Library

Glossary

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Acquis Communautaire 

The acquis communautaire comprises the common set of laws and acts adopted by the European Union since the outset. It is, in some way, the contract to which the EU-25 Member States subscribe and represents an estimated total amount of 80,000 pages legislation.

Active Substance

An active substance (AS), is the substance in a medicinal product that is biologically active

Active substance of biotechnological origin

A product that contains an active substance of biological or biotechnological origin

Adherence

The extent to which the patient follows medical instructions and takes prescribed medicines according to treatment recommendation. In developed countries, adherence to long-term therapies in the general population is around 50% and much lower in developing countries.

ADR - Adverse Drug Reaction

A response to a drug which is noxious and unintended and which occurs at doses normally used in man for prophylaxis, diagnosis or therapy for diseases or for modification of physiological function.

Advanced Therapy IMP (ATIMP) Advance Therapy Investigational Medicinal Products

are medicinal products involving cell or gene therapy or tissue engineering.

Adverse Event

Any untoward medical occurrence in a patient or clinical trial subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.

Amino acids

the chemical building blocks of proteins. At least 20 different amino acids occur in nature.

Animal modeling

Refers to a laboratory animal useful for medical research because it has specific characteristics that resemble a human disease or disorder. Scientists can create animal models, usually laboratory mice, by transferring new genes into them.

antibodies

y_shaped proteins that are part of the human immune system. They bind to foreign substances in the body and thereby mark them for destruction.

Antimicrobial resistance

The use of anti-microbial medicines (i.e. any substance used to kill or inhibit the growth of micro-organism, including antibiotics and other antibacterials, antiviral, antifungal and antiparasitical agents) has greatly contributed to improvements in health, in treating communicable diseases and preventing infections. However, overuse and misuse of antibiotics (especially in children with respiratory infections) have favoured the growth of resistant organisms. The emergence and spread of antimicrobial resistance has become a major public health problem, within the EU and world-wide.

apoptosis

Greek noun for programmed cell death. Cells whose genetic material is irrevocably damaged commit suicide to protect the rest of the body from the effects of the genetic alteration.

ATC

Anatomic, therapeutic, chemical. International system for classification of medicines maintained by World Health Organisation.

ATC/DDD

Anatomical Therapeutic Clinical Classification/Defined Daily Dose

Attrition Rate

Refers to the rate of new chemical entities that are dropped out of the three phases of clinical trials. For example out of 100 new chemical entities, 25% will drop out at phase I, of the 75 that remain for phase II, 52% would drop out. Of the 36 that remain for phase III, 36% would drop out that phase, leaving about 23 of the orginal 100 new chemical entities that actually made it through the process.
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Benchmarking

Benchmarking is the search for industry best practices that will lead to superior performance.

Bioequivalence study

Bioequivalence is a term in pharmacokinetics used to assess the expected in vivo biological equivalence of two preparations of a Medicinal Product.

Bioinformatics

computer_assisted analysis of biological data by special databases, applications and programmes.

Biologic

Biologics include a wide range of medicinal products such as vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins created by biological processes (as distinguished from chemistry).

Biological similarity

The principles of biological similarity have not been adequately defined. Previous studies have not been fully successful, mainly because the search for such principles centered around the idea that they would apply to all animals, just as NewtonÕs principles of mechanical similarity apply to all inanimate objects. However, this is not possible.

Biomarker

a substance used as an indicator of a biologic state.

Biotechnology

Biotechnology is used in two ways: first it describes the traditional biological methods which living organisms use to produce or modify chemical compounds (e.g. antibiotics and vitamins). Secondly, it includes gene technology, which is based on the ability to isolate, replicate, cut and recombine DNA and to transfer DNA from one cell to another. Organisms whose genetic material has been altered in a way that does not occur naturally are called genetically modified organisms (GMOs).

Blockbuster

Refers to a drug which generates over $ 1 billion in global sales.

Bolar

In the US, the "Roche versus Bolar"" amendment to the Watchman-Hatch Act (1984) allows generic medicines manufacturers to use a patented product, before its patent expires, in the preparation of their marketing authorisation application. This amendment was part of the political balance reached when agreeing on measures to restore effective patent protection in the USA. In Europe, this balance was reached by limiting, in the SPC regulation, the effective marketing authorisation to a maximum of 15 years. Introducing a Bolar provision (also called ""early working"") in Europe would go against this equilibrium."
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Cadreac - Collaboration Agreement between Drug Regulatory Authorities in European Union Associated Countries

Informal voluntary co-operation of drug regulatory authorities of accessing countries, represented by their directors, that should help to prepare the area of drug regulation for accession to the European Union.

carcinogen

any substance or agent directly involved in the promotion of cancer or in the facilitation of its propagation.

CAS number

Chemical Abstract Services (CAS) are unique numerical identifiers for chemical elements, compounds, polymers, biological sequences, mixtures and alloys. A service of the American Chemical Society that indexes and compiles abstracts of worldwide chemical literature called Chemical Abstracts.

CAT

Procedural advice on the provision of scientific recommendation on CAT (classification of advanced therapy medicinal products).

