The federal Right to Try Act is a 2018 U.S. law that lets certain terminally ill patients obtain access to certain unapproved investigational drugs directly from a manufacturer, without going through the U.S. Food and Drug Administration (FDA) authorization step that governs FDA’s separate Expanded Access program (often called “compassionate use”). Its full name is the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, and it was signed into law on May 30, 2018 as Public Law 115-176. It is codified as a new Section 561B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), 21 U.S.C. § 360bbb-0a. (Source: Public Law 115-176, congress.gov.)
For a research administrator or clinical-trial sponsor, the practical significance of the Act is narrower than the “right to try” framing suggests: it removes FDA’s individual-patient authorization step from one specific access route, but it creates no obligation on any manufacturer to provide a drug, imposes no price controls, and does not touch the separate Expanded Access pathway FDA has operated for decades. Understanding what the law actually changes — and what it leaves exactly as it was — matters for anyone advising a sponsor, an investigator, or a patient’s care team on which pathway applies.
What the Federal Right to Try Act Does
Before the federal Act, a terminally ill patient seeking an unapproved investigational drug outside a clinical trial had one route: FDA’s Expanded Access program, which requires the treating physician to obtain the manufacturer’s agreement, get FDA authorization (via a request, most commonly FDA Form 3926 for a single patient), and secure Institutional Review Board (IRB) concurrence. The Right to Try Act created a second, parallel route that removes the FDA-authorization and IRB-review steps for a narrower set of eligible patients and eligible drugs, provided a treating physician certifies eligibility and obtains written informed consent. (Source: FDA, “What is Right to Try?” fact sheet.)
The law followed, and was modeled in part on, a wave of state right-to-try laws. Colorado enacted the first, in 2014, based on model legislation drafted by the Goldwater Institute, and dozens of other states passed similar laws in the years that followed. Those state laws could not bind FDA itself — FDA regulation of drug distribution is federal — which is a large part of why advocates pushed for a federal statute that could actually remove the FDA-authorization step rather than merely declare a state-law right that FDA wasn’t bound by.
Patient Eligibility Requirements
Under FD&C Act Section 561B, a patient is eligible for the federal Right to Try pathway only if all of the following are true:
- The patient has been diagnosed with a life-threatening disease or condition.
- The patient has exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (as certified by a physician, in consultation with the treating physician if different).
- The patient’s treating physician has provided written certification of the above and obtained the patient’s (or legally authorized representative’s) written informed consent regarding the eligible investigational drug.
These criteria roughly track — but are not identical in wording to — the eligibility standard FDA applies under Expanded Access, which is discussed further below.
Investigational Drug Eligibility Requirements
Not every unapproved drug qualifies as an “eligible investigational drug” for Right to Try purposes. The drug must:
- Have completed a Phase 1 clinical trial;
- Not be approved or licensed for any use by FDA;
- Either have an active investigational new drug (IND) application on file with FDA, or be under investigation in a clinical trial that is intended to form the primary basis of a manufacturer’s claim of effectiveness in support of approval; and
- Have ongoing active development or production by the manufacturer — i.e., development has not been discontinued, and the drug is not under an FDA clinical hold.
This scopes the pathway to drugs already inside the FDA development pipeline with at least preliminary human safety data — it does not open access to anything a patient or physician independently believes might help, and it does not apply to devices (a device seeking pre-effectiveness market access under a small-population pathway would instead look at the Humanitarian Use Device (HUD) / Humanitarian Device Exemption pathway, which is a separate FDA mechanism built around a population-size threshold rather than terminal illness).
What the Act Does Not Require or Guarantee
This is the point most often misunderstood, including in early political framing of the law:
- No manufacturer obligation. The Act does not require any sponsor or manufacturer to make an eligible investigational drug available to any patient who requests it. A manufacturer can decline, and most manufacturers evaluate Right to Try requests against the same kind of capacity, liability, and supply constraints they’d weigh for any early-access request.
