From Bench to Bedside to Community:Statistical Issues in the Early to Late Assess

描述(由申请人提供)：持续需要开发科学合理、有效、道德和安全的方法来评估人类干预。这项建议的动机是通过持续的一系列有针对性的研讨会，促进临床试验的统计设计、实施和生物统计分析方法的进步。每一次研讨会都将完全集中在普遍接受的干预发展阶段定义范围内的一个领域(动物和实验室研究、早期人类和剂量发现研究、早期疗效和过渡研究、最终疗效研究、上市后研究)。 每个研讨会的主题将每年根据一个地区是否适合从集中的研讨会中受益，以及它对临床试验实践的潜在影响来选择。我们期望，专题讨论会将解决问题，并提出易于跨疾病领域翻译的解决方案，这在历史上一直是生物统计学方法的情况，无论是哪个疾病领域激发了它的发展。每一次研讨会都期望在与所考虑的问题相关的临床、伦理和实践环境的背景下审查统计方法方法。会议的结构模式大致包括将当时特定领域的有说服力的思想家聚集在一起进行正式演讲，创造一个积极讨论的环境，重要的是，提供快速发布(目标，会议一年后)。我们的具体目标是：开发一系列临床试验研讨会，每个研讨会都侧重于干预评估的五个阶段之一的一个领域；召集顶尖科学家参与介绍和讨论这些领域的最新进展和预期发展；组织者进行会前审查，以便在目标领域建立争议和平衡，并将这些纳入计划；寻找既有专业知识又有承诺参加研讨会的受众；尽快在同行评议期刊上发表研讨会记录，包括讨论。我们的期望是，实现这一系列会议的这些目标将不仅仅是传递信息，还能通过对比想法和讨论内容来扩大现有的最先进信息。

DESCRIPTION (provided by applicant): There is a continuing need for the development of scientifically sound, efficient, ethical, and safe methods for the assessment of human interventions. The motivation for this proposal is to facilitate advances in approaches to the statistical design, implementation and biostatistical analysis of clinical trials, through a continuing series of targeted symposia. Each symposium will focus entirely on an area approximately within commonly accepted definitions of stages in the development of an intervention (animal and laboratory studies, early human and dose finding studies, early efficacy and transition studies, definitive efficacy studies, post marketing studies). The topic for each symposium will be chosen annually, based on the suitability of an area to benefit from a concentrated symposium, and its potential impact on the practice of clinical trials. We expect that the symposia will address problems and produce solutions that translate readily across disease areas, which historically has been the case for biostatistical methodology, regardless of the disease area that inspired its development. Every symposium is expected to examine statistical methodological approaches in the context of the clinical, ethical and practical environments attendant to the problems being considered. The structural model for the conference roughly involves getting the cogent thinkers of the day in a particular area together to make formal presentations, create an environment for active discussion, and importantly, provide rapid publication (goal, one year from conference). Our specific objectives are to: develop a series of clinical trials symposia, each focused on an area within one of the five stages of assessment of an intervention; bring together leading scientists to be involved in the presentation and discussion of the state of the art and expected developments in these areas; to have pre-conference review by organizers in order to establish controversy and equipoise in the targeted area and incorporate such into program; to seek an audience with both expertise and commitment to participate in the symposia; to publish the proceedings of the symposia, including discussion, as quickly as possible in a peer reviewed journal. Our expectation is that meeting these objectives for the conference series will provide more than just a transfer of information, allowing for an expansion of the available state-of-the-art information through the contrast of ideas and the elements of discussion.