A manuscript detailing this study is under revision and awaiting acceptance by the British Journal of Clinical Pharmacology.  The study was funded using an unrestricted grant from myTomorrows, a global company that connects patients to expanded access programs and helps drug companies collect data from these programs. Polak is a real-world data lead at myTomorrows.

One reason why gathering expanded access data may be the only route for drug approval is if conducting a clinical trial would be unethical, as was the case for uridine triacetate. “You can’t deliberately overdose a person and then try to undo the overdose,” said Dr Bateman-House. “It was only in expanded access that they could collect [these] data.”

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