Statistics and Methodology in Clinical Trials

描述(由申请人提供)：人们普遍认为，在精神病学研究的方法和内容方面训练有素的生物统计学家太少了。在儿科治疗结果研究领域，这一点最为真实。这项以患者为导向的导师研究职业发展奖(K23)旨在为已经作为儿科临床试验人员拥有丰富专业知识的候选人提供统计和研究方法方面的高级培训。总体目标是将候选人确立为一名独立的生物统计调查员，能够在儿科干预研究的研究设计、实施和统计方法的界面上服务，并为新知识和新的统计和研究方法的发展做出贡献。具有互补技能的模范导师将建议和监督候选人的教学议程，其中包括高级生物统计学的正式课程作业、定向阅读、教程以及参加研讨会和会议。主要赞助人苏珊·席尔瓦博士将为候选人在研究设计、统计和临床试验管理方面的培训提供建议。联合发起人约翰·马奇博士将在候选人从临床试验人员过渡到具有研究设计和先进统计方法专业知识的独立研究员时提供监督，并将在他担任首席研究员的NIMH资助的多个试验中与候选人合作。共同赞助人萨蒂什·莱恩加博士、詹姆斯·罗雄博士、玛丽·戴维迪安博士和托马斯·滕博士将监督候选人在与其专业领域相关的先进数据分析方法方面的培训。候选人将使用来自NIMH资助的两个儿科比较治疗试验的可用数据集来解决与临床医生和个人做出有关患者护理决策的直接相关的研究问题。候选人研究议程的主要目的是应用和扩展适用于从复杂的多部位儿科比较治疗试验中得出因果推断的数据分析方法[例如，编译器平均因果效应(CAGE)和结构平均模型(SMM)j]。研究计划还广泛地侧重于应用先进的定量方法来分析临床试验数据。这些分析将有助于该领域更好地了解与儿童焦虑症成功的循证治疗相关的基本治疗要素和机制。补充的职业发展和研究计划将有助于将候选人定位为一名独立的生物统计研究员，他将能够在应用定量和临床试验方法学领域做出独到的贡献，并成为多学科研究团队的统计和方法学顾问，并在应用定量方法学和临床试验方法学的科学领域做出独到的贡献。

DESCRIPTION (provided by applicant): It is widely acknowledged there are too few biostatisticians well-trained in the methods and content of psychiatric research. No where is this more true than in the area of pediatric treatment outcome studies. This Mentored Patient-Oriented Research Career Development Award (K23) is designed to provide the candidate, who already has substantial expertise as a pediatric clinical trialist, with advanced training in statistical and research methodology. The overall goal is to establish the candidate as an independent biostatistical investigator who can serve at the interface of research design, implementation, and statistical methods in pediatric interventions research and contribute to the development of new knowledge and new statistical and research methods. Exemplary mentors, with complementary skills, will advise and supervise the candidate's didactic agenda, which includes formal coursework in advanced biostatistics, directed readings, tutorials, and participation in seminars and conferences. Primary sponsor, Dr. Susan Silva, will advise the candidate's training in the area of research design, statistics, and clinical trails management. Co-sponsor, Dr. John March, will provide oversight as the candidate transitions from clinical trialist to an independent investigator with expertise in research design and advanced statistical methods, and will collaborate with the candidate on multiple NIMH- funded trials for which he is the Principal Investigator. Co-sponsors Drs. Satish lyengar, James Rochon, Marie Davidian, and Thomas Ten Have will supervise the candidate's training in advanced data analytic methods related to their area of expertise. The candidate will use available data sets from two NIMH-funded pediatric comparative treatment trials to address research questions that are of direct relevance to clinicians and individuals making decisions regarding patient care. The primary aim of the candidate's research agenda is to apply and extend data analytic methods appropriate for drawing causal inference from complex multisite pediatric comparative treatment trials [e.g., compiler average causal effect (CAGE) and structural mean model (SMM)j. The research plan also focuses broadly on applying advanced quantitative methods for analyzing clinical trials data. These analyses will help the field better understand the essential treatment elements and mechanisms associated with successful evidence-based treatments for pediatric anxiety disorders. The complementary career development and research plans will help position the candidate as an independent biostatistical investigator who will be able to make original and unique contributions in the areas of applied quantitative and clinical trials methodologies and serve as a statistical and methodological consultant to multidisciplinary research teams and make original and unique contributions to the scientific field of applied quantitative methodology and clinical trials methodologies.