A new phase in the effort to close research gaps in rare diseases was marked with the launch of the T4RD Toolkit, a practical and co-created resource to catalyse collaboration between European Reference Networks (ERNs) and life sciences companies. Presented at a joint webinar on 23 June 2025 by Together4RD and ERDERA, the Toolkit responds to a clear need: turning the promise of public-private partnerships in rare disease research into tangible, impactful action.
The Toolkit’s launch is directly aligned with the strategic goals of ERDERA Work Package 25.4, which aims to enhance the research capacity of ERNs and to establish a collaborative “ERN Living Lab.” With input from over 40 stakeholders (including ERN coordinators, industry representatives, researchers and patient advocates) the Toolkit is rooted in real-world lessons and aims to overcome the operational bottlenecks that have long hindered collaboration.
The Toolkit is Together4RD’s most comprehensive effort to date to strengthen research collaboration between ERNs and industry. ERNs, established to improve the diagnosis and care of people living with rare diseases, are recognised as key enablers of rare disease research due to their pan-European networks, data infrastructure, and deep disease-specific expertise. Yet, many of these networks operate in fields with limited prior R&D and lack experience in engaging with private sector partners.
“Although there’s widespread recognition of the value ERN-industry collaboration can bring, there’s been a lack of practical guidance on how to initiate, structure and govern these partnerships—especially in less research-mature ERNs. This Toolkit aims to fill that gap.” Dr Victoria Hedley, policy manager at Newcastle University.
A modular resource
The Toolkit is structured into three core sections, each responding to a specific stage in the collaboration journey:
Section A: Building common ground
Provides foundational knowledge for both ERNs and companies, including tools on how ERNs are structured, what kinds of research they can support, and how to understand each other’s operating realities and motivations.
Section B: Shaping collaboration
Offers guidance on forming research questions, aligning priorities, and structuring a partnership. It includes templates, examples of past collaborations, and summaries of ERN-industry pilot projects supported by Together4RD.
Section C: Implementation and Delivery
Focuses on operationalising a project, covering legal considerations, governance, and communication.
Strategic alignment with ERDERA
The Toolkit directly supports ERDERA WP25.4, which aims to align the research roadmaps of the 24 ERNs with ERDERA’s strategic objectives. As part of this, the Toolkit will be integrated into ERDERA’s Innovation Management Toolbox, a broader platform offering mentoring, expert services and capacity-building to European research actors.
The Toolkit will also serve as a resource for the ERN Living Lab, a new governance and exchange platform being developed under ERDERA to foster joint action and accelerate the translation of research into improved care pathways.
“At ERDERA, we are building a structured ecosystem that connects research, clinical infrastructure and private sector innovation. This Toolkit helps bridge a critical interface by providing the clarity and confidence needed to make partnerships work.” Daria Julkowska, ERDERA Coordinator
Learning from early pilots
A highlight of the Toolkit is its practical grounding in the experiences of three early ERN-industry pilot projects. These collaborations, co-developed through a call issued by T4RD in 2022, tackle challenges ranging from real-world evidence generation to diagnostic algorithm development. The lessons are candid and concrete—covering co-creation practices, mismatched timelines, and strategies for trust-building across institutional cultures.
The pilot projects are:
- BOND & Sanofi – evaluating disease burden and unmet needs in osteogenesis imperfecta.
- ERKNet & Novo Nordisk – repurposing registry data to explore phenylketonuria care gaps.
- EuroBloodNet & Takeda – applying AI to enhance early diagnosis of thrombotic thrombocytopenic purpura (TTP).
Recommendations from these experiences emphasise early alignment of expectations, transparency in project design, and realistic scoping of deliverables.
Looking ahead
Speakers at the webinar unanimously called for the Toolkit to be viewed as a starting point and not a finished product. Future work could include:
- Developing shared legal templates for data use or collaboration governance.
- Hosting in-person forums to foster relationships and matchmaking.
- Establishing a trusted third party to provide neutral coordination in sensitive collaborations.
- Clarifying policy signals from the Board of Member States to explicitly encourage ERN-industry cooperation.
“This Toolkit is not just a guide. It’s an instrument to unlock Europe’s competitive potential in rare disease R&D and to transform the landscape for patients who still wait for diagnosis, treatment and hope.” Professor Maurizio Scarpa, Coordinator of MetabERN
With the ERDERA partnership providing long-term continuity and T4RD’s multi-stakeholder energy driving uptake, the Toolkit marks a key milestone in turning collaborative intent into shared impact.
You can access the Toolkit here.