Case Studies: Navigating Approvals in Canada

the situation

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

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

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