CE mark

European conformity Marking: A mandatory European marking for products falling under one of the New Approach Directives (including medical devices, but excluding cosmetics, chemicals, pharmaceuticals, foodstuffs) to indicate conformity with the health and safety requirements set out in European Directive.

Cell

The cell is the basic unit of all living organisms. Some living organisms exist only as a single cell. This is the case for bacteria , as well as certain animals and plants. An average sized man contains from 60 to 100 trillion cells. Cells nourish themselves, produce energy, exchange information with their neighbours, multiply, and eventually die when their time has come.

Centralised Procedure

Since 1995, medicinal products can be assessed via the Centralised procedure. Medicinal products that have been approved via this procedure are issued a marketing authorisation that is valid throughout the EU. This marketing authorisation is granted by the European Commission. The use of this procedure is compulsory for medicinal products derived from a biotechnological process. For other innovative products, such as products with a new active substance, a company can choose whether to follow this procedure or the Mutual recognition procedure. In the case of the Centralised procedure, a dossier must be submitted to the European Agency for the Evaluation of Medicinal Products (EMEA) in London.

Classification code (MedDRA)

An eight digit unique numeric code assigned to a MedDRA term. The code is non-expressive and is intended to fulfil a data field in various electronic submission types.

Clinical Trials

A clinical trial is a research study conducted in human participants to evaluate the safety and efficacy of a medicine to improve patient's health. Clinical trials conducted on new medicines and sponsored by pharmaceutical companies can only be started after a compound has survived rigorous pre-clinical development work, which involves laboratory testing (chemical/biological/pharmacological/toxicological). It is only when these tests show favourable and promising results that a company can proceed to assess the medicine in humans.

Combination ATIMP An ATIMP

(Advanced Therapy Investigational Medicinal Product) involving a medical device.

Committee for Medicinal Products for Human Use -ÊCHMP

Within the European Agency for the Evaluation of Medicinal ProductsÊ(EMEA), the Committee for Proprietary Medicinal Products is the scientific committee responsible for preparing the Agency's opinions on questions relating to the evaluation of medicinal products for human use. It is made up of 30 members nominated by the Member States.

Comparator

An investigational or marketed product (i.e. active control) or placebo, used as a reference in a clinical trial.

Compassionate Use

Refers to situations where a drug is provided to a patient on humanitarian grounds prior to the drug's receiving regulatory approval.

Competent Authority (CA)/ National Competent Authority (NCA)

Also referred as National Medicine Regulatory Authorities A regulatory agency in an EU Member State or for medical devices, a Competent Authority is the organization with the authority to act on behalf of the government of a Member State to ensure that all medical devices meet the essential requirements laid down in the Directives prior to marketing authorisation.

Compulsory Licensing

The TRIPS agreement provides strict conditions under which governments can allow the production or sale of a product without the permission of the patent holder (for example in cases of extreme urgency).

Concentration unit

The unit of measurement used for the concentration of the active substance.

Controlled

In a controlled trial, the tested product is compared to a reference treatment. The reference treatment can be, for example, a placebo, a product known to be effective, a surgical procedure, or a different dose of the same product.

Counterfeit pharmaceutical (WHO definition)Ê

(A fake medicine) which is deliberately and fraudulently mislabelled with respect to identity and/ or source. The term counterfeiting can apply to both branded and generic products, and counterfeit products may include products:

CRO (Clinical Research Organisation)

Organisation which provides services to the pharmaceutical industry and specialised in the conduct of clinical trials.Ê

Cross over

Comparison of two (or more) treatments in which patients are switched to the alternative treatment after a specified period time.

Current sponsor code

The current code in use by the sponsor for an active substance.
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DALY

Disability-Adjusted Life Year( DALY) measures overall burden of a disease by combining the year of potential life lost due to premature death and the year of productive life lost due to the disability.One DALY is one lost year of healthy life.

Data Exclusivity (also referred to as Data Protection)

Pharmaceutical registration data are the proprietary (i.e. confidential) data generated by scientific research conducted to demonstrate the efficacy and safety of new medicines and submitted to regulatory authorities for marketing approval. Data protection periods refer to the period of time during which a company is able to keep its rights for clinical data related to a given medical product, without another company being able to use that data when applying for an authorisation to market a generic medical product. Data exclusivity is an independent intellectual property right, subject to protection under Article 39.3 of the TRIPs Agreement.

Data Monitoring Committee

A Data Monitoring Committee is a group of independent experts external to a study assessing the progress, safety data and, if needed critical efficacy endpoints of a clinical study.

Data Privacy

Individually identifiable information, e.g. medical background and personal health data, that may not be disclosed or transferred inappropriately without the explicit authorisation of the individual.

Differential Pricing

Sometimes referred to a "tiered pricing"", ""equity pricing"" or ""preferential pricing"". They all refer to the same principle of adapting prices to purchasing power of the world's poorest countries."

Disease

An alteration in the state of the body or of some of its organs, interrupting or disturbing the performance of the vital functions.

dna

deoxyribonucleic acid, the chemical substance of which the genetic material of nature exists.

