Glossary, abbreviations and acronyms

Accelerated assessment

Rapid assessment of medicines in the centralised procedure that are of major interest for public health, especially ones that are therapeutic innovations. Accelerated assessment usually takes 150 evaluation days, rather than 210.

Accountability

Accountability refers to the obligation of an individual or organization to be answerable for their actions and decisions, and to accept the consequences of those actions and decisions. It is a principle that requires transparency and responsibility in decision making and performance, and it is often enforced through formal systems of oversight and control. In the context of individuals, accountability refers to the responsibility to take ownership of one’s actions and decisions, and to accept the consequences of those actions and decisions. This can involve setting and working towards personal goals, taking responsibility for one’s behaviour and decisions, and being accountable to others for the outcomes of one’s actions.

Active Pharmaceutical Ingredient (or Drug substance) (API)

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

Active substance (AS)

The substance responsible for the activity of a medicinal product.

Active substance master file (ASMF)

Documentation providing detailed information on the manufacturing of the active substance of a medicine.

Active substance of biotechnological origin

Product that contains an active substance of biological or biotechnological origin

Active substance of chemical origin

A product that contains an active substance of chemical origin

Adaptive (trial) design

The option to modify the design of an ongoing clinical trial is becoming increasingly common and is known as adaptive design. Data are evaluated before the trial is finished. This is known as interim analysis and might be carried out at several time points. Depending on the circumstances, this may lead to changes to the trial such as stopping one treatment arm or changing the number of participants in a group. The planned number of participants might be reduced if the interim analysis shows that a smaller sample size will still allow a valid result to be obtained. Alternatively, the sample size might be increased if that will allow a valid or reliable result to be obtained within a more acceptable period of time.

Adaptive designs can save time and resources and can reduce the exposure of study participants to the inferior treatment.

Interim analyses and any anticipated changes to a trial should be described and justified in the study protocol.

Adjuvant (ADJ)

An ingredient in a medicine that increases or modifies the activity of the other ingredients. Adjuvants are often included in vaccines to enhance the body’s immune response.

Adolescents (12-17 years)

Subjects are aged 12 to less than 18 years.

Adults (18-64 years in EU, 16-64 years in UK)

Subjects are aged 18 to 64 years in EU, and 16-64 years in UK.

Advanced Therapy IMP (ATIMP)

Advance Therapy Investigational Medicinal Products are medicinal products involving cell or gene therapy or tissue engineering.

Advanced therapy medicinal product (ATMP)

A medicine for human use that is based on genes, cells or tissue engineering.

Adverse drug reaction (ADR)

A noxious and unintended response to a medicine.

Adverse event (AE)

An untoward medical occurrence after exposure to a medicine, which is not necessarily caused by that medicine.

Allometric scaling

Allometric scaling is one of the tools that drug developers use to predict human PK based upon animal data. Prediction methods, like allometric scaling, provide a “sneak peek” at how a drug might behave in humans before any clinical studies are conducted. This is important information for both drug developers and regulators (like the FDA) because it provides a data-driven foundation for establishing a safe starting dose in humans. Allometric scaling comes from two words: ALLOMETRY: The study of size and its consequences SCALING: An engineering term meaning to adjust (or “scale”) dimensions (or other parameters) with size In biology, the basis of allometric scaling lies in the relationship between metabolic rate (defined as the rate of biological life processes and metabolism) and the body size of the animal. The metabolic rate that is used in allometry includes life processes such as number of heart beats or number of breaths in the lifespan of the animal as a function of size. It is critical to understand that as body size increases from one animal species to another, metabolism slows down. To exploit this, allometric scaling uses mathematical models to describe physiological, anatomical, and biochemical changes in animals as their size changes. Of specific interest for drug development, this approach can predict important PK information for humans using experiments conducted in various animal species.

Ancillary human blood derivative

A medicine derived from human blood or plasma that is incorporated within a medical device, where the main mode of action is due to the device.

Ancillary medicinal substance

A medicine that is incorporated within a medical device where the main mode of action is due to the device.

Annual reassessment

An annual review of the benefits and risks of a medicine that has been authorised under exceptional circumstances. As part of the process, the specific obligations imposed on the marketing-authorisation holder are also reviewed.

Article 58 application

An application for a scientific opinion on the use of a human medicine intended exclusively for markets outside of the European Union. Medicines eligible for this procedure are used to prevent or treat diseases of major public health interest.

ATC code

The Anatomical Therapeutic Chemical code: a unique code assigned to a medicine according to the organ or system it works on and how it works. The classification system is maintained by the World Health Organization (WHO).

Attribute

A feature or characteristic of a medical product—such as efficacy or effectiveness, safety, means of administration, duration of effect, or duration of use—that may affect benefit-risk considerations.

Basket (clinical) trial

A basket clinical trial is a type of clinical trial that focuses on testing a single specific treatment (=single targeted therapy) across various diseases or subtypes within a disease (i.e., in cancer, it might involve different types or stages of cancer).  After participants are screened for the presence of a target, target-positive participants are entered into the trial; as a result, the trial may involve many different diseases or disease subtypes.

Benefit

Benefits are the favorable effects of a medical product. Types of benefit include clinical benefit (see definition below). Benefits may also include important characteristics of the medical product, such as convenience (e.g., a more convenient dosing regimen or route of administration) that may lead to improved patient compliance, or benefits that affect those other than the patient.

Benefit-risk assessment

Evaluation of the demonstrated benefits and risks of a medical product and making a judgment as to whether the expected benefits outweigh the potential risks associated with its expected use.

Bioavailability (F)

The extent to which an active ingredient is absorbed from a medicine and becomes available in the body.

Bioequivalence (BE)

When two medicines release the same active ingredient into the body at the same rate and to the same extent under similar conditions.

Bioequivalence study

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

Biological hypothesis

A supposition or tentative explanation about a phenomenon or a narrow set of phenomena observed in the natural world

Biological medicine

A medicine whose active substance is made by a living organism.

Biomarker

A biomarker, or biological marker, is a biological molecule that serves as measurable indicator of some biological state or condition. Biomarkers are often measured and evaluated using blood, urine, or soft tissues to examine normal biological processes, pathogenic processes (diseases), or pharmacologic responses to a therapeutic intervention.

Biomarker qualification

Certification of the acceptability of a biomarker for a specific use in pharmaceutical research and development. The Agency gives opinions on biomarker qualifications.

Biosimilar medicine

A medicine that is similar to a biological medicine that has already been authorised.

Biotechnology (Biotech)

Biotechnology in the medical context refers to the use of biological systems and processes to develop new treatments and therapies for diseases and medical conditions. It encompasses a range of scientific disciplines, including molecular biology, genetics, biochemistry, and microbiology, and utilizes techniques such as recombinant DNA technology and stem cell research to develop innovative medical products and treatments. Medical biotechnology has the potential to revolutionize healthcare by providing new and improved ways to diagnose, treat, and prevent a wide range of medical conditions.

Budget plan (in drug development)

A comprehensive financial plan outlining the projected expenses associated with the various stages of bringing a pharmaceutical product from discovery through to market launch. This plan typically includes costs for research and development, preclinical studies, clinical trials, regulatory submissions, manufacturing, marketing, and post-market surveillance.

Caregiver

A person who helps a patient with daily activities, health care, or any other activities that the patient is unable to perform himself/herself due to illness or disability, and who understands the patient’s health-related needs. This person may or may not have decision-making authority for the patient and is not the patient’s healthcare provider.

CAS number (CAS)

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 classification

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

CE mark (CE)

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

Centralised procedure (CP)

The European Union-wide procedure for the authorisation of medicines, where there is a single application, a single evaluation and a single authorisation throughout the European Union. Only certain medicines are eligible for the centralised procedure.

Centrally authorised product (CAP)

A medicine with a single marketing authorisation issued by the European Commission and valid across the European Union.

Champion

A person who enthusiastically supports, defends, or fights for a person, belief, right, or principle.

Class waiver

An exemption from the obligation to submit a paediatric investigation plan for a class of medicines, such as medicines for diseases that only affect adults. Class waivers are adopted by the Paediatric Committee (PDCO).

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 benefit

A positive clinically meaningful effect of an intervention, e.g., a positive effect on how an individual feels, functions, or survives. 

Clinical development

Clinical Development, also called Drug Development, is a blanket term used to define the entire process of bringing a new drug or device to the market. It includes drug discovery / product development, pre-clinical research (microorganisms/animals) and clinical trials (on humans). Few people still refer to the drug development as mere preclinical development.

Clinical endpoint

A clinical endpoint is an outcome from a clinical trial that represents direct clinical benefit, such as survival, decreased pain, or the absence of disease.

Clinical outcome

An outcome that describes or reflects how an individual feels, functions or survives.

Clinical outcome assessment

Assessment of a clinical outcome can be made through report by a clinician, a patient, a non-clinician observer or through a performance-based assessment. There are four types of COAs: patient-reported outcome (PRO), clinician-reported outcome (ClinRO) measures, observer-reported outcome (ObsRO), and performance outcome (PerfO).

Clinical profile (of a drug)

The clinical profile of a drug refers to the overall information on its safety and efficacy, as well as its pharmacokinetic and pharmacodynamic properties. It includes information on the drug’s dosage and administration, therapeutic indications, target populations, and potential side effects. It also includes data on the drug’s pharmacokinetics, such as its absorption, distribution, metabolism, and elimination, and pharmacodynamics, such as its mechanism of action and its effects on the body.

Clinical relevance

The extent to which an endpoint can capture and measure an aspect of a potential clinical benefit (improvement in how the patient feels, functions, and/or survives) that is important from a clinical perspective and from the patient’s perspective.

Clinical trial (CT)

Any investigation in human subjects intended to discover or verify the safety or efficacy of one or more investigational medical products.  These studies are carried out in human volunteers.

Clinical Trial Application (CTA)

A Clinical Trial Application (CTA) is a submission to the competent National Regularoty Authority(ies) for obtaining authorization to conduct a clinical trial in a specific country. It is an application with necessary information on investigational medicinal products.

Clinical trial authorization

Before a clinical trial can start, the sponsor must apply for and be given clinical trial authorisation (CTA). Each European country has its own regulatory authority that assesses applications for clinical trial authorisations. For clinical trials that will take place in more than one European country, there is a ‘Voluntary Harmonisation Procedure’ which allows one application to be submitted to the authorities in all the relevant countries. In addition to the clinical trial authorisation, a positive opinion from an ethics committee (or institutional review board) is needed before a clinical trial can proceed.

Clinical trial sponsor

A person, company, institution, group, or organization that oversees or pays for a clinical trial and collects and analyzes the data. Also called clinical trial sponsor.

Clinical-reported outcome

A measurement based on a report that comes from a trained health-care professional after observation of a patient’s health condition. Most ClinRO measures involve a clinical judgment or interpretation of the observable signs, behaviors, or other manifestations related to a disease or condition. ClinRO measures cannot directly assess symptoms that are known only to the patient (e.g., pain intensity).

Clinicaltrials.gov

ClinicalTrials.gov is an online resource that provides information about clinical studies on a wide range of diseases. It includes information on whether the studies are recruiting patients, and a summary of the results of studies once they have finished.
Studies can be searched for by disease or by country. Although it is funded by the US government, it includes studies based around the world. The resource is aimed at patients and their families, healthcare professionals, researchers and the public. The website is maintained by the National Library of Medicine (NLM) at the National Institutes of Health (NIH).

Clock stop

A period of time during which the evaluation of a medicine is officially stopped, while the applicant prepares responses to questions from the regulatory authority. The clock resumes when the applicant has sent its responses.

Co-rapporteur

One of the two members of a committee or working party leading the assessment of an application.

