Glossary

In this glossary, you can find the meaning/definition of the concepts used throughout this course. Feel free to go back to it in case you need to clarify a particular word.

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

A

ADMET – Absorption, distribution, metabolism, excretion, and toxicity

• A drug has to reach the intended target(s), elicit the desired functional response with no or limited toxicity and be eliminated from the body (typically via the liver or kidneys). These are critical properties of the drug candidates that are commonly investigated at various stages of the process.

ANDA

• Abbreviated New Drug Application (USA)

API – Active Pharmaceutical Ingredient (sometimes also Active Pharmaceutical Intermediaries) formerly “Raw Material”

• Any substance or mixture of substances intended to be used in the manufacture of a drug (medicinal) product and that, when used in the production of a drug, becomes an active ingredient of the drug product. Such substances are intended to furnish pharmacological activity or other direct effect in the diagnosis, cure, mitigation, treatment, or prevention of disease or to affect the structure or any function of the body. (ICH Q7)

B

Batch Number

• A unique combination of numbers and/or letters which specifically identify a batch or lot and from which the production and distribution history can be determined. Sometimes referred to as a “Lot Number”.

Biologics

• “Any virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product… applicable to the prevention, treatment, or cure of diseases or injuries of man…”.

Biopharmaceuticals

• Ethical pharmaceutical drugs derived through bioprocessing.

Biotechnology

• A process of applying genetic engineering (recombinant DNA), hybrid (monoclonal antibody), hybridization (gene probes), bioelectric, etc. to commercial applications in pharmaceutical, chemical, medical diagnostic device, food, animal and plant industries.

B.P. (British Pharmacopeia)

• A compendium of testing and purity criteria for pharmaceuticals, ancillaries, and raw materials.

C

Calibration

• A comparison of a measurement standard or instrument of unknown accuracy to detect, correlate, report, or eliminate by adjustment of any variation in the accuracy of the unknown standard or instrument.

Carcinogen

• A substance that causes the development of cancerous growths in living tissue. A chemical is considered to be a carcinogen if it has been evaluated by the International Agency for Cancer Research (IARC) and found to be a carcinogen or potential carcinogen, or if it is listed in the Annual Report on Carcinogens published by the National Toxicology Program.

CFR (Code of Federal regulations) Title 21

• The U.S. regulations that directly apply to biopharmaceutical development are contained in Title 21 parts 58 (Good Laboratory Practice for Nonclinical Laboratory Studies), 210 (Current Good Manufacturing Practice in Manufacturing, Processing, Packing, or Holding of Drugs; General), 211 (Current Good Manufacturing Practice for Finished Pharmaceuticals), and 600 (Biological Products: General). Parts 50 (Protection of Human Subjects), 56 (Institutional Review Boards), and 312 (Investigational New Drugs) apply to critical trials. Part 11 provides criteria which will consider electronic records to be equivalent to paper records and electronic signatures to be equivalent to traditional handwritten signatures.

cGMPs (current Good Manufacturing Practices)

• Current accepted standards of design, operation, practice, and sanitization. The FDA is empowered to inspect drug-manufacturing plants in which drugs are processed, manufactured, packaged, and stored for compliance with these standards.

Certificate of Analysis (CoA)

• A listing of the results found on analysis of a sample, taken by a defined procedure, of a batch of Active Substance [Active Pharmaceutical Ingredient (API)], Drug Product, or other material. The CoA will also include the tests performed with the associated methods and specifications and the results of those tests.

Change Control

• A formal system by which qualified representatives of appropriate disciplines review proposed or actual changes that might affect a validated process’s status. The intent is to determine the need for action that would ensure that the system is maintained in a validated state.

Clinical Trials

• Testing of INDs (Investigational New Drugs) in human subjects to prove safety and efficacy prior to the drug’s approval for marketing. The investigation of a previously untested drug is generally divided into three phases:
  1. Phase I: Introducing the product (or drug) into a small number, generally 20 to 80, patients or healthy volunteers to determine the drug’s metabolism, pharmacological actions, and side effects associated with increasing doses.
  2. Phase II: introducing the product (or drug) into a small number, generally no more than several hundred, patients with the disease or condition under study to evaluate the effectiveness of the drug, common short-term side effects and risks associated with its use.
  3. Phase III: Introducing the product (or drug) into several hundred to several thousand subjects. Studies are expanded controlled and uncontrolled trials performed after preliminary evidence suggesting effectiveness of the drug has been obtained. If the results of the Phase III Clinical Trials are favorable, then the FDA will normally license the drug for manufacture and sale. This phase is usually performed using double blind studies with a placebo and the actual drug.
  4. Phase IV: Ongoing testing studies conducted after the drug is approved. This is done to ensure the long-term efficacy of the drug, detect any long-term beneficial and/or detrimental side effects, and to determine additional potential uses for the drug.

