week 6 randomized control trials and pragmatic trials

Question 1

what is the design of a randomized trial?

Answer 1

1. population is randomly assigned
2. treatment or new treat,emt
3. do they improve or not?

Question 1

equipoise definition

Answer 1

genuine doubt about which course of action is better than the other - ethical

Question 2

why is there randomization?

Answer 2

aims to ensure groups are similar and no differences in factors that are known and unknown
- only difference between is the intervention

Question 3

is it randomization if eg: birth date (odd or even) to determine if a person gets a drug?

Answer 3

NO! these are quasi-randomized

Question 4

what is a triple bind

Answer 4

participants, study staff and data analysts are blinded

Question 5

what are placebos used for?

Answer 5

to make the groups as comparable as possible and for studying side effects/benefits of the treatment

Question 6

what is a crossover RCT?

Answer 6

when there is a washout period and then the groups switch, both groups receive both treatments
- controls for confounding and is less money

Question 7

what is a factorial RCT?

Answer 7

2 different treatments and you are wondering about the effects when combined
1. drug a and b group
2. drug a and placebo b
3. drug b and placebo a
4. both placebo
see who get disease

Question 8

what are per protocol analyses

Answer 8

only takes into account the study participants who completed the intervention as intended - perfect participants

Question 9

what is intent to treat

Answer 9

analysis including all participants even if didn’t complete intervention as intended - includes dropout - more real-world

Question 10

what are the problems with RCTs

Answer 10

• randomization is hard to achieve
• control must be the best available care
• ethical issues
• impossible to avoid contamination of control

Question 11

what is a pragmatic trial?

Answer 11

trials that aim to confirm a physiological or clinical hypothesis (real world)

Question 12

what is an explanatory trial?

Answer 12

trial that aims to confirm a physiological or clinical hypothesis - (ideal conditions)

Question 13

explanatory trial vs pragmatic trial: what it does?

Answer 13

E: tests hypothesis under ideal conditions
P: compares treatment under everyday clinical conditions

Question 14

explanatory trial vs pragmatic trial: goals

Answer 14

e: to determine the causes and effects of treatments
P: to improve practice and inform clinical and policy decisions

Question 15

explanatory trial vs pragmatic trial: design

Answer 15

E: test the intervention against placebos, use strict study protocols
P: test two or more real-world treatments using flexible protocols and local customization

Question 16

explanatory trial vs pragmatic trial: participants

Answer 16

e: highly defined and carefully selected
p : more representative and eligibility criteria are less strict

Question 17

explanatory trial vs pragmatic trial: measures

Answer 17

e: require data collection outside of routine clinical care
p : brief and designed so data can be easily collected in clinical setting

Question 18

explanatory trial vs pragmatic trial: results

Answer 18

e: rarely relevant to everyday practice
p : useful in everyday practices, clinical decision making

Question 19

is a trial with blinded interventions not fully pragmatic?

Answer 19

no, it is not, cause it is not representative of the real world

Question 20

what are pragmatic endpoints

Answer 20

1. symptoms
2. disability
3. quality of life

Question 21

when should you do a pragmatic trial?

Answer 21

after a phase 3 clinical trial
or you know the intervention is beneficial, who it benefits most and the adverse effects
- when feasible and sensible and does not compromise trial quality for the ability to answer question of interest