Abstract

• A brief summary of the main features and results of a clinical study.

Adherence (to treatment)

• Following a recommended course of treatment by taking or using a treatment as described in the protocol (or “Study”) (for example, taking a medicine once a day for 5 weeks). 

Adverse event

• An adverse outcome (e.g. side effect) that occurs during a clinical study that may or may not be caused by the treatment being tested.

Adverse reaction

• An adverse outcome (e.g. side effect) that occurs during a clinical study that is thought to be possibly, probably or definitely related to the treatment being tested.

Advisory group 

• A group that often includes consumers, researchers and health professionals, who can provide relevant and varied advice on the project.

Analysis (of data)

• The examination and processing of data, in order to answer the questions that the study is trying to address. Analysis may be qualitative - Looking at what people have said and drawing out the main themes. Or, analysis can be quantitative - Looking at numbers and statistics to identify patterns.

Arm

• Any of the treatment groups in a randomised trial. Many randomised trials have two ‘arms’ or groups, but some may have three or even more.

Attrition

• Refers to those participants that are lost to the study because they drop out of the study.

Basket trial

• A trial that tests, one drug on a single gene mutation in many tumour types.

Benefit-risk assessment

• The continuous assessment of the favourable and unfavourable effects of a treatment. 

Bias

• A type of error that can affect the results of studies by favouring a particular outcome and therefore distorting/confounding the findings. This would make the study results unreliable. Bias often arises from systematic differences in the groups that are compared. In clinical trials, bias is controlled by blinding and randomisation.

Blinding

• Where a person does not know which treatment, is being given. The person who is blinded could be either the person being treated or the researcher assessing the effect of the treatment (single blind), or both of these people (double blind). Blinding reduces bias by ensuring the person is not influenced by knowledge of which treatment they have received.

Carer

• A carer is a relative, friend or partner who provides care to another person on a regular basis.

Clinical study

• Research using human participants that is intended to add to medical knowledge. There are two main types of clinical studies: a clinical trial (also called an interventional study), and an observational study. 

Clinical study (study)

• Clinical research that aims to find out the causes of human illness and how it can be treated or prevented.

Clinical trial

• A type of clinical study that compares the effects of treatments. They may test new treatments to find out whether they are safe and effective or may compare existing treatments to find out which ones are best.

Clinically significant

• A result (for example, the effects of a treatment) that is large enough to be of practical importance to patients and healthcare providers.

Cluster randomised trial

• A clinical trial in which clusters of individuals (e.g. GP practices, clinics, nursing homes, geographical areas), rather than individuals, are randomised to different treatment groups.

Co-applicants

• Co-applicants are individuals with responsibility for the day to day management and delivery of a study. Co-applicants are considered part of the study team and are expected to share responsibility for its successful delivery.

Cohort

• A group of individuals with some characteristics in common.

Cohort study

• A study that identifies a group of people and follows them over a period of time to see how their exposures affect their outcomes. This type of study often looks at possible risk factors (e.g. smoking on lung cancer).

Co-morbidity

• The presence of one or more diseases or conditions other than the main disease or condition under investigation.

Consensus

• A general agreement among the members of a group/committee who all have some decision-making power.

Consent form

• A document that patients/volunteers sign to say that they agree to take part in the clinical trial and understand what it may involve.

Consumer (healthcare)

• Any actual or potential user of the health service.

Control group

• The study arm that acts as the basis for comparison in a study.  In this group, no experimental treatment or intervention is given. For example, the control group may receive standard care for the disease condition being studied or a placebo.

Co-production

• A process where researchers, clinicians and consumers work together throughout a clinical study - sharing power, key decisions and responsibilities.

Cost-benefit analysis

• An analysis that explores how to reach the desired goal at the lowest cost and/or in the most efficient manner.

Cost-effectiveness

• A measure of whether something is of a good value. A study looking at cost effectiveness estimates the costs and health gains of alternative treatments or services.

Critical appraisal

• The process of assessing and interpreting evidence by carefully considering its validity, results, and relevance.

Data

• Information collected during a clinical study. The information may be numbers, written information, audio recordings, pictures.

Data Monitoring Committee

• An independent group that reviews, at intervals, accumulating trial data, in order to monitor the progress of a trial and to make recommendations on whether to continue, modify or stop the clinical trial for safety or ethical reasons.

