Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial data

Area of Science:

• Pharmacovigilance
• Clinical Trial Management
• Regulatory Science

Background:

• Clinical trial registries like ClinicalTrials.gov (CTG) are vital for tracking studies, especially for orphan drugs (ODs) for rare diseases.
• Accuracy of data in CTG is critical, yet discrepancies may exist between registry information and data supporting drug approval (e.g., FDA Medical Reviews).

Purpose of the Study:

• To audit and compare trial-related data (phase, enrollment, enrollment attribute) between FDA Medical Reviews and CTG for FDA-approved ODs.
• To identify the extent of discrepancies in clinical trial data between regulatory documents and public registries.

Main Methods:

• An audit was conducted comparing data from FDA Medical Reviews with corresponding entries in CTG for 63 approved ODs.
• Study identifiers from Medical Reviews were used to match trials in CTG, with specific attention to phase, enrollment numbers, and enrollment attributes.

Main Results:

• Of 422 trials identified in FDA Medical Reviews for 63 ODs, only 202 were found registered in CTG.
• Exact matches for trial phase data were found in only 75% of cases, enrollment data in 70%, and enrollment attribute in 91% when comparing FDA documents to CTG.
• Similar discrepancies were observed for pivotal trials.

Conclusions:

• Significant data discrepancies exist between FDA approval documents and ClinicalTrials.gov for orphan drugs.
• Recommendations include consistently providing trial IDs in FDA Medical Reviews for unambiguous identification in CTG.
• Continuous updating of CTG records with relevant information is essential to ensure data accuracy and facilitate drug development oversight.