The UK’s MHRA proposed pairing trial authorization with market access for very rare diseases, aiming to create a flexible approval pathway while acknowledging that reimbursement remains unresolved. The regulator’s move is designed to reduce delays from development into patient availability. The proposal centers on streamlining governance around when trials can start and how market access can follow, a persistent friction point for ultra-rare drug development. For developers, the change could tighten timelines and improve planning—if payers and reimbursement policy align with authorization decisions.