Every year, some patients learn that the one drug that might help them exists — but it is still in trials, and they don't qualify for any of them. Maybe they're too sick to meet the eligibility criteria. Maybe the nearest open site is a thousand miles away. Maybe enrollment closed last month. Expanded access, still widely called "compassionate use," is the regulatory pathway built for exactly this gap: a way for a patient with a serious or life-threatening condition to receive an investigational drug outside of a formal study. It is not a long shot reserved for the famous or the well-connected. The FDA authorizes the overwhelming majority of the requests it receives. The harder part is almost never the FDA — it's finding a company willing to provide the drug and a physician willing to run the request.
This article is for informational purposes only and does not constitute medical advice. Expanded access eligibility, drug availability, and costs vary case by case. Always consult a qualified healthcare professional about your specific situation.
Summary
Expanded access is an FDA-regulated pathway (21 CFR Part 312, Subpart I) that allows a patient with a serious or immediately life-threatening condition, no comparable therapeutic options, and no ability to enroll in a trial to receive an investigational drug. The FDA authorizes well over 99% of requests it receives, and most non-emergency requests are processed in days — emergency requests can be granted by phone in hours. The real gatekeepers are the drug manufacturer, which is never obligated to provide the drug, and the treating physician, who must agree to supervise treatment and file the request. Understanding who does what in this three-party process is what makes the difference between a request that moves and one that stalls.
ClinicalMetric Analysis
- The FDA is not the bottleneck, and treating it as one wastes time. In its published expanded access data, the FDA allows roughly 99% of the individual-patient requests it receives, and its review of a single-patient IND is often completed in a few days. Families who spend their energy lobbying the FDA are usually aiming at the wrong target. The two decisions that actually determine whether a patient gets the drug are made earlier: whether the company will release it, and whether a physician will take on the request. Direct that energy at the manufacturer's medical affairs team and at your own care team.
- A company's refusal is usually about trial integrity or supply, not indifference. Manufacturers frequently decline expanded access during pivotal trials because a serious adverse event in a compassionate-use patient — often someone sicker than any trial participant — still has to be reported and can complicate the drug's safety record and its path to approval. Limited manufacturing capacity for a drug not yet at commercial scale is the other common reason. Understanding this reframes the conversation: asking "can you provide this under a protocol that protects your trial data?" lands very differently from "why are you denying a dying patient."
- Right to Try and expanded access are not competing options — expanded access is almost always the stronger route. The 2018 federal Right to Try Act created a pathway that bypasses the FDA and the IRB, but it did not create any obligation for a company to provide a drug, which was never the real barrier. What it removed was FDA oversight and independent ethics review — protections that exist for the patient's benefit. In practice, most academic centers and most manufacturers still route requests through expanded access, because the FDA's involvement adds a safety check and, through the "Project Facilitate" program, actual help navigating the process.
What Expanded Access Actually Is
Expanded access is the use of an investigational medical product — a drug, biologic, or device that has not yet received full FDA approval — outside of a clinical trial, to treat a patient rather than to study one. The term "compassionate use" is the older, informal name; the FDA's regulatory term is "expanded access," and the two mean the same thing in ordinary conversation.
The logic is straightforward. Clinical trials are designed to answer scientific questions, and to do that they impose strict eligibility rules, fixed enrollment windows, and a limited number of sites. Those constraints inevitably exclude patients who could still benefit from the drug but don't fit the study. Expanded access exists so that "you don't fit our trial" doesn't have to mean "you can't have the drug at all."
There are three categories, scaled to the number of patients involved: individual patient expanded access (including an emergency version for situations that can't wait for written authorization); intermediate-size population access, for a group of patients who share a condition; and treatment IND or treatment protocol, for widespread access to a drug that is far along in development, often while it awaits final approval. The overwhelming majority of requests are for a single, named patient.
Who Qualifies
The FDA's criteria are consistent across the categories, and all of them must be met:
- Serious or immediately life-threatening condition. The bar is a serious disease — not necessarily terminal, but serious enough that the risk of an unapproved drug is justifiable.
- No comparable or satisfactory alternative. The patient has exhausted, cannot tolerate, or is not a candidate for approved therapies that would reasonably address the condition.
- Enrollment in a clinical trial is not possible. There is no trial the patient can join — because none is recruiting, none is geographically reachable, or the patient doesn't meet trial eligibility.
- Potential benefit justifies the risk. The treating physician judges that the probable benefit outweighs the potential risks, given everything known about the drug so far.
- Providing the drug won't interfere with trials that could support approval. This is the criterion companies weigh most heavily — access can't compromise the studies needed to get the drug approved for everyone.
Notice what's not on this list: being famous, being wealthy, or having a personal connection at the company. The criteria are clinical. Where money and connections have historically appeared to matter, it's almost always at the manufacturer's discretion stage — not the FDA's — which is precisely why the FDA now requires large drugmakers to post their expanded access policies publicly.
How the Request Actually Works: A Three-Party Process
The single most useful thing to understand is that three parties must all say yes, and they say it in a specific order.
