What is it?

In the context of immuno-oncology (IO), Mazzarella et al. argue that traditional trial models are no longer sufficient to address the biological and clinical complexity of modern therapies. Immunotherapies often produce variable, delayed, or atypical responses and toxicities, which challenge the rigid structure of conventional trials. Master protocols—whether umbrella, basket, or platform trials—respond to this by allowing adaptive features such as continuous treatment arm modifications, biomarker-based stratification, and shared control groups. 

How can it be useful? 

This article can be useful as a foundational reference for designing or evaluating innovative clinical trials in the IO field. It provides conceptual clarity on why master protocols are not just methodologically novel but practically necessary. Researchers, sponsors, and regulators can use it to justify protocol adaptations, guide safety oversight structures, and align trial design with modern drug development realities—especially where biomarker complexity and therapeutic uncertainty intersect.

What is it?

This is a video lecture by Dr. John Marshall (University of Toronto) made available by the Canadian Transfusion Trial Group. It explains the evolution of trial design and introduces master protocols—basket, umbrella, and especially platform trials. Using examples like REMAP-CAP and RECOVERY, it shows how adaptive platforms can test multiple interventions, add or remove arms, and deliver rapid evidence. The talk also covers Bayesian methods, response-adaptive randomization, and funding or regulatory challenges.

How can it be useful?

The video is a clear introduction to master protocols, illustrated with real clinical examples. It helps researchers and clinicians understand design choices, adaptive methods, and operational issues, making it a practical learning tool for innovative trial design.

What is it?

This meta-epidemiological study systematically reviewed 98 randomized platform trials published or registered between 2015 and 2022. Platform trials evaluate several interventions within a single protocol for a specific disease, with flexibility to add or drop interventions. The review summarizes trial characteristics, methodological features (e.g., statistical methods, randomization, blinding), and examples across diverse disease areas, notably COVID-19, oncology, and neurodegenerative disorders.

How can it be useful?

This review provides practical insights on adaptive designs, decision rules, and adding new interventions, useful for planning efficient, multi-intervention studies. It summarizes trial characteristics, methodological features (e.g., statistical methods, randomization, blinding), and examples across diverse disease areas, notably COVID-19, oncology, and neurodegenerative disorders (RECOVERY, REMAP-CAP, I-SPY, PRINCIPLE).
 

What is it?

This is a commentary article that examines the operational and human experience of researchers working on platform trials such as STAMPEDE and FOCUS4. Based on qualitative interviews, it describes challenges of workload, complexity, scale, and heightened expectations. Importantly, it shows how molecular stratification “in effect brings the patient into the trial office, as a specific individual, despite anonymisation, who is owed test results and a treatment decision,” creating a direct link between patient needs and researcher responsibilities.

How can it be useful?

The article documentis both staff and patient-related pressures in adaptive platform trials. It highlights the need for adequate resources, recognition of researcher workload, and mechanisms to protect staff well-being. At the same time, it emphasizes how staff experience a compassionate responsibility toward anonymised patients whose biomarker results and treatment options depend on their work. This dual perspective offers practical lessons on building trial environments that support both patients and the research teams delivering complex studies

What is it?

This is a commentary article that examines the operational and human experience of researchers working on platform trials such as STAMPEDE and FOCUS4. Based on qualitative interviews, it describes challenges of workload, complexity, scale, and heightened expectations. Importantly, it shows how molecular stratification “in effect brings the patient into the trial office, as a specific individual, despite anonymisation, who is owed test results and a treatment decision,” creating a direct link between patient needs and researcher responsibilities.

How can it be useful?

The article documentis both staff and patient-related pressures in adaptive platform trials. It highlights the need for adequate resources, recognition of researcher workload, and mechanisms to protect staff well-being. At the same time, it emphasizes how staff experience a compassionate responsibility toward anonymised patients whose biomarker results and treatment options depend on their work. This dual perspective offers practical lessons on building trial environments that support both patients and the research teams delivering complex studies

What is it?

This is a commentary article that examines the operational and human experience of researchers working on platform trials such as STAMPEDE and FOCUS4. Based on qualitative interviews, it describes challenges of workload, complexity, scale, and heightened expectations. Importantly, it shows how molecular stratification “in effect brings the patient into the trial office, as a specific individual, despite anonymisation, who is owed test results and a treatment decision,” creating a direct link between patient needs and researcher responsibilities.

How can it be useful?

The article documentis both staff and patient-related pressures in adaptive platform trials. It highlights the need for adequate resources, recognition of researcher workload, and mechanisms to protect staff well-being. At the same time, it emphasizes how staff experience a compassionate responsibility toward anonymised patients whose biomarker results and treatment options depend on their work. This dual perspective offers practical lessons on building trial environments that support both patients and the research teams delivering complex studies

What is it?

This EUPATI (European Patients’ Academy on Therapeutic Innovation) resource, developed as part of its patient engagement toolbox, explains adaptive clinical trial designs in plain language for patients and the general public. Adaptive designs allow pre-planned modifications during a trial based on interim data while maintaining validity and integrity. The resource describes three types of adaptive trial designs: group sequential designs, adaptive dose-finding designs, and biomarker-adaptive designs.

