Chapter 11 - Clinical Trials

Question 1

The average number of trials per new drug has continued to increase, while patient recruitment has become even more challenging. True or false?

Answer 1

True.

Question 2

What are the 7 ethical requirements for clinical trials?

Answer 2

Social value, scientific validity, fair subject selection, informed consent, favorable risk-benefit ratio, independent review board/independent ethics committee, and respect for human subjects.

Question 3

Phase I is the first experiment in which a drug is tested on the human body. True or false?

Answer 3

True.

Question 4

Phase I

Answer 4

Assess the safety of the new drug.

Question 5

Phase II

Answer 5

Determine the effective dose and the dosing regimen of frequency and duration.

Question 6

Phase III

Answer 6

(pivotal trial) Determines the effectiveness of the drug - dosage, treatment frequency, duration, and target patient groups.

Question 7

Phase IV

Answer 7

Post marketing approval trials to monitor the efficacy and side effects of the drug in an uncontrolled real-life situation.

Question 8

Investigator

Answer 8

Conducts the study. Normally an expert in the field of the disease being investigated.

Question 9

Protocol

Answer 9

Sets out how the trial is to be conducted.

Question 10

Inclusion and Exclusion Criteria

Answer 10

Set out the conditions under which a person may or may not be included in the trial.

Question 11

Surrogate markers

Answer 11

Used when it is not possible to measure the direct effect of the drug. Blood pressure, cholesterol, concentration of certain enymes, proteins, blood glucose levels, etc.

Question 12

What are the three main areas of biostatistics?

Answer 12

Clinical statistics, epidemiology, and bioinformatics.

Question 13

Mean

Answer 13

Sum of observed values divided by the number of observations.

Question 14

Standard Error of the Mean

Answer 14

Describes the variability of the population mean that is measured in several different samples.

Question 15

Incidence

Answer 15

The number of new events that occur during a specified period of time in a population at risk for developing the events.

Question 16

Validity

Answer 16

The extent to which a test or surrogate is measuring what we think it’s measuring.

Question 17

Sensitivity

Answer 17

Ability of the test to correctly identify those who have the disease.

Question 18

Specificity

Answer 18

Ability of the test to identify correctly those who do not have the disease.

Question 19

Predictive values

Answer 19

Help assess how useful a test will be in the clinical setting at the individual patient level. Depend on the prevalence of a disease in a population.

Question 20

Kaplan-Meier analysis and Cox proportional hazards analysis

Answer 20

Two methods of survival analysis that account for inter-subject variation in events and follow up time.

Question 21

Abstract

Answer 21

Road map for the clinical paper.

Question 22

Introduction

Answer 22

Implicitly or explicitly identifies the clinical need that prompted the study.

Question 23

Methods

Answer 23

Provides all the salient details of how the study was designed and conducted.

Question 24

What is an example of prioritization of clinical information?

Answer 24

STEPS approach - Safety, tolerability, efficacy, price, and simplicity.