Pilot-Feasibility

摘要-试点/可行性核心 试点/可行性核心是培育创新的诊断和 尿素循环障碍(UCD)的管理核心提供了患者需求之间的重要联系-作为 由临床医生和患者权益倡导团体代表阐述-以及可能 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，确定和确认最显著的结果衡量标准，以及开发新的 诊断和护理的方法。试点项目的结果将加强收集的纵向数据 研究完善临床试验设计。 申请、审查和遴选程序：从尿素循环障碍中征求建议书 财团(UCDC)成员和其他人通过融资机会公告(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有关 并推进《行动纲领》中确定的UCDC的总体目标和优先事项。符合以下条件的申请者 邀请筛选标准提交完整的申请(5页的研究计划和预算信息)。这个 遴选委员会根据以下方面审查全部申请：UCDC的相关性、重要性、创新和 对每个标准和总体影响使用NIH量表评分系统的方法。其他关键考虑事项 审查中使用的包括使用UCDC的现有资源和基础设施，阐明具体的 成果衡量和里程碑，以及支持初级调查员职业生涯的潜力，包括 代表不足的少数族裔。评选委员会对评审结果进行评分和讨论(包括 国家卫生研究院科学官员)，并根据国家卫生研究院研究部分的格式确定优先顺序。推选委员会 建议被发送给UCDC执行委员会，该执行委员会作为“理事会”来提供资金 将由NIH审查批准的决定。寻求第二年试点资金的申请者是 使用相同的标准进行评判。关于第二年资金的其他考虑因素包括 在第一年成功达到里程碑和成就，证明第二年 资金将促进UCDC的目标和优先事项，并为如何增加一年的 资金将增加取得有意义成果的可能性和获得外部资金的潜力。申请一个 第二年的资助是在慈善基金可用时考虑的，它们不会直接竞争 提出了新的建议。 监督和评价：每个试点项目的结果都作为平台报告在 UCDC年度面对面会议。获奖者每年提交一份进度报告，直到项目完成。这 该报告被纳入美国疾病控制与预防中心提交给美国国立卫生研究院的年度进展报告中。获奖者每季度与Core会面 Pi(Nagamani博士)审查进展情况并解决可能出现的任何挑战。

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.