What expanded access and right to try actually require

The question underneath a lot of this marketing

Peptide product pages describe compounds as promising, as studied, as under investigation. A reader who takes that seriously arrives at a fair question. If something is real but not approved, is there a lawful way to receive it?

There are two written answers. One is a set of regulations. The other is a statute added to the federal drug law in 2018.

Both are short, both are public, and neither is quoted on any page selling anything. Reading them is useful for a reason that has nothing to do with using them. They describe what a supervised route to an unapproved drug looks like, and the comparison is unflattering to almost everything else.

The first route, and the three findings it turns on

The regulations call it expanded access, and they sit in their own subpart of the rules that govern investigational drugs. The subpart states its own aim. It exists to make such drugs available to patients with serious diseases or conditions when there is no comparable or satisfactory alternative therapy.

One section carries the criteria for every kind of expanded access, and the agency has to determine three things before any of it happens.

First, that the patient or patients to be treated have a serious or immediately life-threatening disease or condition, and that no comparable or satisfactory alternative therapy exists to diagnose, monitor or treat it.

Second, that the potential patient benefit justifies the potential risks, and that those risks are not unreasonable in the context of the disease or condition being treated.

Third, that providing the drug will not interfere with the initiation, conduct or completion of clinical investigations that could support marketing approval. The trial comes first, in writing.

Both threshold words are defined, and the definitions are not loose

The subpart defines its own terms rather than leaving them to argument. An immediately life-threatening disease or condition means a stage of disease where there is a reasonable likelihood that death will occur within a matter of months. It also covers a stage where premature death is likely without early treatment.

A serious disease or condition means one associated with morbidity that has a substantial impact on day-to-day functioning. The definition then narrows itself twice. Short-lived and self-limiting morbidity will usually not be sufficient. The morbidity need not be irreversible, provided it is persistent or recurrent.

It closes by saying that whether a disease is serious is a matter of clinical judgment. The factors it names are survival, day-to-day functioning, and whether an untreated condition will progress to something more serious.

A separate regulation defines life-threatening for a related purpose, and the statute below borrows it. It means diseases where the likelihood of death is high unless the course of the disease is interrupted. It also covers conditions with potentially fatal outcomes, where the end point of trial analysis is survival.

What has to be filed, and who becomes responsible

Expanded access is not an informal arrangement. It requires a submission for each type of access, either a new investigational application or a protocol amendment to an existing one, and the mailing cover has to be marked as an expanded access submission.

The contents are specific. A cover sheet. The rationale for the intended use, with a list of the therapeutic options that would ordinarily be tried first, or an explanation of why the investigational drug is preferable. The criteria for patient selection, or for one patient a description of the disease, recent medical history and previous treatments.

Then the parts that describe the product rather than the person. The method of administration, the dose and the duration of therapy. A description of the facility where the drug will be manufactured. Chemistry, manufacturing and controls information adequate to ensure the proper identification, quality, purity and strength of the drug.

It also requires pharmacology and toxicology information adequate to conclude that the drug is reasonably safe at what is proposed. And it requires a description of the clinical procedures, laboratory tests or other monitoring needed to evaluate the effects of the drug and minimize its risks.

The safeguards then attach names to people. A licensed physician administering or dispensing the drug is an investigator, with an investigator's duties. Whoever submits the access application is a sponsor, with a sponsor's duties. Investigators report adverse events, obtain consent under the federal consent rules, and secure review board approval. Sponsors file safety reports and annual reports, and keep drug disposition records.

One patient, some patients, many patients

The subpart is written in three sizes, and each adds conditions to the three findings above.

For an individual patient, the treating physician must determine that the probable risk from the drug is not greater than the probable risk from the disease. The agency must determine that the patient cannot obtain the drug under another application or protocol. Treatment is generally limited to a single course of therapy for a specified duration, and at the end the physician or sponsor must give the agency a written summary of the results, including adverse effects.

That section also has an emergency path. Where a patient has to be treated before a written submission can be made, an agency reviewing official may authorize the use by telephone, and the written submission follows within fifteen working days.

For an intermediate-size population, the agency must find enough evidence of safety to justify a trial in about that many people, and at least preliminary clinical evidence of effectiveness or a plausible pharmacologic effect. The section names the situations it is for, including a drug so rarely needed that no trial can be recruited, and patients who cannot join an open trial.

For widespread use, the drug has to be in a controlled trial designed to support a marketing application, or all its trials have to be finished. The sponsor has to be actively pursuing approval with due diligence. And there has to be sufficient clinical evidence of safety and effectiveness, which the rule says would ordinarily consist of data from late-stage trials.

The second route, and the list it runs on

The statute is titled "Investigational drugs for use by eligible patients," and it defines both halves of its own name.

