139 glossary terms commonly used in FDA regulatory planning and submissions.
A 505(b)(2) application is a New Drug Application that relies partly on FDA's findings for an already-approved listed drug or on published literature, unlike a 505(b)(1) NDA built entirely on the applicant's own studies.
A 510(k) premarket notification is the FDA submission that clears a medical device for US marketing by proving substantial equivalence to a legally marketed predicate device, not by proving safety and effectiveness from scratch.
Abbreviated 510(k) is a premarket notification format in which conformance to an FDA-recognized consensus standard, special control, or guidance document substitutes for the device-by-device test summaries a Traditional 510(k) requires.
Abbreviated New Drug Application (ANDA) is the FDA marketing application for a generic drug, which wins approval by demonstrating bioequivalence to a reference listed drug rather than repeating the clinical safety and efficacy trials behind an NDA.
Accelerated Approval is an FDA pathway that grants marketing approval for serious or life-threatening illnesses on a surrogate endpoint reasonably likely to predict clinical benefit, conditioned on postmarketing studies that verify and describe that benefit.
Active Pharmaceutical Ingredient (API), also called the drug substance, is the component of a drug product intended to furnish pharmacological activity or other direct effect on the body. Every other component of the dosage form is an inactive ingredient.
Adaptive trial design is a clinical trial design whose prespecified rules let interim data change sample size, dose, arms, or enrollment mid-study, unlike a fixed design, which locks every parameter before the first subject is dosed.
Adverse Event (AE) is any untoward medical occurrence in a patient or clinical trial subject administered a pharmaceutical product, recorded whether or not it is considered drug related; causality is assessed separately, not presumed.
Annual Reports are the yearly postmarketing filing required for every approved NDA, ANDA, and BLA, covering distribution, labeling, and minor manufacturing changes that do not rise to the level requiring a supplement.
Audit trail is the secure, computer-generated, time-stamped metadata record of who created, modified, or deleted a regulated record and when, captured independently of the record so that previously recorded values are never obscured.
A basket trial is a master-protocol design that enrolls patients into one study by shared molecular target rather than by disease, testing a single therapy across multiple histologies as separate, independently analyzed cohorts.
A batch record is the controlled document that both instructs and evidences the manufacture of a single batch of drug product, capturing actual materials, equipment, parameters, yields, deviations, and operator signatures step by step.
BIMO is FDA's inspection program for the entities that generate research data, covering clinical investigators, sponsors, CROs, IRBs and nonclinical laboratories, and auditing study conduct and data integrity rather than manufacturing.
Biocompatibility is the property of a finished medical device to contact tissue, blood, or bone without provoking an unacceptable adverse biological response, evaluated for a defined contact category and duration rather than material by material.
Bioequivalence is the regulatory standard requiring a test product to deliver the same rate and extent of active-ingredient exposure as its comparator, unlike pharmaceutical equivalence, which compares only composition, strength, and dosage form.
A Biologics License Application (BLA) is the marketing application a manufacturer submits to obtain an FDA license under section 351 of the Public Health Service Act, granted on a demonstration that the biological product is safe, pure, and potent.
A biosimilar is a biological product licensed under section 351(k) of the Public Health Service Act as highly similar to an existing reference product, with no clinically meaningful differences in safety, purity, and potency.
Biosimilar interchangeability is a statutory FDA determination, one tier above biosimilarity, that a biosimilar produces the same clinical result as its reference product in any given patient and may be substituted without the prescriber's intervention.
Black Box Warning is FDA's most serious prescription drug labeling warning, a bordered box at the start of the prescribing information reserved for contraindications or serious warnings that may lead to death or serious injury.
Blinding is the bias-control measure in a clinical trial that conceals treatment assignment from subjects, investigators, or analysts, distinct from randomisation, which determines how subjects are assigned rather than who knows the assignment.
Breakthrough Therapy Designation is an FDA expedited-development status granted when preliminary clinical evidence indicates a drug may substantially improve on existing therapies for a serious or life-threatening condition on a clinically significant endpoint.
A bridging study is a clinical study run in the region where approval is sought that generates the pharmacodynamic, pharmacokinetic, or clinical data needed to extrapolate foreign pivotal trial results to that region's population.
