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EU Patient- cEntric clinicAl tRial pLatform

Deliverables

List of Non-Clinical, Early Development Biomarkers and Strategy for Validation

Bringing together a comprehensive overview of the current state of scientific validation to guarantee safety and to predict the outcome of late stage phase 2 and 2 clinical studies

Operational Plan for the implementation of the NF IRP

The operational plan for a LNHS and phase 23 IRP trial in NF patients based on WP2

Roadmap for collaboration with other projects/networks

A roadmap of synergies with other projects/networks will be established, followed by implementation of the most relevant collaborations

IRP-Integrated Sustainability Business Plan adapted to NASH-PT

An IRPspecific exploitation and sustainability plan

IRP-Integrated Sustainability and Business plan adapted to TB

An IRPspecific exploitation and sustainability plan

EU-PEARL Sustainability Plan

Sustainability plan defining an operations ready concern including legal regulatory and ethically compliant operations and governance structure ready for exploitation The plan will be based on a Cost Benefit Assessment and Business Impact Assessment to develop a business model for adoption scale up and the creation of a sustainable entity It will include impacts on stakeholders and organisational change recommendations

TRD/PRD final report on Regulatory and Ethics Consultation

Report on meetings held with WP4 and Regulators along with expertise from the biopharmaceutical industry to discuss regulatory and ethical issues involved in MDD platform trial and LNHS with particular reference to TRDPRD populations and studies Formats may include formal EMA regulatory consultation with EMA Innovation Task Force ITF or informal workshops Compound ownersdevelopers may include industry academia or others

Operational Plan to implement TB IRP

Framework protocol will be developed to allow rapid implementation of the IRP trial with the development of a TB specific operating plan to oversee such trials across the networks of clinical sites

Recommended Ethical, Information Governance and Security Policies for IRPs

Final portfolio of policies that need to be adopted by any cluster of companies and sites IRPs as final versions of instruments created in tasks 11 and 13 informed by the project experiences in WPs 27

Digital Phenotyping Tool for TRD/ PRD review

This deliverable will be a general review of the top digital phenotyping tools currently in use in MDD clinical trials The review should contain indepth evaluation of the top 23 tools in the market in addition to DocuMental This review should compare and contract these tools with respect to consensus definition used for TRDPRD as well as other important features relevant for the conduct of clinical trials as opposed to a consumer product or a patient management product targeting physicians A publication may result from this report if company confidential information can be redacted

Report on challenges and clinical trial scenarios in NASH specific IRPs

A manuscript for submission to a peerreviewed journal describing the challenges in designing a NASH specific IRP including various approaches to conducting studies patient subphenotypes outcomes and endpoints and standard assessments such as liver biopsy and other biomarkers

Final Report on Clinical Operations Best Practices

Includes methods for the implementation of interim analyses randomisation patient allocation and blinding safety monitoring and pharmacovigilance data management governance sponsorship site management selection of third party vendors clinical supplies and manufacturing issues around introduction and removal of treatments protocol deviations trial longevity and intervention selection

TRD/PRD final master protocol for IRP

This report will develop protocol templates for POC Phase II and III for TRDPRD IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Operational Plan to implement the NASH IRP

Framework protocol will be developed to allow rapid implementation of the IRP trial with the development of a NASHspecific operating plan to oversee such trials across the networks of clinical sites

Operational Plan to implement TRD/PRD IRP and LNHS

A framework operational plan will be developed to allow rapid implementation of a MDDspecific LNHS and platform trial using the networks of clinical sites being established in D48

Report on the Consensus Definition of Current Challenges in NF

This will define the scope of disease spectrum, patient definition, list and prioritise the main challenges in disease to be addressed by the IRP and the LNHS, define outcomes/endpoints, and how to identify key risk predictors to identify patients that can qualify for future trials and assess potential biomarkers.

Provisional Generic Master Protocol Template and Appendix for IRPs

Definition of the master protocol template structure including diseasespecific appendices statistical analysis plans and additional elements such as charts of data andor safety monitoring committees

NASH final Master Protocol for IRP

This report will develop protocol template for Phase 2 and considerations for Phase 3 for NASH IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

European Certification Programme for IRP Platforms

Organisational and business model for a scalable and sustainable certification programme initially within Europe for certifying IRP products and services operationalising the results in D16

TB final report on Regulatory and Ethics Consultation

Report on meetings held with ECs and Regulators along with expertise from the biopharmaceutical industry to discuss regulatory and ethical issues involved in IRP with particular reference to TB populations and studies

TB final Master Protocol for IRP

This report will develop protocol template for Phase 2a bc and considerations for Phase 3 for TB IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Clinical Operations Best Practices Report

Includes a systematic literature review on integrated research platforms covering methodological research, ongoing and completed platform trials, relevant European and US regulatory guidance and other relevant documents to identify the current state of the art, methodological gaps and major operational challenges.

