Submission & ApprovalLast reviewed May 2026

Expanded Access(Expanded Access)

The pathway for patients with serious or immediately life-threatening diseases to access investigational medical products outside of clinical trials.

Usage Examples

The sponsor submitted an Individual Patient IND for expanded access to the Phase 2 investigational drug.
IRB approval and informed consent were completed before dispensing under expanded access.
Serious adverse events under the Treatment IND were reported to FDA per 21 CFR 312.32.

What is Expanded Access?

Expanded access — also called compassionate use — is a regulatory pathway allowing patients with serious or immediately life-threatening diseases to access investigational drugs, biologics, or devices outside of clinical trials when no satisfactory alternative therapy exists. FDA, sponsors, and physicians work together to authorize expanded access under 21 CFR 312 (for drugs and biologics) or 21 CFR 812 (for devices).

Three categories exist: Individual Patient IND (single patient with immediate need), Intermediate-Size Patient Population IND (small defined patient group), and Treatment IND (large patient population in late development). Each has specific eligibility, protocol, and reporting requirements. The 21st Century Cures Act (2016) and the Right to Try Act (2018) created additional pathways for certain investigational products, though expanded access remains the primary FDA mechanism.

Expanded access is sponsor-initiated and sponsor-supplied: the sponsor must agree to provide the investigational product. Costs can be recovered for manufacturing and direct costs. Data collected during expanded access use is not intended to support approval, but sponsors must report serious adverse events and safety findings to FDA. Physician and institutional requirements include IRB approval and informed consent.

Regulatory Context

This term appears most often in submission & approval workflows where submission quality, regulatory evidence, and audit readiness depend on consistent language. It is commonly referenced alongside 21 CFR 312 305, 21 CFR 812 36, RIGHT TO TRY ACT.

FDAICHEMA

When This Matters

The sponsor submitted an Individual Patient IND for expanded access to the Phase 2 investigational drug.
IRB approval and informed consent were completed before dispensing under expanded access.
Serious adverse events under the Treatment IND were reported to FDA per 21 CFR 312.32.

Common Mistakes

Treating submission readiness as a formatting-only check without lifecycle validation.
Using outdated guidance references across modules and summaries.
Missing cross-functional review between RA, CMC, and quality before submission.

Related Regulations

21 CFR 312 30521 CFR 812 36RIGHT TO TRY ACT

Frequently Asked Questions

Expanded access requires FDA authorization and applies to investigational products with at least some clinical evidence. Right to Try (2018 federal law) allows physicians to access investigational drugs without FDA involvement, but requires the drug to have completed Phase 1 and the sponsor must agree to provide it. Both pathways coexist; expanded access remains more commonly used.

No. Expanded access requires sponsor agreement to provide the product. Sponsors evaluate based on supply availability, clinical trial impact, and program considerations. Some sponsors operate formal expanded access programs; others handle requests case-by-case.

Generally not. Expanded access is not designed as a controlled study — it treats patients outside the trial setting without randomization or standardized endpoints. Safety information collected is reported to FDA. Rare exceptions occur when expanded access data is integrated into approval packages, but this is not the default design intent.