An Approach to Mitigating Challenges of Data Sharing in the Development of Biomarkers

2021 FDA Science Forum

• Authors: VanHeusen, Margaret FDA/CDER; Leptak, Christopher FDA/CDER

• Center: Center for Drug Evaluation and Research

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Abstract

Background

Biomarker integration into drug development can happen through various pathways. These pathways are often executed in parallel with one and other and are not mutually exclusive.

Purpose

This project, beginning January 2020, aims to help address barriers seen with the integration of biomarkers in drug development and to assess if we can use IND/NDA/BLA biomarker data submitted to FDA to answer new drug development questions. Historically access to data has been the largest challenge due its proprietary nature and lack of consistency in data collection, analysis, and interpretation between groups. Therefore, can we explored using data previously submitted to the FDA during the application process to form a metadata repository that can be queried to address specific drug development specific, while defining the context for which this data might be appropriately used.

Methodology

The authors chose to use fecal calprotectin as a test case since here is interest both internally and externally, large amounts of data has been collected by multiple programs. Additionally, FDA staff have discussed possible utility for a fecal calprotectin but has been unable to draw definition conclusions about its use.

Results

Of the three trials identified for this case study, we focused our efforts on the one with the largest number of patients and the longest duration. Preliminary data showed a modest correlation between the raw fecal calprotectin levels and the raw endoscopy score.

Conclusion

Since beginning this project in January 2020, it is encouraging that through this process we have learned there is accessible data that could be utilized to answer current drug development questions. Future steps and challenges of this project include:

• Identify/grouping analysis methods used for fecal calprotectin testing and endoscopy scoring (including hundreds of different testing level cut off/significant value points for fecal calprotectin, some which are specific to a single site, and identifying methods to avoid double counting patients)
• Data standardization/normalization (including grouping analysis methods, time frames, standardizing by time frame, endoscopy scoring system)
• Collaborating with experts to identify appropriate cut offs points

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