Glossary

Danish National Competent Authority. http://laegemiddelstyrelsen.dk/en/"

A Data and Safety Monitoring Board (DSMB) is an independent group of experts set up to protect patient safety during a clinical trial. This board periodically reviews clinical study data (and they have access to unblinded data in case of blinded studies), incidental event reporting, and clinical study performance. The DSMB provides independent advice to ensure participants are not exposed to undue risks, and make recommendations concerning the continuation, modification, or termination of a trial.

Data exclusivity refers to the period during which the data of the original marketing authorisation holder is protected. It is the time during which another company cannot use the originator™s data in support of another marketing authorisation application, i.e.: generics, hybrids, biosimilars. Therefore, competent authorities may not accept such an application during this period of time. In Europe, this protection period lasts for a minimum of eight years and is intended to incentivise innovation.

Data manipulation is the process of taking data and manipulating (reformatting) it to be easier to read or better organised. For example, a list of data entries could be organised in alphabetical order, making it easier to view and find information.

However, these practices should be used carefully as they can lead to the selective incorrect reporting of data or creation of false results (see bias).

Data merging is a process that involves combining data from different sources, and providing users with a single view of these data.

For example:

• data from different hospital sites within a clinical trial will be combined for analysis
  
• data generated from entirely separate scientific studies might be combined if this will provide a better data set for analysis
  

Data mining is the practice of searching through existing large sets of data to find useful patterns or trends. Data mining can generate new hypotheses or new ideas for diagnosing, preventing, or treating diseases. It can, for example, lead to predictions for individual responses to medicines, or help with the design of completely new medicines.

The pharmaceutical industry uses sophisticated computer-driven data-mining techniques in an effort to extract information from the large amounts of chemical, biological, and clinical data available.

Data can be mined from clinical trial data sets, data from biobanks, or any other data set that is accessible “ for example, data sets held by public research organisations or insurance companies.

Data Monitoring Committee

Dear Doctor letters are correspondence • often in the form of a mass mailing from the marketing authorisation holder of a human medicine or biologic • intended to alert doctors and other healthcare providers about important new or updated information regarding a marketed medicine or biologic.

Such information about medicinal products may emerge throughout a product™s life cycle.

The decentralised procedure is a process for authorising medicines in more than one European Union member state at the same time.

Chemical products formed from the breakdown of a medicine due to for example light, temperature, water, reaction with non-active substances, or container and closure systems, etc.

A device used for the delivery of a medicine or therapeutic agent via a specific route of administration (e.g. inhaler, dermal patch or infusion pump).

Medicine delivery systems encompass four main related aspects:

1. Routes of delivery (ways in which the medications can be taken, such as orally, by injection, by inhalation, etc.).
   
2. Delivery vehicles (dosage forms such as pills or slow-release granules).
   
3. Chemical/biological properties of the active substance of the medicine (the cargo).
   
4. Targeting strategies (delivery methods that deliver medicines to specific organs, tissues, tumours or structures inside of cells).
   

Danish Institute for Local and Regional Government Research http://www.kora.dk/temaer-paa-tvaers/sundhedsoekonomi/"

German Agency for Health Technology Assessment of the DIMDI. http://www.dimdi.de/static/de/hta/index.htm"

The Development Safety Update Report is an annual review of safety information during clinical trials of a medicine under investigation “ whether or not it is marketed. The main objectives of a Development Safety Update Report is to:

• Summarise the current understanding and management of identified and potential risks.
  
• Describe new safety issues that could have an impact on the protection of clinical trial participants.
  
• Examine whether any new safety information is in line with previous knowledge of the product™s safety.
  
• Provide an update on the status of the clinical investigation/development programme and study results.
  

Developmental and reproductive toxicity (DART) is studied in animals to test chemical substances or medicines that might interfere with normal reproduction. This includes adverse effects on sexual function and fertility in adult males and females, as well as developmental toxicity in the offspring (child).

An EU Directive is a form of legislation that is directed™ at the member states of the EU. Directives set out a goal or policy which needs to be achieved by the member states. A directive shall be binding upon each member state to which it is addressed, but shall leave to the national authorities the choice of form and methods. The member states are required to make the appropriate changes to their national legislation to implement the Directive. Normally this must be done within two years.

A wide range of issues is dealt with by directives, including some aspects of health and social policy.

The disability-adjusted life year (DALY) is a measure used in health economics. It represents the 'burden' of a disease, expressed as the number of years lost due to ill-health, disability, or early death. One DALY can be thought of as one lost year of 'healthy' life.

The total of DALYs across a population can also be thought of as a measurement of the gap between the population™s current health status and an ideal situation in which every person enjoys perfect health into old age.

Also known as ˜unmet need™ or ˜therapeutic need™. It may be a measure of the number of people affected by a particular disease for whom current treatments are inadequate. It may include the number of new diagnoses of a disease, or the costs to society or a government representing those affected. It may also include more qualitative aspects about the burden of disease and current treatments available to patients.

Dosage is a measured and specific amount of a medicine, with number, and frequency of doses over a specified period of time or prescribed intervals.

Dosage forms of a medicine are the means (or the form) by which drug molecules are delivered to sites of action within the body. There are several types of dosage form, depending on the method/route of delivery of the medicine. These include for instance pills, capsules, syrups, suppositories and solutions for injection. Typically this involves a mixture of the active substance(s) and non-active substances (excipients).

The dosage regimen is the schedule of doses of a medicine, including the time between doses, the duration of treatment and the amount to be taken each time. Dosage regimens also include how a medicine is to be taken, and in what formulation (dosage form).

A dose is a single, measured amount of a medicine to be taken at one time. This can be expressed as the forms (e.g. 1 capsule, 1 suppository), weight (e.g. 250 mg), volume (e.g. 10 mL, 2 drops), or some other quantity (e.g. 2 puffs).

Dose-Range Finding

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.