The Challenge
For a leading multinational pharmaceutical corporation preparing to launch a new route of administration (RoA) for spinal muscular atrophy (SMA), the strategic question was simple: where should we launch first?
The existing global forecast treated ten priority markets largely as a single population scaled by market size. While this approach can work for large primary-care brands, it failed to capture the unique dynamics of SMA, where patient opportunity is shaped by local epidemiology, diagnosis pathways, treatment eligibility, and access.
Key challenges included:
Market size did not reflect patient opportunity
Birth cohorts, SMA prevalence, newborn screening, diagnosis rates, and biomarker eligibility varied significantly across markets.
Time to treatment differed by market
Diagnosis pathways and reimbursement/funding timelines influenced when eligible patients could realistically access treatment.
Launch demand was not purely new-patient driven
A significant portion of early opportunity could come from patients switching from existing therapies.
Uncertainty was not adequately captured
Point estimates for diagnosis, eligibility, switching, access, and uptake created risk around market prioritization.
The client’s leadership team needed a market-specific view of not just how many patients existed, but where and when eligible patients could realistically access treatment.
Our Solution
Chryselys developed a patient-based market prioritization framework that maintained a consistent methodology across all ten markets while capturing local market dynamics.
A systematic approach from patient opportunity to launch sequencing:
Size the patient opportunity
Built market-specific epidemiology models incorporating birth cohorts, prevalence, newborn screening, diagnosis pathways, and biomarker eligibility.
Segment treatment opportunity
Distinguished newly diagnosed/treatment-naïve patients from existing treated patients and potential switchers.
Assess access readiness
Incorporated country-specific reimbursement, funding, coverage restrictions, and expected time to patient access.
Model adoption and switching
Developed market-specific uptake curves informed by rare-disease and RoA launch analogs, competitive dynamics, and switching potential.
Quantify uncertainty
Used scenario analysis and Monte Carlo simulations to assess variability in key assumptions and generate forecast ranges.
Prioritize markets
Combined patient opportunity, access readiness, uptake potential, launch timing, and risk to identify priority markets and sequencing.
Business Impact
The framework shifted launch planning from a market-size-based exercise to an evidence-based, patient-centric sequencing strategy.
- Prioritized markets based on accessible patient opportunities and launch readiness.
- Identified near-term opportunities across both new patients and existing patients eligible to switch.
- Quantified launch risks associated with diagnosis, access, switching, and uptake assumptions.
- Enabled scenario-based decisions using forecast ranges rather than relying on single-point estimates.
- Improved resource allocation by differentiating first-wave, subsequent-wave, and preparation of markets.
- Aligned global and local teams through a transparent and consistent forecasting methodology.
- Accelerated launch planning with actionable, evidence-based recommendations.
