Glossary

Any untoward medicinal occurrence in a subject to whom a medicinal product has been administered, including occurrences which are not necessarily caused by or related to that product.

Any untoward and unintended response in a subject to an investigational medicinal product which is related to any dose administered to that subject.

The decision that the clinical trial has been reviewed and may be conducted at the institution site under regulations of the institution and Good Clinical Practice.

A systematic and independent review of trial related activities and documents to determine whether the evaluated trial was conducted according to the protocol, GCP and the applicable regulatory requirements.

A procedure in which one or more parties to the trial are kept unaware of the treatment assignment. Single-blinding usually refers to the subjects being unaware, and double-blinding usually refers to the subjects, investigators and monitor being unaware of the treatment assignments.

The person who takes overall responsibility for the design, conduct and reporting of a study if it is at one site; or if the study involves researchers at more than one site, the person who takes primary responsibility for the design, conduct and reporting of the study, whether or not that person is an investigator at any particular site. For multi-centre studies the Chief Investigator is normally also the Principal Investigator.

The person employed by the NHS organisation who supervises the clinical aspects of a student's research.

Aims continually to improve standards of clinical care in the NHS and to reduce unacceptable variations in clinical practice.

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

Adherence to all trial-related, GCP and applicable regulatory requirements.

The power to produce the intended results - effectiveness

A database of all clinical trials commencing in the European Community after 1st May 2004.

A data processing network & management system for reporting and evaluating suspected adverse reactions during the development & following the marketing authorisation of medicinal products in the European Community. It stores safety reporting data related to all medicinal products.

Remit: to advise the Committee on Human Medicines on the safety, quality and efficacy of medicines for paediatric use, and on the implementation of the DoH/MRHS paediatric strategy, EU paediatric work sharing project, and the European regulation on Medicines for Paediatric Use.

Believes that the variability of results between studies is exclusively due to random variation. Therefore, if all the studies were infinitely large they would give identical results.

A set of ethical and scientific standards that must be adhered to in the design and conduct of clinical trails involving human subjects. Compliance with these internationally recognised standards ensures that participants inteerets are protected and that the results of the study are credible and accurate.

Composed of unrelated or unlike elements or parts.

A pharmaceutical form of an active substance or placebo being tested or used in a clinical trial.

A statistical procedure that integrates the results of several independent studies considered to be "combinable".

A government agency that is responsible for ensuring that medicines & medical devices work, and are acceptably safe. Aiming to make as much information publicly available as possible enabling greater access to products, and the timely introduction of innovative treatments and technologies that benefit patients and the public.

Research conducted according to a single protocol but at more than one research site, and therefore usually by more than one investigator.

Describes the number of patients needed to be treated with drug A for 1 patient to benefit who would not have benefited with drug B (commonly a placebo). This is specific to the drug, its dose and the clinical situation. The greater the NNT for a drug, the less effective it is likely to be. The closer the NNT is to '1', the better the treatment is.

The odds is the number of patients who fulfil the criteria for a given endpoint divided by the number of patients who do not.

• The odds of diarrhoea during treatment with an antibiotic in a group of 10 patients may be 4 to 6 (4 with diarrhoea divided by 6 without, 0.66); in a control group the odds may be 1 to 9 (0.11). The odds ratio of treatment to control group would be 6 (0.66¸0.11).

Earliest trials in the life of a new drug or treatment. Usually small trials involving healthy male subjects. Aims to find out the safe dose range, side effects and how the body copes with the drug. Dose is gradually increased for each participant.

Often larger than phase 1 and carried out in people who have a certain disease which the new drug can treat. Aims to find out if the new treatment works well enough to test in phase 3, if it is effective against the intended disease, more about side effects and how to manage them and more about the most effective dose to use.

When phase 2 trials show a new treatment may be as good or better than an existing treatment it moves to phase 3. Done to compare new treatments with the best currently available treatment (standard treatment). They may compare a completely new treatment with the standard treatment, different doses or ways of giving a standard treatment. Usually much larger than phase 1 or 2. This is because differences in success rates may be small so you would need many patients in the trial to show the difference.

Begin after the new medicine has been granted a product licence and are used to produce further data on a product. They continue to monitor the effectiveness in the intended population and identify for rare adverse events thus providing more comprehensive information about side effects and safety in long-term use. These studies are often referred to as post marketing surveillance trials, which can be useful in establishing how well the drug works when used more widely than in the more tightly focused phase 3 clinical trials required for licensing.

The leader responsible for a team of individuals conducting a study at a site. One Principal Investigator per site.

A study plan on which clinical trials are based. The plan is carefully designed to safeguard the health of the participant as well as boing specific research questions. The document describes the objective(s) design, methodology, statistical considerations and organisation of a trial. The types of people who may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study are also described here. The term protocol refers to the protocol, successive versions of the protocol and protocol amendments.

Allocating participants to different forms of care that is not truly random, e.g. allocation by date of birth, day of week, medical record number or order of inclusion in study (alternation). Creates a greater risk of selection bias.

Assumes a different underlying effect for each study and takes this into consideration as an additional source of variation.

Similar to tossing a coin to assign patients to different treatment groups. Usually done by using a computer that generates a list of random numbers, which can be used to generate a treatment allocation list.

A group of patients is randomised into an experimental group and a control group.
Avoids any possibility of selection bias. Randomisation has been successful if the different treatment groups have the same characteristics at baseline, e.g. same number of men and women, older or younger people or different degrees of disease severity.

Guidance on good practice in the collaboration between researchers, health & social care teams and their employers and funders; aiming to improve research quality and safeguard the public by setting out principles, requirements and standards. See separate sheet.

The extent to which the research will provide the same results on repeated measures and is therefore concerned with consistency, accuracy & precision.

The risk is the number of patients who fulfil the criteria for a given endpoint divided by the total number of patients. In the odds and odds ratio example above the risks would be 4 in 10 in the treatment group and 1 in 10 in the control group, giving a risk ratio, or relative risk, of 4 (0.4¸0.1).

Any adverse event or adverse reaction that:

• Results in death.
• Is life threatening.
• Requires hospitalisation or prolongation of existing hospitalisation.
• Results I persistent or significant disability or incapacity.
• Consists of a congenital anomaly or birth defect.

Research taking place at one research site only.

Individual, organisation or group taking on responsibility for securing the arrangements to initiate, manage and finance a study.

Used to ensure that equal numbers of participants with a characteristic thought to affect prognosis or response to the intervention will be allocated to each comparison group. E.g. trial of women with breast cancer should have the same number of pre & post menopausal women in each comparison group.

Any adverse reaction that is classed in nature as serious and which is consistent with the information about the medicinal product in question set out:

• In the case of a licensed product, the SmPC for that product.
• In the case of any other investigational medicinal product, the Investigator's Brochure relating to the trial in question.

Any adverse reaction that is classed in nature as serious and which is not consistent with the information about the medicinal product in question set out:

• In the case of a licensed product, the SmPC for that product.
• In the case of any other investigational medicinal product, the Investigator's Brochure relating to the trial in question.

Refers to whether a measurement accurately measures what it is supposed to measure.