What may be said, labeled and charged for a drug still in trials

Three rules, before any of the paperwork

The federal rules for investigational drugs run to dozens of sections about applications, holds, reports and records. Three short ones sit at the front, before any of that.

One says what the package has to be labeled. One says what anyone running the study may say about it. One says what may be charged for it, and how the number has to be built.

They are worth reading on a market like this one for a plain reason. They describe the restraint that applies where a compound really is under investigation, and the comparison to a product page is stark.

The label the rules actually prescribe

The first rule is one sentence long. The immediate package of an investigational new drug intended for human use has to bear a label with a stated caution. The words are fixed: new drug, limited by federal or United States law to investigational use.

That is a different phrase from the one this market prints, and the difference is not stylistic. A research-use label is a statement about the buyer a chemical supplier is selling to. The investigational caution is a statement about a drug inside a federal application, and this site covers the other label separately.

The second paragraph is the one that matters more. The label or labeling must not bear any statement that is false or misleading in any particular. It must not represent that the drug is safe or effective for the purposes for which it is being investigated.

Read that last clause slowly. The sponsor running the study is forbidden from claiming, on the package, the very thing the study exists to find out.

A narrow exception exists. A center director may grant an alternative to the caution statement for specified lots destined for the national stockpile, to the extent the requirement is not fixed by statute. It is a carve-out for a stockpile, not a general escape.

The rule against promotion, and the sentence that stops it being a gag

The second rule opens with a prohibition. A sponsor or investigator, or any person acting on their behalf, must not represent in a promotional context that an investigational new drug is safe or effective. That applies to the purposes for which the drug is under investigation. The same sentence adds that they must not otherwise promote it.

The next sentence is the one people forget, and it changes the shape of the rule. This provision is not intended to restrict the full exchange of scientific information concerning the drug, including dissemination of scientific findings in scientific or lay media.

Then the rule states its own intent. It exists to restrict promotional claims of safety or effectiveness for a use under investigation, and to preclude commercialization of the drug before it is approved for commercial distribution.

So publishing a result is permitted. Discussing it in the press is permitted. Turning it into a claim, or into a business, is not. That line between a finding and a claim is the same line this site keeps drawing from the advertising side, arrived at from a different direction.

One sentence decides the commercial question

The same rule carries a second paragraph, and it is nine words of consequence. A sponsor or investigator must not commercially distribute or test market an investigational new drug.

There is no proviso, no threshold and no exception paragraph attached to it. Selling it, and trying a market to see whether it would sell, are both named and both barred.

A third paragraph closes a subtler gap. A sponsor must not unduly prolong an investigation after finding that the results appear to establish sufficient data to support a marketing application. A study cannot be kept running as a way of keeping a supply going.

The scope has to be stated plainly here. All of this binds a sponsor or an investigator, meaning the people running an investigation under a federal application. A company that has filed nothing is not a sponsor, so this rule is not the thing that reaches it. What the rule shows is the standard the regulated version of the same activity is held to.

What it takes to charge anything at all

The third rule governs money, and it starts by taking the decision away from the sponsor. A sponsor must obtain prior written authorization from the agency to charge for an investigational drug, and must justify the amount.

The agency also keeps the ability to stop it. Authorization is withdrawn if the agency determines that charging is interfering with the development of the drug for approval, or that the criteria are no longer met.

Charging inside a clinical trial takes three separate showings. Evidence that the drug has a potential clinical benefit that would be a significant advantage over available products. A demonstration that the trial data would be essential to establishing effectiveness or safety for an initial approval, or would support a significant labeling change. And a demonstration that the trial could not be conducted without charging, because the cost of the drug is extraordinary to the sponsor.

The rule even lists what makes a cost extraordinary. Manufacturing complexity, scarcity of a natural resource, the large quantity of drug needed, or some combination of those and other extraordinary circumstances.

Charging for expanded access has its own conditions. The sponsor must give reasonable assurance that charging will not interfere with developing the drug for approval. For widespread treatment use that assurance has to include enrollment evidence, evidence of adequate development progress, and the development milestones planned for the next year. Authorization runs for one year unless the agency specifies a shorter period.

Direct costs, and the accountant at the end of it

The rule then defines what may be recovered, and the definition is tight. A sponsor may recover only the direct costs of making its investigational drug available.

Direct costs are the costs that can be specifically and exclusively attributed to providing the drug for the authorized use. The rule names them. One is the per-unit cost to manufacture, meaning raw materials, labor and nonreusable supplies and equipment. Another is the cost of acquiring the drug from another manufacturing source. Direct costs to ship and handle it also count.

Indirect costs are named too, and excluded. Facilities and equipment primarily intended to produce large commercial quantities. Research and development. Administrative, labor and other costs that would be incurred even if the trial or treatment use had never happened.

For the two larger access sizes, a sponsor may add the cost of monitoring the access application, of complying with reporting requirements, and other administrative costs directly associated with it.

The last paragraph is the one worth remembering. The sponsor must provide supporting documentation showing the calculation is consistent with those requirements. That documentation has to be accompanied by a statement that an independent certified public accountant has reviewed and approved the calculations.

That is not a price. It is an audited cost recovery, authorized in advance, capped at what the drug cost to make and move, and reviewed by someone outside the company.

Why these three travel together

The three rules are not usually read as a set, and one statute reads them that way for us.

The right-to-try law exempts an eligible investigational drug from the approval requirement, the labeling requirements, the consent and review board regulations, and the investigational drug regulations as a whole. That is a wide exemption.

It then attaches a condition. The exemption holds only where the sponsor, and every person who manufactures, distributes, prescribes, dispenses or provides the drug, complies with the labeling rule, the promotion rule and the direct-costs paragraph.

When Congress wrote the widest door in the federal drug law, those three were what it kept. A companion article here walks through that route and the older one beside it.

What a reader can do with rules that do not apply

The honest position is that none of this reaches an online seller, and an absence of one rule is not a permission from another. What a seller may say is governed by advertising law, and this site covers that standard separately.

The value here is a set of comparisons that cost nothing to make. When a page says a compound is under investigation, the rules describe what that state normally involves: a named sponsor, a federal application, a fixed caution on the package.

When a page presents a study as proof that a compound works, the rules show that the people who ran the study would be forbidden from saying so while it was under investigation.

And when a page prints a number, the rules show what a lawful figure for an investigational drug looks like. Authorized in writing beforehand, limited to direct cost, and signed off by an outside accountant. A retail price is not a smaller version of that. It is a different thing entirely.

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 312.6 — Labeling of an investigational new drug, prescribing the caution statement and barring any representation that the drug is safe or effective for the use under investigation — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
2. 21 CFR 312.7 — Promotion of investigational drugs, including the scientific-exchange proviso, the bar on commercial distribution and test marketing, and the rule against unduly prolonging an investigation — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
3. 21 CFR 312.8 — Charging for investigational drugs under an IND, including the prior written authorization requirement, the definitions of direct and indirect cost, and the independent accountant review — Office of the Federal Register, Electronic Code of Federal Regulations, August 2026
4. 21 U.S.C. 360bbb-0a — Investigational drugs for use by eligible patients, whose exemption is conditioned on compliance with the labeling rule, the promotion rule and the direct-costs paragraph — Office of the Law Revision Counsel, United States Code, September 2026