Clinical Trial Data Verification to Obtain Registration for Pediatric Indication
A global pharmaceutical company had submitted data for approval of a pediatric indication of an existing drug. Such approvals are often sought to extend exclusivity of the existing registration for another 18 month.
The FDA denied approval on grounds that and audit had revealed that
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There were dosing inconsistnecy at at least one site
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Informed consent and ascent was not obtained for all patients
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some participating physicians did not meet the trial protocol requirements
The client was informed that the FDA would reconsider if all sites were audited and the Intend-To-Treat population would be verified. If the data of the verified cohort were favorable the pediatric inciation would be approved.
The need for analysis analysis was time sensitive. The additional 18 months exclusivity would only be granted if the pediatric registration would occur prior to loosing exclusivity. On the other hand, the 18 months would start as soon as the pediatric registration occured, which had prompted the client to plan the registration at the out edge of the market exclusivity of the original market exclusivity.
Expedited Data Collection
QORM consultants developed an auditing protocol to focuson the required data and to ensure that not other inconsistencies would derail approval.
8 auditors visited 42 US and international sites to review data for almost 300 patients.
The auditing protocol was encoded into an Microsoft Outlook form which the auditors could complete in close-to real time. The forms were submitted through Outlook into a database. This architecture dramatically sped up the auditing completion, likely reducing the time by approximately 4 to 6 weeks compared to a paper based audit.
Data Analysis
An external biostatistics consultant reviewed the data in the database for consistency and integrity. The biostaticistician then adjucated which patient's data were acceptable.
This cohort was then submitted to the client where it was combined with patient outcome data. The statistical analysis of the resulting data set was sufficient to satisfy the protocol requirements and the pediatric indication of the drug was approved.