Contact Information
Registered Office/ Head Office:
Ashford Laboratories Pvt. Ltd.,
31/36, 5th Floor, Dheeraj Heritage,
S. V. Road, Santacruz (West),
Mumbai (Bombay) - 400 054. India.
Tele- fax: + 91 22 6675 8866

Japan Office:
Ashford Laboratories,
4-11-24-1402 Fukae Kitamachi,
Higashi Nada Ku, Kobe 658-0013,
Tele- fax: +81 0784 122579
Title 21, Code of Federal Regulations, Part 11. Part 11 established the criteria under which electronic records and signatures will be considered equivalent to paper records and handwritten signatures in manufacturing processes regulated by the FDA. See Code of Federal Regulations.

483 Letter
See Form 483.

505 (b) (2) submission
A new drug application that contains full reports of investigations of safety and effectiveness but where at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference.

Abbreviated Antibiotic Drug Application (AADA)

A marketing application usually reserved for antibiotics that duplicate products that were previously approved under a full marketing application. Similar to an ANDA.

Abbreviated New Drug Application (ANDA)

Application made to the FDA for a generic version of a branded drug. The duplicate drug must be bioequivalent to the branded version of the drug. The first company to receive FDA approval of an ANDA receives 180 days of market exclusivity for the generic drug. Compare with NDA.

Absorption, Distribution, Metabolism and Excretion/Toxicology (ADME/T)
The objective of ADME/T testing is to measure what happens to a compound in the human physiology. ADME/T tests are done during the preclinical stage of the drug discovery process and are a necessary part of any clinical trial, prior to filing an IND.

Action Letter

An official communication from the FDA to the sponsor of an NDA, regarding a decision before the agency.

Active Pharmaceutical Ingredient (API)
Any substance or mixture of substances intended to be used in the manufacture of a drug product and that becomes an active ingredient of the drug. APIs are intended to cause pharmacological activity or other direct effect in the diagnosis, cure, mitigation, treatment or prevention of disease or to affect the structure and function of the body.

See Absorption, Distribution, Metabolism and Excretion/ Toxicology.


See Adverse Drug Reaction.

Adverse Drug Reaction (ADR)
An adverse drug reaction is an undesirable effect associated with the use of a drug. It may occur as a standard part of the drug's action, or it may be unpredictable.


Agence française de sécurité sanitaire des produits de santé. France’s health regulatory agency.

An agonist is a substance or a drug that can interact with a receptor and initiate a physiological or a pharmacological response characteristic of that receptor.

Amino Acids
Organic compounds consisting of one or more amino groups and a carboxyl group which are the basic building blocks of peptides (i.e. two amino acids is a dipeptide three is a tripeptide etc.) and proteins.

Ampoule (Ampule)
A small glass vial sealed after filling. Ampoules were one of the earliest devices developed for safe storage of a sterile injectable.


See Abbreviated New Drug Application.


An antagonist is a drug or a compound that opposes the physiological effects of another. At the receptor level, it is a chemical entity that opposes the receptor-associated responses normally induced by another bioactive agent.


An organic substance of microbial origin that is either toxic to, or inhibits the growth of other organisms. Examples include penicillin, tetracycline, and erythromycin and semi-synthetic derivatives. Totally synthetic antibiotics (or "antibacterials") also exist, such as the quinolones.


A protein molecule in the blood serum (and other bodily fluids) produced by the immune system in response to exposure to a foreign substance. Antibodies are the body's protective mechanism against infection and disease. An antibody is characterized by a structure complementary to the foreign substance (the antigen that provokes its formation), making it capable of binding specifically to the foreign substance to neutralize it.

A medication used to treat fungal infections such as athlete's foot, ringworm, candidiasis (thrush), serious systemic infections such as cryptococcal meningitis, and others.

Substances capable of inducing a specific immune response and reacting with the products of that response.


Chemotherapeutic reagents produced chemically or via biotechnology and used to treat infections caused by bacteria, fungi, parasites and viruses.

A nucleic acid sequence that complements the coding sequence of DNA or mRNA. An antisense sequence does not code for RNA or protein synthesis, but can match to a coding strand and interfere with its function. Antisense sequences may have therapeutic usage, particularly against some cancers.

An antiseptic agent is one that has been formulated for use on living tissue such as mucous membranes or skin to prevent or inhibit growth or action of organisms.

See Active Pharmaceutical Ingredient.

Aseptic Processing
A method of producing a sterile (devoid of living organisms) product in which sterile bulk drug or raw materials are compounded and assembled with sterile packaging components.

A laboratory test or technique to identify and/or measure the amount of a particular substance in a sample, or for determining characteristics such as composition, purity, activity and weight. Used to determine whether compounds (drugs, chemicals, etc.) have the desired effect either in a living organism, outside an organism, or in an artificial environment. A test method for determining the presence, quantity or efficacy or a test substance.

Asymmetric Synthesis

A synthesis in which one isomer is produced preferentially to others.


An effort to determine through investigation the adequacy of, and adherence to, established procedures, instructions, specifications, codes, and standards or other applicable requirements, as well as the effectiveness of their implementation.

Audit Trail

An automatic feature of some computer programs or operating systems that creates a running record of all transactions.

Authorized Person (AP)

The person recognized by a national regulatory authority as being responsible for ensuring that each batch of finished drug product has been manufactured, tested and approved for release in compliance with the laws and regulations in force in that country. See Qualified Person.

A device used to administer/self-administer a predetermined dose of intramuscular or subcutaneous medication.

Viable small single cell microorganisms without a cell nucleus which proliferate by dividing into two cells.

A specific quantity of material produced in a process (or series of processes) and expected to be homogeneous within specified limits. The batch size may be defined either by fixed quantity or the amount produced in a fixed time interval. In the case of continuous production, a batch may correspond to a defined fraction of the production characterized by its intended homogeneity.


For biotechnological/biological products, a material produced during a manufacturing process that is not the drug substance but which is critical to the successful production of the drug. A bio-intermediate will generally be quantifiable and specifications will be established to determine the successful completion of the manufacturing step before continuation of the manufacturing process.

Bioanalysis (Bioanalytical)

Analytical procedure(s) to quantify results of target analyte(s) in a defined biological matrix.

The determination of the biological activity of a drug by observing its effect on an organism (or organ) compared to a standard preparation.


A measure of the uptake of an ingested substance by the body as assessed by its concentration in the blood. The rate and extent of its appearance in the blood are important determinants of bioavailability.

The level of microorganisms present. For an antimicrobial process, the higher the bioburden, the more aggressive the process must be.

A chemical conversion of a substance by a microorganism or enzyme.

A biological agent such as a microorganism or enzyme that activate or speed up a chemical reaction.

A chemical substance capable of killing microorganisms (i.e. disinfectants, fungicides, bactericides).

A scientific basis on which generic and brand name drugs are compared with one another. Drugs are bioequivalent if they enter circulation at the same rate when given in similar doses under similar conditions. Proof of bioequivalence is crucial for generic drugs, and must be demonstrated in ANDAs.

A biological medicinal product that is pharmaceutically and therapeutically similar or equivalent to licensed/approved product. Sometimes called biosimilar.

The use of computers in the life sciences, electronic databases of genomes and protein sequences, and computer modeling of biomolecules and biologic systems.

According to the FDA's Center for Biologics Evaluation and Research, a biological product is 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 to humans. Biological products include, but are not limited to, bacterial and viral vaccines, human blood and plasma and their derivatives, and certain products produced by biotechnology, such as interferons and erythropoietins.

Biological Pathway
A cellular process serving a specific purpose such as insulin regulation. These sequences of events can reveal the causes of p

Biologics License Application (BLA)

An application to FDA for a license to market a new biological product in the U.S. roblems such as tumor progression and help researchers target particular molecular processes to block with drugs.


The statistical study of biological events.

A biotechnologically produced substance such as proteins, nucleic acids and other naturally occurring compounds derived from living cells for pharmaceutical applications.

A process in which living cells or their components are used to produce a desired product.


Vessel used for bioprocessing. Biosafety in Microbiological and Biomedical Laboratories A document published by the Center for Disease Control that defines the various biosafety levels, practices, procedures, and criteria for which biological agents are required to be managed.


See Biogeneric.

