Sample Size Planning of Clinical Trials, Introduction

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Presentation transcript:

Sample Size Planning of Clinical Trials, Introduction Dr. Gu Ming Gao Department of Statistics, CUHK For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Clinical trials are important in Evidence-based medicine Compare different treatments, quality of care or medicine, study into Chinese medicine Development, improvement of clinical practice guidelines Finding new factors and relationships which effect our health, drug development For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Two types of clinical trials Trials involve immediate responses Trials involve time-to-event responses The statistical procedures for handling those two types of data are very different Survival analysis Cox regression model For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Trials with immediate responses, comparing two samples Control vs. treatment Assumptions on the distribution Statistic: T-test statistic Type-I error and power Formula for sample size calculations under normal distribution assumptions For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Trials with time-to-event responses, comparing two samples Normal assumption on the distribution of the life-time is always violated We usually only have vague ideas about the underlying distribution functions No exact simple size calculation formula Uses of Monte-Carlo simulations For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Censoring Censoring caused by patient withdraw Censoring by other illness, competing risks Censoring by design of the trial Censoring caused by pre-scheduled date of termination of the trial For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Noncompliance Noncompliance is also called crossovers Drop-in rates Drop-out rates For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Log-rank statistics An intuitive view of Log-rank statistics: boys computing with girls General payment functions, Wilcoxon statistic Rank statistics are more robust than the t-test, less assumptions on the distribution functions The Log-rank statistics are very powerful For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Sequential monitoring of clinical trials Trials are monitored for safety reasons Group sequential trials, planning and design Complications caused: what is my type-I error and what is my power? For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

Different stopping rules in clinical trials Pocock boundary O’Brein-Fleming boundary Haybittle boundary Use function approach by Lan & DeMetes Complications caused different stopping rules For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018

The DSSP program Use DSSP program to find the power of the test for a given sample size Use the DSSP program for sample size. The program is window based and easy to use Intro to the program will be given next week Available at http://www.sta.cuhk.edu.hk/minggao/ctrials.asp For PgD / MSc Programme in Epidemiology & Biostatistics, CUHK 11/20/2018