Cross-Contamination

• Contamination of a material or of a product with another material or product (ICH Q7).

D

Drug Candidate

• A molecule suitable for clinical testing. The molecule is expected to bind selectively to a target involved in the disease process, to elicit the desired functional responses in vivo, often in animal models of the human disease, to have adequate bioavailability and bio-distribution within the body to reach the intended target and to pass formal toxicity evaluation in various in vitro and animal models.

Drug formulation

• The process in which the therapeutic agent is combined with different substances to produce a final medicinal product (e.g., a tablet, infusion solution, etc.). Formulation optimization is ongoing throughout pre-clinical and clinical stages. It ensures drugs are absorbed into the body and delivered to the proper organ at the right time and in the right amount.

Drug Product

• The dosage form in the final immediate packaging intended for marketing (ICH Q7).

E

E.P. (European Pharmacopeia)

• A compendium of testing and purity criteria for pharmaceuticals, ancillaries, and raw materials.

Excipient

• Substances other than API which have been appropriately evaluated for safety and are intentionally included in a drug delivery system.

Expiry date

• The date designating the time during which the material is expected to remain within specifications if stored under defined conditions, and after which it should not be used.

F

FDA

• Food and Drug Administration

Final Bulk Product

• The final drug product after chemical or biological processing and purification, ready for concentration, drying, and filling into containers prior to dispensing and final filling.

Finished Product

• A medicinal product that has undergone all stages of production, including packaging in its final container.

First Expired, First Out principle concept (FEFO)

• A distribution procedure that ensures that the stock with the earliest expiry date is distributed and/or used before an identical stock item with a later expiry date is distributed and/or used; earliest expiry/ first out (EEFO) has a similar meaning.

G

GDP – Good Distribution Practice

• Set of guidelines published by the MHRA and the EU in relation to the storage and distribution of pharmaceutical and related products.

Gene

• Genes are macromolecules, made up of deoxynucleic acid (DNA) bases. In humans, genes vary in size from a few hundred DNA bases to more than 2 million bases and about 20,000 to 25,000 genes have been identified.

Generic Drug

• A drug produced and marketed under its chemical or “generic” name (e.g. acetaminophen) as opposed to “Tylenol”, a brand name for the former produced by Johnson & Johnson. A generic drug can be sold only after a proprietary drug goes off patent (i.e. when the patent runs out after 17 years). There are numerous generic drug manufacturers. While generic drugs are cheaper for consumers, they still must meet the standards of GMPs as set out by the FDA.

GLP

• Good Laboratory Practice

GMP

• Good Manufacturing Practice

GTP

• Good Transportation Practice – written by Pharmafreight to demonstrate a series of guidelines, based on GDP, on how to handle pharmaceuticals when shipping on an international basis.

GxP

• The general description for the family of terms like GMP, GDP etc (i.e. A collective term used to refer to the regulations and guidances governing the research, development, testing, and manufacturing of drugs, medical devices, and biologics.

H

HS Tariff code

• Harmonised System tariff code – a numerical code assigned by customs regimes around the world to identify specific types of product.

I

IBC (Intermediate Bulk Container)

• A container for storing, transporting, and handling dry materials. Normally bigger than ½ cubic meter but smaller than 3 cubic meters, dust free, able to receive and discharge a variety of materials, and capable of automation.

Intermediate

• Partly processed material which must undergo further manufacturing steps before it becomes an excipient or an API.

J

K

L

Labelling

• The action involving the selection of the correct label, with the required information, followed by line-clearance and application of the label.

Lot Number

• See batch number

M

Medical Devices

• Any health care product that does not achieve its principal intended purposes by chemical action in or on the body or by being metabolized. The term “devices” also includes components, parts, or accessories of medical devices, diagnostic aids such as reagents, antibiotic sensitivity disks, and test kits for in vitro diagnosis of diseases and other conditions.

