New & Updated Terms

FDA regulations governing electronic records and electronic signatures to ensure their trustworthiness and reliability.

A US NDA pathway that relies, at least in part, on FDA's findings of safety and effectiveness for a previously approved drug or published literature.

A premarket submission demonstrating that a medical device is substantially equivalent to a legally marketed predicate device.

A 510(k) pathway that relies on FDA guidance documents, recognized consensus standards, or special controls to establish substantial equivalence.

An application for FDA approval of a generic drug product that references an already-approved drug.

An FDA pathway allowing earlier approval based on surrogate or intermediate endpoints for serious conditions.

The substance in a pharmaceutical drug that is biologically active and produces the intended therapeutic effect.

A clinical trial design that allows pre-planned modifications to aspects of the trial based on accumulating data, while maintaining trial integrity and validity.

Any untoward medical occurrence in a patient or clinical trial subject administered a pharmaceutical product.

A yearly report submitted to FDA summarizing changes and updates to an approved drug application.

A clinical trial design that tests one investigational therapy across multiple indications or tumor types sharing a common molecular target or biomarker.

A comprehensive document containing all information and instructions for manufacturing a specific batch of drug product.

The ability of a medical device material to perform its intended function without causing adverse biological response in contact with tissue, blood, or other body fluids.

The absence of a significant difference in the rate and extent to which the active ingredient in two pharmaceutically equivalent products becomes available at the site of drug action.

A submission to the FDA requesting approval to market a biological product in the United States.

FDA's program that inspects clinical investigators, sponsors, IRBs, and nonclinical laboratories to verify compliance with applicable regulations.

A biological product highly similar to an FDA-approved reference biologic with no clinically meaningful differences in safety, purity, and potency.

A higher regulatory designation than biosimilarity, requiring demonstration that a biosimilar can be substituted for its reference product without intervention from the prescribing physician.

A clinical trial design technique where participants, investigators, or both are prevented from knowing which treatment each subject is receiving.

The strongest warning that FDA requires in prescription drug labeling, highlighting serious or life-threatening risks.

An FDA designation for drugs showing substantial improvement over existing treatments for serious conditions.

A clinical study conducted to allow extrapolation of efficacy and safety data from one region or population to another.

The FDA center responsible for regulating biological products including vaccines, blood products, and cell and gene therapies.

The FDA center responsible for ensuring the safety and effectiveness of medical devices and radiation-emitting products.

The FDA center responsible for ensuring that drugs marketed in the United States are safe and effective.

The section of a regulatory submission describing a drug's composition, manufacturing process, and quality controls.

Low-risk medical devices subject to general controls and, in most cases, exempt from 510(k) premarket notification.

Moderate-risk medical devices subject to general controls plus special controls, typically requiring 510(k) premarket notification.

Highest-risk medical devices requiring Premarket Approval (PMA) based on clinical evidence of safety and effectiveness.

An FDA order to a sponsor to delay a proposed or suspend an ongoing clinical investigation due to safety concerns or deficiencies.

A regulatory submission to EU or non-US authorities seeking authorization to conduct a clinical trial of an investigational medicinal product.

A therapeutic product combining two or more regulated components (drug, device, biologic) with a primary mode of action determining the lead FDA center.

An internationally harmonized format for organizing pharmaceutical regulatory submissions into five standardized modules.

An analytical method published in an official pharmacopoeia (USP, Ph. Eur., JP) that serves as the recognized standard for testing a specific article.

An FDA letter indicating that an application review is complete but the application is not approved as submitted.

A company that provides outsourced research services to pharmaceutical and biotechnology companies.

The planned set of controls, derived from product and process understanding, that ensures process performance and product quality.

A systematic approach to identifying, investigating, and addressing the root causes of quality problems.

A process parameter whose variability has an impact on a Critical Quality Attribute and therefore must be monitored or controlled to ensure product quality.

A physical, chemical, biological, or microbiological property of a drug substance or product that must be within an appropriate range to ensure quality.

The current quality standards FDA enforces for the manufacture, processing, packing, and holding of drug products to ensure identity, strength, quality, and purity.

The completeness, consistency, and accuracy of data throughout its lifecycle, following ALCOA+ principles.

An independent group of experts that periodically reviews accumulating clinical trial data to monitor participant safety and trial integrity.

A pathway for novel low-to-moderate risk medical devices that lack a predicate but do not require PMA-level review.

A clinical trial where some or all trial activities take place at locations other than traditional research sites, often including patient homes.

Systematic practices applied during product design and development to ensure devices meet user needs and intended uses.

The multidimensional combination of input variables and process parameters demonstrated to provide assurance of quality, enabling operational flexibility within the defined region.

An annual comprehensive safety report submitted by sponsors of investigational drugs to regulatory authorities summarizing the safety profile during the reporting period.

A departure from an approved procedure, specification, or established standard during manufacturing or testing.

A confidential document submitted to FDA containing detailed information about manufacturing facilities, processes, or components.

Inorganic impurities, principally heavy metals, that may be present in drug products and require assessment under ICH Q3D.

Analytical testing to quantify bacterial endotoxins in parenteral drugs, medical devices, and other products where endotoxin presence could cause pyrogenic reaction.

The formal written report FDA issues at the conclusion of a facility inspection documenting findings and classification.

The EU GMP annex establishing requirements for the manufacture of sterile medicinal products.

The EU GMP annex establishing requirements for computerized systems used in GMP-regulated activities.

The European Union agency responsible for the scientific evaluation, supervision, and safety monitoring of medicines.

An inactive substance used as a carrier or vehicle for the active pharmaceutical ingredient in a drug product.

The pathway for patients with serious or immediately life-threatening diseases to access investigational medical products outside of clinical trials.

An FDA program designed to facilitate development and expedite review of drugs treating serious conditions and filling unmet medical needs.

A list of inspectional observations issued by FDA investigators at the end of a facility inspection when conditions may violate the FDCA or related acts.