Case unit 2

Question 1

Describe phase 1 of clincial trails - size, lenght

Answer 1

20-100 healthy volunteers

last several months to a year

Question 2

what are they testing for in phase 1

Answer 2

does - may gradually increase throughout
side effects
bodys reaction
process in the body

Question 3

how many drugs move from phase 1 into 2

Answer 3

approx 70%

Question 4

what is the purpose of phase 2

Answer 4

asses the safety, efficacy and side effects

Question 5

how big is phase 2

Answer 5

upto several hundred patients

Question 6

how long is phase 2

Answer 6

6 months to 3 years

Question 7

describe the two steps of phase 2

Answer 7

phase IIa - small number of patients to demostrate saftey and first signs of efficacy

Phase IIb established does and overall efficacy, establish inital benefit to risk ratio

Question 8

purpose of phase 3

Answer 8

efficacy and monitoring of adverese reactions

Question 9

number of partcipants in phase 3

Answer 9

300-3000 volunteers who have the condition

Question 10

how long does phase 3 last

Answer 10

1-3 years

Question 11

describe the two stages of phase 3

Answer 11

phase IIIa - evidence of saftey, efficiacy and side effects, often include pivotal trails

Phase IIIb - conducted after the drug has been submitted for marketing approval
compare to current treatments
use in additonal populations

Question 12

describe phase IV

Answer 12

commerially orinentated trails conducted after the drug has been approved for marketing
expand testing to broader patient population
look at long term effectiveness

Question 13

what is an observational study

Answer 13

address questions that randomsied control studies cannot
descriptive
lead to a determaination of associations
idenitfy patterns

Question 14

describe the translational pipeline

Answer 14

idea - generated by oberservation 
basic research - idea development 
clinical trials - drug development and testing 
regulatory approval 
patients care

Question 15

example of a randomaised control trail

Answer 15

effectiveness and sustainaible mutilmedia education for children with asthma
control gorup pedatric pateints given standard educational rescourse
experimental group pedriatic pateints given standard and mulitmedia rescources
reduction in daily symptoms, emergrnecy vists, school missed and days of limited activty in experimenal group

Question 16

what is a single blind trail

Answer 16

patients dont know the treatment but doctors do

Question 17

what is a double blind trail

Answer 17

researches and patients do not know

Question 18

what are the benefits of blind trails

Answer 18

prevents doctors, researches and pateints from knowing which study group they are in so they cannot influnce results
protects against bias - reduces performance and ascertainment bias

Question 19

what should you do if you cant blind

Answer 19

measure objective as possible
duplicate
treat groups in the same manner
assign indivduals to differnt surgeons each performing

Question 20

what is intention to treat analysis

Answer 20

all patients who were enrolled and randomly allocated to treatment are included in the analysis and are analysed in the group to which they were randomsied

Question 21

inclusions in ITT happen even if ..

Answer 21

• withdawal
• protocol violations
• losses to follow up

Question 22

pros of intention to treat analysis

Answer 22

more reliable estimate of true treatment by replicating what happens in the real world
simplifies task of dealing with suspiciuos outcomes
prevents bias when incomplete data is related to outcome
preserves sample size
when ITT analysis and per protocol analysis come to the same conclusions, confidence in the study is increased

Question 23

cons of intention to treat analysis

Answer 23

estimate of treatment effect is conserative becuse of dilutaion to non compliance and more prone to type 2 errors (false negatives)
does not asses treatment efficacy accutately unless there is negligable protocol violations
protocol violations and poorly conducted trails may cause results obtained from two different treatment groups to appear similar

Question 24

what is the role of NICE

Answer 24

act as a rationaing body to reduces varaiation in acess to new interventions
guideline development group, on the use of drugs and spending

Question 25

what is the cost effective analysis

Answer 25

is looks at the ratio of cost to health effect (QALYS)

compares the price and effectivness of treatments

Question 26

what are QALYS

Answer 26

‘currency’ for health
a measure of the overal effictiveness, combines
- lenght of life
- quality of life (1=full health, 0=dead)

Question 27

what is the equation for QALYs

Answer 27

life expectancy x (HR-QoL)

Question 28

what is the EQ-5D questionnaire

Answer 28

pateinst are asked 5 questions referring to 5 domains of health e.g mbolity, pain
this gives as overall score
asses life today

Question 29

time trade off

Answer 29

asks how long you would like to live at full health compare to living in your current state
- the worse the health state the more time one will be willing to sacrifes in order to avoid it e.g rather than living for 10 years at 50% health you would rather live for 2 years at full health

Question 30

what is the standard gamble

Answer 30

the worst the health state the higher the risk of possible immediate death one will accept in order to avoid current state

Question 31

incremental cost effictiveness ration equation

Answer 31

difference in cost/difference in QALYs
e.g avg cost of B - avg cost of A/avg QALY B - Avg QALT A
= £500 per QALY gained

Question 32

what is the current ICER threshold

Answer 32

1 QALY = £20,000