R&D vocabulary

A placebo is a product that is similar to a drug that is being studied but does not have the active ingredient of the actual drug. Do you know what a lead or hit means in pharmaceutical research? We have compiled a glossary of key terms in drug development and their explanations.

Biomarker: Measureable characteristic that is an indicator of normal biological processes, pathogenic processes and/or response to therapeutic interventions.

Candidate: Drug candidate is a compound that has been selected for development and will be taken in to clinical phase studies. The efficacy and safety of the candidate compound will be studied and documented thoroughly.

Clinical Study: Drug is administered to humans and its efficacy and safety are tested.

Hit: A compound having better activity than predefined activity in a primary activity assay. Many times they have to fulfil some basic predefined properties e.g. solubility and molecular weight requirements.

Lead: A compound that can be optimized to drug candidate. Its scaffold should be patentable. It should fulfil activity and selectivity criteria and it should have promising kinetic and safety profile.

Metabolism: The sum of all the chemical reactions for biotransformation of endogenous and exogenous substances which take place in the living cell.

Pharmacogenetics: A subset of pharmacogenomics. The study of variations in DNA sequence as related to drug response.

Pharmacogenomics: The study of variations in DNA and RNA characteristics as related to drug response.

Pharmacokinetics: The changes of drug and/or metabolite concentration in the different body fluids and tissues in the dynamic system of absorption, distribution, metabolism and excretion.

Phase: Development of a drug is devided to four phases. Certain information on the drug has to be obtained during the phase before starting the next one.

Placebo: a product that is similar to a drug that is being studied but does not have the active ingredient of the actual drug.

Polymorphism: Single nucleotide variation in the DNA sequence that can affect how humans develop diseases and respond to drugs.

Proof-of-concept (POC): During the phase II of development, the drug concept is tested in dozens of patients. The aim is to see the expected effect in the illness.

Randomisation: Study subjects get randomly either the investigational or the reference product.

Sample size: Based on literature and earlier results on the product, the required number of study subjects to show the drug effect is calculated by biostatistical methods.

Statistical analysis: The study results, e.g. differences between the investigational and reference products, are calculated from the collected data with statistical methods.