Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such cases, expanded access (or "compassionate use") provides a potential means of accessing unapproved investigational medicines. It is also possible to capture and...
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| Main Authors: | , , , , |
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| Format: | Book |
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Frontiers Media S.A.,
2022-05-01T00:00:00Z.
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A1234.567 |
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