lecture 12 - drug development and clinical trials

Question 1

phases of drug development

Answer 1

phase 0 = predictions for humans

phase 1 = tolerability

phase 2 = effectiveness

phase 3 = safety

phase 4 = post marketing

Question 2

define biomarker, surrogate, outcome

Answer 2

biomarker = readily measurable marker of response

surrogate = biomarker used for regulatory approval

outcome = how the patient functions/feels/survives

Question 3

clinical trial design

Answer 3

A = assignment
B = blinding
C = comparison
S = sequence

Question 4

assignment

Answer 4

way of determining which subject gets which treatment

• first come, first serve
• randomized

Question 5

blinded

Answer 5

open trials (unblinded)

single-blind (investigator knows subject doesn’t)

double-blinded (investigator and subject are both unaware)

triple blinded (randomized sequence lost - no one knows)

Question 6

comparison

Answer 6

use of control group to account for factors that might influence the outcome that are not experimentally assigned

• active
• placebo
• standard treatment

Question 7

sequence

Answer 7

sequence of treatment can influence what is learned from a trial and the kind of bias that can arise

• parallel group design = groups all treated in same time course
• crossover = uses two or more treatments for each individual; cons include carryover effect of treatment
• titration = giving fixed sequences of doses to learn about dose-repose relationship

Question 8

analysis perspective

Answer 8

• intention to treat perspective = assume adherence to treatment in study, assumes use effectiveness (pharmacoeconomic perspective)
• as treated = looks at what patients in trial actually took, gives method effectiveness (development science perspective)