System for Cost Effective Clinical Trial Design

该项目的长期目标是开发软件，以促进成本效益高的临床试验的设计。1999年，制药业和NIH在临床试验上花费了超过230亿美元。临床试验设计理论和优化算法的发展为更具成本效益的设计打开了可能性，这些设计可以以更低的总成本、更短的时间和/或需要更少的患者来执行。拟议的成本效益试验系统(SCET)将是一个软件包，通过比较替代试验设计的功率、样本量要求和成本来指导试验设计者。这些方法在整个医学研究中都未得到充分利用，但特别适用于治疗方案相对较短、终点确定较快的试验，如许多癌症治疗试验。SCET第二阶段的目标是构建系统，按照FDA的软件验证法规对其进行验证，在一系列目标客户组织中执行Beta测试，并使用Beta测试结果来生产适销对路的版本。拟议的商业应用：该软件系统的潜在市场包括世界上几乎每一家制药公司(每家公司都有多个许可证)、每一家参与临床试验的生物技术公司、每一家参与临床试验设计或实施的合同研究机构、NIH赞助的多中心临床试验协调中心、进行临床试验的个别大学研究人员、设计临床试验的个别生物统计顾问，以及进行动物研究的农业企业和研究人员。

The long-term objective of this project is to develop software to facilitate the design of cost effective clinical trials. In 1999, the pharmaceutical industry and NIH spent more than $23 billion on clinical trials. Developments in clinical trial design theory, and in optimization algorithms have opened possibilities for more cost- effective designs that can be executed for lower total cost, over shorter periods of time and / or requiring fewer patients. The proposed System for Cost Effective Trials (SCET) will be a software package to guide the trial designer through comparisons of the power, sample size requirements, and cost of alternate trial designs. These methods are under-used throughout medical research, but are particularly applicable to trials with relatively short treatment regimens and rapid ascertainment of endpoints, such as many cancer treatment trials. The aims of SCET Phase II are to build the system, validate it in compliance with FDA regulations for software validation, perform Beta testing at a range of target client organizations, and use the Beta test findings to produce a marketable release. PROPOSED COMMERCIAL APPLICATION: The potential market for this software system includes virtually every pharmaceutical company in the world (multiple licenses to each), every biotech company involved in clinical trials, every contract research organization involved in the design or conduct of clinical trials, coordinating centers of NIH-sponsored multi-center clinical trials, individual university-based investigators who conduct clinical trials, individual biostatistical consultants who design clinical trials, and agricultural businesses and researchers that conduct animal research.