Combination ATIMP

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

Commercially confidential information (CCI)

Information whose publication might prejudice the commercial interests of individuals or companies to an unreasonable degree. The Agency cannot disclose commercially confidential information unless there is an overriding public interest in disclosure.

Committee for Advanced Therapies (CAT)

The committee that is responsible for assessing the quality, safety and efficacy of advanced-therapy medicines, including medicines classified as gene therapy, somatic-cell therapy or tissue-engineered products.

Committee for Medicinal Products for Human Use (CHMP)

The committee that is responsible for preparing the Agency’s opinions on questions concerning human medicines.

Committee for Orphan Medicinal Products (COMP)

The committee that is responsible for reviewing applications for orphan designation for medicines that are intended for the diagnosis, prevention or treatment of rare diseases.

Committee for Proprietary Medicinal Products (CPMP)

The name of the Committee for Medicinal Products for Human Use (CHMP) until May 2004.

Committee for Veterinary Medicinal Products (CVMP)

The committee that is responsible for preparing the Agency’s opinions on all questions concerning veterinary medicines.

Committee on Herbal Medicinal Products (HMPC)

The committee that is responsible for establishing Community herbal monographs and preparing the Agency’s opinions on questions relating to herbal medicines.

Common technical document (CTD)

A common format for submitting scientific information when applying for marketing authorisations in the European Union, Japan and the United States.

Comparator

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

Compassionate use (CU)

The use of an unauthorised medicine outside a clinical study in individual patients under strictly controlled conditions. This helps to make medicines that are still under development available to patients.

Competent Authority (CA (NCA))

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.

Concentration unit

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

Concept paper (CP)

A document prepared by a European Medicines Agency working party prior to the drafting of a guideline, setting out the problem, the scope of the work, the resources needed and the timeframe.

Concerned Member State (CMS)

A European Economic Area country in which an application has been submitted for authorisation of a medicine through the mutual recognition or decentralised procedure. The assessment of the medicine is performed by another country called the reference Member State.

Condition

A “condition” or “medical condition” refers to the health issue or disease affecting a patient, such as diabetes or Hepatitis C. “Condition” and “disease” are related but not exactly the same. “Condition” is a broader term that can includes diseases but also refers to other states of health that may not be classified as diseases, such as pregnancy or allergies. In summary, while all diseases can be considered conditions, not all conditions are classified as diseases.

Conditional marketing authorisation (CMA)

The approval of a medicine that addresses unmet medical needs of patients on the basis of less comprehensive data than normally required. The available data must indicate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data in the future.

Controlled Clinical Trial (CCT)

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.

Coordination Group for Mutual Recognition and Decentralised Procedures – Human (CMDh)

The group responsible for the examination and coordination of questions relating to the marketing authorisation of human medicines in two or more Member States in accordance with the mutual recognition or decentralised procedure.

Coordination group for mutual recognition and decentralised procedures for veterinary medicinal products (CMDv)

The group responsible for the examination and coordination of questions relating to the marketing authorisation of veterinary medicines in two or more Member States in accordance with the mutual-recognition or decentralised procedure.

Corporate Social Responsibility (CSR)

CSR refers to the responsibility of businesses and corporations to consider the impact of their operations on society and the environment, and to take actions that promote sustainable development and improve the well-being of individuals and communities.

Counterfeit medicine

A medicine made by someone other than the genuine manufacturer, by copying or imitating an original product without authority or rights. Counterfeit medicines infringe trademark law.

Countries in which trial sites are planned

Country(s) where clinical trials are planned to be conducted

Country which granted the Marketing Authorisation

Name of the country where the holder was granted the Marketing Authorisation of the actual IMP to be used in the clinical trial in the member state concerned by the application.

Critical path

Group of tasks and steps that are necessary for the completion of a project.

Cross over

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

Current sponsor code

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

Data curation

Data curation is the process of creating, organizing and maintaining data sets so they can be accessed and used by people looking for information. It involves collecting, structuring, indexing and cataloging data for users in an organization, group or the general public. Data can be curated to support business decision-making, academic needs, scientific research and other purposes.

Data exclusivity

The period of eight years from the initial authorisation of a medicine during which the marketing-authorisation holder benefits from the exclusive rights to the results of preclinical tests and clinical trials on the medicine. After this period, the marketing authorisation holder is obliged to release this information to companies wishing to develop generic versions of the medicine.

Data Management Plan

A written document that describes the data you expect to acquire or generate during the course of your research study; how you intend to manage, describe, analyze, and store data; and what mechanisms you will use at the end of your study to preserve and share your data.

Data Monitoring Committee (DMC)

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 processing agreement (DPA)

A data processing agreement (“DPA”) needs to be in place when a data controller engages a data processor. The DPA sets out the relationship between the two parties and the data being processed.

Data protection impact assessment (DPIA)

Data protection impact assessment (DPIA) is a process to identify and mitigate data protection risks. If a processing activity is likely to result in a high risk to individuals, you need to do a DPIA. The main goal is to minimise the identified and predicted risks of the processing activity.

Data sharing agreement (DSA)

A Data Sharing Agreement is a contract that documents what data is being shared and how it can be used. It can be used to make data sharing lawful.

Date of Competent Authority
Decision

This is the date on which the National Competent Authority Decision was made on clinical trial.

Date of Ethics Committee Opinion

This is the date on which the Independent Ethics Committee Opinion was given on clinical trial.

Date of the global end of the trial

This is the date on which the trial is ended in all countries.

Decentralized procedure (DCP)

The procedure for authorising medicines in more than one European Union Member State in parallel. It can be used for medicines that do not need to be authorised via the centralised procedure and have not already been authorised in any Member State.

Declaration of interests (DOI)

The provision of information on all potential conflicts of interests by an individual, including recent work activities, investments and family connections with the pharmaceutical industry.

Deferral (paediatric)

The possibility to defer a measure in a paediatric investigation plan until after studies in adults have been conducted. This ensures that research in children is done only when it is safe and ethical to do so. Deferrals are adopted by the Paediatric Committee (PDCO).

Deliverable

Deliverables are additional outputs (e.g., information, special report, a technical diagram brochure, list, a software milestone or other building block of the project) that must be produced at a given moment during the action (Milestones are, by contrast, control points in the project that help to chart progress).

Diagnosis

1. the determination of the nature of a case of disease.
2. the art of distinguishing one disease from another.

Direct healthcare-professional communication

A letter sent directly to individual healthcare professionals by a marketing authorisation holder or a regulatory authority, containing information or advice regarding the use of a medicine.

Disease burden

For patients and their close environment: The impacts, direct and indirect, of the patient’s health condition that has a negative effect on his or her health, functioning, and overall well-being. Disease burden includes (but is not limited to): the physical and physiologic impacts of the disease and its symptoms; co-morbidities; emotional and psychological effects of the disease, its management, or prognosis; social impacts; effects on relationships; impacts on the patient’s ability to care for self and others; time and financial impacts of the disease and its management; and considerations on the impacts on the patient’s close environment (family, friends, carers communities and residence).

– For society at the regional or global level: The impact of the disease in terms of incidence, prevalence, morbidity and mortality in a population. – For the health and social care system: The impact of the disease in terms of health & social care resource use and costs.

Disincentive

Something that makes a person or organization less willing to do something.

Dissemination plan

A structured strategy for sharing information, knowledge, or content with specific target audiences or stakeholders. This plan outlines how, when, and through which channels information will be distributed to ensure maximum reach, engagement, and impact. Dissemination plans are commonly used in various fields such as research, public relations, marketing, and education to effectively communicate messages and achieve organizational goals.

Dosage form

The form of the completed pharmaceutical product, e.g. tablet, capsule, injection, elixir, suppository.

Dose

The highest single oral IR dose recommended for administration in the summary of Product Characteristics (also known as the Prescribers’ Information) (based on EMA definition).

Dose-response (DR)

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) don’t know which treatment is given.

Drug effectiveness

Ability of a drug to produce the desired therapeutic effect under real-world circumstances.

Drug efficacy

Ability of a drug to produce the desired therapeutic effect under ideal circumstances.

Drug master file (DMF)

A Drug Master File (DMF) is a submission to the Food and Drug Administration (FDA) that may be used to provide confidential detailed information about facilities, processes, or articles used in the manufacturing, processing, packaging, and storing of one or more human drugs. The submission of a DMF is not required by law or FDA regulation. A DMF is submitted solely at the discretion of the holder. The information contained in the DMF may be used to support an Investigational New Drug Application (IND), a New Drug Application (NDA), an Abbreviated New Drug Application (ANDA), another DMF, an Export Application, or amendments and supplements to any of these.

Drug product (DP)

Finished dosage form, e.g., tablet, capsule, or solution, that contains a drug substance, generally, but not necessarily, in association with one or more other ingredients.

Drug redirection

See Drug Repurposing or repositioning/rescue definition

Drug rediscovery

See Drug Repurposing or repositioning/rescue definition.

Drug repositioning/rescue (DR)

Finding a new use/indication for a (de-risked) drug molecule (i.e., drug substance for which human safety data are available). Generally, requires pharmaceutical product development (e.g., reformulation/dose adjustment), following redirection/bridging of an active drug development program, revival of an abandoned/shelved compound/off-market drug product, or a drug screening campaign to exploit alternative drug pathways/targets. May include drug combinations.

Drug reprofiling

See Drug Repurposing or repositioning/rescue definition

Drug repurposing (DR)

Finding a new use/indication for an existing/licensed medicinal drug product (applying approved dosage forms/strengths). Generally, does not require additional pharmaceutical product development. May include drug combinations.

Drug reuse

See Drug Repurposing definition.

Drug safety

Drug safety refers to the frequency and severity of adverse drug effects (i.e., physical or laboratory toxicity that could possibly be related to the drug) that are treatment emergent—that is, they emerge during treatment and were not present before treatment, or they become worse during treatment compared with the pre-treatment state.

Drug substance (DS)

See API definition.

Early Career Researcher/Early Stage Researcher (ECR/ESR)

ECR or ESR are researchers in the first four years (full-time equivalent research experience) of their research careers that have not been awarded a doctoral degree.

Efficacy

The measurement of a medicine’s desired effect under ideal conditions, such as in a clinical trial.

Elderly

Subjects are aged 65 years or more.

Electronic common technical document (eCTD)

A common technical document in electronic format.

EMA Decision number of PIP

European Medicines Agency’s decision number for the Paediatric Investigation Plan (PIP)

Emergency situation

Situation where urgent care is needed for the patient and this involves enrolment in the trial (for example: head injury).

Endpoint

A precisely defined variable intended to reflect an outcome of interest that is statistically analyzed to address a particular research question. A precise definition of an endpoint typically specifies the type of assessments made, the timing of those assessments, the assessment tools used, and other details. When a trial evaluates the efficacy of a new medical product or a new use for an approved product, investigators may choose endpoints that directly measure the clinical outcome they want to study. Alternatively, they may choose an endpoint that is a substitute, or “surrogate”, for the outcome they want to study (see Surrogate endpoint).

Environmental risk assessment (ERA)

Analysis of the potential risk that the use of a medicine poses to the environment.

Equity (Health equity)

Equity is the absence of unfair, avoidable or remediable differences among groups of people, whether those groups are defined socially, economically, demographically, or geographically or by other dimensions of inequality (e.g. sex, gender, ethnicity, disability, or sexual orientation). Health equity is achieved when everyone can attain their full potential for health and well-being.

Ethics Committee (EC)

Independent reviewing body that considers and approves/disapproves biomedical research involving human subjects. Ethics Committee approval is required for each clinical study protocol and Principal Investigator prior to study initiation.

