Important tool in our rare disease toolbox: hybrid retrospective-prospective natural history studies serve well as external comparators for rare disease studies
Natural history studies (NHS) can support regulatory decision-making at different stages of the drug product life cycle and are especially important in the context of rare diseases, which are associated with not only delayed or erroneous diagnoses but also a lack of approved treatments. Real-world e...
Saved in:
| Main Authors: | , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2024-12-01
|
| Series: | Frontiers in Drug Safety and Regulation |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fdsfr.2024.1418050/full |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|