SITUATION
A biotech company developing a single-dose gene therapy for a rare neuromuscular disorder had secured orphan drug designation in the US and EU. With pivotal trial results expected within the year, the leadership team needed to prepare for global rollout — starting with a launch sequencing strategy that balanced commercial opportunity, regulatory complexity, and real-world system readiness.
The asset had strong clinical differentiation, but the company lacked prior experience launching cell and gene therapies across multiple regions. The team needed a data-driven plan to determine which countries to prioritize, how to align functions internally, and how to avoid delays post-approval.
CHALLENGE
Gene therapy launch planning is complex due to unique factors: small patient populations, high one-time costs, manufacturing constraints, and payer hesitancy. In this case, the client faced several strategic challenges:
- No clear framework for sequencing country-level launches based on access and regulatory dynamics
- Uncertainty about which HTA markets would accept surrogate endpoints or RWE in initial submissions
- Operational gaps in identifying treatment-capable centers and supply chain infrastructure per region
- Pressure from leadership to align investor timelines with realistic market readiness
The team needed a unified roadmap that connected regulatory strategy, commercial planning, and local feasibility.
APPROACH
ConsaInsights was engaged to build a comprehensive launch sequencing strategy for 12 key markets across North America, EU5, APAC, and LATAM. The approach integrated competitive intelligence, market access benchmarking, and operational readiness insights.
Regulatory & HTA Pathway Mapping
We analyzed regulatory timelines, submission routes, and HTA precedents for 15 rare disease therapies with orphan designation across the same regions. This allowed us to anticipate review durations, data acceptance hurdles, and conditional approval opportunities.
Market Readiness & Site Capability Scoring
We assessed each country’s real-world readiness to deliver gene therapy, including treatment center accreditation, patient identification infrastructure, and supply chain robustness. Countries were scored using a proprietary readiness model.
Commercial Impact & Uptake Modeling
We modelled forecasted uptake based on prevalence data, local diagnosis rates, payer feedback, and physician familiarity with advanced therapies. This helped prioritize not just size but launch timing sensitivity.