CAPA is the quality-system element that investigates a detected problem to root cause and eliminates that cause so it cannot recur, unlike a correction, which only fixes the affected batch.
CBER is the FDA center that licenses biological products under section 351 of the Public Health Service Act, covering vaccines, blood, allergenics, human tissues, and cell and gene therapies, rather than the therapeutic proteins CDER reviews.
CDER is the FDA center that reviews human drugs, covering prescription, over-the-counter, and generic products plus most therapeutic biologics, while vaccines, blood products, and cell and gene therapies are reviewed by CBER.
CDRH is the FDA center that regulates medical devices, in vitro diagnostics, and radiation-emitting electronic products, reviewing premarket submissions such as the 510(k) and enforcing quality-system and adverse-event reporting duties after market entry.
Current Good Manufacturing Practice (cGMP) is the FDA-enforced minimum standard for the methods, facilities, and controls used to manufacture, process, pack, and hold drugs. Drugs made outside cGMP are adulterated by law, regardless of how the batch tests.
Class I is FDA's lowest-risk medical device class, covering devices for which general controls alone provide reasonable assurance of safety and effectiveness, without the special controls or premarket approval higher classes require.
Class II medical devices are the FDA device class subject to special controls, the additional device-type-specific requirements necessary to reasonably assure safety and effectiveness for a generic device type, beyond the general controls every device must meet.
Class III medical devices are the FDA's highest-risk device class, covering devices that are life-supporting, life-sustaining, or present a potential unreasonable risk of illness or injury, and require premarket approval rather than 510(k) clearance.
A clinical hold is an FDA order under an IND that delays a proposed clinical investigation or suspends an ongoing one, halting dosing until the sponsor corrects the deficiencies FDA cited in writing.
Clinical Trial Application (CTA) is the authorisation dossier a sponsor files with a national or EU regulator before dosing the first subject in a trial of an investigational medicinal product, the non-US counterpart to an FDA IND.
CMC is the quality section of a drug application, covering the composition, manufacturing process, and controls for drug substance and drug product, as opposed to the nonclinical and clinical sections that establish safety and efficacy.
A combination product is a medical product composed of two or more regulated constituent parts - drug, device, or biological - that FDA regulates through one lead center, assigned by the constituent part providing the product's primary mode of action.
Compendial Method is an analytical procedure published in an official pharmacopoeia such as USP-NF that a manufacturer may cite by reference instead of documenting its own accuracy and reliability data. Suitability still must be verified in the using laboratory.
Complete Response Letter is the FDA action letter that closes a review cycle without approval, describing every deficiency the agency identified that a sponsor must resolve before an NDA, ANDA, or BLA can be approved.
A Contract Research Organization is an independent contractor that assumes, under written transfer from a sponsor, one or more of that sponsor's regulatory obligations for a clinical investigation and must itself comply with the regulations governing them.
A control strategy is the complete planned set of controls, spanning input materials, process parameters, in-process tests, and release specifications, that together assure process performance and product quality. No single specification is the control strategy.
Critical Process Parameter is a manufacturing process input whose variability measurably impacts a critical quality attribute, and which therefore must be monitored or controlled within a justified range so the process delivers the intended quality.
A Critical Quality Attribute is a physical, chemical, biological, or microbiological property of a drug substance, excipient, intermediate, or drug product that must be held within a defined limit, range, or distribution because its variation would compromise product quality.
Common Technical Document is the ICH-harmonized dossier structure that fixes what content belongs in each of five modules of a drug application, letting one technical package serve regulators across the ICH regions.
Data integrity is the property of regulated records being complete, consistent, and accurate across their full lifecycle, from first capture through retention, so a reviewer can reconstruct an activity without relying on the people who performed it.
A Data Monitoring Committee is an independent group of clinicians and statisticians that reviews unblinded accumulating data during a trial and recommends to the sponsor whether to continue, modify, or terminate it.
De Novo Classification Request is an FDA premarket submission that asks the agency to classify a novel device into class I or class II when no legally marketed device exists to support a 510(k) substantial equivalence review.
Decentralized Clinical Trials are clinical investigations in which some or all participant activities, including consent, dosing, assessments and safety monitoring, happen at the participant's home or a local provider instead of the investigator's research site.
