TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases
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DOI: 10.1186/s13023-020-01370-3
Abstract: Background 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 based… read more here.
Keywords: evidence generation; specialised treatments; rare diseases; evidence ... See more keywords