Spotlight Session - Cell & Gene Therapy

(B41) A Framework for Considering Regulatory Likelihood of Acceptance of a Single-Arm Trial

(B42) A retrospective cohort study of Gaucher Disease Type 1 to describe sub-optimal responders to standard of care treatments in the United States

(B43) A systematic review of the efficacy of sirolimus and methotrexate after hematopoietic stem cell transplantation

(B44) Assessing Risk of Bias in Comparative Effectiveness Studies of Chimeric Antigen Receptor T-Cell Therapies for Relapsed or Refractory Large B-Cell Lymphoma

(B45) Feasibility of Assessing Comparative Long-Term Safety for Gene Therapy Products by Creating an External Control Arm using Real-World Data Sources

(B46) Maximising the use of real-world data from multiple sources to generate comprehensive evidence: insights from retrospective cohorts of allogeneic hematopoietic cell transplant recipients with adenovirus infection