More Effectively Using Registries to suppOrt PAtient-centered Regulatory and HTA decision-making

Projektinformationen

More-EUROPA

ID Finanzhilfevereinbarung: 101095479

DOI

10.3030/101095479

EK-Unterschriftsdatum 24 November 2022

Startdatum 1 Januar 2023

Enddatum 31 Dezember 2027

Finanziert unter

• Health

Gesamtkosten

€ 6 997 523,75

EU-Beitrag

€ 6 997 523,25

Koordiniert durch

ACADEMISCH ZIEKENHUIS GRONINGEN
Netherlands

CORDIS bietet Links zu öffentlichen Ergebnissen und Veröffentlichungen von HORIZONT-Projekten.

Links zu Ergebnissen und Veröffentlichungen von RP7-Projekten sowie Links zu einigen Typen spezifischer Ergebnisse wie Datensätzen und Software werden dynamisch von OpenAIRE abgerufen.

Leistungen

Documents, reports (17)

Guidelines for data volume & quality assessment

Recommendations for data volume and quality assessment, including key questions to answer, recommended statistical and machine learning tools

Study on heart failure treatments

Report on the effectiveness/safety of various heart failure treatments. This will include the presentation/publication of the results of the analyses testing the associations novel HF medications and mortality/morbidity and onset of common safety issues.

Online training materials

Online training materials will be made available on CORDIS that include videos, interactive presentations, and quizzes, as well as a manual for the use of the machine learning-powered registry screening tool.

Evidence needs across lifecycle

Report on evidence needs defined by stakeholders across the lifecycle of medicinal products. Report contains summary of evidence needs formulated such that they can be translated to quantifiable concepts for use in desig and analyses of trials and RWD from registries.

KI-Trials-App

Report on the development and validation of the open access trial design KI-Trials-App. The app will allow users to specify combinations of inclusion and exclusion criteria, and to receive back numbers for proportion of patients eligible, and event rates. The report will present the steps taken in the development and validation and the results.

Registries identification machine learning tool

Natural language processing-powered tool for identifications of the registries and key opinion leaders based on automated systematic literature review (including, code, documentation, and methodology description)

Data collection (feasibility)

report on the description of data from multiple sclerosis patients treated with rituximab and dimethyl fumarate from all contributing registries. This will, in addition to treatment information, include personal, clinical and other treatment data and be specified per country.

Dissemination and exploitation plan

A report of the communication and dissemination plan will be written on the dissemination strategy towards key stakeholders to ensure an uptake of the project outcomes. The plan will include the set-up of More-EUROPA's website with content relevant for both the project members and the wider, lay-men’s audience, and will cover social media activities presentation templates and networking activities.

Registries identification guidelines

Recommendations for registries identification; modelling plan for the tool development

Stakeholder kick-off

A report will be written on the kick-off stakeholder conference with a project overview and appraisal of key stakeholders’ perspectives on implementing registry-based RWD/RWE in regulatory and HTA decision making.

Framework early-stage evidence

Framework to assess level of evidence needed for exploratory development decisions. Framework includes exploratory development process steps, key decision points and proposed metrics that express uncertainty in decision making, that can be used in guide trial design and use of RWD registry data.

Patient considerations

Report about patient relevant information and current practices. The report will present the results of the conducted mixed method research. The current practices regarding the assessment of information from patients (PROMs, demographic information) in the included registries will be presented, as well as patients' perspectives toward these results and possible lacking relevant PROs. The report will also include an advise about PROs in the registries.

Report on the state of the art and unmet needs

Detailed document discussing the current practices of registry identification and assessment in Europe and worldwide.

SWOTs themes for RWD decision-making

Report on the Strengths, Weaknesses, Opportunities and Threats (SWOTs) of using RWD in regulatory/HTA decision making. The report presents the results from a systematic search of literature and policies as well as interviews with relevant patient/stakeholder groups.

Representativeness of registries

A report about the representativeness of patients in registries. The report will document on the assessment of: - the representativeness of patients enrolled in RCTs vs. the disease registries used in this project and other RWD- the application of in- and exclusion criteria of RCTs to disease registries - the influence of registries' selection criteria to other RWD sources - associations between the representativeness and characteristics of the registries- where possible, whether the drop-out rates are higher among specific groups.

