Rare Diseases Clinical Research Network (RDCRN)

Published by NCATS

Clinical DevelopmentNetworkNCATSRDCRN

The RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.

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The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary model centred upon an international group of academic and industry drug development experts, including patient representatives. TACT gives advice on the translational and development pathway of therapeutic programs in rare Neuromuscular Diseases. Due to its success in the neuromuscular field, a toolkit to replicate the TACT model has been produced. The Advisory Committee for Therapeutics (ACT) toolkit is intended to support rare disease communities outside of Neuromuscular to establish their own ACT, by providing them with step-by-step guidance and template documentation.

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Participating in research provides patients with access to cutting-edge treatments and benefit the population and the health services as a whole by leading to the development of more effective, high quality, cost-efficient treatments and healthcare delivery models. ERNs represent an unicum in the clinical and research worldwide collaborative panorama, indeed 24 ERNs span the over 6000 rare diseases by involving 300 EU HCPs and over 1000 specialized units in 25 EU countries

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