Production of a chemical by a living organism. Also, the route by which the organism synthesizes a chemical.


The industrial use of living organisms (or parts of living organisms) to produce foods, drugs, or other products. The oldest biotechnologies include fermentation and plant and animal hybridization.


Generally used to refer to chemical modification of a drug molecule or xenobiotic by a biologically based process, i.e., drug metabolism, microsomal metabolism, or plasma esterase mediated hydrolysis.

See Biologics License Application.

Blister Pack
A unit dose package commonly constructed from a formed cavity containing one or more individual doses.

Blow (Form), Fill, Seal
Refers to machines that combine formation of a plastic container by blow molding, aseptic filling of a liquid product, and sealing of the final package.

See Biosafety in Microbiological and Biomedical Laboratories.

See Bulk Pharmaceutical Chemical.

British Pharmacopoeia (BP)
The authoritative, current collection of standards for UK medicinal substances and the official source of all UK quality standards. It is used by individuals and organizations involved in pharmaceutical research, development, manufacture and testing.

Bulk Pharmaceutical Chemical (BPC)
A pharmaceutical product derived by chemical synthesis, in bulk form, for later dispensing, formulation or compounding, and filling in a pharmaceutical finishing facility.

Capacity Utilization

The percentage of available capacity used for production.

A class of antibiotics with a broad spectrum of antibacterial activity. Used in hospital settings to treat serious infections.


The potential of any substance to cause cancer.

Case Report Form (CRF)
A record of pertinent information collected on each subject during a clinical trial, as outlined in the study protocol.

See Center for Biologics Evaluation and Research.

See Center for Drug Evaluation and Research.


See Contract Development and Manufacturing Organization.


Smallest viable component of a living organism.

Cell Banks
Uniform pools of preserved cells, typically in vials and frozen in liquid nitrogen.

Cell Culture
A method for growing cells isolated from living organisms under laboratory conditions.

Cell Lines
When cells from a first culture (taken from the organism) are used to make subsequent cultures, a cell line is established.

Cell Therapy

A form of therapy in which cells are grown or manipulated in culture and then administered to a patient to treat a disease.

Cell Culture

A method for growing cells isolated from living organisms under laboratory conditions.

Cell Lines
When cells from a first culture (taken from the organism) are used to make subsequent cultures, a cell line is established.

Cell Therapy
A form of therapy in which cells are grown or manipulated in culture and then administered to a patient to treat a disease.

Center for Biologics Evaluation and Research (CBER)

The FDA successor to the Bureau of Biologics concerned with biologic drugs and with the new protein and peptide drugs arising from biotechnology. According to its charter, CBER is responsible for ensuring
  The safety of the U.S. entire blood supply and the products derived from it
  The production and approval of safe and effective childhood vaccines, including any future AIDS vaccines
  The proper oversight of human tissue for transplantation
  An adequate and safe supply of allergenic materials and anti-toxins
  The safety and efficacy of biological therapeutics, including an exciting new array of biotechnology-derived products used to       treat diseases such as cancer and AIDS.

Center for Drug Evaluation and Research (CDER)

The successor to the Bureau of Drugs of the FDA concerned with all SVPs (Small Volume Parenterals), LVPs (Large Volume Parenterals), and non-biological or small-molecule drugs.


A class of antibiotics with activity against a wide variety of bacterial infections. The most prescribed class of antibiotics.


See Code of Federal Regulations.


See current Good Manufacturing Practice.

Change Control

The processes, authorities for, and procedures to be used for all changes that are made to the computerized system and/or the system's data. Change control is part of the QA program and should be clearly described in SOPs.

Change Order

Process to be defined in the MSA or Supply Agreement where by changes to the Work Order will be initiatied, agreed and approved.

Precisely describing the characteristics of a drug substance that affect its efficacy and its purity. Also, the chemical, physical and biological properties of a specific drug substance.

Chemical Library
A collection of stored chemicals usually used ultimately in high-throughput screening or industrial manufacture. Each chemical has associated information stored in some kind of database with information such as the chemical structure, purity, quantity, and physiochemical characteristics of the compound.

Chemical Process Development

A step in the drug discovery process in which the chemical reactions necessary for the production of a drug are streamlined. Reactions are optimized, scaled up, and sometimes completely new routes are devised.

Chemical Synthesis and Scale-Up

A step in the drug discovery process in which a chemical process is expanded to produce adequate amounts of a potential drug compound for preclinical and clinical testing.


A term that encompasses the design, creation, organization, management, retrieval, analysis, dissemination, visualization and use of chemical information.

Chemistry, Manufacturing, and Controls (CMC)
The section on a BLA, IND, NDA or ANDA describing the composition, manufacture, and specifications of a drug product and its ingredients.

Child Resistant (CR)
A package that will pass a test protocol administered by the U.S. Consumer Product Safety Commission (CPSC).

An organism combining tissues derived from two or more genotypes.

Chinese Hamster Ovaries (CHO)

A type of cell line that can grow continuously in culture and is very widely used for the production of recombinant proteins.

Chiral Compound

Molecules that have the same chemical and structural formulae but differ in the third dimension (spatially). CHO See Chinese Hamster Ovaries


Separation of a mixture of substances by size, charge, or other characteristic by allowing the mixture to partition between a moving phase and a stationary phase.


Chemical packages of hereditary information, made up of long coiled chains of DNA. Humans have 22 different pairs of chromosomes plus either two x-chromosomes or one x and one y-chromosomes depending on the gender.


See Clean In Place

Clean In Place (CIP)

Cleaning involving cleaning equipment without first disassembling the equipment.


A specially constructed space environmentally controlled with respect to airborne particles (size and count), temperature, humidity, air pressure, airflow patterns, air motion, and lighting. ISO 14644-1 defines a cleanroom as "a room in which the concentration of airborne particles is con-trolled, and which is constructed and used in a manner to minimize the introduction, generation and retention of particles inside the room, and in which other relevant parameters -- e.g. temperature, humidity and pressure -- are controlled as necessary."

Clinical Research Associate

Also known as a monitor, a CRA is an individual who oversees the progress and conduct of a clinical trial to ensure the scientific integrity of the data collected, and the protection of the rights, safety and well-being of human study subjects.

Clinical Trials

Carefully designed investigations of the effects of investigational new drugs, medical treatments, or devices on a group of patients. There are typically three phases of clinical trials prior to the filing of an NDA, sometimes followed by ongoing, post-approval studies. See Phase I, Phase II, Phase III, and Phase IV.


A population of organisms produced from a single parent cell where the derived cells are genetically identical.


The maximum concentration of a drug in the body after dosing.

See Chemistry, Manufacturing, and Controls.

The minimum concentration of a drug in the body after dosing.


S 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 Manufactur-ing 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. See also 21 CFR Part 11.

ee Contract Manufacturing Organization.

A triplet of nucleotides in the DNA or RNA molecules that code for one of the 20 amino acids in proteins or as a signal to stop or start protein production.


A gram negative bacterium associated with filth that when present can serve as an indicator of potential fecal contamination, as in polluted water or adulterated food or drug.

Combination Product
According to FDA:
  A product comprised of two or more regulated components, i.e., drug/device, biologic/device, drug/biologic, or      drug/device/biologic, that are physically, chemically, or otherwise combined or mixed and produced as a single entity
  Two or more separate products packaged together in a single package or as a unit and comprised of drug and device      products, device and biological products, or biological and drug products.
  A drug, device, or biological product packaged separately that according to its investigational plan or proposed labeling is      intended for use only with an approved individually specified drug, device, or biological product where both are required to      achieve the intended use, indication, or effect and where upon approval of the proposed product the labeling of the      approved product would need to be changed, e.g., to reflect a change in intended use, dosage form, strength, route of      administration, or significant change in dose; or
  Any investigational drug, device, or biological product packaged separately that according to its proposed labeling is for use      only with another individually specified investigational drug, device, or biological product where both are required to achieve      the intended use, indication, or effect.

Combinatorial Chemistry

An approach to chemical synthesis that creates large numbers of organic compounds by putting chemical "building blocks" together in every possible combination. Combinatorial chemistry is used in the drug discovery process to create many lead compounds that are screened against biological targets.

Commodity Chemical

A chemical produced in high volumes with low value and used for a broad spectrum of applications.


