TOOLBOX

The Adaptive Platform Trial Toolbox

Objective of this toolbox

The Adaptive Platform Trial Toolbox aims to collect the accumulated knowledge, experience, and resources (collectively termed as ‘tools’) from multiple projects and trials into a practical and guided toolbox to facilitate planning and conduct of future adaptive platform trials in any therapeutic area

Why should you use the toolbox?

Adaptive platform trials constitute a dynamic topic in the current clinical research environment. Design, conduct, and even the naming of adaptive platform trials are in active discussion. Therefore, the toolbox has been structured as a practical aid to support complex clinical research. If you need resources regarding adaptive platform trials including, general information as well as specific resources for the different steps in the design or conduct, you can look for recommendations within the toolbox. You can consult the toolbox for training purposes as well.

How should you use the toolbox?

The toolbox has been structured with different categories for different trial steps. Each tool is presented with a brief explanation and a resource type, indicating the tool's format and level of validation. The tools are grouped into categories, each with its own tab. On the reference for each tool, the link to access the outside source is included. Further information is also available within the references section. The tools have been selected for their relevance and utility, but the views and opinions expressed in the tools may not represent the specific opinions of the EU-RESPONSE and RECOVER consortia.  

The toolbox is still under development and is currently available in a beta version. If you would like to provide feedback or gather more information about the rationale and methodology of the toolbox, please contact us.

