Main glossary


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A

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.


Animal model

Humans and animals share many similarities in physiology, anatomy, and genetics. Animals can therefore act as an effective model for humans and are especially useful in studying genetic conditions. 


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.



B

Baseline data

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


Blinding

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.


Budget impact

The change in levels of spending because of a decision.


Budget impact

The change in levels of spending because of a decision


C

Carrier

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 a health-related research study involving volunteers who are usually patients with a specific disease. The aims of these trials are to test whether potential new treatments are effective and safe.


Clinical trials

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



Comparators

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.


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


Cost-effectiveness

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


Cost-effectiveness

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)


D

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.

De novo

Latin term for 'new'.


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.


Dissemination

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.


DNA

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


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

Drug repurposing is taking a drug already approved for human use in one disease and testing it to see if they will help 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.



E

Efficacy

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.


Ethics

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.


Exclusion

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. 


Extrapolate

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


G

Gene

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


Genotyping

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. 


H

Heterogeneous

A state of having different characteristics and qualities.


I

Inclusion

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.



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.


Intervention

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


Intervention

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. 


L

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. 


M

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.



Mutation

A change in a gene.


N

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. 


O

Off-label

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


On-label

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.


Orphan

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.


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. 


Outcomes

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



P

Participant

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.


Pathways

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

A patient group is 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. They are set up by patients, for patients. 


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. 

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.

Perspective

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.


Phenotyping

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.


Placebo

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.


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. 


Prevalence

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


Productivity

The extent to which an individual can work effectively.


Protocol

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.


Q

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 year

A numerical measurement that combines quality of life and life expectancy



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


R

Randomisation

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.


Rare disease

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


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.


S

Scenarios

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


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.



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. 


T

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.


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


U

Ultra-orphan

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).



Unrestricted funding

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



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