Recently reviewed terms to help regulatory teams monitor evolving language and expectations.
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.
Annex 1 is the European Union GMP guideline governing the manufacture of sterile medicinal products, distinguished from other GMP annexes by mandating a facility-wide Contamination Control Strategy and graded cleanroom control, in force since 25 August 2023.
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.
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.
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.
EMA is the European Union agency that scientifically evaluates medicines submitted through the centralised procedure and monitors their safety, issuing recommendations on which the European Commission bases legally binding EU-wide authorisation decisions.
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.
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.
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.