OVERSIGHT AND EVALUATION: Results of each pilot project are reported as platform presentations at the annual in-person UCDC meeting. Awardees submit an annual progress report until the project is complete. This report is incorporated into the annual UCDC progress report to the NIH. Awardees meet quarterly with the Core PI (Dr. Nagamani) to review progress and to resolve any challenges that may occur.