Introduction Of Clinical Trials

Question 1

Clinical trial is?

Answer 1

Human experiments involving medical intervention ( I.e drugs, surgical procedures etc)

Question 2

Importance of clinical trials

Answer 2

To obtain approval for new drug/procedure from a regulatory body
To obtain a new indication for an established drug
Compare 2 treatments
Determine it the new medicine is safe and effective

Question 3

Phase 0 referred to as?

Answer 3

Pre - clinical

Question 4

Phase 1 role

Answer 4

Assess the safety of the drug

Question 5

Phase 2 role

Answer 5

Efficacy and safety of the drug

Question 6

Phase 3 role

Answer 6

Drug effectiveness and possible adverse receptions

Question 7

Phase 4 role

Answer 7

Testing drug after market approval

Question 8

Investigational new drug (IND) application: must include information on?

Answer 8

Animal pharmacology and toxicology studies
Preclinical data used to assess whether the drug is sate for testing in humans
Chemistry and manufacturing information whether the drug is stable, able to be manufactured with high qualify
Clinical protocols describing study designs, safety assurance
Investigators brochure

Question 9

Investigators brochure contains?

Answer 9

The information investigators need to know in order to conduct a safe and appropriate clinical trial

Question 10

What happens in phase 1?

Answer 10

New drug tested on a small number of healthy volunteers
Participants given cow dose of drug then incrementally increase the dose of the drug until which could potentially cause some sort of a side effect
Typically last several months, can be longer up to 2 years

Question 11

What is required in phase 7 before progressing to phase 2?

Answer 11

Drug and food interactions

Question 12

What happens in phase 2?

Answer 12

Studies safety and effectiveness of the drug to treat a disease
Randomised /non- randomised and often blinded / double-blinded
Can Last for months to years (2-3 years)

Question 13

What % of drugs successfully complete phase 2 studies?

Answer 13

25%

Question 14

Phase 2 can be subdivided into what phases?

Answer 14

Phase 2a ( proof of concept)
Phase 2b ( pivot trails)

Question 15

What happens in phase 2a:

Answer 15

Pilot trials to evaluate safety and efficacy in selected groups of patients with the condition to be treated
- goal is to refine frequency of dosing or particular aspects of safety and efficacy

Question 16

What happens in phase 2b:

Answer 16

Typically represents the most rigorous demonstration of a drug’s efficacy
- tells us what type of dose to use in phase z

Question 17

What happens in phase 3?

Answer 17

Large number of participants, diverse patient population which is representative of the main target population
Double blinded and randomised- give existing therapy in market instead of placebo
Confirmation of efficacy, safety and benefit :risk ratio at the selected dose(s)

Question 18

What does phase 3 provide investors and regulatory body?

Answer 18

With most complete information about the drug’s effectiveness and possible adverse reaction

Question 19

What phase is a regulatory requirement for substantive evidence?

Answer 19

Phase 3
Usually at least 2 well designed studies (reproducible)

Question 20

If phase 3 is successful?

Answer 20

Investigators can seek regulatory approval to market the drug

Question 21

Phase 4 known as?

Answer 21

Post market surveillance trial

Question 22

What happens in phase 4?

Answer 22

Drug is approved for marketing by regulatory body an can be used in wider community
Strong test of drug safety and effectiveness

Question 23

Aim of phase 4?

Answer 23

Compare a drug with other drugs already in the market
Monitor a drug’s long-term effectiveness
Determine the cost-effectiveness

Question 24

In which phase can result in drug being taken off market/restricted?

Answer 24

Phase 4

Question 25

What to do to get strongest study design

Answer 25

Ensure that study is blinded Randomised groups Controlled trial: standard vs new drug compared to standard vs placebo

Question 26

Examples of clinical trials eligibility criteria

Answer 26

Certain medical condition Certain stage of condition/disease Age Other co-morbidities and drugs that could alter results

Question 27

Benefits of participating in a clinical trial

Answer 27

Receive new treatment Close supervision from clinical staff Be part of research breakthrough that may help others

Question 28

Risk of participating in a clinical trial

Answer 28

May not benefit from treatment: no effect, unsafe Side effects Trials car be angry and require extra treatments, and time in clinic May not receive new treatment al all

Question 29

Underrepresented populations in clinical trials

Answer 29

Older adults - may new co-morbidities Pregnant women-can harm baby Children under 16-ethical issues Ethnic minorities

Question 30

What can be done to increase participation in underrepresented populations?

Answer 30

Require strong justification for exclusion criteria Encourage clinical tricks specific to the underrepresent group Publicise trends in the inclusion of these groups in clinical trials to assess progress Consider adaptive platform trial designs

Question 31

What are adaptive platform clinical trials?

Answer 31

Based on predefined criteria, you conduct a clinical trial and may decide to adapt it in some way e.g dose, and then analyse data

Question 32

Population is?

Answer 32

The entire group that you want to draw conclusions about

Question 33

Sample is?

Answer 33

A group of people who will participate in the study that is representative of the population

Question 34

Considerations in designing a clinical trial

Answer 34

Safety Avoid bias Measurable endpoints Allows for statistical analysis

Question 35

Good clinical practice

Answer 35

International ethical and scientific quality standard for designing, conducting, recording and reporting trials that involve the participation of human subjects

Question 36

Sponsor

Answer 36

An individual, company, institution organisation or group a organisations that take on responsibility for initiation, management and financing (or arranging of financing) of research

Question 37

Research questions for phase o

Answer 37

Does the drug bind the target protein? PK