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Natural history

Coverage of natural history across PatientSpotlight - the developments worth watching, the access reality, and what the next twelve months may bring. 1 piece on this topic across therapy areas, newest first within each collection.

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Explained

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ExplainedApr 26, 2026FDA · EMA · patient-advocacy

How natural-history studies became regulatory currency for rare-disease approvals

In conditions where a randomized controlled trial is impractical or unethical, well-conducted natural-history studies have become the comparator of record for FDA submissions. The methodology, the patient-advocate organizations that often run these studies, and the regulator-sponsor dialogue have all matured into a recognizable framework.

RegulatoryNatural historyRare diseaseTrial design