About Us

What is Together4RD?

ERNs

Improving care for people living with a rare disease across Member States

In 2017 24 European Reference Networks (ERNs) were launched, virtual networks involving more than 900 highly specialised healthcare units from over 300 hospitals across 26 EU countries.

The aim of the ERNs is to improve care for patients with rare or low-prevalence complex diseases, by facilitating discussion on such conditions and concentrating knowledge and resources across Member States. The criteria for establishing and evaluating the work of ERNs is set out in EU legislation.

Bringing together experts to address unmet need

95% of rare diseases do not have any authorised treatment. Unlocking solutions for patients with unmet medical need is a priority for the European Commission, as well as the wider industry and clinical community.

As clinical research networks, ERNs hold an incredible amount of knowledge and data on rare diseases, which can support the research and development of new therapies and support greater understanding of these conditions. However, there is no overarching framework for ERNs to engage with industry or other researchers to exchange and collaborate using this knowledge.

In 2019 the Board of Member States of ERNs published a Statement outlining the principles of ERN and industry collaboration, but there are restrictions on funding and governance arrangements which make collaboration practically challenging.

Together4RD will work to identify frameworks and structures to enable closer and more targeted partnership in the areas that can make a difference.

Together for Rare Diseases (Together4RD) is an agile multi-stakeholder initiative aimed at supporting ERNs to collaborate with stakeholders to pursue opportunities that will address unmet medical needs of people living with rare diseases.

Our Aim

Unlocking opportunities

Our aim is to unlock opportunities for partnerships, particularly with the pharmaceutical industry, in areas such as:

Basic to translational research

Clinical trials for rare & ultra-rare conditions

Testing and accelerating innovative approaches to diagnosis

Development and implementation of data/evidence generation initiatives

Our multi-stakeholder Steering Group brings together experts from across the EU to guide our work.

Together4RD

Steering Group

Professor Dr Hélène Dollfus

Coordinator of ERN-EYE and Chair of the ERN Coordinators Group, Strasbourg University Hospital (France)

Dr Dorothée Leroux

Project Manager of ERN-EYE, Strasbourg University Hospital (France)

Professor Maurizio Scarpa

Coordinator of MetabERN, Regional Coordinating Centre for Rare Diseases, Udine University Hospital (Italy)

Professor Dr Franz Schaefer

Coordinator of ERKNet, Universitätsklinikum Heidelberg (Germany)

Professor Dr Alain Verloes

Coordinator of ERN-ITHACA, Hopitaux de Paris, Hopital Robert Debre (France)

Matt Bolz-Johnson

ERN and Healthcare Advisor at EURORDIS

Dr Daria Julkowska

Scientific Coordinator of the European Joint Programme on Rare Diseases

Dr Ana Rath

Director of Orphanet, French National Institute of Health and Medical Research (INSERM)

Anton Ussi

Operations & Finance Director at EATRIS ERIC

Maciej Gajewski

Executive Director, Head of International Government Affairs and Policy at Alexion Pharmaceuticals

Toon Digneffe

Head Public Affairs & Public Policy – Europe & Canada at Takeda

Anne-Sophie Chalandon

Global Rare Public Affairs – Rare Diseases Policy Head at Sanofi Genzyme

Tina Taube

Director Market Access & Orphan Drug Policy Lead at EFPIA

Vittoria Carraro

Executive Director, Government Affairs at EUCOPE

Andrzej Rys

Director for Health Systems, medical products and innovation

Martin Dorazil

Deputy Head of Unit, Digital Health, European Reference Networks

Sheela Upadhyaya

Rare Diseases & RAPID-C19 Strategic Advisor at NICE, Special Advisor to FIPRA

Victoria Hedley

Rare Disease Policy Manager at Newcastle University

Together4RD on Twitter

@eurordis's @yann_eurordis will deliver a keynote speech at the launch event for Together4RD's policy asks. 👉 Thursday 10 November 2022 👉14:00-16:00 👉European Parliament, Brussels Secure your place now: https://t.co/BZEryO3ByZ Follow @Together4RD for further announcements https://t.co/W4OsmOPnNM
28 September at 1:06pm
🗣️ Come join us in the European Parliament on November 10th as we launch our Policy Asks: A new model for collaboration to address areas of unmet need in rare diseases. Don't miss out, register here 👉 https://t.co/JSo81mp4Xn
16 September at 10:02am
📢We are proud to announce that our first in-person event on the 10th of November will be held under the patronage of the @CZECHIAinEU! 🇨🇿 Secure your place👉https://t.co/LI44NecHGr https://t.co/XKvYC49EwE
05 September at 10:59am