May 17, 2026
Explore how real-world evidence can inform value, affordability, and access decisions for cell and gene therapies.
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.
LEVEL: Intermediate
TRACK: Real World Data & Information Systems
This short course is offered in-person at the ISPOR 2026 conference. Separate registration is required. Registration coming soon. Visit the ISPOR 2026 Program page to learn more.
FACULTY MEMBERS
Schedule:
LENGTH: 4 Hours | Course runs 1 day
Tuesday, 17 May 2026 | Course runs 1 Day
ISPOR short courses are designed to enhance knowledge and techniques in core health economics and outcomes research (HEOR) topics as well as emerging trends in the field. Short courses offer 4 or 8 hours of premium scientific education and an electronic course book. Active attendee participation combined with our expert faculty creates an immersive and impactful learning experience. Short courses are not recorded and are only available during the live course presentation.