How to access investigational drugs outside of clinical trials when approved treatments have been exhausted.
Sometimes a promising treatment exists but you can't access it through a clinical trial. Maybe you don't meet the eligibility criteria. Maybe the trial isn't recruiting near you. Maybe the trial has closed enrollment. For patients with serious or life-threatening conditions who have exhausted approved treatments, compassionate use (also called expanded access) provides a legal pathway to receive investigational drugs outside of a clinical trial.
This is not a loophole or an unusual request. The FDA approves the vast majority of compassionate use applications, often within hours for emergency cases. In fiscal year 2023, the FDA allowed about 98% of the expanded access requests for drugs and biologics it received to proceed.
Individual patient access is for a single patient with a serious or immediately life-threatening condition. It can be an emergency or non-emergency request, and most requests are non-emergency. Your physician contacts the drug manufacturer and files FDA Form 3926, a simplified two-page application. In emergencies, the FDA can authorize treatment by phone within hours, with paperwork submitted within 15 working days afterward.
Intermediate-size expanded access is for a group of patients smaller than a treatment IND would serve, such as patients who cannot join an ongoing trial or who have a disease too rare for a trial. The sponsor submits a protocol, an IRB reviews it, and the FDA must find enough safety evidence and at least early signs that the drug may work.
Treatment IND/protocol is for widespread access, typically when a drug is close to FDA approval and there is sufficient safety and efficacy data. This pathway can serve larger patient populations and is often established by the manufacturer during the period between completing pivotal trials and receiving formal FDA approval.
The process needs agreement from your physician, the drug manufacturer, and the FDA, and an institutional review board (IRB) must also review the request. Your doctor initiates the request by contacting the manufacturer's medical affairs team. The manufacturer decides whether to provide the drug (they are not required to). If the manufacturer agrees, your physician submits FDA Form 3926 along with relevant clinical information.
If the manufacturer declines, ask them to explain why in writing. Some manufacturers have formal compassionate use policies published on their websites. Others delegate expanded access decisions to independent review committees. The Reagan-Udall Foundation maintains a publicly available list of manufacturer expanded access policies for investigational drugs.
Cost is a real consideration. The drug itself may be provided free by the manufacturer, but associated medical costs (administration, monitoring, lab work) may be billed to your insurance. Some manufacturers cover all costs; others cover only the drug. Clarify this before starting treatment.
The federal Right to Try Act (2018) created an alternative pathway that bypasses the FDA. Under Right to Try, patients with life-threatening conditions who have exhausted approved treatments can request investigational drugs directly from manufacturers if the drug has completed a Phase I trial. The key difference is that no FDA authorization is needed.
In practice, most patients and physicians still use the FDA's expanded access program rather than Right to Try, because the FDA process includes FDA review and IRB oversight, which Right to Try does not require. Right to Try does limit the legal liability of manufacturers and doctors, and manufacturers must report serious side effects to the FDA once a year. The FDA pathway also has a faster turnaround than many people realize.
Compassionate use (formally called expanded access) is a legal FDA pathway that lets patients with serious or life-threatening conditions receive investigational drugs outside of a clinical trial when approved treatments have been exhausted and trial enrollment is not possible.
The FDA allows the vast majority of expanded access requests to proceed, about 98% for drugs and biologics in fiscal year 2023. In emergency cases the FDA can authorize treatment by phone within hours, with paperwork submitted within 15 working days afterward. The most common barrier is not the FDA but the drug manufacturer, which must agree to provide the drug and is not required to.
Individual patient access covers a single patient with a serious or immediately life-threatening condition, in an emergency or not, using FDA Form 3926. Intermediate-size access covers smaller groups of patients who do not qualify for existing trials, under a sponsor treatment protocol reviewed by an IRB. Treatment protocol access serves larger populations, typically when a drug is close to FDA approval with substantial safety and efficacy data.
Your physician initiates the request by contacting the drug manufacturer's medical affairs team. If the manufacturer agrees to provide the drug, your physician submits FDA Form 3926 along with relevant clinical information. If the manufacturer declines, ask for the reason in writing; the Reagan-Udall Foundation maintains a public list of manufacturer expanded access policies for investigational drugs.
The federal Right to Try Act (2018) lets patients with a life-threatening condition, who have tried approved treatments and cannot join a trial, request investigational drugs that have completed Phase 1 testing directly from manufacturers, without FDA review. In practice most patients and physicians still use the FDA's expanded access program because it includes FDA review and IRB oversight, which Right to Try does not require. FDA's public summary shows only a few drugs supplied under Right to Try each year (6 in 2025).
The manufacturer often provides the drug itself free of charge, but associated medical costs such as administration, monitoring, and lab work may be billed to your insurance. Some manufacturers cover all costs and others cover only the drug, so clarify the cost arrangement before starting treatment.