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...
Saved in:
| Main Authors: | , , , , |
|---|---|
| Format: | Book |
| Published: |
Frontiers Media S.A.,
2022-05-01T00:00:00Z.
|
| Subjects: | |
| Online Access: | Connect to this object online. |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|