How can it be useful?

As a patient-focused educational tool, this EUPATI resource helps patients, advocates, and the public understand how adaptive trials work, why flexibility can improve efficiency, and how such trials can still safeguard scientific validity. It explains three specific adaptive design types, enabling non-specialists to better follow discussions about trial planning, especially in rare diseases. It also shows how patient input can guide researchers in choosing the most appropriate design by clarifying patient needs, and how patients can participate in Data Monitoring Committees to oversee trial conduct.

What is it?

It reviews alternative trial designs to conventional randomized controlled trials, including pragmatic trials, cluster trials, stepped wedge designs, practice-preference randomisation, adaptive designs, registry-based randomized trials, and specifically platform, basket, and umbrella trials. The article explains the strengths, weaknesses, and methodological challenges of each approach.

How can it be useful?

This article provides a concise overview of modern innovative trial designs for researchers and sponsors planning complex studies. It highlights when and why alternative methods may be appropriate, outlines practical trade-offs, and identifies challenges in implementation and analysis. As part of the toolbox, it serves as a reference for selecting and comparing trial designs, especially adaptive platform, basket, and umbrella trials, in order to improve efficiency, reduce cost, and enhance generalisability.

What is it?

It reviews alternative trial designs to conventional randomized controlled trials, including pragmatic trials, cluster trials, stepped wedge designs, practice-preference randomisation, adaptive designs, registry-based randomized trials, and specifically platform, basket, and umbrella trials. The article explains the strengths, weaknesses, and methodological challenges of each approach.

How can it be useful?

This article provides a concise overview of modern innovative trial designs for researchers and sponsors planning complex studies. It highlights when and why alternative methods may be appropriate, outlines practical trade-offs, and identifies challenges in implementation and analysis. As part of the toolbox, it serves as a reference for selecting and comparing trial designs, especially adaptive platform, basket, and umbrella trials, in order to improve efficiency, reduce cost, and enhance generalisability.

What is it?

It reviews alternative trial designs to conventional randomized controlled trials, including pragmatic trials, cluster trials, stepped wedge designs, practice-preference randomisation, adaptive designs, registry-based randomized trials, and specifically platform, basket, and umbrella trials. The article explains the strengths, weaknesses, and methodological challenges of each approach.

How can it be useful?

This article provides a concise overview of modern innovative trial designs for researchers and sponsors planning complex studies. It highlights when and why alternative methods may be appropriate, outlines practical trade-offs, and identifies challenges in implementation and analysis. As part of the toolbox, it serves as a reference for selecting and comparing trial designs, especially adaptive platform, basket, and umbrella trials, in order to improve efficiency, reduce cost, and enhance generalisability.

What is it?

This is a systematic review published in BMJ Open (2024; 14:e077132) on the operational complexities of international clinical trials. It synthesises evidence from 38 studies on challenges including sponsorship and insurance requirements, funding constraints, lack of harmonisation in ethics and regulatory approvals, lengthy contracts, site training, monitoring, communication, recruitment, data management, drug procurement and distribution, biospecimen processing, and adaptive trial-specific issues.

How can it be useful?

The review provides practical solutions for planning multinational and adaptive platform trials. It outlines strategies to address funding delays, contract bottlenecks, regulatory incompatibility, drug supply issues, and protocol amendments, offering a consolidated reference of barriers and solutions to improve the planning, coordination, and execution of international complex clinical trials.

What is it? 

This article is a real-world commentary on the plasmaMATCH platform trial, a multi-cohort, biomarker-guided study in advanced breast cancer. It explains in detail how a phase IIa platform trial was implemented, including molecular screening via ctDNA, adaptive cohort inclusion, protocol management, regulatory navigation, and data handling.

How it is useful? 

It is highly useful for clinical trial designers and research units preparing to implement complex, biologically stratified, multi-cohort trials—particularly when integrating liquid biopsy, multiple stakeholders, and adaptive trial governance. It provides practical solutions to operational hurdles, making it a valuable methodological and operational reference."

What is it? 

The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project (2019–2023), funded under the Innovative Medicines Initiative 2, developed the Platform for Patient and Community Engagement in Platform Trials (PaCEPT). Led by the European Patients’ Forum (EPF) and Novartis, this online repository explains platform trials through training materials, tools, and guidance for engaging patients and communities in platform trials, and offers resources for Patient and Community Advisors to actively shape trial design and conduct.

How can it be useful?

PaCEPT gives researchers, sponsors, and patient/community representatives free access to practical engagement resources, including infographics, videos, recommendation documents, best practice tools, and a repository of previous platform trials. It supports involvement of patients and communities from trial design to dissemination, helping align research with patient needs, improve informed consent processes, and enhance recruitment and retention. By hosting on the Patient Focused Medicines Development-PFMD Synapse network, it connects users to over 4,700 members and 1,450 organisations for collaboration. This resource can be directly integrated into a clinical trial toolbox to guide stakeholder engagement in patient-centric platform trials.

