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Adverse reaction

An unwanted or harmful reaction which occurs after administration of a drug


Artificial intelligence (AI) is the simulation of human intelligence processes by machines 

Amino acid

Amino acids are the building blocks to forming proteins: they are the acids formed from the coding of a triplet of bases in the DNA. There are 20 amino acids, and most have several triplets which code for them.


Evidence based on personal accounts rather than facts or research 

Animal model

An animal model utilises a non-human species to be studied to understand particular biological phenomena, to provide insight into the workings of other organisms.


The expression of agreement 

Audit trail

A record of the changes that have been made to a database or a file 


Baseline data

This is data that is collected on patients at the beginning of the clinical trial.

Big data

Extremely large data sets that may be analysed computationally to reveal patterns, trends and associations 


A biobank is a place that stores biological samples for use in research


Relating to the chemical processes and substances that occur within living organisms


An interdisciplinary field of science that combines biology, computer science, information engineering, mathematics and statistics to analyse data

Biological sample

Any sample or specimen of material derived from an organism


A naturally occurring molecule, gene or characteristic by which a biological process can be identified


The area of biology involving living systems and organisms used to develop a product


Blinding means setting up a trial in a way which prevents anyone from knowing which participants are in the control group and which are in the treatment group.

Boilerplate paragraph

It is the small section at the end of the press release that provides some background information about the organisation or group, including what the group does, its mission and past accomplishments. It comes under the “Notes to Editor” section along with relevant links to the website and social media channels.

Bowed legs

When the leg curves outward at the knee


Drugs that have been given a name by a company for the purpose of advertising

Budget impact

The change in levels of spending because of a decision



A drug that has been identified as having strong therapeutic potential and will be taken into clinical trials


A person who is generally not affected with the condition (at that moment), but carries one faulty copy of a gene

Clinical research

Clinical research is any research that requires human participation. This could include giving biological samples (e.g. blood or tissue), answering health questionnaires, giving researchers access to medical records, or taking part in a clinical trial

Clinical trial

A clinical trial is any research study that assigns human participants to health-related interventions to evaluate the effects on health outcomes.


A medical professional that has direct contact with patients


The presence of more than one disorder in the same patient

Coding region

The coding region of a gene, also known as the CDS, is that portion of a gene's DNA or RNA that codes for protein. 


A group of people with a shared characteristic


The alternative to the proposed treatment or strategy


The alternatives to the proposed treatment or strategy.

Compound rescue

Compound rescue is taking a potential drug compound at advanced stage of pharma development but abandoned before drug approval, and testing it a disease that is different than the one it was originally developed for and then abandoned.


Providing permission for something to happen or agreement to do something

Contract research organisation

Any organisation that provides services relating to clinical research, such as the development of medicines, clinical trials and commercialisation

Control group

A control group in a clinical trial is given either an existing standard treatment or a placebo, to serve as a base line for the new medicine's effects to be tested against.

Corporate Social Responsibility

Corporate Social Responsibility (CSR) is the voluntary activities undertaken by a company to operate in an economic, social, and environmentally sustainable manner.

Cost of illness

A measure of the economic burden of a disease on society, incorporating all costs of the disease.

Cost-consequence model

A model that details the costs and outcomes of an intervention separately, without combining the two elements


A measure of the value a new treatment provides by looking at the costs per unit of benefit.


A measure of the value a new treatment provides by looking at the costs per unit of benefit.

Cost-effectiveness model

A model that compares the costs and benefits of two or more interventions

Cost-utility model

A model type that measures health benefits in a preference-based measure, such as quality-adjusted life years (QALYs)


A way of raising funds by asking a large number of people each for money


Obtaining information or input into a task/project by enlisting the help of a large number of people


Data integrity

The maintenance and assurance of the accuracy and consistency of data over its entire life cycle. 

Data protection

All personal information is protected in the UK by the Data Protection Act (1998). This means that researchers have to put in all the necessary safeguards to protect the confidentiality of the information they collect about research participants. They should explain the patient information sheet:

  • how the participants' data will be collected
  • how it will be stored securely
  • what it will be used for
  • who will have access to the data that identifies participants
  • how long it will be kept
  • how it will be disposed of securely.

