The Situation
Strategic solutions to obtain a clinical “yes”
- Limited pivotal trial data with NOC/c
- Significant data gaps
- Complex dosing
- GVD not finalized
- Overly complex BIA
- Economic model lacked relevant Canadian components
The Solution
- Implemented a project management plan with key touch points with client to ensure alignment throughout process
- Conducted a comprehensive assessment of clinical trial data of product vs historical orphan drug comparators from a CDR perspective:
- Identified previous concerns CDR/CDEC have had with similar trial design
- Assisted in development of clinical summary by proactively addressing perceived gaps in data and concerns from previous orphan drug submissions to the CDR
- Supported the adaption of economic model for Canada
- Supported US based economics team to finalize reports to meet tight CDR deadline
- Streamlined manufacturer’s budget impact model to allow for greater transparency and simplicity
- No limits on reviews or revisions – needed to get this right the first time!
The Results
- Consolidated GVD, NICE submission and CTD data into a comprehensive clinical summary highlighting key clinical benefits and demonstrating the robustness of clinical data despite small numbers and trial design
- Attained a positive listing recommendation from CDEC with clinical criteria similar to indication