Consortium

  • 1 research organization (Inserm)
  • 1 platform (French Health Data Hub)
  • 2 EU networks (Teddy Network, European Clinical Research Infrastructure Network ECRIN)
  • 1 SME (Pharmetheus)
  • 2 Pharma companies (Novartis, Roche)
  • 1 of the largest RD registries in the world (AP-HP-BNDMR)
  • 5 universities/medical university centers (Radboud University Medical Center, Uppsala University, University Medical Center Göttingen, Vienna Medical University, Warwick University)
  • 1 technology transfer office (Inserm-Transfert)
  • 1 regulatory agency (Swedish Medical Products Agency)

This consortium benefits from an excellent work environment providing cutting-edge platforms for data access provided by Novartis and HDH, as well as access to 27 clinical datasets (provided by Novartis and Roche) and 2 national registries (access provided by APHP-BNDMR).

INSERM – INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Paris, France

UU – UPPSALA UNIVERSITET
Uppsala, Sweden

MUW – MEDIZINISCHE UNIVERSITÄT WIEN
Vienna, Austria

HDH – PLATEFORME DES DONNEES DE SANTE
Paris, France

TEDDY – EUROPEAN NETWORK OF EXCELLENCE FOR PEDIATRIC CLINICAL RESEARCH
Pavia, Italy

MUNI – MASARYKOVA UNIVERZITA
Brno, Czechia

MPA – LÄKEMEDELSVERKET
Uppsala, Sweden

ROCHE – F. HOFFMANN-LA ROCHE AG
Basel, Switzerland

RUMC – STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM
Nijmegen, Netherlands

UMG – UNIVERSITY MEDICAL CENTER GÖTTINGEN
Göttingen, Germany

AP-HP – ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS
Paris, France

PMX – PHARMETHEUS AB
Uppsala, Sweden

ECRIN – EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK
Paris, France

IT – INSERM TRANSFERT
Paris, France

NOVARTIS – NOVARTIS PHARMA AG
Basel, Switzerland

UOW – THE UNIVERSITY OF WARWICK
Coventry, United Kingdom

INVENTS will propose a comprehensive clinical development framework, starting with enhancing the robustness of model-based treatment effect models through innovative clinical trial designs, as well as evidence synthesis methods augmented and improved by computational models for small sample evidence. These methods and designs will be incorporated into tailored evidence tools for regulatory decision-making.

WP2

Cope with missing knowledge

 

Objectives:

To evaluate, to assess and to propose a workflow for using in silico methods, and approaches to generate virtual patients using various data sources and computational models

WP Leader

Main contributors

WP3

Increasing robustness of small population confirmatory trials 

 

Objectives:

To develop and assess innovative clinical trial designs that make use of mature computational models such as PKPD and in silico trials to improve the efficiency and robustness of these trials

WP Leader

Main contributors

WP4

Proposing evidence synthesis approach 

 

Objectives:

To deepen the understanding of how information originating from very different data sources should be synthesized, in particular, when in silico trials generating virtual patients and cohorts are involved. 

WP Leader

Main contributors

WP5

Developing evidence tools for regulatory decision making in RD

 

Objectives:

To provide applicants and assessors with a framework, assessment criteria and associated decision tools use of computational models with high impact on decision making for marketing approval and reimbursement for small populations. Models, spanning from fundamental to real-world data integration.

WP Leader

Main contributors

WP6

Ethical, legal issues and patients’ engagement in a regulatory perspective

 

Objectives:

To ensure the Ethical, Legal and Regulatory requirements and Patients’ perspective implementation

WP Leader

Main contributors

WP7

Use cases and data sharing

 

Objectives:

To provide use cases for the methods developed in work packages 1-5 and provide relevant data and disease area expertise. 

