A natural history study collects information about the course of a disease in people who are not receiving the investigational treatment, to understand how it develops, which outcomes matter and how to design trials for it.
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Years
In short
Explained
For common diseases, decades of trials and clinical experience show how the disease progresses and which endpoints work. For many rare diseases that knowledge does not exist. A natural history study fills the gap: it shows how fast the disease progresses, how variable it is between patients, which measures change meaningfully over a trial-length period and which patient subgroups progress fastest.
Good natural history data can shape every later decision, from the choice of primary endpoint to the sample size, and in some cases provide an external comparator where a concurrent control arm is not feasible. Because the data may later support regulatory decisions, collect it with the same attention to consistency, audit trail and definitions as trial data. See EDC for rare disease trials and EDC for registry studies.
It is a clinical study, but observational: participants are not assigned an investigational intervention.
They provide the disease knowledge needed to select endpoints, size trials and sometimes serve as external controls.
In some circumstances, as an external control. Its suitability depends on data quality and comparability, and should be discussed with regulators.
Often years, because progression is slow. Long follow-up needs low-burden, consistent data collection.
Long schedules, ePRO and a full audit trail. Free sandbox.