RESEARCH HIGHLIGHTS
The megafund model to finance development of orphan drugs: analysing the NCATS portfolio
March 25, 2015 A paper published in Science Translational Medicine demonstrates how financing a programme used by the National Center for Advancing Translational Sciences (NCATS) – the megafund model – could reduce the risk associated with investing in developing orphan drugs. A megafund is a “financial investment fund in…
Orphanet Report Series update on Registries and Research Infrastructures useful to Rare Diseases in Europe
March 23, 2015 Patient registries and databases constitute key instruments to develop clinical research in the field of rare diseases, to improve patient care and healthcare planning. Additionally, registries of patients treated with orphan drugs are relevant as they allow post marketing authorisation monitoring.…
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