Glossary

A biological marker is something that can be measured which points to the presence of a disease, a physiological change, response to a treatment, or a psychological condition. For example, glucose levels are used as a biomarker in managing diabetes, and brain images can provide information about the progression of multiple sclerosis.

Biomarkers are used in many scientific fields. They are used in different ways at different stages of medicines development, including in some cases as a surrogate endpoint to indicate and measure the effect of interventions, such as medicines, in trials.

For example, haemoglobin levels have been used in Phase III trials to support development of therapies for Type 1 Gaucher disease (a rare disease that affects multiple organ systems and shortens life expectancy, but which can take years to show changes in clinical symptoms).

Biosimilar Medicinal Products Working Party (BMWP) http://www.ema.europa.eu/ema/index.jsp?curl=pages/contacts/CHMP/people_listing_000024.jsp"

A biosimilar medicine is a biological medicine which is similar to another biological medicine that has already been authorised for use. Biosimilar medicines are commonly known as biological generic medicines. The existing biological medicine is known as the 'reference medicinal product'. Biosimilars may only be marketed after the patent for the reference medicinal product has expired, although they may be developed earlier. A biosimilar medicine and its reference medicinal product are expected to have the same safety and efficacy profile.

Biosimilar medicines are developed to have the same mechanism of action, and to treat the same diseases as the reference medicinal product. Standards of the EU Good Manufacturing Practice (GMP) apply to the manufacture of biosimilar medicines in the same way as for any other biological medicinal product. Biosimilar medicines may offer a less costly alternative to existing biological medicinal products that have lost their exclusivity rights.

Any technological application of living systems, biological processes or organisms, to develop or make useful products or new technologies.

Biotransformation is the process whereby a substance is changed (transformed) from one chemical to another by a chemical reaction within the body. Biotransformation is vital to survival because absorbed nutrients (food, oxygen, etc.) are transformed into substances required for normal body functions. For some pharmaceuticals, it is a metabolite that is therapeutic and not the active pharmaceutical ingredient of a medicine itself.

Blinding is a way of making sure that the people involved in a research study, such as the participants in clinical trials, do not know which trial arm they are assigned to. For example, in a trial with one treatment arm and one placebo arm, blinding means that the participants do not know if they are receiving the treatment or the placebo.

Blinding is used to remove bias that can be caused intentionally or unintentionally if participants or the research team are aware of which trial group participants are in.

Sometimes the term 'single-blind' is used to describe studies where the participants are unaware of which arm they are in, but the research team does know. In a double-blind trial, both the research team and participants do not know which participant is assigned to which arm.

A blind trial is the opposite of an open or open-label trial.

Carried out in good faith; honest, without fraud or deception.

Branded medicines are medicines which have a name given to them by a company for the purpose of advertising. The names of branded medicines are different from the International Nonproprietary Name (INN), also known as the generic name. Branded medicines may be the original medicine developed by a company or several companies may make the same generic medicine, to which each company gives its own brand name.

Costs within a particular timeframe and related to a particular healthcare budget rather than a country's overall budget. This assumes robust data on epidemiology and treatment patterns, along with assumptions of uptake and displacement of current treatments.

Bulgarian National Competent Authority. https://www.bda.bg/en/"