Right to Try vs Expanded Access: What Each Law Lets Patients Do

Right to Try is a 2018 federal law that lets some terminally ill patients get an unapproved drug directly from its maker without asking the FDA for permission. Expanded access, sometimes called compassionate use, is the older FDA-run route in which the agency reviews the request. Both depend on the manufacturer agreeing to supply the drug, and neither guarantees treatment. This article covers US federal law and was checked on October 10, 2026.

The difference in one table

What Right to Try requires

The statute defines an eligible patient as someone with a life-threatening disease or condition who has exhausted approved treatment options and cannot join a clinical trial of the drug. A physician in good standing must certify that, and the physician may not be directly paid by the manufacturer. The patient, or a legally authorized representative, must give written informed consent.

The drug side is just as specific. An eligible investigational drug must have finished a Phase 1 trial as defined at 21 CFR 312.21, must not be approved for any use, and must either have a pending marketing application or an active investigational new drug application for a trial meant to support approval. Development or production must be ongoing, and the drug cannot be discontinued or on clinical hold.

FDA’s own Right to Try page states that the agency does not review or approve individual requests, and that the company decides whether to make its product available. The statute adds that nobody incurs liability merely for declining to provide access.

What expanded access involves

FDA describes expanded access as a route for patients with a serious or immediately life-threatening disease to receive an investigational drug, biologic or device outside a trial. FDA says it may be appropriate when all of these hold: the patient has a serious or immediately life-threatening condition, no comparable or satisfactory alternative exists, trial enrollment is not possible, the potential benefit outweighs the risks, and providing the product will not interfere with trials that could support approval.

There are three types: individual patients (including emergency use), intermediate-size groups, and widespread treatment use. For an individual patient under 21 CFR 312.310, the physician must judge that the drug’s probable risk is no greater than the risk of the disease, and FDA must determine the patient cannot obtain the drug under another IND or protocol. Treatment is generally one course for a set duration unless FDA allows more, with a written summary of results at the end.

A physician who will be the sponsor submits the request to FDA, usually on Form FDA 3926 for drugs and biologics, and files the protocol with an institutional review board that complies with 21 CFR part 56. The Reagan-Udall Foundation runs an eRequest site for preparing and submitting these forms electronically.

Emergencies

If a patient must be treated before paperwork can be finished, FDA may authorize use by telephone. The physician or sponsor then agrees to submit the written request within 15 working days of authorization. FDA lists an after-hours emergency line for all product types, 866-300-4374, and separate business-hours numbers for drugs, devices and biologics.

Where the two overlap, and where they do not

Practical experience of the two routes is usually closer than the law suggests, because the manufacturer is the gatekeeper in both. A company that does not want to supply an unapproved drug is not required to under either path. The differences lie in oversight and paperwork.

• Oversight. Under Right to Try, FDA is not part of the individual decision, and FDA’s page says individual Right to Try requests need no IRB review or approval. Expanded access puts FDA, and typically an IRB, in the loop.
• Reporting. Right to Try sponsors send FDA an annual summary of doses supplied, patients treated, the target disease and serious adverse events, due by March 31, through the CDER NextGen Portal for drugs or by email for CBER-regulated products. FDA posts an annual summary report.
• Effect on approval. Under the Right to Try statute, FDA generally may not use those outcomes to delay or adversely affect approval, unless it finds them critical to assessing safety, with written notice and a public health justification, or the sponsor asks.
• Liability. Right to Try provides liability limits. Sponsors and manufacturers generally cannot be held liable for acts or omissions in compliance with the section, and prescribers get the same protection unless there is reckless or willful misconduct, gross negligence or an intentional tort under state law. The law does not affect private product liability, tort, consumer protection or warranty actions in general. I did not find equivalent language on FDA’s expanded access pages.

How a request usually moves, step by step

Expanded access, as FDA lays it out for physicians

1. The physician confirms there is no comparable alternative and that trial enrollment is not feasible.
2. The physician confirms the patient’s condition is serious or immediately life-threatening.
3. The physician gets the manufacturer’s agreement to supply the product. A manufacturer can allow FDA to cross-reference its IND through a letter of authorization.
4. If the physician will be the sponsor, the request goes to FDA, and the protocol goes to an IRB along with informed consent.
5. Treatment begins once regulatory and institutional requirements are met.

Right to Try, as the statute lays it out

1. A physician in good standing certifies that the patient meets the eligibility terms.
2. The patient or representative signs written informed consent.
3. The manufacturer decides whether to supply a drug that has completed Phase 1 and remains in active development.
4. The sponsor later reports the use in its annual summary to FDA.

Read side by side, the two lists show where FDA sits. In the first it is a reviewer at the front of the process. In the second it appears at the end, as a recipient of reports. That is the real structural difference, and everything else follows from it.

Which route fits which situation

This section describes how the rules line up, not a recommendation for any person.

1. The drug has not finished Phase 1. Right to Try is closed by its own definition. Expanded access may still be possible, since FDA looks at each request on its own facts.
2. The condition is serious but not life-threatening in the narrow sense. Expanded access covers serious conditions as well as immediately life-threatening ones. Right to Try refers to life-threatening disease or condition.
3. The patient wants to avoid an FDA and IRB process. Right to Try removes both for individual requests, though the company must still agree.
4. A trial is open and the patient qualifies. Both routes assume the patient cannot join a trial of the drug, so enrolling is the first question.
5. The drug is an approved product used for a new purpose. A Right to Try drug cannot be approved for any use. That limit can matter.

What neither law does

Neither one makes a company supply a drug, neither promises the drug will work, and neither requires insurers to pay. FDA notes that products under expanded access have not been found safe and effective for their intended use and may cause serious side effects. Costs of a drug supplied outside a trial, and who bears them, are not addressed in the sources I read. A patient or family weighing either route should ask the treating physician three plain questions: what is known about the drug’s safety so far, what the realistic goal of treatment is, and who handles care if a serious side effect appears. The legal pathway only opens the door; those answers decide whether walking through it makes sense.

Related questions

Does Right to Try replace expanded access?

No. FDA describes expanded access as a separate program active for more than 30 years, and the two coexist.

Can I sue if a company says no?

The statute says no liability arises from declining to provide access under Right to Try. I found nothing creating a right to compel supply under either route.

Do state laws matter?

State right-to-try laws exist, but I did not review them for this article, so check your state’s statute and speak to a lawyer if the details matter. This is general information, not legal or medical advice.

Sources

• U.S. Food and Drug Administration, Right to Try
• Cornell LII, 21 U.S.C. 360bbb-0a
• U.S. Food and Drug Administration, Expanded Access
• U.S. Food and Drug Administration, Expanded Access: Information for Physicians
• Cornell LII, 21 CFR 312.310
• Cornell LII, 21 CFR 312.21