Biopharmaceutical Software Working Group

Category: Clinical trial simulations.

Working group

The Biopharmaceutical Software Working Group is sponsored by the Biopharmaceutical Section of the American Statistical Association. The Working Group is planning to solicit ideas for new software tools with biopharmaceutical applications (including R-based software and free Windows-based software packages with a graphical user interface), set up a network of testers and reviewers, and ultimately cultivate a community focused on the development of software tools for biopharmaceutical statisticians by biopharmaceutical statisticians.


Core team: Neby Bekele (Gilead), Alun Bedding (Roche), Thomas Brechenmacher (IQVIA), Fei Chen (J&J), Greg Cicconetti (AbbVie), Alex Dmitrienko (Mediana) [Chair], Jessica Hu (FDA), Dacheng Liu (Boehringer Ingelheim), Gautier Paux (Sanofi), Kyle Wathen (Gilead) [Vice Chair].


The current set of the Working Group’s goals includes:

  • Identify the gaps in the availability of software tools that facilitate the design, conduct and analysis of clinical trials with emphasis on tools that enable efficient clinical trial simulations.
  • Develop best practices for clinical trials simulations.
  • Develop general guidelines and more detailed specifications for statistical software tools, including software testing/validation guidelines.
  • Identify and collaborate with software developers to build free statistical software tools based on the specifications created by the working group.
  • Create a network of reviewers for testing the statistical software tools designed by the working group and gathering feedback to inform the next steps of the software development process.
  • Develop documentation and other instructional materials, including free training videos on the Biopharmaceutical Section’s YouTube channel, for the software tools.
  • Prepare publications, organize conference presentations and develop training courses to introduce the software tools designed by the working group to the biostatistical and clinical trial communities.

The goals are expected to be reviewed and revised on a regular basis.

Software tools

Mediana package (R package for general simulation-based power and sample size calculations in fixed-sample trials).
Mediana Designer (free Windows-based software tool for traditional and simulation-based power/sample size calculations in fixed-sample and group-sequential trials).
OCTOPUS (R package for simulation of platform clinical trials, including designs with multiple interventions entering the trial).
MedianaDesigner (R package for efficient simulation-based power and sample size calculations in a broad class of early-stage and late-stage clinical trials).
MCPModPack (R package for a simulation-based design and analysis of dose-finding trials with normally distributed, binary and count endpoints using the MCPMod method).

Papers and presentations

Clinical trial simulation based on the Clinical Scenario Evaluation framework (presentation by Alex Dmitrienko at the Duke Industry Statistics Symposium, April 2019).
Biopharmaceutical Software Working Group (article by Alex Dmitrienko, J. Kyle Wathen, Gautier Paux, Thomas Brechenmacher, Kaushik Patra, B. Nebiyou Bekele and Dacheng Liu published in the Biopharmaceutical Report, Spring 2019).
Simulation design and reporting with applications to drug development (short course by Greg Cicconetti and Inna Perevozskaya presented at the ASA Conference on Statistical Practice, April 2019). Download the R code for this short course.
Simulation practices for adaptive clinical trial design in drug and device development (short course by Inna Perevozskaya and Greg Cicconetti presented at the ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop, September 2019).