Overview
- The MHRA launched proposals on November 2 to redesign oversight so rare-disease treatments can reach patients years earlier.
- The plan would accept real-world outcomes from NHS records as comparators, with computer-modeled digital twins replacing placebo groups in some trials.
- Interim trial results could justify earlier use, supported by a single authorization that spans clinical study and early-access stages to accelerate full approval.
- Leaders cite ethical concerns over placing children in placebo arms and stress that safeguards and standards will remain in place.
- About one in 17 people in Britain—roughly 3.5 million—live with one of some 7,000 rare diseases, and many currently face four to six years from diagnosis to treatment as industry talks continue after some firms pulled investment.