Building a Global Patient Funnel to Predict Uptake for a Gene Therapy Across 15 Countries
Context: A global biotech developing an ex-vivo gene therapy for a rare neuromuscular disorder engaged Thelansis to build a cross-market patient funnel and uptake forecast. The therapy was approaching regulatory submissions in the US, EU5, and select APAC markets. While early clinical data showed durable functional gains and reduced hospitalization burden, the commercial challenge was more structural: The disease lacked consistent epidemiology across markets Diagnostic rates varied from >70% in Germany to <25% across several APAC markets, driven by limited NGS & enzyme testing access Payers were already signaling demand for outcomes linked contracting, making precise patient identification essential for budget-impact planning The sponsor needed a unified, evidence-based model that could: Quantify the true “treatable” population across 15 markets Identify points of friction in the diagnostic and referral pathw...