Doha Declaration

Refers to the "Declaration on the TRIPS Agreement and Public Health"", adopted on 14 November 2001 at the Doha (Qatar) Ministerial conference of the World Trade Organization. It is a political commitment of all WTO members governments to the TRIPS Agreement which further clarifies the flexibility already available under TRIPS, enabling developing countries to address national health emergencies.Ê"

Dose response

The dose-response describes the change in effect caused by differing doses to a Medicinal Product after a certain exposure time. This may apply to individuals, or to populations.

Double blind

A trial where the investigators and the subjects included in the trial (healthy volunteers or patients) donÕt know which treatment is given.

DTCI - Direct-to-Consumer Information

In contrast with Direct-to-Consumer Advertising (DTCA) which is forbidden in Europe, the pharmaceutical industry considers that all patients should have the same right to truthful, accurate and easily understandable information that enables them, in consultation with their doctors, to make well-informed choices about their health. Europeans should have access to quality medicine information from all sources, including from pharmaceutical companies (see "information to patients"")."
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E-health

The application of Information and Communication Technologies (ICT) across a range of functions in the healthcare sector with a view to enhancing continuity of care and ensuring access to safe and highquality healthcare.

EDLÊ

The WHO Model List of Essential Drugs was developed in the 1970s to stimulate the rational availability of medicines in developing countries. Essential Drugs are those that meet the health needs of the majority of the population. They should be available at all times in adequate amounts and in appropriate dosage forms. The WHO Model List of Essential Drugs enables developing countries to indentify priorities and make their own drug selection. It is to be noted that over 90% of the essential drugs contained in the WHO Model List are off-patent and can therefore be legally copied, at virtually no cost, by generic companies.

EMEA

The European Agency for the Evaluation of Medicinal Products, now called European Medicines Agency, began its activities in 1995. Based in London, the Agency coordinates the existing scientific resources of the Member States in order to evaluate and supervise medicinal products for both human and veterinary use throughout the European Union.

enzyme

a biological catalyst, generally a protein, than can accelerate and combine certain chemical reactions.

EPAR

European Public Assessment Report - Reflects the scientific conclusion reached by the Committee for Proprietary Medicinal Products (CPMP) at the end of the centralised evaluation process and provides a summary of the grounds for the CPMP opinion in favour of granting a marketing authorisation for a specific medicinal product.

Epidemic

A widespread occurence of a disease affecting many persons simultaneously in a community or area.

Epigenetics

Variations in the way genetic material is packaged and read can influence gene activity without altering the sequence of DNA. These patterns of modifications in identical twins are different despite their having the same DNA.

Extractive medicinal product

A medicinal product derived from human tissue such as blood or plasma.
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Falsified Medicines

A falsified medicine gives a false representation of its identity and/ or source and/or record keeping for traceability; pretends to have been assessed and approved by the competent regulatory authority, pretending to be a genuine quality product; has an intention to deceive by a fraudulent activity; is falsified for profit motives, disregarding public health and safety; and that disputes concerning patents or trademarks must not be confused with falsification of medicines.

First human administration

Conducting the first dose in human phase I clinical trial.

Free Trade Agreement

An agreement between partner countries which aims to eliminate tariffs, import quotas, and preferences on most (if not all) goods and services traded between them, whilst ensuring market access (e.g. through transparency, IPR protection and enforcement, regulatory harmonisation).
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GATT (General Agreement on Tariffs and Trade)Ê

A comprehensive free-trade treaty signed in 1947 by 117 nations, including almost every developed country. The goal of GATT has been to promote global economic growth by encouraging and regulating world trade. Among other things, member countries are required to treat all other member countries equally in the application of import and export tariffs, offer basic copyright protection to authors from member countries, consult with each other about trade matters and attempt to resolve differences in a peaceful manner. GATT created an international regulatory body known as the World Trade Organization (WTO) to enforce compliance with the agreement.

GCP (Good Clinical Practice)

International ethical and scientific quality standard for designing, conducting, recording and reporting trials that involve the participation of human subjects. Compliance with this standard provides public assurance that the rights, safety and well-being of trial subjects are protected, consistent with the principles that have their origin in the Declaration of Helsinki, and that the clinical trial data are credible.

Gene Therapy

The replacement of a defective gene in an organism suffering from a genetic disease. Recombinant DNA techniques are used to isolate the functioning gene and insert it into cells. Over 300 single-gene genetic disorders have been identified in humans. A significant percentage of these may be amenable to gene therapy.

Gene therapy medical product

Product aimed at the transfer of a prophylactic, diagnostic or therapeutic gene to human and its subsequent expression in vivo.

Generic

An off-patent medicine. Until the patent expires, only the company that discovered a new medicine may produce it. After patent expiry any company may produce the same generic compound.

Genes

Genes are formed from DNA, carried on the chromosomes and are responsible for the inherited characteristics that distinguish one individual from another. Each human individual has an estimated 100,000 separate genes. More generally the term 'gene' may be used in relation to the transmission and inheritance of particular identifiable traits.

genetics

the study of inheritance; deals with the laws of inheritance from one generation to the next and the prop_erties of genes.

Genome

A genome contains all of the information needed to build and maintain that organism, it contains the entirety of an organismÕs hereditary information.

