Navigating the regulatory landscape can be challenging if you’re not familiar with all the terms. Find a list of some of the key regulatory terms, processes, acronyms, and their definitions below.
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A
An application seeking the FDA’s approval of a generic drug product. These types of applications do not generally need to include preclinical and clinical data, but instead must demonstrate that the product performs in the same manner as the innovator drug.
Also known as the drug substance, the API is the biological molecule that is responsible for the action or efficacy of the drug.
An international standards organization that establishes voluntary consensus technical standards for a wide range of materials, products, systems, and services.
A type of immunotherapy that’s either a gene therapy medicinal product, a somatic cell therapy medicinal product, or a tissue engineered product.
B
The marketing approval application (MAA) that’s filed in the US for the commercial manufacturing of biologics. This application contains specific data on the manufacturing processes, chemistry, pharmacology, clinical pharmacology, and medical effects of the biologic product.
A written record that documents all the instructions to be followed when manufacturing pharmaceuticals, as well as history of a product batch. It includes information like the product name, weight, and count of each component in the medication. This is also sometimes referred to as production batch record (PBR) or master batch record (MBR).
The levels of biocontainment precautions needed to isolate dangerous biological agents in an enclosed facility. The four levels are BSL-1, BSL-2, BSL-3, and BSL-4, with BSL-4 requiring maximum containment.
C
How to maintain compliance and achieve operational excellence in your quality system through a formalized process to document, address, and eliminate causes and recurrence of non-conformances or other deviations of the quality management system (QMS).
The center within the FDA that regulates over-the-counter and prescription drugs, including biological therapeutics, biological products, and generic drugs.
The center within the FDA that regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs.
A service provider for biotherapeutics and small molecule development and manufacturing.
A database including a codification of the general and permanent rules published in the Federal Register by the Executive departments and agencies of the Federal Government. Title 21 of the CFR is reserved for rules of the Food and Drug Administration, including electronic signatures.
An alternative way of referring to good manufacturing practice (GMP).
Two examples of advanced therapy medicinal (ATM) products, often spoken about together. Cellular therapy is the administration of living whole cells into a patient to treat a disease. Gene therapy seeks to alter either the expression of a gene or the biological properties of living cells for therapeutic use.
A property or characteristic of an input (raw) material that should be kept in an appropriate range to ensure the desired product quality. A CMA can be physical, chemical, biological, or microbiological. Examples: ligand densities and purity/impurity levels in iron carriers used to make cell culture media.
This term can refer to a major part of the ‘quality’ module within the common technical document (CTD). This section is there to ensure consistency within the manufacturing process, and it should contain all relevant data on the active pharmaceutical ingredient (API), the drug product, and the placebo (if used).
A document provided in manufacturing that reports on the quality assessment of the production lot.
A process parameter whose variability has an impact on a CQA and therefore should be monitored or controlled to ensure the process produces the desired quality.
A physical, chemical, biological, or microbiological property or characteristic that should be within an appropriate range to ensure the quality of the product.
Defines the overall scope of what is needed to qualify facilities, systems, and utilities in order to ensure they are GMP ready. This can be referred to as just the qualification master plan.
Defines the overall scope of what is needed to qualify facilities, systems, and utilities in order to ensure they are GMP ready. This can be referred to as just the qualification master plan.
The regulatory application format used internationally to seek new drug approval. This must contain all information relevant to demonstrate that the proposed clinical trial can be conducted safely as planned, and later on, that the drug will be safe and effective for market.
A special emergency program created by the FDA (US) for possible coronavirus therapies. This uses every available method to get new treatments to patients as quickly as possible, whilst also establishing whether they are helpful or harmful.
The extensive electronic or paper document where the CTA (or IND in the USA) is organized, which is submissible within all regions and to all regulatory bodies.
D
A systematic method to determine the relationship between factors affecting a process and the output of that process. It is used to find cause-and-effect relationships between multiple factors across various levels. The DoE approach minimizes the number of experiments by varying several parameters at the same time.
A submission to the Food and Drug Administration (FDA) that may be used to provide confidential detailed information about facilities, processes, or articles used in the manufacturing, processing, packaging, and storing of one or more human drugs.
A documented verification process that checks whether the design of the facilities, systems, and equipment is fit for intended use.
E
An electronic submission of the common technical document (CTD).
The European Union’s decentralized agency that is in charge of evaluating and supervising medical products for use in the European Union.
The Food and Drug Administration’s (FDA) ESG is an agency-wide solution for accepting electronic regulatory submissions. The FDA ESG enables the secure submission of premarket and post-market regulatory information for review.
F
Tests performed at the vendor’s test facility (factory) to verify that all the specifications for newly manufactured and packaged equipment have been met.
A federal agency of the Department of Health and Human Services (US) that protects and promotes public health through the control of various areas of manufacturing.
The first tests conducted in humans as part of the regulatory process. This will occur in clinical Phase I.
G
Internationally recognized ethical and scientific quality requirements that must be applied when designing, conducting, recording, and reporting clinical trials involving people. The ICH provides guidance on this.
The standard to follow when recording raw data entries in a legible, traceable, consistent, and reproducible manner.
A quality system concerned with the organizational process and the conditions under which preclinical health and environmental safety studies are planned, performed, monitored, recorded, archived, and reported.
The quality regulations system designed to minimize the risks involved in any manufacturing process that cannot be eliminated through testing the final product.
The part of quality assurance that adequately controls the storage of pharmaceutical products.
The specific requirements a company outlines for its warehouse, storage and shipping facilities, and departments.
