Compassionate Use / Expanded Access
An FDA pathway allowing patients with serious or life-threatening conditions to access unapproved drugs outside of clinical trials when no comparable alternatives exist.
In Depth
Compassionate use (formally "expanded access") allows individual patients to receive investigational drugs before FDA approval. GW Pharmaceuticals provided Epidiolex to hundreds of pediatric epilepsy patients through expanded access before its 2018 approval. The data collected from these patients contributed to the safety database supporting approval. Compassionate use programs require physician and FDA approval and do not substitute for rigorous clinical trials.
Related Terms
More in Regulatory
Schedule I
The most restrictive classification under the US Controlled Substances Act (CSA), applied to substances deemed to have high abuse potential, no accepted medical use, and lack of accepted safety under medical supervision.
IND (Investigational New Drug)
An application submitted to the FDA allowing a pharmaceutical company or researcher to begin clinical trials of an unapproved drug in humans.
NCT Number
A unique identifier assigned by ClinicalTrials.gov to every registered clinical study. Format: NCT followed by 8 digits (e.g., NCT04849910).
GMP (Good Manufacturing Practice)
A system of regulations ensuring that products are consistently produced and controlled according to quality standards. Required for pharmaceutical-grade cannabinoid products.
DEA (Drug Enforcement Administration)
The US federal agency responsible for enforcing controlled substances laws. Administers the scheduling system under the Controlled Substances Act.