Applying statistical and programming expertise to overcome FDA challenges
Case study
Challenge
A biotech company, specialising in infectious disease vaccines addressing unmet medical needs had reached the critical phase of preparing for FDA submission after finalising two pivotal phase 3 Clinical Study Reports (CSRs). Unexpectedly, the FDA’s pre-BLA meeting introduced comments that necessitated revisions in statistical analysis and data collection. This unforeseen challenge posed a significant roadblock to the client’s regulatory timeline, especially since they lacked an in-house statistician to address these concerns.
Solution
To tackle this challenge head-on, ICON immediately mobilised a team of experts, including a lead statistician and a programming manager with extensive study knowledge. Their primary objective was to engage directly with the FDA during the sponsor’s scheduled meeting to comprehensively understand, discuss, and align on the required modifications to derivations and reporting of solicited adverse events. Our strategic approach involved the following key steps:
- FDA meeting participation: Our lead statistician and programming manager actively participated in the FDA meeting, representing the sponsor’s interests and ensuring clear communication with the regulatory body.
- Dataset revisions: Post-meeting, our team promptly updated the Study Data Tabulation Model (SDTM) and Analysis Data Model (ADaM) datasets to address the specific FDA comments, ensuring data integrity and compliance.
- Additional sensitivity analysis: Recognising the importance of robust statistical analysis, our experts developed and incorporated additional sensitivity analyses, by creating four new sensitivity analysis tables, to provide a comprehensive view of the data and enhance its robustness.
- Regeneration of tables, figures, and listings: A meticulous effort was made to regenerate all impacted Tables, Figures, and Listings (a total of 118), ensuring they accurately reflected the revised data and statistical analyses.
Sponsor collaboration: Throughout the process, we maintained a collaborative relationship with the sponsor, ensuring their insights and feedback were integrated into the revisions.
Outcome
The collaboration between our team and the biotech sponsor yielded substantial benefits:
- Timely BLA submission: Despite the unexpected challenges, our efficient response ensured that the revised CSR was approved within just three months, aligning perfectly with the client’s BLA submission timeline.
- Comprehensive data: Our team’s expertise in statistical analysis and data management resulted in a comprehensive and robust dataset that met the FDA’s stringent requirements.
- FDA alignment: Through direct engagement with the FDA, our experts successfully clarified uncertainties, gaining alignment on data derivations and reporting of solicited adverse events.
Enhanced data quality: The addition of sensitivity analyses and thorough regeneration of tables and figures enhanced the overall quality and reliability of the CSR.
This case study exemplifies our commitment to providing tailored solutions to our biotech clients, especially in situations where unforeseen challenges threaten regulatory timelines. By leveraging our expertise, collaborating closely with the sponsor, and effectively engaging with the FDA, we not only overcame obstacles but also ensured a seamless BLA submission, ultimately advancing our client’s drug development objectives.
For more information, visit:
ICONplc.com/biostats