WP Leader

Main contributors

WP8

Management and knowledge transfer

 

Objectives:

To provide efficient management and coordination of the project and the consortium and to ensure progress of the project towards its planned objectives. To also address dissemination, communication and training activities as well as the protection, exploitation and further use of the project results

WP Leader

Main contributors

ECRIN - EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK

Paris, France

ECRIN, the European Clinical Research Infrastructure Network, is a not-for-profit intergovernmental organisation that focuses on investigator initiated multinational clinical trials as well as clinical trials initiated by biotech and medical device small and medium enterprises (SMEs). It provides sponsors and investigators with advice, management services and tools to navigate Europe’s fragmented health and legal systems on clinical studies. ECRIN was listed on the European Strategy Forum on Research Infrastructures (ESFRI) roadmap in 2006, was awarded ERIC status in 2013 and was listed as a Landmark Infrastructure on the 2016 ESFRI Roadmap.

Currently ECRIN has thirteen member and observer countries, the Czech Republic, France, Germany, Hungary, Ireland, Italy, Norway, Portugal, Spain, and Slovakia that include over 120 Clinical Trial Units (CTUs) and cover more than 360 million citizens. Multi-country clinical trials mean greater access to patients, resources, and expertise, and, in turn, faster and potentially more robust results.

ECRIN is involved in pan-European collaborative projects addressing important issues in data management for health research. In INVENTS, ECRIN will work together with its Czech node, Masaryk University, on dissemination activities via training and workshops targeting two major stakeholders in the field of clinical trial methodology: the clinical trial units (CTUs) and the regulators and health technology assessment (HTA) bodies.

Jacques Demotes
Director general of the European Clinical Research Infrastructure Network (ECRIN)

Jacques Demotes is the director general of the European Clinical Research Infrastructure Network (ECRIN), which he founded in 2004. A neurologist and professor of cell biology, Jacques is an advisor to the biology and health research department at the French Ministry of Higher Education and Research.

While at ECRIN, he has contributed to numerous initiatives and collaborative projects related to multinational clinical trials. In particular, he chaired the working group that drafted the Organisation for Economic Co-operation and Development (OECD) Council Recommendation on the Governance of Clinical Trials.

Prior to ECRIN, he worked as a clinical neurologist and basic neuroscientist, then as director of the clinical investigation centre in Bordeaux. Jacques is a MD (with a specialisation in neurology), has a PhD in neuroscience, a Master of Science (MS) in neuroscience, and a Bachelor of Arts (BA) in mathematics and computer science from the University of Bordeaux. He also received an MBA from IAE Paris, and completed a training course in science policy with the Institute of Advanced Studies in Science and Technology in Paris.

UMG - University Medical Center Göttingen – Department of Medical Statistics

Göttingen, Germany

The University Medical Center Göttingen provides high-quality patient care, excellent research as well as modern educational opportunities. As part of the Georg August University of Göttingen it is situated within a dynamic network of research facilities and provides access to a range of scientific resources. Thus, the Department of Medical Statistics is embedded in a strong environment for clinical research and includes working and service groups closely working together. These collaborative efforts extend to statistical advisory services encompassing biostatistics as well as bioinformatics for the analysis of clinical and genomic data. Moreover, the department is actively engaged in intensive research across several key areas:

  • Methodological research focusing on innovative designs and analyses for clinical trials, with a specific emphasis on adaptive designs
  • Advancing methods of evidence synthesis, with a primary focus on synthesis techniques for health economic evidence, meta-analytical approaches for a small number of studies, and systematic review methodologies tailored to non-randomized study designs and diagnostic investigations
  • Exploring computational statistics methods to develop and implement statistical methodologies and applications using e.g. observational data, simulations, or data from published trials with a particular emphasis on techniques capable of addressing heterogeneous and distributional effects
Tim Friede

Tim Friede

Christian Röver

TEDDY - EUROPEAN NETWORK OF EXCELLENCE FOR PEDIATRIC CLINICAL RESEARCH

Pavia, Italy

TEDDY ‘European Network of Excellence for Paediatric Research’ was funded in 2005 under the European Sixth Framework Programme (FP6), with the aim of promoting research and development of children-tailored medicine to address children’s unmet needs.