Name Explanation Type Year Reference
Report on terminology, references and scenarios for platform trials and master protocols The project deliverable contains a selective glossary of terms for complex trials and platform trials. It also lists the current published platform trials and contains a curated and rated set of seminal publication references for Platform Trials. Developed within EU-PEARL project. Document, project deliverable, review 2020 Parke et al. 2020
Project: master protocol studies. Using master protocol studies to drive medical product development A set of project resources that guide the appropriate use of master protocols, developed by CTTI. Document, project outcome, guide 2020 CTTI 2020a
The evolution of master protocol clinical trial designs: a systematic literature review The journal review identifies existing master protocol studies and summarizes their characteristics. The review also identifies articles relevant to the design of master protocol trials, such as proposed trial designs and related methods. Document, journal article, systematic review 2020 Meyer et al. 2020
Clinical operations best practices report The deliverable is a project review of the current platform trial landscape to understand which challenges have the most complexity and impact. It is developed within EU-PEARL project and includes a literature search, website review, and an outreach survey to trial teams. Document, project deliverable, review 2020 Parke et al. 2020ba
Advancing the use of complex innovative trial designs (CID): introduction This video introduces FDA’s Complex Innovative Trial Designs (CID) program. It also covers some key aspects of complex trial designs with examples. Video, regulatory guide by health authority 2019 FDA 2019a
Adaptive designs for clinical trials This journal review focuses on adaptive designs of confirmatory clinical trials. It discusses the benefits and limitations of such designs, using four case studies that highlight the statistical and operational considerations. Document, journal article, review 2016 Bhatt and Mehta 2016
Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs The journal article introduces the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials. Document, journal article, correspondence 2020 Burnett et al. 2020
Adaptive trial designs - introduction for non-statisticians This video introduces adaptive trial design options for non-statisticians with trial examples. Presented by Cytel. Video, presentation by private company 2019 Cytel 2019
New approaches to clinical trials: adaptive designs The document and attached presentation discuss adaptive design elements for clinical trials, including advantages, disadvantages, rare diseases, and involvement of patients. Prepared by EUPATI. Document, website, article and presentation 2015 EUPATI 2015
Clinical trials in global health This Lancet Global Health Series of four papers and a commentary show the challenges in clinical trial research in global health and offers solutions that can optimise statistical efficiency and sustainability, including adaptive trial designs and master protocols, with considerations for the required clinical research infrastructure. Document, journal article, discussion and comment 2021 Bhandari et al. 2021, Dron et al. 2021, Park et al. 2021a, Park et al. 2021b, Park et al. 2021c
The COMET handbook: version 1.0 A key consideration for platform trials is to designate appropriate outcomes. These outcomes must be relevant for new interventional arms and suitable for adaptive elements. This journal article of Core Outcome Measures in Effectiveness Trials (COMET) Initiative addresses issues to consider when selecting or developing a core outcome set. Available outcome or data element repositories are also identified. Document, journal article, review 2017 Williamson et al. 2017
Effective delivery of Complex Innovative Design (CID) cancer trials—a consensus statement The journal article provides ten consensus recommendations to improve the conduct, quality and acceptability of complex oncology trials. While it has been prepared for oncology within the United Kingdom setting, the recommendations could be of general use for adaptive platform trials. Document, journal article, consensus statement 2020 Blagden et al. 2020
Delivering complex and innovative trials: case study library This website includes podcast series and case studies of adaptive platform trials performed in the United Kingdom. Hosted by the National Institute of Health Research. Document, video, and podcast, website, case study library 2020 NIHR 2020
PARADIGM patient engagement toolbox This project deliverable centralises all PARADIGM’s co-created recommendations, tools and relevant background information to make patient engagement in medicines development easier for all. The toolbox could help develop clinical trials with a further enhanced patient-focus and improve the experience of patients participating in the trials. Developed by PARADIGM project. Document, project deliverable, guide, checklist, and article 2020 PARADIGM 2020
Name Explanation Type Year Reference
Recommendation paper on the initiation and conduct of complex clinical trials This key document provides recommendations for sponsors regarding the authorisation and conduct of complex clinical trials. It describes the current perspective of the CTFG on these trials and outlines the major issues that sponsors should address in the process of initiating and conducting complex clinical trials in the EU/EEA. Document, regulatory guide by health authority 2019 CTFG 2019
Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design The document outlines some general considerations for studies incorporating interim analyses. A set of minimal requirements is outlined that must be fulfilled whenever confirmatory clinical trials are planned with an adaptive design. Later sections comment on specific design modifications. Published by EMA. Document, regulatory guide by health authority 2007 CHMP 2007
Adaptive designs in clinical trials: from scientific advice to marketing authorisation to the European Medicine Agency The journal article assesses scientific advice letters containing questions related to adaptive clinical trials in phases II or III, issued by EMA. It also examines relevant regulatory aspects. Document, journal article, research paper 2018 Collignon et al. 2018
Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics - draft guidance for industry This FDA guidance provides advice to sponsors of drugs and biologics for cancer treatment regarding the design and conduct of clinical trials, other than first-in-human trials, intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. Document, regulatory guide by health authority 2018 FDA 2018
Interacting with the FDA on complex innovative trial designs for drugs and biological products - guidance for industry This FDA document provides guidance to sponsors and applicants on interacting with the FDA on complex innovative trial design proposals for drugs or biological products. It also covers some key aspects of complex trial designs. Document, regulatory guide by health authority 2020 FDA 2020
COVID-19: master protocols evaluating drugs and biological products for treatment or prevention FDA is issuing this guidance to provide recommendations to sponsors of master protocols evaluating drugs for the treatment or prevention of COVID-19. Document, regulatory guide by health authority 2021 FDA 2021
Name Explanation Type Year Reference
Master protocol content development guide A step-by-step tool to develop an engagement strategy for engaging all relevant stakeholders early in the master protocol development process, developed by CTTI. Document, project outcome, guide 2020 CTTI 2020b
Clinical ICF - Study information and informed consent form An informed consent form template harmonised for clinical studies and further research. It is meant to cover all information required by the General Data Protection Regulation (GDPR) within a traditional (non-electronic) informed consent document to be signed by patients or healthy volunteers before participating in a clinical study; it does not address country specific requirements. Developed within DO->IT project. Document, project deliverable, template 2019 Norwegian Institute of Public Health and Boehringer Ingelheim 2019
Guidelines for tailoring the informed consent process in clinical studies These guidelines have been designed to provide information and evidence to assist with the development, or review of the consent process for use in clinical studies with human participants. Developed within i-CONSENT project. Document, project deliverable, guide and checklist 2021 i-CONSENT 2021
Adaptive platform trials: scalable from breast cancer to COVID This video with attached slides summarizes how the adaptive platform I-SPY trials has been developed for breast cancer and how it is replicated as a COVID trial. It depicts the life cycle of an adaptive platform trial with rationale, design, operations, infrastructure, intervention selection, data collection, added value, and publications. Hosted by NIH Collaboratory. Video, presentation, discussion 2020 NIH Collaboratory 2020
Lessons from COVID-19: the first year of the REMAP-CAP global adaptive platform trial The video with slides discusses the accumulated experience from REMAP-CAP COVID-19 platform trial with a focus on design, Bayesian statistical model, data management, interpretation of results, funding, and challenges for publication and trial logistics. Hosted by NIH Collaboratory. Video, presentation, discussion 2021 NIH Collaboratory 2021
Flexible trial design in practice - stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial The journal article describes methodological and practical experience arising with stopping recruitment to research arms following a pre-planned intermediate analysis and adding a new research arm during STAMPEDE, an adaptive platform oncology trial. It also includes criteria for assessing potential new intervention arms. Document, journal article, methodology 2012 Sydes et al. 2012
Mind the gap? The platform trial as a working environment In this commentary journal article, trial management and data management teams of STAMPEDE and FOCUS4 trials summarise the operational challenges and personal experience of individual researchers, to enable the broader clinical trials community to learn from their experiences. Document, journal article, commentary 2019 Morrell et al. 2019
This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols The journal article provides experiences gathered in FOCUS4 and STAMPEDE adaptive platform trials from a trial management perspective, highlighting the challenges and successes encountered in running adaptive platform trials. Document, journal article, methodology 2019 Schiavone et al. 2019
Name Explanation Type Year Reference
Statistical simulation in master protocols: components & communications considerations 

