Drug Discovery Flashcards
FDA
Food and Drug Administration
The 1938 Federal Food, Drug, and Cosmetic Act gives the FDA its current authority to regulate medications. Signed by FDR
Drug
All drugs are chemicals. Most drugs either enhance or diminish an existing physiologic function of an organism.
Pharmacognosy
The study and discovery of drug substances from natural sources
Example: caffeine, morphine, cocaine
Pharmacokinetics
The branch of pharmacology concerned with the movement of drugs within the body
ADME
Lead Compound
The initial step in the drug approval process is identification of a chemical compound from a larger group of compounds which is most likely to be an effective and safe drug
Preclinical Testing
Before the lead compound can be administered to humans, it must go through a series of in-vitro and animal tests
ADME
Absorption
Distribution
Metabolism
Excretion
IND
Investigational New Drug
If IND status is granted, the investigational drug is cleared to begin human testing
Clinical Trial
Drug studies performed in human subjects
Biomarker
Something that is measured as an indicator of health, disease, or response to a therapeutic intervention. They may be used to monitor the effects of a medication to determine if it has an effect or not
Endpoint
A clinical trial’s “endpoints” are measurements of what happens to people in the trial
NDA
New Drug Application
How drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the US
Supplemental NDA
A supplemental new drug application based on an existing new drug application filed and approved by the FDA with respect to the existing product
How much does it cost for the drug approval process?
Around $800 million, going from lead compound to FDA approval
Clinical Trial Phase I
Safety
Is the investigational medication/treatment safe?
- Are there side effects?
- How does it affect or move through the body?
- Is it safe to use at the same time as other medications?
Group size: generally less than 100 healthy people
Clinical Trial Phase II
Efficacy
Is the investigational medication/treatment effective in treating the targeted condition?
- Does it relieve, reverse, or stop the progression of the condition?
- How safe is it?
- What is the most effective dosage?
Group size: generally 100-300 people with the condition being studied
Clinical Trial Phase IV
Follow Up
After the investigational medication/treatment is approved, how does it work for other patients with the condition?
- More safety/efficacy information is gathered
- Are there long-term benefits?
- Are there long-term risks?
Group size: often several thousand people who have been prescribed the investigational medication
Clinical Trial Phase III
Confirmation
How does the investigational medication/treatment compare to the standard treatment for the condition?
- More effective, less effective, or the same?
- Longer-term adverse effects?
- How does it affect quality of life or survival?
- How might it be used along with existing treatment?
Group size: 300-3,000 people with the condition being studied
Small Molecule Drug
MW of about 1000 or less
Most currently marketed drugs are small molecule
Large Molecule Drug
MW weight in the thousands, recombinant DNA technology
How long is the drug approval process?
10-15 years, going from lead compound to FDA approved drug
Drug Classification
Most drugs can be placed into large groups based on their pharmacologic action or clinical use. Within these larger groups, drugs are typically placed into classes based on common characteristics. Often, drugs are placed into classes based on their target in the body
Sources of New Drugs
- Natural
- Random Screenings
- Rational Screenings
Endogenous Protein
Another natural source of new drugs are the many different proteins that our bodies produce
Example: insulin
AKA: protein drugs, biopharmaceuticals, biological drug
