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 2023, the FDA authorized over 99% of expanded access requests it received.
Individual patient access (emergency IND) is for a single patient with an immediately life-threatening condition. 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 days afterward.
Intermediate-size expanded access is for a smaller group of patients (typically fewer than several hundred) with a serious condition who don't qualify for existing trials. The drug company sponsors a treatment protocol, an IRB reviews it, and the FDA evaluates safety data before authorizing access.
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 requires three parties to agree: your physician, the drug manufacturer, and the FDA. 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 provides regulatory oversight, liability protections, and adverse event monitoring that Right to Try does not. 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 authorizes over 99% of the expanded access requests it receives. In emergency cases the FDA can authorize treatment by phone within hours, with paperwork submitted within 15 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 (emergency IND) covers a single patient with an immediately life-threatening condition using the simplified two-page 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 terminally ill patients request investigational drugs that have completed Phase 1 testing directly from manufacturers, bypassing the FDA. In practice most patients and physicians still use the FDA's expanded access program because it provides regulatory oversight, liability protections, and adverse event monitoring that Right to Try does not. Right to Try has been used in fewer than 100 documented cases since enactment.
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.