February, 9, 2023

Jennifer Fulton, Senior Biostatistician and Stephen Black, Manager of Statistics

February 2023

Implementing the Clinical Data Interchange Standards Consortium (CDISC) standards required for data management, programming, documentation, and eCTD data submission can be challenging for many reasons but is essential for increased quality and efficiency, leading to clinical research breakthroughs. If you approach your CDISC projects the way we do at Westat, you likely dive first into the SDTM and ADaM Implementation Guides.  These are valuable documents and should definitely be your primary tools through the process of programming your SDTM and ADaM data files.  But the guides do not hold all the answers you will need.  CDISC standards are a set of complex and multifaceted criteria with different components that are guided by a variety of reference resources from regulatory agencies and process organizations as well as CDISC documents and CDISC standards validation software.

Understanding What You Need from CDISC Standards

Before consulting these resources, it is important to understand the difference between “conformance” and “compliance” in the context of what is required vs. what is recommended or a best practice but is not likely to risk an all-out rejection of your submission. Conformance is voluntary adherence to a standard, rule, or requirement and use of CDISC conformance guidances is absolutely recommended but not required specifically by laws and regulations. After all, what is the point of developing standards if we don’t all use them. Compliance is required adherence to a law, regulation, or rule and must be followed or penalties will be incurred. CDISC compliance is regulated by the FDA (U.S.) and PMDA (Japan), and following compliance rules is not optional for a successful regulatory submission.

There are other considerations in deciding how to approach the level of adherence to rules and guidelines. As mentioned, if the data is intended for regulatory submission, then FDA or PMDA compliance rules are required.  But if the data is intended for a journal publication, grant application, or data warehousing, the requirements are less stringent. Sometimes the sponsor must make a judgement call.  Perhaps a Phase 1, exploratory clinical trial is not intended for immediate submission, but if the intervention proves worthy of further research, it will be more efficient in the long run if the data is standardized and CDISC compliant from the beginning.

Finding the Right Resources

How can you navigate the rules and understand the best path forward for your needs? Start with the valuable resources, references, and learning tools directly from the CDISC website including: Models and Implementation Guides for CDASH, SDTM, ADaM as well as non-clinical data and medical devices; Therapeutic Area User Guides (TAUGs); Conformance Rules; Controlled Terminology; Define-XML release information with files and links, Metadata Submission Guidelines, and Knowledge Base articles.

Documents such as the Data Standards Catalog, Technical Conformance Guide, validation rules, and other information critical to CDISC standards conformance and compliance are available on the FDA website. For more information on Japan’s PMDA regulations, see

Technical information on electronic submission of application data (FAQs, data standard catalogs, etc.) | Pharmaceuticals and Medical Devices Agency (pmda.go.jp)

There are also process organizations and CDISC standards data validators that facilitate useful direction and checks, including:

• PHUSE — The Global Healthcare Data Science Community, which provides templates for the Study Data Reviewer’s Guide and Analysis Data Reviewer’s Guide as well as white papers and a community forum
• TransCelerate – Pharmaceutical Research and Development, a nonprofit organization with a mission to collaborate across the global biopharmaceutical research and development community
• Pinnacle 21, providing CDISC standards validation software along with forums and informative articles

Adapting to an Ever-Changing Landscape

Certainly, our lives would be easier if there was a single, definitive resource that held the answer to every CDISC standards question we have.  But health research serves a variety of different needs, different customers, and different sources.  CDISC standards are an ever-growing and evolving set of criteria, and staying abreast of the wealth of available knowledge will continue to be key to successful data submissions.

Contact Information

Your comments and questions are valued and encouraged. Contact the authors at:

Jennifer Fulton
JenniferFulton@westat.com

Stephen Black
StephenBlack@westat.com