Sample Size Method and Software Development in Survival Trial with a Cure Rate

描述(申请人提供)：设计随机对照临床试验的关键问题之一是计算研究所需的样本量，这在生存试验中更为复杂。现有的生存分析样本量计算方法和软件只考虑无治愈率的生存试验。就我们所知，没有现有的方法和软件可以提供在治疗中存在治愈率时的功率和样本量计算。为了填补这一空白，我们提出了用于混合固化模型中样本量计算的统计合理方法，其中包括现有的基于比例风险模型的样本量方法作为特例，我们将在此授权期内用R开发用户友好的软件，并提供完整的用户手册。因此，该方法将为研究人员探索生存试验中的最优样本量提供一种灵活的工具。用户界面友好的软件解决了样本量计算中的计算问题，便于研究人员和实践者将所提出的方法应用于研究。 公共卫生相关性：项目简介这个项目将为研究人员提供一个灵活的工具，在有或没有治愈率的情况下探索生存试验中的最佳样本量，并且用户友好的软件解决了所提出的方法在样本量计算中的计算问题。因此，研究者和实践者可以很容易地将所提出的方法应用于他们的研究中。

DESCRIPTION (provided by applicant): Abstract One of the crucial issues in designing randomized controlled clinical trials is to calculate sample size needed in the study, which is more complicated in survival trial. The existing method and software for the sample size calculation in survival analysis only consider the survival trial without a cure rate. To the best of our knowledge, no existing method and software can provide the power and sample size calculation when there is a cure rate in the treatment. In order to fill in this gap, we propose the statistically sound method for the sample size calculation in the mixture cure model, which include the existing sample size method based on the proportional hazards model as a special case, and we will develop the user friendly software in R during this grant period with a complete user manual. Therefore, the proposed method will provide a flexible tool for investigators to explore the optimal sample size in survival trial. The user friendly software solves the computational issue in the sample size calculation, and thus the researchers and practitioners can apply the proposed method in their study easily. PUBLIC HEALTH RELEVANCE: Project Narrative This project will provide a flexible tool for investigators to explore the optimal sample size in survival trial with or without a cure rate, and the user friendly software solves the computational issues in the sample size calculation from the proposed method. Thus the researchers and practitioners can apply the proposed method in their study easily.