Protocol for a Longitudinal Natural History Study (LNHS) in NF

Continuing from D71 we will define the content and time intervals of evaluation within the LNHS including standardised tools to evaluate complications

NASH final Report on Regulatory and Ethics Consultation

Report on meetings held with ECs and Regulators along with expertise from the biopharmaceutical industry to discuss regulatory and ethical issues involved in IRP with particular reference to NASH populations and studies

Interim Report on TRD/PRD master protocol and regulatory requirements

It includes the results of the first TRDPRD master protocol synopsis It will also describe the first outcome of the consultation regarding the minimum regulatory requirements of a platform study design

Outcome of workshops delivered by EPF and Patient Representatives

This deliverable will inform on the outcomes of two workshops delivered to the consortium by EPF with input from patient representatives

Trial feasibility in federated hospital network

This deliverable will document the outcomes of the evaluation for the tools and scaffolding developed extended or integrated during the project

Functional requirements for a federated network of patient cohorts for clinical trial feasibility and recruitment

This deliverable will describe architecture and processes for establishing an IRP involving real world clinical data sources disease registries and other patientlevel data sources

Interim Report on NASH master protocol and regulatory requirements

It includes the results of the first NASH master protocol synopsis and corresponding operational plan for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements of the NASH platform study design

Project Communication and Dissemination Strategy Report and Initial Toolset

Plan for communicating the project progress and its results. It will comprise dissemination objectives, target audiences, tools and planned activities

Interim Report on TB master protocol and regulatory requirements

It includes the results of the first TB master protocol synopsis and corresponding site readiness for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements for the TB platform study design

Report on Consensus Disease Definition for TRD and PRD

A oneday Scientific Meeting on definitions for TRD and PRD will be organised followed by a written report An openaccess publication with disease definitions will be pursued subsequently

Generic process for an EHR based patient cohort for IRPs

This report will define the standards and processes for IRPs patient identification selection and recruitment including multisponsors Long Term Registries to support patient recruitments

Interim Report on NF master protocol and regulatory requirements

It includes the results of the first NF master protocol synopsis and corresponding operational plan for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements of the NF platform study design

Report on stakeholders' event

This deliverable will contain a summary of the discussions and outcomes of the stakeholders event that WP3 will organize as a satellite session of WP2stakeholder event to share results and present methodologies developed for clinical trial feasibility

Capacity Building and Assessment Handbook for site readiness

Based on need assessment and requirements from platform short guide for site implementation focusing on the laboratory aspects human resources data management ethics integrity

Framework for Ensuring and Benchmarking Acceptable Data Quality Standards for Platform Trials

Organisational and business model for a scalable and sustainable data quality assessment service to assure future IRP studies and platforms that the data they reuse eg from EHR systems is of a satisfactory quality drawing on the results and experiences in WPs 27

Final Generic Master Protocol Template and Appendix for IRPs

See D23 Definition of the master protocol template structure including diseasespecific appendices statistical analysis plans and additional elements such as charts of data andor safety monitoring committees

NF Specific Report on Regulatory and Ethics Consultation

Report that identifies the NFspecific adaptations to the generic regulatory endorsement of WP2 eg age 18 years learning and behavioural disabilities patients with multiple complications offering themselves for multiple trials

Report on Terminology and Scenarios for Platform Trials

Includes a definition of common terminology for IRP and description of a number of disease and product development scenarios (including those of DSWPs) where a platform trial would be appropriate. These scenarios will be considered in the production of the generic master protocol template.

IRP-Integrated Sustainability and Business Plan Adapted to NF

An IRPspecific exploitation and sustainability plan will be created

Report on Methodological Tools for Intervention Selection

Includes tools and best practice advice for presentation assessment and decisionmaking of additional intervention to already running platform trial

Clinical Network of NF Sites Report

This will identify the required and available facilities of sites for trial and patient data and define minimal criteria for a viable network of sites for IRP and LNHS

Report on Regulatory Issues of IRPs

Includes requirements of regulatory authorities and ethics committees for approval of IRPs informed consent protocol amendments openingclosing of arms and issues in assessment of IRPs for regulatory approval of new medicinal products by regulatory authorities eg European National Authorities EMA and FDA and assessment by HTAs

NF Master Protocol for IRPs

This report will develop protocol template for Phase 2 and considerations for Phase 3 for NF IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Patient Engagement Platform

