Case In Point
ISS/ISE and Regulatory Success for a GI Therapeutic
About Client
The sponsor initially engaged Everest for ad hoc statistical support based on a word-of-mouth referral.
Following the success of that early collaboration, Everest was brought in to support an NDA submission for what is now a well-known Ulcerative Colitis treatment, owned by a top-5 pharmaceutical company. Everest remains a long-term partner in the product’s development and global regulatory submissions.
Challenge
- Legacy datasets that were not CDISC-compliant
- Preparing robust and defensible statistical methodology for ISS and ISE with limited timeframes
- Supporting submissions across multiple global regulatory bodies (EMA, PMDA, DCGI), each with differing requirements
Solution
- Converted legacy datasets into CDISC-compliant SDTM formats, including preparation of all required documentation for submission
- Executed thorough programmatic validation and data remediation to ensure alignment with FDA guidance
- Developed a comprehensive Statistical Analysis Plan (SAP) for both ISS and ISE, including statistical methodologies and mock TLGs
- Delivered BIMO/OSI listings for pivotal studies
- Provided submission deliverables including 120-day and 180-day post-submission safety updates (DSUs), along with subgroup analyses
- Continued regulatory support extended to EMA, PMDA, and DCGI submissions
Results
- Achieved FDA agreement on ISS and ISE statistical methodology during the pre-NDA meeting
- The NDA was approved with no FDA inspection findings
- Client praised Everest’s responsiveness, flexibility, attention to detail, and consistent delivery quality
