Drug Discovery
Two reasons why a drug candidate does not become a drug?
What is either it is not effective and/or not safe?
Determine if the candidate drug (and its metabolite/s) is safe enough for human testing
What is pre-clinical testing?
Microdose studies prior to clinical trial phase 1
What is Phase 0
Clinical trial phase conducted in vast groups (100-300) designed to analyze working of drug along with Phase 1 assessment of safety in the larger participants. Genetic testing is very much common if there is enough proof of variation in metabolic rate.
What is clinical trial phase 2?
Clinical trial phase conducted randomly on large number of patients (300-3000 or more), having the target to achieve the definite assessment of the new drug, by comparison with the standard drug treatment.
What is clinical trial phase 3?
Allows a drug researcher to quickly conduct thousands of chemical, genetic, or pharmacological tests?
What is high throughput screening?
Filed with the FDA before clinical testing can begin
What is an Investigational New Drug Application?
Clinical trial phase with a small number of volunteers (20-100) selected in order to analyze the safety, tolerance, pharmacodynamic and pharmacokinetic properties of a drug.
What is clinical trial phase 1?
Clinical trial phase for designing a rigorous and focused Phase II trial
What is clinical trial phase IIb?
Drug application filed after Clinical Trial Phase 3. Contains highly organized and complete presentation of all pre-clinical and clinical data
Average pages of data = 120,000 (300 binders with 500 pages each)
What is a New Drug Application (NDA)?
In drug discovery implies discovering one thing while looking for something else as in the case of the six drugs: aniline purple, penicillin, lysergic acid diethylamide, meprobamate, chlorpromazine, and imipramine.
What is serendipity
The unit within the FDA where the IND is filed and processed
What is the Center for Drug Evaluation and Research (CDER)?
Trial of arms where neither the patients nor the doctors knew who was in which group until after the results had been assessed
What is double blind?
A remarkable phenomenon in which a fake treatment, an inactive substance can sometimes improve a patient's condition simply because the person has the expectation that it will be helpful to them.
What is a Placebo effect?
Treats a rare disease affecting <200k Americans or for for the diagnosis, prevention or treatment of life-threatening or chronically debilitating condition affecting < 5:10,000 persons in European Union
What is an orphan drug?
The process of uncovering new indications of the approved or failed/abandoned compounds for use in a different disease
What is drug repurposing or drug repositioning?
A series of protocol-driven studies, aimed specifically at detecting possible undesirable or dangerous effects of exposure to the drug in therapeutic doses
What is safety pharmacology?
Experiments in which the effects of various biological entities are tested on whole, living organisms usually animals and plants
What is in-vivo testing?
Trial of arms where there is no comparison group
What is a single arm?
An Incentive to companies that go into orphan drug development that will enable it to market the drug without competitors.
What is market exclusivity?
Chemically modified ‘hits’ which are considered drug candidates
What are leads?
An adverse drug effect that can lead to fatal arrhythmias
What is long QT syndrome?
The difference between the therapeutic dose and the toxic dose of the drug
What is the therapeutic margin?
Neither the pharmacist or statistician, medical doctor (principle investigator) or the study participant are aware of specific intervention a given
What is a triple blind?
“The science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other drug-related problem”
What is pharmacovigilance?