Clinical Trial Design Flashcards Preview

Principles of Disease > Clinical Trial Design > Flashcards

Flashcards in Clinical Trial Design Deck (61)
Loading flashcards...
1

Why are clinical trials important

They provide evidence and most medical practice is evidence based

2

Name some drug treatments which are based on clinical trial evidence

Treatment of:
Myocardial infarction
Stroke
Many cancers
Rheumatoid arthritis

3

What type of questions should be asked when developing a new drug/clinical trial

Does it work?
What dose is therapeutic?
What dose is toxic?
Is it safe?
Is it necessary?

4

Name some drugs used today that were developed before clinical trials

Digoxin
Warfarin

5

Name some false positives which have occurred with observational studies

High cholesterol diet and rectal cancer
Smoking and breast cancer
Vasectomy and prostate cancer
Red meat and colon cancer
Red meat and breast cancer
Drinking water frequently and bladder cancer
Not consuming olive oil (reduces bp in women) and breast cancer
HRT and cardiovascular risk

6

What are observational studies difficult to replicate

Due to bias/different criteria

7

Why should robust clinical trials be conducted

What works in theory may not be best in practice

8

Give some examples of treatments which have changed due to robust clinical trials

Intermittent positive pressure ventilation (IPPV) - reduced use as no benefit
Tonsillectomy -
unnecessary in most cases

9

What acts and regulations are in place for clinical trials

UK Medicines Act 1968
The Medicines for Human Use (Clinical Trials) Regulations 2004

10

What should clinical trials test

Efficacy
Saftey

11

What should the drug efficacy be compared with in clinical trials

Placebo
Another drug

12

What are the stages in development

Drug discovery
Pre-clinical development
Clinical development

13

Name some drugs and how they were discovered

Fox gloves and digoxin
Poppies and morphine
Dogs and insulin

14

What is done in pre-clinical development

Animal pharmacology
Animal toxicology
Tissue culture

15

What does animal pharmacology test

Dose
Adverse effects

16

What does animal toxicology test

Teratogenicity
Fertility
Mutagenicity

17

How many phases are there in clinical trials

4

18

What does phase 1 (volunteer studies) in clinical development involve

Clinical pharmacology in normal volunteers generating pharmacokinetic, metabolic and pharmacodynamic data.
Usually involves around 100 subjects

19

What type of drugs can bypass phase 1

Cytotoxics

20

Give an example of a phase 1 trial

Tegenero drug which used 8 volunteers
Six given active drug intravenously
Two given placebo
Given in regulated environment
According to the protocol approved by MHRA
Volunteers got paid

21

What does phase 2 clinical trials involve

About 500 PATIENTS

Clinical investigation to confirm kinetics and dynamics in patients
Provides some evidence of efficacy and identifies a likely dosage range

22

What are phase 3 clinical trials

Formal therapeutic trials where efficacy will be established and evidence of safety obtained (so does it work for the condition we are testing)
Involves 1000-3000 patients

23

What happens at the end of phase 3 clinical trials

All data (pre-clinical, pharmaceutical and clinical data) is submitted as an application to the regulatory authority for a license to sell the drug

24

What occurs in a phase 4 clinical trial

Post-marketing surveillance to produce evidence of long term safety (can involve tens or hundreds of thousands of patients)

25

What are pilot studies for

To test study design not estimate outcome

26

What can trials be

Double blind
Single blind
Prospective
Retrospective

27

What does a placebo controlled study compare

Compare the outcome in two groups (one given active drug and other given placebo)

28

What does an other therapy study compare

Compare the end points with the use of two different drugs

29

What is a cross over design

When one group of patients start of with the study drug and the other with the compared therapy for a certain amount of time then they have a wash out period and swap drugs

30

What is a randomised control clinical trial (RCCT)

When patients are assigned at random to either treatment(s) or control
This is the gold standard