De-identified data

• Data from which an the person cannot be identified.

Dissemination

• Communicating the findings of a study to a wide range of people who might find it useful, for example, through reports, newsletter, articles published in journals, press releases, talks at conferences.

Effectiveness

• The extent to which a specific treatment, when it is used under ordinary circumstances, does what it is intended to do.

Efficacy

• The extent to which a treatment produces a beneficial result under ideal conditions.

Eligibility criteria

• The key requirements that people who want to participate in a clinical study must meet, or, the characteristics that they must have. These comprise inclusion criteria and exclusion criteria  (see further below). For example, a study might only accept participants who are above or below certain ages.

Endpoint

• The overall outcome that the study is designed to evaluate. Endpoints are pre-planned measurements of a participant's status after a treatment has been given that are used to assess the effectiveness of the treatment.

Equipoise

• A state of uncertainty where a person believes it is equally likely that either of two treatment options is better.

Ethics committee

• A group of professionals and lay people that review the ethical considerations of studies and make sure that the potential benefits outweigh the risks.

Evidence based medicine

• The use of the best current available scientific research in making decisions about the care of patients. Evidence-based medicine (EBM) is intended to integrate clinical expertise with the research evidence and patient values.

Exclusion criteria

• A type of eligibility criteria. The characteristic reasons that prevent/disqualify a person from taking part in a study.

False negative

• A falsely drawn negative conclusion - the analysis of a study concludes a treatment does not work, when in truth it does.

False positive

• A falsely drawn positive conclusion - the analysis of a study concludes a treatment works, when in truth it does not.

Focus Group (study method)

• A small group of people brought together to talk about a particular subject. The purpose is to find out how people feel or think about an issue, or to come up with possible solutions to problems. This method is used particularly within qualitative research.

Grant

• Money awarded given to researchers by funding organisations (for example, governments, or charities) to enable them to carry out their research. To get a grant, researchers often have to write a research proposal and receive a positive peer review (feedback from other researchers).

Health literacy

• The skills, knowledge, motivation and ability of a person to access, understand, evaluate and apply information to make effective decisions about health and health care and take appropriate action.

Hypothesis

• An unproved theory that can be tested through research.

Inclusion criteria

• A type of eligibility criteria. The characteristics that a person must have to be eligible to take part in a study.

Informed consent

• The process of learning the key facts about a study before freely deciding whether or not to participate.

Intention to treat analysis

• Analysis of study results that includes all data from participants in the groups to which they were randomised even if they never received the treatment.

Interview (study method)

• A conversation between two or more people, where a researcher asks questions to obtain information from the person (or people) being interviewed.

Investigator

• A researcher involved in a clinical study.

Literature review

• An examination of the existing research publications on the topic area of a new study, to discuss their research designs, data collection methods, findings, strengths, limitations and contexts as relevant to the new study.

Mean

• An average value, calculated by adding all the observations and dividing by the number of observations.

Median

• The value of the observation that comes half way when the observations are ranked in order.

Medical journal

• A publication where researchers formally report their results to people with similar interests.

Morbidity

• Illness or harm.

Multicentre trial

• A clinical trial conducted at more than one location (e.g. in more than one hospital).

Non-inferiority trial

• A clinical trial designed to find out whether the effect of a new treatment is not worse than a standard treatment by more than a certain pre-decided amount. 

Observational study

• A type of study in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome (for example, no treatment is given by the researchers).

Open label

• A study where researchers and participants in a randomised controlled trial are aware of what treatment/intervention is being given (the study is not blinded).

Outcome

• Outcomes are the results of treatment that patients care about most (Also see Endpoint).

Participant

• A person who takes part in a study. 

Participant Information Sheet

• The information sheet that describes all the important facts about a study and the treatments that it is testing so that patients/volunteers can make a decision whether or not to take part.

Patient advocacy or support group

• Organisations and groups that share common interests and experiences and provide a forum to share information and discuss ways to support patients and their families.

Peer review

• A review of the merits of a study by experts.

Personal information

• Information that may include a person’s name, initials, address and phone number, blood type, age, ethnic background or information about their health.

Pilot study

• A small study that aims to check whether or not the methods will work before deciding to conduct the main study. Pilot studies help identify problems and glitches in the way a study is designed or managed.

