Version 1.0 of the Rare Diseases Therapeutic Area User Guide was developed under the CDISC Standards Development Process and provides advice and examples for CDASH, SDTM, and ADaM, including:
- Guidance on the use of domains and variables.
- Sample annotated case report forms (aCRFs).
- Examples of SDTM datasets, with text describing the situational context and pointing out records of note.
- Guidance on the use of ADaM.
Therapeutic Area User Guides (TAUGs) extend the Foundational Standards to represent data that pertain to specific indications within disease areas. CDISC Standards and TAUGs specify how to structure the data; they do not specify what data should be collected or how to conduct clinical trials, assessments or endpoints.
Public Review Comments
CDISC posts public review comments and resolutions to ensure transparency and show implementers how comments were addressed in the standard development process.
TA Specifications
TA Specifications show how to modify TAUG examples for various versions of the SDTM and SDTMIG. These specifications assist the FDA and the Japanese PMDA with testing to enable support of the standards and inclusion in their respective Technical Conformance Guides.1,2
- https://www.fda.gov/industry/fda-resources-data-standards/study-data-standards-resources
- https://www.pmda.go.jp/english/review-services/reviews/0002.html
Partnerships
The Rare Diseases Therapeutic Area User Guide was developed in partnership with, and funded by, the National Organization for Rare Disorders (NORD®), the first national nonprofit to represent all individuals and families affected by rare disease.