Shifting clinical trial and value demonstration models for cell and gene therapies: present & future critical success factors

Background & Aim Clinical trial models have been changing over the past decade, influenced by emergence of innovative technologies, increasing focus on subgroups and niche populations, competition over patients, and need to capture transformative health outcomes. Approximately half of rare disease and oncology therapies today are launched from fast-track pathways with a single-arm study, creating challenges in shaping a comprehensive value proposition sufficient to meet the needs of clinicians, regulators and payers. In addition, uncertainties remain around demonstration of transformative magnitude, duration and patient-centric effects, which require strong data-analytic and real-world evidence development strategies. This session will discuss how the evolution of clinical trial models is shifting us towards requirements to consider a comprehensive approach to value demonstration that meets provider, regulator, payer decision needs. Methods, Results & Conclusion Mr. Faulkner will open the session by reviewing key clinical design issues in cell and gene therapy trials and common gaps that must be filled outside the trial, including leveraging outcomes research and real-world evidence solutions, highlighting recent global research on cell and gene therapy regulatory and HTA decisions in North America and the EU. Dr. Theocharous will consider key clinical trial design and execution best practices from a manufacturer perspective, drawing from common development challenges, and Dr. Koh will compliment this with clinical trial and patient treatment considerations from the perspective of a practicing physician executing cell and gene therapies in the field. Mr. Morgese will discuss recent US and EU policy issues that flow from cell and gene therapy evidence issues and highlight steps that the cell and gene therapy community are taking to address. The panel will debate the top critical success factors necessary for linking clinical trial designs to other gap-fill strategies to meet downstream approval, access and uptake requirements.