ROAR Pilot/ Feasibility Core

ABSTRACT – PILOT/FEASIBILITY CORE The Pilot/Feasibility Core (PFC) of the ROAR consortium will serve as a hub for innovation that drives research in the ROAR consortium, in the organic acidemia research community, and in the field of inborn errors of metabolism. The PFC will integrate clinical expertise, scientific innovation, and research priorities from patient advocacy groups to solicit, select, and oversee pilot projects that align with the mission of the ROAR consortium and meet the needs of patients in the organic acidemia community. The PFC will achieve these goals with the following specific aims 1) implement a process for the selection of innovative pilot projects that will advance the diagnosis, clinical trial readiness, management, and treatment of rare organic acidemias and 2) provide oversight for selected pilot projects to ensure that NIH prior approval requirements are met, that subawards are executed efficiently, and that results are disseminated to the larger community. The PFC will broadly circulate the request for pilot applications to solicit a large pool of applications for one-year pilot funding (renewable for a second year by competitive application if appropriate progress is documented). A team consisting of ROAR investigators, NIH program representation, and patient advocacy group leaders will systematically score, prioritize pilot project proposals for funding in an unbiased way, and provide constructive feedback to all applicants. The PFC will work collaboratively with the RDCRN Data Management & Coordinating Center (DMCC), the ROAR lead study site, and the pilot project principal investigator and their institution to ensure that all regulatory approvals are met and subawards are executed in a timely manner. To ensure scientific rigor and alignment with the Career Enhancement Core, the PFC will monitor the selection process closely and encourage applications from new and junior investigators. Upon completion of funding, we expect that the data generated from ROAR pilot projects will be developed into larger grant applications and larger projects within and outside the consortium and lead to publications and presentations to the scientific, medical, and patient communities. Moreover, we expect that results from pilot studies will inform our data collection and analyses in the longitudinal study, will inform the design of future clinical trials and clinical studies in organic acidemias, and will ultimately positively impact the lives of patients with these disorders.