Natural history data may one day define your endpoints or serve as an external comparator. Capture collects it with the same controls as a trial: structured assessments, caregiver reports, remote questionnaires and a complete audit trail over years.
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| Assessment | BL | M6 | Y1 | Y2 | Y3 | Y4 | Y5 |
|---|---|---|---|---|---|---|---|
| Clinical assessment | |||||||
| Functional scale | |||||||
| Caregiver report | Q | Q | Q | Q | Q | Q | |
| Patient questionnaire | Q | Q | Q | Q | Q | Q | |
| Biomarker sample |
Q = quarterly remote questionnaires between visits
What natural history studies need
Data quality
Natural history studies are observational, and they are sometimes run with lighter systems than interventional trials: spreadsheets, survey tools or registries without audit trails. That can be a costly shortcut. If the data later informs a regulatory submission, reviewers will ask how it was collected, whether definitions changed, who entered and changed values, and how complete it is. A dataset that cannot answer those questions has limited value as evidence.
Using a trial-grade system from the start costs little more. In Capture every value has a field-level audit trail, approved forms are locked and versioned, edit checks raise queries at entry, and exports include a data dictionary and annotated CRF. See the natural history study glossary entry for how these studies fit into development programmes.
Many rare diseases affect children, and much of the meaningful information comes from parents: sleep, feeding, seizures, behaviour, daily function. Assessments can be set to be completed by a caregiver on their own phone, alongside clinician-rated forms completed at visits. See the parent proxy ePRO template.
Patient-reported
Participant, own phone
Observer-reported
Caregiver
Clinician-reported
Site staff, clinic tablet
Performance outcome
Site-run test, e.g. chair rise
Long schedules
Visits anchored to baseline, recurring questionnaires between them, and windows and reminders that keep a years-long schedule on track. The status overview shows every participant against every visit, and the dashboard flags participants falling behind.
Build visits and remote questionnaires in the free sandbox.
Study build
Instrument versions and administration methods written down.
Disease milestones and events defined in the protocol.
Who completes which assessment, by age.
Including possible regulatory use of the data.
Remote questionnaires and reminders between visits.
CDASH-style forms and a data dictionary for later use.
Later use
| Question | What answers it |
|---|---|
| Were assessments done the same way throughout? | Locked, versioned forms and a record of every form change and when it took effect. |
| Who entered and changed each value? | A field-level audit trail with user, time, old and new value and reason. |
| How complete is the dataset? | Visit and questionnaire completion by participant, with reasons for missed assessments. |
| How were events defined? | Protocol definitions reflected in structured fields, not free text. |
| Is the population comparable to the trial? | Eligibility-relevant characteristics captured at baseline in structured form. |
| Can the data be traced to source? | Source documents at sites and SDV on critical fields. |
Endpoint development
In rare diseases, natural history studies often show which measures change meaningfully over a realistic trial duration and which are too noisy or too slow. That makes them the evidence base for endpoint selection and for sample size assumptions in the pivotal trial. To serve that purpose, collect candidate measures at consistent intervals, record assessor and administration conditions, and capture caregiver and patient views of what matters, so the chosen endpoint can be linked to meaningful change. See clinical trial endpoints.
Ideally the same kind of validated data capture system used for trials, so the data can support regulatory use later.
Yes. Assessments can be set to be completed by a caregiver on their own phone.
In some circumstances. Suitability depends on data quality and comparability, and should be discussed with regulators.
Often several years. Long schedules with remote questionnaires help retention.
Yes, in the free sandbox.
Keep exploring
Trial-grade controls for observational studies. Free sandbox.