A state of operations that ensures activities are following documented protocols.

Computational Biology

The process of analyzing and interpreting biological data.

Computational Chemistry,

A discipline used for computer-aided drug design. Computer modeling attempts to predict the type of compounds (and the structures) most chemically suitable for binding to a drug target.

Computer Systems Validation
Confirmation by examining and providing objective evidence that computer system specifications conform to user needs and intended uses, and that all requirements can be consistently fulfilled.

Contract Development and Manufacturing Organization
A company that offers both contract drug development and manufacturing services. Comprehensive services range from early-stage R&D services such as formulation development, stability studies and method development all the way through manufacturing services, ranging from preclinical R&D material for clinical trial purposes and commercial production (Phases I-IV).

Contract Manufacturing Organization (CMO)

A company that offers manufacturing services, with volume capabilities ranging from small amounts for preclinical R&D to larger volumes necessary for clinical trials purposes and commercialization.

Contract Research and Manufacturing Services (CRAMS)

Catch-all term for contract services offerings, used predominantly among Indian providers.

Contract Research Organization (CRO)

A company involved in performing clinical research on a contract basis for a pharmaceutical company, research organization, or other health organization. CROs are contracted to perform some or all of the duties by the sponsor for a clinical trial; examples include monitoring the trial, enrolling patients, performing statistical analysis, and writing the protocols.

Contract Service Provider

Organization that provides specific cGMP service(s) according to a written contract and to mutually agreed upon conditions; services provided include manufacturing product, laboratory services, packaging and more.

Controlled Release Formula

A drug formulation that releases the active ingredient at a steady rate. This can be done by a variety of technologies, including liposomes, subcutaneous injections of a polymer matrix, and transdermal patches. Controlled release of a drug can increase patient comfort and compliance by reducing the number of injections or oral doses that must be taken, as well as avoiding the “peak and valley” profile of drug concentration in the blood that is usually seen after a dose of conventional formulations.

Cooperative Research and Development Agreement (CRADA)

A written agreement between a private company and a government agency to work together on a project. The collaborating partner agrees to provide funds, personnel, services, facilities, equipment or other resources needed to conduct a specific research or development effort while the federal government agrees to provide similar resources (but not funds) directly to the partner.


A cosmetic product claimed to have medicinal or drug-like benefits.

COX-2 Inhibitor

A class of anti-inflammatory drug that blocks Cox-2 enzyme activity without impeding the activity of Cox-1. Vioxx is the best-known drug in this class, due to Merck’s withdrawal of it from the marketplace, after a possible link to cardiac episodes was uncovered.


See Child Resistant.


Catch-all term for Contract Research & Manufacturing.


See Clinical Research Associate.


See Cooperative Research and Development Agreement.


See Contract Research and Manufacturing Services


See Case Report Form.


See Contract Research Organization.


The determination and characterization of the structure of crystalline materials, typically through the use of analytical instruments. Two key types are macromolecular crystallography, which crystallizes proteins to discover their structure, and “small molecule” crystallography, which focuses on drugs and pigments. See X-Ray Crystallography.


See Contract Service Provider.

Current Good Manufacturing Practices (cGMP)
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. See also Good Manufacturing Practices.

Any agent or process that kills cells. Includes chemotherapy and radiotherapy.

In sterilization, the amount of energy or the length of kill time at a certain concentration or temperature to lower microbial content one decade or 1-log-10.

Data and Safety Monitoring Board (DSMB)

A group of researchers who periodically review data from blinded, placebo-controlled clinical trials. A DSMB board can stop a trial if toxicities are found or if the treatment is proved beneficial.

Drug Delivery Systems

Closely related compounds. In Structure-Activity Relationship (SAR) work, after a lead compound is identified, derivatives are made to try to maximize activity and minimize toxicity.

Drug Controller General of India. India’s health regulatory body.

Disease Pathway
The set of proteins and their regulators whose activation or inactivation lead to a particular disease. By defining a disease pathway, scientists can learn which genes and proteins contribute to the disease and are therefore potential drug targets.

The abbreviation for deoxyribonucleic acid, which holds the genetic information for all living creatures.

Dosage Form
The form in which the drug is delivered to the patient. Dosage forms include parenteral, topical, tablet, oral (liquid or dissolved powder), suppository, inhalation and transdermal.

Double Blind
A procedure in which the subjects, investigators, and sometimes also the data analysts in a clinical trial are not told of the treatment assignments (experimental vs. control). This is done to prevent bias of the results, because neither the investigator nor investigated know what interaction is being conducted in a clinical study.

Downstream Processing (DSP)

The stages of processing that take place after the fermentation or bioconversion stage; includes separation, purification, and packaging of the product.

Drug Delivery

The process by which a formulated drug is administered to the patient. Traditional routes are oral or intravenous perfusion. New methods deliver through the skin with a transdermal patch or across the nasal membrane with an aerosol spray. Optimized compliance and increased prescription writing tends to occur when agents are developed in ways to ease adverse and uncomfortable effects associated with delivery and systems thereof.

Drug Discovery
Identification of a biological, genetic or protein target linked to a particular disease, and subsequent lead identification of a potential drug that interacts with the target to help cure the disease or halt its progression.

Drug Product
A finished dosage form, for example, tablet, capsule, solution, etc., that contains an active drug ingredient generally, but not necessarily, in association with inactive ingredients.

Drug Product
A finished dosage form, for example, tablet, capsule, solution, etc., that contains an active drug ingredient generally, but not necessarily, in association with inactive ingredients.

See Drug Substance.

See Data and Safety Monitoring Board.


See Downstream Processing.

E-Pedigree (alt. ePedigree)
An electronic document that enables a single view of the product and order and is used for drug authentication. The pedigree goes with the drug and tracks the change of custody as the drug passes through the supply chain.

or electronic common technical document, an FDA term for electronic submission format.

See Electronic Data Capture.

Measures the power to produce, in a controlled setting such as a clinical trial, a stated effect typically attributable to a known physiologic phenomenon. It is important to derive measures of efficacy because, with appropriate statistical achievements, these data can approximate real-world effectiveness.

See Environmental, Health and Safety.

Electronic Data Capture (EDC)
Collection of data in an electronic format, typically used in reference to data obtained from clinical trials.

See European Agency for the Evaluation of Medicinal Products.

Molecules with the same empirical formula that are mirror images of each other.

A complex lipopolysaccharide molecule shed from dead gram negative bacteria that when present in process water or a pharmaceutical or device product can produce a pyrogenic reaction when injected.

In clinical trials, a parameter used to compare the results in different arms of the trial. Endpoints may be directly related to the condition (such as progression of the disease) or may be measurements of surrogate markers.

Engineering Batch
Batches which are manufactured prior to full scale cGMP batches. Used to fine-tune processes before committing to full scale manufacture.

Environmental Health and Safety (EHS)
Intra-company collections of policies, systems and programs to protect the aforementioned environment, assure worker safety and exhibit responsible behavior.

European Agency for the Evaluation of Medicinal Products (EMEA)
The European FDA, the EMEA is responsible for protecting public health by mobilizing scientific resources within the European Union, promoting health care through the regulation of new pharmaceuticals, and supporting the pharmaceutical R&D industry by developing efficient, effective and responsive operating procedures.

European Pharmacopeia (EP)
Official compendium of the member states of the Council of Europe, including all EC and European Free Trade Association (EFTA) countries. It lists a wide range of active substances and excipients used to prepare pharmaceutical products in Europe.

A more or less inert substance added in a prescription drug compound. Excipients serve as diluents or vehicles or to give form or consistency when the remedy is given in a pill form. Examples include simple syrup, aromatic powder and honey. Also known as Inactive Pharmaceutical Ingredient.

The formation of proteins or peptides in a cell by transcription of the genetic information and its translation into an amino acid sequence.

An antibody fragment that contains the regions that bind to antigens.

See Fluorescence-Activated Cell Sorter.

Fast Track
Designation given by the FDA to programs that are designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast track emphasizes the critical nature of close early communication between the FDA and sponsor, procedures such as pre-IND and end of Phase I meetings as methods to improve the efficiency of preclinical and clinical development, and focuses on efforts by the FDA and sponsor to reach early agreement on the design of the major clinical efficacy studies that will be needed to support approval.

