Jay J H Park1, Ofir Harari2, Louis Dron2, Richard T Lester3, Kristian Thorlund2, Edward J Mills4. 1. Department of Medicine, Experimental Medicine, University of British Columbia, Vancouver, British Columbia, Canada; Cytel, Vancouver, British Columbia, Canada. 2. Cytel, Vancouver, British Columbia, Canada; Department of Health Research Methodology, Evidence, and Impact (HEI), McMaster University, Hamilton, Ontario, Canada. 3. Department of Medicine, Experimental Medicine, University of British Columbia, Vancouver, British Columbia, Canada. 4. Cytel, Vancouver, British Columbia, Canada; Department of Health Research Methodology, Evidence, and Impact (HEI), McMaster University, Hamilton, Ontario, Canada. Electronic address: Edward.mills@cytel.com.
Abstract
OBJECTIVES: The objective of the study was to outline key considerations for general clinical readers when critically evaluating publications on platform trials and for researchers when designing these types of clinical trials. STUDY DESIGN AND SETTING: In this review, we describe key concepts of platform trials with case study discussion of two hallmark platform trials in STAMPEDE and I-SPY2. We provide reader's guide to platform trials with a critical appraisal checklist. RESULTS: Platform trials offer flexibilities of dropping ineffective arms early based on interim data and introducing new arms into the trial. For platform trials, it is important to consider how interventions are compared and evaluated throughout and how new interventions are introduced. For intervention comparisons, it is important to consider what the primary analysis is, what and how many interventions are active simultaneously, and allocation between different arms. Interim evaluation considerations should include the number and timing of interim evaluations and outcomes and statistical rules used to drop interventions. New interventions are usually introduced based on scientific merits, so consideration of these merits is important, together with the timing and mechanisms in which new interventions are added. CONCLUSION: More efforts are needed to improve the scientific literacy of platform trials. Our review provides an overview of the important concepts of platform trials.
OBJECTIVES: The objective of the study was to outline key considerations for general clinical readers when critically evaluating publications on platform trials and for researchers when designing these types of clinical trials. STUDY DESIGN AND SETTING: In this review, we describe key concepts of platform trials with case study discussion of two hallmark platform trials in STAMPEDE and I-SPY2. We provide reader's guide to platform trials with a critical appraisal checklist. RESULTS: Platform trials offer flexibilities of dropping ineffective arms early based on interim data and introducing new arms into the trial. For platform trials, it is important to consider how interventions are compared and evaluated throughout and how new interventions are introduced. For intervention comparisons, it is important to consider what the primary analysis is, what and how many interventions are active simultaneously, and allocation between different arms. Interim evaluation considerations should include the number and timing of interim evaluations and outcomes and statistical rules used to drop interventions. New interventions are usually introduced based on scientific merits, so consideration of these merits is important, together with the timing and mechanisms in which new interventions are added. CONCLUSION: More efforts are needed to improve the scientific literacy of platform trials. Our review provides an overview of the important concepts of platform trials.
Authors: Suzan Cochius-den Otter; Jan A Deprest; Laurent Storme; Anne Greenough; Dick Tibboel Journal: Front Pediatr Date: 2022-04-15 Impact factor: 3.569
Authors: Elias Laurin Meyer; Peter Mesenbrink; Tobias Mielke; Tom Parke; Daniel Evans; Franz König Journal: Trials Date: 2021-03-04 Impact factor: 2.279
Authors: Jay J H Park; Robin Mogg; Gerald E Smith; Etheldreda Nakimuli-Mpungu; Fyezah Jehan; Craig R Rayner; Jeanine Condo; Eric H Decloedt; Jean B Nachega; Gilmar Reis; Edward J Mills Journal: Lancet Glob Health Date: 2021-05 Impact factor: 38.927
Authors: Gilmar Reis; Eduardo Augusto Dos Santos Moreira Silva; Daniela Carla Medeiros Silva; Lehana Thabane; Gurmit Singh; Jay J H Park; Jamie I Forrest; Ofir Harari; Castilho Vitor Quirino Dos Santos; Ana Paula Figueiredo Guimarães de Almeida; Adhemar Dias de Figueiredo Neto; Leonardo Cançado Monteiro Savassi; Aline Cruz Milagres; Mauro Martins Teixeira; Maria Izabel Campos Simplicio; Luciene Barra Ribeiro; Rosemary Oliveira; Edward J Mills Journal: JAMA Netw Open Date: 2021-04-01
Authors: Craig R Rayner; Louis Dron; Jay J H Park; Eric H Decloedt; Mark F Cotton; Vis Niranjan; Patrick F Smith; Michael G Dodds; Fran Brown; Gilmar Reis; David Wesche; Edward J Mills Journal: Am J Trop Med Hyg Date: 2020-10 Impact factor: 3.707