Development of new effective therapies for rare diseases

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 3 “Tackling diseases and reducing disease burden”. To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:

Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.

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Designated Intractable Diseases (Designated Nan-byo)

Published by IRDiRC

Act on Medical Care Early Access Support MHLW or PMDA Regulatory Nan-byo Designation Rare Disease Regulatory Affairs

Definition of designated Nan-byo is provided in the law named “Act on Medical Care for Patients with Intractable/Rare Diseases”. Patients with designated Nan-byo can receive financial support for medical treatment

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Rare disease research: what industry contributes in knowledge and resources

Published by

European Reference Network Industry collaboration Rare Disease Research and Drug Development

This webinar highlighted how industry contributes to PPPs beyond funding, and provided attendees with examples of how industry has brought value to research projects conducted with European Research Networks (ERNs) in recent years.

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ERNs: a key EU infrastructure to partner for research activities, the why and the how

Published by

European Reference Network Industry collaboration Rare Disease Research and Drug Development

The resource explains that European Reference Networks (ERNs) are a major EU healthcare and research infrastructure that bring together clinical expertise, data and specialised resources across Europe to tackle rare and complex diseases and are valuable partners for collaborative research activities. It emphasises the untapped potential of ERNs to engage with the pharmaceutical and biotech sectors, advocating for greater awareness and public–private partnerships to accelerate rare disease research and development and benefit patients

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What can the conect4children Stichting (c4c-S) do for ERNs

Published by

C4C Clinical Research Network European Reference Network Network Orphan Drug paediatric Rare Disease Research and Drug Development

conect4children Stichting (c4c-S) is a non-profit, pan-European organisation that supports paediatric clinical research by providing expert advice and trial support services, including feasibility assessments and site identification, for ERNs and other stakeholders. Building on a successful public–private partnership, it mobilises a large network of experts and research-ready sites to improve the efficiency and quality of paediatric trials across Europe.

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Tool 2: Examples of Initiatives which Foster Public-Private Partnerships in Rare Disease and Complementary Areas

Published by

European Reference Network European Union Industry collaboration Network Rare Disease Research and Drug Development

This resource presents examples of programmes and structures which facilitate public private partnerships in rare disease or a complimentary area.

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Tool 1: The Importance of Public Private Partnerships in Rare Diseas

Published by

European Reference Network Industry collaboration Private Public Rare Disease Research and Drug Development

This resource explains the importance of public-private partnerships or collaborations for the rare disease community, in the light of the needs of the rare disease field and the current climate around research and innovation

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Together4RD Toolkit Master Document

Published by

European Reference Network Industry collaboration Rare Disease Research and Drug Development Research Collaboration

In this Master Document, you can navigate between the tools and understand the background, rationale and value behind the Toolkit, which aims to support ERNs and Industry to create and sustain public-private partnerships in rare disease research.

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Position statement on collaboration between ERNs and industry

Published by

European Reference Network Industry collaboration Rare Disease Research and Drug Development

Together4RD’s Position Statement highlights that although European Reference Networks (ERNs) have significant potential to accelerate rare disease research and innovation, collaboration with industry has been limited due to structural, regulatory and governance barriers that hinder robust partnerships. It calls for strategic action, frameworks and pilot models to enable transparent, well-supported ERN–industry collaborations that harness collective expertise and resources to better address unmet needs in rare disease research and improve Europe’s competitiveness in the field.

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Tool 3: What are ERNs?

Published by

Clinical Collaboration European Reference Network European Union Rare Disease Research Collaboration

This tool explains the origins of ERNs and highlights key resources on their conceptualisation, set-up and operations, including the legal documents on which they are based. It also points to reports and recommendations that analyse the achievements and added value of ERNs to date, indicating where there may be room for improvement, and to key resources showcasing the achievements of ERNs overall as well as where to learn more about the achievements of specific ERNs.

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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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