An online portal and resources that support patientpublic awareness about IRP trials enable their engagement in design and participation in relevant studies

Publications

An Adaptive-Discard-Graph for online error control

Author(s): Fisher, L., Bofill Roig, M., & Brannath, W
Published in: Arxiv gov, Issue N/A, 2023, Page(s) --
Publisher: --
DOI: 10.48550/arxiv.2301.11711

On model-based time trend adjustments in platform trials with non-concurrent controls

Author(s): Marta Bofill Roig, Pavla Krotka, Carl-Fredrik Burman, Ekkehard Glimm, Katharina Hees, Peter Jacko, Franz Koenig, Dominic Magirr, Peter Mesenbrink, Kert Viele, Martin Posch
Published in: 2021
Publisher: Cornell University

Type I Error Considerations in Master Protocols With Common Control in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion

Author(s): ,Olga Marchenko,Qi Jiang,Richard Pazdur,Martin PoschORCID Icon,Mary Redman,Yevgen Tymofyeyev,Xiaoyun (Nicole) Li,Marc Theoret,Yuan Li Shen,Thomas Gwise,Lorenzo Hess,Michael Coory,Andrew Raven,Naoto Kotani,Kit Roes,Filip Josephson,Scott Berry,Richard Simon &Bruce Binkowitz
Published in: Statistics in Biopharmaceutical Research, Issue 4 issues per year, 2021, ISSN 1946-6315
Publisher: Taylor & Francis Group
DOI: 10.1080/19466315.2021.1906743

Platform trials: the impact of shared controls on type one error and power

Author(s): Nguyen, Q., Hees, K., Hofner, B
Published in: Arxiv gov, Issue N/A, 2023, Page(s) --
Publisher: --
DOI: 10.48550/arxiv.2302.04713

Commentary: Two approaches to analyze platform trials incorporating non-concurrent controls with a common assumption

Author(s): Bofill Roig, M., König, F., Meyer, E., & Posch, M.
Published in: Clinical Trials, Issue Bimonthly, 2022, Page(s) Volume 19, Issue 5, October 2022, Pages 502-503, ISSN 1740-7745
Publisher: Sage Publications
DOI: 10.1177/17407745221112016

The Online Closure Principle

Author(s): Fischer, L., Bofill Roig, M., & Brannath, W
Published in: Arxiv gov, Issue N/A, 2022, Page(s) --
Publisher: --
DOI: 10.48550/arxiv.2211.11400

Platform trials and the future of evaluating therapeutic behavioural interventions

Author(s): Stefan M. Gold, Marta Bofill Roig, J. Jaime Miranda, Carmine Pariante, Martin Posch & Christian Otte.
Published in: Nature Reviews Psychology, 2022, ISSN 2731-0574
Publisher: Springer Nature Limited
DOI: 10.1038/s44159-021-00012-0

CohortPlat: Simulation of cohort platform trials investigating combination therapies

Author(s): Meyer E., Mesenbrink P, Duner-Baldauf C, Glimm E., Li Y, König F.
Published in: Arxiv gov, Issue N/A, 2022, Page(s) 1-24
Publisher: ARXIV.GOV
DOI: 10.48550/arxiv.2202.02182

Lost in translation. The quest for definitions of treatment-resistant depression with a focus on inflammation-related gene expression.

Author(s): Luca Sforzini
Published in: Brain, Behavior, & Immunity - Health, Issue 2, 2021, ISSN 2666-3546
Publisher: Elsevier Inc
DOI: 10.1016/j.bbih.2021.100331

Adaptive clinical trial designs with blinded selection of binary composite endpoints and sample size reassessment

Author(s): Bofill Roig, M., Melis, G. G., Posch, M., & Koenig, F.
Published in: Biostatistics, Issue Quarterly, 2022, ISSN 1465-4644
Publisher: Oxford University Press
DOI: 10.1093/biostatistics/kxac040

Systematic review of available software for multi-arm multi-stage and platform clinical trial design

Author(s): Elias Laurin Meyer, Peter Mesenbrink, Tobias Mielke, Tom Parke, Daniel Evans, Franz König
Published in: Trials, Issue 22/1, 2021, ISSN 1745-6215
Publisher: BioMed Central
DOI: 10.1186/s13063-021-05130-x

Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and Cons

Author(s): Juan M. Pericàs, Frank Tacke, Quentin M. Anstee, Nicholas A. Di Prospero, Mette Skalshøj Kjær, Peter Mesenbrink, Franz Koenig, Joan Genescà, Vlad Ratziu
Published in: Journal of Hepatology, Issue Monthly, 2022, Page(s) Vol.78 442–447, ISSN 0168-8278
Publisher: Elsevier BV
DOI: 10.1016/j.jhep.2022.09.021

The use of external controls: To what extent can it currently be recommended?