Medicinal Product

• Any substance or combination of substances presented for treating or preventing disease in human beings or animals. Any substance or combination of substances that may be administered to human beings or animals with a view to making a medical diagnosis or to restoring, correcting or modifying physiological functions in human beings or in animals is likewise considered a medicinal product.

MSDS (Material Safety Data Sheet)

• Document describing the chemical and physical properties of a substance as related to its safe handling and storage. The substance manufacturer originates it.

N

New Drug Application (NDA)

• The New Drug Application contains most of the information included in the IND. Only after FDA approval of the NDA, can distribution and marketing of a new drug begin.

NDC

• National Drug Code, required for imports to the USA of most pharmaceutical products.

O

Off-target activity

• Action of a drug on targets other than the intended biological target. Such events commonly contribute to adverse effects or toxicity, however, in some cases, off-target activity can be valuable for therapeutic purposes.

On-target toxicity

• A drug is usually designed to interact with its intended target. In some situations, the drug induces exaggerated and adverse pharmacological effects at the target of interest. This is commonly referred in the literature to as on target toxicity.

P

Patent

• A patent is an exclusive right granted by the governments for an invention. Patents give an inventor (academic group or a private company) the exclusive right to prevent others from making, using, selling, or importing a product or process based on the patented invention without the inventor’s prior permission, such as through a patent license.

Pharmaceutical

• A medicinal drug, or relating to or engaged in pharmacy or the manufacture and sale of pharmaceuticals. A pharmaceutical product is generally one that is made up using available chemical compounds.

Peptides

• Peptides are short chains of amino acids. They can be modified to include non-natural amino acids (up to hundreds of atoms). Very short and modified peptides can behave like small molecules while longer peptides (e.g., insulin which is used to manage diabetes) fit in the category of biologics. At present, there are very few approved peptides that can be given by oral route but important work is ongoing in this field to enable oral delivery.

Proteins

• Proteins are large biomolecules (up to thousands of atoms) that comprise one or more chains of amino acids. Proteins perform a vast array of functions within organisms, often through interactions with other macromolecules. Proteins are products of genes; they generally fold into a specific three-dimensional (3D) structure that determines their activities. There are different types of proteins with different functions and locations in the body.

Proteomics

• Proteomics is the process of separation and characterization of all the proteins of a biological system. Target identification with proteomics can be performed by comparing the protein expression levels in normal and diseased tissues.

PK

• Pharmacokinetics is the time-concentration profile of drugs administered in vivo to living organisms. PK parameters include clearance, volume of distribution, peak plasma concentration.

PD

• Pharmacodynamics refers to the relationship between drug concentration at the site of action and the resulting effect, including the time course and intensity of therapeutic and adverse effects. PD parameters include minimum effective concentration, maximum safe concentration, onset of action, therapeutic range and therapeutic index. PD describes how biological processes in the body respond to or are impacted by a drug.

Q

QC(Quality Control)

• Checking or testing, that specifications are met, or the regulatory process through which the industry measures actual quality performance, compares it with standards, and acts on the difference.

R

Regulatory Affairs

• Drug companies must show that their products consistently meet standards set by government agencies. Regulatory affairs departments document those activities, submit proposals, and follow those proposals through completion or approval.

Retest Date

• The date when the material should be re-examined to ensure that it is still suitable for use. (ICH Q7)

S

Side effects

• Secondary unwanted effects that occurs due to the drug therapy. Side effects are usually known and patients are informed about such effects.

Small molecules

• Any organic compound with around 80–100 atoms. Most are made synthetically (aspirin), while others can be derived from natural product (morphine).

Solvent

• An inorganic or organic liquid used as a vehicle for the preparation of solutions or suspensions in the manufacture of an intermediate or API (Active Pharmaceutical Ingredient).

Specification

• A list of tests, references to analytical procedures, and appropriate acceptance criteria that are numerical limits, ranges or other criteria for the tests described for a material.

T

Targets or drug targets

• A therapeutic target is in general a macromolecule (typically a protein), which may cause or be associated with a particular disease, that can be modulated by a therapeutic agent in a measurable way.

Traceability

• Ability to determine the history, application or location that is under consideration, for example, origin of materials and parts, possession history or distribution of the product after delivery.

U

U.S.P. (United States Pharmacopeia)

• A compendium of testing and purity criteria for pharmaceuticals, ancillaries, and raw materials.

V

W

X

Y

Z