What is it?

This is a discussion paper published in the CADTH Health Technology Review (2022; Vol. 2, Issue 7). It summarises a workshop hosted by CADTH (Canada) examining the use of platform, basket, and umbrella trials in oncology and precision medicine. The paper discusses challenges for health technology assessment (HTA), including lack of comparator arms, small heterogeneous subgroups, use of real-world data (RWD), survival analysis limitations, health economic modelling, incremental cost-effectiveness ratios (ICERs), patient preferences, and ethical issues such as informed consent, justice, and equity. While the HTA framework is Canada-specific, the methodological and ethical challenges are internationally relevant.

How can it be useful?

This paper provides practical insights into data quality, economic evaluation, patient-centred value, and ethical principles when assessing new therapeutics. As part of the toolbox, it highlights how HTA decision-making is affected by trial design limitations, heterogeneity, and uncertainty, and suggests ways to integrate real-world evidence, health economics, and patient perspectives into evaluations of complex clinical trials. The lessons can be relevant for international researchers and policy-makers, beyond Canada, facing similar challenges.

What is it?

This is a discussion paper published in the CADTH Health Technology Review (2022; Vol. 2, Issue 7). It summarises a workshop hosted by CADTH (Canada) examining the use of platform, basket, and umbrella trials in oncology and precision medicine. The paper discusses challenges for health technology assessment (HTA), including lack of comparator arms, small heterogeneous subgroups, use of real-world data (RWD), survival analysis limitations, health economic modelling, incremental cost-effectiveness ratios (ICERs), patient preferences, and ethical issues such as informed consent, justice, and equity. While the HTA framework is Canada-specific, the methodological and ethical challenges are internationally relevant.

How can it be useful?

This paper provides practical insights into data quality, economic evaluation, patient-centred value, and ethical principles when assessing new therapeutics. As part of the toolbox, it highlights how HTA decision-making is affected by trial design limitations, heterogeneity, and uncertainty, and suggests ways to integrate real-world evidence, health economics, and patient perspectives into evaluations of complex clinical trials. The lessons can be relevant for international researchers and policy-makers, beyond Canada, facing similar challenges.

What is it?

This is a discussion paper published in the CADTH Health Technology Review (2022; Vol. 2, Issue 7). It summarises a workshop hosted by CADTH (Canada) examining the use of platform, basket, and umbrella trials in oncology and precision medicine. The paper discusses challenges for health technology assessment (HTA), including lack of comparator arms, small heterogeneous subgroups, use of real-world data (RWD), survival analysis limitations, health economic modelling, incremental cost-effectiveness ratios (ICERs), patient preferences, and ethical issues such as informed consent, justice, and equity. While the HTA framework is Canada-specific, the methodological and ethical challenges are internationally relevant.

How can it be useful?

This paper provides practical insights into data quality, economic evaluation, patient-centred value, and ethical principles when assessing new therapeutics. As part of the toolbox, it highlights how HTA decision-making is affected by trial design limitations, heterogeneity, and uncertainty, and suggests ways to integrate real-world evidence, health economics, and patient perspectives into evaluations of complex clinical trials. The lessons can be relevant for international researchers and policy-makers, beyond Canada, facing similar challenges.

What is it?

This is a review article published in Therapeutic Innovation & Regulatory Science (2021; 55:1145–1154). It provides an overview of master protocol frameworks—basket, umbrella, and platform trials—covering their definitions, design considerations, statistical methods, operational aspects, and regulatory perspectives. The paper highlights major challenges including control arm selection, non-concurrent controls, type I error control and multiplicity, data sharing, transparency, cross-treatment comparisons, tumor agnostic indications, and operational complexity.

How can it be useful?

This article provides practical recommendations for designing and implementing basket, umbrella, and platform trials, with examples from oncology and regulatory feedback from both the US and EU. It offers guidance on managing issues such as control arm design, type I error, and data sharing, while addressing broader concerns like regulatory acceptance, transparency, and efficiency. As part of the toolbox, it supports informed decision-making on the design, conduct, and compliance of complex clinical trials.

What is it?

This is a review article published in Therapeutic Innovation & Regulatory Science (2021; 55:1145–1154). It provides an overview of master protocol frameworks—basket, umbrella, and platform trials—covering their definitions, design considerations, statistical methods, operational aspects, and regulatory perspectives. The paper highlights major challenges including control arm selection, non-concurrent controls, type I error control and multiplicity, data sharing, transparency, cross-treatment comparisons, tumor agnostic indications, and operational complexity.

How can it be useful?

This article provides practical recommendations for designing and implementing basket, umbrella, and platform trials, with examples from oncology and regulatory feedback from both the US and EU. It offers guidance on managing issues such as control arm design, type I error, and data sharing, while addressing broader concerns like regulatory acceptance, transparency, and efficiency. As part of the toolbox, it supports informed decision-making on the design, conduct, and compliance of complex clinical trials.

What is it?

