Accelerating Drug and Device Evaluation through Innovative Clinical Trial Design

通过创新的临床试验设计加速药物和器械评估

• 批准号: 8323322
• 负责人: WILLIAM G BARSAN
• 金额: $ 56.69万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-09-27 至 2015-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8323322
• 关键词: Acute; Address; Applications Grants; Belief; Benzodiazepines; Cardiopulmonary Arrest; Categories; Characteristics; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Clinical Trials Design; Coma; Complex; Data; Data Protection; Decision Making; Development; Device or Instrument Development; Devices; Drug Evaluation; Emergency Care; Emergency treatment; Environment; Ethics; Evaluation; Feedback; Funding; Futility; Goals; Health Food; Human Resources; Infusion procedures; Injury; Instruction; Insulin; Ischemic Stroke; Laboratories; Lead; Medical; Medical Device; Methodology; Methods; Modeling; National Institute of Neurological Disorders and Stroke; Neurologic; Neurological emergencies; Outcome; Patient Care; Patients; Peer Review; Performance; Pharmaceutical Preparations; Pharmacotherapy; Plague; Principal Investigator; Probability; Process; Public Health; Randomized; Randomized Clinical Trials; Refractory; Relative (related person); Resources; Risk; Safety; Science; Spinal cord injury; Statistical Methods; Status Epilepticus; Survivors; United States Food and Drug Administration; United States National Institutes of Health; Work; base; design; drug development; effective therapy; human subject; improved; induced hypothermia; innovation; natural hypothermia; primary outcome; response; simulation; success; treatment trial; willingness

The use of randomized clinical trials to evaluate drugs and medical devices is a complex and increasingly expensive process, which is plagued by disappointingly low rates of success. Innovations in clinical trial methodology, and specifically adaptive clinical trials, can address many ofthe limitations of conventional trial designs and have the potential to improve the efficiency and success rates of clinical research, the quality of resulting scientific data, and the protection of human subjects. Optimal approaches-including both statistical methods and organizational processes-iforthe collaborative development and implementation of confirmatory, adaptive clinical trials remain unclear. This project will address that gap. The process of clinical trial development within the NINDS-supported Neurological Emergencies Treatment Trials (NETT) networl< will be used as the "laboratory" in which to study the development of these trials. The overall objective ofthe proposed worl< is to illustrate and explore how best to use adaptive clinical trial designs to improve the evaluation of drugs and medical devices and to use mixed methods to characterize and understand the beliefs, opinions, and concerns of key stal<eholders during and after the development process. The first specific aim is to design four innovative, adaptive clinical trials for the evaluation of drugs and devices used in the care of patients with acute neurological illness or injury. The adaptive methods will include frequent interim analyses, probability-based decision rules, longitudinal modeling of unl<nown outcomes, and response-adaptive randomization. The second specific aim is to identify and qualitatively characterize l<ey steps and barriers related to the acceptance and implementation of adaptive clinical trials. After acceptance of one or more adaptive clinical trial designs by key stakeholders, the designs will be incorporated into grant applications submitted for funding and peer review. RELEVANCE (See Instructions): Randomized clinical trials are the cornerstone for acceptance of new therapies by regulatory agencies and clinicians. Currently, this process is slow and cumbersome and may lead to false conclusions about the utility of new treatments. This project will identify effective processes for the design and initiation of high-quality, confirmatory, adaptive clinical trials. These methods can be used to accelerate the evaluation of promising new therapies, conserve resources and support public health by improving the rate and quality of drug and device development.

使用随机临床试验来评估药物和医疗器械是一项复杂且日益复杂的工作。 整个过程成本高昂，而且成功率低得令人失望。临床试验的创新 方法论，特别是适应性临床试验，可以解决传统方法的许多局限性 试验设计并有可能提高临床研究的效率和成功率， 所产生的科学数据的质量以及对人类受试者的保护。最佳方法-包括 统计方法和组织流程——为了协作开发和 验证性、适应性临床试验的实施仍不清楚。该项目将解决 那个差距。 NINDS 支持的神经紧急情况下的临床试验开发流程 治疗试验 (NETT) 网络<将用作研究治疗进展的“实验室” 这些试验。所提出的世界的总体目标是说明和探索如何最好地使用自适应 临床试验设计以改进药物和医疗器械的评价并使用混合方法 描述并理解关键利益相关者在项目期间和之后的信念、观点和担忧 发展过程。第一个具体目标是设计四个创新的、适应性的临床试验 评估用于治疗急性神经疾病或损伤患者的药物和设备。这 适应性方法将包括频繁的中期分析、基于概率的决策规则、纵向分析 未知结果的建模和响应自适应随机化。第二个具体目标是 识别并定性描述与接受和实施相关的步骤和障碍 适应性临床试验。关键接受一项或多项适应性临床试验设计后 利益相关者，这些设计将被纳入为资助和同行提交的赠款申请中 审查。 相关性（参见说明）： 随机临床试验是监管机构和机构接受新疗法的基石 临床医生。目前，这个过程缓慢且繁琐，并且可能导致关于效用的错误结论 新的治疗方法。该项目将确定设计和启动高质量、 验证性、适应性临床试验。这些方法可用于加速有前途的评估 新疗法，通过提高药品和药物的利用率和质量来节省资源并支持公共卫生 设备开发。

项目成果

Reflections on the Adaptive Designs Accelerating Promising Trials Into Treatments (ADAPT-IT) Process-Findings from a Qualitative Study.

对自适应设计加速有希望的治疗试验 (ADAPT-IT) 过程的反思——来自定性研究的发现。

• DOI: 10.3109/10601333.2015.1079217
• 发表时间: 2015
• 期刊: Clinical research and regulatory affairs
• 作者: Guetterman,TimothyC;Fetters,MichaelD;Legocki,LaurieJ;Mawocha,Samkeliso;Barsan,WilliamG;Lewis,RogerJ;Berry,DonaldA;Meurer,WilliamJ
• 通讯作者: Meurer,WilliamJ