Video-on-Demand Library

Play

This content is restricted to members of PSI. If you are already a member please login. To join PSI or to see all the benefits of membership click here.

PSI Conference 2019: Sample size calculation in non-inferiority trials with binary endpoint: scale matters

03 June 2019

Sample size calculation in non-inferiority trials with binary endpoint: scale matters

It is becoming increasingly difficult to justify testing the efficacy of a new treatments against placebo. Instead, active controlled trials are being used to test whether the new treatment has not much worse efficacy than an already known effective treatment. Choosing the margin defining ‘not much worse’ is a pivotal step in designing such non-inferiority trials. It is well known that the size of the margin strongly influences the required sample size. What is largely ignored however, is that also the scale of the margin (for binary endpoints absolute risk difference, risk ratio or odds ratio) strongly influences the power of the trial and thus the required sample size.

Using the same margin for the minimum allowed success proportion in the trial arm receiving the new treatment, but a different choice of the parameter used for evaluating the distance between this proportion and the success proportion in the active control arm could easily require two times larger sample size.

By exploring the exact size of the power differences in various scenarios, I will show in what kind of trials this scale choice is particularly influential for sample size requirement. Also I will show which parameter choice has highest power in which situation. Alongside, I will discuss other considerations than power when choosing between risk difference, risk ratio and odds ratio as primary estimand in a non-inferiority trial.  

Related Documents

Part of Collection(s)

Topic(s)

Upcoming Events

Joint PSI/EFSPI Visualisation SIG 'Wonderful Wednesday' Webinars

Our monthly webinar explores examples of innovative data visualisations relevant to our day to day work. Each month a new dataset is provided from a clinical trial or other relevant example, and participants are invited to submit a graphic that communicates interesting and relevant characteristics of the data.

PSI Webinar: Drug Development in Women's Health

The event will open with an overview on drug development in women’s health from a clinician perspective. This talk is followed by talks about statistical challenges when planning IVF studies and analysing the menstrual cycles.

PSI Webinar: Surrogacy

This webinar will provide an overview of surrogacy for licensing and reimbursement. In turn, the need of extensions of the SPIRIT and CONSORT statement will be defined and outlined, with case studies to support.

Joint PSI/EFSPI Pre-Clinical SIG Webinar: Virtual Control Groups in Toxicity Studies

Lea Vaas will present how replacement of concurrent control animals by Virtual Control Groups (VCGs) in systemic toxicity studies may help in contributing to the 3R's principle of animal experimentation: Reduce, Refine, Replace.

Joint PSI/EFSPI Data Science SIG Webinar: Developing Digital Measures (Digital Biomarkers) in Drug Development – insights from Mobilise D consortium

We will share a brief overview of what Mobilise D is and why it is an important step stone in the development of digital biomarkers, and how Mobilise D outputs can be relevant for you.

PSI Career Young Virtual Event (Q2 2024)

This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.

PSI EIWG Webinar: Estimands framework in action, the Alzheimer's disease case

The webinar will show how the estimand framework was applied in a Phase 3 trial in Alzheimer's disease.

PSI Webinar: Development of Gene Therapies: Strategic, Scientific, Regulatory and Access Considerations

This webinar will cover the history of cell/gene therapy, major regulatory advances, the role of quantitative scientists in drug development of these novel therapeutics, and discuss opportunities for innovation and product advancement.

PSI Career Young Virtual Event (Q3 2024)

This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.

PSI Introduction to Industry Training (ITIT) Course - 2024/2025

An introductory course giving an overview of the pharmaceutical industry and the drug development process as a whole, aimed at those with 1-3 years' experience. It comprises of six 2-day sessions covering a range of topics including Research and Development, Toxicology, Data Management and the Role of a CRO, Clinical Trials, Reimbursement, and Marketing.

Joint PSI/EFSPI Causal Inference SIG Webinar: Opportunities in applying a causal inference framework during the analysis of an RCT

This webinar will discuss opportunities in applying causal inference methodology during the analysis of RCT data.

PSI Career Young Virtual Event (Q4 2024)

This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.

Latest Jobs

Statistician, Data Science

We have exciting new opportunities available for Study Statisticians, Support Statisticians and Exploratory Statisticians at various levels.