Expanded Access

Key facts

Regulation

21 CFR Part 312, Subpart I

Categories

Individual patients, intermediate-size populations, widespread treatment use

Individual patient request form

Form FDA 3926

Charging for the drug

Requires prior FDA authorization under 21 CFR 312.8

Separate federal pathway

Right to Try Act, signed in 2018

What is expanded access?

Expanded access allows use of an investigational medical product for treatment rather than research. It is meant for patients who cannot enroll in a clinical trial, for example because they do not meet the eligibility criteria or no trial is within reach, and whose physician believes the potential benefit justifies the risk.

Access requires a manufacturer willing to supply the product, and FDA cannot compel a company to provide it. Under the 21st Century Cures Act, manufacturers of investigational drugs for serious diseases must make their expanded access policies publicly available.

How expanded access works

The general requirements in 21 CFR Part 312, Subpart I apply across all three categories of expanded access:

• The patient has a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy.
• The potential benefit justifies the potential risks, and those risks are not unreasonable in the context of the disease.
• Providing the product will not interfere with clinical investigations that could support its approval.
• An Institutional Review Board (IRB) reviews the use and informed consent is obtained; in an emergency, treatment can begin before IRB review.

Expanded access vs Right to Try

The federal Right to Try Act, signed in 2018, created a second route for patients with life-threatening conditions who have exhausted approved options and cannot join a trial. It applies to investigational drugs that have completed a Phase 1 trial and remain in active development, and requests go to the manufacturer without FDA review of each request.

Many states passed their own right-to-try laws before the federal act. Neither pathway obligates a company to supply its product.

Why expanded access matters

Expanded access programs show that patient demand exists before approval, and ClinicalTrials.gov carries expanded access records as their own study type, so analysts can track which investigational products are available outside trials. For manufacturers, program design affects drug supply, safety reporting obligations and relationships with patient advocacy groups.