EudraCT number

When registered, each trial is issued with a unique EudraCT number, which identifies the protocol and trial throughout its lifespan.

EudraVigilance

A centralised European database of suspected adverse reactions to medicines that are authorised or being studied in clinical trials in the European Economic Area (EEA).

European Commission decision

The legally binding decision issued by the European Commission at the end of a regulatory procedure, such as a marketing authorisation application or arbitration procedure. A European Commission decision comes after an opinion from one of the Agency’s scientific committees.

European Drug Regulatory Network (EudraNet)

A secure communication infrastructure linking European medicines regulatory authorities.

European Economic Area (EEA)

The EEA consists of the Member States of the European Union (EU) and three countries of the European Free Trade Association (EFTA) (Iceland, Liechtenstein and Norway; excluding Switzerland).
It seeks to strengthen trade and economic relations between the contracting parties and is principally concerned with the four fundamental pillars of the internal market, namely: the free movement of goods, people, services and capital.

European medicines regulatory network

The system for regulating medicines in Europe is based on a closely coordinated regulatory network of national competent authorities in the Member States of the European Economic Area working together with the European Medicines Agency and the European Commission.

European Network for Health Technology Assessment (EuneHTA)

Network established to create an effective and sustainable structure for health technology assessment (HTA) across Europe that could develop and implement practical tools to provide reliable, timely, transparent and transferable information to contribute to HTAs in Member States.

European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP)

A network of European centres in the fields of pharmacoepidemiology and pharmacovigilance, which aims to strengthen the postauthorisation monitoring of medicines by facilitating the conduct of multicentre, independent, postauthorisation studies focusing on safety and on benefit-risk balance. It is coordinated by the European Medicines Agency.

European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA)

A network of research networks, investigators and centres with recognised expertise in performing clinical studies in children.

European Pharmacopoeia (EP)

A compendium of texts on the qualitative and quantitative composition of medicines, and on the tests to be carried out on medicines, on the raw materials used in the production of medicines and on the intermediates of synthesis.

European public assessment report (EPAR)

A set of documents describing the evaluation of a medicine authorised via the centralised procedure and including the product information, published on the European Medicines Agency website. European public assessment reports include the product information.

European Review System (EURS)

An electronic tool for the receipt, validation, storage and provision of electronic documents for marketing authorisation applications and postauthorisation applications.

European Union Clinical Trials Register (EUCTR)

A website that contains public information on interventional clinical trials on medicines.

European Union Drug Regulating Authorities Clinical Trials Database (EudraCT)

EudraCT is a database that includes information on clinical trials taking place in the European Union and clinical studies conducted worldwide in accordance with a paediatric investigation plan. A subset of the data is publicly accessible via the European Clinical Trials Register.

European Union reference dates (EURD) list (EURD list)

The European Union reference dates (EURD) list is a comprehensive list of active substances and combinations of active substances contained in medicinal products subject to different marketing authorisations, together with the corresponding EU reference dates, frequencies for submission of periodic safety update reports and related data lock points.

EV Product Code

Marketed Product’s EudraVigilance Number.

EV Substance Code

Active Substance’s EudraVigilance Substance Code.

Exceptional circumstances

A type of marketing authorisation granted to medicines where the applicant is unable to provide comprehensive data on the efficacy and safety under normal conditions of use, because the condition to be treated is rare or because collection of full information is not possible or is unethical.

Excipient

A constituent of a medicine other than the active substance.

Exploratory clinical trial

Exploratory trials are developed to select compounds or targeted medicines or approaches based on human data rather than animal data. They are conducted early in Phase I of the clinical development process, which involve limited human exposure, have no therapeutic intent, and are not intended to define a maximum tolerated dose.

Extractive medicinal product

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

Failure Mode and Effect Analysis (FMEA)

FMEA is an ad hoc risk analysis tool wherein all project-related risks are listed and reviewed in terms of their severity, probability of occurrence and probability of detection. When assigning arbitrary numbers to these parameters, the product resulting from them gives a risk prioritization number that in turn allows the classification of risk. Early risk mitigating actions will lower this number and therefore minimize the associated risk.

FAIR guiding principles for scientific data management and stewardship (FAIR)

Guidelines to improve the Findability, Accessibility, Interoperability, and Reuse of digital assets. The principles emphasise machine-actionability (i.e., the capacity of computational systems to find, access, interoperate, and reuse data with none or minimal human intervention) because humans increasingly rely on computational support to deal with data as a result of the increase in volume, complexity, and creation speed of data.

FAIRification

Process that aims at addressing the translation of raw datasets into FAIR datasets in a general way, without considering specific requirements and challenges that may arise when dealing with some particular types of data.

Falsified medicine

A fake medicine that passes itself off as a real, authorised medicine.

First human administration

Conducting the first dose in human phase I clinical trial.

First stage researcher (Up to the point of PhD) (R1)

This profile includes individuals doing research under supervision in industry, research institutes or universities. It includes doctoral candidates.
Researchers with this profile will:

• Carry out research under supervision;
• Have the ambition to develop knowledge of research methodologies and discipline;
• Have demonstrated a good understanding of a field of study;
• Have demonstrated the ability to produce data under supervision;
• Be capable of critical analysis, evaluation and synthesis of new and complex ideas;
• Be able to explain the outcome of research (and value thereof) to research colleagues.

Desirable competences:

• Develops integrated language, communication and environment skills, especially in an international context.

First-in-human clinical trial (FIH)

First-in-human trials are a key step in medicines development, where a medicine already tested in vitro, in animals or in other preclinical studies is administered to people for the first time. Participants in these trials, often healthy volunteers, face an element of risk as the ability of researchers to predict the effects of a new medicine on people is limited before it is actually studied in humans.

Formulation

Any mixture or substance prepared according to a particular formula

Freedom to operate (FTO)

Freedom to operate (FTO) is the ability of your Company to develop, make, and market products without legal liabilities to third parties (e.g., other patent holders).

Full title of the trial

The title as specified in the study protocol and other documents 
submitted as part of the Clinical Tral Application.

Functional name of contract point

The point of contact for further information on the trial (e.g. “Clinical Trial Information Desk”).

Gene therapy medicinal product

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

Gene therapy medicine

A medicine that delivers genes into the body.

General Data Protection Regulation (GDPR)

The General Data Protection Regulation (GDPR) is a legal framework that sets guidelines for the collection and processing of personal information from individuals who live and outside of the European Union (EU). Approved in 2016, the GDPR went into full effect two years later. Its aim is to give consumers control over their own personal data by holding companies responsible for the way they handle and treat this information. The regulation applies regardless of where websites are based, which means it must be heeded by all sites that attract European visitors, even if they don’t specifically market goods or services to EU residents.

Generic drug/medicine

Generic medicines are those where the original patent has expired and which may now be produced by manufacturers other than the original innovator (patent-holding) company.

Generic medicine

A generic medicine is a medicine that is developed to be the same as a medicine that has already been authorised. Its authorisation is based on efficacy and safety data from studies on the authorised medicine. A company can only market a generic medicine once the 10-year exclusivity period for the original medicine has expired.

Genetically Modified Organism (GMO)

A genetically modified organism (GMO) or genetically engineered organism (GEO) is an organism whose genetic material has been altered using genetic engineering techniques.

Good clinical practice (GCP)

A code of international standards concerning the design, conduct, performance, monitoring, auditing, recording, analysis and reporting of clinical trials. Good clinical practice provides assurance that a study’s results are credible and accurate and that the rights and confidentiality of the study subjects are protected.

Good distribution practice (GDP)

A code of standards ensuring that the quality of a medicine is maintained throughout the distribution network, so that authorised medicines are distributed to retail pharmacists and others selling medicines to the general public without any alteration of their properties.

Good laboratory practice (GLP)

A code of standards concerning the testing of medicines in laboratories during their development.

Good Manufacturing Practice (GMP)

GMP is that part of quality assurance which ensures that medicinal products are consistently produced and controlled to the quality standards (concerning manufacturing, processing, packing, release and holding) appropriate to their intended use and as required by the marketing authorisation (MA) or product specification.

Good Manufacturing Practice Certificate (GMPC)

GMP Certificate. A Certificate issued to a manufacturer who has met the standards laid out under the GMDP guidelines.

Good Pharmacovigilance Practice (GVP)

A set of measures drawn up to facilitate the performance of the safety monitoring of medicines in the European Union. Abbreviated as GVP.

Good x Practice (GxP)

The GxP is a collection of quality guidelines and regulations to ensure medical product safety; their intended use; and quality processes during clinical development, manufacturing, and distribution

Grouping

The possibility for a marketing authorisation holder to submit more than one variation for a medicine in a single application.

Guideline

A document providing guidance on the scientific or regulatory aspects of the development of medicines and applications for marketing authorisation. Although guidelines are not legally binding, applicants need to provide justification for any deviations.

Heads of Medicines Agencies (HMA)

The network of the heads of the regulatory authorities responsible for the regulation of human and veterinary medicines in the European Economic Area.

Health technology assessment (HTA)

Health technology assessment (HTA) refers to the systematic evaluation of properties, effects, and/or impacts of health technology. It is a multidisciplinary process to evaluate the social, economic, organizational and ethical issues of a health intervention or health technology.The main purpose of conducting an assessment is to inform a policy decision making.

Health technology assessment body

A public organisation that provides recommendations on the medicines and other healthcare interventions that can be paid for or reimbursed. These organisations look at the relative effectiveness and cost effectiveness of medicines that have been authorised.

Healthy volunteers (HVs)

Clinical trial includes subjects in good health

Herbal medicinal product (HMP)

Any medicinal product, exclusively containing as active ingredients one or more herbal substances or one or more herbal preparations, or one or more such herbal substances in combination with one or more such herbal preparations.

Human Pharmacology

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.

Hybrid medicine

A medicine that is similar to an authorised medicine containing the same active substance, but where there are certain differences between the two medicines such as in their strength, indication or pharmaceutical form.

Hypothesis

A supposition or proposed explanation made on the basis of limited evidence as a starting point for further investigation.

Hypothesis (biological)

A supposition or tentative explanation about a phenomenon or a narrow set of phenomena observed in the natural world

Hypothesis (mechanistic)

A hypothesis about the biological processes that are thought to have given rise (or are thought to give rise) to a certain outcome.

Hypothesis (therapeutic)

Therapeutic hypothesis is a scientifically reasonable association of a Druggable Target with a disease/therapeutic area where such association is based on scientific evidence.

Immunological Medicinal Product

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

Impact investor

An impact investor in the biomedical field is an individual or organization that invests capital into companies or projects that are focused on developing and commercializing innovative medical technologies and treatments. The goal of impact investing in the biomedical field is to support the development of new treatments and therapies that have the potential to improve human health, while also generating a financial return on investment. Impact investors in the biomedical field typically focus on investments in early-stage companies or projects that have the potential to have a significant impact on human health, such as companies developing new treatments for diseases with high unmet medical needs, or companies working on innovative medical technologies with the potential to revolutionize the way that healthcare is delivered. The focus of impact investing in the biomedical field is not just on financial returns, but also on creating positive social and environmental impact. For example, an impact investor in the biomedical field might invest in a company that is working on developing a new treatment for a neglected tropical disease, or a company that is working to make healthcare more accessible and affordable in low- and middle-income countries. Overall, impact investing in the biomedical field provides an important source of capital for early-stage companies and projects, and it helps to support the development of innovative treatments and therapies that have the potential to make a significant impact on human health.

In silico drug repurposing

In silico drug repurposing/repositioning is the use of bioinformatics tools and resources to predict relationships between drugs, their treatment efficacy and side effects and to systematically identify interaction networks between drugs and protein targets based on literature mining and experimental screenings.

