Expanded access is a pathway that allows patients with serious or life-threatening conditions to use investigational drugs or medical devices that haven’t yet been approved by the FDA. Sometimes called “compassionate use,” it exists for people who have no other treatment options and can’t enroll in a clinical trial. The FDA authorizes the vast majority of these requests: in fiscal year 2023, more than 2,300 individual expanded access applications for drugs and biologics were submitted, and nearly all were allowed to proceed.
Who Qualifies for Expanded Access
Expanded access isn’t available to anyone who simply wants to try an experimental treatment. Five conditions must all be met. The patient must have a serious or immediately life-threatening disease. No comparable or satisfactory alternative therapy can exist to diagnose, monitor, or treat their condition. Enrollment in a clinical trial must not be possible, whether because no trial is recruiting, the patient doesn’t meet trial criteria, or the trial site is too far away. The potential benefit of the treatment must justify the risks. And providing the drug to the patient must not interfere with clinical trials that could lead to the drug’s eventual approval for everyone.
That last requirement matters more than it might seem. If granting expanded access would slow down a drug’s path to market, it could ultimately harm far more patients than it helps. The FDA weighs this tradeoff for every request.
Types of Expanded Access
The FDA offers several categories depending on how many patients need treatment and how urgent the situation is.
Individual patient access is the most common type. A physician submits a request for a single patient using a streamlined two-page form (Form FDA 3926). In non-emergency situations, there’s a 30-day waiting period after the FDA receives the application before treatment can begin, though the FDA often grants permission to start sooner. If the request is added as a new protocol to an existing application the drug manufacturer already has on file, there’s no 30-day wait, but the submission still needs FDA receipt and ethics board approval before treatment starts.
Emergency access covers situations where a patient needs treatment before paperwork can be completed. A physician can call the FDA directly, provide the necessary information by phone, fax, or email, and receive authorization to begin treatment immediately. The written application must follow within 15 business days.
Intermediate-size population access covers groups of patients, typically when a drug shows enough promise that multiple people with the same condition need it but a full clinical trial isn’t available to them. Treatment INDs serve even larger populations, sometimes as a bridge while a drug awaits final FDA approval.
How a Physician Requests It
Patients can’t apply for expanded access on their own. A licensed physician must initiate the process, and the first step isn’t contacting the FDA. It’s contacting the drug manufacturer. The company must agree to provide the investigational drug before anything else can move forward. If the manufacturer says no, the process stops there, because the FDA cannot compel a company to supply its product.
Once the manufacturer agrees, the physician submits a request to the FDA. For individual patients, Form FDA 3926 was designed specifically for this purpose and is significantly simpler than the standard research application forms. For requests involving more than one patient, the older, more detailed forms (1571 and 1572) are still required.
An Institutional Review Board, the independent ethics committee that oversees research involving people, must also review the request. For individual patients, the FDA has simplified this step considerably. Instead of requiring a full board meeting, a single IRB chairperson or designated member can review and concur with the request. In true emergencies, treatment can begin before IRB review as long as the board is notified within five working days. For medical devices, only IRB chair concurrence is needed regardless of circumstances.
Why a Manufacturer Might Say No
The drug company’s agreement is often the biggest hurdle. Manufacturers deny expanded access requests for several legitimate reasons. The most common is limited supply. Drugs still in development are typically manufactured in small quantities, just enough for clinical trial participants. Any commitment to provide the drug outside of trials requires having enough product on hand without shortchanging the research.
Companies also weigh whether granting access could delay or complicate clinical trials. If expanded access draws potential participants away from a trial, or if a patient has a bad outcome that creates regulatory complications, the drug’s path to approval for the broader public could slow down. Some companies also lack the infrastructure to manage individual treatment requests while running complex trials. These decisions are made case by case, and there is no appeals process if a manufacturer declines.
What It Costs
Expanded access isn’t necessarily free. Manufacturers are allowed to charge patients for investigational drugs, but only after receiving written FDA authorization and only to recover direct costs: raw materials, labor, supplies, shipping, and storage. They cannot profit from providing drugs through expanded access. Unless the FDA specifies otherwise, charging authorization lasts one year and can be renewed.
Insurance generally does not cover the investigational drug itself or the medical costs associated with administering it. This means patients may face out-of-pocket expenses for the drug, doctor visits, lab work, and any monitoring related to the experimental treatment. The financial burden varies widely depending on the drug and the complexity of treatment.
FDA Approval Rates
Once a request actually reaches the FDA, approval is nearly guaranteed. In fiscal year 2023, the FDA allowed 99% or more of expanded access requests to proceed across almost every category. For emergency requests specifically, every single one was authorized. For medical devices, the approval rate was 98.6% for standard requests and 100% for those submitted under investigational device exemptions.
These numbers can be misleading, though. They only reflect requests that made it to the FDA, meaning the manufacturer already agreed to provide the drug. The real bottleneck is getting a company to say yes in the first place, a step that happens before the FDA is ever involved and for which no public data exists.
How It Differs From Right to Try
The federal Right to Try Act, signed into law in 2018, created a separate pathway for patients to access investigational drugs. Both programs target patients with life-threatening illnesses who have exhausted approved treatments and can’t join a clinical trial. But the similarities largely end there.
Right to Try bypasses the FDA entirely. A patient and their physician negotiate directly with the drug manufacturer, with no FDA review of whether the treatment is appropriate and no IRB oversight of the process. The drug must have completed at least one Phase I safety trial and have an ongoing effectiveness trial. Manufacturers can charge for the drug, and as with expanded access, insurance typically does not cover any costs.
Expanded access, by contrast, keeps the FDA in the loop. The agency evaluates whether the drug has potential to benefit the patient, an IRB reviews the treatment plan, and the FDA monitors safety reporting. This oversight adds a layer of protection but also adds time, which is why the emergency expanded access process exists for situations where hours matter.
In practice, expanded access remains far more widely used. The FDA processes thousands of requests annually, while Right to Try usage has been minimal since the law’s passage, partly because manufacturers still have the same concerns about supply and liability regardless of which pathway a patient uses.