Genomics

The study of genes and their function. Recent advances in genomics are bringing about a revolution in our understanding of the molecular mechanisms of disease, including the complex interplay of genetic and environmental factors. Genomics is also stimulating the discovery of breakthrough healthcare products by revealing thousands of new biological targets for the development of drugs, and by giving scientists innovative ways to design new drugs, vaccines and DNA diagnostics. Genomics-based therapeutics include "traditional"" small chemical drugs, protein drugs, and potentially gene therapy."

genotoxicity

the degree to which something causes dam_age to or mutation of DNA.

GMP (Good Manufacturing Practice)

Under this regulation, all pharmaceutical manufacturing companies in Europe are required to comply with rules setting controls over manufacturing processes and inspections of manufacturing sites.
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Hatch-Waxman ActÊ

In the US, the Hatch-Waxman Act (1984) struck a balance by making generic drugs more widely available and continuing incentives for pioneer companies to develop new drugs. Generic companies have a limited right to infringe on a patent, so they can get an early start on the approval process. Since the law's passage, the generic drugs' share of the market has grown to nearly 50 percent.

Health - WHO definition

Health is a state of complete physical, mental and social well-being and not merely the absence of disease or infirmity. Article 152 ofthe Treaty on the European Union states that a "high level of human health protection shall be ensured in the definition and implementation of all Community policies and activities""."

Health Outcome

Changes in health status (mortality and morbidity) which result from the provision of health (or other) services.

Health Outcomes Research

Refers to a discipline/methodology to assess the value added - including direct, indirect and intangible benefits - of a particular medicine. Industry considers that such considerations (also referred to as "pharmacoeconomics"" or ""cost-effectiveness studies"") are not appropriate in the marketing approval process which must focus solely upon quality, safety and efficacy."

Health Technology Assessment

is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to inform the formulation of safe, effective, health policies that are patient focused and seek to achieve best value.

Healthy volunteers

Clinical trial includes subjects in good health.

HMO (Health Maintenance Organization)

Is a health plan that is also involved in how your health care is delivered. Managed care refers to health plans coordinating your health care with you and the providers that participate in the health plan. HMOs are the most common type of managed care.Ê

Hormone

A hormone is a molecule produced by a gland or tissue. Usually transported by the blood, a hormone affects an organ or other tissue located at a distance. For example, the pancreas produces insulin, which regulates the level of sugar throughout the organism. Hormones are ÒmessengersÓ that, in concert with the nervous system, coordinate the activities of billions of cells in the human body.

hts

high_throughput screening; a highly automated meth_od of identifying potential medicines in chemical libraries.

Human Genome

The entirety of a humanÕs hereditary information.

Human pharmacology (Phase I)

Human pharmacology (Phase I) trials are the first stage of testing in human subjects, generally comprising a small group of healthy volunteers. This phase includes trials designed to assess the safety (pharmacovigilance), tolerability, pharmacokinetics, and pharmacodynamics of a drug
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ICH (International Conference on Harmonisation)

The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) is a unique project that brings together the regulatory authorities of Europe, Japan and the United States 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 harmonisation in the interpretation and application of technical guidelines and requirements for product registration.

Immunological medicinal product

Any medicinal product consisting of vaccines, toxins, serums or allergen products used to produce an immunological response

IMP (Investigational Medicinal Product)

A pharmaceutical form of an active substance or placebo being tested or used as a reference in a clinical trial, including products already with a marketing authorisation but used or assembled in a different way from the authorised form, or when used for an unauthorised indication, or when used to gain further information about the authorised form.

in silico

expresses the meaning that an experiment is per_formed on computer or via computer simulation. Contrary to widespread belief, the term is not derived from Latinin vitro: Latin for Òwithin the glassÓ; refers to the technique of performing a given experiment in a test tube.

In utero

Subjects are unborn infants, still in the womb.

in vivo

Latin for Òwithin the livingÓ; describes experimenta_tion done on a whole, living organism..

In-silico

Analysis performed using computers in conjunction with informatics.

In-vitro

Literally means Òin glassÓ, as in a test tube. A test that is performed in-vitro is one that is done in glass or plastic vessels in the laboratory. In vitro is the ÔoppositeÕ ofÊin vivo.

In-vivo

Biological processes or experiments occurring or carried out in the living organism, as opposed toÊin-vitro, which is outside the body (in a test tube)

Incidence

The number of new patients affected by the disease per annum

Informed consentÊ

The concept of informed consent (required to participate in clinical trials) is based on the principle that a physician/doctor has the duty to disclose to a patient information (e.g. potential risks, benefits and alternatives) that allows the patient to make reasonable decision regarding his or her participation in a clinical study.

INN

When a molecule is shown to have some useful activity, it is given an international non-proprietary name (INN), based on internationally agreed rules, which is submitted for approval to a special committee at the World Health Organisation.

Innovation

Technological progress that leads to the creation of an entirely new product (product innovation) or a reduction in the cost of producing (process innovation) or an increase in the therapeutic value of an existing product for patients. Innovations can lead to new active substances, new indications for existing products or new ways of administering the same product.

