CML+Imatinib

Question 1

What studies are carried out before clinical trials and why?

Answer 1

Preclinical studies –> checks safety in primates

Question 2

What is the purpose of phase I trials?

Answer 2

test drug safety

Question 3

What are the key steps/characteristics of phase I trials?

Answer 3

• Small scale
• Low does initially –> gradually increased to therapeutic level
• Don’t give all patients drug at same time

Question 4

What is the purpose of phase II trials?

Answer 4

test drug efficacy

Question 5

What are the characteristics of phase II trials?

Answer 5

• Sometimes RCT

- small scale –> quick

Question 6

What happens in a phase III trial?

Answer 6

• Full scale RCT
• 1 group test drug, other group current therapy
• test patients withe the believed optimum dose
• prospective with continuous monitoring
• individual/s doing worse on test drug must swap groups

Question 7

What are the main purposes of NICE?

Answer 7

• reduce variation in access to new treatments

- judge value of new interventions using health technology assessments and cost-effectiveness analysis

Question 8

How can utility be measured?

Answer 8

EQ-5D questionnaire 
- patients answer 5 questions about 5 domains of health 
- answers coded as levels 1-3
Disease specific questionnaires
Time trade off
VAS method

Question 9

What type of questionnaire may capture impact of poor health better?

Answer 9

Disease specific questionnaires

Question 10

What do Time trade offs evaluate?

Answer 10

The desirability of an individual in living the remainder of their life in the current health state VS living shorter in excellent health

Question 11

When modelling costs what must be included?

Answer 11

• cost of NHS resources
• treatment/administration costs
• hospital care costs
• end of life palliation
• long-term costs

Question 12

What is the purpose of an ICER?

Answer 12

• summarise results of cost-effectiveness studies
• compare 2 strategies
• give cost-effectiveness esitmate

Question 13

How might 30,000 per QALY be justified by NICE?

Answer 13

• certainty around ICER
• reasons to suggest HR-QOL inadequately captured
• innovative nature of technology

Question 14

What is an intention to treat analyses

Answer 14

• Technique used in RCTs
• Patients are compared in terms of their final results according to their randomised group
• it is independent of whether or not people violated the initial protocol/dropped out of the study etc.

Question 15

What is the advantage of using allocation concealment to assign participants to groups in RCTs?

Answer 15

prevents selection bias