23 February 2026 - The U.S. FDA today issued draft guidance for sponsors seeking approval for targeted individualised therapies by generating substantial evidence of effectiveness and safety when randomised controlled trials are not feasible due to small patient populations.

The draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, specifically discusses genome editing and RNA-based therapies such as antisense oligonucleotides but leaves open the potential that this framework may apply to additional tailored therapeutics provided they directly address the underlying specific cause of the disease.

Read FDA press release