International Exhaustion of International Property Rights

A theory which is based on the territoriality of rights and implies that after the legitimate first sale of a protected good in a country anywhere in the world, the owner of the intellectual property right on this product loses his right to restrict its (re-)exportation in any other country. In Europe, international exhaustion is not in place and the current Community-wide exhaustion regime for trademarks allows trademarks holders in the EU to ban imports of branded goods from outside the territory of the EU into the Community. The same situation prevails for patented goods. By contrast, the EU authorities consider the European Union as a single and unified territory where protected goods can freely circulate between the Member States. Consequently, parallel import is allowed within the territory of the EU.

International Reference Pricing

The practice of using the price(s) of a medicine in one or several countries in order to derive a benchmark or reference price for the purposes of setting or negotiating the price of the product in a given country. In some countries, the referencing pricing system is applied rigidly, while in other countries, it is simply one of many elements of information used to inform the pricing decision. The basket of countries chosen varies, based on a range of criteria used to justify the selection of countries.

Interventional trial

An interventional trial sets up to discover or verify the effects of one or more investigational medicinal product(s) (IMP), to ascertain its (their) safety and/or efficacy. The assignment of the patient to a particular therapeutic strategy is decided in advance by a trial protocol. The way the IMP(s) are used, and the way the patients are selected for the trial and followed up are not as per current practice, and the data from the trial are systematically analysed.

Investigator

A doctor or a person following a profession agreed in the Member State for investigations because of the scientific background and the experience in patient care it requires. The investigator is responsible for the conduct of a clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the leader responsible for the team and may be called the principal investigator.

IPRs (Intellectual Property Rights)

The area of law that regulates the ownership and use of creative works, including patent, copyright and trademark law. Intellectual property rights have been developed to protect creative people who have disclosed their work for the benefit of humankind from having it copied or imitated without their consent.

IVRS Interactive Voice Response System

commonly used for randomisation of treatment and controlling the shipment of stock of product.
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LDC

Least Developed Countries (currently 49 countries).Ê Least Developed Countries (currently 49 countries). Developing countries are distributed under income groups : high income, middle income, low income
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MedDRA

Medical Dictionary for Regulatory Activities Terminology. This critical medical resource, was developed by the International Conference on Harmonisation (ICH) and is owned by the International Federation of Pharmaceutical Manufacturers Association (IFPMA). By providing one source of medical terminology, MedDRA improves the effectiveness and transparency of medical product regulation worldwide.

Medical research / biomedicalÊ

Biomedical research (or experimental medicine), in general is known as medical research, and is basic research, applied research, or translational research conducted to aid and support the body of knowledge in the field of medicine. Biomedical research comprises of three main categories:- The scientific investigation required to understand the underlying life processes which affect disease and human well-being, including such areas as cellular and molecular bases of diseases, genetics, immunology. - The study of specific diseases and conditions (mental or physical), including detection, cause, prophylaxis, treatment and rehabilitation of persons.- The design of methods, drugs and devices used to diagnose, support and maintain the individual during and after treatment for specific diseases or conditions - A full list of such activities includes clinical trials and laboratory investigations, the study of exposure to environmental agents and various behavioural hazards.

Medicinal Product

Under EU law a medicinal product is defined as any substance or combination of substances presented for treating or preventing diseases in human beings or animals. Any substance or combination of substances which 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 is likewise considered a medicinal product. See Directive2001/83/EECÊamending directive 65/65/EEC setting out the framework of the pharmaceutical regulation.

MEP

Member of the European Parliament

metabolism

the transformation of chemical substances in the body or within a cell.

Microdosing

The concept of microdosing involves the use of extremely low, non-pharmacologically active doses of a drug to define the pharmacokinetic profile of the medication in humans. By definition, microdosing means the use of Ôless than 1/100th of the dose calculated to yield a pharmacological effect of the test substance to a maximum dose of <100 microgramsÕ (European Medicines Agency paper).

Millennium Development Goals (MDGs)

The Millennnium Development GoalsÊ(MDGs), adopted at the Millennium Summit of the United Nations in September 2000, call for a dramatic reduction in poverty and marked improvements in the health of the poor. The goals and targets represent a partnership between the developed countries determined to "create an environment which is conducive to development and the elimination of poverty"""

Monopsomy

A situation in which the entire market demand for a product or service consists of only one buyer

Morbidity

The relative incidence of a particular disease in a specific locality

Mortality

Number of deaths in a given period

mrna

messenger ribonucleic acid, the working copy of a gene that acts as a blueprint for the synthesis of pro_teins. unlike DNA, the molecule is able to leave the cell nucleus.

Multinational trial

A multinational research trial is a clinical trial conducted in more than one country at the same time.

Mutual Recognition

Under this procedure a marketing authorisation granted by one Member State is extended to one or more other Member States selected by the applicant. This procedure applies to the majority of conventional medicines.
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Neurodegenerative diseases

An umbrella term for diseases, which result in the progressive loss of structure or function of neurons, including ParkinsonÕs, AlzheimerÕs, and HuntingtonÕs.

New Science

A general term used for, biotechnological advances in the pharmaceutical industry, including personalised medicines, epigenetics, diagnostic tools such as biomarkers and nanotechnology.

NGO

Non Governemental Organisation

NMEs

New molecular entities: products (including new chemical entities (NCEs), biological products, vaccines and products of biotechnology) that have not been previously available for therapeutic use in humans and are destined to be made available as prescription-only medicine, to be used for the cure, alleviation, treatement, prevention or in vivo diagnosis of diseases in humans.

