Flashcards in McPhail Deck (75)
What was important about the discovery of the ability to synthesize salicylic acid?
It showed that we can get a large amount of a product without using all of our natural resources
Potential therapeutic target
genomic sequence specific to disease
Compound that was effective in the assay
Hit validation with follow up assays
Different assays to make sure the hit actually works and wasn't just in the right conditions
Shows potential but hasn't been tested
Drug discovery process
Elucidation and selection of a potential therapeutic target, target validation, screening assay development, high throughput screening, hits, hit validation with follow up assay, lead compound, lead optimization, clinical candidate, pre-clinical and clinical studies, approval, new chemical entity as a therapeutic candidate
Testing how good a lead compound is
What is the difference between hit and lead?
A hit worked for that particular assay, but a lead works for several.
What is the mission of the FDA?
To promote and protect public health by guiding safe and effective drug products to the market in a timely manner
What are phase IV clinical trials?
What are the four functions of the FDA?
(1) To monitor and protect public health by guiding safe and effective drug products to the market in a timely manner (2) To monitor products for continued safety after they're on the market (3) To regulate biologicals, medical devices, veterinary drugs, food products, and animal feed (4) To monitor the safety of cosmetics, other medical and consumer products, and devices which emit radiation
What FDA centers monitor human pharmaceuticals?
Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and the Office of Regulatory Affairs
What does the Center for Drug Evaluation and Research do?
Evaluates and monitors prescription, generic, and OTC drugs.
What does the Center for Biologics Evaluation and Research do?
Regulates biologics not reviewed by the CDER (many are reviewed by both)
What does the Office of Regulatory Affairs do?
Monitors sites and facilities for drug manufacture
What is preclinical testing and what is its purpose?
In vitro and in vivo laboratory animal experiments to evaluate toxicity, pharmacology, and ADME
What is the purpose of an Investigational New Drug application?
It allows a drug candidate to become an investigational drug and proceed to human trials after approved
How many drugs that enter phase I will make it to FDA approval?
Describe Phase 1 clinical trials
20-100 healthy volunteers with gradually increasing doses over the course of a year. The goal is to assess safety and obtain general pharmacokinetic data
Describe Phase 2 clinical trials
100-500 patients with the targeted disease/condition. Dosage and route of administration are varied over two years. The goal is to evaluate effectiveness and further check on safety
Describe Phase 3 clinical trials
1000-5000 patients that may have some comorbid conditions and is typically a longer study. The goal is to confirm efficacy (that it's better than the existing options) and safety.
What populations are left out of clinical trials?
Pediatric, Geriatric, and Pregnant/Nursing mothers
How long does it take a new drug application o be reviewed?
6 months - several years
What are the three phases of drug design?
Discovery, Optimization and Development
Describe the discovery phase of drug design
Identification of a pharmacologically active compound (lead compound)
Describe the optimization phase of drug design
Modification of the structure of the lead compound to accentuate desired characteristics (selectivity, potency, absorption, metabolism, low toxicity, duration of action, ADME)
Describe the development phase of drug design
Developing the formulation taking into account different salt forms, solubility characteristics, binders, fillers, dosage form, ease/cost of preparations, taste/smell, etc
What constitutes a drug in the drug development process?
The entire formulation