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.
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