Non-durables

Consumable medical supplies are non-durable medical supplies that are usually disposable in nature, cannot withstand repeated use and are primarily and customarily used to serve a medical purpose.

Non-human primates

Lemurs, monkeys and apes are non-human primates. These mammals have highly developed brains, eyes facing forward, a shortened nose and muzzle, and opposable thumbs.

Non-vertebrate / invertebrate

Any animal that lacks a vertebral column or backbone. More than 90% of living animals are invertebrates. Worldwide in distribution, they range in size from minute protozoans to giant squids. Apart from the absence of a vertebral column, invertebrates have little in common. They are generally soft-bodied and have an external skeleton for muscle attachment and protection. They include the protozoans, annelids, cnidarians, echinoderms, flatworms, nematodes, mollusks, and arthropods
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ODA

Official Development Assistance

Off-Patent Pharmaceuticals

A medicine that has come to the end of its patent term and is open to generic competition.

Omics

A short-hand term used to refer to a field of study in biology. For example, genomics is the study of genomes.

oncogene

a gene that plays a role in the development of cancer.

ontology

the study of being or existence.

Open Trial

A Trial where the investigators and the subjects know which treatment is actually given.

Orphan Drug

A drug which only has a limited target population or which treats a rare disease thus limiting its commercial and financial potential. First enacted in the US (in 1983), the Orphan Drug Act was developed to help these patients and provides incentives (a period of market exclusivity and a tax credit for research expenses) to help make it commercially feasible to develop these drugs. In Europe, the Orphan Medicinal Product Regulation was adopted much later (in December 1999), to stimulate the development in the EU of pharmaceuticals for the diagnosis, prevention and treatment of some 5,000 diseases that are classified as "rare"" and affect not more than 5 in 10,000 persons in the European union."

OTC (Over the counter)

A medicine available without a prescription.

OutpatientÊCare

Medical services that are provided without the need for an overnight stay in a hospital, such as through an ambulatory care clinic or emergency department.
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Paediatric Investigation Plan (PIP)

Document upon which the development and authorisation of medicinal products for the paediatric population is based. It is presented by an applicant early during the development of a product to the EMA Paediatric Committee in order to agree a paediatric development plan.

Pandemic

Disease affecting persons over a wide geographical area (extensively epidemic).Ê

Parallel group

A trial in which two or more treatments are evaluated concurrently in separate groups of patients.

Parallel Trade

Parallel trade is the purchase of goods at low prices in one country and the subsequent resale of those goods at higher prices in another country.

Patent

A patent is a legal, public document granting an intellectual property right to the patent holder. At the same time the information relating to the invention is made public, thereby enhancing the public good and protecting the rights of the inventor. In exchange for publication of his or her patent, the inventor is rewarded a limited monopoly allowing the patent holder to prevent others from making, using or selling the patented product. The monopoly is limited as to time (now 20 years from the date of filing of the patent application) and by national boundaries (where the patent has been granted). Patents for inventions are granted only if three conditions are met : Novelty, industrial applicability and inventive step.

Patient adherence

The degree to which patients adhere to medical advice and take medicines as directed.

pcr

polymerase chain reaction; a technique for rapid copy_ing and amplification of even minute amounts of DNA.

Personalised medicines

Tailored treatment to patient subgroups based on their biological characteristics.

Pharmacodynamic

Pharmacodynamics is the exploration of what the Medicinal Product does to the body.

Pharmacoeconomic

Pharmacoeconomics refers to the scientific discipline that compares the value of one pharmaceutical drug or drug therapy to another.

pharmacogenetics

describes the influence of gene vari_ations in individuals on the efficacy and side effects of medicines.

Pharmacokinetics

Investigates the update, conversion and breakdown of medicines in the body overtime. Environmental factors, diet and genetic predisposition all play a role.

Pharmacology

Pharmacology is a broad discipline encompassing all aspects of the study of drugs, including their discovery, development and actions.

Pharmacovigilance

Refers to the careful monitoring and continuous surveillance of the safety of an authorised medicinal product during its life on the market.

Phase I

Phase I is the first stage of a clinical trial. It is to ensure a treatment is safe for people to take, rather than to try to treat a condition. These trials are very small, (typically around 30 people), and usually involve healthy volunteers or sometimes patients

Phase II

The second phase in clinical trials aims to investigate the safety and effectiveness of a potential therapy. Usually between 100 and 300 people will be enlisted to take part with the aim of determining whether the treatment will be safe and effective to treat a condition.

Phase III

If previous trials have indicated a treatment is safe and that it also shows promise in being able to treat a condition, phase III clinical trials begin. These involve large numbers of participants usually from several hundred to several thousand subjects, and are often spread between different hospitals and countries. If these trials show that a drug is safe and effective, the manufacturers can apply for a drug license.

Phase IV

Post marketing studies to delineate additional information including the drug's risks, benefits, and optimal use. These studies are designed to monitor effectiveness of the approved intervention in the general population and to collect information about any adverse effects associated with widespread use.