An eligible patient is one diagnosed with a life-threatening disease or condition as the regulation defines that term. The patient must have exhausted approved treatment options and be unable to participate in a clinical trial involving the drug. A physician has to certify that, and the statute adds two conditions to the certifier: good standing with a licensing organization or board, and no direct compensation from the manufacturer for certifying. The patient must have given written informed consent.

An eligible investigational drug carries four requirements. A first-phase trial has to have been completed. The drug must not be approved or licensed for any use. An application must have been filed, or the drug must be under investigation in a trial intended to form the primary basis of an effectiveness claim and be the subject of an active investigational application. And its active development or production has to be ongoing, not discontinued and not on clinical hold.

Read those together and the route is narrower than its name suggests. It is for a specific person with a specific kind of disease, and for a specific drug that is already deep inside the regulated system.

What even the permissive route refuses to give up

The statute grants a real exemption. A drug provided this way is exempt from the adequate-directions labeling requirement and from the prescription-labeling paragraph. It is also exempt from the approval requirement, from the investigational-application section, and from the biologics licensing provision. The consent, review board and investigational drug regulations drop away entirely.

Then comes the condition, in the same sentence. That exemption applies only where the sponsor, and any person who manufactures, distributes, prescribes, dispenses or provides the drug, complies with three specific regulations.

Those three are the labeling rule for an investigational drug, the rule against promoting one, and the paragraph limiting any charge to direct costs. So the most permissive federal access route in existence keeps the investigational caution on the package, the ban on selling the idea, and the ceiling on the price. A companion article here reads all three.

That is the sentence worth carrying away from the whole statute. When Congress wrote the widest door, it left those three things standing.

Nobody has to say yes

The older expanded access statute is written as a permission rather than a duty. A person acting through a licensed physician may request an investigational drug from a manufacturer or distributor, and the manufacturer or distributor may provide it after the conditions are met.

The right-to-try statute makes the point in the form of a liability note. No liability lies against a sponsor, manufacturer, prescriber, dispenser or other entity for its determination not to provide access to an eligible investigational drug.

The same note limits liability the other way for acts or omissions in compliance with the section, except where conduct amounts to reckless or willful misconduct, gross negligence or an intentional tort under state law. And it says that apart from those two paragraphs, nothing modifies anyone's right to bring a private action under state or federal product liability, tort, consumer protection or warranty law.

There is also a reporting duty. The manufacturer or sponsor has to submit an annual summary of any use under the section. It covers the number of doses supplied, the number of patients treated, the uses the drug was made available for, and any known serious adverse events. The agency posts an annual summary report on its own website.

What this does and does not settle

Both routes turn on a finding about a disease. One asks for a serious or immediately life-threatening condition with no satisfactory alternative. The other asks for a life-threatening one, with approved options exhausted and no trial available.

A purchase made for recovery, sleep, skin, energy or longevity is not inside either description, and no paperwork converts it into one. Both routes also require a named licensed physician and a federal file. Neither is a category a product page can join by describing itself well.

Whether any particular purchase is lawful is not a question this site answers. It turns on a specific transaction, a specific substance and a specific place, and it is a question for a lawyer.

What the two rules do give a reader is a yardstick. This is what it looks like when the system deliberately lets an unapproved drug reach a person. A defined disease, a physician on the record, an agency determination, a monitoring plan, a written summary at the end, and a price capped at cost.

Sources

Each document below is named as it names itself, with the date printed on that document rather than the day it was read.

1. 21 CFR 312.300 — General, stating the aim of expanded access and defining "immediately life-threatening disease or condition" and "serious disease or condition" — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
2. 21 CFR 312.305 — Requirements for all expanded access uses, including the three criteria, the contents of a submission, and the safeguards placed on sponsors and investigators — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
3. 21 CFR 312.310 — Individual patients, including for emergency use, with the single-course limit, the written summary of results, and the telephone authorization procedure — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
4. 21 CFR 312.315 — Intermediate-size patient populations, including the situations it is written for and its own safety and effectiveness criteria — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
5. 21 CFR 312.320 — Treatment IND or treatment protocol, including the trial status, marketing status and evidence conditions for widespread treatment use — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
6. 21 CFR 312.81 — Scope, defining "life-threatening" and "severely debilitating" for the regulations the right-to-try statute points at — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
7. 21 U.S.C. 360bbb-0a — Investigational drugs for use by eligible patients, defining eligible patient and eligible investigational drug, and setting the exemption and its three conditions — Office of the Law Revision Counsel, United States Code, September 2026
8. 21 U.S.C. 360bbb — Expanded access to unapproved therapies and diagnostics, the statute behind the regulations, written as a permission to supply rather than a duty — Office of the Law Revision Counsel, United States Code, September 2026