- No insurance mandate. Nothing in the Act requires an insurer to cover the cost of the drug, associated care, or complications, and the Act does not require the manufacturer to provide it for free (though sponsor policies on this vary).
- No FDA pre-authorization for the individual request — this is the actual mechanical change: FDA does not have to approve a specific patient’s Right to Try request before treatment begins, unlike Expanded Access.
- No IRB review of the individual request is required under the Act itself, in contrast to Expanded Access.
The practical effect is that Right to Try shifts the gatekeeping role away from FDA and the IRB and onto the manufacturer’s own willingness to supply the drug — which is also the basis of the most common criticism of the law: bioethicists including Arthur Caplan have argued that because the Act creates no supply obligation, it primarily removes a regulatory step without materially expanding actual patient access, since the limiting factor in practice is usually manufacturer willingness, not FDA turnaround time.
Sponsor Reporting Obligations
The Act does impose one concrete, ongoing compliance obligation on manufacturers who use it: under FD&C Act Section 561B(d)(1) (21 U.S.C. § 360bbb-0a(d)(1)), a sponsor or manufacturer that provides an eligible investigational drug under Right to Try must submit an annual summary to FDA, due no later than March 31 for the preceding calendar year, covering the number of doses supplied, the number of patients treated, the use(s) for which the drug was supplied, and any known serious adverse events. This requirement was implemented through a final rule at 21 CFR § 300.200. FDA also posts its own aggregate annual summary of program use. (Sources: Federal Register final rule, 2020-16016; FDA, Right to Try Annual Reporting Summary.)
For a sponsor or a technology-transfer office advising an early-stage drug developer, this reporting duty is worth building into regulatory-affairs planning from the point a Right to Try request is granted, not treated as an afterthought — missing the March 31 deadline is a real compliance failure against a real annual regulatory deadline, distinct from a discretionary courtesy report.
Right to Try vs. FDA Expanded Access: The Real Difference
Expanded Access is the older, broader pathway — codified in FDA regulation (21 CFR Part 312, Subpart I) since a 2009 rule that formalized decades of prior agency practice, well before the 2018 Act existed. It remains fully in effect and, in practice, is used far more often than Right to Try.
| Right to Try (FD&C Act § 561B) | Expanded Access (21 CFR Part 312, Subpart I) | |
|---|---|---|
| Legal basis | Federal statute, 2018 | FDA regulation, formalized 2009; agency practice since the 1980s |
| FDA authorization for the individual request | Not required | Required — FDA reviews and authorizes (commonly via Form 3926 for a single patient) |
| IRB review | Not required by the Act | Required, though a single IRB member (chair or designee) can often provide expedited concurrence for a single-patient emergency request |
| Patient eligibility standard | Life-threatening disease; exhausted approved options; unable to enroll in a relevant trial | Serious or immediately life-threatening disease; no comparable alternative therapy; potential benefit justifies the risk |
| Drug eligibility standard | Completed Phase 1; active IND or pivotal trial; active ongoing development | No fixed development-stage floor in regulation, though FDA weighs available safety data case by case |
| Sponsor obligation to provide the drug | None | None — manufacturer participation is voluntary under both pathways |
| FDA authorization rate (single-patient requests) | Not centrally tracked the same way | FDA has authorized over 99% of the roughly 1,000+ single-patient requests it receives annually in recent years |
The 99%-plus Expanded Access authorization rate is the detail that undercuts the assumption that Right to Try’s main value is bypassing a slow or restrictive FDA. FDA very rarely refuses an Expanded Access request outright; where it does intervene, it more often asks for a dosing or monitoring change than a denial. (Source: FDA Expanded Access program reporting, summarized in agency and peer-reviewed literature on Expanded Access authorization rates.) The genuine practical difference the federal Act offers is procedural speed and reduced administrative burden on the treating physician and manufacturer for the narrower set of requests that meet its stricter eligibility criteria — not a materially different chance of ultimately getting the drug, since manufacturer willingness is the binding constraint either way.
