How to Use a Clinical Trial Run in Brazil to Support US FDA Approval

Yes, and there are two routes. The one you choose shapes the entire submission.

Route one: run the trial under a US IND. The study follows FDA’s IND requirements regardless of where the sites sit. You get early FDA feedback on design, dose, and endpoints, and the data enters your program the same way US data would.

Route two: run the trial outside a US IND and submit it later under 21 CFR 312.120. Most foreign studies take this route. FDA accepts a well-designed, well-conducted non-IND foreign study as support for an IND or a marketing application when it meets the conditions below.

Under 21 CFR 312.120, the study has to clear four bars:

1. Good Clinical Practice across design, conduct, monitoring, recording, and reporting, so the data are credible and accurate and subjects are protected.
2. Independent ethics committee review, with a favorable opinion before the study starts and continuing review while it runs. The committee has to meet the definition of an IEC in 21 CFR 312.3.
3. Freely given, documented informed consent from every subject.
4. Data FDA can validate on inspection, if it decides an onsite inspection is warranted.

If the study is meant to support effectiveness, it also has to be adequate and well controlled under 21 CFR 314.126.

Running under an IND buys early FDA alignment. Before you commit a Phase 2 design or a starting dose, you can put the question to FDA directly and get a documented answer. For a program headed to the US market, that feedback often prevents an expensive bridging request later.

Running outside an IND is lighter at the front and heavier at submission. You assemble a 21 CFR 312.120 package: a description of how the study conformed to GCP, a protocol and results summary, a description of the drug substance and product used, the adequate-and-well-controlled information where effectiveness is at stake, and the name of the ethics committee with a statement that it meets the IEC definition. FDA keeps the option to inspect.

Neither route is a shortcut. Both put the burden of proof on the sponsor.

It is possible. 21 CFR 314.106 governs approval based solely on foreign clinical data. In practice the harder question is applicability: whether the study population, medical practice, and endpoints map onto the US population and US clinical practice. Approvals resting on data from a single country outside the US have drawn closer scrutiny recently, so the strongest position is usually not a solely-foreign submission. It is Brazilian data sitting inside a multiregional program under ICH E17, with a clear bridge showing the results extrapolate to the US setting.

Brazil earns its place in that program on the merits: large treatment-naive populations, concentrated high-enrolling centers, and a framework modernized under Lei 14.874/2024 and ANVISA’s RDC 945/2024.

The pathway is rarely the problem. The data integrity and inspection-readiness bar is.

An FDA investigator arriving at a Brazilian site is checking whether the record supports the result: source data traceable end to end, informed consent documented to standard, the ethics committee’s constitution and approvals on file and matching the 21 CFR 312.3 definition, and every data point attributable, legible, contemporaneous, original, and accurate. A study that was clinically sound but cannot produce that record on inspection is a study FDA can decline to rely on.

This is a documentation and evidence problem, and it is solvable before you file rather than after FDA asks.

This is the problem NexTrial’s platform is built to address. Celina, our Trial Activation Intelligence layer, checks a protocol and its regulatory package against the destination framework and produces an evidence trail rather than a confidence score. The method rests on formal validation and uncorrelated evidence: each requirement is checked structurally, and the checks do not share a single point of failure. Gate 2 of the Three-Gate Verification System, the structural proof layer, is shipped and in first validation with a sponsor. The remaining gates are designed to carry the same standard through compliance checking and human attestation.