Data quality standards

Development of an evidence-base for data quality/density/completeness for RWE in multiple sclerosis. Data quality indicators will be identified and an assessment of data quality and quantity across several MS registries will be created.

Network meta-analysis

Report on the network meta-analysis: Relative cost-effectiveness of new heart failure drugs. This will include the presentation/publication of the results regarding the comparison novel/more tradition HF medications in terms of cost/effectiveness.

Websites, patent fillings, videos etc. (1)

Website

A More-EUROPA dedicated public website will be launched.

Demonstrators, pilots, prototypes (1)

Text mining

A pilot on how to extract the minimal dataset, focusing on unstructured patient data and a test of the validity of data gathered by this new innovative technique. Data mining techniques will be further explored and compared to existing registry data.

Data sets, microdata, etc (1)

Minimal dataset - content

Determine the minimal dataset necessary for RWE for HTA. The content of a minimal dataset will be identified per disease group and compared to an expanded dataset as described per available register. Minimal datasets will be used to compare RCTs and RWE effects.

Veröffentlichungen

Peer reviewed articles (20)

Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024

Autoren: Diogo Almeida; Diana Mandslay; Peter G. M. Mol; Bruno Sepodes; Carla Torre
Veröffentlicht in: BioDrugs, 2025, ISSN 1179-190X
Herausgeber: Springer Nature Link
DOI: 10.1007/S40259-025-00737-X

Timing of Cardiac Resynchronization Therapy Following Stable Medical Therapy in Patients With Heart Failure

Autoren: Alessandro Villaschi, Christian Basile, Lina Benson, Paolo Gatti, William T. Abraham, Michael Böhm, Biykem Bozkurt, Javed Butler, Gianluigi Condorelli, Cecilia Linde, Marco Metra, Wilfried Mullens, Peter Mol, Shelley Zieroth, Raffaele Scorza, Lars H. Lund, Fredrik Gadler, Gianluigi Savarese
Veröffentlicht in: JACC: Heart Failure, Ausgabe 13, 2025, ISSN 2213-1779
Herausgeber: Elsevier BV
DOI: 10.1016/J.JCHF.2025.102515

Glucagon-like peptide-1 receptor agonists use and associations with outcomes in heart failure and type 2 diabetes. Data from the Swedish Heart Failure and Swedish National Diabetes Registries

Autoren: Markus Wallner, Mattia Emanuele Biber, Davide Stolfo, Gianfranco Sinagra, Lina Benson, Ulf Dahlström, Soffia Gudbjörnsdottir, Francesco Cosentino, Peter G M Mol, Giuseppe M C Rosano, aved Butler, Marco Metra, MD, Lars H Lund, Giulia Ferrannini, Gianluigi
Veröffentlicht in: European Heart Journal - Cardiovascular Pharmacotherapy, ISSN 2055-6845
Herausgeber: Oxford
DOI: 10.1093/EHJCVP/PVAE026

Associations between cardiac resynchronization therapy and clinical outcomes according to the atrial fibrillation status in patients with heart failure with reduced ejection fraction

Autoren: Renzo Laborante, Valeria Valente, Lina Benson, Paolo Gatti, Christian Basile, Alessandro Villaschi, Peter Moritz Becher, Domenico D’Amario, Carin Corovic-Cabrera, Fredrik Gadler, Gianluigi Savarese, Raffaele Scorza
Veröffentlicht in: Europace, Ausgabe 27, 2025, ISSN 1099-5129
Herausgeber: Oxford University Press (OUP)
DOI: 10.1093/EUROPACE/EUAF296

Stakeholders’ Perspectives Toward the Use of Patient Registry Data for Decision-Making on Medicines: A Cross-Sectional Survey

Autoren: Fabian Windfuhr; Sieta T. de Vries; Maria Melinder; Tanja Dahlqvist; Diogo Almeida; Bruno Sepodes; Carla Torre; Björn Wettermark; Peter G. M. Mol
Veröffentlicht in: Drug Safety, 2025, ISSN 1179-1942
Herausgeber: Springer Nature Link
DOI: 10.1007/S40264-025-01528-7