Do State Right-to-Try Laws Still Matter?
Yes, in the sense that most states still have their own right-to-try statute on the books, and some state laws include provisions the federal Act does not (for example, some state laws address physician liability protections in more detail). But because FDA drug distribution is governed by federal law, a state right-to-try law was never able to compel a manufacturer to bypass FDA on its own — the federal Act is what actually changed FDA’s role. In practice, the federal pathway is now the operative one for the FDA-facing part of any Right to Try request; state law is more relevant to the state-level physician-liability and licensing questions that sit alongside it.
Why It Matters for Clinical Research Administrators
- It is not a substitute for enrollment. A patient’s inability to enroll in a relevant clinical trial is itself part of the Right to Try eligibility standard — administrators fielding a Right to Try inquiry should first confirm whether an open, enrolling protocol the patient could actually join exists, since that changes which pathway (or neither) applies. See clinical trial patient recruitment for how enrollment eligibility is normally screened.
- It does not relieve consent or documentation rigor. Written physician certification and patient informed consent are still required; the pathway removes FDA/IRB review, not the underlying consent and documentation obligations that also govern standard trial participation (see the IRB/REC approval process for comparison).
- It creates a real annual FDA filing obligation for any sponsor whose drug is supplied under the pathway, which regulatory-affairs and technology-transfer offices supporting an early-stage sponsor should track on the same calendar as other FDA reporting deadlines.
- It sits alongside, not inside, standard IND and clinical trial phase administration — a drug’s Right to Try eligibility depends on its IND and Phase 1 status, so the people managing that trial’s regulatory file are the ones who can actually answer whether a given drug qualifies.
Frequently Asked Questions
What is the Right to Try Act?
It is a 2018 federal law (Public Law 115-176, codified at FD&C Act § 561B) that lets eligible terminally ill patients obtain an eligible investigational drug directly from a manufacturer without FDA pre-authorization or IRB review of the individual request, provided the patient’s physician certifies eligibility and obtains written informed consent.
Who qualifies for Right to Try under federal law?
A patient with a life-threatening disease or condition who has exhausted approved treatment options and is unable to enroll in a clinical trial involving the eligible investigational drug, with physician certification and written informed consent.
Does a manufacturer have to provide the drug if a patient qualifies?
No. The Act creates no obligation for any sponsor or manufacturer to supply an investigational drug to a requesting patient, and it does not require insurance coverage of the drug or related care.
Is IRB review required for a Right to Try request?
Not under the federal Act itself. This is one of the two procedural steps (along with FDA authorization) the Act removes for requests that meet its eligibility criteria, distinguishing it from FDA’s Expanded Access pathway, which does require IRB concurrence.
How is Right to Try different from FDA’s Expanded Access (“compassionate use”) program?
Expanded Access is the older, FDA-regulation-based pathway (21 CFR Part 312, Subpart I) and requires both FDA authorization and IRB review of each request; FDA authorizes over 99% of the single-patient requests it receives. Right to Try is the narrower, federal-statute-based pathway that removes those two review steps for patients and drugs meeting its specific eligibility criteria, but does not change manufacturer willingness to supply the drug, which remains the binding constraint under either pathway.
What does a sponsor have to report after providing a drug under Right to Try?
Under FD&C Act § 561B(d)(1) and implementing regulation 21 CFR § 300.200, the sponsor or manufacturer must submit an annual summary to FDA by March 31 covering doses supplied, patients treated, the use(s) involved, and any known serious adverse events.
Do state right-to-try laws still apply now that there is a federal law?
Most states still have their own right-to-try statute, and some address issues like physician liability protection in more detail than the federal Act. But FDA’s role in the process is governed by federal law, so the federal Act is what actually changed the FDA/IRB review requirement; state law was never able to do that on its own.