See Food and Drug Administration.

The FDA Modernization Act of 1997 reauthorized the collection of user fees by the FDA and amended the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act to improve the regulation of food, drugs, devices and biological products, and to facilitate the development and evaluation of new drugs designed to treat serious and life-threatening illnesses.

Field Alert
Any incident that causes the drug product or its labeling to be mistaken for or applied to another article, bacterial contamination, a significant chemical, physical, or other change, deterioration in the distributed drug product, and failure of one or more distributed batches of the drug product to meet the specifications established in its application.

Fill and Finish
Some secondary elements of the drug manufacturing process. Includes formulation, vial preparation, filling, capping and inspection.

Fluorescence-Activated Cell Sorter
A machine that can rapidly separate the cells in a suspension on the basis of size and the color of their fluorescence.

Food and Drug Administration
The U.S. federal agency charged with promoting and protecting the public health by helping safe and effective products reach the market in a timely way and monitoring products for continued safety after they are in use. Founded in 1938.

Form 483
The official form of notification prepared at the conclusion of an inspection (without review by FDA management) listing observations of perceived violations of Good Manufacturing Practices that may constitute violations of law in the opinion of an inspector. Originally intended to inform companies of possible product adulteration, they must be replied to satisfactorily and/or corrective action taken in order to alleviate any offensive notification and avoid action from the FDA. Form 483s can lead to withholding of product approvals and plant shut-downs, may come into play in due diligence phases of acquisitions and mergers, and can p

Form, Fill, Seal
See Blow, Fill, Seal. otentially cost companies money.

Freeze Dry and/or Freeze Dryer
See Lyophilization and Lyophilizer

See Full-Time Equivalent.

Full-Time Equivalent
A way to measure a worker's involvement in a project. An FTE of 1.0 means that the person is equivalent to one full-time employee, while an FTE of 0.5 signals that the worker is only half-time.

Functional Genomics
Applying genomic information to determine gene function, commonly using microarrays and model organisms. An important part of target validation.

See Good Automated Manufacturing Practice.

Gas Chromatography (GC)
A process by which the components of a mixture are separated by volatilizing the sample into a carrier gas stream and passing the gas through a column containing a substance that retains (absorbs) and releases the volatile constituents.

See Good Clinical Practice.

A natural unit of hereditary material that is the physical basis for the transmission of the characteristics of living organisms from one generation to another. The basic genetic material is fundamentally the same in all living organisms. It consists of deoxyribonucleic acid (DNA) in most organisms and ribonucleic acid (RNA) in certain viruses, and is usually associated in a linear arrangement that constitutes part of the chromosome.

Gene Therapy
According to the FDA's Center for Biologics Evaluation and Research, gene therapy is the use of normal genes or genetic material to replace or cancel out the "bad" or defective genes in a person's body that are responsible for a disease or medical problem. In early studies of gene therapy for cancer, researchers were trying to improve the body's natural ability to fight the disease or to make the tumor more sensitive to other kinds of therapy. This treatment may involve the addition of a functional gene or group of genes to a cell by gene insertion to correct a hereditary disease.

Generic Drug
A drug product sold under the provisions of Section 505(j) of the Federal Food, Drug and Cosmetic Act. The Abbreviated New Drug Application (ANDA) was enabled through the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman). The ANDA must contain
  information to show conditions of the proposed generic drug's labeling have been previously approved for the "reference      listed drug" (RLD),
  that the generic drug is pharmaceutically equivalent to the RLD (same active ingredient, route of administration, dosage      form and strength),
  that the generic drug is bioequivalent to the RLD,
  the generic drug must have the same labeling as the RLD, and
  the generic drug must be manufactured in compliance with cGMPs. Finally, with regard to patents, the sponsor of a generic      drug application must certify to the FDA that (I) information on any patents were not filed by the NDA holder with FDA, (II)      relevant patent(s) have expired, (III) relevant patent(s) will expire on some future date, or (IV) relevant patent(s) are invalid or      would not be infringed by the manufacture of a generic version.

Genetic Engineering
Refers to all methods used for the isolation of DNA molecules of a living organism and their analysis, modification and introduction into a cell.

A genome is all of the DNA in an organism, which includes genes and a great deal of DNA that does not carry genetic code (known as introns and exons). Each animal or plant has its own unique genome. Genetic DNA is the molecular code that carries information for making all the proteins required by a living organism.

The study of genes and their function. Genomics has revealed many new biological targets for the development of drugs and has given scientists innovative ways to design new drugs, vaccines and DNA diagnostics. Genomic-based therapeutics may include small molecule drugs, biologics and gene therapy.

See Good Laboratory Practice.

The addition of a carbohydrate (sugar) residue to a protein often essential for biological activity.

See Good Manufacturing Practice.

GMP Facility
A production facility or clinical trial materials pilot plant for manufacturing drug products or APIs. It includes the manufacturing space, the storage warehouse for raw and finished product, and support lab areas, as well as laboratories that are independent of production facilities. A GMP facility operates under the guidelines established by the CFR (Code of Federal Regulations) Title 21, Parts 210 and 211 (cGMP in Manufacturing, Processing, Packing or Holding of Drugs and Finished Pharmaceuticals), Part 225 (Current Good Manufacturing for Medicated Feeds - Subpart B), and Part 226 (Current Good Manufacturing Practice for Type A Medicated Articles - Subpart B).

Good Automated Manufacturing Practices
A forum that exists to promote the understanding of the regulation and use of computer and control systems within the pharmaceutical manufacturing industry.

Good Clinical Practice (GCP)
The international ethical and scientific quality regulations for designing, conducting, monitoring, recording, auditing, analyzing and reporting studies. It insures that the data reported is credible and accurate, and that the subject’s rights and confidentiality are protected. Refers to 21 Code of Federal Regulations Parts 50 and 56.

Good Laboratory Practice (GLP)
A set of rules and criteria for a quality system concerned with the organizational process and the conditions under which non-clinical health and environmental safety studies are planned, performed, monitored, recorded, archived and reported. The GLP principles have been developed to promote the quality and validity of data generated in the testing of chemicals in order to facilitate their recognition for purposes of assessment and other uses relating to the protection of human health and the environment. U.S. GLPs are found in 21 CFR Part 58.

Good Manufacturing Practice (GMP)
A set of principles and procedures which, when followed by manufacturers of drugs and other therapeutics, helps ensure that the products manufactured will be of the required quality. GMP is based on the premise that quality cannot be tested into a batch but must be built into each batch of product during all stages of manufacturing. See also current Good Manufacturing Practice.

Good Tissue Practice (GTP)
Regulations that govern methods used in, and facilities used for manufacturing human cellular and tissue-based products. Current Good Tissue Practice (cGTP) requirements are less extensive in scope than current Good Manufacturing Practice (cGMP). cGTP requirements are limited to preventing circumstances concerning the introduction, transmission and spread of communicable disease. cGTPs are intended to assure that products do not get contaminated during manufacturing, and that product function and integrity are not impaired through improper manufacturing processes.

GS Expression System
A proprietary highly efficient mammalian expression system (glutamine synthetase) combining the use of selection via glutamine independence with a powerful promoter to allow the rapid creation of highly productive cells.

See Good Tissue Practice.

Amount of time necessary for 50% of a drug to be eliminated from the recipient’s system.

Height Equivalent to the Theoretical Plate. Numerically equal to column length divided by the number of theoretical plates in the column. The HETP is the theoretical link between the Plate Theory and the Rate Theory as the HETP is numerically equal to the variance per unit length of the column as determined from the Rate Theory. As the HETP is a function of both the properties of the column and the solute, it will vary from one column to another and, more importantly, between different solutes eluted from the same column in the same chromatogram. See Plate Theory and Rate Theory.

High Pressure Liquid Chromatography (HPLC)
A separation technique based on a solid stationary phase and a liquid mobile phase. Separations (into distinct bands) are achieved by partition, adsorption, or ion-exchange processes, depending upon the type of stationary phase used. Each band is then profiled as the solvent flows through a UV detector, or by fluorescence, or refractive index detectors. Sometimes called high-performance liquid chromatography.