This tool provides early adopters with an overview of components of statistical simulation and tips for how to organize presentations of simulated trials to key stakeholder groups. Developed by CTTI.

Document, project outcome, guide  2020 CTTI 2020c
Systematic review of available software for multi-arm multi-stage and platform clinical trial design  This systematic research reviews existing software for the design of platform trials. Developed within EU-PEARL project. Document, journal article, systematic review  2021 Meyer et al. 2021
Adaptive design clinical trials for drugs and biologics - guidance for industry This FDA document provides guidance to sponsors and applicants submitting investigational new drug applications, new drug applications, biologics licensing applications, or supplemental applications on the appropriate use of adaptive designs for clinical trials to provide evidence of the effectiveness and safety of a drug or biologic. Document, regulatory guide by health authority 2019 FDA 2019b
Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats The journal article reviews the impact on statistical inference of adding arms. It also covers changing the control arm and how patient eligibility would complicate the trial design and analysis, the more generic statistical literature, and statistical considerations for different phases and funders. Document,journal article, commentary 2021 Lee et al. 2021
The adaptive platform trial - the statistical efficiencies The video provides statistical and methodological aspects of adaptive platform trials. It also covers case studies. Prepared by Berry Consultants. Video, presentation by private company 2015 Berry Consultants 2015
Data, data all around This news article from the Lancet Digital Health highlights the complex routine data collection machinery behind the RECOVERY trial, simplifying site operations. Document, journal article, news 2021 McCall 2021
Individualized screening trial of innovative glioblastoma therapy (INSIGhT): a Bayesian adaptive platform trial to develop precision medicines for patients with glioblastoma This journal article presents the Bayesian, adaptive, platform trial INSIGhT to develop precision medicine approaches in glioblastoma. Detailed information on the statistical model, simulations, and randomization algorithm is provided with expected benefits and challenges. Document, journal article, methodology 2019 Alexander et al. 2019
Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons The journal article shares experiences from FOCUS4 and STAMPEDE trials from the operational aspects of running these adaptive trials, focusing on data management. Adding and closing intervention arms, number of case report form changes, database amendments and database growth are discussed. Document, journal article, methodology 2019 Hague et al. 2019
Name Explanation Type Year Reference
The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design This CONSORT extension is meant to provide reporting guidance for any randomised trial that was designed using an adaptive design. Document, website and journal article, statement and checklist 2020 Dimairo et al. 2020
An overview of platform trials with a checklist for clinical readers The objective of this journal review is 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. Supplementary tables provide example use of the checklist for platform trials. Document, journal article, review and checklist 2020 Park et al. 2020
Name Explanation Type Year Reference
Accelerating COVID-19 therapeutic interventions and vaccines (ACTIV) trials A public-private partnership in the United States led by the National Institutes of Health (NIH) and coordinated by the Foundation for the National Institutes of Health (FNIH) to develop a research strategy for prioritizing and speeding development of the most promising COVID-19 vaccines and treatments. Document, trial website, protocol 2020 NIH 2020
A randomised, embedded, multi-factorial, adaptive platform trial for community-acquired pneumonia (REMAP-CAP) REMAP-CAP is a randomised, embedded, multifactorial, adaptive platform trial for community-acquired pneumonia. In addition, REMAP-CAP provides and adaptive research platform for evaluation of multiple treatment modalities in the event of a respiratory pandemic resulting in critical illness. Sponsored by University Medical Center Utrecht. Document, video, and podcast, trial website, protocol, statistical plan, and statistical model 2015 University Medical Center Utrecht 2015
Investigation of serial studies to predict your therapeutic response with imaging and molecular analysis (I-SPY) trials Adaptive platform phase 2 trials family with a Bayesian design to investigate treatments for breast cancer or COVID-19. Sponsored by QuantumLeap Healthcare Collaborative. Document and video, trial website, presentation 2010 QuantumLeap Healthcare Collaborative 2010
Randomised evaluation of COVID-19 therapy (RECOVERY) An adaptive platform clinical trial design to accelerate the process of identifying effective treatments for COVID-19 patients who enter the United Kingdom’s National Health Service hospitals. Sponsored by University of Oxford. Document and video, trial website, protocol, statistical plan, consent form, manual, patient engagement material, and presentation 2020 University of Oxford 2020a
Helping alleviate the longer-term consequences of COVID-19 (HEAL-COVID) A national platform trial jointly Sponsored by Cambridge University Hospitals NHS Foundation Trust and The University of Cambridge. It aims to evaluate the impact of treatments on longer-term morbidity, mortality, re-hospitalisation, symptom burden and quality of life associated with COVID-19. Document, trial website, protocol and consent form 2021 Cambridge University Hospitals NHS Foundation Trust 2021
Platform randomised trial of interventions against COVID-19 In older people (PRINCIPLE) This study is a platform, randomised controlled trial in primary care that can rapidly test low-risk treatments for people at higher risk of complications from COVID-19. The intent is to establish an on-going trial infrastructure within a master protocol. Sponsored by University of Oxford. Document, trial website. protocol, guidance, and patient information sheet 2020 University of Oxford 2020b
Molecular selection of therapy in colorectal cancer: a molecularly stratified randomised controlled trial programme (FOCUS4) FOCUS4 is an adaptive, molecularly stratified trial which aims to improve the efficiency with which new novel agents can be tested in the colorectal cancer setting. It has a multi-arm, multi-stage design with a master protocol. Sponsored by University College London. Document, trial website, protocol and patient information sheet 2013 University College London 2013
Seamless phase I/IIa platform for the rapid evaluation of candidates for COVID-19 treatment (AGILE) AGILE is a multicentre, multi-arm, multi-dose, multi-stage open-label, adaptive, seamless phase I/II Bayesian randomised platform trial to determine the optimal dose, activity and safety of multiple candidate agents for the treatment of COVID-19. Sponsored by University of Liverpool. Document and video, trial website, protocol and manual 2021 University of Liverpool 2021
An open-label, multicentre, randomised, adaptive platform trial of the safety and efficacy of several therapies, including antiviral therapies, versus control in mild/moderate cases of COVID-19 (ANTICOV) ANTICOV is an open-label, randomised, comparative, adaptive platform trial that will test the safety and efficacy of treatments in 2,000 to 3,000 mild-to-moderate COVID-19 patients. ANTICOV aims to identify early treatments that can prevent progression of COVID-19 to severe disease and potentially limit transmission. Sponsored by Drug for Neglected Diseases Initiative and others. Document and video, trial website, protocol, patient information, informed consent form, and standard operating procedure 2020 Drugs for Neglected Diseases Initiative et al. 2020

