Building a Payer-Aligned Access Strategy for a High-Uncertainty Launch
A US-based gene therapy manufacturer is preparing to launch a first-in-class treatment for a rare metabolic disease across the EU5. There are no approved therapies, with standard of care limited to dietary management.
The asset represents a significant advance, but would face high scrutiny around price, durability and evidentiary uncertainty – trial data is promising but immature, with small patient numbers and a reliance on surrogate endpoints.
The Challenge
The health economic case was difficult given the negligible cost of existing care. Competitors were progressing in development, and unmet need was not consistently rated as high.
A structured, cross-market strategy was required ahead of launch to develop the client’s internal capability to design innovative contracting approaches capable of addressing payer concerns across multiple European markets.
Verpora Approach
Verpora began with a structured evidence review, synthesising clinical data, socioeconomic context, competitor positioning and the broader value narrative from both client and independent sources.
This was followed by internal stakeholder interviews and qualitative discussions with EU5 payers to test assumptions around value, willingness to pay and access barriers. Early concepts for value-based and innovative contracting constructs were generated and iteratively pressure tested.
Non-viable options were eliminated quickly. Feasible constructs were aligned against payer appetite and client commercial constraints, resulting in a structured playbook of contracting frameworks tailored to different EU5 payer archetypes. Each framework incorporated clear value narratives alongside financial and commercial terms to support launch readiness.
Client Impact
The client entered launch preparation with clear, payer-informed contracting frameworks across all five major European markets. These frameworks provided structure and confidence in engagement planning and aligned internal stakeholders around a consistent access strategy eighteen months ahead of launch.
The approach was subsequently extended to inform evaluation of contracting options within US payer settings.
“The Verpora team have undertaken invaluable work to support both the global and US launch strategy of our new gene therapy. Their due diligence and comprehensive methodological process has led to the synthesis of a phenomenal go-to VBA/IC resource for our access teams”
VP Head of Market Access & Pricing EMEA
Key Takeaway
High-cost gene therapies in rare disease settings often launch with limited long-term data and significant uncertainty. Early development of payer-informed contracting frameworks can reduce risk, strengthen credibility and improve launch preparedness across markets.