High-Throughput Screening (HTS)
A process in which batches of compounds are tested for binding activity or biological activity against target molecules. High-throughput screening seeks to evaluate large numbers of compounds rapidly and in parallel. Compounds are tested as inhibitors of target enzymes, as competitors for binding of a natural ligand to its receptor, and as agonists or antagonists for receptor-mediated intracellular processes.

HIPAA stands for the Health Insurance Portability and Accountability Act. This law, passed by Congress in 1996, helps to protect citizens' rights to health coverage during events such as changing or losing jobs, pregnancy, moving, or divorce. It also provides rights and protections for employers when getting and renewing health coverage for their employees.

A positive result from high throughput screening. Compounds resulting in hits are collected for further testing in which further potency or effectiveness may be determined. If hits demonstrate further potency, they become lead compounds.

Host Range
The number of cell cultures or animals that can be infected by a virus.

Host System
A cell or organism used for the expression of foreign DNA.

See High Pressure Liquid Chromatography.

See High Throughput Screening.

The engineering of an antibody protein sequence to make it more human like and less antigenic during therapeutic use.

A cell line resulting form the fusion of a continuously dividing cancer cell with a normal blood cell secreting a specific antibody to produce an immortal hybridoma cell line manufacturing a specific antibody or monoclonal antibody.

See International Conference on Harmonization.

Abbreviation for immunoglobulins: gamma globulin proteins that are found in blood or other bodily fluids, and are used by the immune system to identify and neutralize foreign objects, such as bacteria and viruses.

In silico
Test performed in a computer model.

In vitro
Literally, “in glass.” Test performed in a test tube or other laboratory apparatus.

In vivo
Test performed in a living organism.

Obtaining the right from another company to develop, produce and commercialize a particular compound.

Inactive Pharmaceutical Ingredient
See Excipient.

See Investigational New Drug.

Infectious Diseases
Diseases caused by bacteria and viruses which can be transmitted from one person to another (i.e. cholera, typhoid, HIV, influenza).

The study of the application of computer and statistical techniques to the management of information. In genome projects, informatics includes the development of methods to search databases quickly, to analyze DNA sequence information, and to predict protein sequence and structure from DNA sequence data.

Installation Qualification (IQ)
Documented verification that all key aspects of hardware installation adhere to appropriate codes and approved design intentions and that the recommendations of the manufacturer have been suitably considered.

Proteins secreted in human and other animals during viral infections that have antiviral and immunological properties.

International Conference on Harmonization (ICH)
Full name: The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. ICH brings together the regulatory authorities of Europe, Japan and the U.S. and experts from the pharmaceutical industry in the three regions to discuss scientific and technical aspects of product registration. The purpose is to make recommendations on ways to achieve greater harmonization in the interpretation and application of technical guidelines and requirements for product registration in order to reduce or obviate the need to duplicate the testing carried out during R&D. Also known as International Committee on Harmonization.

International Organization for Standardization (ISO)
Non-governmental organization responsible for developing international standards, founded in 1947. ISO’s 13,700 standards are voluntary and market-driven. The ISO 9000 series has become an international reference for quality requirements in business-to-business dealings, and the ISO 14000 standards address environmental issues.

Investigational New Drug Application (IND)
An application to the FDA to begin clinical trials of a new drug or biologic on humans. The IND gives the plan for the study and contains formulation, manufacturing and animal test result information.

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted with a team of individuals at a trial site, the investigator is the leader of the team and may also be called the principal investigator.

See Installation Qualification.

Japanese Pharmacopoeia (JP)
A listing of a wide range of active substances and excipients used to prepare pharmaceutical products in Japan.

Joint Services Document
The JSD is a living document agreed upon by the company and CSP that facilitates the implementation of the Commercial Supply and Quality Agreements. The goal of this document is to coordinate and manage the exchange of information, the movement of materials and documents, the resolution of issues, and the ongoing evaluation of performance. The purpose of this JSD is to explain how the company and CSP will work together to manage the logistics of the relationship. This document is intended to serve as a reference manual that describes the specifics around who, what, when, and how of the day-to-day interactions.

See Joint Services Document.

kilogray: a unit of irradiation administered to a drug or raw material from a cobalt-60 or e-beam reactor for the purpose of lowering bioburden or producing sterility.

Laboratory Information Management Systems (LIMS)
Software that is used in the laboratory for the management of samples, laboratory users, instruments, standards and other laboratory functions such as invoicing, plate management, and work flow automation.

Large Volume Parenteral (LVP)
A parenteral product packaged in a volume of 100 ml or more.

See Liquid Chromatography/Gas Chromatography.

Liquid Chromatrography/Mass Spectrometry involving sequential ionization/fragmentation.

Chemical species that have migrated from packaging or other components into the dosage form under normal conditions of use or during stability studies. Leachables are substances identified in a defined laboratory regimen by simulating use conditions. Leachables are a subset of Extractables or can be related to reaction or degradation products.

A compound with validated biological activity, both in primary and secondary screens, against known targets.

Lead Compound
A peptide or small molecule that can bind to a target and bring about a therapeutic effect. A successful lead compound will become a drug candidate for clinical trials. See Derivative.

Lead Discovery
The process of identifying a lead compound from a pool of candidates. Lead candidates may be discovered randomly using high-throughput screening techniques or structure-based drug design, which is intended to streamline the process.

Lead Optimization
The process of creating the most advantageous lead compound in terms of its binding affinity for the discovery and production of drugs.

Legacy Systems
Hardware and software applications in which a company has already invested considerable time and money. Legacy systems typically perform critical operations in companies for many years even though they may no longer employ state-of-the-art technology. See 21 CFR Part 11.

Molecule that binds to a receptor protein.

See Laboratory Instrument Management Systems.

An artificial phospholipid vesicle. Liposomes can be useful for the enclosure of macromolecules such as nucleic acids or after loading with an appropriate drug. They may be used therapeutically to achieve slow release of the drug into circulation. See drug delivery.

Liquid Chromatography/Gas Chromatography (LC/GC)
Chromatography involves a sample (or sample extract) dissolved in a mobile phase (a gas, a liquid, or a supercritical fluid). The mobile phase is then forced through an immobile, immiscible stationary phase. The phases are chosen so that components of the sample have differing solubilities in each phase. As a result of these differences in solubilities, sample components will become separated from each other as they travel through the stationary phase. This process, in addition to separating components, also permits analysis. See also High Pressure Liquid Chromatography.

Liquid Chromatography/Mass Spectrometry (LC/MS)
An analytical chemistry technique that combines the physical separation capabilities of liquid chromatography with the mass analysis capabilities of mass spectrometry. Tends to be used for the specific detection and potential identification of chemicals in the presence of other chemicals in a complex mixture.

A batch, or a specific identified portion of a batch, having uniform character and quality within specified limits; or, in the case of a drug product produced by continuous process, it is a specific identified amount produced in a unit of time or quantity in a manner that assures its having uniform character and quality within specified limits

See Large Volume Parenteral.

Also known as freeze drying, lyophilization is a means of stabilizing wet substances by freezing them, then evapor-ating the resulting ice, to leave a substantially dry, porous residue which has the same size and shape of the original frozen mass.

An apparatus used to freeze dry or lyophilize products. Units are capable of freezing products, applying a vacuum and removing water by sublimation.

See Monoclonal Antibody.

Mass Spectrometer
An analytical instrument that measures the mass of charged particles or ions. Mass spec works only on molecules that are charged or ionized; therefore, most biological samples must be subjected to conditions inside the spectrometer that cause the individual molecules to ionize.

Master Cell Bank
A single pool of cells or microorganisms prepared from a selected homogenous population of cells, parent cells, distributed into vials and preserved under defined conditions (usually at -80°C or below). A vial is thawed to grow sufficient cells to create a larger number of vials of the working cell bank from which these vials are used to initiate new production batch.

Master Service Agreement
An umbrella contract between two companies which defines the terms of business, under which work-orders can be incorporated. Enables more agile execution. Must be integrated with the Quality Agreement and often includes Quality Agreement terms and definitions.

Medicinal Chemistry
A field of study that combines organic chemistry, analytical chemistry and other chemical disciplines for the discovery, optimization, and production of drugs. See Structure-Activity Relationship.

Medium (pl. Media)
A formulation of nutrient substances used in the culture of microorganisms or cell cultures.

Metabolic Induction
The effect of a one drug, chemical or biochemical on another such that the biotransformation of the article of interest is enhanced.

