Comment from Robert Hebron
AnonymousSupportIndividual
Summary: The commenter suggests that the guidance should provide more specific criteria for determining when a control arm can be depowered, particularly regarding changes in the standard of care. They also recommend including language on how master protocols can support combined Phase 1, 2, and 3 trials for genetic-based treatments to encourage smaller companies to develop gene therapies.
I would offer two comments: (can support 1) the guidance should be more specific on how to judge whether a control is concurrent - it should depend on the disease and when there is a significant change in current standard of care that affects the primary endpoint. The latter should be required before one depowers the control arm. (2) mention should be made about how a master protocol can support genetic based treatments where a Phase 1, 2 and 3 set of trials may be combined (See: the Ionis Pharma FUS trial in ALS) This wouild encoiurage smaller companies to take on the development of gene therapies.