Increasing the Quality and Efficiency of Clinical Trials (R18)

摘要 自1962年以来，国会(Kefauver-Harris修正案)要求FDA有责任确保 在美国被批准用于医疗的每一种药物都被证明是安全有效的临床试验 构成了理解与使用 医疗产品。然而，近几十年来，临床试验变得昂贵得令人望而却步，进展缓慢 结业，而且往往招生不足，给医生和患者带来了关于 哪些治疗方法对哪些患者有效。通过提高效率和质量来解决这些差距 临床试验是临床试验转化倡议(CTTI)的首要目标。 自2007年成立以来，CTTI-由杜克大学和美国食品和药物管理局共同创立 (FDA)，并由杜克大学主办-寻求确定和促进将提高质量和 临床试验的效率。CTTI项目通过以下方式解决广泛的试验设计和操作问题 注重质量、相关性和效率。这些问题的例子包括知情同意、主体 招聘、使用中央机构审查委员会(IRBs)、研究新药(IND)安全报告、 临床试验质量、操作和监测以及抗菌药物开发，所有这些都是直接 与FDA的使命相关(附录A：CTTI项目组合)。临床试验包括大量的 利益相关者被医学研究所统称为临床试验企业(CTE)。所有CTE 在反恐倡议项目中有利益攸关方的代表，以便能够执行建议并达成共识 在提高质量和效率方面的潜力方面可以达成共识。CTTI的战略 涉及三管齐下的方法：1)逐步改进临床试验方法和流程；2) 更好地了解国家研究组合，以改进试验与公共卫生的匹配 优先事项；以及3)推动变革，提供的不仅仅是渐进式的改进 (附录B：战略计划)。反恐执行委员会的工作不仅限于建议，还包括创建 便利采纳其建议的工具和其他方法。 这项提案的总体目标有两个： 1)维持可持续的行政和科学基础设施，为实现以下目标指明道路 通过利用广泛和透明的公共-私营部门，提供高质量、高效和有影响力的临床试验 合作伙伴关系(PPP)，提供减少研究障碍的最佳做法和建议 进行临床试验；以及 2)随后传播和便利通过已发布的建议和调查结果。

Abstract Since 1962, when Congress (Kefauver-Harris Amendment) charged the FDA with the responsibility to ensure that each pharmaceutical approved for medical use in the US be shown to be safe and effective, clinical trials have formed the foundation for understanding the balance of benefits and risks associated with the use of medical products. In recent decades, however, clinical trials have become prohibitively expensive, slow to finish, and are often under-enrolled, presenting physicians and patients with major gaps in knowledge about what treatments work for which patients. Addressing these gaps by improving the efficiency and quality of clinical trials is the overarching purpose of the Clinical Trials Transformation Initiative (CTTI). Since its inception in 2007, CTTI—cofounded by Duke University and the U.S. Food and Drug Administration (FDA), and hosted by Duke—has sought to identify and promote practices that will improve the quality and efficiency of clinical trials. CTTI projects address a broad range of trial design and operational issues by focusing on quality, relevance, and efficiency. Examples of these issues include informed consent, subject recruitment, use of central institutional review boards (IRBs), investigational new drug (IND) safety reporting, clinical trial quality, operations and monitoring, and antibacterial drug development, all of which are directly relevant to FDA's mission (Appendix A: CTTI Project Portfolio). Clinical trials comprise a vast number of stakeholders collectively identified by the Institute of Medicine as the Clinical Trials Enterprise (CTE). All CTE stakeholders are represented in CTTI projects, so that recommendations can be implemented and consensus can be reached regarding the potential to encompass improvements in quality and efficiency. CTTI's strategy involves a three-pronged approach: 1) incremental improvement to clinical trial methods and processes; 2) better understanding of the national research portfolio to improve the matching of trials to public health priorities; and 3) a push for transformational change that will provide more than incremental improvements (Appendix B: Strategic Plan). CTTI's work is not limited to recommendations, but also includes the creation of tools and other approaches to facilitate adoption of its recommendations. The overarching goals of this proposal are twofold: 1) To maintain a sustainable administrative and scientific infrastructure that will show the path forward to high-quality, efficient, and influential clinical trials by leveraging a broad and transparent public-private partnership (PPP) to provide best practices and recommendations for reducing research impediments in the conduct of clinical trials; and 2) To subsequently disseminate and facilitate adoption of issued recommendations and findings.