This is a review article published in Therapeutic Innovation & Regulatory Science (2021; 55:1145–1154). It provides an overview of master protocol frameworks—basket, umbrella, and platform trials—covering their definitions, design considerations, statistical methods, operational aspects, and regulatory perspectives. The paper highlights major challenges including control arm selection, non-concurrent controls, type I error control and multiplicity, data sharing, transparency, cross-treatment comparisons, tumor agnostic indications, and operational complexity.

How can it be useful?

This article provides practical recommendations for designing and implementing basket, umbrella, and platform trials, with examples from oncology and regulatory feedback from both the US and EU. It offers guidance on managing issues such as control arm design, type I error, and data sharing, while addressing broader concerns like regulatory acceptance, transparency, and efficiency. As part of the toolbox, it supports informed decision-making on the design, conduct, and compliance of complex clinical trials.

What is it?

This is a methodology and practical guidance article published in Trials (2022; 23:757). It summarises the collective experience of six UK-based Clinical Trials Units (CTUs) in running late-phase platform protocols, including basket, umbrella, multi-arm multi-stage (MAMS), and master protocols. Based on discussions from a one-day workshop and examples such as STAMPEDE, FOCUS4, Add-Aspirin, and CompARE, it presents operational recommendations covering the entire life cycle of a platform trial. 

How can it be useful?

This paper provides practical lessons and recommendations to run late-phase platform protocols more effectively. It gives guidance on communication, funding models, regulatory engagement, database and randomisation systems, PPI involvement, staffing, and oversight structures, and highlights specific challenges compared with traditional two-arm trials. It can serve as a concise operational reference for planning, managing, and maintaining complex adaptive and master protocol trials.

What is it? 

This is a project deliverable developed by ECRIN as part of the ERA4Health project (2025). It provides an overview of master protocol frameworks—basket, umbrella, and adaptive platform trials—covering their definitions, design features, and methodological considerations. The booklet addresses challenges such as funding models, sponsorship and governance, regulatory and ethical aspects, trial and data management, biomarker assay quality, patient engagement, and statistical approaches including Bayesian methods, error control, and adaptive features.

How can it be useful?

The booklet offers practical recommendations for the planning and conduct of master protocols, focusing on infrastructure setup, governance, recruitment strategies, consent processes, and data quality assurance. It also points to relevant EU and US regulatory guidance. As part of the toolbox, it serves as a hands-on reference for navigating regulatory compliance, operational complexity, funding mechanisms, and methodological rigor in complex clinical trials.

What is it? 

This is a project deliverable developed by ECRIN as part of the ERA4Health project (2025). It provides an overview of master protocol frameworks—basket, umbrella, and adaptive platform trials—covering their definitions, design features, and methodological considerations. The booklet addresses challenges such as funding models, sponsorship and governance, regulatory and ethical aspects, trial and data management, biomarker assay quality, patient engagement, and statistical approaches including Bayesian methods, error control, and adaptive features.

How can it be useful?

The booklet offers practical recommendations for the planning and conduct of master protocols, focusing on infrastructure setup, governance, recruitment strategies, consent processes, and data quality assurance. It also points to relevant EU and US regulatory guidance. As part of the toolbox, it serves as a hands-on reference for navigating regulatory compliance, operational complexity, funding mechanisms, and methodological rigor in complex clinical trials.

What is it? 

This is a project deliverable developed by ECRIN as part of the ERA4Health project (2025). It provides an overview of master protocol frameworks—basket, umbrella, and adaptive platform trials—covering their definitions, design features, and methodological considerations. The booklet addresses challenges such as funding models, sponsorship and governance, regulatory and ethical aspects, trial and data management, biomarker assay quality, patient engagement, and statistical approaches including Bayesian methods, error control, and adaptive features.

How can it be useful?

The booklet offers practical recommendations for the planning and conduct of master protocols, focusing on infrastructure setup, governance, recruitment strategies, consent processes, and data quality assurance. It also points to relevant EU and US regulatory guidance. As part of the toolbox, it serves as a hands-on reference for navigating regulatory compliance, operational complexity, funding mechanisms, and methodological rigor in complex clinical trials.

What is it?

This deliverable (EU-PEARL D1.7) sets out the Recommended Ethical, Information Governance and Security Policies for Integrated Research Platforms (IRPs). It describes the portfolio of policy documents needed to ensure compliance in areas such as ethics, informed consent, data protection, GDPR, transparency, and internal governance when designing and running IRPs. The report also points to templates and examples (e.g., master protocol, informed consent forms, data management plans) that can be adapted for platform trials.

How can it be useful?

This resource provides a practical checklist of governance and compliance instruments needed when planning or operating Integrated Research Platforms or other master protocol trials. It helps to identify required documents (e.g., DPIAs, transparency statements, data-sharing policies), understand their rationale, and locate existing templates. It is also useful for ensuring ethical compliance, GDPR alignment, and clear governance frameworks when developing multi-sponsor, multi-arm platform trials.

What is it?

A phase II, multi-arm multi-stage (MAMS) adaptive platform trial protocol evaluating reduced-frequency dosing of immune checkpoint inhibitors (ICIs) in patients with sustained responses across various advanced cancers, including renal cancer. The article outlines trial methodology, economic modelling, biomarker sub-studies, and patient and public involvement (PPI), providing a comprehensive overview of implementing an adaptive design to assess extended-interval immunotherapy.