In utero

Subjects are unborn infants, still in the womb.

In vitro

“Within the glass”. A phenomenon or procedure that happens or is performed outside a living organism.

In vivo

Within the living body (animal/man). A phenomenon or procedure that occurs or is performed within a living organism.

Incentives

Something, especially money, that encourages a person or organization to do something:

Independent Ethics Committee (IEC)

Independent Ethics Committee means an independent body (a review board or a committee, institutional, regional, national or supranational), consisting of medical/scientific professionals and non-medical/non-scientific members, whose responsibility is to ensure the protection of the rights, safety and well-being of human subjects involved in a clinical trial and to provide public assurance of that protection, by, among other things, reviewing and approving/providing favourable opinion on the Protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting the informed consent of Study Patients.

Indication

An “indication” refers to the specific reason or purpose for which a drug, treatment, or procedure is used or developed. Example: Aspirin is indicated for the prevention of heart attacks in individuals at risk for cardiovascular disease.

A single medical condition can have several indications, meaning that different treatments may be used to address various aspects of the condition, depending on the specific symptoms, severity, or stage of the disease. Example In Chronic Obtrusive Pulmonary Disease (COPD):

• Albuterol is indicated for acute relief of bronchospasm in COPD, providing immediate symptom relief during flare-ups.
• Tiotropium is indicated for the long-term management of COPD, helping to control symptoms on a daily basis and prevent exacerbations.

Individual case safety report (ICSR)

A document providing information related to an individual case of a suspected side effect due to a medicine.

Infants and toddlers

Subjects are aged 28 days to less than 2 years.

Informed consent application

An application where the reference medicine’s marketing authorisation holder has consented to the use of the reference medicine’s data for the application.

INN – proposed INN (INN)

The International Non-proprietary name for an active substance.

Innovation task force (ITF)

The Innovation Task Force (ITF) is a multidisciplinary group that includes scientific, regulatory and legal competences. It was set up to ensure coordination across the Agency and to provide a forum for early dialogue with applicants on innovative aspects in medicines development.

Innovative Licensing and Access Pathway (ILAP)

The Innovative Licensing and Access Pathway ( ILAP ) aims to accelerate the time to market, facilitating patient access to medicines. These medicines include new chemical entities, biological medicines, new indications and repurposed medicines.

Intellectual property (IP)

Intellectual property is a legal right to control the application of an idea in a specific context (through a patent) or to control the expression of an idea (through a copyright). Patent and copyright protections are legal mechanisms that seek to strike a balance between private gains and public benefits.

Interactive Voice Response system (IVRS)

Interactive Voice Response System: commonly used for randomisation of treatment and controlling the shipment of stock of product.

International birth date (IBD)

The date of the first marketing authorisation for a medicine in any country in the world.

International Council for Harmonisation (ICH)

The International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) brings together regulatory authorities and the pharmaceutical industry. It makes recommendations towards achieving greater harmonisation in the interpretation and application of technical guidelines and requirements for pharmaceutical product registration.

International non-proprietary name (INN)

The globally recognised name used to identify the active ingredient in a medicine.

International Standard Randomised Controlled Trial Number (ISRCTN)

International Standard Randomised Controlled Trial Number is used for the identification of clinical trials worldwide. The randomly generated, eight-digit ISRCTN is unique to a registered trial at current controlled trials, thereby ensuring that the trial can be simply and unambiguously tracked throughout its lifecycle from initial protocol to results publication.

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.

Investigational medicinal product (IMP)

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 (formulated or packaged) in a way different from the authorised form, or when used for an unauthorised indication, or to gain further information about the authorised form.

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.

Investigator-Initiated Studies (IIS)

IISs are clinical studies initiated and managed by a nonpharmaceutical company researcher/s who could be an individual investigator, an institution or a group of institutions, and a collaborative study group or a cooperative group.  IISs are also known by several other names that include Investigator-Initiated Trials, Investigator-Sponsored Trials, Noncommercial Trials, Academic Clinical Trials, Physician-Led Studies and Investigator-Driven Clinical Trials, Academic. The term investigator can also be substituted by the term “Academic” or “Physician” and the term “Clinical trial” can be replaced by “Study.”The fundamental premise here is that the responsibility of being both the sponsor and investigator lies with the person who conceives of and conducts the study.

Investigator-sponsored Trial (IST)

ISTs are clinical studies initiated and managed by a nonpharmaceutical company researcher/s who could be an individual investigator, an institution or a group of institutions, and a collaborative study group or a cooperative group.  ISTs are also known by several other names that include Investigator-Initiated Trials, Investigator-Initiated studies, Noncommercial Trials, Academic Clinical Trials, Physician-Led Studies and Investigator-Driven Clinical Trials, Academic. The term investigator can also be substituted by the term “Academic” or “Physician” and the term “Clinical trial” can be replaced by “Study.”The fundamental premise here is that the responsibility of being both the sponsor and investigator lies with the person who conceives of and conducts the study.

Killer experiment

Experimental study addressing a key research question designed in such a way that the conclusions from this study allow a decision to either stop or progress a project, before significant resources are put into it.

Labelling

Information on the immediate or outer packaging of a medicine.

Legal Representative of Sponsor

An individual, company, institution, or organisation authorised to act on behalf of the sponsor of a clinical trial. Must be based in the EU or the EEA, which includes Iceland, Norway, and Liechtenstein.

License (Drug license)

A drug license, also known as a drug registration or marketing authorization, is a legal document or approval granted by the regulatory agency of a country or region that allows a pharmaceutical company or manufacturer to legally produce, market, and sell a drug within that jurisdiction. The drug license indicates that the drug has undergone appropriate testing and evaluation for safety, quality, and efficacy and meets the necessary standards and regulations required for its use in that specific country or region. Obtaining a drug license is an essential step for pharmaceutical companies to bring their products to the market and ensure that they comply with regulatory requirements to protect public health.

Licensing

In the pharmaceutical industry, licensing is the transfer of rights to a third party (the Licensee) to use the Intellectual Property owned by an innovator (the Licensor) under mutually agreed terms and conditions. Licensing can be for various transactions like manufacturing and/or marketing rights in select geographies, renewable after the initially agreed period.

List of Outstanding Issues (LoOI)

A set of questions addressed to a company during a procedure, such as the evaluation of a marketing authorisation application. Lists of outstanding issues are prepared after a company has already responded to a list of questions.

Low-Intervention clinical trial

A low-intervention clinical trial studies an authorised medicine. Its use according to the trial protocol follows the terms of the marketing authorisation, or published scientific evidence on safety and efficacy. Any additional procedures must not pose more than minimal additional risk or burden to the safety of the participants compared to normal local clinical practice. Low-intervention clinical trials are used for example to investigate safety and efficacy questions that have arisen since authorisation.

Magistral medicine/Magistral preparation/Magisterial preparation

Magistral medicines are medicinal products prepared in a pharmacy for an individual patient or animal in accordance with a prescription from a doctor or veterinarian

Manufacture

All operations of purchase of materials and products, Production, Quality Control, release, storage, distribution of medicinal products and the related controls

Manufacturer

Holder of a Manufacturing Authorisation as described in Article 40 of Directive 2001/83/EC for human products and Article 44 of Directive 2001/82/EC for veterinary products.

Manufacturer’s/Importation Authorization (MIA)

A Manufacturer’s/Importation Authorisation (MIA) is required if a company is involved in manufacturing and/or importation activities relating to human, veterinary or investigational medicinal product, within the EU.

Manufacturing Authorization

Required prior to the commencement of production – application will trigger a GMDP inspection.

Market Exclusivity

The 10-year period after the marketing authorisation of an orphan medicine when similar medicines for the same indication cannot be placed on the market.

Market protection

The protection of an approved medicine against competition from generic medicines that extends beyond the protection conferred by data exclusivity. During this period, applications for generics can be accepted and authorised, but the generic medicines cannot be placed on the market.

Marketing authorization (MA)

The approval to market a medicine in one, several or all European Union Member States.

Marketing Authorization Application (MAA)

Marketing authorization Application. Across all European markets, plus Australia, New Zealand, South Africa, and Israel (exceptions amongst major markets include USA, Canada, China and Japan), the Marketing authorisation Application (MAA) is a common document used as the basis for a marketing application (an application for approval to market the product based on a full review of all quality, safety, and efficacy data, including clinical study reports). In the USA, the New Drug Application (NDA) is the MAA equivalent. In Canada, the New Drug Submission (NDS) is the MAA equivalent. An MAA is comprised of 4 parts:

Part 1: Summary of the Dossier includes application forms, summary of Product Characteristics, packaging, Expert Reports.

Part 2: Chemical, Pharmaceutical, and Biological Documentation: drug substance and drug product

Part 3: Pharmacological and Toxicological (Preclinical) Documentation is a report of all animal pharmacology, toxicology, and pharmacokinetics studies.

Part 4: Clinical Documentation is a report of all Phase I, II, III, IV, V clinical studies conducted up to the time of submission.

Marketing authorization holder (MAH)

The company or other legal entity that has the authorisation to market a medicine in one, several or all European Union Member States.

Master data

Any non-transactional information considered to play a key role in the core operation of a business and re-used for multiple purposes.

Master protocol

A master clinical protocol is an overarching clinical trial protocol designed to answer clinical multiple questions. Master protocols may involve one or more interventions in multiple diseases or a single disease. There are three distinct entities included under this broad definition: umbrella, basket, and platform clinical trials.

Material Transfer Agreement (MTA)

A Material Transfer Agreement (MTA) is a contract that governs the transfer of tangible research materials between two organizations, when the recipient intends to use it for his or her own research purposes. The MTA defines the rights of the provider and the recipient with respect to the materials and any derivatives. Biological materials, such as reagents, cell lines, plasmids, and vectors, are the most frequently transferred materials, but MTAs may also be used for other types of materials, such as chemical compounds and even some types of software.

Maximum residue limit (MRL)

The maximum concentration of a medicine residue that is considered acceptable in food produced from an animal that has been treated with that medicine.

Mechanism of action (for a drug/medicine) (MOA)

In pharmacology, the term mechanism of action (MOA) refers to the specific biochemical interaction through which a drug substance produces its pharmacological effect. A mechanism of action usually includes mention of the specific molecular targets to which the drug binds, such as an enzyme or receptor. Simplified, MOA is a term used to describe how a drug/medicine produces an effect in the body.

Mechanistic hypothesis

A hypothesis about the biological processes that are thought to have given rise (or are thought to give rise) to a certain outcome.

Medical condition(s) investigated

Description of intended indication for the product under development.

Medical Dictionary for Regulatory Activities (MedDRA)

Key to MedDRA hierarchical levels (highest to lowest) SOC (System Organ Class) HLGT (High Level Group Term) HLT (High Level Term) PT (Preferred Term) LLT (Lowest Level Term).

Medicinal product

A substance or combination of substances that is intended to treat, prevent or diagnose a disease, or to restore, correct or modify physiological functions by exerting a pharmacological, immunological or metabolic action.

Medicine repurposing

Finding a new use/indication for an existing/licensed medicinal drug product (applying approved dosage forms/strengths). Generally, does not require additional pharmaceutical product development. May include drug combinations.

Member State (MS)

A country member of the European Union.

Milestone

A milestone is an event of special importance. Milestones are predefined, important incidents during project conduct with special interest to the project, the user or the project organization. Milestones represent the project situation and mark a key development stage at a given time point.

Minor use

A type of veterinary medicine that is to be used in small numbers of animals or in animals of low individual economic value, such as honey bees.

Mock-up

A copy of the flat artwork design for a medicine’s inner and outer packaging, in full colour, that can be assembled into a three-dimensional replica.

