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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Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives

Published by IACMHR Co., Ltd.

Orphan Drug Regulatory Science

This paper describes the current status of the regulation of rare diseases and orphan drugs in Asia and we comparatively analyze the regulation of rare diseases and orphan drugs worldwide in order to examine the challenges to and future perspectives on promoting research on rare diseases and development of orphan drugs in China and other Asian countries.

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Ethic’s Committees assessment of protocols for trials with orphan medications

Published by IRDiRC

Ethic and Legal Issue Guideline Ethical Assessment Orphan Drug Protocol Assessment Regulatory Sciences

Ethical assessment of clinical trials in orphan diseases should be done by experienced committees, and should ensure that all relevant aspects of this particular type of trials is properly assessed.

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Orphanet database

Published by IRDiRC

Database Database or Tool Orphanet Orphan Drug Rare Disease Research and Drug Development

Orphanet is a unique public resource worldwide, gathering and improving knowledge on all rare diseases (RD), affecting less than 1 in 2’000 people in the European population.

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IRDiRC Recognized Resources

Published by IRDiRC

Consulting & Advice IRDiRC Recognised Resource Orphan Drug Orphan Drug Development Guideline Research and Drug Development Tranlational Medicine Use Case

IRDiRC Recognized Resources is a quality indicator, which is expected to help accelerate the pace of translating discoveries into clinical applications. It therefore lists a number of resources important for Orphan Drug Development.

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Use of Biomarkers in Orphan Drug Development

Published by IRDiRC

Biomarker Development Clinical Development Endpoint EndpoInternationalBiomarker Orphan Drug Regulatory Sciences Research and Drug Development

Biomarkers can be utilized as primary surrogate endpoInternationalas a basis for accelerated approval (US), and as secondary or exploratory endpoints for drug mechanism of action or aspects of Safety monitoring.

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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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