Author(s): Hans Ulrich Burger,Christoph Gerlinger,Chris Harbron,Armin Koch,Martin Posch,Justine Rochon,Anja Schiel
Published in: Pharmaceutical statistics, 2021, ISSN 1539-1612
Publisher: John Wiley & Sons, Inc
DOI: 10.1002/pst.2120

A Delphi-method-based consensus guideline for definition of treatment-resistant depression for clinical trials

Author(s): Luca Sforzini, Courtney Worrell, Melisa Kose, Ian M. Anderson, Bruno Aouizerate, Volker Arolt, Michael Bauer, Bernhard T. Baune, Pierre Blier, Anthony J. Cleare, Philip J. Cowen, Timothy G. Dinan, Andrea Fagiolini, I. Nicol Ferrier, Ulrich Hegerl, Andrew D. Krystal, Marion Leboyer, R. Hamish McAllister-Williams, Roger S. McIntyre, Andreas Meyer-Lindenberg, Andrew H. Miller, Charles B. Nemeroff, Cl
Published in: Molecular Psychiatry, 2021, ISSN 1476-5578
Publisher: Springer Nature Limited
DOI: 10.1038/s41380-021-01381-x

Decision rules for identifying combination therapies in open-entry, randomized controlled platform trials

Author(s): Elias Laurin Meyer,Peter Mesenbrink,Cornelia Dunger-Baldauf,Ekkehard Glimm,Yuhan Li,Franz König,EU-PEARL (EU Patient-cEntric clinicAl tRial pLatforms) Consortium
Published in: Pharmaceutical Statistics, 2022, ISSN 1539-1612
Publisher: John Wiley & Sons Ltd
DOI: 10.1002/pst.2194

Community engagement in tuberculosis research: the EU-Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) experience

Author(s): Dressler S, Tàvora Dos Santos Filho E, Seidel S, Gonzalez Moreno J, Heinrich N, Sanchez-Montalva A, Cirillo D
Published in: International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases,, Issue Monthly, 2023, Page(s) Volume 130 Suppl.1 S-20-24, ISSN 1201-9712
Publisher: Elsevier BV
DOI: 10.1016/j.ijid.2023.03.008

Estimands and Complex Innovative Designs

Author(s): Olivier Collignon,Anja Schiel,Carl-Fredrik Burman,Kaspar Rufibach,Martin Posch,Frank Bretz
Published in: Clinical Pharmacology & Therapeutics, 2022, ISSN 1532-6535
Publisher: John Wiley & Sons, Inc
DOI: 10.1002/cpt.2575

Identifying challenges in neurofibromatosis: a modified Delphi procedure

Author(s): Britt A. E. Dhaenens, Rosalie E. Ferner, Annette Bakker, Marco Nievo, D. Gareth Evans, Pierre Wolkenstein, Cornelia Potratz, Scott R. Plotkin, Guenter Heimann, Eric Legius & Rianne Oostenbrink
Published in: European Journal of Human Genetics, Issue 12 issues per year, 2021, ISSN 1018-4813
Publisher: Natue Publishing Group
DOI: 10.1038/s41431-021-00892-z

Online error rate control for platform trials.

Author(s): Robertson, D. S., Wason, J. M., König, F., Posch, M., & Jaki, T.
Published in: Statistics in Medicine, Issue 30/year, 2023, Page(s) Volume 42, Issue 14Jun 2023 Pages 2475–2495, ISSN 0277-6715
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/sim.9733

Review Article: The need for more efficient and patient-oriented drug development pathways in NASH-setting the scene for platform trials

Author(s): Juan M. Pericàs, Nicholas A. Di Prospero, Quentin M. Anstee, Peter Mesenbrinck, Mette S. Kjær, Jesús Rivera-Esteban, Franz Koenig, Elena Sena, Raluca Pais, Ramiro Manzano, Joan Genescà, Frank Tacke, Vlad Ratziu,
Published in: Alimentary Pharmacology and Therapeutics, Issue Bimonthly, 2023, Page(s) Volume 57, Issue 9 Pages: 948–961, ISSN 1365-2036
Publisher: John Wiley and Sons Ltd.
DOI: 10.1111/apt.17456