Since its foundation, it achieved numerous outcomes thanks to multidisciplinary collaborations across the EU, overall contributing to raise social awareness on the paediatric medicines and research in Europe. These outcomes include: the production of several scientific publications, participation in collaborative paediatric research projects, the development of activities and tools to engage and empower children on their health protection.

TEDDY’s objectives involve the:

  • Promotion of children access to paediatric medicines and medical devices tailored for children’s use
  • Promotion of paediatric research in accordance with high-level ethical and methodological standards
  • Engagement of children and young patients in decisions related to their participation in clinical research and healthcare
  • Enhancement of awareness among stakeholders including authorities, public, health professionals and companies about the persistent unmet therapeutic needs of children

In the context of children involvement in research, Teddy has launched two YPAGs (KIDS Bari and KIDS Albania) composed by young people aged between 12-18 years recognised as iCAN chapters, as depicted in the picture.

MUW – MEDIZINISCHE UNIVERSITÄT WIEN

Vienna, Austria

The Medical University of Vienna is one of the longest-standing medical training and research institutions in Europe. With 8,000 students it is today the largest medical training facility in the German-speaking area. With its 30 university departments and two clinical institutes, 13 medical theory centers and numerous highly specialized laboratories, it is also one of the most important cutting-edge research institutions in the field of biomedicine. The Institute of Medical Statistics within the Center for Medical Data Science has a research focus in statistical methods in adaptive clinical study designs and platform trials, clinical trials in rare diseases, testing multiple hypotheses and regulatory statistics. It cooperates in numerous international research consortia involving academia, industry and regulatory authorities. Currently, the department hosts 18 statisticians and is strongly engaged in the PhD-program “Medical Informatics, Biostatistics and Complex Systems”.

Martin Posch

Martin Posch

INSERM – INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

Paris, France

OrphanDev, the FCRIN expertise network dedicated to rare diseases, is a collaborative and voluntary initiative of stakeholders involved in the fight against rare diseases, representing all parties concerned: researchers and clinicians, patients and entrepreneurs.

Website:  https://www.orphan-dev.org/

Olivier Blin

Olivier Blin

Vincent Montero

Vincent Montero

IT – INSERM TRANSFERT

Paris, France

Founded in 2000, Inserm Transfert SA is the private subsidiary of the French National Institute of the Health and Medical Research (Inserm), dedicated to technology transfer (from invention disclosure to industrial partnership). Inserm Transfert runs a comprehensive set of technology transfer activities from classical value chain activities to unique solutions:

  • Scouting & Intellectual property management, with a philosophy of giving a chance to all innovations (portfolio of 2190 patent families in 2021)
  • Early development & Proof-of concept support for therapeutics and diagnostics (biomarkers), with a philosophy of maximum consolidation of the earliest phases, with more than 21 M€ since 2009 with the objective to create value. In 2021, 250 projects were detected and about 500 contracts and licences were signed.
  • Setting up public multi-private partnerships on cohorts, databases, biobanks & public health management
  • Collaborative Research Funding: setting up proposals and managing collaborative research projects (FP7, H2020, IMI, national programmes, etc.)

