Event

Cell and gene therapies (CGTs) have the potential to deliver transformative, long-term benefits through one-time treatment, often for severe or rare conditions. However, these therapies are frequently supported by limited clinical evidence, such as small or single-arm trials, creating uncertainty for payers related to eligible populations, appropriate comparators, durability of benefit, and relative effectiveness. These uncertainties are compounded by the high upfront costs of CGTs and concerns about affordability and budget impact.

Technical Topics Include:

• The role of RWE in supporting economic evaluation of CGTs
• Addressing evidence gaps related to eligibility, comparators, and natural disease progression
• Evaluating relative effectiveness and durability of benefit using RWE
• Payer archetypes focused on value, cost-effectiveness, and budget impact
• Assessing internal and external validity of real-world data
• Using RWE to inform payment models and outcome-based agreements

This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:

• Practical guidance on planning and generating RWE for CGTs
• Examples of RWE use in payer decision-making and policy development
• Frameworks for balancing access, value, and affordability
• Real-world case examples illustrating RWE-informed economic evaluation

Participants will gain a clearer understanding of how RWE can be used to address payer uncertainty and support informed reimbursement and policy decisions for cell and gene therapies across diverse stakeholder contexts.

PREREQUISITE: This course requires familiarity with basic economic evaluation and HTA concepts and methodologies of pharmaceuticals.

LENGTH: 4 Hours | Course runs 1 day

Sunday, 17 May 2026 | Course runs 1 Day
1:00pm-5:00pm Eastern Daylight Time (EDT)