Design controls are the FDA-mandated design and development requirements for medical devices, governing how a device is planned, specified, verified, validated, and transferred to production, unlike production controls that govern manufacture of an already-approved design.
Design Space is the regulator-approved multivariate region of input material attributes and process parameters demonstrated to assure quality, inside which a manufacturer can move without filing a post-approval change.
Deviation is a departure from an approved instruction, procedure, specification, or established standard in a regulated process, which GMP requires to be recorded and justified, and investigated whenever it is critical or unexplained.
A DMF is a standalone submission of confidential manufacturing, packaging, or excipient information that FDA reviews only when an application incorporates it by reference, letting a supplier support a customer's filing without disclosing trade secrets.
DSUR is the annual clinical-trial safety report a sponsor submits to regulators for an investigational drug, consolidating all trials of that drug worldwide, unlike the PSUR, which covers products already on the market.
eCTD is the standard electronic format for pharmaceutical regulatory submissions, organizing CTD content into five modules with a machine-readable backbone so an agency can navigate an application and track every amendment across its lifecycle.
An EIR is FDA's full narrative record of a completed facility inspection, prepared after the investigator leaves and assigning the inspection a classification that determines FDA's next action. The Form 483 lists observations; the EIR evaluates them.
Elemental impurities are inorganic contaminants, principally metals such as arsenic, cadmium, mercury and lead, that reach a drug product from catalysts, drug substances, excipients, water, manufacturing equipment or container closure systems rather than being added for therapeutic benefit.
Endotoxin testing is the quality control assay that quantifies bacterial lipopolysaccharide in parenteral drugs, biologics, and blood-contacting devices, detecting one specific fever-causing contaminant rather than all pyrogens or viable microorganisms.
eSTAR is FDA's interactive PDF submission template, mandatory for 510(k) and De Novo submissions, that the CDRH Portal rejects until every applicable field and attachment is complete. eSTAR use stays voluntary for PMA, IDE, 513(g), and Q-Submissions.
EU GMP Annex 11 is the EudraLex Volume 4 annex governing computerised systems used in GMP-regulated activities, covering lifecycle risk management, validation, audit trails, access control and periodic evaluation, not only the electronic records and signatures Part 11 addresses.
An excipient is any component of a drug product other than the active ingredient: the binders, diluents, buffers, coatings and preservatives that carry, stabilize or deliver the drug without pharmacological activity of their own.
Expanded access is the FDA-authorized use of an investigational drug, biologic, or device to treat a patient outside a clinical trial, permitted only when the disease is serious or immediately life-threatening and no satisfactory alternative therapy exists.
Fast Track Designation is an FDA status, granted on sponsor request, that expedites development and review of a drug for a serious or life-threatening condition demonstrating potential to address an unmet medical need.
FDA is the United States federal agency within the Department of Health and Human Services that authorizes drugs, biologics, and medical devices before marketing and regulates the safety and labeling of foods and cosmetics.
FDA Form 483 is the written list of inspectional observations an FDA investigator hands to facility management at the close of an inspection, recording conditions that may violate the FD&C Act without constituting a final agency determination.
A first-in-human study is the first administration of an investigational drug or biologic to human subjects, run under an IND in effect, to establish tolerability, pharmacokinetics, and a safe dose range before efficacy is tested.
GMP is the minimum standard for the methods, facilities, and controls used to manufacture a drug, enforced through adulteration: a drug made outside GMP is legally adulterated regardless of whether it passes its release tests.
Good Clinical Practice (GCP) is the international standard governing the design, conduct, recording, and reporting of clinical trials in human participants, binding sponsors and investigators to protect participant rights and safety while making trial data verifiable.
Good Laboratory Practice is the FDA quality system at 21 CFR Part 58 governing nonclinical safety studies submitted for research or marketing permits, distinguishing pivotal toxicology work from exploratory laboratory research that carries no such obligations.
GxP is the collective term for the good-practice regulations and guidelines that govern how regulated companies generate, document, and retain data across laboratory, clinical, and manufacturing activities.
ICH is the global standards body where pharmaceutical regulators and industry associations jointly draft technical guidelines for drug quality, safety, and efficacy; it enacts no law itself, so guidelines apply only once a member authority adopts them.
IEC 62304 is the international standard defining the life cycle processes for medical device software, covering development and maintenance of software that is itself a device or embedded in one, but stopping short of final device validation and release.
