dd

Question 1

is in general a macromolecule (typically a protein), which may cause or be associated with a particular disease, that can be modulated by a therapeutic agent in a measurable way

Answer 1

Targets or drug targets

Question 2

are macromolecules, made up of deoxynucleic acid (DNA) bases. In humans, genes vary in size from a few hundred DNA bases to more than 2 million bases and about 20,000 to 25,000 genes have been identified

Answer 2

Genes

Question 3

is the process of separation and characterization of all the proteins of a biological system. Target identification with proteomics can be performed by comparing the protein expression levels in normal and diseased tissues

Answer 3

Proteomics

Question 4

Diverse molecules from biological origins that include, nucleic-acids, various (recombinant) proteins, antibodies . . . some types of peptides. Biologics typically have a high molecular weight

Answer 4

Biologics

Question 5

are large biomolecules (up to thousands of atoms) that comprise one or more chains of amino acids.

Answer 5

Proteins

Question 6

perform a vast array of functions within organisms, often through interactions with other macromolecules. they are products of genes; they generally fold into a specific three-dimensional (3D) structure that determines their activities. There are different types of proteins with different functions and locations in the body

Answer 6

Proteins

Question 7

are short chains of amino acids. They can be modified to include non-natural amino acids (up to hundreds of atoms). Some peptides belong to the category of glycopeptide or lipopeptide, among others.

Answer 7

Peptides

Question 8

can behave like small molecules

Answer 8

Very short and modified peptides

Question 9

(e.g., insulin which is used to manage diabetes) fit in the category of biologics. At present, there are very few approved peptides that can be given by oral route but important work is ongoing in this field to enable oral delivery

Answer 9

longer peptides

Question 10

Any organic compound with around 80–100 atoms. Most are made synthetically (aspirin), while others can be derived from natural product (e.g., morphine, which is used to relieve moderate to severe pain)

Answer 10

Small molecules

Question 11

A molecule suitable for clinical testing. The molecule is expected to bind selectively to a target involved in the disease process, to elicit the desired functional responses in vivo, often in animal models of the human disease, to have adequate bioavailability and bio-distribution within the body to reach the intended target and to pass formal toxicity evaluation in various in vitro and animal models

Answer 11

Drug candidates

Question 12

is a branch of molecular biology that involves extensive analysis of biological data using computers

Answer 12

Bioinformatics

Question 13

is a field that attempts to solve chemical problems on the computer, including chemical structure coding, properties modeling and development of databases

Answer 13

Chemoinformatics

Question 14

, as used today (the so-called weak-AI), combines computer sciences and mathematics and uses (large) datasets to enable problem-solving. It includes various learning approaches, natural language processing, knowledge representation and reasoning, among others

Answer 14

Artificial Intelligence (AI)

Question 15

. A drug has to reach the intended target(s), elicit the desired functional response with no or limited toxicity and be eliminated from the body (typically via the liver or kidneys). These are critical properties of the drug candidates that are commonly investigated at various stages of the process

Answer 15

Absorption, distribution, metabolism, excretion, and toxicity
ADMET

Question 16

is a computer modeling approach that incorporates blood flow and tissue composition of organs to define the pharmacokinetics (PK) of drug candidates

Answer 16

Physiologically-based pharmacokinetic modeling and simulation (PB/PK)

Question 17

is the time-concentration profile of drugs administered in vivo to living organisms. PK parameters include clearance, volume of distribution, peak plasma concentration . . . PK is sometimes described as “what the body does to a drug”

Answer 17

Pharmacokinetics (PK)

Question 18

refers to the relationship between drug concentration at the site of action and the resulting effect, including the time course and intensity of therapeutic and adverse effects.

Answer 18

Pharmacodynamics (PD)

Question 19

PD parameters include .

