FDA Lays Out Proposed Ground Rules for Natural History Studies

March 29, 2019

Drug sponsors who want to use natural histories to help develop treatments for rare diseases should draw up well-defined statistical analysis plans and probably even hire statisticians to help develop the histories, the FDA said in new draft guidance.

Natural histories are observational studies that track the course of diseases and the FDA has promised to make more room for them in the hopes of speeding up drug development.

“Because the natural history of many rare diseases remains relatively unknown, investing in rigorous models can help advance the development of treatments for these conditions,” outgoing Commissioner Scott Gottlieb said in announcing the draft’s release. “Information obtained from a natural history study can play an important role at every stage of drug development, from drug discovery to the design of clinical studies intended to support a drug's marketing approval.”

Regulators think that, done properly, natural histories can help drugmakers zero in on patient populations, figure out ways to measure clinical outcomes, identify or develop biomarkers and even design externally controlled studies.

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