Data Protection Act

The Data Protection Act 2018 is the UK's implementation of EU General Data Protection Regulation (GDPR), that controls how personal information is used by organisations.

De novo

Latin term for 'new'. De novo drug discovery is the traditional route of generating new treatments from novel (previously unknown) chemical entities with desired activity against a disease

Decision trees

A type of model that details a pathway of a disease as a set of potential outcomes, with different probabilities assigned to each of the outcomes.


Characterised by progressive deterioration and loss of function in the organs or tissues


A particular sector of the population. 


A disorder of structure of function in an organism


Dissemination involves communicating the findings of a research project to a wide range of people who might find it useful. This can be done through:

  • producing reports (often these are made available on the Internet)
  • publishing articles in journals or newsletters
  • issuing press releases
  • giving talks at conferences

It is important to feed back the findings of research to research participants.


A chemical substance which makes up the genes, and which contains the information needed for the body to work. 


The amount of or frequency that a drug is given in


Any substance that causes a change in an organism

Drug reformulation

Drug reformulation is taking an existing approved drug and creating a new delivery form or a new dosage.

Drug repositioning

Drug repositioning is when a pharma company takes one of their patent protected drugs and creates a new disease use for it, and then starts marketing the drug for a new use. 

Drug reprofiling

Drug reprofiling changes an already approved drug by creating tiny variations (called isomers) so that it can be re-patented for a new disease indication

Drug repurposing

Taking a drug already approved for human use in one disease and using it for another disease

Drug target

A drug target is anything in a living organism to which a drug can binds to, causing a change in its behaviour or function. Common biological targets are proteins and nucleic acids.


The act or process of making an exact copy of something 



Efficacy refers to the ability of a medicine to provide a beneficial effect (a positive benefit/risk ratio) when studied in a clinical trial

When talking in terms of efficacy vs. effectiveness, effectiveness relates to how well a treatment works in the real world practice of medicine, as opposed to efficacy, which measures how well a treatment works in clinical trials or laboratory studies.

End of life

Types of terminal medical conditions that have become advanced, progressive and incurable. The criteria required to meet this description differ between decision makers across the different regions.

Endoplasmic reticulum

A network of membranes within animal cells used to facilitate reactions 


An outcome relating to a change in biological processes, sometimes measured to determine the effectiveness of an intervention


Ethics is the name given to the code of practice based on a set of decent, fair, and moral principles and guidelines that researchers should abide by when conducting research. Any research that will seek to gain personal confidential information or to test a new intervention or treatment on people must get ethical approval from a Research Ethics Committee.

European Medicines Agency

The body in the European Union that regulates the use of medicines across all EU states. 

European Reference Networks

Virtual networks of healthcare providers across Europe that aim to facilitate discussions on complex or rare conditions


Not everyone is applicable to take part in a clinical trial. Volunteers can be excluded for a number of reasons such as age, gender, type of condition, and other symptoms. 

Expert testimony

Testimony about a scientific, technical, or professional issue given by a person qualified to  testify because of familiarity with the subject or special training in the field.


To make a prediction about the future, using existing trends.



Information needed for the body to work, stored in chemical form (DNA).


A drug that has the same dosage, safety, efficacy and quality as a branded drug but is no longer under patent


An organisms complete set of DNA 


Genotyping is the process of determining differences in an individual's genetic make-up (genotype) by examining the individual's DNA sequence using biological assays and comparing it to another individual's sequence or a reference sequence. 


Health state

a comprehensive description of an individual’s health, including their quality of life and likely outcome of their condition (known as a prognosis)


Having different characteristics and qualities


A state of having different characteristics and qualities.


The automation of research such that experiments can be conducted on a large scale with repetition


in silico

Performed on computer or via computer simulation

in vitro

When a study is performed with microorganisms, cells or molecules outside of their normal biological context

in vivo

When a study tests the effect of drugs on whole, living organisms


Not everyone is applicable to take part in a clinical trial. Volunteers can be included for a number of reasons such as age, gender, type of condition, and other symptoms. 

Incremental cost-effectiveness ratio

A measure of the economic value of an intervention, in terms of costs and health benefits, compared with an alternative (comparator). It is usually the main output or result of an economic evaluation.


