Right to Try vs. FDA Expanded Access (Compassionate Use)

If you or a loved one has a serious illness and standard treatments have failed, there are two legal pathways to seek a drug that is not yet FDA-approved: Expanded Access (long known as compassionate use) and Right to Try. They sound similar and overlap a lot, but they work differently, and knowing which is which helps you and your doctor choose.

Expanded Access (compassionate use)

Expanded Access is the older, FDA-run program. Your physician applies to the FDA and an institutional review board (IRB) reviews the request. That may sound slow, but in reality the FDA authorizes the vast majority of requests, frequently within days, and emergency requests can be handled even faster. It is available for serious or immediately life-threatening conditions, and, importantly, a drug can be requested at any stage of development, not just after Phase 1. The FDA’s involvement also adds a layer of safety review.

Right to Try

Right to Try, created by the 2018 federal Act, lets a physician go straight to the manufacturer without FDA or IRB review. It is limited to life-threatening conditions and to drugs that have completed Phase 1 and remain in active development. Supporters say it cuts red tape; critics note it removes oversight that can protect vulnerable patients.

What they share

• Both are for patients who have exhausted approved options and generally cannot join a clinical trial.
• Both require the manufacturer to agree to provide the drug, neither can force a company to say yes.
• Neither requires an insurer to pay; manufacturers may charge direct costs.
• Both require a treating physician and the patient’s informed consent.

Key differences at a glance

• Who reviews it: Expanded Access goes through the FDA and an IRB; Right to Try goes directly to the manufacturer.
• Development stage: Expanded Access can apply at any stage; Right to Try requires Phase 1 to be complete.
• Who qualifies: Expanded Access covers serious or life-threatening illness; Right to Try is limited to life-threatening illness.
• Oversight: Expanded Access includes FDA safety review; Right to Try removes it.

How to actually pursue access

• Start with your specialist, who must be willing to request and supervise the treatment.
• Ask first whether a clinical trial is available, since that is usually the preferred route.
• Identify the drug and manufacturer and find out whether the company will provide it, this is often the real bottleneck.
• Work with your doctor to choose Expanded Access (often the default, with FDA help such as its oncology facilitation program) or Right to Try.

This is general information, not legal or medical advice. Talk to your treating physician about the best pathway for your situation.