Opinion: Novartis violated FDA’s sacred principle: In God we trust, all others must bring data

A common saying at the Food and Drug Administration is: “In God we trust, all others must bring data.” The independent evaluation of science is an essential element of the FDA’s dual role of protecting the public health and promoting innovation to bring new therapies into practice. In fact, with certain exceptions, the FDA is the only regulatory agency for drugs and devices that independently evaluates both the science and the source data from relevant studies. In many ways, the FDA serves the world as an arbiter of quality in the global development of medicines.

The recent flurry of news about the in the regulatory submission for Zolgensma (onasemnogene abeparvovec-xioi), a novel gene therapy developed by the Novartis subsidiary AveXis for the treatment of , should remind us all of the FDA’s central role as we grapple with the critical issue of scientific integrity. I have no specific knowledge about this issue other than what I’ve gleaned from the news. But based on those reports it seems that Novartis discovered an issue relating to data integrity while preparing its Biologics and , Novartis followed its usual internal procedure and the FDA is confident that the data integrity issues did not concern clinical trials .