Investigational drugs
Can a patient just ask for 'compassionate use' access to an investigational compound?
Plain English first, then the primary source. Education, not legal advice.
Interpretation
In short: expanded access is a narrow, supervised route through a physician and FDA. It is not a self-service option.
Plain English
Expanded access, sometimes called compassionate use, is a regulated pathway that allows patients with serious or immediately life-threatening conditions to access an investigational drug outside a clinical trial when no comparable alternative exists. It is described by FDA and governed by rules within 21 CFR Part 312.
It is not an informal arrangement. It requires a licensed physician, an FDA submission, and generally the cooperation of the drug's sponsor, who must be willing to provide the product.
It exists for a narrow category of situations and is not a general mechanism for obtaining research chemicals for personal or elective use.
Why it matters
- This addresses a common misconception that 'compassionate use' is an easy or informal route around the approval process.
Where the answer becomes fact-specific
The Foundation does not manufacture legal conclusions. These points change the analysis and may require qualified regulatory counsel.
- Availability depends on the severity of the condition, existence of alternatives, and whether a sponsor will provide the drug.
View the source
- FDA, Expanded Access
- 21 CFR Part 312, Subpart I, expanded access, 21 CFR Part 312