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: 8149979
• 负责人: WILLIAM G BARSAN
• 金额: $ 71.34万
• 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 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8149979
• 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; 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; public health relevance; response; simulation; success; treatment trial; willingness

DESCRIPTION (provided by applicant): 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. PUBLIC HEALTH RELEVANCE: 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.

描述（由申请人提供）：使用随机临床试验评估药物和医疗设备是一个复杂且日益昂贵的过程，这受到令人失望的成功率困扰。临床试验方法的创新，特别是自适应临床试验，可以解决常规试验设计的许多局限性，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。包括统计方法和组织过程在内的最佳方法 - 是否尚不清楚，适应性的，自适应临床试验的协作开发和实施确认。该项目将解决该差距。 NINDS支持的神经系统紧急情况治疗试验（NETT）NetWorl <将用作研究这些试验发展的“实验室”。拟议中的worl <的总体目标是说明和探索如何最好地使用适应性临床试验设计来改善对药物和医疗设备的评估，并使用混合方法来表征和理解关键阶层的信念，观点和关注点在开发过程中和之后。第一个具体目的是设计四个创新的自适应临床试验，以评估用于护理急性神经疾病或损伤患者的药物和设备。自适应方法将包括频繁的临时分析，基于概率的决策规则，UNL结果的纵向建模以及响应自适应随机化。第二个具体目的是识别和定性地表征与接受和实施自适应临床试验有关的L <EY步骤和障碍。在主要利益相关者接受一种或多种自适应临床试验设计之后，这些设计将被纳入提交资金和同行评审的赠款申请中。 公共卫生相关性：随机临床试验是监管机构和临床医生接受新疗法的基石。目前，此过程缓慢而繁琐，可能会导致关于新疗法效用的错误结论。该项目将确定高质量，验证性，自适应临床试验的设计和启动的有效过程。这些方法可用于通过提高药物和设备开发的速度和质量来加速有希望的新疗法，保护资源并支持公共卫生。