Step one: the physician
Expanded access is always physician-initiated. A patient cannot file the request themselves. Your treating physician — usually a specialist already managing your condition — agrees to supervise the treatment, monitor for adverse events, and submit the paperwork. If your current physician is unfamiliar with the process, that is not a dead end: many academic medical centers have research pharmacies and regulatory staff who do this routinely and can guide a community physician through it.
Step two: the manufacturer
The physician (or the patient's advocate) contacts the company that makes the drug — specifically its medical affairs or expanded access team — to ask whether it will provide the product for this patient. This is the step with no guarantees. A company is never legally required to say yes, and its reasons for declining can be legitimate: an ongoing pivotal trial it doesn't want to jeopardize, limited drug supply, or a formal decision not to run any expanded access program. Large manufacturers are now required to make their expanded access policy and a contact point publicly available, which makes this step easier to start.
Step three: the FDA and an IRB
Once the company agrees to supply the drug, the physician submits the request to the FDA — typically as an individual-patient investigational new drug application (a "single-patient IND") using Form FDA 3926, a form the agency deliberately simplified so a physician can complete it in well under an hour. For non-emergency requests, treatment can generally begin after FDA authorization and review by an Institutional Review Board (IRB); the FDA permits a single IRB member's concurrence for individual-patient requests rather than a full board meeting, which removes weeks of delay. For genuine emergencies, an FDA official can authorize treatment over the phone, with the paperwork following afterward.
What It Costs
Expanded access is not automatically free, and this surprises many families. Under FDA rules, a manufacturer is permitted to charge for an investigational drug provided through expanded access — but only to recover the direct costs of making it available (manufacturing, shipping, and monitoring). Companies cannot profit from expanded access, and in practice many provide the drug at no charge. What you should not assume is that "investigational" means "complimentary."
Separately, the routine medical care that surrounds the treatment — office visits, infusions, lab tests, imaging, hospitalization for side effects — is billed the way any other care is, and coverage varies. Health insurers are generally not required to cover an investigational drug obtained through expanded access, and coverage of the associated care depends on the plan. This is a critical conversation to have with both the manufacturer and your insurer before treatment begins, not after the bills arrive. The FDA's Reagan-Udall Foundation and the manufacturer's patient-assistance staff can often help clarify what will and won't be covered.
Expanded Access vs. Right to Try
Since 2018, the federal Right to Try Act has offered a second pathway to investigational drugs, and the two are frequently confused. The practical differences matter:
- FDA involvement. Expanded access is reviewed by the FDA; Right to Try bypasses the agency entirely. That FDA review is a safety check, and through the "Project Facilitate" call center the agency actively helps physicians assemble requests.
- Ethics review. Expanded access requires IRB concurrence; Right to Try does not require IRB review. Independent ethics oversight exists to protect the patient.
- Eligibility. Right to Try is limited to life-threatening conditions and to drugs that have completed Phase 1 and remain in active development. Expanded access covers a broader range of serious conditions and stages.
- The one thing both share. Neither pathway compels a manufacturer to provide the drug. Right to Try removed regulatory steps, but it did not remove the barrier that actually stops most requests — a company's willingness to supply.
Because Right to Try strips out protections without solving the real bottleneck, most academic centers still prefer expanded access. It is worth asking your care team which route they use and why.
Practical Steps If You're Considering This
- Confirm a trial truly isn't an option first. A clinical trial gives you the drug plus structured safety monitoring, and often at no cost. Search recruiting studies thoroughly before treating expanded access as your only path.
- Identify the exact drug and its manufacturer. Expanded access is drug-specific. Know the investigational product's name and the company developing it before you make contact.
- Ask the FDA's Project Facilitate for help. This is a dedicated FDA Oncology Center of Excellence call center that walks physicians and patients through single-patient expanded access, including identifying the right manufacturer contact.
- Bring your physician in early. Because the request must be physician-initiated, your specialist's willingness is foundational. If they're unfamiliar with the process, ask whether a nearby academic center's research office can support the filing.
- Settle cost and coverage before starting. Ask the manufacturer whether it charges, and ask your insurer what surrounding care it will cover. Get the answers in writing.
Key Takeaways
- Expanded access ("compassionate use") lets a seriously ill patient receive an investigational drug outside a trial when no trial or approved therapy is available — and the FDA authorizes the vast majority of requests.
- The FDA is rarely the obstacle. The two decisions that determine access are the manufacturer's willingness to supply the drug and the treating physician's willingness to file and supervise.
- The request is a three-party process in order: physician agrees, manufacturer agrees, then FDA and an IRB authorize. Emergency requests can be granted by phone within hours.
- Expanded access is not automatically free — manufacturers may charge cost-recovery, and insurance coverage of the drug and surrounding care varies. Confirm costs before treatment.
- Right to Try bypasses the FDA and IRB but doesn't obligate companies to provide drugs either — for most patients, expanded access remains the safer and better-supported route.