Phase 3 Clinical Trials

PHAR

what is the main purpose of phase 3 clinical trails

to test safety and effectiveness in large groups of patients

how many patients are usually involved

usually thousands of patients

what are the 3 main parts

enrolment, treatment allocation, and results analysis

what does enrolment mean

testing who is eligible and enters the study

what does treatment allocation mean

deciding what treatment group each participant enters

what does result analysis mean

comparing outcomes to determine what happened and weather the drug worked

what is the target population

the full group of people the drug is intended to treat

what is the study population

the eligible people from the target population who actually participate

what happens if the trials are successful

the evidence can be submitted for regulatory approval

which regulator reviews drug evidence in Canada

health Canada

what is one main reason phase 3 clinical trials are important

they provide large scale evidence before drug approval

what question does result analysis answer

what happened, and did the treatment work

what question does treatment allocation answer

who gets which treatment

what question does enrolment answer

who gets into the study

what is the full phase 3 flow in short form

enrol, blind, randomize, treat/control, measure, analyze

can participants be forced to stay once they consent

no. they can withdraw at any time without penalty

can study population and target population be treated as the same thing?

no. the study pop is only the participating subset

must every control group receive a placebo

no. it may receive the gold standard treatment instead

does a placebo contain a smaller dose of the active drug

no. in contains no active drug

what does randomization NOT mean

it does not mean participants and investigators are unaware of treatment

what does double blind NOT mean

it does not mean participants are assigned to groups by chance

which phase occurs after approval

phase 4

which phase usually uses thousands of patients

phase 3

which phase uses health volunteers most often

phase 1

what were statistics used for in the semaglutide trial

to compare body weigh loss between semaglutide and placebo groups

what else was monitored besides weight loss

adverse effects

how was quality of life measured in the semaglutide example

using a validated questionnaire

what was the main outcome in the semaglutide trial

percent body weight loss

how was compliance monitored in the example

weekly counselling plus tracking medication, diet, and activity

why was placebo considered acceptable in the example

there was no gold standard

what did both example groups ago get

lifestyle interventions

in the example what did the control group get

a placebo

what did the experimental group get in the example

semaglutide

was th3 example double blind

yes

was the example randomized

yes

who was disclosed form the example

people with diabetes or other surgical obesity treatments

what is the example studying

safety and efficacy of semaglutide for obesity

why is phase 4 needed

rare or delayed side effects may not appear during phase 3

what is phase 4

post market surveillance after the drug is approved

what is phase 3 mainly focused on

large scale safety and effectiveness compared with a control

what does phase 2 mainly focus on

early effectiveness and safety in patients with the disease

how many people are often in phase 1

20 - 80 healthy volunteers

who participates in phase 1

healthy volunteers

what does phase 1 focus on

safety, tolerability and pharmacokinetics

what happens after treatment is given

researchers measure outcomes and analyze the results

what happens after the study population is selected

participants are blinded and randomized into treatment groups

what happens first in phase 3 flow

target population is identified, then eligibility and consent are assessed

three things that are important in result analysis

compliance, quality of life and statistics

what do statistics help researchers determine

if group differences are real or due to chance

why is quality of life measured in phase 3

a drug may work but still cause side effects

how can you check compliance with oral medication

counting pills, or asking

why is compliance important

bad compliance can make it hard to see if a drug is working

what is compliance in a clinical trial

weather participants actually followed the treatment instructions

what makes a good clinical trial outcome

it should be objective, reliable and measured consistently

what is an outcome in a clinical trail

the measurement used to determine whether the treatment worked.

why is it unethical to use placebo when gold standard exists

because effective treatment would be withheld from the control group

when is gold standard peferend over placebo

when an effective accepted treatment already exists

what is a gold standered treatment

the treatment currently accepted as the best available treatment

why is placebo used

the separate the drugs true effects from the expectation related effects

what is a placebo

a treatment with no active drug that people think is real

what does the control group receive

a placebo or the current gold standered treatment

what does the experimental group receive

the new drug being tested

what is a confounding variable

another factor that’s not the drug that could influence the results

difference between randomization and blinding

randomization = who goes where…. blinding =. who knows what

main purpose of randomization

to reduce bias and balance confounding variables

what does randomization mean

assigning participants to treatment groups randomly

what is the main purpose of double blinding

to reduce bias

what does double blind mean

both the participants or investigator know the treatment assignment

can you leave a clinical trail after consenting

yes

what must informed consent explain

purpose, procedures, possible risks and possible benefits

what is informed consent

the participant agrees after understanding the study, risks and benefits

why might common comorbidities still be allowed

to keep the group representative of the real population

what is comorbidity

another health condition a patient has at the same time

why are inclusion and exclusion cetera used

to reduce outside factors that could affect the results

what are exclusion criteria

characteristics that prevent a person from entering the study

what are inclusion criteria

characteristics a person must have to enter the study

how are target and study population related

the study population is a subset of the target population

what is the study population

the eligible people from the target population who actually enter the trial

what is target population

everyone the treatment is intended for