FDA Aims to Ease Single-Patient Trials for Rare Disease Drugs (1)

More clinical trials for patients battling life-threatening genetic disorders could open up under an FDA proposal for developing drugs targeting the exact DNA misspellings causing the disease.

The Food and Drug Administration issued draft guidance Monday to help researchers design and pursue a type of “N of 1" clinical trial, in which a single patient is the entire study population. The guidance outlines how and when to talk to FDA staff and keep information confidential. It also discusses ethical reviews and consent requirements.

N of 1 studies come with a unique set of challenges. A single academic researcher, who hasn’t ...