Metabolic Inhibition
The effect of a drug, chemical or biochemical on another such that the biotransformation or excretion of the article of interest is slowed or impeded.

Metabolic Transformation
Chemical modification of a substrate molecule by a metabolic process.

Metered-Dose Inhaler (MDI)
A device that allows delivery of medicine directly into the lungs. The medicine is in the form of a very, very fine powder, and a propellant is used to get the powder out in a cloud to be inhaled.

Methicillin Resistant Staphylococcus aureus
A virulent strain of the organism resistant to several antibiotics, including synthetic penicillins, tetracyclines, macrolides, but sensitive to older drugs such as vancomycin and the sulfonamides.

Methods Validation
Establishing, through documented evidence, a high degree of assurance that an analytical method will consistently yield results that accurately reflect the quality characteristics of the product tested.

Japan's Ministry of Health, Labour and Welfare.

Medicines and Healthcare products Regulatory Agency. The UK's health regulatory body.

Monoclonal Antibody (MAb)
A laboratory-engineered antibody, derived from a single cell, that recognizes and targets a specific antigen.

See Methicillin Resistant Staphylococcus aureus.

See Master Service Agreement.

The ability to cause a change in DNA structure.

National Formulary
An official compendium providing standards and specifications to evaluate the quality of pharmaceuticals and therapeutic agents

See New Chemical Entity.

See New Drug Application.

Formation of any type of tumor, benign or malignant.

New Chemical Entity (NCE)
Any new molecular compound (excluding diagnostic agents, vaccines, and other biologic compounds) not previously approved for human use by CDER. New Drug Application (NDA)
An application to FDA for a license to market a new drug in the U.S. Sponsor companies submit NDAs after completing clinical trials on a new drug.

Non-Research Development Organization (NRDO)
A company that attempts to acquire and develop drugs that have already been discovered. Also known more descriptively as “No Research – Development Only”.

Nucleic Acids
Long chained molecules (DNA and RNA) that encode and control synthesis of proteins in living organisms

Structural component of DNA and RNA.

A medicinally or nutritionally functional food

Occupational Exposure Limit
A generic term used to represent a pair of numbers: (1) the agent concentration or intensity that is allowable (based on health-effects data) and (2) the time period over which one averages workplace concentrations to evaluated whether the measured concentrations are less than the allowable limit. Some substances may have several occupational exposure limits (e.g., one for eight hours, one for 30 minutes, and a not-to-exceed ceiling).

See Occupational Exposure Limit.

Off-Label Use
The use of a drug in a way neither approved by the FDA nor permitted to be put on its label and advertised as its intended purpose. Once a drug is approved by the FDA, physicians are free to prescribe it for any indication they see fit.

Short chains of nucleotides which have been linked together in a specific number and configuration.

The potential of a virus to change DNA and RNA.

Operational Qualification (OQ)
Documented verification that the equipment related system or subsystem performs as intended throughout representative or anticipated operating ranges.

See Operational Qualification.

Oral Solid Dosage
Hard gelatin capsules, tablets, troches (lozenges); the preferred method of drug delivery.

Orange Book
An FDA-issued list of all approved prescription and OTC drug products. The list includes indications of "equivalency" for generic drugs, and also contains pertinent patent information.

Orphan Drugs
Drugs developed for rare diseases and conditions which, in the U.S., affect fewer than 200,000 people or which, in the EU, affect five or fewer per 10,000 people. Because sales of orphan drugs are likely to be small compared to their development costs, pharmaceutical companies are awarded exclusive rights to market these medicines for a period of time (usually seven years) as an incentive to develop them and bring them to market.

See Over the Counter.

Out of Specification (OOS)
OOS results include those that fall outside the specifications or acceptance criteria established in New Drug Applications (NDAs), official compendia, or by the manufacturer.

Selling the rights of a developed products or potential compound to another firm for further development, production or marketing.

Out-of-Trend (OOT)
OOT results occur when a stability result does not follow the expected trend, either in comparison with other stability batches or with respect to previous results collected during a stability study.

Over the Counter (OTC)
Drug that can be purchased without a prescription.

See Pre-Approval Inspection.

Parenteral Drug
A drug intended for injection through the skin or other external boundary tissue, rather than through the alimentary canal (oral), so that active substances they contain are administered directly into a blood vessel, organ, tissue, or lesion. They are infused when administered intravenously, or injected when administered intramuscularly, or subcutaneously into the human body. See LVP, SVP.

See Process Analytical Technology

A grant made by the government giving the creator of an invention the sole right to make, use and sell the invention for a set period of time. In exchange for the temporary monopoly granted under a patent, the inventor is obligated to disclose the nature of the invention such that others may apply that knowledge upon expiration of the patent.

See Polymerase Chain Reaction.

See Prescription Drug User Fee Act.

A class of antibiotics similar to carbapenems. No penems are available in the U.S.; Replidyne's faropenem was rejected by the FDA and the company sold itself shortly after. Faropenem was intended to treat respiratory and other community infections. See Carbapenem.

A group of antibiotics that comprised the first effective treatment against a number of hithero-untreatable diseases. First discovered in 1928 and moved into wide use in the early 1940's, this class is one of the most widely used antibiotics.

A molecule consisting of between two and 20 amino acids connected by peptide bonds; a short segment of a larger protein or a completely functional molecule.

Pharmacodynamics (PD)
Study of the reactions between drugs and living structures. This covers a compound's pharmacologic effect on patients, including the study of uptake, movement, binding and interactions of agents at their tissue and cellular site(s) of action.

Branch of economics that applies cost-benefit, cost-effectiveness, cost-minimization and cost-utility analyses to compare the economics of different pharmaceutical products or to compare drug therapy to other treatments. Sometimes referred to as outcomes research.

The hereditary determinants of how individuals metabolize drugs.

The application of genomics to the discovery and development process for drugs. This use of genetic information can predict the safety, toxicity and/or efficacy of drugs in individual patients or groups of patients. Genomics may aid drug development by defining specific receptive subpopulations of patients, thus simplifying and focusing the clinical trial process. Refined models of disease mechanism based on knowledge of the genome may provide new lines of research and possibly new drugs.

Pharmacokinetics (PK)
The movements of drugs within biological systems, as affected by uptake, distribution, elimination and biotransformation. See also ADME/T.

The study of how drugs produce their effects. Pharmacology relies on knowledge of physiology, biochemistry, molecular biology, and other scientific disciplines.

The official compilation of medicinal substances, with descriptions, tests and formulas for preparing them, selected by a recognized authority. The pharmacopoeia issued for a country is the legal standard of that nation.

The monitoring of the quality, safety and efficacy of marketed medicines.

Phase 0
An exploratory clinical study to gain a better understanding of drug parameters.

Phase I
Initial safety studies in humans. May include as few as 10 subjects, often in healthy volunteers, and includes PK, ADME/T and dose escalation studies to determine some side effects. Usually open label.

Phase II
Following initial safety (Phase I) testing, a drug is tested for efficacy, typically in blind, randomized trials, in which a control group receives a placebo. Phase II testing may last from several months to two years. Phase II trials involve 100-300 subjects with the disease or condition of interest. Includes PK, dose ranging, safety and efficacy.

Phase III
Following Phase II testing, a drug is tested in a large-scale setting (several hundred to several thousand patients) to determine effectiveness, benefits, and the range of possible adverse reactions. Most Phase III studies are randomized and double blinded, and typically last several years. Usually, two well-controlled studies are necessary to establish efficacy. Once Phase III trials are successfully completed, a pharmaceutical company can request FDA approval for marketing the drug, by filing a New Drug Application (see NDA).

Phase IV
Following FDA approval and marketing, drug companies may conduct further studies on their products. These post-approval studies have several objectives, including comparing the drug with other drugs already in the market, monitoring the drug's long-term effectiveness, and determining additional potential uses for the drug.

Description of the overall characteristics of an organism it morphology, physiology and behavior.

Pilot-Plant Scale
The production of drug substance or drug product at a scale intermediate between the laboratory and the full-scale manufacture.

See Pharmacokinetics.

A mock treatment or drug that has no effect on the illness, given in a clinical trial to the control group to help differentiate the specific versus nonspecific effects of an experimental treatment. Is generally made to appear identical to the active drug.