Safety of continuing mineralocorticoid receptor antagonist treatment in patients with heart failure with reduced ejection fraction and severe kidney disease: Data from Swedish Heart Failure Registry

Autoren: Federica Guidetti, Lars H. Lund, Lina Benson, Camilla Hage, Francesca Musella, Davide Stolfo, Peter G.M. Mol, Andreas J. Flammer, Frank Ruschitzka, Ulf Dahlstrom, Giuseppe M.C. Rosano, Oscar Ö. Braun, Gianluigi Savarese
Veröffentlicht in: European Journal of Heart Failure, 2023, ISSN 1879-0844
Herausgeber: Wiley
DOI: 10.1002/ejhf.3049

BMC Medicine

Autoren: Olmo R. van den Akker; Susanne Stark; Daniel Strech
Veröffentlicht in: BMC Medicine, 2024, ISSN 1741-7015
Herausgeber: BioMed Central
DOI: 10.1186/S12916-024-03799-W

Eligibility for vericiguat in a real-world heart failure population according to trial, guideline and label criteria: Data from the Swedish Heart Failure Registry

Autoren: Nguyen NV, Lindberg F, Benson L, Ferrannini G, Imbalzano E, Mol PGM, Dahlström U, Rosano GMC, Ezekowitz J, Butler J, Lund LH, Savarese G.
Veröffentlicht in: European Journal of Heart Failure, ISSN 1879-0844
Herausgeber: Wiley
DOI: 10.1002/EJHF.2939

Lung Cancer

Autoren: Geeske F Grit; Esmée van Geffen; Ruben Malmberg; Roelof van Leeuwen; Stefan Böhringer; Hans JM Smit; Pepijn Brocken; Job FH Eijsink; Esther Dronkers; Pim Gal; Eva Jaarsma; Regine JHM van Drie-Pierik; Anne MP Eldering-Heldens; AN Machteld Wymenga; Peter GM Mol; Juliëtte Zwaveling; Doranne Hilarius
Veröffentlicht in: Lung Cancer, 2024, ISSN 0169-5002
Herausgeber: Elsevier BV
DOI: 10.1016/J.LUNGCAN.2024.107950

RWD16 An AI-Powered Tool to Identify and Assess Fit-for-Use Registries for Drug Development and Evaluation

Autoren: Ghinwa Y. Hayek, Boris Kopin, Sonia Zebachi, Gaëtan Pinon, Basile Ferry, Elisabeth Bakker, Alexandre Macquin, Sieta de Vries, Peter GM. Mol, Billy Amzal
Veröffentlicht in: Value in Health, Ausgabe 28, 2025, ISSN 1098-3015
Herausgeber: Elsevier BV
DOI: 10.1016/J.JVAL.2025.09.2766

On the Concepts, Methods, and Use of “Probability of Success” for Drug Development Decision‐Making: A Scoping Review

Autoren: Cetinyurek-Yavuz, A.; Fayyad, M.B.N.; Jiang, Ce; Brion Bouvier, F.; Beji, C.; Zebachi, S.; Hayek, G.Y.; Amzal, B.; Porcher, R.; Tanniou, J.; Roes, K.C.B.; Rodwell, L.; Rodwell, L.
Veröffentlicht in: Clinical Pharmacology &Therapeutics, 2025, ISSN 1532-6535
Herausgeber: Wiley
DOI: 10.1002/CPT.3571

Characteristics, use of guideline–recommended medical therapies and clinical outcomes of patients with heart failure not enrolled in a quality registry: a comparison with the Swedish Heart Failure Registry

Autoren: Ailema González-Ortiz, Paul Hjemdahl, Faizan Mazhar, Alessandro Bosi, Anne-Laure Faucon, Gianluigi Savarese, Lars H Lund, Juan Jesus Carrero
Veröffentlicht in: European Heart Journal - Quality of Care and Clinical Outcomes, Ausgabe 11, 2025, ISSN 2058-5225
Herausgeber: Oxford University Press (OUP)
DOI: 10.1093/EHJQCCO/QCAF019

Eligibility for omecamtiv mecarbil in a real-world heart failure population: Data from the Swedish Heart Failure Registry

