Like many rare diseases, this one had few reported cases in the literature—and models suggest that a substantial number of patients are not correctly diagnosed. How can a client—who is new to rare diseases—effectively prepare to launch the first gene therapy for affected patients?
Our client needed to both increase global HCP awareness of the disease and establish themselves as a trusted leader in the field.
We began by gathering insights from experts around the world to help our client understand the therapeutic landscape and the diagnostic process. Armed with these insights—and a comprehensive literature review—we developed a strategy to increase global awareness and diagnosis of this disease. A suite of materials educated top-tier and regional neurologists and laboratory specialists about this disease; congress materials communicated developments in the clinical trial program; a private global scientific exchange meeting fostered dialogue among leading physicians from around the world.
Attendance at sponsored presentations resulted in identification of new patients. The global scientific exchange meeting brought together over 300 physicians to share learnings across multiple disease states—and small-group discussions even revealed potential new research partnerships.
“Everyone said the outcome went above and beyond their expectations. Given changes, live to virtual format, we had the A++ team delivering even though the timelines were tight and there were lots of changes.”– VP, Global Scientific Affairs/Global Medical Affairs