FDA proposes rare disease treatment approval path

(CNN, KYMA) - The Food and Drug Administration (FDA) is proposing a new path to approve treatments for ultra-rare diseases.

It would provide a way to get individualized therapies and treatments approved.

The problem right now is it's very difficult to get therapies approved for conditions that affect a small population. That's because there aren't enough people to take part in the necessary trials.

HHS Secretary Robert F. Kennedy Jr. says the new guidance would cut red tape.

It specifically targets genome editing and RNA-based therapies, though it could also be used for other treatments in the future.

Draft guidance from the FDA typically requires a 60-day public comment policy before it is finalized.