May 13, 2025
Explore the role of real-world evidence (RWE) in supporting the economic evaluation of cell and gene therapies (CGTs)
Explore the role of real-world evidence (RWE) in supporting the economic evaluation of cell and gene therapies (CGTs), which are often one-time treatments with the potential for transformative, long-term benefits for patients with severe conditions. Despite their promise, uncertainty in the evidence base, due to small, single-arm trials and unclear long-term effectiveness, creates challenges for payer reimbursement and policy decisions.
Technical Topics Include:
- Challenges in evaluating CGTs:
- Small patient populations and single-arm trial limitations
- Uncertainty in patient eligibility and standard of care definitions
- Durability of benefit and long-term effectiveness concerns
- Payer concerns and economic challenges:
- High up-front costs and affordability issues
- Balancing relative effectiveness, cost-effectiveness, and budget impact
- The role of RWE in addressing uncertainties:
- Informing eligibility criteria and appropriate comparators
- Assessing long-term outcomes and treatment durability
- Supporting outcome-based agreements and value-based pricing
- Internal and external validity of RWE: Strengthening payer confidence in decision-making
- Case studies: Real-world applications of RWE in policy and payment decisions for CGTs
This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:
- Frameworks for integrating RWE into payer decision-making for CGTs
- Best practices for addressing payer concerns related to CGT affordability and reimbursement
- Strategies for developing outcome-based agreements based on real-world data
- Case-based discussions on using RWE to balance access and payment considerations
This course is designed for medical operations, HEOR professionals, and policymakers looking to gain a deeper understanding of how RWE can inform economic evaluations, reimbursement strategies, and payment models for CGTs.
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 2025 conference. Separate registration is required. Visit the ISPOR 2025 Program page to register and learn more.
FACULTY MEMBERS
Schedule:
LENGTH: 4 Hours | Course runs 1 day
Sunday, 13 May 2025 | Course runs 1 Day
1:00pm-5:00pm Eastern Daylight Time (EDT)
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.