Glossary

In a dose-ranging study different doses of a medicine are tested against each other to establish which dose works best and/or is least harmful.

Double blinding is a method used in clinical trials to reduce the risk of bias, which can be caused intentionally or unintentionally when trial participants and/or researchers are aware of which participants are receiving which treatment (or placebo).

For example, in a trial with one treatment group and one placebo group, blinding means that the participants do not know which group they have been assigned to. In a double blind trial, neither the research team nor the participants know which participant is assigned to which group.

Sometimes the term 'single blind' is used to describe studies in which the participants are unaware of which group they are in but the research time is aware.

In medicines development, the drug candidate is the molecule among several that has been shown to have sufficient target selectivity and potency, and favourable medicine-like properties and justifies further development. It will then be subjected to a new series of tests, and non-clinical studies and clinical trials. At this stage it is not yet a medicine.

Drug development is the process of bringing a new medicine to the market once a drug candidate (lead compound) has been identified in drug discovery. It includes non-clinical tests on microorganisms and animals, application to the regulatory authority to initiate clinical trials on humans, and may include the step of obtaining regulatory approval with a Marketing Authorisation Application to market the drug. It is also known as medicines development. EUPATI uses the term medicines development throughout its texts.

The process by which a medicine is distributed from one location to another within the body. See also pharmacokinetics.

An ingredient intended to exert pharmacologic action or other direct effect in the diagnosis, cure, mitigation, or prevention of disease or to affect any function of the body. Along with other ingredients (excipients,) it is used to formulate a medicinal product.

Tolerance of a medicine may be considered as the ability of the body to endure a certain dose of a medicine. In contrast, drug tolerance refers to a decreasing response to repeated constant doses of a medicine, or the need for increasing doses to maintain a constant response. Drug tolerance can lead to physical (physiological) or emotional dependence, which is an adaptive state associated with a withdrawal syndrome on cessation of repeated exposure to a medicine.

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

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.

In the context of health economics, efficiency of a medicine is a measure of its ability to provide a beneficial effect against its costs to individuals or society. The most efficacious treatment may not be the most efficient (cost-effective) option, for example making it unaffordable for the patients or the health system to implement.