Refers to the use of a drug for treating a particular disease

Informed consent

Informed consent requires trial participants to be provided with a clear explanation of the trial and associated risks and benefits to make a fully informed decision about participating.

Intellectual property

Denotes ownership of an idea and can include copyrights, patents and trademarks


A treatment option such as a drug, type of surgery, or medical device.


A treatment option such as a drug, type of surgery, or medical device

Inverted pyramid structure

The inverted pyramid is a writing style used by journalists which follows a standard format. In this pattern, the most important facts are presented at the beginning followed by additional information in the subsequent paragraphs. The opening paragraph answers the 5 W’s and 1 H: who, what, when, where, why, and how. 


Molecules with identical formulas but distinct structures


Lay (person)

The term 'lay' means non-professional. In research, it refers to the people who are neither academic researchers nor health or social care professionals. 


A drug must be granted a licence by a regulatory body, before it can be made available for wider use. Licences confirm the health condition the medicine should be used for and the recommended dosage


When a product has been given a marketing authorisation (product licence) by a medicines regulator, meaning it can be placed on the market

Literature review

A comprehensive summary of previous research on a topic. 


Market Exclusivity

A fixed period of time during which similar medicines for the same indication as the drug granted market exclusivity, cannot be placed on the market. The EMA grant market exclusivity for a 10-year period after the marketing authorisation of an orphan drug

Marketing authorisation

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

Markov models

A type of model that assumes that patients move between different health states through the course of their disease. These health states are associated with particular costs and quality of life levels, and the time spent in these health states determines the overall costs and benefits experienced by the patients.


An intermediate or end product of metabolism


Literature mining is the process of extracting facts/data from text in the scientific literature. Literature mining can generate new hypotheses by scrutinising large numbers of publications


A biological or computational system used to simulate a disease or biological process, on which tests are done to describe and explain phenomena that cannot be experienced directly


A group of two or more atoms held together by chemical bonds


Monotherapy is therapy that uses one type of treatment, such as radiation therapy or surgery alone, to treat a certain disease or condition. In drug therapy, monotherapy refers to the use of a single drug to treat a disease or condition.


An analysis approach in which the data sets are multiple 'omes', such as the genome (all of an individual's genes) or proteome (all of an individual's proteins)


A change in a gene.


National Institute for Health and Care Excellence

Public organisation that develops medical guidelines for health and care in England

Natural history

A natural history study is a medical study of people who have, or are at risk of developing, a specific condition over an extended period of time. 


Typically of an action or activity- wicked or criminal 


Pharmaceutical alternative which claims physiological benefits 



Off-label use is when a drug is used for something other than for what it was approved.


A drug label describes the uses regulatory agencies have approved for the drug

Opportunity cost

The cost in terms of the value of the treatment compared to the best alternative option that was not chosen. For example, if the NHS decided to spend money on a new treatment for a rare disease, an opportunity cost would be that the money spent on this new treatment could not be spent on anything else.


Conditions that only affect a small group of patients. In the European Union (EU), the European Medicines Agency (EMA) defines this as a condition affecting fewer than 1 in 2,000 individuals (European Medicines Agency, 2015).

Orphan disease

Another term for rare disease. They are conditions that only affect a small group of patients. In the European Union (EU), the European Medicines Agency (EMA) defines this as a condition affecting fewer than 1 in 2,000 individuals (European Medicines Agency, 2015)

Orphan drug

A medicine developed for a rare disease.


An online portal containing information related to rare diseases, orphan drugs, patient organisations, professionals and institutions, research projects, clinical trials, biobanks and expert centres. 


A broad term often used to describe a variety of results from trials

Outcome measures

Outcomes measures are measurements of the effects of a treatment or service. They might include physical measurements - for example measuring blood pressure - or psychological measurements - for example measuring people's sense of well-being. So if someone takes part in research, they may be asked questions or may be asked to have extra tests to assess how well the treatment or service has worked. 


A broad term often used to describe a variety of results from trials



The branch of medicine that involves the care of infants, children and adolescents


A participant is someone who takes part in a research project. Sometimes participants are referred to as research "subjects".

