EATRIS Innovation Management Toolbox – Resources for Rare Disease Translational Research

Translational research in the field of rare diseases is a complex process that requires not only scientific expertise, but also extensive knowledge of regulation, intellectual property, project management, funding sources, and innovation management. One of the resources offered by EATRIS is the Innovation Management Toolbox, developed within the framework of the European Joint Programme on Rare Diseases. It is designed as a free online library of resources that brings together practical tools and guidelines to support translational medicine and rare disease researchers, clinicians, entrepreneurs, and other members of the innovation ecosystem.

In addition, within another EATRIS tool – the Innovation Services – a free mentoring support option for rare disease researchers has been developed. This service also provides support for health technology assessment, evaluation of translational feasibility, and offer services to get through the complex regulatory landscape related to a research idea.

Within the Innovation Management Toolbox, the collection of digital tools is structured to support all phases of innovation development – from early discovery to clinical translation and preparation for regulatory approval. The resources are carefully selected and categorised, ensuring convenient access to relevant information.

The Innovation Management Toolbox is part of the European Rare Disease Research Alliance (ERDERA) initiative – a large partnership supported by the European Union’s Horizon Europe programme that promotes rare disease research and innovation in Europe. ERDERA’s goal is to make Europe a global leader in rare disease research, improve diagnosis, treatment, and prevention, and promote data-driven and patient-centred approaches in translational research. The toolbox provides resources that help researchers navigate the complex translational and innovation processes in the field of rare diseases. Materials and functionalities available in the library include:

• Broad categories of resources, including research and drug development, regulatory science, intellectual property, translational project management, and funding opportunities.
• Short, targeted videos and practical examples illustrating specific challenges and solutions along the translational pathway.
• Collections of external tools and links related to specific topics or phases of the research cycle.
• A questions-and-answers section addressing common challenges in the development of rare disease therapies and diagnostics.
• A service catalogue – an overview of support services offered by national and European organisations, including expertise, technologies, databases, and biological sample resources.

Who is it for?

The ERDERA Innovation Management Toolbox is particularly useful for researchers and clinical specialists working on rare disease projects who wish to accelerate the translational process; for start-ups and small and medium-sized enterprises (SMEs) developing diagnostic or therapeutic solutions; and for consortia and research groups preparing project proposals or implementing internationally funded research.

Translational research in rare diseases is especially challenging, as it requires an integrated understanding of scientific, regulatory, commercial, and innovation management aspects. These resources help reduce uncertainty and inefficiency by providing researchers with structured and easily accessible information that can accelerate the transformation of innovations into real clinical solutions.

☞ More information and access to the Innovation Management Toolbox: https://imt.erdera.org/
☞ More information and access to Innovation Services: https://eatris.eu/innovation-services/

Information provided within the project “RSU participation in the Horizon Europe programme” (No. 1.1.1.5/3/25/I/014).

*project no. 1.1.1.5/3/25/I/014