Small Populations SIG

Purpose

The SIG “Small populations” provides a forum for identifying and discussing statistical methodology related to clinical development of treatments in small populations, and for sharing experiences.

Objectives

Our objectives include:

  • Exchange information & share case studies of statistical/methodological challenges in the area of small populations
  • Collaborate and discuss strategies and methodology
  • Support the promotion of new methodology
  • Organise and/or participate in workshops in the area of small populations

Who we are

NameCompany/Institution
Tal Otiker (co-chair)GSK
Aysun Cetinyurek-yavuz (co-chair)Radboud UMC
Alexander PieperChrestos

Alexia Kakourou

GSK

Anais AndrillonSaryga

Arnaud Monseur

Pharmalex

Audrey Iris D'Abrigeon

Servier

Carol Reid

Roche

Elodie BlondiauxVenn Life Sciences

Giles Partington

Phastar

Guido Thoemmes

Merck

Jacquie Christie

Sobi

Joanna in t Hout

Radboud UMC

Maeva Dupuis

Alexion

Maya Marintcheva-Petrova

Danone

Tim Friede

University Medical Center Göttingen

Zerong Chen

Imperial College London

 

How to get in touch

For further information, or to join the Small Population SIG, please contact the co-chairs:

Tal Otiker (GSK), tal.x.otiker@gsk.com

Aysun Cetinyurek Yavuz (Radboud University Medical Center), aysun.cetinyurek-yavuz@radboudumc.nl

Resources

Points to consider in the design and analysis of clinical trials in small populations

Diseases that occur in populations with a low prevalence or in vulnerable populations lead to limitations in the number of available patients for drug development. In such a setting, when sample sizes for clinical development are limited, one usually speaks of small populations. Drug development in small populations poses some special challenges. This applies above all to the statistical planning of the clinical studies, their statistical evaluation and interpretation as well as decision-making. The challenges can often only be addressed using innovative statistical methods and data analysis approaches. This points-to-consider document is aimed at statisticians who are going to work in this exciting field intending to give them an initial introduction and to sensitize them to the peculiarities of small populations. Basic points to consider in clinical development for small populations are addressed and potential useful statistical approaches are proposed.

Historical controls, meta-analytic priors, and extrapolation - some useful publications for small populations

The SIG Small Populations has been working to identify publications that would be useful for statisticians working in studies with small populations. The topics that were chosen as of interest were extrapolation, historical controls, and meta-analytic priors.

Subgroups were set up to work on each of these topics. A literature search was performed to identify publications of potential interest. These were then reviewed within the subgroups and those deemed most relevant and useful were identified. The attached slides list the publications recommended for each topic with a brief description of the content and the reason for the recommendation.

Upcoming Events

PSI Introduction to Industry Training (ITIT) Course - 2025/2026

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 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 Book Club Webinar: Atomic Habits - The Science of Getting Your Act Together

The book club’s usual focus is to read and discuss professional development books. In this short format event you can more easily develop you career without the commitment of reading the whole book - simply listen to the 1-hour long podcast before joining the interactive session on 21 May.

PSI Webinar: Methods and tools integrating clinical trial evidence with historical or real-world data, Bayesian borrowing, and causal inference

This webinar is organised by the RWD SIG and the Historical Data SIG. We will review recent methods, applications, and tools of integrating subject-level-data from clinical trial with external data using Bayesian methods and/or causal inference methods.

PSI Career Young Virtual Event (Q2 2025)

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

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