Participatory research

This is a type of research where researchers and people who use services or carers are partners in a research project. The research addresses an issue of importance to service users or carers, who are involved in the design and conduct of the research, and the way the findings are made available. The aim of the research is to improve people's lives.

This is not a research method - it's an approach to research, a philosophy.


A form of intellectual property that gives the producer of a drug the legal right to exclude others from making, using or selling the drug


Genes do not act in isolation, and do their job by interacting in a network of genes and proteins. These networks are known as signalling pathways. 

Patient group

An organisation set up to represent the views of patients and families around all issues relating to a condition, from diagnosis, care and management, and treatment.

Patient information sheet

Researchers must provide a patient information sheet to everyone they invite to take part in a research study, to ensure people can make an informed decision about this. The sheet explains what taking part will involve and should include details about:

  • why the research is being done, how long it will last, and what methods will be used
  • the possible risks and benefits
  • what taking part will practically involve, e.g. extra visits to a hospital or a researcher coming to interview someone at home
  • what interventions are being tested, or what topics an interview will cover
  • how the researchers will keep participants' information confidential
  • what compensation is available to people if they are harmed as a result of taking part in the research
  • who to contact for further information
  • how the results will be shared with others. 

Patient registry

A patient registry is a centralised database that systematically collects information about people who are affected by a particular condition or have specific interactions with a health service.

Patient reported outcome

A health outcome directly reported by the patient who experienced it

Peer review

Peer reviewing is where a research proposal or a report of research is read and commented on by people with similar interests and expertise to those who wrote the proposal or report. Peer reviewers might be members of the public, researchers, or other professionals. Peer review helps to check the quality of a report or research proposal. Members of the public who act as peer reviewers may choose to comment on:

  • whether the research addresses an important and relevant question
  • the methods used by researchers
  • the quality of public involvement in the research.


The point of view that an analysis is conducted from.

Pharmaceutical company

A pharmaceutical company is a company that develops, produces, and markets drugs for use as medications.


The study of the use and effect of drugs in a well-defined, large population


The study of how a drug enters, moves through and leaves the body. This can include its absorption, distribution, breakdown and excretion


The practice of monitoring the effects of medical drugs after they have been licensed to identify previously unreported adverse reactions


Clinical trial programmes are divided into different stages, called phases. There are three main phases where Phase I trials typically involve testing a drug on a small number of healthy volunteers to assess safety, Phase II trials are testing on a small number of patients to assess the efficacy and Phase III allow this to be scaled up to much larger numbers


The set of observable characteristics in a living organism


The full set of an individual's observable characteristics reflecting genetic inheritance, genetic mutations, and environmental influences. Phenotypic data can include the results of clinical tests, scans, and descriptions.

Pilot study

A small scale preliminary study to evaluate the feasibility, duration, cost and side effects prior to embarking on a full-scale research project


A placebo is an inactive medicine which has no treatment value. 

Pre-clinical research

Pre-clinical research is essentially all medical research that does not require human participation. To name but a few methods, researchers can use bacteria, yeast, animal models, and computational models to investigate human health.


A stage of research that begins before a first-in-man clinical study, during which feasibility and drug safety data are collected. Preclinical studies are required before clinical trials can be started

Predatory price hikes

When marketing authorisation has not been secured for a drug by a company, it is possible for another organisation to buy the drug and drastically increase the price at which it is sold


A greater liking for one option over another

Press release

A press release is an essential communication tool that is used to get your message across to the media in one fell swoop. It follows the structure of a typical news story including relevant quotes and 5 Ws and 1 H (who? what? Where? When? Why? and how?). Whether you want to announce a fundraising event, launching an awareness campaign, promoting an accomplishment or engaging in any clinical trial/research programme, a press release can be used to get the right publicity. 


The proportion of a chosen population that are affected by a single disease.


A biologically inactive compound which can be metabolised in the body to produce a drug

Product registry

Registries set up to collect information related to pharmacovigilance. They are different to patient registries as they only include people taking the medicine in question and only collect information on the drug’s effects. 


The extent to which an individual can work effectively.


To increase or spread at a rapid rate 


A protocol is the plan for a piece of research. It usually includes information about:

  • what question the research is asking and its important / relevance
  • the background and context of the research, including what other research has been done before
  • how many people will be involved
  • who can take part
  • the research method
  • what will happen to the results and how they will be publicised.

