Case Studies
Building a Global Patient Funnel to Predict Uptake for a Gene Therapy Across 15 Countries
- October 27, 2026
- 4 min read
- thelansis20
The question
How many patients could this gene therapy really treat across 15 markets, and how fast?
What we did
Harmonized the epidemiology, mapped a six-step patient funnel, layered in access timelines and ran three scenarios.
The result
A defensible country-level model showing a treatable population 40–55% smaller than assumed.
01 · CONTEXT
A promising therapy facing structural hurdles
A global biotech developing an ex-vivo gene therapy for a rare neuromuscular disorder asked Thelansis to build a cross-market patient funnel and uptake forecast. The therapy was nearing regulatory submissions in the US, EU5 and select APAC markets. Early clinical data showed durable functional gains and a lower hospitalization burden, but the commercial challenges were structural.
Epidemiology was inconsistent across markets.
Diagnostic rates ranged from above 70% in Germany to below 25% in several APAC markets, driven by limited NGS and enzyme testing access.
Payers were signaling interest in outcomes-linked contracting, so precise patient identification was essential for budget-impact planning.
The sponsor needed one evidence-based model that could:
- Quantify the true treatable population across 15 markets
- Identify friction points in the diagnostic and referral pathway
- Predict realistic uptake under multiple access scenarios
02 · THE CHALLENGE
Three teams, three sets of assumptions
Epidemiology and forecasting inputs came from HEOR, medical and commercial teams, each working from its own assumptions.
- There was no standard definition of an eligible patient by genotype, age limits or functional class.
- Several markets lacked structured pre-symptomatic screening data, though newborn screening pilots were running in France, Japan and Brazil.
- Patient leakage wasn't measured along the path from primary care to a neuromuscular specialist, genetic confirmation and treatment-center readiness.
That made pricing, payer review timelines and launch sequencing hard to justify. The brief was to move beyond prevalence figures and capture real-world conversion at every step.
03 · OUR APPROACH
From prevalence to real-world uptake, in four steps
The largest gaps appeared at genetic confirmation, especially where testing is self-pay. Leakage was measured from:
The model accounted for expected HTA durations, anticipated contracting models, capacity limits at authorized treatment centers, and cold-chain and apheresis logistics. Country-specific access start dates were then layered onto the funnel to produce realistic adoption curves.
Typical HTA review time, in months
Each scenario produced a country-level patient funnel aligned with clinical, access and operational realities.
04 · KEY INSIGHTS
What the model revealed
Diagnostic friction varied drastically by region
System readiness was the real bottleneck
Even in high-diagnosis markets, treatment-center capacity limited year-1 uptake. Italy and Japan had strong apheresis and infusion pipelines, while Spain, Brazil and Korea needed infrastructure ramp-up before treating their first patient.
Uptake shifted materially by scenario
Compared with the base case, better diagnostic pathways raised year-5 treated patients by 15–30%, and faster access in Japan and the UK pulled peak uptake forward by 6–9 months.
05 · IMPACT DELIVERED
What the sponsor could do next
Related topics
Share this case study
Planning a launch across multiple markets?
Services behind this work
Market Forecasting
Consulting
Epidemiology Insights and Disease Burden
Reports & Insights
Market Access and Reimbursement Insights
Reports & Insights
EpiLansis
Strategy Product
- Report & Insights
- Strategy Products
Strategy Products
- Consulting Services
- Media
Media
