CHF Accelerated Approval !, page-87

I also think we fall under this particular situation warranting "one trial with confirmatory evidence"

"One adequate and well-controlled clinical investigation with compelling results, supported by additional data from the natural history of the disease

In certain circumstances, FDA accepts one adequate and well-controlled clinical investigation that has generated compelling results as the basis to demonstrate effectiveness, when the single trial is supported by additional data from the natural history of the disease that reinforce the very persuasive finding. For example, a single trial showing marked improvement in survival compared to a control group, either external to the trial or concurrent, could be supported by data from separate sources (e.g., a natural history study, case report forms, or registries) that demonstrate a very limited median survival time or other clinically highly important outcome without treatment. In this case, the natural history data would represent confirmatory evidence."

(See page 12 of the draft guidance)