ISSUE: Many CAR-T therapies are being developed for late-stage rare hemato-oncology diseases, where there is a significant unmet need. Under these conditions, RCTs may not be possible due to lack of clinical equipoise leading to ethical concerns due to emerging data on the effectiveness of these types of therapies; significant unmet need where alternative treatment options are not able to deliver the efficacy to prolong disease progression, improve quality of life and symptoms of disease, and extend survival. Many HTA agencies still have a strong preference for RCT data for comparative effectiveness. In the absence of evidence from RCTs, indirect treatment comparison (ITC) methods can be used to generate comparative data, however, these methods are not always accepted by HTA agencies. There is an urgent need to explore this issue from a multi-stakeholder perspective

OVERVIEW: This panel will debate whether single-arm studies of CAR-T therapies can meet the evidence requirements of global HTA bodies. Dr. Satish Valluri will moderate the panel and provide an overview of the current landscape of HTA requirements on comparative evidence for CAR-T therapies, as well as pose key questions for the panelists to debate, including:

• What are the challenges with conducting RCTs for CAR-T therapies?
• What are alternative approaches to RCTs that can generate robust comparative evidence?
• What role can RWE play in these situations?
• Under what clinical and methodological conditions is it sufficient to generate comparative data using RWE to justify the value of CAR-T therapies?
• Which methods are most acceptable from an HTA perspective, and what are the challenges?

International experts from the US, Canada, and Europe will each describe their unique perspective from industry, HTA and academia on ITC methods to develop comparative evidence in support of HTA submissions involving CAR-T therapies. Target stakeholders include HTA agencies, patient advocacy organizations, and pharma/biotech manufacturers.