Expanded Access Programme

Expanded access programme permits individual patients with serious or life-threatening diseases access to investigational pharmaceuticals before regulatory approval when no adequate alternatives exist. Named-patient use programmes provide access on case-by-case basis. Cohort expanded access programmes treat larger patient groups under single protocols. Treatment investigational new drug applications enable commercial-scale expanded access.

Physician-directed patient requests for experimental drugs raise ethical and legal questions balancing autonomy against safety assurance. Regulatory requirements vary internationally with European Union permitting broader access than United States. Data collection from expanded access programmes informs regulatory submissions providing preliminary safety evidence. Reimbursement for expanded access drugs remains complex with insurance coverage varying. Transition of successful expanded access cases to formal registration expedites market access.