Clinical trials with interim analyses: standardizing terminology to increase clarity

Elina Asikanius; Benjamin Hofner; Lisa V. Hampson; Gernot Wassmer; Christopher Jennison; Tobias Mielke; Cornelia Ursula Kunz; Kaspar  Rufibach

doi:10.1186/s13063-025-08942-3

Clinical trials with interim analyses: standardizing terminology to increase clarity

Elina Asikanius
, Benjamin Hofner
, Lisa V. Hampson
, Gernot Wassmer
, Christopher Jennison
, Tobias Mielke
, Cornelia Ursula Kunz
, Kaspar Rufibach

Finnish Medicines Agency
Paul-Ehrlich-Institut
Friedrich-Alexander-University Erlangen-Nürnberg (FAU) and Universitätsklinikum Erlangen
Advanced Methodology & Data Science, Basel,
RPACT GbR
Janssen-Cilag GmbH
Boehringer Ingelheim International GmbH
Product Development Data Sciences (MCO) Basel

Research output: Contribution to journal › Article › peer-review

5 Citations (SciVal)

Abstract

Interim analyses for group-sequential decision-making are prevalent in clinical trials. Methodology is well established and has been routinely implemented over the last decades. Still, confusions and uncertainties on aspects of how to operationalize and interpret interim analyses exist for many stakeholders. In this paper, a team of statisticians from the pharmaceutical industry, academia, and regulatory agencies provide a multi-stakeholder perspective on the key concepts behind interim analyses, with the aim to introduce standard terminology to mitigate misunderstandings and facilitate clearer discussions.

Original language	English
Article number	247
Number of pages	14
Journal	Trials
Volume	26
Issue number	1
Early online date	14 Jul 2025
DOIs	https://doi.org/10.1186/s13063-025-08942-3
Publication status	Published - 31 Dec 2025

Data Availability Statement

Code to reproduce all the computations in this paper is available as a quarto file on GitHub [26].

Funding

Not applicable.

Access to Document

10.1186/s13063-025-08942-3Licence: CC BY

Cite this

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AU - Wassmer, Gernot

AU - Jennison, Christopher

AU - Mielke, Tobias

AU - Kunz, Cornelia Ursula

AU - Rufibach, Kaspar

PY - 2025/12/31

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AB - Interim analyses for group-sequential decision-making are prevalent in clinical trials. Methodology is well established and has been routinely implemented over the last decades. Still, confusions and uncertainties on aspects of how to operationalize and interpret interim analyses exist for many stakeholders. In this paper, a team of statisticians from the pharmaceutical industry, academia, and regulatory agencies provide a multi-stakeholder perspective on the key concepts behind interim analyses, with the aim to introduce standard terminology to mitigate misunderstandings and facilitate clearer discussions.

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Clinical trials with interim analyses: standardizing terminology to increase clarity

Abstract

Data Availability Statement

Funding

Access to Document

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