References

  • Alexander BM et al. (2019). Individualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT): A Bayesian Adaptive Platform Trial to Develop Precision Medicines for Patients With Glioblastoma. JCO precision oncology, 3, PO.18.00071. https://doi.org/10.1200/PO.18.00071
  • Berry Consultants (2015). The Adaptive Platform Trial - The Statistical Efficiencies. https://youtu.be/LcuEVL0VUso. Upload date: March 6, 2015.
  • Bhandari N et al. (2021). Challenges of adopting new trial designs in LMICs. The Lancet. Global health, 9(5), e575–e576. https://doi.org/10.1016/S2214-109X(21)00168-6
  • Bhatt DL and Mehta C (2016). Adaptive Designs for Clinical Trials. The New England journal of medicine, 375(1), 65–74. https://doi.org/10.1056/NEJMra1510061
  • Blagden SP et al. & Experimental Cancer Medicine Centres (ECMC) CID trials working group (2020). Effective delivery of Complex Innovative Design (CID) cancer trials-A consensus statement. British journal of cancer, 122(4), 473–482. https://doi.org/10.1038/s41416-019-0653-9
  • Burnett T et al. (2020). Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs. BMC medicine, 18(1), 352. https://doi.org/10.1186/s12916-020-01808-2
  • Cambridge University Hospitals NHS Foundation Trust (2021). HElping Alleviate the Longer-term Consequences of COVID-19 (HEAL-COVID). ClinicalTrials.gov identifier: NCT04801940
  • Clinical Trials Facilitation and Coordination Group (CTFG), Heads of Medicines Agencies (2019). Recommendation Paper on the Initiation and Conduct of Complex Clinical Trials. February 12, 2019. https://www.hma.eu/ctfg.html
  • Clinical Trials Transformation Initiative (CTTI 2020a). Project: Master Protocol Studies. https://www.ctti-clinicaltrials.org/projects/master-protocol-studies (accessed on April 12, 2021).
  • Clinical Trials Transformation Initiative (CTTI 2020b). Master Protocol Content Development Guide. September 29, 2020. https://www.ctti-clinicaltrials.org/sites/www.ctti-clinicaltrials.org/files/ctti_master_protocol_mp_protocol_development_map_29sept2020.pdf (accessed on April 12, 2021).
  • Clinical Trials Transformation Initiative (CTTI 2020c). Statistical Simulation in Master Protocols: Components & Communications Considerations. September 29, 2020. https://www.ctti-clinicaltrials.org/sites/www.ctti-clinicaltrials.org/files/ctti_master_protocol_stats_simulation_considerations_29sept2020.pdf (accessed on April 12, 2021).
  • Collignon O et al. (2018). Adaptive designs in clinical trials: from scientific advice to marketing authorisation to the European Medicine Agency. Trials, 19(1), 642. https://doi.org/10.1186/s13063-018-3012-x
  • Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (2007). Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design. CHMP/EWP/2459/02, London, 18 October 2007.
  • CytelVideos (2019). Adaptive Trial Designs - Introduction for Non-Statisticians. https://youtu.be/iA6lUAz_QaA. Upload date: October 17, 2019.
  • Dimairo M et al. (2020). The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. BMJ (Clinical research ed.), 369, m115. https://doi.org/10.1136/bmj.m115
  • Dron L et al. (2021). The role and challenges of cluster randomised trials for global health. The Lancet. Global health, 9(5), e701–e710. https://doi.org/10.1016/S2214-109X(20)30541-6
  • Drugs for Neglected Diseases Initiative et al. (2020). An open-label, multicentre, randomised, adaptive platform trial of the safety and efficacy of several therapies, including antiviral therapies, versus control in mild/moderate cases of COVID-19 (ANTICOV). Pan African Clinical Trials Registry number: PACTR202006537901307
  • European Patients’ Academy on Therapeutic Innovation (EUPATI 2015). New approaches to clinical trials: Adaptive designs. https://toolbox.eupati.eu/resources/new-approaches-to-clinical-trials-adaptive-designs/ (accessed on April 17, 2021).