PIL - Patient Information Leaflet

Provides a set of information, in a particular order, accompanying each medicine. It is usually the only source of information on how to use a medicine safely and effectively, when the patient actually takes the medicine.

PIM - Product Information Management

PIM is a project that was initiated in 1999 to develop a new way of handling product information. Its membership has been drawn from EMEA, Member State competent authorities and EFPIA members. It has gone through several phases, which have culminated in the publication of the PIM standard. Should you need more information please visit the EMEA website atÊhttp://pim.emea.eu.int/whatispim.htm

Placebo

A placebo is a control substance (a dummy treatment) that is given to people taking part in a clinical trial. It allows researchers to test for the 'placebo effect'. This is a psychological response where people feel better even though the substance they are taking has no effect. By comparing people's responses to the placebo and to the drug being tested, researchers can tell whether the drug is having any real benefit.

Plasma derived medicinal product

A medicinal product derived from human blood or human plasma. Preterm newborn infants Subjects are not more than 37 weeks from their conception.

polymorphism

existence in more than one form; in gene_tics, a region of DNA in which differences in the sequence of building blocks occur in a relatively large number of individuals.

Prevalence

The percentage of population which is affected by a diseaseÊ

Primary Care

The "medical home"" for a patient, ideally providing continuity and integration of health care. All family physicians and most pediatricians and internists are in primary care. The aims of primary care are to provide the patient with a broad spectrum of care, both preventive and curative, over a period of time and to coordinate all of the care the patient receives."

Principal inclusion criteria

Reasons for the inclusion of subjects in the clinical trial.

Procedure

itt means any use, invasive or non-invasive, of an animal for experimental or other scientific purposes, with known or unknown outcome, or education purposes, which may cause the animal a level of pain, suffering, distress or lasting harm equivalent to, or higher than, that caused by the introduction of a needle in accordance with good veterinary practice.

Product code

This is a code designated by the sponsor who represents the name routinely used by the sponsor to identify the product in the Clinical Trial documentation.

protein

a molecule consisting of a chain of amino acids. Be_cause of the variety of their building blocks, proteins can differ greatly in form and function.

proteome

the totality of the proteins of an organism.

Proteomics

Proteomics is the systematic analysis of all protein sequences and protein expression patterns in tissues, which involves the isolation, separation, identification and functional characterization of all of the proteins in an organism. Genes encode proteins that perform all of the fundamental activities within cells. Proteins transmit messages, repair damage, provide the building blocks for tissues and carry out reactions essential for life. Proteins are the molecular machines that carry out genetic instructions. Abnormalities in protein production or function have been connected to many diseases and health conditions. To understand how best to treat a particular disease, it is necessary to identify the proteins associated with that disease and to understand how they function.Ê

Protocol

A document that describes the objective(s), design, methodology, statistical considerations and organisation of a trial. The term protocol refers to the protocol, successive versions of the protocol and protocol amendments.

Public Health

Public health is covered by Article 152 of the EC Treaty, which was introduced by the Treaty of Maastricht. This article states that Community action is to focus on the prevention of illnesses, including drug addiction, by promoting research into their causes and their transmission, as well as health information and education. The Treaty of Amsterdam reinforces these objectives by requiring that the definition and implementation of all Community policies and activities ensures a high level of human health protection. Under Article 152 action towards these ends may involve Community measures, complementing action by the Member States. But the main approach should be to encourage cooperation between the Member States, in line with the subsidiarity principle.
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Radiopharmaceutical medicinal product

A radioactive pharmaceutical, nuclide, or other chemical used for diagnostic or therapeutic purposes.

Rare disease

A rare disease concerns a restricted number of patients in the general population and shows evidence of gravity (because it is life-threatening, invalidating or serious and chronic). The limit accepted in Europe is 5 people in 10,000 affected by the disease.

Reference Pricing

The practice of setting the price and/or reimbursement level of medicines according to the price set in other EU Member States. Such practice undermines price competition and jeopardizes incentives for innovation and R&D. Mixing patented and off-patent medicines in the same category of reference pricing is even worse because it prevents price competition both among generics and branded medicines, rewards imitation, and penalizes innovation.Ê

Replacement, Reduction, Refinement (3Rs)

ReplacementÊrefers to methods, which avoid or replace the use of animals in areas where animals would otherwise have been used. RefinementÊrefers to improvements to housing and care and procedures, which minimise actual or potential pain, suffering, distress or lasting harm and/or improve animal welfare in situations where the use of animals is unavoidable. Refinement also refers to the lifetime experience of the animal. There is evidence that refinement not only benefits animals, but also improves the quality of research findings. ReductionÊrefers to methods which minimise animal use and enable researchers to obtain comparable levels of information from fewer animals or to obtain more information from the same number of animals, thereby reducing future use of animals.

rna

ribonucleic acid; the chemical substance of which, among other things, working copies of genes exist.

Routes of administration

A route of administration in pharmacology and toxicology is the path by which a drug, fluid, poison, or other substance is brought into contact with the body.

Rx

Prescription medicine (in the US)
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Scope of the trial

A definition of the general outline of what the clinical trial will investigate.

