FDA Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases (Catapult’s regulatory round-up – October 2024)

CBER will host a public patient listening meeting to better understand patient and carer perspectives on enrollment of rare disease patients into gene therapy clinical trials in the pre-symptomatic or early symptomatic stages of their disease. The aim of this meeting is to explore what patients, and caregivers of patients in the pre-symptomatic or early stages of disease take into consideration when deciding whether to enroll in a gene therapy clinical trial and potentially receive an invest...