Mock-up vaccine

A vaccine that contains a strain of flu virus which could cause a pandemic. The mock-up procedure allows a vaccine to be developed in advance of a pandemic.

Modelling and simulation

Techniques that use mathematical models to understand and predict the outcomes of interventions.

Multinational trial

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

Multiple Member States

This is where the trial will be conducted in more than one Member 
State of the European Economic Area.

Mutual recognition (MR)

A community registration procedure described by Council Directive 75/319/EEC (as amended) for the authorisation of medicinal products. Mutual Recognition Procedure: One of the routes for seeking regulatory approval in the European Union. A submission is first made to a EU Member State authority that assesses, grants a national approval and prepares an assessment report. This report is circulated by the initial authority to the other (concerned) Member States who are expected to recognize this decision and grant their own national authorisation within a period of 90 days following the initial approval. The 90-day period is used to resolve any issues between Member States. If serious objections are raised then the application is referred to the CHMP for arbitration leading to a binding decision.

Note: Concerned Member State: A Member State that is concerned (i.e. included in the mutual recognition phrase) with an application for Mutual Recognition, and expected to recognize the initial approval of the Reference Member State.

Mutual Recognition Agreement (MRA)

An agreement between two regulatory agencies to recognise each other’s regulatory assessments, inspections or reviews.

Mutual-recognition procedure

A procedure through which an authorisation of a medicine in one European Union Member State is recognised by another Member State.

Nanotechnology

The use of tiny structures less than 1,000 nanometres across, which are designed to have specific properties.

National competent authority (NCA) (NCA)

A medicines regulatory authority in a European Union Member State.

Nationally authorised product

A medicine authorised in a Member State in accordance with its national authorisation procedure.

Newborns (0-27 days)

Subjects are newborn babies from birth to less than 28 days of age

Non-compliance report (NCR)

A non-conformance report (also called NCR or non-conformity report) is used to document non-conformances within an organization.
Nonconformance occurs when a product, service, or process is not following the industry specifications.

Note for guidance

A type of document intended to provide guidance on an aspect of a regulatory procedure.

Notified body (NoBo)

An accredited body that conducts conformity assessments for medical devices

Number of sites anticipated in Member State concerned

This is number of sites in the Member State concerned where the trial will take place.

Number of sites anticipated in the EEA

Number of investigator sites in the European Economic Area where the trial is planned to take place

Number of sites anticipated outside of the EEA

Number of investigator sites outside the European Economic Area where the trial is planned to take place.

Number of treatment arms in the trial

Any of the treatment groups in a randomized trial. Most randomized trials have two “arms,” but some have three “arms,” or even more.

Nursing women

Clinical trial includes women subjects who are breastfeeding.

Observer-reported outcome

A measurement based on a report of observable signs, events or behaviours related to a patient’s health condition by someone other than that patient or a health professional. Generally, ObsROs are reported by a parent, caregiver, or someone who observes the patient in daily life and are particularly useful for patients who cannot report for themselves (e.g., infants or individuals who are cognitively impaired). An ObsRO measure does not include medical judgement or interpretation. Examples of ObsROs include a parent report of a child’s vomiting episodes or a report of wincing thought to be the result of pain in patients who are unable to report for themselves.

Off-label use

Use of a medicine for an unapproved indication or in an unapproved age group, dosage, or route of administration.

Off-market use

Use of a medicine for an unapproved indication when this has been withdrawn from the market.

Off-patent drug

Medicine on which there are no exclusive marketing rights because the patent has expired. When a drug’s patent expires, manufacturers other than the initial developer may take advantage of an abbreviated approval process to introduce lower-priced generic versions.

Official medicines control laboratory (OMCLs)

A European laboratory, independent from pharmaceutical companies, that supports regulatory authorities by controlling the quality of medicinal products for human or veterinary use.

On-label use

Use of a medicine in the way described on the approved drug label. Approved drug labels include information on the specific diseases and conditions that a drug is approved to treat or prevent and how to use the drug to treat or prevent those specific diseases and conditions. [Definition slightly modified from the original definition in the source].

On-patent drug

An on-patent drug is a medicine covered under patent protection, meaning  that only the pharmaceutical company that holds the patent is allowed to manufacture, market the drug and eventually make profit from it.

Open trial

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

Oral explanation

A presentation and question-and-answer session in person between representatives of an applicant and a European Medicines Agency committee.

Orphan drug

An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. A drug for the treatment of a rare disease (affecting fewer than 200,000 people in the US or fewer than 5 in every 10,000 individuals in the EU) or for a disease not likely to generate sufficient profit to justify Research and development costs

Orphan Drug Designation (ODD)

A status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the market.

Orphan medicine

A medicine for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare (affecting not more than five in 10,000 people in the European Union) or where the medicine is unlikely to generate sufficient profit to justify research and development costs.

Package leaflet (PL)

The leaflet in every pack of medicine that contains information on the medicine for end-users, such as patients and animal owners.

Packaging

All operations, including filling and labelling, which a bulk product has to undergo in order to become a finished product.Note: Sterile filling would not normally be regarded as part of packaging, the bulk product being the filled, but not finally packaged, primary containers.

Paediatric Committee (PDCO)

The committee that is responsible for assessing the content of paediatric investigation plans, which describe how a medicine should be studied in children, as well as waivers and deferrals.

Paediatric investigation plan (PIP)

A development plan aimed at ensuring that the necessary data are obtained to support the authorisation of a medicine for children, through studies in children. All applications for marketing authorisation for new medicines have to include the results of studies as described in an agreed paediatric investigation plan, unless the medicine is exempt because of a deferral or waiver

Paediatric-use marketing authorisation (PUMA)

A type of marketing authorisation covering the indication(s) and appropriate formulation(s) for the paediatric population, which was introduced by the Paediatric Regulation. Applicants can request paediatric-use marketing authorisations for medicines that are already authorised, are no longer covered by a supplementary protection certificate or a patent that qualifies as such and are developed exclusively for use in children.

Parallel distribution

The distribution of a medicine from one Member State to another by a pharmaceutical company independently of the marketing authorisation holder.

Parallel group

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

Patent

An official document securing the exclusive right to make, use, or sell an invention to its inventor for a defined period.

Patent status of the investigated drug

The legal information about the patent protection status, the intellectual property owners and the market and data exclusivity rights related to the investigated active substance.

Patient

Any individual with or at risk of a specific health condition, whether or not they currently receive any therapy to prevent or treat that condition. Patients are the individuals who directly experience the benefits and harms associated with medical products.

Patient advocacy

Patient advocacy is a process in health care concerned with advocacy and support for patients, survivors, and caregivers. The patient advocate may be an individual or an organization, concerned with healthcare standards or with one specific group of disorders. The terms patient advocate and patient advocacy can refer both to individual advocates providing services that organizations also provide, and to organizations whose functions extend to individual patients. Some patient advocates work for the institutions that are directly responsible for the patient’s care.

Typical advocacy activities are the following: safeguarding patients from errors, incompetence and misconduct; patient rights, matters of privacy, confidentiality or informed consent, patient representation, awareness building, support and education of patients, survivors and their carers.

Patient advocate

An individual or group of individuals, who may or may not be part of the target patient population, who has a role in promoting an interest or cause to influence policy with respect to patients’ health or healthcare.

Patient centricity

Patient-centricity is a healthcare approach that prioritizes the needs, preferences, and values of the patient in all aspects of healthcare delivery. It is centered around the belief that patients should be actively involved in their own care, and that all decisions about their health should be made in collaboration with them. The concept of patient-centricity recognizes that patients are individuals with unique needs and experiences, and that healthcare should be tailored to meet those specific needs. This means that healthcare providers should listen to the patient’s perspective, involve them in decisions about their care, and provide them with the information and support they need to manage their health effectively. The goal of patient-centricity is to improve the quality of care and the overall healthcare experience for patients. By putting the patient at the center of the healthcare process, providers can work together with patients to create individualized treatment plans and improve health outcomes.

Patient champion

A non-profit stakeholder developing or gathering evidence, including the use of scientific advice as the main regulatory tool, for the repurposing of a medicinal product that can be e.g. a patient organisation, academia, collaborative groups or European Reference Networks (ERNs)’. A champion is typically :

• Able to coordinate and/or foster the research programme up until the point of full engagement by a pharmaceutical company.
• Initially responsible for liaising and leading the interactions with regulatory authorities and pharmaceutical companies/other stakeholders.
• Transparent regarding interactions with relevant pharmaceutical company(ies) in charge of
filing the initial request for scientific/regulatory advice on the basis of the available data.

Patient engagement

Patient engagement is the effective and active collaboration of patients, patient advocates, patient representatives and/or carers in the processes and decisions within the medicines lifecycle, along with all other relevant stakeholders when appropriate.
Structured, effective, meaningful and ethical patient engagement should be a standard practice to improve medicines development and deliver results that are focused on patients’ needs.

Patient input

Information that captures patients’ experiences, perspectives, needs, and priorities delivered directly by patients or patient representatives.

Patient partner

An individual patient, caregiver or patient advocacy group that engages other stakeholders to ensure the patients’ wants, needs and preferences are represented in activities related to medical product development and evaluation.

Patient perspective

A type of patient experience data that specifically relates to patients’ attitudes or points of view about their condition or its management. Patient perspectives may include (but are not limited to): perceptions, goals, priorities, concerns, opinions, and preferences.

Patient registry

A patient registry is a collection of information about individuals, usually those with a specific diagnosis or with specific risk factors for a disease. Some patient registries seek people with varying health levels who may be willing to take part in research about a particular disease. Registries can be funded and/or managed by government agencies, non-profit organisations, clinics, or commercial organisations.

Patient reported outcome (Patient reported outcome measures) (PRO/PROM)

A patient-reported outcome (PRO) is a measure of the experience or view of a participant in a clinical study. It is not a clinical measure, or an assessment made by anyone else involved in the study. PROs are commonly collected by asking patients to fill in questionnaires, or by interviewing patients. Questionnaires or interview guides used as part of clinical studies should undergo extensive testing to ensure they are reliable and valid. PROs can be used to assess, for example, symptoms as experienced by the patient, disability, quality of life, and other health perceptions.

Patient representative

An individual, who may or may not be part of the target population, who has direct experience with a disease or condition (e.g., a patient or caregiver) and can provide information about a patient’s experience with the disease or condition.

Patient selection

Patient selection can refer to how patients are matched with proposed treatments (in the clinic), or how patients are selected to take part in clinical trials.

For clinical trials, detailed inclusion and exclusion criteria are documented before recruitment of patients can begin. The inclusion and exclusion criteria are an important part of a trial protocol. If they are properly defined, inclusion and exclusion criteria will increase the chances of a trial producing reliable results. They also protect participants from harm and help avoid exploitation of vulnerable people (such as those without the ability to provide informed consent).

Patient-centric approach

A patient-centric approach is a healthcare strategy that prioritizes the needs, preferences, and values of the patient in all aspects of healthcare delivery. This approach is centered around the belief that patients should be actively involved in their own care and that all decisions about their health should be made in collaboration with them. In a patient-centric approach, healthcare providers take the time to understand the patient’s individual needs, concerns, and goals. They listen to the patient’s perspective, involve them in decisions about their care, and provide them with the information and support they need to manage their health effectively. This can lead to better health outcomes and a more positive healthcare experience for the patient. A patient-centric approach also places emphasis on patient education, empowerment, and engagement. Patients are encouraged to take an active role in their own health and to work with their healthcare providers to make informed decisions about their care. By doing so, patients can become more involved in their own health and more confident in managing their conditions. Overall, a patient-centric approach is a way of delivering healthcare that puts the needs of the patient first and recognizes that patients are unique individuals with unique needs and preferences. It aims to improve the quality of care and the overall healthcare experience for patient.

