An Alternative to Traditional Sample Size Determination for Small Patient Populations

نویسندگان

چکیده

The majority of phase III clinical trials use a 2-arm randomized controlled trial with 50% allocation between the control treatment and experimental treatment. sample size calculated for these normally guarantee power at least 80% certain Type I error, usually 5%. However, calculations, do not typically take into account total patient population that may benefit from investigated. In this article, we discuss two methods, which optimize designs, to maximize patients population. We continuous endpoint, when is small (i.e., rare diseases). One approach uses point estimate effect second distribution on in order uncertainty estimated effect. Both one-stage two-stage trials, using three different stopping boundaries are investigated compared, efficacy ethical measures. A completed anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis used demonstrate method. Supplementary materials article available online.

برای دانلود رایگان متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

منابع مشابه

Sample size determination for logistic regression

The problem of sample size estimation is important in medical applications, especially in cases of expensive measurements of immune biomarkers. This paper describes the problem of logistic regression analysis with the sample size determination algorithms, namely the methods of univariate statistics, logistics regression, cross-validation and Bayesian inference. The authors, treating the regr...

متن کامل

Comparing two Formulas of Sample Size Determination for Prevalence Studies

Background and purpose: Sample size and its determination is one of the most important problems in health researches. Calculating sample size for prevalence studies is one of the common questions of sample size topics. Minimum sample size with least complexity is desirable in order to achieve the basic goal of these studies. This study aims to compare two formulas of sample size calculation for...

متن کامل

Sample size determination.

Scientists who use animals in research must justify the number of animals to be used, and committees that review proposals to use animals in research must review this justification to ensure the appropriateness of the number of animals to be used. This article discusses when the number of animals to be used can best be estimated from previous experience and when a simple power and sample size c...

متن کامل

On Sample Size Determination

One of the questions most frequently asked of a statistician is: how big should the sample be? Managers are anxious to obtain an answer to this fundamental question during the planning phase of the survey since it impacts directly on operational considerations such as the number of interviewers required. There is no magical solution and no perfect recipe for determining sample size. It is rathe...

متن کامل

ذخیره در منابع من


  با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید

ژورنال

عنوان ژورنال: Statistics in Biopharmaceutical Research

سال: 2022

ISSN: ['1946-6315']

DOI: https://doi.org/10.1080/19466315.2022.2107565