Expanded Access ("Compassionate Use")
How U.S. expanded access ("compassionate use") and the Right to Try Act work for people with serious illness, who must agree, what it costs, and why it is not a purchase or an approval.
Content last updated . This page has not been medically reviewed.
Expanded access, sometimes called "compassionate use," is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product. [1] It is not approval, not a clinic service you can buy, and not a promise of benefit. This page describes United States rules, with one note on Europe. Other countries differ. It is not medical or legal advice.
When the U.S. FDA says it may be appropriate
The FDA lists conditions that should all apply, including: [1]- The patient has a serious or immediately life-threatening disease or condition.
- There is no comparable or satisfactory alternative therapy.
- Enrolling in a clinical trial is not possible. [1]
- The potential benefit justifies the potential risks, and providing the product will not interfere with trials that could support approval. [1]
For drugs the federal regulation says the same: no comparable or satisfactory alternative therapy, and potential patient benefit that justifies the potential risks. [2] The FDA also warns that the investigational product may cause unexpected serious side effects. [1] Whenever possible, an investigational product should be used as part of a clinical trial. [3]
Who has to agree
Even if you meet the criteria, the licensed physician, the Institutional Review Board (IRB) and the company all need to agree that expanded access is appropriate for you. [3]| Who | Role |
|---|---|
| You | Talk with a licensed physician about approved options and clinical trials |
| Licensed physician | Oversees treatment, requests the product, files paperwork, reports |
| Company | Chooses whether to supply the investigational product |
| IRB | Reviews the plan and the informed consent |
| FDA | Reviews the request and decides whether it may proceed |
(Roles as described on the FDA patient page. [3]) The company is not required to say yes: there is no law or policy requiring a company to provide access outside a clinical trial. [3] The FDA says that, on average, the vast majority of expanded access requests it reviews may proceed. [3] That figure does not tell you how often companies or physicians agree in the first place.
Informed consent and leaving
Your physician must obtain your informed consent, or that of your legal representative. [3] You may stop at any time, even after signing. [3] For more, see Informed Consent in Emerging Treatments.Costs
Costs of the product and related medical services are usually not covered by private insurance or Medicare. [3] A company may give the product free; if it charges, the cost must be justified and limited to recovering the costs of manufacture, research, development and handling. [3] The FDA also notes that access can end: it cannot compel a sponsor to keep providing an investigational drug. [3]The Right to Try Act is a different route
The Right to Try Act allows eligible patients to seek certain investigational drugs from manufacturers, with the FDA not reviewing individual requests. An eligible patient has been diagnosed with a life-threatening disease or condition, has exhausted approved options, and cannot take part in a clinical trial; they also must give written informed consent. [4] An eligible drug must have completed a Phase 1 trial and must not be approved for any use. [4] The FDA says it does not review or approve requests under this Act. [4] The Act does not require a sponsor to provide the drug. [4]Europe
The European Medicines Agency describes compassionate use programs as put in place only if the medicine is expected to help patients with life-threatening, long-lasting or seriously debilitating illnesses. [5] It says that, in general, unauthorised medicines are first made available through clinical trials. [5] Access is arranged through national authorities, so ask your own doctor how it works where you live.For physicians
The FDA lists contact routes for emergency requests, including its Emergency Call Center at 866-300-4374 after hours and on weekends. [1] This number is for physicians' requests, not for patients in an emergency.What expanded access is not
It is not a way to obtain a product from a clinic that sells it, and it does not make a product approved. For products offered commercially, read Unapproved Commercial Interventions. To understand trials, read Clinical Trials Explained for Participants, and for status words, Established vs Investigational Use.Frequently asked questions
Is expanded access the same as "compassionate use"?
In the FDA's wording, expanded access is sometimes called "compassionate use." [1]
Can I request an investigational drug myself?
No. A licensed physician requests it for you; the company must be willing to supply it, and the IRB and FDA are involved. [3]
Will insurance pay?
Costs are usually not covered by private insurance or Medicare, according to the FDA. [3]
Does it mean the product works?
No. The FDA says the product may or may not be effective and may cause unexpected serious side effects. [1]
Is Right to Try approved by the FDA?
The FDA says it does not review or approve Right to Try requests. [4]
References
- U.S. FDA, Expanded Access (page current as of 10/22/2026). Read 2026-10-22.
- Electronic Code of Federal Regulations, 21 CFR 312.305, Requirements for all expanded access uses. Read 2026-10-22.
- U.S. FDA, Expanded Access: Information for Patients (page current as of 10/22/2026). Read 2026-10-22.
- U.S. FDA, Right to Try (page current as of 12/12/2024). Read 2026-10-22.
- European Medicines Agency (EMA), Compassionate use. Read 2026-10-22.