Patient-focused drug development (also referred to as patient-focused medical product development) (PFDD)

A systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into the development and evaluation of medical products throughout the medical product life cycle.

Performance outcome (PerfO)

A measurement based on a standardized task(s) performed by a patient that is administered and evaluated by an appropriately trained individual or is independently completed. PerfOs require patient cooperation and motivation. These include measures of gait speed (e.g., timed 25 foot walk test), memory recall (e.g., word recall test), or other cognitive testing (e.g., digit symbol substitution test).

Periodic safety update report (PSUR)

A report prepared by the marketing-authorisation holder describing the worldwide safety experience with a medicine at a defined time after its authorisation.

Personalised medicine/Precission medicine

A medicine that is targeted to individual patients, based on their genetic characteristics.

Personalized medicine

Term synonym to precision medicine but superceeded by it and dismissed with the argument that physicians have always treated patients on a personalised level.

Pharmaceutical form

The way a medicine is presented, e.g. tablet, capsule, solution for injection, cream, etc.

Pharmacodynamic (in vivo) (PD)

Focuses on determining concentration profiles and the fate of drug molecules in the body

PharmacoEconomics

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

Pharmacogenetics

Pharmacogenetics is generally regarded as the study or clinical trial of genetic variation that gives rise to differing response to drugs.

Pharmacogenomics

Pharmacogenomics is the broader application of genomic technologies to new drug discovery and further characterization of older drugs.

Pharmacokinetic (in vivo) (PK)

Examines the effect of the drug in the body.

Pharmacovigilance (PV, or PhV)

Science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other medicine-related problem.

Pharmacovigilance Risk Assessment Committee (PRAC)

The committee that is responsible for assessing all aspects of the risk management of medicines for human use.

Phase 0 clinical trial

Phase 0 trials are conducted with sub-therapeutic doses to see if a medicine behaves in the body in the way that earlier laboratory studies (non-clinical trials) predicted.

Phase I clinical trial

Normally, the first studies in humans with a new medicine are Phase I trials.

Phase I trials are usually conducted in a small number of healthy volunteers (although some trials recruit patients). The aim of Phase I trials is to find out the safe dose range, and to look for any side effects. The initial dose given will be very small, and gradually increased if no or only mild side effects are observed. A new medicine has to meet certain pre-set requirements before it can continue to Phase II trials. Phase I, II, and III trials are commonly known together as ‘clinical development’. See clinical trials figure overview in: Phase I Trials – EUPATI Toolbox

Phase II clinical trial

Phase II studies are designed to find out if the medicine has a beneficial effect on the disease in question: They might compare the new medicine to an existing treatment or to a placebo. They also set out to determine the best dose range and how often the medicine should be given, and investigate the best way to manage any side effects. These trials are often larger than Phase I trials.

A new medicine has to meet certain pre-set requirements before it can continue to Phase III trials. Phase I, II, and III trials are commonly known as ‘clinical development’. See clinical trials figure overview in: Phase II Trials – EUPATI Toolbox

Phase III clinical trial

Phase III trials are generally large (comprising thousands of patients) and involve several study sites, sometimes in different countries. They compare the new medicine to existing treatments or a placebo, in order to show the safety and efficacy of the new medicine. Most Phase III trials are randomised.

Phase I, II, and III trials are commonly known as ‘clinical development’. Phase III studies are critical to applications for marketing authorisation. See clinical trials figure overview in: Phase III Trials – EUPATI Toolbox

Phase IV clinical trial

Phase IV trials are usually conducted after marketing authorisation is granted and the medicine is in general use.

Phase IV studies are also known as post-authorisation safety studies (PASS) and may be voluntary or imposed by the regulatory authorities. The possibility also exists of requesting the marketing authorisation holder to conduct post-authorisation efficacy studies (PAESs) in order to complement efficacy data that are available at the time of the initial authorisation. Phase IV studies collect additional information about side-effects and safety, long-term risks and benefits, and/or how well the medicine works when used widely. See clinical trials figure overview in: Phase IV Trials – EUPATI Toolbox

Philantropy

Philanthropy refers to the act of giving time, resources, or money to support causes and organizations that are dedicated to making a positive impact in the world. It is an important form of social investment that is focused on creating lasting change and improving the lives of individuals and communities. Philanthropy can take many forms, including charitable donations, volunteering, and community service. It can support a wide range of causes, from education and healthcare to environmental protection and social justice. Philanthropic activities can be carried out by individuals, foundations, corporations, and other organizations.

PK-PD modeling

PK/PD modeling (pharmacokinetic/pharmacodynamic modeling) (alternatively abbreviated as PKPD or PK-PD modeling) is a technique that combines the two classical pharmacologic disciplines of pharmacokinetics and pharmacodynamics.[3] It integrates a pharmacokinetic and a pharmacodynamic model component into one set of mathematical expressions that allows the description of the time course of effect intensity in response to administration of a drug dose. PK/PD modeling is related to the field of pharmacometrics. The rationales for in vivo PK-PD-efficacy relationship studies in preclinical models are to:

1. provide a quantitative analysis of dose–response relationships;
2. describe and predict the time-course effects resulting from one or multiple drug doses;
3. understand for how long and how much the target must be modulated to observe statistically significant efficacy;
4. Ultimately identify efficacious dosing regimens with an acceptable therapeutic index.

Inclusion in vivo PK-PD-efficacy studies at early stages during the lead identification and optimization stages of a drug development program can significantly accelerate the selection of the most promising compounds. This is done by informing on the precise mechanisms of action and by rapidly identifying unexpected toxicity and/or off-target activities in vivo.                                                                                                                               Properly planned and executed PK-PD-efficacy studies requires a team of experts in:

1. The establishment of the most relevant and appropriate animal model(s);
2. Animal dosing and handling;
3. The measurement of efficacy in different models;
4. The identification of the most appropriate PD readout for the drug and for the model being tested;
5. The design, optimization and execution of quantitative PD assays and/or measurements;
6. The measurement of drug concentrations in different tissue matrices;
7. the evaluation and analysis of PK data;
8. the analysis of relationships between PK, PD and efficacy.

Placebo (PLCB)

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.

Planned Inspection

A planned inspection is a set of additional information that is provided when a GMDP certificate is first created. The Planned Inspection may be considered as the first step in the creation of a GMP Certificate, allowing inspectors from multiple NCAs to work together on planning inspections on third country site. The Planned Inspection falls into two categories:

1. It remains as a Draft GMP Certificate until the planned inspection is actually concluded and the certificate is submitted.
2. It is accessible to all NCAs with appropriate access (EUDRANET) via the ‘Search Planned Inspections’ module.

It is hoped that users will search for any planned inspection on a particular site in order to provide more efficient collaborative inspection coverage of distant sites. Planned Inspections may also be found within the Draft GMP Certificates area for colleagues of the author of the Planned Inspection (i.e. belonging to the same NCA).

Plasma master file (PMF)

Documentation providing detailed information on the characteristics of the human plasma used in a medicine or a medical device.

Plasma-derived medicinal product (PDMPs)

A medicinal product derived from human blood or human plasma

Platform (clinical) trial

A platform clinical trial is a type of clinical trial that focuses on testing multiple targeted therapies in the context of a single disease in a perpetual manner (ongoing over time with no fixed stopping date), with therapies allowed to enter or leave the platform on the basis of a decision algorithm.

Policy Board REMEDi4ALL (PB)

Composed of leading experts in policy and practice of repurposing, from all relevant sectors. The members of the PB will bring their knowledge and experience to bear to formulate policy innovations that are directed at solving current limitations in the system. To that end the PB will inventorise the current bottlenecks and propose solutions for improvements to the policy and regulatory environment. This will build on and work with existing efforts, such as in the Observatory Group (EMA and others), STAMP, ILAP (MHRA), EUnetHTA, and European Commission. The PB will work together to define positions, and on the basis of broad consultation and consensus-building will subsequently build buy-in and generate momentum using their own networks and the REMEDi4ALL community.  Conclusions, opinions and policy innovations will be disseminated through publication of white papers and position papers as relevant. The core principle underlying the efforts is inclusiveness, consensus and consultation, so that ideas can become reality using stakeholder momentum.

Polypharmacology

Polypharmacology is the design or use of drugs/medicines that act on multiple disease pathways.

Pre-term newborn infants

Subjects are not more than 37 weeks from their conception.

Precision medicine

Medical care/treatment designed to optimize efficiency or therapeutic benefit for particular groups of patients, especially by using genetic or molecular profiling.

Preclinical development

In drug development, preclinical development, also termed preclinical studies or nonclinical studies, is a stage of research that begins before clinical trials (testing in humans) and during which important feasibility, iterative testing and drug safety data are collected, typically in laboratory animals.

The main goals of preclinical studies are to determine a starting, safe dose for first-in-human study and assess potential toxicity of the product, which typically include new medical devices, prescription drugs, and diagnostics.

Prescription only Medicine (POM)

A treatment that must be prescribed by a doctor and is not licensed for sale to the general public.

Primary Endpoint(s)

The main result that is measured at the end of a study to see if a given treatment worked (e.g., the number of deaths or the difference in survival between the treatment group and the control group). What the primary endpoint will be is decided before the study begins.

Principal exclusion criteria

Reasons for exclusion of subjects from the clinical trial from among the exclusion criteria described in the protocol.

Principal inclusion criteria

Reasons for the inclusion of subjects in the clinical trial.

Principal Investigator (PI)

The person(s) in charge of a clinical trial or a scientific research grant. The principal investigator prepares and carries out the clinical trial protocol (plan for the study) or research paid for by the grant.

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.

Product Information document (PI)

Documents providing officially approved information for healthcare professionals and patients on a medicine. The product information includes the summary of product characteristics, package leaflet and labelling.

Product name

In the absence of trade name this is the name routinely used by a sponsor to identify the IMP in the Clinical Trial Documentation.

Product-specific waiver

An exemption from the obligation to acquire data, through a paediatric investigation plan, in some or all subsets of the paediatric population for a given condition, route of administration and pharmaceutical form of a specified medicine. Product-specific waivers are adopted by the Paediatric Committee (PDCO).

Prognostic biomarker

A prognostic biomarker is one that indicates an increased (or decreased) likelihood of a future clinical event, disease recurrence or progression in an identified population. Prognostic biomarkers are measured at a defined baseline, which may include a background treatment.

Proof of Concept (PoC)

The earliest point in the drug development process at which the weight of evidence suggests that it is ‘reasonably likely’ that the key attributes for success are present and the key causes of failure are absent (Cartwright et al 2010). The interpretation of this definition, however, is context dependent. For industry, POC almost always applies to clinical trials. POC studies are usually small and designed so as to provide early statistical evidence allowing drug developers to make the decision whether or not to proceed into larger, more expensive Phase 2b or 3 clinical trials. To the academic researcher however, POC often represents the results of mechanistic in vitro studies, or at best, studies in an animal model. While the scientific rationale may be proved, other questions that are important in the broader drug development pipeline are often not addressed, such as safety, efficacy, dosing regime, cost of goods, and commercial and regulatory issues that could threaten the ultimate success of a novel drug.

Prophylaxis

Medical or public health measure taken whose purpose is to prevent, rather than treat or cure a disease. Primary prophylaxis is generally intended to prevent the development of a disease, while secondary prophylaxis is intended to prevent the disease, once contracted by a patient, from worsening.

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.

Protocol assistance

A type of scientific advice for developers of orphan medicines.

Quality Control (QC)

Quality Control. Quality Control enacts the SOP, monitoring and recording the activity of the process.

