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Development of new effective therapies for rare diseases

Published by European Commission (EC)

Rare DiseaseGrant

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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Patient Community Advisory Boards are groups established and operated by patient advocates. They facilitate discussions in a safe harbour setting and under strict rules, an MOU and CA on the latest developments and challenges related to medical research and procedures in a disease area with the company or body conducting the research. The CAB can cover from preclinical to post marketing topics. The CAB depending on the areas of discussion can invite scientific expert to serve as advisors.

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The purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.

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Rare Diseases: Natural History Studies for Drug Development Guidance for Industry

Published by FDA

Rare DiseaseNatural History

This guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products
for rare diseases. Although the focus of this guidance is rare diseases; the recommendations in the guidance may be applicable to drug development for nonrare diseases.

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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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