Expanded Access Versus Right to Try: Two Doors to an Unapproved Drug

A patient who has run out of approved options and cannot join a clinical trial may still reach an investigational drug through one of two federal routes. FDA Expanded Access, often called compassionate use, adds a layer of federal and ethics-committee review before treatment starts. The federal Right to Try pathway, enacted in 2018, removes those prospective gates so a physician and manufacturer can arrange access directly. Neither route means the drug has been proven safe or effective, and neither obligates a company to provide it. What follows describes the mechanisms and their trade-offs, not a recommendation to pursue either one.

What the two pathways share

Both routes exist for the same situation: a serious or immediately life-threatening condition, no satisfactory approved therapy, and no available or appropriate clinical trial. Both involve a drug that is still investigational, meaning it has not been approved by the FDA for the use in question. And in both cases the manufacturer holds the key. As the review published in the peer-reviewed literature and indexed by the National Library of Medicine notes, a company can decline a request under either pathway and is under no legal obligation to supply an investigational product. The FDA authorizing a request does not force a manufacturer to fill it.

That last point is easy to miss. Public discussion sometimes frames access as a problem of government permission, but the more frequent bottleneck is that the drugmaker must first agree to provide the drug, often in limited supply, before any regulatory step matters.

How FDA Expanded Access works

Expanded Access runs through an Investigational New Drug (IND) submission to the FDA. For a single patient, the treating physician typically files Form FDA 3926, which the agency built specifically to simplify individual-patient requests. The application states the diagnosis, a brief clinical history, the physician's qualifications, and confirmation that the manufacturer will supply the drug.

Two oversight layers sit on top of that submission. First, the FDA reviews the request and weighs whether the potential benefit justifies the potential risks; for a non-emergency individual request, treatment may generally begin 30 days after the agency receives the application, or sooner if the FDA notifies the physician that it may proceed. Second, an institutional review board (IRB) must review and approve the treatment use. For an individual patient, the rules allow a streamlined step in which the IRB chair or a designated member concurs before treatment begins, rather than waiting for a full convened meeting. The FDA describes these forms and steps on its Expanded Access submission page.

Informed consent is required. The FDA treats expanded-access use as a clinical investigation, so the protections in its human-subjects regulations apply, and the agency has published a single-patient consent template to help physicians document the required elements. The practical effect is that a second physician-reviewer and an ethics body both see the plan before an unapproved drug is given, and the FDA continues to receive safety information as treatment proceeds.

How the federal Right to Try pathway works

The 2018 Right to Try Act created a parallel route in which the prospective FDA and IRB steps do not apply. An eligible drug must have completed a Phase 1 clinical trial, must not be approved or licensed by the FDA for any use, and must be in active development, meaning it is the subject of an active application or an ongoing trial intended to support approval. If those conditions are met, the patient, physician, and manufacturer can arrange access without submitting the request to the FDA in advance and without IRB review.

Oversight does not vanish entirely, but it shifts from before treatment to after. Under the framework the FDA describes on its Right to Try pages, a manufacturer that provides a drug this way must submit an annual summary to the agency, reporting the drug and IND number, the number of doses supplied, the number of patients treated, the conditions treated, and any known serious adverse events. Those annual summaries are due by a fixed date each year and are consolidated by the FDA. What is absent is the case-by-case federal risk-benefit judgment and the independent ethics review that Expanded Access builds in at the front end.

Why neither pathway signals proof

The most important thing both routes have in common is what they do not establish. Completing Phase 1, the threshold for Right to Try eligibility, tests mainly for early safety signals and dosing in a small group; it says little about whether a drug works. The published review underscores how far that is from proof by noting how few drugs that enter early human testing ever reach approval. A drug available through either door remains investigational precisely because the evidence needed to conclude it is safe and effective does not yet exist.

That is the core trade-off. Expanded Access trades speed for two independent checks and continued FDA visibility. The Right to Try pathway trades those checks for a more direct arrangement between the patient, physician, and manufacturer. A patient weighing an investigational drug is accepting genuine uncertainty about benefit and harm under either route, which is why informed, unhurried conversation with the treating clinical team matters so much. This article is educational and is not medical advice.