87 glossary terms commonly used in ICH regulatory planning and submissions.
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.
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.
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.
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.
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.
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.
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.
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 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.
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.
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.
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 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.
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.
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.
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.
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.
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.
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.
ICH M7 is the harmonised guideline governing assessment and control of DNA reactive (mutagenic) impurities in pharmaceuticals, setting acceptable intake limits that ICH Q3A and Q3B leave undefined for genotoxic impurities.
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.
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.
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.
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.
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.
MHRA is the United Kingdom's regulator of medicines, medical devices, and blood components for transfusion, granting UK authorisations through its own assessment or by recognising a named reference regulator, not through the EU centralised procedure.
Minimum Anticipated Biological Effect Level (MABEL) is the human exposure predicted to produce the lowest biologically observable effect, calculated from pharmacology rather than toxicology, and used by EMA as one of three permitted anchors for a first-in-human starting dose.
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.
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.
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.
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 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.
PMDA is Japan's incorporated administrative agency that scientifically reviews drugs, medical devices, and cellular and tissue-based products for marketing approval, inspects the data and plants behind them, and runs post-marketing safety and relief services alongside MHLW.
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.
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.
Quality by Design is a systematic pharmaceutical development approach that fixes quality targets before experimentation, then builds product and process understanding until quality is designed into the manufacturing process rather than tested into the finished batch.
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 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.
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.
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.
RMP is the EU regulatory document that records a medicinal product's risk management system: the important identified risks, important potential risks, and missing information, plus the pharmacovigilance and risk-minimisation activities that address them.
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.
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.
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.
TGA is Australia's national regulator of therapeutic goods, applying the Therapeutic Goods Act 1989 to decide which medicines, biologicals and medical devices may enter the Australian Register of Therapeutic Goods and be supplied lawfully.