Pilot-Feasibility

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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.