How can it be useful?

REFINE troal is highly applicable for trial teams designing adaptive oncology trials that test non-inferiority or duration-based interventions. It provides a framework for extending adaptive MAMS platforms to dose scheduling questions. Its inclusion of economic and pharmacodynamic components, modular expansion across indications, and intention to inform a phase III trial makes it a highly translatable and scalable model.

What is it?

This is a regulatory reflection paper developed by the European Medicines Agency (EMA), focusing on the methodological issues involved in confirmatory clinical trials using adaptive designs. It outlines regulatory expectations, defines key concepts, discusses statistical control requirements (e.g. Type I error), and evaluates potential design modifications such as sample size reassessment, early stopping, and changes to primary endpoints.

How can it be useful?

This reflection document informs us how to incorporate adaptive design elements in late-phase (confirmatory) clinical trials. It provides clear regulatory expectations and cautions against misusing adaptive flexibility without pre-planning. It emphasizes the need to preserve trial integrity, especially when modifying trial elements mid-course. Practical insights on interim analysis handling, protocol amendments, and acceptability of design modifications are include

What is it?

This article, Regulatory Issues of Platform Trials: Learnings from EU-PEARL, draws on the  EU-funded Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU) EU-PEARL project (2019–2023) to outline key regulatory considerations for platform trials. Focusing mainly on European regulations (EMA requirements, EU Clinical Trials Regulation No. 536/2014, GDPR) with some US FDA comparisons, it addresses regulator interactions, submission strategies, trial authorisations, amendments, safety reporting, data protection, and ethics.

How can it be useful?

This resource offers practical, directly applicable insights for designing and conducting platform trials in compliance with regulatory expectations in Europe. It can help trial sponsors and investigators navigate multi-country authorisation processes, manage protocol amendments, and ensure safety reporting and data protection. By including concrete lessons learned from EU-PEARL and highlighting jurisdictional differences, it serves as a ready reference for teams planning efficient, regulatorily compliant platform trials.

What is it?

This article, Regulatory Issues of Platform Trials: Learnings from EU-PEARL, draws on the  EU-funded Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU) EU-PEARL project (2019–2023) to outline key regulatory considerations for platform trials. Focusing mainly on European regulations (EMA requirements, EU Clinical Trials Regulation No. 536/2014, GDPR) with some US FDA comparisons, it addresses regulator interactions, submission strategies, trial authorisations, amendments, safety reporting, data protection, and ethics.

How can it be useful?

This resource offers practical, directly applicable insights for designing and conducting platform trials in compliance with regulatory expectations in Europe. It can help trial sponsors and investigators navigate multi-country authorisation processes, manage protocol amendments, and ensure safety reporting and data protection. By including concrete lessons learned from EU-PEARL and highlighting jurisdictional differences, it serves as a ready reference for teams planning efficient, regulatorily compliant platform trials.

What is it?

This deliverable, produced for the EU-PEARL, EU Patient-cEntric clinical tRial pLatforms project (2019–2023), funded by the Innovative Medicines Initiative 2, addresses the emerging field of Integrated Research Platforms and Platform Trials, where terminology is still evolving. It provides a selective glossary of terms for complex and platform trials to promote consistent usage.

How can it be useful?

The glossary harmonises terminology for platform trials, improving communication among diverse clinical research stakeholders. By aligning language and concepts, it helps meet regulatory expectations and reduces duplication of effort. This fosters collaboration, streamlines trial implementation, and accelerates adoption of innovative trial designs in Europe and beyond.

What is it?

This deliverable, produced for the EU-PEARL, EU Patient-cEntric clinical tRial pLatforms project (2019–2023), funded by the Innovative Medicines Initiative 2, addresses the emerging field of Integrated Research Platforms and Platform Trials, where terminology is still evolving. It provides a selective glossary of terms for complex and platform trials to promote consistent usage.

How can it be useful?

The glossary harmonises terminology for platform trials, improving communication among diverse clinical research stakeholders. By aligning language and concepts, it helps meet regulatory expectations and reduces duplication of effort. This fosters collaboration, streamlines trial implementation, and accelerates adoption of innovative trial designs in Europe and beyond.

What is it?

This deliverable, produced for the EU-PEARL, EU Patient-cEntric clinical tRial pLatforms project (2019–2023), funded by the Innovative Medicines Initiative 2, addresses the emerging field of Integrated Research Platforms and Platform Trials, where terminology is still evolving. It provides a selective glossary of terms for complex and platform trials to promote consistent usage.

How can it be useful?

The glossary harmonises terminology for platform trials, improving communication among diverse clinical research stakeholders. By aligning language and concepts, it helps meet regulatory expectations and reduces duplication of effort. This fosters collaboration, streamlines trial implementation, and accelerates adoption of innovative trial designs in Europe and beyond.

What is it?