A protocol describes in great detail what the researchers will do during the research. Usually, it cannot be changed without going back to a research ethics committee for approval.


Pseudonymisation is a process that removes or replaces information that identifies an individual within a data set. This may involve replacing names or other identifiers with a less easily recognisable reference number. 


A condition caused or aggravated by a mental factor such as internal conflict or stress



Measured by the quality of something rather than its quantity 

Qualitative research

Qualitative research is used to explore and understand people's beliefs, experiences, attitudes, or behaviours. It asks questions about how and why a "quality" is being measured by the research. Often the term "holistic" is used, meaning that the complexities of human behaviour are preserved in the study. Qualitative research might ask questions about why people want to stop smoking. It would not ask how many people have tried to stop smoking. It does not collect data in the form of numbers. Qualitative researchers use methods like focus groups and interviews.

Quality-adjusted life year

A numerical measurement that combines quality of life and life expectancy

Quality-adjusted life years

a numerical measurement that combines quality of life and life expectancy


Measured by the quantity of something rather than its quality 

Quantitative research

In quantitative research, researchers collect data in the form of numbers. So they measure things or count things a "quantity" is being measured. Quantitative research might ask a question like how many people visit their GP each year, or what proportion of children have had an MMR vaccine, or whether a new drug lowers blood pressure more than drugs that are usually used. Quantitative researchers use methods like surveys and clinical trials



Randomisation randomly assigns patients to a control group or treatment group in clincial trials. It removed bias from trials, which could make the test unreliable.


A person who is appointed by an organisation to report on the proceedings of its meetings.

Rare disease

The EU defines a condition as 'rare' if it affects less than 1 in 2000 people.


A collection of information about individuals. 


Following regulatory approval, a reimbursement body will decide whether the manufacturer will be paid the cost of the drug, based on a cost-effectiveness calculation. In the UK, the National Institute for Health and Care Excellence (NICE) decide whether a drug will be reimbursed, i.e. whether the NHS will fund patients' treatments


The reduction or disappearance of the signs and symptoms of a disease

Research Ethics Committee

Groups of professionals and service users who review the ethical considerations of research studies. 

Restricted funding

Funding provided to a charitable organisation with restrictions around its use to a specific project.



Alternative situations that a model can generate results for, as opposed to the default settings of the model.

Sensitive data

Under General Data Protection Regulation (GDPR), health data are considered sensitive data and are therefore subject to specific processing conditions

Sensitivity analyses

A group of methods which try and test how reliable the results of a model are. This is done by making small changes to the model and measuring the effect on the results.


A drug for which development has been halted, sometimes because tests are not successful, but often because pursuing them simply does not present a tangible  business opportunity


In the conduct of a clinical trial, the sponsor is an individual or organisation that takes responsibility to initiate, manage or finance the clinical trial, but does not actually conduct the investigation


A person's natural height


The preparation of a drug/compound from its chemical components

Systematic review

Systematic reviews aim to bring together the results of all studies addressing a particular research question that have been carried out around the world. They provide a comprehensive and unbiased summary of the research. For example, one clinical trial may not give a clear answer about the effectiveness of a treatment. This might be because the difference between the treatments being tested was very small, or because only a small number of people took part in the trial. So systematic reviews are used to bring the results of a number of similar trials together, to piece together and assess the quality of all the evidence. Combining the results from a number of trials may give a clearer picture. 


Time horizon

The length of time that is examined in a model, typically in terms of years.

Translational research

The process of turning research findings and discoveries into practical applications that are of benefit to patients.

Treatment pathway

The procedures for treatment of an illness. This will often take the form of a decision tree, which includes all the possible outcomes for a medical condition and all the appropriate elements of care a patient would receive at each stage.



Conditions that only affect an even smaller group of patients than orphan diseases, typically affecting fewer than 1 in 50,000 individuals (All Wales Medicines Strategy Group, 2015).

Umbrella Registry

Registries that are set up for multiple conditions that a particular rare disease could fit into

Unrestricted funding

Funding provided to a charitable organisation without any restricted around its use.

Utility values

Utilities are used to represent the strength of an individual's preferences for different health state

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