Early human studies that primarily assess safety, tolerability, dosing, and pharmacokinetics. They often enroll healthy volunteers, but some programs (for example oncology) enroll patients.

Mid-stage studies in patients that further assess safety and provide preliminary evidence of efficacy, dose selection, and regimen design for later-stage trials.

Usually larger, late-stage studies intended to provide pivotal evidence of efficacy and safety. Many NDA/BLA submissions rely on Phase III data, but the required evidence package varies by disease and pathway.

A key trial intended to provide the main evidence supporting approval (sometimes called a registrational study). Pivotal trials are often Phase III, but may occur in other designs or phases depending on disease and pathway.

A trial design that combines phases in one protocol, such as Phase 1/2 or Phase 2/3. It may start with safety or dose-finding, then move into efficacy testing using prespecified rules. This can save time but requires careful statistical planning.

A post-approval or late-stage trial designed to verify and describe clinical benefit, especially after Accelerated Approval. Failure to confirm benefit can lead to label changes or withdrawal.

The main prespecified outcome measure used to evaluate a drug's effectiveness in a clinical trial. Statistical significance on the primary endpoint is often central to FDA approval decisions.

A prespecified outcome measure used to evaluate additional effects of a treatment beyond the primary endpoint. It can support interpretation and labeling, but formal claims often depend on multiplicity control and study design.

A measurable marker (like tumor response or a lab value) used as a substitute for a direct clinical outcome. It can support faster development when the surrogate is adequately validated or accepted for the context.

The formal submission to FDA requesting approval to market a new pharmaceutical drug. Contains all preclinical and clinical data demonstrating safety and efficacy.

An amendment to an existing NDA for changes like new indications, dosage forms, manufacturing changes, or new patient populations.

An NDA resubmitted after a Complete Response Letter (CRL). The sponsor addresses FDA deficiencies and resubmits for another review cycle.

FDA submission required for approval of biological products including vaccines, blood products, gene therapy, and other complex molecules derived from living organisms.

An amendment to an existing BLA for changes such as new indications, labeling updates, manufacturing changes, or new patient populations for a biologic product.

A BLA that has been resubmitted after a Complete Response Letter (CRL). The sponsor addresses the deficiencies cited by the FDA and resubmits the application for further review.

Application for generic drug approval. It generally relies on pharmaceutical equivalence and bioequivalence to a reference drug rather than new efficacy trials, though some products require additional studies.

A type of NDA that can rely in part on existing studies or FDA findings for a previously approved drug, plus new bridging or supportive data as needed. Common for reformulations or new routes of administration.

A drug containing an active ingredient never previously approved by FDA. Distinct from new formulations, combinations, or new uses of existing drugs.

FDA determination (usually within 60 days of submission) that an application is too incomplete for substantive review. If it is not filed, the review cycle does not proceed.

FDA action granting marketing authorization for the submitted application or supplement. Approval may include final labeling, a REMS, and postmarketing requirements or commitments.

FDA communication indicating that the review cycle is complete but the application is not ready for approval. Lists deficiencies that must be addressed.

Safety strategy required for certain drugs with serious risks. May include medication guides, patient registries, or prescriber certifications.

A study or clinical trial FDA legally requires after approval (or under certain pathways) to further assess safety, efficacy, or optimal use.

A post-approval study the sponsor agrees to conduct that is not legally required in the same way as a PMR. FDA may still track progress and reporting.

Law authorizing FDA user fees for human drug review and setting performance goals for review timelines. It is the basis for many commonly cited NDA/BLA review goal dates.

FDA review designation with a shorter goal timeline (often about 6 months from filing for qualifying applications). It does not lower the evidentiary standard for approval.

Default FDA review designation. For many original NDA/BLA applications, the goal timeline is about 10 months from filing, while supplemental applications may have different goals.

Common shorthand for the FDA target action date on many NDA/BLA reviews under PDUFA. It is a target date for FDA action, not a guaranteed approval date.

Allows companies with Fast Track designation to submit completed portions of their application for review before the entire submission is complete.

Independent expert panel that reviews some applications and gives non-binding recommendations to FDA. Meetings are usually public and may include votes.

Regulatory and technical information describing how a drug is made, tested, and controlled for quality. CMC issues are a common reason for review delays or CRLs.

A CRL resubmission category for lower-complexity responses (for example certain labeling or minor changes). FDA review goal is typically 2 months from receipt.

A CRL resubmission category for more substantial responses, such as significant new analyses, manufacturing updates, or new clinical information. FDA review goal is typically 6 months from receipt.

FDA pilot pathway that can compress review timelines for selected drugs or biologics tied to national priorities. In Endpoint Arena, CNPV may appear as the application type when the public FDA record has not yet disclosed NDA or BLA specifics.

FDA designation for drugs treating serious conditions with unmet need. Enables more frequent FDA communication and rolling submission.

FDA designation for serious conditions when preliminary clinical evidence suggests substantial improvement over available therapy. Includes Fast Track features plus intensive FDA guidance.

Allows approval based on surrogate endpoints (like tumor shrinkage) rather than clinical outcomes (like survival). Requires post-marketing confirmatory trials.