Approaches to reduce the estimated sample size in clinical trials – A checklist

The following approaches can be considered to reduce the estimated sample size in clinical trials: Objective Choose another objective, e.g. Reduce objectives of study, e.g. phase 2 instead of phase 3, fewer primary/key secondary endpoints, descriptive instead of inferential Change target concept, e.g. physical instead of psychological QOL Operating characteristics: Relax type 1 error Relax…

Comments on the 3-year work plan
for the Methodology Working Party (MWP) of the EMA

The Methodological Working Party (MWP) of the EMA has published a draft 3-year work plan [1] on October 30, 2023, which I have commented below. Content L43-45: The strategic goals of The Methodological Working Party (L43-45) are supported. However, innovation and best-practice efforts should be complemented by efforts to provide a standardized, “same practice” harmonized…

Macro “ExtractComments” –
Export comments from MS Word to MS Excel

Introduction The development of clinical documentation in drug development requires interdisciplinary communication and collaboration regarding complex, comprehensive text documents over multiple revision cycles resulting in documents of 50 to 200 pages with several hundreds of comments and replies. Reviewing such documents is time-consuming because it is often not clear: To answer these questions, text-related comments…

Hype, not hope – A comment on Ruberg et al. (2023) “Application of Bayesian approaches in drug development: starting a virtuous cycle”

Wilmar Igl, PhD1 & John Constant, PhD2 1Biostatistics Consulting Services, ICON PLC, Sweden2Scientific Affairs, ICON PLC, Canada “It’s about getting things down to one number.Using the stats the way we read them,we’ll find value in players that no one else can see.”Peter Brand, Moneyball [1] Introduction Ruberg et al.2 have recently published a perspective on…

Federated Analysis with R/DataSHIELD –
Installation, Data Import, Data Analysis [Tutorial]

Introduction Federated Analysis (FA) has received rapidly increasing attention over the last three years in the scientific literature (Figure 1). It describes the centralized analysis of de-centralized databases while preserving the privacy of personal data. FA was proposed for the identification of rare adverse events in international post-marketing studies to overcome legal barriers [1]. This…

The Dandelion Hub – A (technical) review of an open-source web app (R/Shiny, MariaDB) to support global civil-rights movements

In the following blog I am taking a critical look a the The Dandelion Hub platform (https://dhub.global), which seems a promising open-source project to support the global civil-right movements, or in the words of the developers “to create a movement of movements for science, people power, and a livable future”. Can’t be wrong, can it?…

Tech suffers from lack of humanities, says Mozilla head

Considering myself an academic with a social science (MSc, PhD in psychology) and tech (MSc Medical Statistics, Post-doc in genomics) background, I find it highly interesting that Mitchell Baker, head of the Mozilla Foundation (cf Firefox browser), is arguing that hiring employees who mainly come from STEM (Science, technology, engineering and maths) will produce a…