This document is the EU-PEARL Statistical Analysis Plan (SAP) Template V3 (25 April 2023), developed as part of the EU-PEARL project (IMI2 grant 853966). It provides a standardized framework for drafting statistical analysis plans for platform trials conducted under a Master Protocol. The template builds on the TransCelerate SAP V3.0 model and includes core backbone sections, appendices, and guidance for both master SAPs (mSAP) and intervention-specific appendices (ISA-SAP).

How can it be useful?

This resource is highly practical for researchers designing and conducting adaptive platform trials, as it ensures harmonization, regulatory compliance, and consistency across intervention-specific studies. By following the template, users can structure their SAPs in line with regulatory expectations (EMA,FDA,ICH E9) while accommodating trial-specific needs such as estimands, multiplicity adjustments, sensitivity analyses, and interim analyses. It provides ready-to-use text blocks, suggested formats, and instructions, which can directly save time and ensure quality in trial planning and reporting.

What is it?

This is a commentary that reviews the statistical issues when adding new arms to ongoing platform trials. It discusses methodological challenges such as bias, error rates, multiplicity, and interpretability, as well as practical considerations like changes in control arms, patient eligibility, and operational bias. The paper also examines differences in platform use between phase II and phase III trials and highlights areas for future methodological research.

How can it be useful?

This resource is useful for trialists and statisticians designing or conducting platform trials. It provides clear insights into the statistical implications of protocol amendments, decision-making about error rate control, and strategies for robust inference. Readers can use it to anticipate methodological pitfalls, strengthen trial protocols, and align designs with regulatory expectations.

What is it?

A review article in Annals of Oncology that analyses statistical controversies in basket, umbrella, and platform trials. It highlights misuse of terminology, debates on replacing tumor histology with biomarkers, challenges in small heterogeneous cohorts, Type I error control, and the use of Bayesian adaptive methods. Examples such as NCI-MATCH, FOCUS4, LUNG-MAP, SHIVA, and I-SPY2 are used to illustrate these issues.

How can it be useful?

This article offers a clear explanation of the statistical and methodological pitfalls in master protocols, to guide researchers in selecting the right design, avoiding common misuses of terminology, and anticipating regulatory/statistical concerns such as error control, feasibility in rare populations, and validity of biomarker-driven hypotheses. The practical trial examples help translate these controversies into lessons for designing robust master protocol studies.

What is it?

A review article in Annals of Oncology that analyses statistical controversies in basket, umbrella, and platform trials. It highlights misuse of terminology, debates on replacing tumor histology with biomarkers, challenges in small heterogeneous cohorts, Type I error control, and the use of Bayesian adaptive methods. Examples such as NCI-MATCH, FOCUS4, LUNG-MAP, SHIVA, and I-SPY2 are used to illustrate these issues.

How can it be useful?

This article offers a clear explanation of the statistical and methodological pitfalls in master protocols, to guide researchers in selecting the right design, avoiding common misuses of terminology, and anticipating regulatory/statistical concerns such as error control, feasibility in rare populations, and validity of biomarker-driven hypotheses. The practical trial examples help translate these controversies into lessons for designing robust master protocol studies.

What is it?

A review article in Annals of Oncology that analyses statistical controversies in basket, umbrella, and platform trials. It highlights misuse of terminology, debates on replacing tumor histology with biomarkers, challenges in small heterogeneous cohorts, Type I error control, and the use of Bayesian adaptive methods. Examples such as NCI-MATCH, FOCUS4, LUNG-MAP, SHIVA, and I-SPY2 are used to illustrate these issues.

How can it be useful?

This article offers a clear explanation of the statistical and methodological pitfalls in master protocols, to guide researchers in selecting the right design, avoiding common misuses of terminology, and anticipating regulatory/statistical concerns such as error control, feasibility in rare populations, and validity of biomarker-driven hypotheses. The practical trial examples help translate these controversies into lessons for designing robust master protocol studies.

What is it?

This is a video presentation by Scott Berry (Berry Consultants), introducing the concept of adaptive platform trials and contrasting them with traditional trials. The educational video explains key ideas such as heterogeneity of disease, umbrella trials (multiple subsets of disease tested with a single drug), platform trials (multiple drugs tested within one disease), and their combination in adaptive platform trials. The presentation also covers statistical efficiencies, shared control designs, perpetual platforms, and response adaptive randomization, using examples from PREPARE (EU, pneumonia/influenza), I-SPY2 (oncology), and IMI Alzheimer’s initiatives.

How can it be useful?

This video provides clear, accessible explanations of trial structures and their efficiencies, showing how adaptive platform trials reduce patient exposure to placebo, accelerate drug testing, and improve statistical power. It also situates platform trials in real-world contexts (pandemic preparedness, oncology, Alzheimer’s disease, stroke models), making it a practical educational tool for understanding why platform trials are considered the future of clinical research.

What is it?

This is a video presentation by Scott Berry (Berry Consultants), introducing the concept of adaptive platform trials and contrasting them with traditional trials. The educational video explains key ideas such as heterogeneity of disease, umbrella trials (multiple subsets of disease tested with a single drug), platform trials (multiple drugs tested within one disease), and their combination in adaptive platform trials. The presentation also covers statistical efficiencies, shared control designs, perpetual platforms, and response adaptive randomization, using examples from PREPARE (EU, pneumonia/influenza), I-SPY2 (oncology), and IMI Alzheimer’s initiatives.