Quality Control Lab (QC Lab)

A laboratory that undertakes role of validating the quality of the products produced by manufacturers (Active substance and medicinal).

Randomised clinical trial

A trial in which subjects are randomly assigned to one of the treatment arms.
It uses randomisation when allocating people to different arms of the study. For example, in a trial comparing a new medicine with a placebo, each person has an equal chance of being allocated to the medicine or the placebo group.

Randomised controlled clinical trial (RCT)

A randomised controlled trial is a trial in which people are allocated at random (by chance alone) to receive one of several clinical interventions such as a new medicine. One of these interventions is the control group, for example a placebo may be given, no intervention at all, or the current best treatment available.

Rapid Alerts

The process by which information is disseminated to the field on the event of a product failure.

Rapporteur

One of the two members of a committee or working party who leads the evaluation of an application.

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

Re-examination

A review of an opinion by a scientific committee of the Agency, triggered at the request of the applicant.

Re-labelling

Drug relabelling is defined as the act of changing the existing label or labels on a drug or drug package or changing or altering the existing labeling for a drug or drug package, without repacking the drug or drug package.

Real World Data (RWD)

Real-world data are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. RWD can come from a number of sources, for example:

• Electronic health records (EHRs)
• Claims and billing activities
• Product and disease registries
• Patient-generated data including in home-use settings
• Data gathered from other sources that can inform on health status, such as mobile devices

Real World Evidence (RWE)

Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).

Really Simple Syndication (RSS)

RSS is a family of web feed formats used to publish frequently updated works—such as blog entries news headlines, audio, and video—in a standardised format.

Recognised Researcher (PhD holders or equivalent who are not yet fully independent) (R2)

Recombinant

A cell or organism in which genetic recombination has occurred.

Reference Member State (RMS)

The European Union Member State that leads the review of an application in a mutual recognition procedure or decentralised procedure.

Referral

An evaluation conducted by a European Medicines Agency committee following a referral from the European Commission or a Member State. Referrals are used to address particular issues, such as safety concerns, to resolve disagreements between Member States on issues related to the authorisation of medicines or to give an opinion on an issue of Europe-wide interest.

Reflection paper

A document outlining the view of the European Medicines Agency or one of its committees, working parties or other groups on a particular issue.

Reformulation

Reformulations are changes in a drug’s “molecular formulary,” including any combination of changes in active ingredient concentrations, inactive components, or changes in the means of drug delivery. When drugs that have been FDA-approved are reformulated, the data used for the original approval can often be used to support the new application (examples appear in Table 1). In this way reformulation can represent a shortcut past some regulatory hurdles faced by drugs classified as NMEs. While patent protection is certainly a factor in the prevalence of reformulations, improvements in therapeutic efficacy are the overriding motivator. Often these improvements are the result of knowledge of a drug’s properties gained following approval, when the number of patients (and physicians) with first-hand experience grows from clinical-trial levels to tens of millions.

Regulatory Authority (RA)

A body that carries out regulatory activities relating to medicines, including the processing of marketing authorisations, the monitoring of side effects, inspections, quality testing and monitoring the use of medicines.

Regulatory compliance (RC)

The process and activities that a company undertakes to ensure that it adheres to all applicable laws and regulations. Compliance is a broad term that can encompass everything from ensuring that products are safe and effective to making sure that marketing materials are accurate.

Regulatory pathway

In the context of regulatory affairs in drug development, a regulatory pathway refers to the series of steps and requirements set by regulatory agencies, such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA), for the approval and commercialization of a new drug. This pathway involves various stages of pre-clinical testing, clinical trials, and regulatory submissions, as well as post-marketing surveillance and monitoring. The goal of the regulatory pathway is to ensure the safety and efficacy of new drugs before they are made available to the public. The specific requirements and steps involved in the regulatory pathway can vary by country and can be influenced by factors such as the type of drug, its intended use, and the target patient population. The regulatory pathway is a crucial aspect of drug development and can have a significant impact on the timeline, costs, and success of a new drug.

REMEDi4ALL

EU-funded REMEDi4ALL project that aims to establish Europe’s leadership globally in the repurposing of medicines by creating a vibrant community of practice. Bringing together all sectors and involving the right experts at the right time, REMEDi4ALL will create new connections to test and validate existing medicines for a new purpose. Four preclinical and clinical phase demonstrators in a variety of disease areas (including oncology, and rare and infectious diseases) will validate the tools and processes developed by the project.

Renewal

An extension of the validity of a marketing authorisation, which can be for a fixed or indefinite period of time. Initial marketing authorisations are usually valid for five years.

Reporter

In research studies designed to collect patient experience data, the reporter is the individual, group of individuals, or entity providing patient experience data. Reporters may be patients, parents, sexual/romantic partners, caregivers, physicians, or other healthcare professionals. Selection of an appropriate reporter in a given research study will depend on the definition of the target patient population of interest. If a patient in the target population can be reasonably expected to reliably self-report, then one would expect the patient herself/himself to be the reporter in that research study.

Representativeness

Confidence that a sample from which evidence is generated is sufficiently similar to the intended population. In the context of patient experience data, representativeness includes the extent to which the elicited experiences, perspectives, needs, and priorities of the sample are sufficiently similar to those of the intended patient population.

Repurposing Development Plan (REMEDi4ALL) (RDP)

A repurposing development plan is a comprehensive strategy for evaluating an existing drug’s potential for use in a new therapeutic indication. The plan typically includes preclinical and clinical studies designed to assess the safety, pharmacokinetics, and efficacy of the drug in the new indication, as well as any necessary formulation or dose adjustments. The development plan also includes regulatory and intellectual property considerations, as well as a commercialization strategy. Overall, a repurposing development plan aims to efficiently and effectively bring an existing drug to market for a new indication, leveraging the drug’s established safety and efficacy data.

Repurposing Development Team (REMEDi4ALL) (RDT)

A repurposing development team is a group of experts from various disciplines, including scientists, clinicians, patient representatives, regulatory professionals, intellectual property lawyers, and commercialization specialists, who work together to evaluate an existing drug’s potential for use in a new therapeutic indication. The team typically develops and executes a repurposing development plan, which includes preclinical and clinical studies to assess the drug’s safety and efficacy in the new indication, as well as regulatory, intellectual property, market entry route, pricing and reimbursement considerations. The team works collaboratively to ensure that the repurposing effort is scientifically sound, financially viable, and meets regulatory requirements, with the goal of bringing the repurposed drug to market as quickly and efficiently as possible.

Repurposing Hypothesis

A drug repurposing hypothesis is a proposal or conjecture suggesting that an existing drug could be used for a novel therapeutic application, either by identifying new targets for the drug or by applying the drug to different diseases or conditions than it was originally intended for. This hypothesis is based on the premise that a drug with established safety and efficacy in one context may also be effective in treating other diseases, potentially leading to new treatment options and improved patient outcomes.

Request for supplementary information

A list of questions addressed to a marketing authorisation holder during the evaluation of a variation to an existing marketing authorisation.

Research infrastructure (RI)

Research infrastructure refers to the physical, organizational, and technological resources necessary for conducting scientific research. It encompasses the facilities, equipment, data, and personnel required to carry out research projects, as well as the policies, procedures, and systems that support and coordinate research activities. Research infrastructure can include a wide range of resources, such as laboratory facilities and equipment, information technology systems and databases, libraries and archives, animal care facilities, and clinical research facilities. It also includes support services such as core facilities, which provide specialized services such as analytical testing and imaging, and administrative services that manage funding and grant applications, research contracts and agreements, and ethical review and compliance.

Research project

A Research project is an undertaking defined by a unique goal and it is limited concerning resources, budget, time, etc. A research project always allows differentiation against other undertakings and is characterized by a specific project organisation.

Research protocol

A document that describes the background, rationale, objectives, design, methodology, statistical considerations, and organization of a clinical research project. A research protocol guides the study and associated data collection and analysis in a productive and standardized manner.

Return on investment (ROI)

ROI is a measure of the profit or loss generated by an investment compared to the amount of money invested. It is calculated as a ratio or percentage, and it provides a way to evaluate the performance of an investment over a given period of time.

Risk

Risks are adverse events and other unfavorable effects associated with a medical product. Risks include drug interactions, risks identified in the non-clinical data, risks to those other than the patient (e.g., fetus, those preparing and administering the medical product), and risks based on pharmacologic class or current knowledge of the product. Factors such as potential misuse, abuse, or diversion of the product may also be considered.

Risk Management Plan (RMP)

A detailed description of the activities and interventions designed to identify, characterise, prevent or minimise risks relating to medicines.

Risk management system

A set of activities and interventions designed to identify, characterise, prevent or minimise risks relating to medicines, including the assessment of the effectiveness of those activities and interventions.

Risk minimisation activity

A public health intervention intended to prevent or reduce the probability of the occurrence of an adverse reaction associated with exposure to a medicine or to reduce its severity if it occurs.

Risk tolerance

The degree to which a patient would accept increased probability or severity of a harm in exchange for a specific expected benefit.

Route of administration

The way in which a medicine is given, e.g. orally (by mouth), intravenously (into a vein), subcutaneously (under the skin), etc.

Safe and Timely Access to Medicines for Patients (STAMP)

The STAMP expert group is set up to provide advice and expertise to the Commission services in relation to the implementation of the EU Pharmaceutical legislation, as well as programmes and policies in this field. The STAMP will exchange views and information about the experience of Member States, examine national initiatives and identify ways to use more effectively the existing EU regulatory tools with the aim to further improve safe and timely access and availability of medicines for patients.

Safety

The potential of a drug to be endured. Also known as ‘Tolerability’.

Safety profile (of a drug)

The safety profile of a drug refers to the risks and adverse effects associated with its use. It encompasses information on the potential harm that a drug may cause to the patient, including its side effects, contraindications, interactions with other drugs, and any other negative effects that may be associated with its use. The safety profile is determined through pre-clinical testing and clinical trials, as well as post-marketing surveillance and monitoring. The safety profile of a drug is a crucial aspect of drug development and must be considered by regulatory agencies, healthcare providers, and patients when evaluating the benefits and risks of a new drug. The goal is to ensure that the benefits of a drug outweigh its risks, and that the drug is safe for use in the intended patient population. See fifference with clinical profile in section C.

Safety signal

Information on a new or known adverse event that is potentially caused by a medicine and that warrants further investigation. Signals are generated from several sources such as spontaneous reports, clinical studies and the scientific literature.

Scientific advice (SA)

The provision of advice by the European Medicines Agency on the appropriate tests and studies required in the development of a medicine or on the quality of a medicine.

Scientific advisory group (SAG)

A group of European experts brought together to provide advice in connection with the evaluation of specific medicines.

Secondary end point(s)

Results that are measured at the end of a study, in addition to the main result (primary endpoint) to see if a given treatment worked. Secondary endpoints can explore other aspects of the treatment

Secondary objectives of the trial

A description of the secondary objectives of the trial as defined by secondary end points.

Secondary Packing

Secondary packing refers to additional packaging beyond the actual container in which a medicinal product is stored.

Serious adverse reaction (SAR)

An adverse reaction that results in death, is life-threatening, requires hospitalisation or prolongation of existing hospitalisation, results in persistent or significant disability or incapacity, or is a birth defect.

Service provider (external)

An external service provider is an individual or company that offers a service or services to clients or customers (usually) in exchange for payment.

Service provider (internal)

An internal service provider is a department or team within an organization that offers services to other departments or business units within the same organization. A common example of internal service provider within a scientific research institution is a Core Facility. Core facilities may offer services such as genomics sequencing, mass spectrometry, flow cytometry, imaging, animal care, and other specialized research techniques.

Single blind

A trial where the subjects (healthy volunteers or patients) included 
in the trial doesn’t know which treatment they are given but the investigator does.

