Core A: Program Management and Biostatistics Core

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Abstract – Core A The Program Management and Biostatistics Core is designed to function as a drug development Project Team decision-making body. Thus, it will oversee all aspects of the drug development paradigm. This activity will involve two integrated and overlapping functions: (1) Oversight/Assessment of new directions of the scientific topics that form the basis of the tPPG target/drug development paradigm; and (2) Oversight of the drug development process. The oversight of scientific paradigms will focus on delineation of the ideal profile for mucolytics, need for mucus hydrating agents for co-formulation with mucolytics, selection of mucolytic clinical candidates, and identification and validation of biomarkers for use throughout the tPPG development program. Oversight of the drug development process will include oversight of the composition of the DSMB and DSMB Charter, flow of data to the DSMB, and the role of DSMB in clinical development decision making. Core A will also provide biostatistical assistance to the pre-clinical projects and extensive services for the clinical projects in the context of the creation of REDCap databases for clinical data collection, oversight of clinical database management , and creation of tables and listings for analyses of all studies. Note, an external biostatistician will be named by Core A for overall final review of safety and efficacy data. The external statistician will also be part of the External Advisory Board. Finally, Core A will provide administrative functions to the tPPG, including budgetary and programmatic support, oversight of human subjects, protocols, and oversight of conflict of interest reviews. These activities should provide for an effective Project Management in Biostatistics Core support function for the tPPG.