PBI38 CHARACTERIZING THE USE OF CLINICAL OUTCOME ASSESSMENTS IN APPROVED GENE THERAPY TREATMENTS.

Gene therapy has the potential to transform patients' lives and appropriate assessment methods are needed to capture the benefits of this type of treatment. The objective of this research was to characterize the current use of clinical outcome assessments (COAs) for U.S. Food and Drug Administration (FDA)- and European Medicines Agency (EMA)-approved gene therapies to describe ways in which the gene therapy patient experience is captured. [Extracted from the article]