Lecture 2 Flashcards
(26 cards)
Pharmacy
The science of preparing, compounding, and dispensing substances for
medicinal use.
Symptom
Effect reported by the patient
Sign
Effect observed by the clinician
Palliative
Drug used to relieve symptoms
Therapeutic
Drug used to cure disease
Prophylactic
Drug used to prevent disease
Toxicity
Any adverse effect of a xenobiotic on a biological system
Xenobiotic
A molecule that is foreign to the organism. From the Greek xeno (ξένο)
for “foreign” and bios (βίος) for “life”.
Hazard
The likelihood that a xenobiotic will cause toxicity at a specific dose or
exposure level.
Risk
A quantitative measure of hazard
Safety
The reciprocal of hazard; the likelihood that toxicity will not occur.
Dose Formula
μg/kg
Dose Rate Formula
μg/kg-day
IND Application Process Definition
IND (Investigational New Drug) is a new drug that is proposed to
be used in a clinical trial.
What does the IND contain?
– Data on chemistry, manufacturing, and control.
– Pre-clinical safety information.
– Description of proposed first human trial. (Mainly used to decide what dose would be used).
Blind Trial
– Patient doesn’t know if getting drug or “placebo”.
Double Blind Trial
– Patient, doctor, and investigator don’t know if patient is getting
drug or placebo.
Phase 1 Trials
- “First-in-human” study.
- Usually 20-50 healthy volunteers.
- Several months in length.
Phase 1 Trials Objectives
– Focus on toxicity (tolerated dose range).
– Determine metabolic and excretory pathways.
– Assess variability between individuals; effect of route of
exposure; bioavailability.
Phase 2 Trials
“Proof-of-concept” phase.
* Usually 150-350 people with disease.
* Several months to two years in length.
* Often includes patients for whom other treatments have
failed.
Phase 2 Trials Objectives
– Focus on efficacy. (will it work?)
– Perform pharmacokinetic studies in diseased people.
– Identify nature and severity of side effects.
– Dose Range: not everyone gets the same dose.
Phase 3 Trials
- Several hundred to several thousand patients with disease.
- Often multicenter, replicated; length up to 1 – 4 years.
- Most expensive component of drug development.
Phase 3 Trials Objectives
– Focus on safety, dosage, efficacy.
– Identify interactions between drugs in the larger population.
– Supports NDA.
NDA
New Drug Application (NDA)
NDA is an application for marketing the drug.
* NDAs are submitted for:
– New molecular entities.
– New formulation of previously approved drug.
– New combination of two or more drugs.
– New indication (claim) for already marketed drug.
* 50-400 volumes (30,000-150,000 pages)
