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There is a growing interest in the implementation of platform trials, which provide the flexibility to incorporate new treatment arms during the trial and the ability to halt treatments early based on lack of benefit or observed…

统计方法学 · 统计学 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

There is growing interest in platform trials that allow for adding of new treatment arms as the trial progresses as well as being able to stop treatments part way through the trial for either lack of benefit/futility or for superiority. In…

统计方法学 · 统计学 2021-12-14 Peter Greenstreet , Thomas Jaki , Alun Bedding , Chris Harbron , Pavel Mozgunov

Platform trials offer a framework to study multiple interventions in a single trial with the opportunity of opening and closing arms. The use of a common control in platform trials can increase efficiency as compared to individual control…

统计方法学 · 统计学 2023-02-10 Quynh Nguyen , Katharina Hees , Benjamin Hofner

Platform trials gained popularity during the last few years as they increase flexibility compared to multi-arm trials by allowing new experimental arms entering when the trial already started. Using a shared control group in platform trials…

Multi-Arm Multi-Stage (MAMS) platform trials are an efficient tool for the comparison of several treatments. Suppose we wish to add a treatment to a trial already in progress, to access the benefits of a MAMS design. How should this be…

应用统计 · 统计学 2021-05-25 Thomas Burnett , Franz König , Thomas Jaki

Platform trials are randomized clinical trials that allow simultaneous comparison of multiple interventions, usually against a common control. Arms to test experimental interventions may enter and leave the platform over time. This implies…

统计方法学 · 统计学 2024-04-12 Marta Bofill Roig , Ekkehard Glimm , Tobias Mielke , Martin Posch

Though platform trials have been touted for their flexibility and streamlined use of trial resources, their statistical efficiency is not well understood. We fill this gap by establishing their greater efficiency for comparing the relative…

统计方法学 · 统计学 2022-01-28 Tzu-Jung Huang , Alex Luedtke , the AMP Investigators Group

Platform trials can evaluate the efficacy of several treatments compared to a control. The number of treatments is not fixed, as arms may be added or removed as the trial progresses. Platform trials are more efficient than independent…

Shared controls in platform trials comprise concurrent and non-concurrent controls. For a given experimental arm, non-concurrent controls refer to data from patients allocated to the control arm before the arm enters the trial. The use of…

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

统计方法学 · 统计学 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

统计方法学 · 统计学 2021-08-31 Elja Arjas , Dario Gasbarra

The analysis of platform trials can be enhanced by utilizing non-concurrent controls. Since including this data might also introduce bias in the treatment effect estimators if time trends are present, methods for incorporating…

统计方法学 · 统计学 2026-05-21 Pavla Krotka , Martin Posch , Marta Bofill Roig

Background: Experimental treatments pass through various stages of development. If a treatment passes through early phase experiments, the investigators may want to assess it in a late phase randomised controlled trial. An efficient way to…

The standard paradigm for confirmatory clinical trials is to compare experimental treatments with a control, for example the standard of care or a placebo. However, it is not always the case that a suitable control exists. Efficient…

统计方法学 · 统计学 2024-10-29 Thomas Burnett , Thomas Jaki

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Major Depressive Disorder (MDD) is one of the most common causes of disability worldwide. Unfortunately, about one-third of patients do not benefit sufficiently from available treatments and not many new drugs have been developed in this…

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

统计方法学 · 统计学 2022-11-14 Martin Law

Clinical trials with a hybrid control arm (a control arm constructed from a combination of randomized patients and real-world data on patients receiving usual care in standard clinical practice) have the potential to decrease the cost of…

统计方法学 · 统计学 2021-08-20 Joanna Harton , Brian Segal , Ronac Mamtani , Nandita Mitra , Rebecca Hubbard

A platform trial with a master protocol provides an infrastructure to ethically and efficiently evaluate multiple treatment options in multiple diseases. Given that certain study drugs can enter or exit a platform trial, the randomization…

统计方法学 · 统计学 2025-07-15 Tianyu Zhan , Jane Zhang , Lei Shu , Yihua Gu

Platform trials have become increasingly popular for drug development programs, attracting interest from statisticians, clinicians and regulatory agencies. Many statistical questions related to designing platform trials - such as the impact…

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