IND is the FDA application a sponsor submits before shipping or administering an unapproved drug to human subjects, authorizing a clinical investigation rather than granting marketing approval. Studies of a lawfully marketed drug may be exempt.
IND-enabling studies are the nonclinical pharmacology, toxicology and ADME work a sponsor runs to support the conclusion that human dosing is reasonably safe, distinguishing them from exploratory discovery studies never submitted to FDA.
Informed Consent is the legally effective authorization a research subject gives before any study procedure, obtained under conditions that allow unhurried consideration, minimize coercion, and use language the subject actually understands.
Investigational Device Exemption is FDA authorization to ship and use an uncleared or unapproved device in a clinical investigation, exempting it from premarket approval and performance-standard requirements while safety and effectiveness data is collected.
The Investigator's Brochure is the sponsor-maintained compilation of clinical and nonclinical data on an investigational product, functioning as its pre-approval labeling: the reference investigators use to judge risk-benefit and comply with the protocol.
An IRB is the committee a research institution formally designates to approve, require modification of, or disapprove human-subject research before it begins, and to re-review it at least annually under FDA rules.
ISO 14971 is the international consensus standard for applying risk management to medical devices, covering the identification, evaluation, and control of risk to patients and users across the product lifecycle rather than at a single design stage.
IVDs are reagents, instruments, and systems intended for use in diagnosing disease or other conditions, including determining a state of health. They are regulated as medical devices but act on specimens taken from the body, not the patient.
Market Exclusivity is a statutory bar FDA enforces after approving a drug or biologic, blocking the submission or approval of competing generic, 505(b)(2), and biosimilar applications for a fixed period independent of any patent.
Master Protocol is a single overarching clinical trial protocol under which multiple sub-studies run simultaneously, evaluating several investigational products, several populations, or both within one shared infrastructure, control group, and statistical framework.
MDR (Medical Device Reporting) is the FDA post-market surveillance regime requiring manufacturers, importers, and device user facilities to report device-related deaths, serious injuries, and specified malfunctions to FDA on fixed deadlines.
Medical Dictionary for Regulatory Activities (MedDRA) is the standardized medical terminology ICH regions use to code adverse events, indications, and medical history in safety reports, structured as a five-level hierarchy from Lowest Level Term up to System Organ Class.
MedWatch is FDA's safety reporting program and form set for human medical products, covering both voluntary reports from clinicians and patients and the mandatory postmarket reports sponsors and manufacturers must file under 21 CFR.
New Chemical Entity Exclusivity is a five-year FDA marketing exclusivity awarded on the first approval of a drug whose active moiety has never been approved, blocking ANDA and 505(b)(2) submissions rather than merely their approval.
A New Drug Application (NDA) is the marketing application a sponsor submits to obtain FDA approval to market a new drug in the United States, carrying the complete safety, effectiveness, manufacturing, and labeling record supporting that decision.
Nitrosamine impurities are N-nitroso compounds that form in or contaminate drug substances and drug products; ICH M7(R2) places them in the cohort of concern, so each needs a substance-specific acceptable intake, not the generic 1.5 microgram threshold.
No Observed Adverse Effect Level is the highest dose in a nonclinical toxicology study producing no biologically significant adverse finding relative to controls, and it anchors the human starting dose for a first-in-human trial.
Orange Book is FDA's published list of drug products approved as safe and effective under the Federal Food, Drug, and Cosmetic Act, paired with therapeutic equivalence codes and the patent and exclusivity information that governs generic entry.
Orphan Drug Designation is an FDA status, not an approval, granted to a drug for a disease affecting fewer than 200,000 people in the United States, carrying seven years of marketing exclusivity if the drug is later approved.
Out of Specification (OOS) is a laboratory test result that falls outside an established specification or acceptance criterion, obligating a documented investigation and a batch disposition decision before the material can be released.
A patient narrative is the case-level prose account of one trial subject's death, serious adverse event, or other significant adverse event, written for the clinical study report rather than for expedited safety reporting.
Patient-reported outcome (PRO) is an outcome measure captured directly from the patient about their own symptoms, function, or health status, with no interpretation, filtering, or amendment by a clinician, caregiver, or investigator.
