Clinical Trials Development Resource for Hematologic Disorders (U24)

描述（由申请人提供）：RTI International正在提交血液病临床试验开发资源（U24）的申请，RFA-HL-12-016。该资源将支持研究人员开展临床试验，这些研究人员对血液疾病的新治疗方法有想法，但缺乏设计试验以测试这些想法所需的资源。RTI资源团队包括具有不同背景的经验丰富的个人，涵盖多中心临床试验的各个方面。我们的生物统计学家与临床研究人员在试验设计、监测和分析方面进行了广泛的合作，包括血液病（镰状细胞病、血友病和TTP）的试验。临床研究管理人员将对试验实施的实际方面（研究地点和受试者的招募，操作手册和其他研究材料的开发）提出建议。该团队包括为数据输入和数据管理系统提供建议的专家程序员，与FDA和其他监管机构互动的经验丰富的监管专家，以及为临床试验的道德行为提供建议的生物伦理学家。我们的团队还包括几位在设计各种血液疾病的临床试验方面经验丰富的血液学顾问。最后，我们的团队包括具有丰富经验的团队，提供试验设计和管理方面的正式培训。利用这种基础广泛的专门知识，RTI资源将实现两个广泛的目标。首先，来自资源的建议将为R34/U34研究人员提供他们在试验计划阶段结束时制定高质量，有效的建议所需的工具和知识。此外，这些建议将促使R34/U34研究人员制定可行、科学合理、设计良好的方案，这些方案可以通过R01/U01机制有效实施。我们将提供培训和指导，以提高研究人员的设计技能，以便在资源期限之外改进未来的研究。Donald Brambilia博士将担任资源首席调查员，他在临床试验领导方面拥有超过25年的统计学家经验，其中包括20年的血液学研究，与全国许多领先的血液学研究专家合作。Nigel Key博士是我们的首席血液学顾问，与其他5位血液学专家合作支持U34/R34研究人员。我们还包括在临床试验设计的各个方面都有经验的人员，

DESCRIPTION (provided by applicant): RTI International is submitting this application for the Clinical Trials Development Resource for Hematologic Disorders (U24), RFA-HL-12-016. The Resource will support the development of clinical trials by investigators who have ideas for new treatments of blood disorders but lack access to the resources needed to design trials to test these ideas. The RTI Resource team includes highly experienced individuals with diverse backgrounds that cover all aspects of multicenter clinical trials. Our biostatisticians have collaborated extensively with clinical investigators on trial design, monitoring, and analysis, including trials in hematological diseases (sickle cell disease, hemophilia and TTP). Clinical study managers will advise on the practical aspects of trial implementation (recruitment of study sites and subjects, and development of manuals of operations and other study materials). The team includes expert programmers to advise on data entry and data management systems, regulatory specialists with experience interacting with FDA and other regulatory agencies, and bioethicists to advise on ethical conduct of clinical trials. Our team also includes several hematology consultants experienced in designing clinical trials for a wide range of blood disorders. Finally, our team includes groups with considerable experience offering formal training on trial design and management. Drawing on this broad-based expertise, the RTI Resource will accomplish two broad Aims. First, advice from the Resource will provide the R34/U34 investigators with the tools and knowledge that they need to develop high-quality, effective proposals at the end of the trial planning phase. Also, that advice will lead to development by the R34/U34 investigators of feasible, scientifically sound, well-designed protocols that can be implemented efficiently through an R01/U01 mechanism. We will offer training and mentoring to improve the design skills of the investigators so as to improve future research studies beyond the term of the Resource. Scientific leadership will be provided Dr. Donald Brambilia as the Resource Principal Investigator who has over 25 years of experience as a statistician in leadership of clinical trials including 20 years in hematological studies working with many of the leading research hematologists in the nation. Dr. Nigel Key is our lead hematology consultant cooperating with 5 other hematologists to support the U34/R34 investigators. We also include personnel with experience in all aspects for clinical trials design, development, implementation and operations. (End of Abstract)