Our Work

Collaboration Case Studies

During the course of our work, stakeholders have shared with us examples of successful collaboration between clinical research networks and industry.

Together for Rare Diseases wants to use its platform to amplify these case studies, and promote the sharing of intelligence around how others have achieved success – for the benefit of the whole rare disease community.

Examples of clinical research and industry partnerships


TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.

Innovative Therapies for Children with Cancer in Europe (ITCC)

ITCC gathers 63 European Paediatric Oncology Departments with expertise in conducting early phase trials in children and adolescents, and 25 European research laboratories.

The aim of this organisation is to develop novel therapies for the treatment of paediatric and adolescent cancers in cooperation with regulatory bodies, pharmaceutical enterprises, parents and patients.

European Collaboration for Epilepsy Trials (ECET)

ECET is a non-for-profit organization registered in Ireland that brings together European professional and lay stakeholders associated with the International League Against Epilepsy (ILAE) and with the International Bureau for Epilepsy (IBE) to promote prevention, treatment and research of epilepsy.

Examples of registries that are involved in collaborations with industry

The European Society for Blood and Marrow Transplantation (EBMT) Registry

The EBMT Registry, established in 1974, is the backbone of the EBMT’s research and educational activities. As of 2021, the EBMT Registry has acquired data on over 700,000 patients that received a haematopoietic stem cell transplantation (HSCT) procedure procedure as well as data on over 3,000 patients that received CAR T-cell therapy.

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