The power of a prospective RWE study

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 The FDA’s 2019 Draft Guidance on Rare Disease Natural History Studies illustrates what agency reviewers consider best practices.

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