An all-encompassing term that refers to the various types of good practice, including good laboratory practice (GLP), good manufacturing practice (GMP), good clinical practice (GCP), good documentation practice (GDP), and more.
I
A brief description of the product being manufactured, including a summary of the animal studies and any clinical evidence. This is common to all countries and is used by the physician leading the trial to capture what has been learned about the drug.
A committee that joins regulatory authorities such as the FDA and EMA and the pharmaceutical industry together to discuss the scientific aspects of pharmaceutical development and registration.
Allows the investigational device to be used in a clinical study in order to collect safety and effectiveness data. (Investigational use also includes clinical evaluation of certain modifications or new intended uses of legally marketed devices.) An approved IDE means that the independent review board (IRB) has approved the sponsor’s study application.
The specific documentation used to seek new drug approval from regulatory bodies in the EU and UK. This will use the clinical trial application (CTA) format.
The specific documentation used to seek new drug approval from regulatory bodies in the US. This will use the clinical trial application (CTA) format.
An exit to an investment whereby the company sells its shares to public shareholders, resulting in liquidity for shareholders.
A documented verification process that the equipment and its systems have been properly delivered, installed, and configured.
A group formally designated by an institution to review biomedical research involving human subjects, in order to assure the protection of the rights, safety, and welfare of human subjects.
A not-for-profit association that leads scientific, technical, and regulatory advancement throughout the whole pharmaceutical product life cycle.
An independent, non-governmental international organization composed of representatives from various national standards organizations. Together, they establish voluntary, consensus-based standards.
M
The end approval sought in the regulatory process so that the drug can be sold commercially using an approved manufacturing process.
A written record that documents all the instructions to be followed when manufacturing pharmaceuticals, as well as the history of a product batch. It includes information like the product name, weight, and count of each component in the medication. This is also sometimes referred to as a product batch record (PBR) or batch master record (BMR).
The process by which a molecule functions to produce a pharmacological effect. Knowing MOA of a drug may help provide information about the safety of the drug and how it affects the body. It may also help identify the right dose of a drug and which patients are most likely to respond to treatment.
N
This application will be submitted to the FDA once the sponsor believes they have obtained enough evidence on the drug's safety and effectiveness to meet the FDA's requirements. If approved, the product may be marketed in the United States.
Describes the operating range around the target operating conditions that contain common operational variability in the process, for example due to uncontrolled factors.
The difference between the present value of cash inflows and outflows over a period of time.
O
A series of tests checking that all equipment and associated systems will work within their defined operational specifications.
The organization within CBER (FDA) that oversees development of biological
products including cellular and gene therapy products.
P
An acceptable parameter range for a process parameter that will produce drug of the right quality when all other parameters are kept constant. The range is independent of any other CPP.
A system for designing, analyzing, and controlling manufacturing through timely measurements (i.e., during processing) of critical quality and performance attributes of raw and in-process materials and processes, with the goal of ensuring final product quality. It is important to note that the term analytical in PAT is viewed broadly to include chemical, physical, microbiological, mathematical, and risk analysis conducted in an integrated manner.
A written record that documents all the instructions to be followed when manufacturing pharmaceuticals, as well as the history of a product batch. It includes information like the product name, weight, and count of each component in the medication. This is also sometimes referred to as batch master record (BMR) or master batch record (MBR).
A detailed diagram showing the piping and process equipment with the instrumentation and control devices, including the sequences of branches, reducers, valves, and control interlocks.
The realization of a certain idea to demonstrate its feasibility.
Part of process qualification that produces commercial batches by combining the actual facility, utilities, equipment, and trained personnel with the commercial manufacturing process, control procedures, and components.
A documented collection of tests that confirm whether the finished product or process meets all release requirements for functionality and safety, and that the procedures can be reproduced.
Testing to verify the process performs as intended. A report is then generated to go into the CMC section of the common technical document (CTD) to demonstrate the manufacturing processes are validated.
Q
Any systematic process of determining whether a product or service meets specified requirements. QA establishes and maintains set requirements for manufacturing reliable products.
A systematic approach to pharmaceutical development and manufacturing which aims to ensure quality by applying statistical, analytical, and risk-management methodology in product design, development, and manufacturing.
A system of maintaining standards in manufactured products by testing a sample of the output against the specification.
A system of maintaining standards in manufactured products by testing a sample of the output against the specification.
An alternative way of referring to the current qualification master plan (CQMP).
An overall master set of processes and regulations for how a business is run, including areas such as ensuring a product has the appropriate strength, purity, quality, and other requirements.
Within European markets and the UK, a QP stands for a qualified person and is a specific role that must be appointed to release pharmaceutical batches. The QP is a registered job and the QP signs off based on that registration.
The systematic process for assessing, communicating, and reviewing risks to the quality of a pharmaceutical product across its life cycle.
The description of the drug profile (including for example, the type of drug, the dosage form and design, dosage strength) that needs to be achieved to ensure drug quality, safety, and efficacy.
R
An installation providing an enclosed, but not closed, environment meeting defined cleanroom conditions by use of a rigid-wall enclosure and an air overspill that separates the interior from the environment.
The way a substance enters the body.
S
Tests performed at the buyer’s (site) location to verify that all the equipment and associated systems meet the requirements and standards.
A set of instructions outlined by an organization for workers to carry out routine operations, as part of the overarching QMS.
An agreement that indicates concurrence by the FDA, with the adequacy and acceptability of specific critical elements of overall protocol design (for example, entry criteria, dose selection, endpoints, and planned analyses) for a study intended to support future marketing.
T
A collection of documents and images related to the clinical trial that must be stored and maintained in order to comply with governmental regulations.