Autoren: Lindberg F, Øigaard N, Metra M, Rosano GMC, Dahlström U, Mol P, Hage C, Lund LH, Savarese G.
Veröffentlicht in: PLoS ONE, ISSN 1932-6203
Herausgeber: PLOS
DOI: 10.1371/JOURNAL.PONE.0303348

European Journal of Heart Failure

Autoren: Benedikt N. Beer; Lina Benson; Christian Basile; Benedikt Schrage; Peter Moritz Becher; Stefan Blankenberg; Paulus Kirchhof; Barna Szabó‐Söderberg; Marco Metra; Anne Lindberg; Egidio Imbalzano; Giuseppe M. C. Rosano; Patric Karlström; Peter G.M. Mol; Raffaele Scorza; Lars H. Lund; Felix Lindberg; Gianluigi Savarese
Veröffentlicht in: European Journal of Heart Failure, 2025, ISSN 1388-9842
Herausgeber: Elsevier BV
DOI: 10.1002/EJHF.70046

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Autoren: Almeida D, Umuhire D, Gonzalez-Quevedo R, António A, Burgos JG, Verpillat P, Bere N, Sepodes B, Torre C.
Veröffentlicht in: Frontiers in Medicine, ISSN 2296-858X
Herausgeber: Frontiers
DOI: 10.3389/FMED.2024.1408636

Sex differences in the prognostic role of achieving target doses of heart failure medications: Data from the Swedish Heart Failure Registry

Autoren: Amerigo Ferrari, Davide Stolfo, Alicia Uijl, Nicola Orsini, Lina Benson, Gianfranco Sinagra, Peter Mol, Sieta T. de Vries, Ulf Dahlström, Giuseppe Rosano, Lars H. Lund, Gianluigi Savarese
Veröffentlicht in: European Journal of Heart Failure, ISSN 1879-0844
Herausgeber: Wiley
DOI: 10.1002/EJHF.3272

Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real‐World Data Sources for Fit‐for‐Purpose Studies

Autoren: Sonia Zebachi, Julien Tanniou, Elisabeth Bakker, Sieta T. de Vries, Rossella Di Bidino, Entela Xoxi, Anna Glaser, Gianluigi Savarese, Jan Hillert, Peter G. M. Mol, Kelly Plueschke, Billy Amzal, Ghinwa Y. Hayek, Jeverson Moreira
Veröffentlicht in: Clinical Pharmacology & Therapeutics, Ausgabe 118, 2025, ISSN 0009-9236
Herausgeber: Wiley
DOI: 10.1002/CPT.3746

Withdrawal of Guideline-Directed Medical Therapy in Patients With Heart Failure and Improved Ejection Fraction

Autoren: Christian Basile, Felix Lindberg, Lina Benson, Federica Guidetti, Ulf Dahlström, Massimo Francesco Piepoli, Peter Mol, Raffaele Scorza, Aldo Pietro Maggioni, Lars H. Lund, Gianluigi Savarese
Veröffentlicht in: Circulation, Ausgabe 151, 2025, ISSN 0009-7322
Herausgeber: Ovid Technologies (Wolters Kluwer Health)
DOI: 10.1161/CIRCULATIONAHA.124.072855

Eligibility for sotagliflozin in a real-world heart failure population based on the SOLOIST-WHF trial enrolment criteria: data from the Swedish heart failure registry

Autoren: Becher PM, Savarese G, Benson L, Dahlström U, Karlström P, Mol PGM, Metra M, Bhatt DL, Pitt B, Lund LH.
Veröffentlicht in: European Heart Journal - Cardiovascular Pharmacotherapy, ISSN 2055-6845
Herausgeber: Oxford
DOI: 10.1093/EHJCVP/PVAD012

Decision‐Making Criteria and Methods for Initiating Late‐Stage Clinical Trials in Drug Development From a Multi‐Stakeholder Perspective: A Scoping Review

Autoren: Ce Jiang, Céline Beji, Sonia Zebachi, Ghinwa Y. Hayek, Aysun Cetinyurek‐Yavuz, Muhammad Bergas N. Fayyad, Laura Rodwell, Kit C. B. Roes, Billy Amzal, Christoph Gerlinger, Raphaël Porcher, Julien Tanniou
Veröffentlicht in: Clinical Pharmacology & Therapeutics, Ausgabe 117, 2025, ISSN 0009-9236
Herausgeber: Wiley
DOI: 10.1002/CPT.3566