Single site in the Member State concerned

A trial is conducted in a single centre (clinical trial site) in the EU Country concerned by the application.

Social Return on Investment (SROI)

SROI is a method for measuring the social, environmental, and economic impact of an investment, program, or project. The goal of SROI is to provide a more comprehensive and meaningful measure of impact, beyond financial returns, by considering the impact of an investment on the well-being of individuals, communities, and the environment.

Somatic cell based medicine

A medicine containing cells or tissues that have been manipulated to change their biological characteristics, which is used to cure, diagnose or prevent a disease.

Somatic cell therapy medicinal product

Means the use of autologous (emanating from the patient himself), 
allogeneic (coming from another human being) or xenogeneic 
(coming from animals) somatic living cells, the biological 
characteristics of which have been substantially altered as a result 
of their manipulation to obtain a therapeutic, diagnostic or 
preventive effect.

Source(s) of Monetary or Material Support for the Clinical Trial

Organisation or Pharmaceutical company providing monetary or 
material support for the conduct of the trial.

Specific obligations

Requirements imposed on holders of conditional marketing authorisations or marketing authorisations granted under exceptional circumstances.

Specimen

A sample of the actual printed outer and inner packaging materials and package leaflet for a medicine.

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

Sponsor’s protocol code number

Unique Identifier number for the Protocol e.g. Trial acronym and Year (MAG 98).

Stakeholder

Any individual, group, or organization that has an interest in or is affected by the activities, decisions, or outcomes of a particular project, initiative, or organisations. Stakeholders in drug repurposing may include: patients, patient carers, academic researchers, healthcare providers, regulatory agencies, pharmaceutical companies, nonprofit organisations, investors, government agencies, payers, ethics committees.  

Standard Operating Procedure (SOP)

Detailed, written instructions to achieve uniformity of the performance of the special functions. These provide a general framework enabling the efficient implementation and performance of the functions and activities for a particular process.

Status of the sponsor

Indication of whether the sponsor is commercial or non-commercial

Sub-study

A sub-study, or ancillary study, is a study performed on a subgroup of the subjects included in the clinical trial. For example, a pharmacokinetics or pharmacogenetics sub-study may include a sample of the patients participating in the clinical trial.

Subgroup

A subset of the study population or study sample defined by specific baseline characteristics. For example, demographic subgroups are commonly defined by subject sex, race, and age.

Subgroup analysis

The separate analysis of clinical trial results in subsets of the trial’s participants.

Subject

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

Subject-matter expert (SME)

A subject-matter expert (SME) is a person who has accumulated great knowledge in a particular field or topic and this level of knowledge is demonstrated by the person’s degree, licensure, and/or through years of professional experience with the subject.

Summary of opinion

A document summarising the opinion issued by one of the European Medicines Agency’s committees at the end of the evaluation process.

Summary of product characteristics (SmPC)

A document describing the properties and the officially approved conditions of use of a medicine. Summaries of product characteristics form the basis of information for healthcare professionals on how to use the medicine safely and effectively. Abbreviated as SmPC.

Sunset clause

A legal provision stating that the marketing authorisation of a medicine will cease to be valid if the medicine is not placed on the market within three years of the authorisation being granted or if the medicine is removed from the market for three consecutive years.

Supplementary protection certificate (SPCs)

A means of extending the term of patent exclusivity for a new medicine for a fixed period from the date of the first marketing authorisation in a European Union Member State.

Surrogate endpoint

In clinical trials, an indicator or sign used in place of another to tell if a treatment works. Surrogate endpoints include, for example, a shrinking tumor or lower biomarker levels. They may be used instead of stronger indicators, such as longer survival or improved quality of life, because the results of the trial can be measured sooner. The use of surrogate endpoints in clinical trials may allow earlier approval of new drugs to treat serious or life-threatening diseases. Surrogate endpoints are not always true indicators or signs of how well a treatment works.

Suspected Unexpected Serious Adverse Reactions (SUSAR)

A SUSAR is defined as an untoward and unintended response to a study drug, which is not listed is the applicable product information, and meets one of the following serious criteria: results in death, is life-threatening, requires hospitalisation or prolongation of an existing hospitalisation, results in persistent or significant disability or incapacity, or is a congenital anomaly or birth defect

Target patient population

Refers to the patients the medicine is intended for.

Target Product Profile (TPP)

A target product profile (TPP) outlines the desired ‘profile’ or characteristics of a target product that is aimed at a particular disease or diseases. TPPs state intended use, target populations and other desired attributes of products, including safety and efficacy-related characteristics. Such profiles can guide product research and development (R&D). In industry, in-house target product profiles (TPPs) are used as planning tools that guide development towards desired characteristics. In the regulatory context, TPPs are considered as tools to frame development in relation to submission of product dossiers.
In the context of public health, TPPs set R&D targets for funders and developers.

Technology Transfer Organization (TTO)

A TTO is associated to an academic institution and dedicated towards the identification, securing Intellectual Property (IP), and commercialization of academic research results. TTOs act as information brokers between academic research groups, patent attorneys, and commercially focused parties (e.g., venture capital, biotech, Pharma companies, etc.).

Therapeutic hypothesis

Therapeutic hypothesis is a scientifically reasonable association of a Druggable Target with a disease/therapeutic area where such association is based on scientific evidence.

Therapeutic window (TW)

Therapeutic window (TW) can be defined as the range of drug concentrations that provide therapeutic response without significant adverse effects. (Clinical Pharmacy Education, Practice and Research, 2019).

Third country

A country outside the European Union and European Economic Area.

Tissue-engineered product

A medicine containing engineered cells or tissues, which is intended to regenerate, repair or replace a human tissue.

Toll-Gate

Toll-gates are similar to milestones since they reflect an important event during the course of the project. Toll-gates, however, are critical events that need special attention of and go/no-go decision-making by the User, the Steering Committee (SC) and other stakeholders because the fulfilment and approval to continue the project has significant impact on time and resource allocation for the next project phase. It is therefore required to prepare a Toll-Gate-Decision Document (TDD), summarizing the work performed to date and describing the planned work (incl. budget, resources, risk, and deliverables) until the next toll-gate. Examples for toll-gates in pharmaceutical development are:

• Progress a lead candidate to in vivo preclinical studies
• Initiate the nonclinical development programme, incl. GMP-production
• Initiate clinical phase I (first-in-man) studies
• Initiate clinical phase II (efficacy in target population) studies
• Initiate clinical phase III (pivotal) studies, incl. full GMP-process validation and manufacturing of GMP-validation batches

Trade name

Name of the MA holder of the actual IMP used in the member state concerned by the application

Transfer of marketing authorisation

A change to a marketing authorisation where the ownership of a marketing authorisation is moved from one marketing-authorisation holder to another.

Translational research

Translational research (also called translation research, translational science, or, when the context is clear, simply translation) is research aimed at translating (converting) results in basic research into results that directly benefit humans. The term is used in science and technology, especially in biology and medical science. As such, translational research forms a subset of applied research.

Treatment burden

The impacts of a specific treatment or treatment regimen that have a negative effect on the patient’s health, functioning, or overall well-being. Treatment burden includes (but is not limited to): side effects, discomfort, uncertainty about treatment outcomes, dosing and route of administration, requirements, and financial impacts.

Treatment effect

The amount of change in a disease/condition, symptom, or function that results from a medical intervention (as compared to not receiving the intervention or receiving a different intervention).

Treatment outcome

The benefits or harms to a patient who receives an intervention; the impact on a patient’s health, function, or well-being—or on a clinical indicator thereof—that is assumed to result from an intervention.

Trial arm

A trial arm is a group of participants that receives the same interventions, or no intervention, according to the study protocol. Many randomised trials have two arms, but some may have three or even more. This is decided before the trial begins.

Trials with several arms (multi-arm) allow more than one treatment to be tested at once, and can reduce the costs and time needed during clinical development. Multi-arm, multi-stage (MAMS) trials take this idea a step further and allow the recruitment of participants in a particular arm to be stopped partway through if that treatment is not producing satisfactory results. MAMS can also allow for new treatments to be added to the trial as they become ready for testing.

Trial protocol

An agreed plan issued by the sponsor of a clinical trial giving details of the objectives, design and organisation of the study.

Type IA variation

A minor change to a marketing authorisation that has a minimal or no impact on the quality, safety or efficacy of the medicine and does not require prior approval before implementation by the marketing authorisation holder.

Type IB variation

A minor change to a marketing authorisation that the marketing-authorisation holder must notify to the regulatory authority before implementation, but which does not require formal approval.

Type II variation

A major change to a marketing authorisation that may have a significant impact on the quality, safety or efficacy of a medicine, but does not involve a change to the active substance, its strength or the route of administration. Type II variations require a formal approval.

Umbrella (clinical) trial

An umbrella clinical trial is a type of clinical trial that focuses on the study of multiple targeted therapies for a single disease. Patients with the disease are screened for the presence of a biomarker or other characteristic and then grouped based on the results. Different drugs are then tested within these groups.

Unmet medical need (UMN)

An unmet medical need is a condition or symptom whose treatment or diagnosis is not addressed adequately by available therapy. An unmet medical need includes an immediate need for a defined population (e.g., to treat a serious condition with no or limited treatment) or a longer-term need for society (e.g., to address the development of resistance to antibacterial drugs).

Urgent Safety Restriction (USR)

Urgent regulatory action triggered by the marketing authorisation holder, a national regulatory authority or by the European Commission in the event of, or to prevent, a risk to human or animal health or to the environment.

Value-added medicine (VAM)

Value added medicines are medicines based on known molecules that address healthcare needs and deliver relevant improvements for patients, healthcare professionals and/or payers.

Variation

A change to the terms of a marketing authorisation.

Venture capital fund (VC)

A venture capital (VC) fund is an investment fund that pools money from institutional investors, wealthy individuals, and other sources to provide capital to early-stage, high-growth companies. These companies typically have not yet achieved profitability and may require significant capital investment to grow and succeed. In exchange for the investment, the VC fund receives an equity stake in the company and may play an active role in guiding the company’s growth and development. VC funds typically invest in a range of industries and sectors, with a focus on companies with the potential for high returns. The goal of a venture capital fund is to identify promising startups, provide them with the capital and resources they need to grow and succeed, and generate strong returns for the fund’s investors.

Well-established use

When an active ingredient of a medicine has been used for more than 10 years and its efficacy and safety have been well established. In such cases, application for marketing authorisation may be based on results from the scientific literature.

Withdrawal period

The time that must elapse between the last administration of a veterinary medicine and the slaughter or production of food from that animal, to ensure that the food does not contain levels of the medicine that exceed the maximum residue limit.

Work Breakdown Structure (WBS)

WBS is an overview of the major project activities in a structured manner. Depending on the nature of the project the WBS may be based on organisational, process, or other procedural parameters. The WBS structures shall describe the time-, and organizational aspects of the overall project. The WBS is therefore structured into and is described by sub-projects and working packages. Sub-projects are broken down further to result in WPs with clearly assigned responsibilities are defined.

Working party

A group of European experts that can be consulted by the European Medicines Agency’s committees on scientific issues in their area of expertise. Working parties are often given tasks linked to the evaluation of applications and the drafting or revision of guidance.

Working/Work Package (WP)

A WP is a defined unit for assigning work to a responsible party. During project planning and project implementation, it is the RP manager’s task to break down the project plan and assign responsibilities to the single WPs. This breakdown process is documented in the work breakdown structure. This term could be aligned or overlap with the Work Package definition used in large EU projects and consortia.

Worksharing

The submission of a single application for a variation that affects more than one marketing authorisation from the same marketing authorisation holder.