Core A: Program Management and Biostatistics Core

摘要-核心A 项目管理和生物统计学核心旨在作为药物开发项目团队发挥作用 决策机构。因此，它将监督药物开发模式的所有方面。本次活动将 （1）监督/评估科学研究的新方向; 构成tPPG目标/药物开发范式基础的主题;以及（2）药物的监督 发展过程对科学范式的监督将侧重于描绘理想的轮廓， 粘液溶解剂，需要粘液水合剂与粘液溶解剂共同配制，粘液溶解临床选择 候选物，以及用于整个tPPG开发计划的生物标志物的鉴定和验证。 药物开发过程的监督将包括对DSMB和DSMB组成的监督 章程、数据流向DSMB以及DSMB在临床开发决策中的作用。核心A将 并为临床前项目提供生物统计学协助，为临床项目提供广泛服务 在创建用于临床数据收集的REDCap数据库的背景下，监督临床数据库 管理和创建表格和列表，用于分析所有研究。注：外部生物统计学家 将由核心A指定，对安全性和疗效数据进行总体最终审查。外部统计员还将 外部咨询委员会的一部分。最后，核心A将为临时方案规划小组提供行政职能，包括 预算和方案支持，对人类受试者的监督，协议，以及对冲突的监督 兴趣评论这些活动应提供有效的生物统计学核心项目管理 tPPG的支持功能。

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.