PDUFA is the US statute authorizing FDA to collect user fees from human drug and biologic sponsors, tying that revenue to negotiated review performance goals that produce the target action date known as the PDUFA date.
Pediatric exclusivity is a six-month extension of a drug's existing FDA exclusivity and patent-based approval bars, earned by completing the pediatric studies FDA specified in a Written Request under FD&C Act section 505A.
A Pediatric Investigation Plan is the EU development programme for studying a medicine in children, assessed by EMA's Paediatric Committee. Marketing authorisation applications for new medicines must report results from an agreed PIP unless a waiver or deferral applies.
Pediatric Study Plan is the FDA-agreed development document a sponsor submits before its NDA or BLA, outlining the pediatric studies it will run, their age groups and endpoints, and any deferral or waiver request.
The Periodic Benefit-Risk Evaluation Report (PBRER) is the ICH E2C(R2) periodic safety report for marketed medicines, distinguished from the earlier PSUR format by a formal evaluation of benefit alongside risk to support an explicit benefit-risk conclusion.
Periodic Safety Update Report is a pharmacovigilance document a marketing authorisation holder submits at fixed intervals to re-evaluate an authorised medicine's benefit-risk balance against the safety data accumulated worldwide since the previous report.
Pharmacodynamics is the branch of clinical pharmacology that quantifies what a drug does to the body: target engagement, biochemical effect, and clinical response, measured against dose and blood concentration rather than the body's handling of the drug.
Pharmacokinetics is the quantitative description of what the body does to a drug over time: absorption, distribution, metabolism, and excretion, summarized as exposure parameters such as Cmax, AUC, clearance, and half-life that set dose and dosing interval.
Pharmacovigilance is the regulated safety discipline covering detection, assessment, understanding, and prevention of adverse drug reactions, and the reporting obligations that bind sponsors during trials and marketing authorisation holders for as long as a product stays on the market.
Phase 1 clinical trials are the first human studies of an investigational drug, designed to characterize metabolism, pharmacologic actions, and dose-related side effects rather than to prove effectiveness, typically enrolling 20 to 80 subjects.
Phase 2 clinical trials are the controlled studies that evaluate whether a drug is effective in patients with the target disease and determine the dose, regimen, and endpoints that Phase 3 will confirm.
Phase 3 clinical trials are expanded controlled studies run after preliminary evidence of effectiveness, sized at several hundred to several thousand subjects to establish the benefit-risk relationship and supply the substantial evidence supporting approval and physician labeling.
PMA is the FDA's most stringent device marketing application, the pathway by which a Class III device wins approval on its own clinical and nonclinical evidence rather than by demonstrating equivalence to a marketed predicate.
Post-marketing commitments are studies or clinical trials a sponsor agrees in writing to conduct after FDA approval, not compelled by statute or regulation the way post-marketing requirements are, yet still reported annually until FDA agrees they are fulfilled.
Pre-Approval Inspection is an FDA facility inspection conducted while a drug marketing application is pending, testing whether the site can manufacture the product under cGMP and whether the application's data matches what the site actually did.
Pre-Submission Meetings are formal, sponsor-requested meetings with FDA held before an application is filed, securing agency feedback on study design, evidence standards, and submission content while the plan can still change rather than after review begins.
The primary endpoint is the pre-specified outcome measure a clinical trial is powered and statistically tested against, carrying the trial's efficacy claim; secondary and exploratory endpoints add supporting evidence but cannot substitute for it.
Priority Review is an FDA marketing-application designation that shortens the agency's review-and-action target to six months, compressing the review clock only; it changes neither the evidence required nor the standard for approval.
Process Analytical Technology is a system for designing, analyzing and controlling pharmaceutical manufacturing through measurements taken during processing rather than after it, so quality is assured by in-process control instead of end-product testing alone.
Process Validation is the documented, data-based demonstration that a manufacturing process consistently produces drug product meeting predetermined specifications, spanning process design through routine commercial production rather than a one-time qualification exercise.
Process validation is the documented, lifecycle-long demonstration that a commercial manufacturing process reliably yields conforming product, distinguished from equipment qualification by proving the process itself, not the machinery, performs within defined limits.