  • Food and Drug Administration (2018). Master Protocols: Efficient Clinical Trial Design Strategies To Expedite Development of Oncology Drugs and Biologics - Draft Guidance for Industry. Docket number: FDA-2018-D-3292. September 2018.
  • Food and Drug Administration (2019a). Advancing the Use of Complex Innovative Trial Designs (CID): Introduction. https://www.youtube.com/watch?v=zLuvSQkG6TU. Upload date: July 3, 2019. Parent website: https://www.fda.gov/drugs/development-resources/complex-innovative-trial-designs-pilot-program (accessed on April 17, 2021).
  • Food and Drug Administration (2019b). Adaptive Design Clinical Trials for Drugs and Biologics - Guidance for Industry. Docket number: FDA-2018-D-3124. November 2019.
  • Food and Drug Administration (2020). Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products - Guidance for Industry. Docket number: FDA-2019-D-3679. December 2020.
  • Food and Drug Administration (2021). COVID-19: Master Protocols Evaluating Drugs and Biological Products for Treatment or Prevention - Guidance for Industry. Docket Number: FDA-2021-D-0409. May 2021.
  • Hague D et al. (2019). Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons. Trials, 20(1), 294. https://doi.org/10.1186/s13063-019-3322-7
  • i-CONSENT Consortium (2021). Guidelines for Tailoring the Informed Consent Process in Clinical Studies. Improving the guidelines for informed Consent, including vulnerable populations, under a gender perspective (i-CONSENT), Project number: 741856. https://cordis.europa.eu/project/id/741856. DOI: 10.5281/zenodo.4563938, 25/03/2021
  • Lee KM et al. (2021). Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats. Trials, 22(1), 203. https://doi.org/10.1186/s13063-021-05150-7
  • McCall B (2021). Data, data all around. The Lancet. Digital health, S2589-7500(21)00063-7. Advance online publication. https://doi.org/10.1016/S2589-7500(21)00063-7
  • Meyer EL et al. (2020). The Evolution of Master Protocol Clinical Trial Designs: A Systematic Literature Review. Clinical therapeutics, 42(7), 1330–1360. https://doi.org/10.1016/j.clinthera.2020.05.010
  • Meyer EL et al. (2021). Systematic review of available software for multi-arm multi-stage and platform clinical trial design. Trials, 22(1), 183. https://doi.org/10.1186/s13063-021-05130-x
  • Morrell L et al. (2019). Mind the gap? The platform trial as a working environment. Trials, 20(1), 297. https://doi.org/10.1186/s13063-019-3377-5
  • National Institutes of Health (NIH) Health Care Systems Research Collaboratory (2021). Lessons from COVID-19: The First Year of the REMAP-CAP Global Adaptive Platform Trial (Derek Angus, MD, MPH). https://vimeo.com/533147442. Date: April 2, 2021. Parent website: https://rethinkingclinicaltrials.org/news/april-2-2021-lessons-from-covid-19-the-first-year-of-the-remap-cap-global-adaptive-platform-trial-derek-angus-md-mph/ (accessed on April 19, 2021).
  • National Institutes of Health (NIH 2020). Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV). https://www.nih.gov/research-training/medical-research-initiatives/activ (accessed on April 12, 2021).
  • National Institutes of Health (NIH) Health Care Systems Research Collaboratory (2020). Adaptive Platform Trials: Scalable from Breast Cancer to COVID (Laura Esserman, MD, MBA). https://vimeo.com/451305585. Date: August 21, 2020. Parent website: https://rethinkingclinicaltrials.org/news/august-21-2020-adaptive-platform-trials-scalable-from-breast-cancer-to-covid-laura-esserman-md-mba/ (accessed on April 19, 2021).