Independent self-replicating DNA molecules commonly found in bacterial cells. Plasmids and their genetic information are inherited as part of the hosts genome but are separate from chromosomes.

Plate Theory
A method of describing chromatography behavior. It postulates that the chromatographic column contains a large number of separate layers, called "theoretical plates." Separate equilibrations of the sample between the stationary and mobile phase occur in these "plates". The analyte moves down the column by transfer of equilibrated mobile phase from one plate to the next. Note: the plates don't actually exist. See Rate Theory and HETP.

Polymerase Chain Reaction (PCR)
Technique used to quickly increase the amount of a specific DNA sequence or to detect the existence of a defined sequence within a particular DNA sample.

Post-translational Modification
Describes changes that may occur to a protein in a cell after translation has taken place to produce a defined amino acid sequence. A common example is glycosylation the addition of sugar molecules to the protein.

Potent Compound
A pharmacologically active ingredient or intermediate with biological activity at approximately 15 micrograms per kilogram of body weight or below in humans or a daily therapeutic dose of 1 mg or below per day; or an active pharmaceutical ingredient or intermediate with an occupational exposure limit (OEL) at or below 10 micrograms per cubic meter of air as an eight-hour time weighted average; or a pharmacologically active ingredient or intermediate with high selectivity (as in the ability to bind to specific receptors or inhibit specific enzymes) and/or the potential to cause cancer, mutations, developmental effects or reproductive toxicity at low doses; or a novel compound of unknown potency and toxicity.

See Process Qualification.

Pre-Approval Inspection (PAI)
An inspection of an establishment that performs manufacturing steps in an approved BLA or NDA and for which the applicant has submitted a supplement for a significant manufacturing change or other change that ordinarily requires on-site review of the change.

Preclinical studies
Studies that test a drug on animals and in other nonhuman test systems. Safety information from such studies is used to support an Investigational New Drug (IND) application.

The utilization of biopharmaceutical principle to determine the physicochemical properties of a drug substance. Through the process of choosing the substance's best form, evaluating its physical properties and understanding its stability under various conditions, one can develop an optimal drug delivery system.

Prescription Drug User Fee Act (PDUFA)
In 1992, Congress passed the PDUFA, authorizing the FDA to collect fees from companies that produce certain human drug and biological products. Any time a company wants the FDA to approve a new drug or biologic prior to marketing, it must submit an application along with a fee to support the review process. In addition, companies pay annual fees for each manufacturing establishment and for each prescription drug product marketed. In exchange for industry fees, the FDA was to meet drug-review performance goals, which emphasize timeliness.

Primary Packaging
Primary packaging refers to packaging that immediately envelopes a product. It provides most of the strength and the moisture, vapor or grease barrier needed to safeguard a product's purity, potency and integrity from the time it leaves the assembly line until it's used by the consumer.

Process Analytical Technologies (PAT)
According to the FDA, Process Analytical Technologies are systems for analysis and control of manufacturing processes based on timely measurements of critical quality parameters and performance attributes of raw and in-process materials and processes to assure acceptable end product quality at the completion of the process. PATs involve optimal applications of process analytical chemistry tools, feedback process control strategies, information management tools, and/or product/ process optimization strategies to the manufacture of pharmaceuticals.

Process Qualification (PQ)
Documented verification that the process and/or the total process related system performs as intended throughout all anticipated operating ranges.

Process Validation
Establishing, through documented evidence, a high degree of assurance that a specific process will consistently yield a product that meets predetermined specifications and quality characteristics.

A large, complex molecule composed of amino acids. The sequence of the amino acids, and thus the function of the protein, is determined by the sequence of the base pairs in the gene that encodes it. Proteins are essential to the structure, function and regulation of the body. Examples are peptide hormones, enzymes and antibodies.

The set of proteins encoded by a genome. That is, the collection of proteins found in a cell.

The study of gene expression at the protein level, by the identifying and characterizing proteins present in a biological sample. By comparing samples from healthy and diseased tissues, it is possible to identify proteins that are specific to diseased cells. These may be potential diagnostic markers for particular diseases, or targets for drug development. Unlike the genome, which is constant regardless of cell type, the proteome varies in each cell type.

A plan that sets guidelines for a trial and usually involves several different trial locations. A protocol is usually designed by the sponsor of a clinical trial.

See Quality Assurance

See Quality by Design

See Quality Control

Qualification batches
Qualification batches are batches that are manufactured in order to qualify a process, API, or excipient according to a protocol that was developed and authorized.

Qualified Person (QP)
A Qualified Person is a technical term used in EU pharmaceutical regulation. The regulations specify that no batch of medicinal product can be released for sale or supply prior to certification by a QP that the batch is in accordance with the relevant requirements. The QP is typically a licensed pharmacist, biologist or chemist (or a person with another permitted academic qualification) who has several years experience working in pharmaceutical manufacturing operations, and has passed examinations attesting to his or her knowledge. In countries that are part of the Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme (PIC/S), the same role may be termed Responsible Person (RP) or Authorized Person (AP).

Quality Agreement
A contract between two companies which is to record the quality standards for goods or services that are purchased through a Supply Agreement

Quality Assurance (QA)
The procedures established to ensure that a product is manufactured, or a clinical trial is performed, in compliance with the appropriate standards and regulatory requirements, and that the process or results are properly documented.

Quality by Design
A two-fold program to [1] develop a pharmaceutical, biologic or medical device to meet predefined product quality, safety and efficacy specifications; and [2] design the manufacturing processes and controls around that pharmaceutical, biologic or medical device to meet predefined product quality, safety and efficacy specifications. Quality by Design begins in the preclinical, research and development phase, and carries forward through post-market and into retirement of the product. Non-medicinal influences that conceptualized Quality by Design started in the 1970’s in the automotive, telecommunications and aeronautics industries.

Quality Control (QC)
Checking or testing that specifications are met, or the regula-tory process through which the industry measures actual quality performance, compares it with standards, and acts on the difference.

Quantitative Whole Body Autoradiography
A method of detecting and visualizing radioactivity concentration in an intact test subject.

Radio Frequency Identification (RFID)
RFID tags are small integrated circuits connected to an antenna, which can respond to an interrogating RF signal with simple identifying information, or with more complex signals depending on the size of the circuits.

Rate Theory
A method of describing chromatography behavior. It uses the process of peak dispersion (band spreading) and provides an equation that allows the calculation of the variance per unit length of a column (the height of the theoretical plate, HETP) in terms of the mobile phase velocity and other physical chemical properties of the solute and distribution system. See Plate Theory and HETP.

Rational Drug Design
Using the known three-dimensional structure of a molecule, usually a protein, to design a drug molecule that will bind to it. Usually viewed as an alternative to drug discovery through screening many molecules for biological activity.

A protein or group of associated proteins in a cell or on its surface that selectively binds a specific substance (called a ligand). Upon binding its ligand, the receptor triggers a specific response in the cell. See Ligand.

Registration batches
Three commercial scale consecutive batches that are manufactured and placed on stability which are used as input for the NDA.

See Risk Evaluation and Mitigation Strategy.

To introduce a product back into the process and repeat steps that are part of the established manufacturing process. Continuation of a process step after an in-process control test shows the process to be incomplete is not considered reprocessing. Further, reprocessing does not include subjecting a product to one or more processing steps that are different from the established manufacturing process

Request For Information (RFI)
A formal request made by potential contract clients for information regarding the capabilities of a contract manufacturer. Used as a preliminary screen for potential contractors. Is more general in nature and does not include a request for a price of the services requested.

Request for Proposal (RFP)
A request for the manufacturer or service provider to submit a proposal supported by cost breakdown. The RFP provides a description of the items to be procured or services to be provided. It may include specifications, quantities, time and place of delivery, method of shipment, packaging and instruction manual requirements, materiel to be furnished, and data requirements, both support and administrative.

Request for Quotation (RFQ)

Collection and testing of a new sample from the batch. Resampling is conducted if the investigation concludes that the original sample was prepared improperly and was therefore not representative of the batch.

Responsible Person (RP)
See Qualified Person

Repeated testing of a portion of the orginial sample. The sample is typically taken from the same homogeneous material used for the original test.

See Radio Frequency Identification.

See Request for Proposal.

