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WASHINGTON (AP) -- Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the pharmaceutical industry has long considered unprofitable.
The preliminary Food and Drug Administration guidelines, if implemented, would create a new pathway for bespoke therapies that have only been tested in a handful of patients due to the challenges of conducting larger studies. The FDA announcement specifically mentions gene editing, although agency officials said the new approach could also be used by other drugs and therapies.
It's a shift long sought by patients, advocates and researchers focused on rare diseases, which often do not fit within the pharmaceutical industry's business model or the FDA's traditional drug-approval system.
"It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases," FDA Commissioner Marty Makary said in a release.
The announcement comes a week after Makary said the FDA would drop its decades-old standard of requiring two clinical trials for standard drug reviews. That was the latest in a series of changes to FDA norms and standards, many which have not gone through federal procedures traditionally used to update agency rules.