FDA drug approval policies draw criticism over rigor and rare disease access

3 articles · Updated · The Wall Street Journal · May 10

In letters published on 10 May, Arnold Ventures defended rigorous, transparent trials, while other writers urged a lower efficacy bar for rare-disease therapies and cited UniQure's Huntington's drug.
Critics said excessive caution can block potentially life-saving treatments, while supporters argued weak evidence may expose patients to harm, divert care and raise costs unless post-market studies confirm benefit.
The exchange reflects a wider dispute over how the FDA should balance safety, efficacy, flexibility and patient access, especially for small populations with few treatment options.

Sources

Is the FDA’s scientific 'gold standard' a safeguard for patients or a roadblock to life-saving cures?

With new flexible policies, why is the FDA demanding stricter trials for some breakthrough drugs?