Valle Montalvo Romeral

HDH – PLATEFORME DES DONNEES DE SANTE

Paris, France

The HDH was conceived as the unique gateway to health data in France and has a role similar to that of a Health Data Access Body foreseen by the draft EHDS regulation. Created in November 2019, it interacts with a variety of actors within the health ecosystem. The Health Data Hub has set up a highly efficient technological platform enabling public interest project leaders to mobilise very large data sources, to cross-reference them, and to use enough computing power to run complex search algorithms. The HDH supports ambitious project leaders who contribute to finding tomorrow's solutions to improve all citizens’ health.
The Health Data Hub is a major player in the French health data ecosystem. Indeed, it has developed more than a hundred active partnerships with various partners (national and international, academic, industrial, university hospitals, data suppliers, research teams, foreign data platforms, patient associations, academic societies) and has assisted more than 100 projects in their administrative procedures, via the "one stop shop". The Health Data Hub has also made available a secure, state-of-the-art platform with advanced data storage, computation, matching and analysis capabilities, which is currently being used by about 15 projects. It has also created a "data catalog" with 10 first databases, allowing access to these partner databases (replication of the cataloged databases to allow access and interfacing). Finally, with more than 300 public interventions since 2021 and participation in major projects such as the Parisanté Campus, the Health Data Hub participates in the animation of the ecosystem to accelerate innovation by promoting the sharing of experience and knowledge.
The Health Data Hub is a major player in the French health data ecosystem. Indeed, it has developed more than a hundred active partnerships with various partners (national and international, academic, industrial, university hospitals, data suppliers, research teams, foreign data platforms, patient associations, academic societies) and has assisted more than 100 projects in their administrative procedures, via the "one stop shop". The Health Data Hub has also made available a secure, state-of-the-art platform with advanced data storage, computation, matching and analysis capabilities, which is currently being used by about 15 projects. It has also created a "data catalog" with 10 first databases, allowing access to these partner databases (replication of the cataloged databases to allow access and interfacing). Finally, with more than 300 public interventions since 2021 and participation in major projects such as the Parisanté Campus, the Health Data Hub participates in the animation of the ecosystem to accelerate innovation by promoting the sharing of experience and knowledge.
Website : www.health-data-hub.fr 

ROCHE

Switzerland

F. Hoffmann-La Roche Ltd is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalised healthcare.

As part of the Consortium, Roche will share anonymised clinical trial data, supporting documentation, and scientific and regulatory expertise to enable the development of novel approaches to paediatric and rare drug development. The acceptability to global regulators will be a particular focus of the discussion. Roche will work closely with all partners on all methodological areas and also co-supervise students or junior researchers.

Website: https://www.roche.com

Markus-Elze

Markus Elze

David-Pau

David Pau

Francois-Mercier

François Mercier

Diane-Uschner

Diane Uschner

Gerardo-Machnicki

Gerardo Machnicki

Nicolas-Frances

Nicolas Francés

Yumi-Cleary

Yumi Cleary

Sylvie-Retout

Sylvie Retout

Cheikh-Diack

Cheikh Diack

PMX - PHARMETHEUS AB
Uppsala, Sweden

Pharmetheus specializes in quantitative approaches to drug research, development, and usage.

  • A value-driven partner to big pharma, biotech, and life sciences organizations, on contexts such as trial analyses and design, program strategies, special populations, and regulatory strategy
  • A vision to improve health and quality of life by realizing the full potential of quantitative approaches
  • A portfolio of efficient and timely model-informed drug development services enabling reliable decision making in healthcare
  • A team of ~60 experts present in 15 countries, serving >100 clients and having co-authored >100 publications
  • An internal R&D organized in three platforms – Pharmacometrics, PBPK-QSP, and Model Informed Drug Development (MIDD) – to maintain good practice and further develop the methodology for a wide range of approaches

Read more at www.pharmetheus.com

Marylore Chenel
Chief Research Officer

  • Joined Pharmetheus in 2021, serving as Chief Research Officer, part of the leadership team, and leading the PBPK-QSP Business Development Platform
  • Expertise includes model-informed drug development, clinical pharmacology and regulatory advices in therapeutic areas such oncology/immune-oncology, immuno-inflammation, cardiology, and diabetes
  • Previously worked as Head of Global Pharmacometrics and Pharmacokinetics at Servier, France, where she led a team supporting the drug development portfolio from research to submission including lifecycle management, as Head and Deputy Head of Clinical Pharmacometrics and Clinical Pharmacokinetics, as a Pharmacometrician at Servier, France, and as Postdoctoral Fellow at the University of Manchester, UK
  • PharmD (2000) and M.Sc. in Pharmacokinetics (2000) from the University of Paris V, France, and Ph.D. in Pharmacokinetics – PKPB modeling from the University of Poitiers, France

 

WP1

Increasing robustness of model-based treatment and extrapolation methods

 

Objectives:

To improve the robustness of extrapolation, modeling and simulation methods, to propose tools helping implement these approaches, and to provide evaluation criteria to use them in the decision-making process.

WP Leader

Main contributors