The described initiative aimed to gain valuable insights into patient experiences and the overall burden of a specific disease. The collaboration involved MedNavigate, a Medical Affairs consultancy, and a rare disease patient organization, collectively conducting research to support a potential label expansion for a prominent pharmaceutical corporation. Working closely with all stakeholder groups, MedNavigate facilitated a comprehensive understanding of research objectives and spearheaded lead design of a retrospective study protocol and accompanying statistical analysis plan. Multiple check-ins were conducted with stakeholders with iterative document updates. Additionally, MedNavigate took charge of establishing a HIPAA-compliant AWS infrastructure for secure data storage and analysis. Seeking and obtaining IRB approval from a renowned academic institution was a crucial milestone in the process. With all necessary approvals now in place, MedNavigate is poised to commence data analysis activities in early 2024, marking a significant step toward enhancing understanding and addressing the challenges associated with the targeted disease.
