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TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases
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 on a small or uncontrolled trial, as randomised controlled trials (RCTs) of s...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7251888/ https://www.ncbi.nlm.nih.gov/pubmed/32456653 http://dx.doi.org/10.1186/s13023-020-01370-3 |