As soon as a company has identified
a compound with biological activity
in a rare disease, it’s time to plan
a natural history study. For rare
diseases, collecting natural history
data is as essential as preclinical work
or a Phase I trial. It’s not just nice-tohave context; it’s the starting point for
efficient development. Natural history
data can inform pivotal development
decisions if the study is conducted
in parallel with the clinical program.
The FDA’s 2019 Draft Guidance on
Rare Disease Natural History Studies
illustrates what agency reviewers
consider best practices.
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