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

RWD differs fundamentally from the structured data collected in clinical trials. RWD often lacks the uniformity required for FDA submissions. ATHN’s team faced the additional challenge of having limited experience in FDA regulatory compliance and the technical expertise needed to map data to SDTM format.

ATHN faced three primary challenges:

1. Urgent timelines: Both submissions needed to be completed in under four months — a task that typically takes a year — posing significant logistical hurdles.
2. Lack of resources: With a small team, many of whom were unfamiliar with SDTM standards, ATHN needed rapid upskilling or external support to ensure success.
3. Gaps in data: The ATHNdataset was not collected for use in a clinical study, which meant data wasn’t collected using standardized eCRFs.

ATHN had already engaged Formedix, now part of Pinnacle 21 by Certara, to help their in-house team understand and implement CDISC SDTM mapping. Using our Pinnacle 21 clinical data management and automation suite, we provided hands-on training on designing the SDTM outputs using our platform’s built-in templates and using the MDR mapping engine to run the conversions.

Given the tight timeline and the linear process of preparing submission-ready datasets (progressing serially from raw data to SDTM, analysis datasets (ADaM), > Tables, Listings & Figures (TLFs), and finally clinical reports), Certara delivered this project as a managed, tech-enabled service, powered by the Pinnacle 21 MDR.

1. Training and Skill Development

Certara worked closely with ATHN’s biostatistics and data science staff helping them build foundational knowledge of SDTM standards. This allowed the team to observe and understand the mapping and conversion process using the Pinnacle 21 MDR, ensuring long-term capability development.

2. SDTM Mapping and Conversions

Certara took on the responsibility for SDTM mapping, conversions into ADaM, and creating all associated tables, listings, and figures necessary for regulatory submissions.

3. Collaborative and Agile Execution

Biweekly calls between Certara and ATHN allowed quick resolution of challenges, such as additional data requirements from clinicians or adjustments to the analysis process. This collaboration, as well as automation through use of the MDR, ensured both FDA and sponsor deadlines were met.

Unlike clinical data, which is collected under controlled conditions, Real World Data can be especially messy and complex. The FDA asked ATHN for a presubmission to help them understand the RWD, how the data was collected, and the quality of the data. Certara’s expertise in harmonizing RWD into the standardized CDISC SDTM format helped overcome this significant barrier.

Despite the inherent challenges of working with RWD under a tight deadline, Certara and ATHN successfully delivered both datasets within the mandated four-month timeline. Key outcomes included:

• Regulatory Compliance Achieved
  Both submissions aligned with FDA standards, enabling the sponsor to use ATHN’s data for analysis and regulatory submission.

• Enhanced Capability for Future Submissions
  By partnering with Certara, ATHN not only met immediate project goals but also upskilled their team on regulatory requirements and software competency, building a foundation for independent standardized data submissions in the future.

• Advancing Research and Care in Rare Diseases
  The successful standardization of ATHN’s Real World Data into SDTM format ensures that this valuable dataset can now be more effectively used by researchers, sponsors, and healthcare providers to improve patient outcomes and support new therapies.

Rare diseases like Von Willebrand often lack the robust clinical trial data available for more common conditions. ATHN’s RWD provides a critical lens into how treatments perform in everyday clinical settings. By converting ATHN’s RWD into SDTM format, Certara helped unlock the potential of this data for advancing research, developing treatment standards, and ultimately improving patient care.

ATHN’s partnership with Certara demonstrates how strategic collaboration, expertise in RWD transformation, use of technology to accelerate timelines, and a commitment to excellence can drive successful outcomes, even under challenging circumstances.

If your organization is working with Real World Evidence and seeks to unlock its full potential, contact Certara today to discover how our expert data standardization and regulatory compliance services can help you achieve your goals.