FDA presents new rare disease drug approval pathways

The U.S. Food and Drug Administration has introduced a new method for the approval of personalized therapies in patients with rare genetic diseases that cause severe disability or death.

The “plausible mechanism pathway” will allow drug manufacturers to use positive findings — including biological plausibility and clinical improvement — from smaller studies rather than randomized trials, according to a news report from Reuters. After showing benefit in several consecutive patients, the drugs would then be eligible for marketing authorization. Drug manufacturers would then be responsible for demonstrating real-world efficacy and safety.

Read more: Reuters

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