“Our goal is to see how we can accelerate methods that improve our ability to use advanced tools to meet FDA’s gold standard for regulation.
These methods are being applied to both common and rare diseases. FDA is also collaborating with scientists to use similar computational tools to develop natural history models, based on placebo arms in Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, and muscular dystrophy. If we’re able to make better use of rigorous, reliable natural history models, especially for rare diseases, it can help make clinical trial recruitment more efficient.”]