Risk Evaluation and Mitigation Strategy (REMS)
A strategy to manage a known or potential serious risk associated with a drug or biological product.

The abbreviation for ribonucleic acid. It forms a complex family of biochemical molecules some of which carry a copy of the genetic information during the process of gene expression to make a particular protein molecules. In other words, “DNA makes RNA makes protein.”

See Structure-Activity Relationship.

See Summary Basis of Approval.

Transition from small-scale production to production of large commercial quantities.

Scale-Up and Post-Approval Changes (SUPAC)
The FDA-recommended testing and filing actions to be taken by a pharmaceutical firm when it changes the manufacturing processes of a drug product that has already been approved via an NDA or ANDA.

Secondary Metabolism
The part of metabolism which is not essential for growth or maintenance.

Secondary Metabolites
Products or secondary metabolism of microorganisms and plants. Most pharmaceuticals, antibiotics are secondary metabolites or derivatives of secondary metabolites.

Secondary Packaging
Material used primarily to give additional physical protection to the outside of a proximity package.

Single Nucleotide Polymorphism (SNP)
A SNP is a place in the genetic code where DNA differs from one person to the next by a single letter. These slight genetic variations among human beings may predispose some people to disease and explain why some respond better to certain drugs.

Small Molecule Drug
This is a natural or synthetic drug which is not a protein, or a large molecular weight or polymeric material.

Small Molecule Library
A collection of prepared small molecule compounds. Such libraries are maintained in order to facilitate screening and target validation. Small molecules interacting favorably with a target may become drug compound candidates, and so the target may become validated.

Small Volume Parenteral (SVP)
A "catch-all" for all non-LVP parenterals products, except biologicals. See LVP.

see Standardized Measurement of Particulate Airborne Concentration.

See Supplemental New Drug Application

See Single Nucleotide Polymorphism.

Solid Dosage
Capsules, tablets, suppositories.

See Standard Operating Procedures.

Special Protocol Assessment (SPA)
A procedure by which the FDA provides official evaluation and written guidance on the design and size of proposed protocols that are intended to form the basis for a new drug.

Specialty Pharma
Meant to differentiate companies that market drugs for highly specific markets, in contrast with the broad approach of Big Pharma companies. This term is rapidly becoming overused to the point of meaninglessness, as many non-Big Pharma companies have taken to calling themselves Specialty Pharmas

Refers to the physico-chemical condition of a parenteral, biological, or shelf life of other drugs. Certain drugs must pass USP or CFR stability tests. Manufacturers must have documentation of potency of products under labeled storage conditions.

Standard Operating Procedures
The description of activities necessary to respond to normal and abnormal situations in an operating system. SOPs may include a troubleshooting checklist, list of personnel to contact, etc. They should also describe normal operation, maintenance, and cleaning of the system, as well as normal operating parameters.

Standardized Measurement of Particulate Airborne Concentration
A committee that is made up of an independent group of professionals that has development the ISPE Good Practice Guide on Assessing the Particulate Containment Performance of Pharmaceutical Equipment.

Any of several drugs known to inhibit the activity of the cholesterol synthetic enzyme, 3-hydroxy-3-methylglutaryl-Coenzyme A (HMG CoA) reductase. Examples include cholesterol drugs cerivastatin (Baycol) and simvastatin (Zocor).

Structural Genomics
The generation of three-dimensional structural information about proteins and the use of that information, using advanced computational methods, to predict compounds capable of interacting with the proteins.

Structure-Activity Relationship (SAR)
The relationship between chemical structure and pharmacological activity for a series of compounds.

Structure-Based Drug Design
The process of drug discovery that uses a high-resolution picture of the drug target's structure. This can help develop a profile of the compound that would be the most suitable for that target.

Summary Basis of Approval
Documentation on which a regulatory approval (for marketing) is based.

See Scale-Up and Post-Approval Changes.

Supplemental New Drug Application (sNDA)
Application submitted for an already approved NDA for any changes in packaging, labeling, dosages, ingredients or new indications.

Supply Agreement
A contract between two companies which is to record obligations to sell and buy quantities of goods or services over time. If the purchaser of the goods or services is restricted as to where or to whom the goods may be sold, the arrangement is referred to as a distribution Agreement.

Sustained Delivery
A drug delivery system that releases API during an extended period of time. Also known as sustained release.

See Small Volume Parenteral.

Target Product Profile
A summary of the attributes of the intended commercial product.

Target Validation

Technology Transfer
The process of moving inventions, patents, or processes from one site to another. The process of demonstrating that a drug interacting with a given target could have a therapeutic effect.

Terminal Sterilization
Sterilization of a product at the final stage of production. The desire for increased levels of sterility assurance has led the FDA to promote the use of terminal sterilization for aseptically filled products. The FDA has stated that terminal sterilization processing is the method of choice unless the manufacturer can show that it is detrimental to the product.

Therapeutic Goods Administration. Australia’s drug regulatory body.

Compounds that are used to treat specific diseases or medical conditions.

Topical formulations are formulations made to be applied onto skin. Topical formulations include pharmaceuticals, skin care, and cosmetic products. Products can be formulated in various consistencies including creams, lotions, and serums. Product base can consist of oil, water, oil-in-water emulsion, and water-in-oil emulsion.

See Target Product Profile.

The process by which genetic information contained in DNA is transcribed or copied into a molecule of RNA.

Transdermal systems deliver drugs directly through the skin and into the bloodstream, offering an alternative to traditional delivery methods. Patches are used in several therapeutic areas, including pain management, smoking cessation, treatment of heart disease, hormone replacement, and management of motion sickness.

A process for transferring recombinant genetic information into a recipient cell or host organism.

Transgenic Organism
Transgenic animals or plants are organisms living with an altered gene or genes as a result of genetic engineering.

Altering the genes of an animal or plant so that it expresses a specific, engineered protein. The protein can then be purified from its expression medium (for example, the milk of a cow) and used to create biologic drugs.

The process by which information is transferred from DNA by RNA specifying the amino acid sequence in a protein.

Transmissible Spongiform Encephalopathy (TSE)
A neurological disease in mammals of many species believed to be caused by prions.

Ultra Performance Liquid Chromatography
Version of liquid chromatography that uses columns packed with sub-2 nanometer particles for greater speed, resolution and sensitivity. "UPLC" is a registered trademark of Waters Corporation, but is also used to describe the process generally.

United States Pharmacopeia (USP)
A non-government, not-for-profit organization that promotes the public health by establishing standards to ensure the quality of medicines and other health care technologies. These standards are developed by a publicly involved process and are accepted worldwide. USP provides standards for more than 3,400 prescription and non-prescription drugs, nutritional and dietary supplements, veterinary drug standards, and health care products. These standards are published in the United States Pharmacopeia and the National Formulary (USP-NF).

See Ultra Performance Liquid Chromatography.

See United States Pharmacopeia.

A substance that closely resembles a particular disease-causing agent, to the body's immune system. The similarity primes the immune system to recognize the agent and fight against it.

Validated Target
A drug target, usually a protein, that has been demonstrated by any of a variety of methods to have therapeutic potential.

The establishment of documented evidence (for example, data derived from rigorous testing) that provides a high degree of assurance that a specific process or system will consistently yield a product meeting predetermined specifications and quality attributes.

Validation batches
Validation batches are commercial scale batches that are manufactured consecutively in order to validate a process according to a validation protocol that was developed and authorized.

A general term for a number of essential substances the human body cannot synthesize itself.

Warning Letter
An FDA post-audit letter notifying the manufacture of adverse inspections findings requiring a reply with defined plans for remediation.

Water For Injection (WFI)
Water purified by distillation or by reverse osmosis, so that it contains no added substance. WFI meets the purity requirements under Purified Water (USP). Although not intended to be sterile, it meets a test for a limit of bacterial endotoxin.

See Water For injection.

Working Cell Bank
A cell bank made form a single vial of the master cell bank.

World Health Organization (WHO)
The agency of the United Nations founded in 1947 for the purpose of promoting health.

X-Ray Crystallography
A technique used to determine protein structure in which proteins in a crystalline form are exposed to X-rays. Advanced computer programs analyze the patterns of the X-rays bending through the crystals to create a picture of the protein in three dimensions.

A compound foreign to an organism.