Glossary

Pediatric Committee (PDCO) http://www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/general/general_content_000265.jsp"

An analysis that is restricted to the participants who fulfil the protocol in terms of the eligibility, interventions, and outcome assessment. This analysis restricts the comparison of the treatments outcomes to the participants who adhered perfectly to the clinical trial instructions as stipulated in the protocol, i.e. completed the full treatment. If done alone, this analysis leads to bias because it does not consider participants who did not follow the protocol completely for any reason.

A performance-linked access system is a system used to minimise known safety risks of a medicine, once it is on the market. These systems guide prescribing, dispensing, and use of the medicine.

They are put in place when products have significant or unique benefits in a particular patient group or condition, but when unusual risks also exist, such as irreversible disability or death. These systems can include:

• 
  
• prescription only by specially certified healthcare practitioners,
  
• product dispensing limited to pharmacies or practitioners that are specially certified,
  
• product dispensing only after patients have, for example, undergone certain laboratory tests.
  

A period of exclusivity refers to a time after a medicine is authorised during which no other similar medicines with the same indications (intended uses) may be authorised. This protects the medicine from competition during the period of exclusivity. There can be several separate market exclusivities relating to designated conditions.

The period of market exclusivity is extended by two years for medicines that have also complied with an agreed paediatric investigation plan (PIP).

A Periodic Benefit Risk Evaluation Report is a format of safety report described by the ICH-E2C(R2) guideline which is used as a basis for the EU Periodic Safety Update Report (PSUR). The report is produced by the marketing authorisation holder (the individual or business that is granted authorisation to market a medicine) at defined time points after a medicine has been given marketing authorisation.

The purpose of the report is to provide comprehensive and up-to-date information about the safety of a medicine. The report should summarise any new evidence on safety, efficacy and effectiveness that might affect the balance of risks and benefits. The PSUR communicates risk to regulatory authorities and identifies where risk management initiatives may be required.

A Periodic Safety Update Report (PSUR) is EU terminology for a Periodic Benefit Risk Evaluation Report (PBRER). It is produced by the marketing authorisation holder (the individual or business that is granted authorisation to market a medicine) at defined time points after a medicine has been given marketing authorisation.

The purpose of the report is to provide comprehensive and up to date information about the safety of a medicine. The report should summarise any new evidence on safety, efficacy and effectiveness that might affect the balance of risks and benefits. The PSUR communicates about risk to regulatory authorities and identifies where risk management initiatives may be required.

Personalised medicine (PM) is a medical model that proposes to customise medical decisions, practices, and treatments for the individual patient. It uses targeted medicines aimed at specific molecules that are involved in the patient's disease and takes genetic, clinical, environmental, and lifestyle information about the patient into account. The aim is to select the best therapies for the individual patient to ensure the best outcome and reduce the risk of side effects.

Progress in understanding the link between genomics (and other molecular factors) and disease is an important part of the development of personalised medicine. Pharmaceutical companies are already producing some targeted medicines as a result.

Pharmaceutical form is the physical characteristics of the combination of active substance and excipients (non-active ingredients) forming a medicinal product (tablet, liquid, capsule, gel, cream, sprays, etc.).

Pharmaceutical Research and Manufacturers of America (PhRMA) http://www.phrma.org/"

Pharmacodynamics is the branch of pharmacology that studies what the medicine does to the body. Pharmacodynamics looks at the biological and physiological effects of a medicine, and their mechanisms of action at organ and cellular level.