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SILVER SPRING, Md. — Food and Drug Administration statisticians pondered changing how the agency uses statistics to approve drugs for rare disease at a public workshop Monday.

The challenge, regulators said, is that there are so few patients who suffer from certain rare diseases that it might not be possible or feasible to do many rounds of traditional clinical trials. So, regulators and drug developers are looking at ways to import data collected in early studies — which are used to initially assess drug safety and efficacy — into the Phase 3 studies that the FDA uses to determine whether drugs should make it onto the market.


“We really need to be able to squeeze every ounce of information from every single piece of data we have in this setting,” said Laura Lee Johnson, acting director of one of the FDA’s Center for Drug Evaluation and Research’s biometrics divisions.

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