Standardizing Real World Data in Rare Disease: ATHN's Journey

The American Thrombosis and Hemostasis Network (ATHN), a nonprofit supporting rare blood disorder patients, needed to standardize real-world data from its ATHNdataset (covering over 69,000 individuals across 146 treatment centers) for FDA submission, facing three obstacles: a four-month deadline versus the typical one year, no internal FDA/CDISC SDTM expertise, and data gaps since the data wasn't originally collected for clinical studies. Certara provided hands-on training on its Pinnacle 21 platform and delivered the project as a managed service, handling SDTM mapping, ADaM dataset conversion, and submission documents through biweekly collaborative calls. Both datasets were completed within the four-month timeline, achieving FDA-compliant regulatory readiness, building ATHN's internal capability for future independent submissions, and enabling broader research use for Von Willebrand disease treatments.

Certara is a biosimulation software company (Simcyp, Phoenix) whose PK/PD and QSP models predict drug behavior in the body; the FDA itself licenses Certara's software to review sponsor submissions.

This sits in the Build stage of the AI use-case lifecycle: Named examples from the foundation-model labs and platforms pharma teams use to actually construct, train and generate the model or application.