The research question
Which measures, clinical confirmations and follow-up intervals could support a useful natural-history cohort?
Proposed design
Partner-led protocol feasibility with patient interviews, a pilot diary and a separately designed longitudinal registry phase.
Illustrative design: a 4-week feasibility pilot before any longer registry; setup and recruitment are scoped separately.
Data to collect
- Disease-specific symptoms and daily function
- Treatment and care history
- Milestones and caregiver observations
- Potential clinical-record and biomarker linkage
Planned outputs
- Cohort and measurement feasibility report
- Proposed longitudinal data specification
- Recruitment, confirmation and retention requirements
What we confirm with you
We agree the population, sample size, country, collection tools, analysis plan and budget. Collection starts after the required consent, ethics or other permissions and a signed scope are in place.
- Require disease specialists and confirmed diagnostic criteria
- Demonstrate access to an eligible consenting population
- External-control or regulatory use requires a separate fit-for-purpose design
Clinical records, laboratory results, photographs and device integrations are included only when access, collection quality and permitted use have been verified. Self-reported findings are identified as such.
Research context
These published sources explain the research need or method. They are not RIDLY customers, endorsements or purchase commitments.
- FDA rare-disease natural-history research workshop
FDA examined the design and potential uses of registries and natural-history studies in rare-disease development.
Related dataset profiles
For an existing-data inquiry, start with a coverage review.