Lessons learned from drug trials in neurofibromatosis: A systematic review

Author(s): Britt A.E.Dhaenens, Rosalie E.Ferner, D. Gareth Evans, Guenter Heimann, Cornelia Potratz, Edwin van de Ketterij, Angela M.Kaindl, Geesje Hissink, Charlotte Carton, Annette Bakker, Marco Nievo. Eric Legius; Rianne Oostenbrink
Published in: European Journal of Human Genetics, Issue Volume 64, Issue 9, 2021, ISSN 1769-7212
Publisher: Elsevier BV
DOI: 10.1016/j.ejmg.2021.104281

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Author(s): Franz Koenig, Cécile Spiertz, Daniel Millar, Sarai Rodríguez-Navarro, Núria Machín, Ann Van Dessel, Joan Genescà, Juan M. Pericàs, Martin Posch, Adrian Sánchez-Montalva, Ana Belén Estevez, Àlex Sánchez, Anna Sanjuan, Elena Sena, Emma Granados, Esther Arévalo de Andrés, Fátima Nuñez, Gara Arteaga, Gabriela Perez Fuentes Ruiz, Guillermo Fernández, Jesus Rivera-Esteban, Joan Comella, J
Published in: eClinicalMedicine, Issue 11, 2024, Page(s) n/a, ISSN 2589-5370
Publisher: Elsevier
DOI: 10.1016/j.eclinm.2023.102384

Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19

Author(s): Nigel Stallard, Lisa Hampson, Norbert Benda, Werner Brannath, Thomas Burnett, Tim Friede, Peter K. Kimani, Franz Koenig, Johannes Krisam, Pavel Mozgunov, Martin Posch, James Wason, Gernot Wassmer, John Whitehead, S. Faye Williamson, Sarah Zohar, Thomas Jaki
Published in: Statistics in Biopharmaceutical Research, Issue 12/4, 2020, Page(s) 483-497, ISSN 1946-6315
Publisher: Taylor & Francis Group, LLC.
DOI: 10.1080/19466315.2020.1790415

On the use of non-concurrent controls in platform trials: a scoping review.

Author(s): Bofill Roig, M., Burgwinkel, C., Garczarek, U., Koenig, F., Posch, M., Nguyen, Q. and Hees, K.,
Published in: Trials, Issue Upon Acceptance, 2023, Page(s) 1-17, ISSN 1745-6215
Publisher: BioMed Central
DOI: 10.1186/s13063-023-07398-7

Platform trials as the way forward in infectious disease’ clinical research: the case of coronavirus disease 2019

Author(s): Juan M.Pericàs; Lennie P.G. Derde; Scott M.Berry
Published in: Clinical Microbiology and Infection, Issue Monthly, 2022, Page(s) 277-280, ISSN 1198-743X
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.cmi.2022.11.022

NCC: An R-package for analysis and simulation of platform trials with non-concurrent controls

Author(s): Pavla Krotka, Katharina Hees, Peter Jacko, Dominic Magirr, Martin Posch, Marta Bofill Roig
Published in: Software X, Issue Quarterly, 2023, ISSN 2352-7110
Publisher: Elsevier B.V.
DOI: 10.1016/j.softx.2023.101437

Online control of the False Discovery Rate in group sequential platform trials

Author(s): Zehetmayer, S., Posch, M., & Koenig, F.
Published in: Statistical Methods in Medical Research, Issue Bimonthly, 2022, Page(s) Volume 31, Issue 12, December 2022, Pages 2470-2485, ISSN 0962-2802
Publisher: SAGE Publications
DOI: 10.1177/09622802221129051

Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints

Author(s): Elias Laurin Meyer, Peter Mesenbrink,Nicholas A. Di Prospero,Juan M. Pericàs,Ekkehard Glimm,Vlad Ratziu,Elena Sena,Franz König
Published in: PLOS one, 2023, ISSN 1932-6203
Publisher: Public Library of Science
DOI: 10.1371/journal.pone.0281674

An interactive R-Shiny app for quickly visualizing a tidy, long dataset with multiple dimensions with an application in clinical trial simulations for platform trials

Author(s): Meyer, E. L., Kumaus, C., Majka, M., König, F
Published in: Software X, Issue Quarterly, 2023, Page(s) Volume 22, ISSN 2352-7110
Publisher: Elsevier BV
DOI: 10.1016/j.softx.2023.101347

EU-PEARL: Changing the paradigm of clinical trials in Europe

Author(s): L Cash-Gibson, JM Pericàs, C Spiertz, E van de Ketterij, E Molero, F Patalano, D Kalra, A Ussi, A Van Dessel, J Genescà
Published in: European Journal of Public Health, Issue Volume 31, Issue Supplement_3, October 2021, 2021
Publisher: Oxford University Press
DOI: 10.1093/eurpub/ckab165.657

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