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

从实验室到临床到社区：早期到晚期评估中的统计问题

• 批准号: 8197585
• 负责人: JONAS H ELLENBERG
• 金额: $ 3.38万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-12-17 至 2012-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8197585
• 关键词: Address; Animals; Area; Clinical; Clinical Trials; Communities; Development; Disease; Dose; Elements; Environment; Equipoise; Ethics; Goals; Human; Intervention; Journals; Laboratory Study; Methodology; Methods; Motivation; Peer Review; Publications; Publishing; Scientist; Series; Solutions; Staging; Structural Models; Text; Translating; base; bench to bedside; design; expectation; meetings; post-market; programs; sound; symposium; therapy development

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.

持续需要开发科学合理、高效、道德和安全的方法来评估人类干预措施。该提案的动机是通过一系列持续的有针对性的研讨会，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。每次研讨会将完全集中于干预措施开发过程中普遍接受的阶段定义范围内的一个领域（动物和实验室研究、早期人类和剂量探索研究、早期疗效和过渡研究、最终疗效研究、上市后研究）。 每次研讨会的主题每年都会根据某个领域是否适合从集中研讨会中受益及其对临床试验实践的潜在影响来选择。我们期望研讨会将解决问题并提出易于跨疾病领域转化的解决方案，这在历史上一直是生物统计方法的情况，无论激发其发展的疾病领域如何。每一次研讨会都应在临床、伦理和实践环境的背景下审查统计方法学方法，以解决所考虑的问题。会议的结构模型大致包括将当今特定领域的有说服力的思想家聚集在一起进行正式演讲，创造积极讨论的环境，并且重要的是，提供快速出版（目标，会议后一年）。我们的具体目标是： 举办一系列临床试验研讨会，每个研讨会重点关注干预评估的五个阶段之一内的一个领域；汇集顶尖科学家，参与介绍和讨论这些领域的最新技术和预期发展；组织者进行会前审查，以便在目标领域建立争议并平衡，并将其纳入议程；寻找既具有专业知识又愿意参加研讨会的观众；尽快在同行评审期刊上发表研讨会记录，包括讨论内容。我们的期望是，实现会议系列的这些目标将提供的不仅仅是信息传输，还可以通过思想对比和讨论要素来扩展可用的最新信息。