Placebo (or sham treatment)

• An inactive substance or procedure given to a participant, usually to compare its effects with those of a real treatment. It looks like the treatment being tested, so that participants do not know if they are on a placebo or the treatment itself.

Platform

• A trial that tests several drugs within one disease (potentially with many types of that disease)

Power calculation

• Calculates the minimum sample size required to detect a significant difference between the treatment and control groups in a randomised trial.

Pragmatic trial

• Clinical trials that are designed to test the effectiveness of the treatment in routine clinical practice.

Primary outcome

• The outcome of most interest or greatest importance.

Prospective study

• A study in which people are identified according to current risk status or exposure, and followed forwards through time to observe outcomes.

Protocol (or proposal)

• The plan for a clinical study that describes in detail the way the study is done.  It includes the study's objectives, design, and methods. It may also include relevant scientific background and statistical information.

Qualitative Research

• Research that explores and seeks to understand people’s beliefs, experiences, attitudes or behaviours. This type of research is based on description and observation (rather than numbers) gathering information using methods such as interviews or focus groups.

Quantitative Research

• Research that is used to measure or count things to find out whether there is a relationship between two or more variables. For example, will Drug A cause a fewer heart attacks than Drug B?

Randomisation

• The process of randomly allocating participants into one of the treatment groups of a clinical trial. Randomisation reduces bias.

Randomised controlled trial

• A study in which participants are allocated at random (by chance), to receive one or more interventions. One of these interventions may be a standard treatment for the condition being investigated.

Recruitment

• Recruitment is the process of enrolling participants into a clinical study that is based on the eligibility criteria that are described in the protocol.

Registry

• A type of observational study that collects information about patients’ medical conditions and/or treatments to better understand how a condition or treatment affects patients in the real world.

Research

• Research is defined as a well-ordered investigation that aims to find new knowledge and reach new conclusions. Health research aims to find new knowledge that could lead to changes to treatments, policies or care.

Retention (strategies)

• Method used to minimise the number of participants that drop out of a trial (i.e. strategies designed to motivate and keep participants (or researchers) engaged in a trial. High drop-out rates can introduce bias and reduce trial quality.

Retrospective study

• A study that looks backwards at outcomes that have occurred to the participants in the past.

Saturation

• The point when there is nothing new arising from the qualitative data.

Semi-structured interviews

• Interviews consisting of a range of closed and open-ended questions but also allowing some freedom in terms of interview content.

Sponsor

• The individual, company, institution that takes responsibility for managing and financing a clinical study.

Statistical analysis

• A set of maths rule used to analyse quantitative data.  It helps decide the likelihood that the results/findings from a study are reliable, or whether they happened by chance.

Statistical significance

• A result that is unlikely to have happened by chance. Of this often judged by saying that there is less than a 5% chance that the study result happened by chance.

Statisticians

• People trained to analyse quantitative data.

Steering Group/Committee

• A special group or committee that is set up to manage a project (for example, a large clinical trial or some other type of organisation). This will include overseeing aims and progress. Members of steering groups usually share decision making.

Study Design

• The specific way a study is undertaken (e.g. a clinical trial or an observational study).

Surrogate outcomes

• Outcome measures that are not of direct practical importance, but are believed to reflect outcomes that are important; for example, blood pressure is not directly important to patients but it is often used as an outcome in clinical trials because it is a risk factor for stroke and heart attacks.

Systematic review

• A method used by researchers to identify, analyse and combine the findings of many studies in a rigorous way.

TGA

• Therapeutic Goods Administration: The Australian regulatory body that ensures that health products, such as medicines and medical devices are safe and effective to be used by the public. 

Themes

• Patterns identified by researchers that occur in qualitative data and are related to the topic of interest.

Treatment (or intervention) 

• The focus of a research project. They include drugs, medical devices, procedures, vaccines, and other products that are either experimental or already used in routine care. They can also include non-invasive approaches, such as education or modifying diet or exercise.

Treatment group (or treatment arm)

• A group or sub-group of participants in a study that receives a specific treatment, or no treatment, according to the study’s protocol.

Validity

• The degree to which a result (of a measurement or study) is likely to be true and free from bias.

Variables

• A factor that can be controlled, changed or measured in a study.