Other (1)

Ethics practices associated with reusing health data: An assessment of patient registries

Autoren: Olmo van den Akker, Susanne Stark, Daniel Strech
Veröffentlicht in: medRxiv, ISSN 0000-0000
Herausgeber: x
DOI: 10.1101/2024.04.26.24306459

Conference proceedings (8)

What Are Unmet Needs for the Use of Registries in Regulatory/HTA Decision-Making in Europe? A Survey-Based Approach From the More-EUROPA Project

Autoren: J. Moreira, J. Tanniou, P. Tang, P. Godbillot, F.Ahrens, B. Amzal
Veröffentlicht in: Value in Health, ISSN 1098-3015
Herausgeber: Blackwell Publishing Inc.
DOI: 10.1016/J.JVAL.2023.09.2150

Criteria and Methods Applied to Decision-Making to Embark on Late-Stage Clinical Trials With a Multi-Stakeholder Perspective

Autoren: C. Jiang, J.Tanniou, B. Amzal
Veröffentlicht in: Value in Health, ISSN 1098-3015
Herausgeber: Blackwell Publishing Inc.
DOI: 10.1016/J.JVAL.2023.09.2148

Decision-Making Criteria and Methods for Initiating Late-Stage Clinical Trials from a Multi-Stakeholder Perspective: A Scoping Review

Autoren: Ce Jiang, Céline Beji, Sonia Zebachi, Ghinwa Hayek, Aysun CetinyurekYavuz, Bergas Fayyad, Laura Rodwell, Kit C.B. Roes, Billy Amzal, Christoph Gerlinger, Raphaël Porcher, Julien Tanniou
Veröffentlicht in: 2025
Herausgeber: PSI 2025 Conference

Decision-Making Criteria and Methods for Initiating Late-Stage Clinical Trials in Drug Development From a Multi-Stakeholder Perspective: A Scoping Review

Autoren: Ce Jiang, Céline Beji, Sonia Zebachi, Ghinwa Hayek, Aysun CetinyurekYavuz, Bergas Fayyad, Laura Rodwell, Kit C.B. Roes, Billy Amzal, Christoph Gerlinger, Raphaël Porcher, Julien Tanniou
Veröffentlicht in: 2025
Herausgeber: SnB Conference

An AI powered tool to identify and assess fit-for-use registries for real-world evidence

Autoren: Ghinwa Y. Hayek, Pascal Godbillot, Coriande Clemente, Sonia Zebachi, Gaëtan Pinon, Boris Kopin, Elisabeth Bakker, Sieta T. de Vries, Peter G.M. Mol, Billy Amzal
Veröffentlicht in: European Drug Utilization Conference 2025, 2025
Herausgeber: European Drug Utilization Conference

Existing Automated Tools to Assist Evidence Generation and Better Qualification of Registries and Real-World Data: A Systematic Review From the More-EUROPA Project

Autoren: P. Godbillot, P. Tang, J. Moreira, J. Tanniou, S. Zebachi, B. Amzal
Veröffentlicht in: Value in Health, ISSN 1098-3015
Herausgeber: Elsevier
DOI: 10.1016/J.JVAL.2023.09.2120

Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies

Autoren: Sonia Zebachi, Julien Tanniou, Elisabeth Bakker, Sieta T. de Vries, Rossella di Bidino, Entela Xoxi, Anna Glaser, Gianluigi Savarese, Jan Hillert, Peter G.M. Mol, Kelly Plueschke, Ghinwa Hayek, Billy Amzal, Jeverson Moreira
Veröffentlicht in: European Drug Utilization Conference 2025, 2025
Herausgeber: European Drug Utilization Conference 2025

Existing Automated Tools to Assist Evidence Generation and Better Qualification of Registries and Real-World Data: A Systematic Review From the More-EUROPA Project

Autoren: S. Zebachi, J. Moreira, J. Tanniou, P. Godbillot, P. Tang, B. Amzal
Veröffentlicht in: Value in Health, ISSN 1098-3015
Herausgeber: Blackwell Publishing Inc.
DOI: 10.1016/J.JVAL.2023.09.2089