pharmaceutical synthesis stuff

Question 1

drug

Answer 1

medicine or substance with a physiological effect (to prevent, treat, diagnose, or relieve symptoms of a disease or condition)

Question 2

qualities of good drugs

Answer 2

• effective
• safe
• selective
• potent
• allow patient to live long (longevity)

Question 3

metabolites and types

Answer 3

• primary: key component in organism
• secondary: not essential, but give an evolutionary advantage

Question 4

natural products

Answer 4

drugs from plant or animal sources

Question 5

opiums

Answer 5

• sedative and analgesic
• laudanum: opium mixed with ethanol to increase solubility (also increased addiction)

Question 6

typical process of drug discovery

Answer 6

• thousands of compounds tested against target to find lead compounds (potential target drug)
• lead compounds changed slightly to create drug candidates (potentially usable as a therapeutic)
• preclinical testing of drug candidate (animals)
• clinical trials of drug candidate
• FDA approval
• post-marketing monitoring

Question 7

levels of clinical trials

Answer 7

• phase 1: few healthy volunteers; tests safety
• phase 2: people with the condition to figure out dosing and specifics; tests efficacy
• phase 3: larger group with the condition; confirms efficacy

**90% failure rate in clinical trials

Question 8

why are drugs so expensive?

Answer 8

• big pharma is greedy
• low success in research and development
• generic competition
• high cost of drug discovery and clinical trials

Question 9

features of clinical trials

Answer 9

• control group (placebo or standard of care)
• randomized treatment (ideally blinded)
• replicates conducted
• patients grouped into smaller units, and effects of different factors are assessed
• results must be statistically significant!

Question 10

placebo and nocebo effects

Answer 10

• placebo effect: difference in clinical outcomes between placebo treatment and no treatment
• nocebo effect: negative symptom from a placebo (e.g. withdrawal)

Question 11

Hepatitis C case study

Answer 11

• HPC targets liver, causing cirrhosis over a long time period (permanent liver scarring)
• initially spread through reused needles and blood transfusions
• difficult to develop treatments because it doesn’t impact other animals (no animal studies) and there is a high mutation rate, with 7 genotypes

Question 12

treatments for Hep C

Answer 12

• many antiviral pills developed in 2010s that only need to be taken for 12 weeks
• high cost (around 100k) but much cheaper than the alternative of needing a liver transplant