Pilot projects

Building on previous Together4RD work on collaboration case studies, we identified achievable pilots for ERN – Industry partnerships as tangible examples of how collaboration can work and be scaled-up in future.

Together4RD aims to create a forum for all ERNs to present their respective areas of work and expertise to pharmaceutical companies, to build new relationships and facilitate the identification of relevant partnerships in future.

Our role as partner in the pilots is to monitor progress to extract key learnings and best practices, demonstrating how these public-private partnerships work to improve the life of people living with rare diseases through multi-stakeholder research. 

Osteogenesis Imperfecta, natural history and innovative clinical trial measures

Powered by Sanofi and ERN-BOND
Thanks to already collected data, the project aims to better understand the disease burden with cross-sectional data, and its evolution across disease domains with longitudinal data while defining genotype-phenotype correlations. Prospectively, the project will also measure the impact of disease on patient activity and quality of life using digital technologies in the real world setting, with a combined approach of gait analysis and sensors. Lastly, the project partners will jointly engage in early scientific dialogues with other stakeholders (such as regulators and HTA) to gain insights into approaches to foster patient access to innovative medicines. 
More information to come

Improving time to diagnosis for unmet patient needs in rare hematologic diseases

EuroBloodNet, Innova, ENROL and Takeda in partnership
This initiative aims to drive progress in the diagnostic pathways for rare hematologic diseases by utilizing algorithms for early detection. The project, in its early stages, employs an AI federated platform with the goal of enhancing patient outcomes. The initial phase of the pilot will focus on scoping activities and identifying centres for the upcoming implementation phase.
More information to come

RHINE Project: Rare Renal and PH International Network

Powered by Novo Nordisk, ERKnet and OxalEurope
The RHINE Project aims to unlock potential in Rare Renal through a comprehensive harmonization and interoperability model, focusing on primary hyperoxaluria (PH) to improve care pathways and patient outcomes. This involves establishing a Rare Kidney Network data registry and collaborating with the Hyperoxaluria Consortium for seamless data connection across ERKnet and PH-specific registries. The project aims to understand PH diagnostic and referral pathways locally, measuring metrics like time-to-diagnosis, referring center numbers, and the percentage of patients diagnosed before ESRD. Educational activities target identified diagnostic pathway gaps at regional and local levels, incorporating KPI testing to assess initiative effectiveness and changes in clinical practice over time.
More information to come

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01 January at 12:00am