How can it be useful?

This video provides clear, accessible explanations of trial structures and their efficiencies, showing how adaptive platform trials reduce patient exposure to placebo, accelerate drug testing, and improve statistical power. It also situates platform trials in real-world contexts (pandemic preparedness, oncology, Alzheimer’s disease, stroke models), making it a practical educational tool for understanding why platform trials are considered the future of clinical research.

What is it?

This is a video presentation by Scott Berry (Berry Consultants), introducing the concept of adaptive platform trials and contrasting them with traditional trials. The educational video explains key ideas such as heterogeneity of disease, umbrella trials (multiple subsets of disease tested with a single drug), platform trials (multiple drugs tested within one disease), and their combination in adaptive platform trials. The presentation also covers statistical efficiencies, shared control designs, perpetual platforms, and response adaptive randomization, using examples from PREPARE (EU, pneumonia/influenza), I-SPY2 (oncology), and IMI Alzheimer’s initiatives.

How can it be useful?

This video provides clear, accessible explanations of trial structures and their efficiencies, showing how adaptive platform trials reduce patient exposure to placebo, accelerate drug testing, and improve statistical power. It also situates platform trials in real-world contexts (pandemic preparedness, oncology, Alzheimer’s disease, stroke models), making it a practical educational tool for understanding why platform trials are considered the future of clinical research.

What is it?

This editorial presents the evolution of oncology trial design from traditional RCTs to innovative methods including Bayesian design, adaptive trials, single-arm trials using historical data, and the threshold-crossing (or counterfactual) model. It emphasizes the role of data-sharing platforms and collaborative initiatives like the Oncology Research Information Exchange Network (ORIEN) in improving patient-trial matching. Case examples include I-SPY2, vemurafenib in BRAF-mutant melanoma, and crizotinib in ALK-positive lung cancer.

How can it be useful?

It offers a framework for designing trials that reduce sample sizes, reuse validated data, and accelerate drug approvals using alternative methods grounded in real-world oncology contexts.

What is it?

This is a systematic literature review published in Clinical Therapeutics (2020; 42:1330–1349). It covers the evolution, definitions, and characteristics of master protocol clinical trial designs, focusing on basket, umbrella, and platform trials. Major themes include design considerations, statistical methods, operational complexities, and regulatory perspectives for these innovative frameworks.

How can it be useful?

This article offers hands-on recommendations for designing and conducting basket, umbrella, and platform trials with examples from oncology and other disease areas. It directly addresses operational and statistical challenges such as control arm choice, adaptive randomization, multiplicity, data management, and regulatory issues. It is valuable for structuring, planning, and implementing complex clinical trials that require flexibility and efficiency, and is relevant for both US and EU settings.
 

What is it?

This is a systematic literature review published in Clinical Therapeutics (2020; 42:1330–1349). It covers the evolution, definitions, and characteristics of master protocol clinical trial designs, focusing on basket, umbrella, and platform trials. Major themes include design considerations, statistical methods, operational complexities, and regulatory perspectives for these innovative frameworks.

How can it be useful?

This article offers hands-on recommendations for designing and conducting basket, umbrella, and platform trials with examples from oncology and other disease areas. It directly addresses operational and statistical challenges such as control arm choice, adaptive randomization, multiplicity, data management, and regulatory issues. It is valuable for structuring, planning, and implementing complex clinical trials that require flexibility and efficiency, and is relevant for both US and EU settings.
 

What is it?

This is a systematic literature review published in Clinical Therapeutics (2020; 42:1330–1349). It covers the evolution, definitions, and characteristics of master protocol clinical trial designs, focusing on basket, umbrella, and platform trials. Major themes include design considerations, statistical methods, operational complexities, and regulatory perspectives for these innovative frameworks.

How can it be useful?

This article offers hands-on recommendations for designing and conducting basket, umbrella, and platform trials with examples from oncology and other disease areas. It directly addresses operational and statistical challenges such as control arm choice, adaptive randomization, multiplicity, data management, and regulatory issues. It is valuable for structuring, planning, and implementing complex clinical trials that require flexibility and efficiency, and is relevant for both US and EU settings.
 

What is it?

This article explores ethical challenges in basket trials, umbrella trials, and other master protocols within precision medicine. It examines the role of genetic screening, biopsy, and molecular stratification in allocating patients based on tumor heterogeneity.

How can it be useful?

It discusses the ethical complexity of informed consent, therapeutic expectations, and maintaining scientific validity while balancing risk-benefit considerations. Real-world examples like NCI-MATCH and Lung-MAP illustrate how designs relying on surrogate endpoints raise important questions in clinical oncology trial ethics.

What is it?