  • National Institute for Health Research (NIHR 2020). Delivering complex and innovative trials: Case study library. Date: 15/01/2020. https://www.nihr.ac.uk/documents/delivering-complex-and-innovative-trials-case-studies/23637 (accessed on May 17, 2021).
  • Norwegian Institute of Public Health and Boehringer Ingelheim (2019). D4.10 Level 3 Clinical ICF - STUDY INFORMATION AND INFORMED CONSENT FORM (in D4.12 Level 3 Biobanking ICF). Big Data for Better Outcomes, Policy Innovation and Healthcare System Transformation (DO->IT), Project number: 116055. V1.4, 31/01/2019. https://cordis.europa.eu/project/id/116055
  • Patients active in research and dialogues for an improved generation of medicines (PARADIGM) project (2020). PARADIGM Patient Engagement Toolbox. Project number: IMI2 777450. https://cordis.europa.eu/project/id/777450
  • Park J et al. (2021a). Urgently seeking efficiency and sustainability of clinical trials in global health. The Lancet. Global health, 9(5), e681–e690. https://doi.org/10.1016/S2214-109X(20)30539-8
  • Park J et al. (2021b). Randomised trials at the level of the individual. The Lancet. Global health, 9(5), e691–e700. https://doi.org/10.1016/S2214-109X(20)30540-4
  • Park J et al. (2021c). How COVID-19 has fundamentally changed clinical research in global health. The Lancet. Global health, 9(5), e711–e720. https://doi.org/10.1016/S2214-109X(20)30542-8
  • Park J et al. (2020). An overview of platform trials with a checklist for clinical readers. Journal of clinical epidemiology, 125, 1–8. https://doi.org/10.1016/j.jclinepi.2020.04.025
  • Parke T et al. (2020a). D2.1. Report on Terminology, References and Scenarios for Platform Trials and Master Protocols. EU Patient-cEntric clinical tRial pLatforms (EU-PEARL), Project number: 853966. V2.2, 03/06/2020. https://cordis.europa.eu/project/id/853966
  • Parke T et al. (2020b). D2.2. Clinical Operations Best Practices Report. EU Patient-cEntric clinical tRial pLatforms (EU-PEARL), Project number: 853966. V2.3, 10/07/2020. https://cordis.europa.eu/project/id/853966
  • QuantumLeap Healthcare Collaborative (2010). I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer and I-SPY COVID-19 TRIAL: An Adaptive Platform Trial for Critically Ill Patients. ClinicalTrials.gov identifiers: NCT01042379 and NCT04488081
  • Schiavone F et al. (2019). This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols. Trials, 20(1), 264. https://doi.org/10.1186/s13063-019-3216-8
  • Sydes MR et al. (2012). Flexible trial design in practice - stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial. Trials, 13, 168. https://doi.org/10.1186/1745-6215-13-168
  • University Medical Center Utrecht (2015). Randomized, Embedded, Multifactorial, Adaptive Platform trial for Community-Acquired Pneumonia (REMAP-CAP). EudraCT number: 2015-002340-14, ClinicalTrials.gov identifier: NCT02735707
  • University College London (2013). FOCUS4 – Molecular selection of therapy in colorectal cancer: a molecularly stratified randomised controlled trial programme. EudraCT number: 2012-005111-12, ISRCTN registry: ISRCTN90061546, https://doi.org/10.1186/ISRCTN90061546
  • University of Liverpool (2021). AGILE (Early Phase Platform Trial for COVID-19). ClinicalTrials.gov identifier: NCT04746183
  • University of Oxford (2020a). Randomised Evaluation of COVID-19 Therapy (RECOVERY). EudraCT number: 2020-001113-21, ClinicalTrials.gov identifier: NCT04381936, ISRCTN registry: ISRCTN50189673, https://doi.org/10.1186/ISRCTN50189673
  • University of Oxford (2020b). Platform Randomised trial of Interventions against COVID-19 In older people (PRINCIPLE). EudraCT number:    2020-001209-22, ISRCTN registry: ISRCTN86534580, https://doi.org/10.1186/ISRCTN86534580
  • Williamson PR et al. (2017). The COMET Handbook: version 1.0. Trials, 18(Suppl 3), 280. https://doi.org/10.1186/s13063-017-1978-4