Introduction to Drug Design and Discovery

Question 1

Outline the various steps or phases in drug approval from the chemical synthesis of a new compound through FDA approval for the final drug product.

Answer 1

1. Preclinical Animal Testing - tests efficacy (in vivo, in vitro) and safety (actute + chronic toxicity)
2. Investigational New Drug (IND) application - Investigator, Emergency, Treatment
3. Phase I Clinical Studies (20-80) - shows drug is acutely safe (1 dose)
4. Phase II Clinical Studies (50-300) - multiple dose to show effectiveness again disease
5. Phase III Clinical Studies (up to 3000ppl) - shows data to see how populations respond
6. New Drug Application (NDA)
7. FDA review team assigned to NDA
8. Labeling Review
9. Facilities Inspection
10. Approved or complete response letter

Question 2

Describe the roles of patent protection for new drugs and required testing for marketing of a generic drug product.

Answer 2

Patent lasts for 20 years after synthesis:
Invitro testing (2 yrs) - Animal testing (2 yrs) - Clinical testing (Phase I,II,III) (4-5 yrs) - Marketing (11 years) - generic becomes available

Question 3

Explain the meaning of the term “orphan drug”.

Answer 3

The Orphan Drug Act was passed in 1983 - made drug development for rare diseases (affecting

Question 4

Compare and Contrast the terms branded drug, generic drug, and dietary supplement

Answer 4

Branded drug - drug that is discovered, developed, and marketed by a pharmaceutical company

Generic Drug - can be developed after 20 years after patent is approved, Submit Abbreviated New Drug Application (ANDA) to prove the product’s bioequivalence (the generic acts essentially the same as the branded

Dietary Supplement - a product intended to supplement the diet that bears or contains: a vitamin, a mineral, a herb, an amino acid

Question 5

Identify the major legislation & governmental bodies regulating the development, manufacture, prescribing, dispensing, and administration of drugs in the U.S.

Answer 5

1902-1950 - alot of acts that changed the purity of vaccines & medication & banned misleading labeling regulated by FDA, FTC, NRC, DEA, and state bureaus

Question 6

Describe the five drug categories used by the FDA to stratify drug risk during pregnancy

Answer 6

Category A - the possibility of fetal harm appears remote (multivitamins)
Category B - If there is a clincal need for this drug, they are considered safe to use (amoxicillin)
Category C - These drugs should be given only if potential benefit outweighs potential risk to fetus (most antibiotics)
Category D - There is positive evidence of human fetal risk bu use in pregnant women may be acceptable if alternatives are worse (pregnant woman is considered 2 patients)
Category X - Animal and human studies show fetal abnormalities
Category N - Unclassified by FDA