A protocol amendment is the IND submission that adds a new clinical protocol, changes an existing protocol, or adds an investigator; protocol changes must be submitted to FDA and approved by the IRB before implementation.
The Purple Book is FDA's searchable list of biological products licensed under the Public Health Service Act, recording each product's licensure date, reference-product exclusivity, and biosimilarity or interchangeability status.
Q-Submission is FDA's umbrella mechanism for a device sponsor to request feedback or a meeting rather than a marketing decision, covering Pre-Submissions, Submission Issue Requests, Study Risk Determinations, and Informational Meetings.
A Quality Agreement is a written cGMP contract between a drug owner and a contract facility that assigns each quality responsibility (batch release, deviation investigation, change control, audit rights) to one named party, separate from the commercial supply agreement.
Quality System is the documented management framework a regulated manufacturer uses to direct product quality across the lifecycle, distinct from GMP's minimum production rules by adding management responsibility, monitoring, and continual improvement obligations.
Quality System Regulation (QSR) was FDA's device current good manufacturing practice framework at 21 CFR Part 820, governing design and manufacture of finished devices until the Quality Management System Regulation superseded it on February 2, 2026.
Quality Target Product Profile is a prospective summary of the quality characteristics a drug product must achieve, covering dosage form, strength, delivery, release and stability, from which every critical quality attribute in the control strategy is derived.
Real-World Evidence is clinical evidence about a medical product's usage, benefits, or risks derived from data generated outside traditional clinical trials, such as health records, claims, and registries, rather than from a protocol-driven randomized study.
Refuse to Accept is FDA's administrative decision that a device premarket submission is too incomplete to enter substantive review, a completeness gate applied before any scientific assessment of the device begins.
Refuse to File is FDA's determination, made within 60 days of receiving an NDA, that the application is not sufficiently complete to permit a substantive review, so no filing date and no review clock begin.
Risk Evaluation and Mitigation Strategy (REMS) is an FDA-mandated drug safety program that imposes enforceable conditions on prescribing, dispensing, or patient monitoring whenever FDA determines those conditions are necessary to ensure a drug's benefits outweigh its risks.
An RLD is the FDA-approved listed drug an ANDA relies on as its legal basis, setting the active ingredient, dosage form, strength, route, conditions of use, and labeling the generic must match.
An SAE is any adverse event or suspected adverse reaction that results in death, a life-threatening event, inpatient hospitalization or its prolongation, persistent or significant incapacity, or a congenital anomaly, in the view of either the investigator or sponsor.
Signal detection is the pharmacovigilance activity of searching safety data for evidence that a medicine may cause a new adverse reaction, or that a known reaction has changed, before that evidence is confirmed.
Signal management is the pharmacovigilance process that carries a suspected safety issue from detection through validation, confirmation, prioritisation and assessment to a documented regulatory decision, with every step, date and rationale tracked.
A Special 510(k) is the streamlined premarket notification format for a change the submitter makes to its own previously cleared device, where design control activities can verify and validate the change against well-established methods.
Stability testing is the formal study program that measures how a drug substance or product changes under defined temperature, humidity, and light conditions, generating the evidence that sets shelf life, storage conditions, and the expiration date on the label.
Substantial equivalence is the FDA finding that a device has the same intended use as a legally marketed predicate and either the same technological characteristics or different ones that raise no new questions of safety and effectiveness.
Supplier qualification is the documented assessment that establishes a supplier's ability to consistently provide materials or services meeting specification, and the ongoing verification that keeps that conclusion current.
SUSAR is a serious adverse reaction observed in a clinical trial that is both suspected to be caused by the investigational drug and not already described in the current investigator brochure, triggering expedited regulatory reporting.
Technology transfer is the transfer of product and process knowledge between development and manufacturing, or between manufacturing sites, so the receiving site can reproduce the process and consistently achieve product realisation.
A Traditional 510(k) is the default premarket notification format, in which the submitter argues substantial equivalence by presenting and discussing each supporting test on its own terms rather than relying on a standard or a prior clearance.
UDI is the unique numeric or alphanumeric code FDA requires on medical device labels to identify a device through its distribution and use, built from a fixed device identifier plus a conditional production identifier.
An umbrella trial is a master protocol design that enrols a single disease and assigns participants to different investigational therapies by molecular subtype, running those arms as sub-studies under one governing framework.