Secondary care

Secondary care is the term for care in hospital

Serendipity

The faculty of making fortunate discoveries by accident

Single blind

A trial where the subjects (healthy volunteers or patients) included in the trial doesnÕt know which treatment they are given but the investigator does.

SITC

Standard International Trade Classification

SMEs

Small and medium size enterprises

SmPC (Summary of Product Characteristics)

In order for a medicinal product to be evaluated either by the competent authorities of Member States or by the EMEA, a company must submit a dossier. This dossier, which in terms of content and presentation must comply with current European guidelines (Directive 65/65/EECÊamended by Directive 83/570/EEC), is comprised of four parts. The first part contains administrative information and a summary of the dossier, which includes the Summary of Product Characteristics. In European jargon, this information is usually referred to as the SPC or SmPC, which forms an intrinsic and integral part of the marketing authorisation

snps

single nucleotide polymorphisms; differences in in_dividual building blocks of DNA that are distributed ran_domly over the genome and passed from generation to generation.

SPC (Supplementary Protection Certificate)

The SPC applies to pharmaceutical products and was brought into effect by Regulation in the European Union on January 2 1993. The SPC has a maximum term of 5 years to give a maximum effective patent life of 15 years from the date on which a product is authorized for first marketing in a EU country.

Specific paediatric formulation

Formulation specifically developed for paediatric use.

Specific vulnerable populations

Clinical trial includes subjects (healthy volunteers or patients), who are considered to be part of a population at risk.

Sponsor

An individual, company, institution, or organization that takes responsibility for the initiation, management, and/or financing of a clinical trial.

Sponsor Country

Country name of the organisation or individual who is providing the finance or resources for the clinical trial.

Stem cell

Stem cells are unspecialized ÒmasterÓ cells that differ from other kinds of cells in the human body in that they have a unique capacity to multiply and differentiate into many types of specialized cells and tissues. Stem cells exist at all stages of human development from early embryos to foetuses to adults. There are three types of stem cells: embryonic, foetal and adult. While the use of adult stem cells is well accepted and non-controversial, research into embryonic and foetal stem cells raises a number of ethical questions.Ê

Subject

A person participating in a trial - the subject may be a patient or a healthy volunteer.

Subsidiarity Principle

It is a fundamental principle of European Union law. According to this principle, the EU may only act (i.e. make laws) where member states agree that action of individual countries is insufficient. The principle was established in the 1992 Treaty of Maastricht, and is contained within the proposed new Treaty establishing a constitution for Europe.Ê
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targets

the molecules, mostly proteins, upon which medi_cines act in the human body.

Testing (animal testing)

Use of animals in experiments and development projects usually to determine toxicity, dosing and efficacy of test drugs before proceeding to human clinical trials.

Therapeutic Reference Pricing

A method of comparing the prices for a range of different medicines, which are deemed by the founder to be similar in as much as they are part of the same therapeutic area and in some circumstances, are interchangeable. However, they are not the same medicine.

Tissue Engineered Product

A product that contains or consists of engineered cells or tissues, is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue.

Transgenic

A plant or animal containing a gene or genes from another species. Also refers to plants or animals that have altered genes.

transgenic animals

animals containing genes derived from other organisms.

Transparency Committee

The Transparency Committee, established under Directive 89/105/EEC (and often referred to as "the Transparency Directive""), is made up of Member State representatives responsible for the pricing and reimbursement of pharmaceuticals and advises the Commission on matters relating to the Transparency Directive."

TRIPS (Trade-Related Aspects of Intellectual Property Rights)

The WTO TRIPS Agreement is a major achievement of the GATT's so-called "Uruguay Round"", which sets up minimum international standards of intellectual property rights, including patents, and for enforcement of those rights, which each member of the WTO must incorporate into its national laws."

tumour suppressor

a molecule which prevents cancer from developing.
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Vaccine

Refers to the introduction of vaccine - attenuated or killed micro-organisms (bacteria or viruses) - into the body for the purpose of inducing protective immunity against infectious diseases. Conventional prophylactic vaccines aim at stopping people becoming infected. Therapeutic vaccines are products that stimulate the immune system of people with chronic infection to eliminate the virus from their bodies. Vaccine preparations can be natural, synthetic or derived by recombinant DNA technology.

Vertebrate

Having a backbone or spinal column. Vertebrates include fishes, amphibians, reptiles, birds, and mammals, all of which are characterized by a segmented spinal column and a distinct well-differentiated head.

Virus

A microorganism smaller than a bacteria, which cannot grow or reproduce apart from a living cell. A virus invades living cells and uses their chemical machinery to keep itself alive and to replicate itself.
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WHO (UTN)

World Health Organisation Universal Trial Number. The UTN is a number, obtained by the trial's sponsor or principal investigator and is used for the identification of clinical trials worldwide.

World Health Organisation

Founded in 1948 with headquarters based in Geneva, the World Health Organisation is a specialised agency of the United Nations with 191 Member States, which promotes technical cooperation for health among nations, carries out programmes to control and eradicate disease and strives to improve the quality of human life.

World Trade Organisation

Created by the Uruguay Round negotiations and established since 1 January 1995 in Geneva, the World Trade Organisation (WTO), with a membership of 153 governments, aims at being a place where member governments try to sort out the trade problems they face with each other.