The Ethics & Science of Controls in GT Studies: Placebos, Shams, Natural History & Delayed Crossover

While ethical dilemmas regarding the selection of control groups can arise in various clinical trials, they become especially prominent in early-phase pediatric GT trials. Decisions regarding the control group in pediatric GT trials are a critical aspect of trial design where ethical and scientific issues are closely intertwined. Due to the vulnerability of children and their parents, when investigating therapies for rare, life-threatening diseases affecting minors, the scientific and ethical considerations surrounding the choice of suitable controls become notably significant. Panelists will discuss the ethics of using various trial designs in the GT space, and what advocates ought to know.
Moderator:
Emma James, PhD; VP Clinical and Medical Affairs; Head of Medical Office, Encoded Therapeutics
Panelists:
Peter Marks, MD, PhD; Director, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration (FDA)
Allyson Berent, DVM, Dipl. ACVIM; CSO, Foundation for Angelman Syndrome Therapeutics; Co-Director, Angelman Syndrome Biomarker and Outcome Measure Consortium
Suyash Prasad, MBBS, MSc, MRCPCH, FFPM, FRCP, GFMD; Principal/Chief Medical Officer, Suyash Prasad Consulting LLC