Answer 19

minimum effective concentration, maximum safe concentration, onset of action, therapeutic range and therapeutic index

Question 20

describes how biological processes in the body respond to or are impacted by a drug

Answer 20

PD

Question 21

Relationship of the drug effect (pharmacodynamics) to the drug concentrations in the body compartments (e.g., blood, organs) as a function of time after drug administration

Answer 21

PK/PD

Question 22

Action of a drug on targets other than the intended biological target. Such events commonly contribute to adverse effects or toxicity, however, in some cases, off-target activity can be valuable for therapeutic purposes

Answer 22

Off-target activity

Question 23

A drug is usually designed to interact with its intended target. In some situations, the drug induces exaggerated and adverse pharmacological effects at the target of interest. This is commonly referred in the literature to as on target toxicity

Answer 23

On-target toxicity

Question 24

Unintended pharmacological effects that occur when a medication is administered correctly. There are different types of reactions (mild, moderate or severe) that can be dose-dependent or not

Answer 24

Adverse events

Question 25

Secondary unwanted effects that occurs due to the drug therapy. Side effects are usually known and patients are informed about such effects

Answer 25

Side effects

Question 26

are a stage of research that precedes clinical trials (testing in humans). The therapeutic agents are tested in animal models of human diseases or in systems that simulate human diseases.

Answer 26

Preclinical development
Preclinical studies

Question 27

The main goals are to determine a starting, safe dose for first-in-human study and assess potential toxicity. Research into early formulations (e.g., tablet, capsule, intramuscular injection, intravenous, sublingual…) is also performed

Answer 27

Preclinical studies

Question 28

Research studies performed in humans aiming at evaluating the efficacy (does the drug cure or slow the progression of a disease?) and safety (does the drug cause undesired effects, or toxicity?) of drug candidates.

Answer 28

Clinical trials

Question 29

Human clinical research is tightly regulated by authorities around the world like____________.Pharmaceutical companies and other organizations developing drugs have to conduct extensive preclinical evaluations, propose the design of clinical trials and formally submit these data and a clinical plan to regulatory authorities. If regulatory authorities approve the proposed strategy, Phase I (first-in- humans) clinical trial can start.

Answer 29

(e.g., US Food and drug Administration or US FDA and European Medicines Agency or EMA)

Question 30

Each study has its own pre-defined rules about which patients can or cannot participate, which is called

Answer 30

eligibility

Question 31

Aka “first-in-humans” trial. Test on 20-80 healthy volunteers to assess the safety and pharmacokinetics, absorption, metabolism, and elimination, actions on the body, as well as possible side effects, formulation, and dose. In some cases, a placebo can be used.

Answer 31

Phase I

Question 32

For some drugs, a________can be sometime performed before phase I to evaluate some properties of the drug on few patients or on healthy individuals

Answer 32

phase 0

Question 33

Assesses drug safety and efficacy on about 100-500 patients (suffering from a specific disease), some of which may receive a placebo or an approved drug for that disease, called “standard of care”. Analysis of optimal dose is performed while adverse events and risks are recorded

Answer 33

phase ll

Question 34

enrolls numerous patients (e.g. 1,000-5,000), enabling medication labeling and instructions for proper drug use. Efficacy, dose, and toxicity are observed and adjustments to the final medication label are being made based on such informati

Answer 34

Phase IIl

Question 35

Following drug approval and manufacturing, regulatory agencies require companies to monitor the safety of the approved drug. Drug makers, health professionals, hospitals and patients report adverse events occurring when taking the approved drug

Answer 35

Phase IV or pharmacovigilance or “real world evidence”

Question 36

The dosage (a range of concentrations) of a drug that provides efficacious therapy and is safe (without serious side effects)

Answer 36

Therapeutic window

Question 37

The process in which the therapeutic agent is combined with different substances to produce a final medicinal product (e.g., a tablet, infusion solution, etc.). Formulation optimization is ongoing throughout pre-clinical and clinical stages. It ensures drugs are absorbed into the body and delivered to the proper organ at the right time and in the right amount

Answer 37

Drug formulation

Question 38

is an exclusive right granted by the governments for an invention. Patents give an inventor (academic group or a private company) the exclusive right to prevent others from making, using, selling, or importing a product or process based on the patented invention without the inventor’s prior permission, such as through a patent license.

Answer 38

patent

Question 39

is limited to the country or region where it was issued and limited in time, typically 20 years from the date of patent application filing. Pharmaceutical patents can be extended for new indications or novel formulations

Answer 39

Patent protection