This is a systematic review published in Therapeutic Innovation & Regulatory Science (2024; 58:634–644). It analyses 38 pediatric master protocol studies identified through PubMed and ClinicalTrials.gov over the past 10 years. The review covers basket, umbrella, and platform trials, most of them in oncology and early-phase research, with increasing use since 2020 including in COVID-19. The paper highlights challenges such as small patient populations, ethical concerns, use of placebo controls, pediatric dosing strategies (weight-based, BSA-based), incorporation of pediatric arms into adult master protocols, lack of adaptive randomisation, sponsor collaboration, and trial infrastructure needs.

How can it be useful?

The review provides a comprehensive picture of how master protocols are being applied in pediatric drug development. It identifies barriers such as infrastructure setup, trial governance, recruitment, and interpretation of findings, while pointing to opportunities like shared control groups, multi-sponsor collaborations, and integration with adult protocols. It offers insights into the design, conduct, and ethical considerations of pediatric basket, umbrella, and platform trials, supporting efforts to expand their use and reduce delays in pediatric drug approvals.

What is it?

This is a systematic review published in Therapeutic Innovation & Regulatory Science (2024; 58:634–644). It analyses 38 pediatric master protocol studies identified through PubMed and ClinicalTrials.gov over the past 10 years. The review covers basket, umbrella, and platform trials, most of them in oncology and early-phase research, with increasing use since 2020 including in COVID-19. The paper highlights challenges such as small patient populations, ethical concerns, use of placebo controls, pediatric dosing strategies (weight-based, BSA-based), incorporation of pediatric arms into adult master protocols, lack of adaptive randomisation, sponsor collaboration, and trial infrastructure needs.

How can it be useful?

The review provides a comprehensive picture of how master protocols are being applied in pediatric drug development. It identifies barriers such as infrastructure setup, trial governance, recruitment, and interpretation of findings, while pointing to opportunities like shared control groups, multi-sponsor collaborations, and integration with adult protocols. It offers insights into the design, conduct, and ethical considerations of pediatric basket, umbrella, and platform trials, supporting efforts to expand their use and reduce delays in pediatric drug approvals.

What is it?

This is a systematic review published in Therapeutic Innovation & Regulatory Science (2024; 58:634–644). It analyses 38 pediatric master protocol studies identified through PubMed and ClinicalTrials.gov over the past 10 years. The review covers basket, umbrella, and platform trials, most of them in oncology and early-phase research, with increasing use since 2020 including in COVID-19. The paper highlights challenges such as small patient populations, ethical concerns, use of placebo controls, pediatric dosing strategies (weight-based, BSA-based), incorporation of pediatric arms into adult master protocols, lack of adaptive randomisation, sponsor collaboration, and trial infrastructure needs.

How can it be useful?

The review provides a comprehensive picture of how master protocols are being applied in pediatric drug development. It identifies barriers such as infrastructure setup, trial governance, recruitment, and interpretation of findings, while pointing to opportunities like shared control groups, multi-sponsor collaborations, and integration with adult protocols. It offers insights into the design, conduct, and ethical considerations of pediatric basket, umbrella, and platform trials, supporting efforts to expand their use and reduce delays in pediatric drug approvals.

What is it? 

A systematic review of global trial registries identified 62 eligible trials up to April 2021. Results showed increasing use of the MAMS approach since 2001, with acceleration during the COVID-19 pandemic, mainly in infectious disease and oncology treatment trials.

How can it be useful?

By documenting global uptake, disease focus, and operational characteristics of MAMS platform trials, this review provides an evidence base for teams considering this design in late-phase RCTs. It highlights patterns of use, potential efficiencies, and operational considerations, supporting better planning and delivery. The findings may assist researchers, funders, and policy makers in evaluating whether the MAMS approach could optimise trial efficiency and speed in answering critical clinical questions.

What is it?

A practical introduction to adaptive clinical trial designs, presented by an experienced biostatistician from Cytel—a consultancy group working in statistical software, advanced analytics, and strategic consulting for clinical trial design and execution. The webinar outlines the adaptive design concept, regulatory acceptance, and practical benefits, focusing on widely used approaches such as sample size re-estimation, endpoint switching, seamless phase II/III trials, dose selection, and population enrichment. Real-world case studies show how adaptive designs improve decision-making, manage uncertainty, and enhance trial efficiency while maintaining scientific validity and operational integrity.

How can it be useful?

This resource is designed for clinical development teams, project managers, and non-statistical stakeholders who want to understand when and how to apply adaptive trial designs effectively. It explains how to integrate adaptive approaches into an overall development plan, address regulatory considerations, and safeguard trial integrity. By applying the principles and examples shared, organizations can shorten development timelines, reduce costs, optimize resource use, and improve the likelihood of success in regulatory submissions and patient outcomes.

What is it? 

This is a podcast series developed by NHS, Health Research Authority on Complex Innovative Designs. In this episode, Professor Pam Kearns, Professor of Clinical Paediatric Oncology, Director of the Cancer Research UK Clinical Trials Unit and Institute of Genomic Sciences in Birmingham explains what are Complex Innovative Designs and the difference in different trial methodologies, Basket, Umbrella, Multi Arm Multi Stage (MAMS) Trials.