TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases | Orphanet Journal of Rare Diseases | Full Text

TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases | Orphanet Journal of Rare Diseases | Full Text

TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases

Many treatments developed for rare diseases will have an Orphan Medicinal Product (OMP) designation, indicating that they are likely to deliver benefit in an area of high unmet need. Their approval may be base...

Authors:Lieven Annemans and Amr Makady

Citation:Orphanet Journal of Rare Diseases 2020 15:127

Content type:Position statement

Published on: 26 May 2020

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